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THE PRIVATE

COMPENDIUM
Page 1 : explanatory notes
Page 2 : corporations

BIOTECH COMPANIES
April 2007
US BIOTECH

 We do not make a market in any of the corporations named in this document.


2

A note on "fast track":


- can be requested at any time (even before IND), requires theoretical improvement over marketed products, must
demonstrate the disease is serious, and development program also;
- consequences :
- eligibility for priority review
- with hence rolling filing
- and accelerated approval and possible softer endpoints.

page I
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


A AGILENT TECHNOLOGIES (A) Also patient monitoring, ultrasound imaging, mass spec.
was Hewlett Packard
Aug 22/01 : slashes 4000 jobs
ABBI K Abraxis Biosciences, was American - Injectable non branded generics (especially anti K and
Pharmaceutical Partners ( APPX) ABs) 70% of sales
- ABI 007, Abraxane, Taxol microencapsulated in human ASCO
IPO filed Oct 3/01 (CIBC) albumin. Ph III in metaststic breast N = 460, 260 mg/m2 partnered w/AZN
Merged (Nov 28/05) w/American versus Taxol 175, doubles response rate. No cremaphor..
Bioscience, to become "Abraxis Bioscience". Filed March 04 for 2nd line. Feb 25/05 Miami Breast Conf
Abraxane copromoted w/AZN (April 27/06). 2nd line > Taxol , signif; 1st line NS
Sales 2006 $ 150m Approved 2nd line Jan 7/05
ABBI to copromote AZN analgesic & SABCS Dec 18/06, ph II 1st line mBC, N = 300, interim. A
anesthetic line (April 06) 100 mg/Kg/M2 qw vs Taxotere q3w. ORR 58%/36%. Data
ASCO June 07. But A label is 260 mg q3w. and trial
Feb 15/06 : plant problems largely in RUSSIA. Ph III to init 1H07
Sept 7/06 ODAC votes 13/1 against extension to
"adjuvant" because no trial done
EU filing end 06 ? NOPE
G-CSF FROM Biocon (India) July 20/07 for US & EU
(approved in India)
ABLX IPO ABLYNX NV(Ghent) ALX 0081 nanobodies that bind Willebrand; ACS, ph I, Practically no other willebrand competitor except
positive interim July 2/07. Ph II to init 2008 Archemix, also in advanced ph I ACS
ABLX pricing early Nov 07 - vs TNF w/WYE
JP Morgab, KBC - target in ALzheimer
- SEpt 9/07 deal w/ B. Ingelheim
ABPI IPO ACCENTIA filed Feb 14/05 (ABPI) - SinuNase : amphotericin B solution , for sinusitis, IND
K Robert Baird for ph III to be filed ( PR Feb 17/05)
Jefferies Broadview - Biovaxid , ph III, NHL, Keyhole limpet hemocyanin +
GM-CSF + autologous lymphoma derived vaccine
AB
ACAD ACADIA (ACAD) Genomics based drug discovery
IPO (R. Stephens) filed dec 21 drugs for Park, schizo, neuropathic pain :
June 2/04 to merge w/Virologic - ACP 103 ph II akathisia
- ACP 104 ph II schizo, desmethylclozapine (neutropenia
??)? iiB INIT June 26/07

ACAM ACAMBIS (ACM/LSE; ACAM Nasdaq) SEE EUROBIOTECH. May 17/07 Adv Com Vaccines OK
ACEL K ALFACELL Onconase (ranpirnase), mesothelioma, ph IIIb, +
doxorubicine vs Doxo alone
ACHN ACHILLION - Elvucitabine, NRTI, ph II, long half life
- ACH 806, ph 1 Hep C, lic to GILD. Terminater Feb 9/07
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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ACOR ACORDA (ACOR) - Zanaflex, mkt
- Fampridine SR,4opyridine (pesticide) ph III MS fatigue
(K channels), Feb 07 seeks EU partner. 2nd ph III to init
May 22/07
- spinal cord injury, precl
ACLA ACLARA (ACLA) -Lab on a chip (microfluidics) : OASIS Packard BioScience
(infringement issue w/Caliper) -SNP typing : GeneMate→→→→→→→→→→→→→→ →PEB

ACTI K ACTIVE BIOTECH (Sweden) -Laquinimod, ph II, oral immunomodulator, lic to TEVA Amer Acad Neurol April 28 May 5 2007
886624; SE; ACTI-B -ANYARA :
CEO: Sven Andreasson - ABR 214936 fusion prot. F(ab) anti 5T4 (oncofoetal Ac 5T4 gene therapy competition Sanofi + ??
Aug 01 : vaccine business (SBL) sold to RCC) with ??, ph II
Powderject - ABR 217620 F(ab) anti 5T4 fused to ???
ACTC C Advanced Cell Technology ( ACTC) h stem cell technology

ACTV ActivBiotics, IPO filed Aug 06 Rifalazil, oral antibacterial, ph III, PAD
ACU K ACUSPHERE (ACU) AI 850 Taxol without cremophor, K, Ph I/II
ADLR ADOLOR (ADLR) analgesics & opioids
-ADL 10-0101 injectable κ opioid agonist, ph II, efficacy ++ on poison
trades since nov 15/00 ivy itch, ph II pancreatic pain
Many κ agons :GSK,MRK,PFE,AVE
-ADL 82698 alvimopan/Entereg (piperidine derivative),oral (inconvenient April 2/01∏ return to ph I (poor knowledge of MTD)
post op)mu antag ,phIII postsurgical ileus delayed : too many
hysterectomies/colectomies & slipped again from June to 4Q02;enrol Lic to GSK April 15/02 ($ 270m, 50m upfront), lic
compl 1st ph III dec 5/02, data April 2/03; N = 450, endpoint recovery GI
May 04 : class action suits function: signif at 6 mg not 12!3 2nd III N + 510 (no simple
from Roberts who had it from LLY
hysterectomy), OK at 6 & 12 (but less). 3rd III (Jan 13 04), N = 450, only - more effective at 6mg than 12
22% hyst., 6 signif, 12 NS. Feb 23/04 fast track. Filing "June 28 04". May - µ antag like Naloxone or Naltrexone : issues
7/04 file pharmacotox for post op ileus. w/memory
PDUFA extended to July 25/05; July 25/05 approvable
Feb 8/06 topline data improves 20h. May 31/06 FDA
Competition : oral methylnatrexone (PGNX)
resumes review; FDA action YE : Nov 6/06
"approvable" again : CV risks.
. III init July 01 opioid bowel dysfunction ($5/pill) data Nov 11/02, N =
168 bowel movement within 8 hours p = 0.001 (but cramps, nausea,
vomiting, diarrhea). 3 ph III init summer 05 : sept 06 : 3 failures !!
-ADL 2-1294 (imodium !), ph II, topical analgesic; w/GSK Puerto Rico
GSK discont on DB failure (dec 24/01)
ADLS Advanced LifeSciences (ADLS) Cethromycin (acq from ABT 2004), ketolide from ABT part. w/BMY
(already had 6 ph III). Ph III CAP N = 522, non
IPO filed April 05 (Unterberg) inf to clarythro (June 21/07)
Traded Aug 5/05

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ADNX IPO ADNEXUS Adnectins (pseudo antibodies)

K filed Aug 22/07 (Lehman); withdrawn. Acq Angiocept, anti VEGF, ph I


by BMY Sept 27/07

ADPX ADERIS ( ADPX) was Discovery Ritigotine CDS, transderm. DA agon, Park, ph III w
Therapeutics.
IPO filed Jan 15/02 (UBSW)

AEGN AEROGEN (AEGN) - Tobi (AeroDose), ph I w/Pathogenesis. Chiron terminates Dec 5/01
∗DEVICES based on proprietary aerosol
generator (for liquid or powder with - Insulin (AeroDose) -w/Becton Dickinson (which is developing patient
calibrated aperture plates : one ph I trial completed adjustable container);Aug 24/01 sues BD for failure to
- AeroDose : hand held breath activated ph II initiated in type II (dec 14/00) perform; sttled Oct 2/01
- Home portable nebulizer -no contract for insulin yet
- Ventilator nebulizer - Albuterol (AeroDose), phII
∗PEOPLE : - Ipratropium (AeroDose), ph I Patents : 5
Jane Shaw was CEO af ALZA from 70 to 94 Headcount : 103
- Home nebulizer, 510k cleared, launch 1H/01 Manufacturing : outsourced
June 7/02 : 30% staff cut -Ventilator nebulizer , 510k cleared, launch 1H01 except aperture plates

AEZS K AETERNA (AEL/TSE, AEZS) - Impavido (Miltefosine), MKT, leishmaniosis ASCO 02


- Cetrorelix (LHRH antag), IVF, MKT, w/Serono, For EU : Ferrer, Medac GmbH
endometriosis ph II Solvay, BPH ph III ti init 2H06.
owns 48 % of Atrium (TSX) csmetics, Regained May 8/07
profitable - Ozarelix, 4th generation LHRH antag, ph HRPC
- Neovastat (AE941) extract from cartilage, oral
antiangiogenesis, renal K, ph III ( data 2007)
endpoint placebo 8m survival/verum 12m ( but mean
spontaneous is 11 m, and w/IFN 15m). Ph III NSCLC
interrupted Feb 18/06 (slow enrol). Interim efficacy to
come. Terminated
- Perifosine, anti AKT, oral, ph II, NSCLC, BC, ASCO 06
- D 63153 (SPI 153) w/ Spectrum for US, LHRH antag Ph
II
AFFX AFFYMETRIX (AFFX) GENE CHIPS ( 53% of sales, instruments 23%) GLX (14.5%∏13.4%)
HGS
Annual EPO sales June 05 (IMS) 13B; 9 in THE FUNDAMENTAL MICROARRAY PATENTS ARE Boehringer Ingelheim
US (3 in dialysis, 6 in onco & predial). PROPERTY OF OXFORD GENE TECHNOLOGY LTD Aventis

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


BY UK HIGH COURT RULING APRIL7, 00, BUT AFFX IS BMY
RULED TO HAVE A LICENSE (NOV 1). SETTLEMENT Incyte
MARCH 26/01 MRK
PFE
May 7/03 : to market SARS resequencing array. ROCHE (P450 gene chip)
SGP
WLA
AHP
AFFY AFFYMAX - Hematide, 50 aa pegylated peptide, ph II, EPO
traded Dec 15/06 like,IV/SC, once a month, to develop & commercialize
w/Takeda. July 16/07 4 ph III init Oct 12/07 vs EPOs in
renal failure (2 dial, 2 predial). Filing 2010
- all others very early
AGEN ANTIGENICS (AGEN) -K Oncophage vaccine (heat shock prot 96 + autoK cells) AACR & ASCO 02
V kidney K,ph II→III (N=300), , fast track granted, enrol to Orphan drug status, May 30/02
June 13/02 : shelf filing end mid 02. Interim data 4Q03; 2nd RCC ph III starting - Clin Canc Res Aug 15/03 : more than 50% of 29 pts
K 1H05. Data 1H06 : March 25/06 : failure
Aug 21 : acq Aquila : Adjuvants had signic immunol response in CRC
…..colorectal ph I/II
Apr 24/01 :acq ARNX $60m(July 01)
…..melanoma pivotal started May 2/02, fast track (Feb
Ags for vaccines→SBH,BMY,AVE, 5/02), partial hold (better characterization) Sept 3/03,
provides QS 21 adjuvant to ELN for cleared Nov 24/03. Fails, but stage IVa fare better. Final
Alzheimer vaccine. 4 severe cerebral inflam. data
(Jan 18/02). Window ? ……ph II initiated in sarcoma (oct 17)
Secondary (UBSW) priced Jan 11/02, nets $ - AG 858 Heat shock protein 70, phII CML w/ Gleevec, N
56m = 120, init April 9/03
-AG 702 : rHu Heat shock protein + synthetic HSV2
July 02 CEO Garo Armen becomes peptide Herpes vaccine, ph I (Nov 01)
Chairman of Elan -QS 21 in antimalaria (GSK) & anti HIV (VaxGen)
∗Aronex products :
-Atragen (liposomal trenitoin for APL),ph III, FDA turns
down (jan8/01)
-Niotran (antifungal),ph III
-Annamycin (anti MDR anthracyclin),ph II
- Aroplatin, liposomal Pt analogue, ph II
AGGX K AngioGenex antiangiogenesis, K, Id genes (interfere with transcription
factors)

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


AGIX AtheroGenics (AGIX) -AGI 1067,oral, blocks V-CAM prod→restenosis, ph II→ SGPreturns rights Oct 5/01, Amer Heart (Nov 11-14)
Oct 4/02 meets endpoint post angioplasty stenosis. CART study
Secondary planned Jan 21/03 $ 43m. Morgan Another ph II (echographic total plaque volume endpoint)
Stanley, Lehman, Lazard interim Sept 27/04 reduced 3.8% (signif), Ph III init Jan
14/03. N = 4000, follow up 18m, in pts w/coronary dis._ Lic to AZN Dec 22/05 $ 1B Deal. AZN says "low
Endpoint : various vascular outcomes (MI, Stroke, etc), probability of success". Data March 07 ACC.
against placebo. Jan 10/01/07 CEO at H&Q . Terminated April 07
- AGIX-4207(dithicarbamate represses VCAM-1gene
expression), oral ph II, RA; IV phI. Terminated Oct 13/04
- AGI 1096, oral, prevention transplant reject

w/Fujisawa
AGNT ARGONAUT TECHNOLOGIES (AGNT) High speed parallel synthesis

AHYS AHYS, was ATHERSYS (ATHX) Functional genomics MEDX, ELN


IPO filed 10/13 (CS) withdrawn april 17/01
AIMM AUTOIMMUNE (AIMM) working with Teva on Copaxone, and RA only 1 staff member left
AKC ACCESS Pharmaceuticals * Vit B12 as oral transporter for drugs

ALKS ALKERMES (ALKS) PROLEASE :


- Nutropin Depot (HGH), MKT, injected every 2-4 weeks DNA for US, Schwarz for Europe term 06/01
POLYMERS & PROCESSES for DRUG instead of every day.
DELIVERY : -EPO, ph II JNJ discont (june 00), won't compete with
™ SC/IM slow release NESP/AMGN
- Prolease : big molecules - rFSH ph I
- Medisorb : small molecules w/Serono
™ CEREBRAL : Cereport MEDISORB :
™RESPIRATORY, "AIR" for local or - Risperdal(Respiradol) , approved in more than 50
countries, once every 2 weeks IM, NDA filed Sept w/JNJ (10% royalties)
systemic delivery of dry powders.
Hopes FDA OK Sept 02. PDUFA Aug 02. Sales could be $ 3B in
3/01.Data presented May 2/01 (int soc schiz) & Int Soc Psy 06. Extrapyramidal sds. July1st Non Approvable letter
20/5/02
(hyperprolactinemia → K in rodents) Approved Aug 9/02
Aug 26/02 : staff ¬ 23% - Naltrexone (once a month)Vivitrex Vivitrol, for Germany & UK. Response to FDA April 30/03Compet : Zyprexa
alcoholism, ph III init April 02, filed April 3/05; June depot to enter ph III
23/05 lic to CEPH. FDA OK April 14/06
w/Amylin
- Exendin, diabetes, ph I
w/Alza
CEREPORT carboplatin for brain cancer
- ph III discont in 04/99
- ph II on going in children

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


AIR : w/LLY who discont the Dura/ELN project that could
- Insulin (short & the only long acting); not adapt to long acting(April 2/01)
first ph II. w/LLY, 3rd ph III init April 26/06 w/LLY
- HGH, ph I w/MEDI
Acquiring RELIANT (since March 21/02), -MAb to RSV (Numax), pre a major has an option
750 reps, stock deal about $ 1B -Albuterol slow release (Albulast), ph II w/GSK
(could they do a sort of King/Altace/Hope -3 small resp.molecules, pre, 1 in ph I
job with Lescol???) RELIANT
Aug 15/02 : terminated because off -Lescol (fluvastatin) from Novartis (for US) $ 228m in
Risperdal 2001
-Dynacirc (isradipine, Ca blocker) $ 32m/01
- Axid (Nitazidine, H2 antag), off patent April 02
- MIV 606 (from Medivir), ph II, shingles (was returned by
ABT in 96 (< acyclovir)

ALLP ALLIANCE (ALLP) perfluorocarbone derivatives oxygen carriers : →Baxter, Nycomed


Aug 30/02 staff¬ 40% -Oxygent, ph III:blood substitute. Ph III in general surgery high frequency of stroke;CV surgery trial halted
CEO J. Roth shows (april 9/01)signif reduct in blood units needed jan9/01 (CEO says linked to rapid blood
Underwriter : Roth capital Management (comparative, but not DB!!) harvesting ?)

Acq Molecular Biosystems (Optison, ultrasound


-Liquivent, ph III: ARDS. Mortality (May 23/01) 19%, contrast agent)
placebo 15%∏halted

ALNY K ALNYLAM (ALNY) siRNA : Other siRNA firms Combimatrix (CLMX), Cytrx
RNA interference is a natural process for - ARMD (anti VEGF w/MRK clinicals 2H05), term Sept (CYTR), SIRna Therapy (RNAI), Benitec (Austral),
gene silencing 20/07 Nastech, Silence (was SR Pharma Plc & acquired
Claim to have predominant IP position -Park (α synuclein targeted w/Medtronic pump), Atugen), Acuity
Sept 7/05 Novartis acq 20%, & $ 750m deal. -Apo B,
- Huntington, pre, w/Medtronic (July 30/07)
March 27/06 : deal w/Inex (IEX/CND) for - CF (target genes involved in CFTR folding, demonstrated
liposome delivery rescue of deltaF 508)
- ANTI TNF α nasal w/Nastech ICAAC 2005; Arrow Ltd has small molecule anti
July 9/07 Roche $ 1B deal (excludes - RSV clinic Dec 7/05 US, Dec 19 EU, intranasal, more RSV A-60444, ph II, lic to Novartis June 30/05
virology, infection, C-V, auto-immunity, potent than Mab in animals (outside of NOV deal). IND
CNS) filed Nov 1/05, init Dec 19/05. Intrasal ph I OK April
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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


30/06 (N = 100), inhaled ph I to init 2H06: Oct 11/06. Ph
Sept 8/07 mi RNA JV w/ISIS (JV called II in adults experimentally infected init June 26/07 vs
Regulus) : placebo. Data YE (Nov 1/07 : delayed to 1Q 08), N = 90
- mir 122 Hep C, res - ALN-FLU.01 Influenza : May 25/05
- many others : K, etc - H5N1 Novartis partnership Feb 21/06 $ 700m
- miRNAOct 23 06 efficacy of miRNA in mice : antagomir
that silences microRNAs (could be applied to K)
ALN-PCS01 Dec 13/06 IND for SYSTEMIC to be filed
anti PCSK9 (proprotein convertase subtilisin/kexin type 9)
lowers LDL cholesterol, also expressed in liver, intestine,
kidney, cortical neurons. W/Lipidoid nanoparticle
technol from MIT
- siRNA for PML (due to Tysabri), w/BIIB)
- ALN-VSP01, anti VEGF & kinesin spindle protein,
could be delivered systemically to liver w/Inex liposomes .
IND 2H07 (Nov 1/07 : delayed 1Q08)
- siRNA for Hemorrhagic fevers, Aug 9/07, $ 38M grant
- si RNA for obesity w/ Southwestern, Sept 7/07
ALT ALTEON (ALT) -ALT 711,pimagedine, alagebrium, cardiovasc aging,ph on hold Feb 25/05 rat carcino
IIb since March 04 (SPECTRA) on syst BP :improves
vascular elasticity. July 18/03 failure.. On hold June 10/05
-Ressuscitating fluid, pre : HemoMax
ALTH ALLOS THERAP (ALTH) - RSR 13( efaproxiral, efaproxin),phIII,brain by allosteric increase of PO2/50 by 10 mm Hg,
K March 5/04 files shelf $ 75m metastases.Open, contr. random. improves radiotherapy™ph II survival increase from
May 29/03 : staff ↘ 30% Enrol. compl. (N=538 6 m follow-up, will file based on 4.1 m to 6.4 m, +56%,p=0.027
- 30 min IV infusion 35% (all they need is signif diff.)increase in median Competition : Xcytrin (PCYC) photodynamic therap.
survival time)& in lung/breast subgroup. Nov 8/00:FDA (whose ph III fails Dec 13/01), Nimorazole
fast track designation. Manuf. by Hovione (Portugal, FDA
-5 times a week for 2 weeks inspected, make Mevacor)fill & finish was Acorn, will be → ASTRO, Nov 6/01 : 43 % 2 years survival
-pts receive 02 nasal canula 30 min prior Baxter; enrol compl. Aug 7/02. Interim May 30/02 (after Top line results 2Q03 if 402 deaths in intent to treat
Rx, during & 1h after 204 deaths) OK by independent Com.(may30/02). April group & 308 in breast/lung. Feb 13/03 CEO says MRI
24/03 : global survival + 17%, NS but breast N = 115 + analyses longer than expected..
Dec 17/01 : Steve Hoffman (sold 12% of 101%, p = 0.006 (8.67 m > 4.57) Rolling fiiling complete
his shares in 02)becomes Chairman, Mike Dec 4/03. Feb 3/04 accepted w/Priority review..
Approval June 4 ??, ODAC May 3/04 : NO (16/to 1), FDA June 2/04 : approvable
Hart President & CEO June 4/04 EMEA filing; Oct 2/05 withdrawn
US : 170,000 cases brain meta/year; price but Donna Przepiorka votes yes.
per ptn/year : $ 10,000 ($ 500 per dose, 10 Feb 12/04 w/Nat Breast K Coalition new ph III ENRICH, → ASCO 02 (2 year survival rate: 35%)
doses for brain, 32 for lung) rand, open, N = 360. Primary endpoint survival (powered → ESTRO 02 : Sept 19/02 : median survival 20.6m
for 50% diff). Interims at 94 & 188 deaths. 1st interim
2H05 (March 05 50 of planned ww centers open). Total (hist compar. 14m, → + 47%)
April 25/02 : $ 15m PIPE with Perseus
Soros fund (Dennis Purcell). Nov 02 have enrol. 2 years : 3Q06; 2nd interim mid 2007 (at 282

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


12.5 %. deaths). June 19/07 FAILS Aug 12/03 : World Conf Lung Cancer. Median
If no partner & $ 200 m sales in 2005 ( NSCLC :ph II, N = 52, 83 % of responders w/paclitaxel & survival 20.6m (best historical : 17m).
20000pts) net income $ 25m (US Bancorp carboplat (ASCO/01). FDA meeting agreed on SPA Aug 3/04 : Orphan drug status for Brain breast metas
Sept 26 : $ 29m) (special protocol assessment) w/ sequential chemo/Rx
In June 02 JNJ invest arm sold shares → SEC rather than simult →ph III,some enrol.1H03; N = 600
13G Feb 4/03 (DJ press release) (Europe), interim & will calculate if need to increase
Screening suspended, partner seeked.Nov 10/06 ph II
March 05 : $ 50m PIPE (W. Pincus) ASTRO, median survival 20.6m (matched RTOG group
13.3m)

Glioblastoma :ph II;pivotal III /or NSCLC


RSR 13+ BCNU™glioma, ph I/II (jan 17/01)
Cervix K ph I/II w/cisplat & Rx init Aug 15/02
modest side effect : 8% of pts have creatinine ∨

- BGP 15,oral chemotherapy protectant, lic from N-Gene(


commercial arm of Hungarian Innov. Technol, invested in
by Soros fund), March 13/02. Ph 1 to start 2H03 (with
platin). Suspended May 29/03
- PDX pralatrexate (antifolate), ph II, lic from MSKCC Jan
8/03. Maintained in NHL & solid K w/Taxotere
A LTU K ALTUS (ALTU) - ALTU 135, 2 ph III init May 07 efficacy top line 2Q08, Competitors (Creon Solvay, Ultrase Axcan (but
spin off from Vertex. safety & filing 2009, CF N= 150, microbially derived refiled Aug 1/07), Pancrease McNeil) have to
Pancreatic enzyme mkt ww 2006 $ 750m crystallized pancreatic enzymes, capsules & liquid, only 1 "renew" their older NDAs for April 2008. Eurand is
HGH mkt 2004 2.2 B pill/meal, supported by CF foundation. June 11/07 EU new but porcine.
rights reacq from Dr Falk Pharma
July 3/07 FDA revokes orphan drug status, - ALTU 238, ph II, long acting HGH thanks to
because ALTU 135 can also be used in AIDS crystallization (no polymer or fusion), lic to DNA Dec
with malabsorption 20/06. DNA has to opt in for ex-US. Nutropin patent
expires 2008
- ALTU 237, pre, oral oxalate degrading enzyme, not
absorbed. IND filed June 19/07. Ph I init Aug 14/07
- ALTU 236 for PKU, pre
ALXA ALEXZA AZ-001, aerosol of prochlorperazine ph II, March 07 very
good data in migraine pain
ALXN M ALEXION (ALXN) - Soliris 5 G1(Hu)eculizumab (anti C5). 1 → R.A.,ph II,12w To be manuf by Lonza
Anti-complement monoclonals, humanized data as good as Remicade
membranous nephritis,ph II, fails endpoint Nov/02, also
Asthma
PG deal revamped Dec 12/01 : ALXN PNH endpoints Hb stabilization & reduced
Parox. noct. hemoglobinuria, ph III (SPA)
invests more in Dev & Mkt, &nd gets 50% of transfusions (p < 0.05). Prior papers show signif
initiated Nov 04, N = 75X2. Enrol compl 1st trial Triumph
profits. stabilization or increase of Hb, and very clear
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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- Manuf by Lonza (DB N = 87) April 12/05. Tmt end Jan 4/06 (see JAMA reduced transfusions.
April 6/05). Data Jan 26/06 : transfusion rate drops to 0 vs ASH 05 : 2 papers : remarkable efficacy
Nov 8/05 : have set up Alexion Europe for 10 (p< 10 – 7 ) Hb stabilized 49% vs 0% p < 10 – 7 ;2nd trial
eculizumab. EMEA filing 2H06 Shepherd (open N = 95) enrol compl Sept 21/05 Data June
US cases : 5 to 10,000 12/06 : safe , all primary (reduct intravasc hemol, but
MStanley gives price 200 to 300,000 $/y, and transfusions not disclosed)& secondary endpoints signif, Lic from SCA (Enzon)
3,000 pts on drug (Feb 07) filing US sept 20/06, EU Sept 26 (accelerated procedure Lic to PG
150 d granted); March 16/ 07 : FDA approves (black box AHA Nov 18/02; ph II, N = 814, infarct size not ¬ but
NEXT RISK : doesn't C5 protect against meningo). EU OK June 07. 90 days mortality ¬ 70% p = 0.014
bacterial infection ? 2Q07 $ 9.8M
MDS : explor study June 18/06 Sept 26/00: acq. Prolifaron the Scripps Mab res. Corp,
with Phage Display patents
- 5 G1. 1-SC (Hu single chain) pexelizumab
→CABG, ph III: FAILS Nov Competitor NEUROGEN has a C5a receptor
25/05 antag ph II RA
→MI.ph II: results jan 23/01: death &
MI :41% at d30;ph IIIw/PTCA (COMMA) init dec 01 (for 12-18m):
PRIMOCABG (w/CABG) ph III fully enrol Feb 24/03. N = 3000, results
Aug 4/03 prelim data mixed. Full data AHA (Nov 03) narrowly misses
endpoint, but very + in subset with 2 or more risk factors. PRIMOCABG
2, with 2 or more risk factors, N = 4250. Enrol compl. Aug 12/05, results
4Q05
April 3/07 PG terminates
Xenotransplatation : res
AMGN M AMGEN (AMGN) -Epogen : $ 1.759 B/99;1.960/2000;2.1/01(+ Eprex $ AMGN prevails (jan 22/01) on TKTX in US
K 3.4B). 2002 $ 2260; FY 03 : 2.4B. 1Q04 590, 2Q 633, 3Q litigation
Aug 15/07 plan to reduce staff 2,600 681, 4Q 697, FY 04 2.6, 1Q05 583 , 2Q 647, 3Q 599 , FY ditto April 11/01 in UK
What if erythroblastopenias & 05 2.5B; 1Q06 604, 2Q 613, 3Q 633, 4QUS 661, 1Q07 Oct 18/03 Lancet paper on decreased survival of H
EPO K worsening gain momentum 625, 2Q 07 624; Nov 20/01 AFSSAPS : 40 & N w/Neorecormon.
erythroblastopenias w/Eprex (JNJ), 10 Neorecormon, 10 Roche to start ph III CERA : pegylated " continuous
? Epogen. EU patent : 2004 epo receptor agonist)
March 7/07 black box warning Hb AMGN has US rights for dialysis, JNJ for US oncology &
>12 both ww.
In Germany Fresenius distributes (Jan 22/02)
EU( 35% of sales), EPO patent expires Dec -NESP/Aranesp darbepoetin SC/IV (once a week
04 (US exclusivity until 2012. hyperglycosylated Epo)→filed FDA/CPMP, NESP : $ 860every 1 or 2 or 3 w for 12-18w
4Q99→CPMP OK march 12 01,FDA delays Aug 7/04 EU appoves Q3W/cancer & Q4W for
Nov 6/00 : Elanex releases data on long term (april30/01).Approved Sept 18/01for dialysis. US onc OK
predialysis
stability of its EPO (sold to Baxter Oct 01, July 19/02; EU OK Aug 28/02. Abs on package insert.
MKT in central Europe). Also EPO from Sales 4Q01 : $ 37m, 2002 : 416;FY 03 : 1.5B (35% in EU),
GMX (GeneMedix LSE who sells GMCSF 1Q04 542, 2Q 617, 3Q 608, 4Q 704; FY 04 2.5B, 1Q05 CERA PDUFA Feb 20/07
in China) partn w/Skyepharma. 723, 2Q837, 3Q 840, FY 05 3.3B; 1Q06 893, 2Q 1.1 B,
Jan 22/01 : Infimed has a SR EPO in pre (& 3Q 1.067, 4Q 1.106, 1Q07 1.02 (US 654), 2Q 949

- 10 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


part w/Novartis). Epo franchise 02 $ 2676 (JNJ Eprex 3.03, Procrit 1.23) Neupogen : $ 2000/10 days
Oct 07 : HOSPIRA -Neupogen : $ 1.256 B→1.220/2000;1.3/01;3Q02:332m,
Retard CERA & Neose 4Q02 : 310. FY 03 1.266, 1Q04 269, 2Q 295, 3Q 608, 4Q
Roche (NeoRecormon) is approved since Oct 309, FY 04 1.2B, 1Q05 294, 2Q 312, 3Q 305, FY 05 1.2B; Neulasta : $ 2360 equivalent period
01 (EU) for 1/week in dialysis. 1Q06 288 2Q 312, 3Q 308 4Q 373, 1Q07 299
Feb 13/03 : Dragon Pharm. (CND) ships -Neulasta SD-01 Peg(Shearwater/INHL) Once a week., Early 05 Centres for Medicare & Medicaid Services
EPO to Itaca Labs (Brazil) BLA filed March 01. FDA OK Jan 31/02,EU Aug 28/02 will publish average sales prices forAranesp, Neulasta
Teva/Sicor G-CSF approved Lithuania 2003? sales 2Q02 Neupo + Neulasta + 39%. Neulasta 3Q02 : & Neupogen
Will file Mexico 2004. 142m, FY 03 1.3B, 1Q04 395, 2Q 426, 3Q 450, 4Q 469,
Dec 13/01 : to acq IMNX $ 16B (AHP will reduce to
8%); Feb 7/02 : FTC extends review period. OK July FY 04 1.7B, 1Q05 501 2Q 587, 3Q 577, FY 05 2.3 B;
17/02 1Q06 608 (US 497) 2Q 567, 3Q 690 4Q 711, 1Q07 719
Also generics from Stada, BioGenerix (RatioPharm),
BioPartners (Merck AG + LG Korea who also hasHGH,
-IL1-ra, Kineret/Anakinra (was the Synergen sepsis
G, GM & IFN α, Pliva, Novartis Austria.
drug)→ R.A., filed FDA/CPMP 4Q99(6 months
May 8/02 : AMGN acq EU rights to Neupogen from
Roche efficacy/500 pts, & signif reduct joint destruction), less
efficacy than Enbrel or Remicade (need for daily shots,
Neulasta generic from BioGenerix AG, ph I, Nov 7/07
Sept 14/04 : FDA (after CHMP) refuses could address their failure, painful inject?., high dosage
Omnitrope (Novartis generic of HGH) (price ?),™new data(1300 pts safety) filed April 23/01.
IV 3 days befor and 3 days after chemoablation for
FDA adv panel Aug 16 OK 6/2. OK Nov 14 in pts who
PBSC
failed DMARD. Sales 4Q01 $ 12m; EO OK March 14/02
June 5/07 acq Ilypsa. Ph II Renagel like FY 05 : $ 77m, FY 06 60
drug : ILY 101. - sTNF type I receptor pegylated inhib (PEGsTNF-RI) ph
June 6/07 acq Alantos $ 300 m : DPP inhib II.
diabetes ph II. - Kepivance / Palifermin KGF mucositis filed June 23/04
(BMT), very signif : Grade IV goes from 62% pts to 20% (
N = 212) FDA OK Dec 16/04. EMEA OK Oct 28/05
FY 05 26 To be covered by Medicare in 2005
see NPSP
- AMG 073 Sensipar, Mimpara cinecalcet, oral Amer soc nephrol Nov12/ 03
calcimimetic, 1st ph III init Dec 01 : April 23/03 signif Amer soc bone metab res Sept 22/03
metab endpoints, 2 more running. Reduces PTH in renal
failure, but pain, emesis, URTI : competition for
Renagel.Filing Sept 9/03. Approval March 8/04 Price $
5,400/y. EU (Mimpara) OK July 31/04. 2004 26 m. 1Q05 :
13.7, 2Q05 : 16.8, 3Q 51, FY 05 147; 1Q06 61 2Q 79, 3Q
83 4Q 98, 1Q07 105, 2Q 108

- AMG 655, fully human Mab, TRAIl receptor 2


agonist

- AMG 162, IgG2, denosumab, fully Hu anti RANKfrom


- 11 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ABGX (receptor activator of NF kappa B) ligand, inhibits
osteoclasts, Ph II, 1/6m sc = Fosamax QW; 2 ph III init
Aug 10/04.. Filing Q3/07Launch 08/09, July 20/05 enrol
compl 10,000; data 2H06 : probl of rebound osteoporosis
(2Q press release) that biphosphonates do not have
K : phases III prostate and breast metas
- GDNF (from Synergen), Park, ph II halted Feb 10/05
inefficacity + cerebellar toxicity in animals.
-BVT 3498 ph II diab, inhib 11β OH steroid dehydro,
Biovitrum.
- AMG 706, phII, oral mixed kinase inhibitor (VEGF 1,2
& 3, PDGF, Kit, RET) : Imatinib resistant GIST, NSCLC,
CRC. GALL BLADDER TOX: To init vs Avastin 1st line
NSCLC 4Q06 : delayed. ASCO 07 thyroid ph II not
impressive, will not pursue this indic. NSCLC Ib Sept 07
w/carbo Taxol w/ or wo Vectibix
- AMG 714 HuMax IL15 ph II, RA lic from GenMab (July
1/03), ACR Oct 04
- AMG 531 : peptibody (Fc peptide fusion protein) that
stimulates TPO receptor, ph III for ITP, to file 2007
- ALS 2-0426, diabetes, DPP IV inhib, ph II from Alantos
(in France, Seevier)
AMGN AVIDIA Protein structures that bind more than 1 site, like Mabs. DEALS
acq Sept 30/06, $ 290m + $ 90m milestones Manuf in e.coli (B. Mannheim)
Anti IL6, ph 1
AMGN IMMUNEX (IMNX) -Enbrel/etanercept (sTNFr, Fc CH1 + CH2 + hinge fused by AHP, selling its shares ($367m→653/00; 762/01;
to p 75 portion of TNFr : active on α & β ),FDA OK 802/02 (Remicade 1200/02; Humira 1Q03 $ 26m, 2Q
M Aug 11 : secondary that decreases AHP from 98,CPMP OK 12/99→RA→ 57 YE forecast 250).FY 03 1.3B, 1Q04 397; 2Q 440,
55% to 41% Also Enbrel ph IIICHF discont March 23/01, subgroup 3Q 496, 4Q 567, FY 04 1.9B, 1Q05 592, 2Q 639, 3Q
analysis ready in June 01 ??? 668, FY 05 2.6 B; 1Q06 658 2Q 724, 3Q 705 4Q
Manuf Capacity issues for Enbrel!!!, Rhode PSO : ph III jan 17/03 50% PASI 12w ; PSO arthritis 792, 1Q07 730, 2Q 823
Island approved Dec 26/02. B.I.essential BLA filed July 17/01, approved Jan 17/02 FDA & Dec FDA approv.early use in RA (June 7/00)
17/02 EU. PSO dermatol filed July 8/03, OK May 3/04 - Ankylosing spondylitis (350000 US pts) : Adv Com
RISK : Remicade could take their patients oct 3/00 : CPMP posts warning on cytopenias + June 24/03. Approved July 25/03
but it is IV (but 1 a month), & Enbrel OCT 11/00: warning on demyelinating disorders - EULAR June 03 50 mg QW = 25 BIW
subcut (BIW or QIW, QIW for RA), -Novantrone→MS, FDA OK (but cytotoxic!)
Humira twice a month subcut → AASLD Nov 02 : ph II DB, Hep C : Ifn α/Rebetol
alone 28%, + Enbrel 58% viral + ALT normalisation,
-Nuvance,inhalable sIL4r→asthma, ph II(but infect.?no ) p = 0.04
Merger declared July 17/02

- 12 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS

-ABX-EGF→K, phII, fh
- Avrend (CD 40 L), ph II, kidney K (w/DNA) FEV1>placebo (signif); 2 ph II w/β- :no efficacy, but
- Mobista (Flt 3 ligand), K, ph II;ex-vivo dendritic cells x steroid sparing ongoing
20; tumor bulk¬.
- Trail/Apo2L, K, pre w/ABGX (competition : OSI, AZN)
- Anti IL15 from GenMab, II, RA (Roche has CRB 15
precl)
- HuMax from GenMab, Lymphoma, pre,

w/CAT : fh MAbs
AMGN TULARIK (TLRK) Partnerships on regul of gene expression :
IPO Dec 99 Roche(antiinflam), , Taisho, Yamanouchi, JNJ, JT
Nov 18/02 : Jack Anthony VP Bus Dev (PPAR)
(from Cell Therapeutics) - T 64 ,lometrexol(antifolate), ph II,NSCLC, H&N,
Founded by guys from DNA melanoma, breast, lic from LLY. Term June 02
-T67, ph II irreversible tubulin binder(taxol is not), &
Hepatocellular K : 1M (US 20 000)cases per resistant to MDR,& enters brain (neuropathy at 11 presentations at ASCO 02
year, no approved therapy, 6m survival 50%, high dose),
2 years : 5%. Mostly post Hep C ph II hepatocellular K 2 studies at 165mg/m2 , 1st line 9 PR, 38% stabil
First line completed, 2nd line also. Meet FDA Aug N = 750/survival (& interim)/ IV every week
PharmaVision owns 24.7% 26/02 OK for ph III at 250 mg/m2 ( supported by compared to Doxo 60 mg/M2 every 3w,started March
MEDX acq. 0.2% (Jan 02) PK 12 patients to start). Ph I/II wDoxo 25/03. Could end 2H04, filing 1H05 ????, sales 1H06
ph II in Glioma, breast, NSCLC, colorectal at 330 (faster if fast track : granted Oct 22/03)
mg/m2 FeRx had a ph II/III
Oct 02 : recruit clin VP from Roche, & -T 607, like 67, but does not cross BBB,ph II HCC, Ovary,
regulatory VP gastric, oesophageal.
NHL halted
Shelf priced at $ 25.7m Oct 18/02 - Geldanamycin, res, inhibits IKK (inducible kappa kinase)
-T 611 : oral anti CMV, phII. Term June 02
May 21/03 : w/AMGN acq 21.3% ($ 35m) + -Antibacterials/antifungals : research
125 m committed funding. Could buy more - Anti Xa oral, w/LLY, ph I ∏ milestone Nov 12/02
over 3 years. After shelf AMGN : 10.8% research
-obesity (Knoll) : term Sept 01
March 29/04 : being acq by AMGN $ 1.3B -cholesterol (Sumitomo) : term Jan 01
Completed Aug 16/04 - GPCR targets w/Sankyo June 02
- T 131, PPAR γ agonist ( like Avandia, witout heart
side-effects ) , Diab II ph II UK Dec 2/02
- T 487, phII, antiCXCR3, PSO, RA

- 13 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


w/MEDX : Mabs to 3 oncogenes from TLRK

-ABX CBL,m antiCD147 →GVHD, ph III


AMGN ABGENIX (ABGX),CEGE owns 10.5 % ABX RB2,fhu antiCD147∏GVHD,pre
→SangStat, term Feb 18/03
Dec 14/05 : AMGN to acq $ 2.2B cash. Compl April →SangStat
M 3/06 -ABX IL8 IV/IM →PSO,IV (every 3w;subcut in dev) phII DB. PASI 75:50% or better (Lancet → Amer Acad dermat : march 15/01:significant++
remicade paper)
Fully human monoclonals →RA, ph I/IIֱ∏ph II term. Jan 4/02. No ADR but poor efficacy
→ AADFeb 02(1 abstract,N= 94, 3mg/6/0, DB, PASI ∨ P=0.02, no rebound)) & Soc Inv
Oct 30/02 : staff¬ 15% (N=450) but +4% in ∏ COPD, ph II (results 1Q03), IL8 responsible for excess leucocyte extravasation
∏ melanoma, ph II (Jan 3/02) w/dacarbazine, data 4Q03 : time to progression
3Q/02 Derm May 18/02: PASI 75/15 weeks reached by 3% placebo, 2% 200mg, 6% 300mg
(XenoMouse,acq. from J.T.) -ABX EGF panitumumab Vectibix IV/2.5mg/kg/qw, 2w or JV (restructure?)IMNX/AMGN (resp for ph III)
XenoMouse +SLAM∏XenoMax 3w(anti EGFR, IgG2, but IgG3 very linked to C’) isotype :competition:OSI,IMCL,AZN(Iressa ph
Was spun-off by CEGE in 96 → no C'(this is bad for Rituxan, ASH02 n° 2256),5 times III),MDRX,PFE (CI 1035,TK inhib,phII), ICR 62(phI,
Nov 00 acq ImmGenics (SLAM) more affinity than IMCL C225 (see GenMab data) MAb),YM Biosciences (TheraCIM,MAb,phI), GSK.
Thanks to SLAM , no need to use Hybridomas : can
manuf. in CHO. Feb6/01: lic to Celltech -renalK,phI→phII(april18/01) IL2/IFN failures → ASCO 02 : N = 88,8 weeks, 58% have minor
SLAM(selective lymphocyte Ab method) Accrual complete year end, endpoints tumor shrinking,T. response or stable;( IFN/IL combi gives 18%)ORR 3
-Jan 30/01 : manuf agreement w/LONZA (exclusive
to prgr.,survival, PhI ASCO 01. N=28, 9w. trial, rashes at PR, 2 minor R; TTP & survival available when?.
access to part of Slough site)
Fremont pilot plant will be ready (for 100kg/year)4Q02 high doses, no allergy. EORTC nov 12/02 N = 88; "very encouraging, not quite enough a signal to go to
∏ customers ????? 1/1.5/2/2.5 mg/kg. Acne in 70/91/95/100%. Consistent phase III"says chief med officer. my comment 8 w
At present manuf in hybridoma but will make economic kinetics. At 2mg/kg blood levels > IC90 of xenograft stabilization is not unusual in RCC → EORTC Nov 03
decision on hybridoma or CHO. model
May 24/01 granted US patent "Human MAbs to EGFR" NSCLC 1st line ongoing ph II, rand, data 2H05)
-Scott Green to go from CEO to Chairman mid 02, - NSCLC w/taxol-carbo, ph II (Aug 1/01) data ASCO 04 :
N = 19, ORR 25%, 2/3rd line, Part II N = 175 ongoing .
→ colorectal MonoASCO 03 : 10% PR, 55%SD (will
Withy (COO becomes CEO)
Sept 07 w carbo Taxol +/-706 rd
-Feb 26/02 : $ 200 m convertibles issued -Colorectal refract mono N = 100 ph II (Dec 29/01) data 1H03 response enrol 150). FDA SPA: Jan 20/04 init 3 line
- Feb 27/02 : SEC filing of ownership of declining
shares in CRGN & IMGN. A charge is recorded : $72 m
& TTP (will disclose interim of 40 first if ORR < 10%). Feb 20/03
interim OK !
oxaliplat refractory; Mono, N = 300, 6m
for first 9m/02. rd
ASCO 04, N = 148, 2/3 line, 10% ORR. enrol, 6 m to measure ORR. BLA Filed
ESMO 04, 1st part open ph II, 1st line CRCm w/Saltz, N = 19, PFS 8.2m
Resteering might be AMGN's influence : the anti EGFR ASCO 05 ph II N = 95 CRC, Prostate, RCC, NSCLC, pancreas, March 31/06; FDA OK 3rd LINE Sept
mkt could be the most important K mkt in 5 years esophagus, gastric, same efficacy & safety q1, 2 or 3w
Aug 26/04 : CEO resigns 1st line CRC ph III init April 2005 28/06. AACR : OS NS, PFS = Erbitux.
- Colorectal combo (+ campto 5FU)
- Prostate ph II Feb 24/04 fails internal threshold
Also EU trial ( data 4Q05; endpoint PFS, vs BSC
NOV 05 : increase in shorts Aug 06 : H & N delayed
which is usually 8 w; ABGX at ASCO 05 had 13 w in N =
Competition Genmab HuMax fully human anti EGFR, -Anti MUC18 : melanoma ph I since Feb 03 148). Enrol. full March 18/05. EU Data Nov 3/
ph I/II H & N ASCO 05 - AMGN 162 fully Hu anti RANK ligand , 5% royalties, init July 10/01, 05
ph I osteoporosis, ph II metastasis
- ABX-PTH, ABX 10241, ph I, for dialysis & PTH cancer, IV, good data N = 463, 46% decrease in T progression
VECTIBIX : $ 8,000/month ASBMR Oct 04 rate (p < 10 -9 : time to disease progr or
- anti CTLA4 ticilimumab w/PFE, ph II, CP 675,206, melanoma
- anti IGF-1R/AKT, w/PFE death 96d vs 60 (bsc). Precise PFS ?
-Anti CD28 (w/Diabetogen, CND)∏Diabetes I, res (Erbitux = 6w).
- Anti properdin f hu, pre, lic from Gliatech Nov 2/01 Also ECCO Nov 3/05 1st line CRC ph II, N = 24, ORR 33%, PFS

- 14 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


10.9m. 1 eyelid edema, some CV tox.; Avastin has PFS 9m & ORR
- Smallpox MAb w/US army : Sept01 17%
PACCE : Panitu Advanced CRC evaluation, 1st line, oxali or irino
+/- panitu, N = 1000, init April 30/05,
interim 2H06
(some data YE : increased side-effects,
terminated March 24/07 signif lower PFS &
OS). SALES 4Q06 : 39, 1Q07 51, 2Q 45
EMEA : NO May 25/07 Sept 23/07 OK but
only in non KRAS mutations
AMLN AMYLIN (AMLN) -Pramlintide(Symlin,amylin analog, synergistic to JNJ discont.
insulin), diabetes, ph III failure→ filed dec 7/00; ADV amylin is a natural hormone from pancreas that
Class action suit for false statements (Aug COM JULY 26/01: rejected (severe hypoglycemia & not contributes to postprandial glucose lowering
9/01)
GCP).Approvable in assoc w/insulin Oct 12/01 require ⇑inject. long action w/ALKS : ph I -once a month
Jan 13/03 : shelf for $ 141.7 m (Goldman, more tests, to be filed 1H03.! CPMP filing May 31/01,
Morgan Stanley, Lehman), sold by jan 17 withdrawn Oct 24/02.
Dose rising trial underway (June 02), full enrol (N = 300). FDA asked for "additional clin data to identify a
Feb 03 . Refiled June 16/03. Dec 17/03 approvable letter patient population and method of use where there
asks for sensitivity to hypoglycaemia. Data submitted Sept is no increased risk of hypoglycemia
20/ 04., PDUFA March 20/05 OK March 17/05. Sales 06 $
43.8m, 2Q07 15.2. Oct 1/07 "non approvable" for assoc w
basal insulin
Symlin in Obesity, ph II
-Exendin/Exenatide, Byetta (GLPr agon) SC BID /
AC2993 (39 aa peptide) →diabetes,phII,
HbA1c¬ & fructosamine¬ (ADA june23/01); ph III init
Dec 10/01. Results 1Q03; 2nd ph III in type II init Jan 02, lic to LLY (Sept 20/02), value $ 325 m, upfront 80 +
3rd ph III init 1Q02 w/Metformin. All ph III data (N= 1600) stock 30 + 80% dev costs
filing June 30 04 PDUFA April 30/05.April 29 approved
in combi, approvable alone. EMEA OK Sept 21/06.. Sales
06 $ 430m, 2Q02 152, 3Q07 177 Aug 7/03 partial results of 1st ph III OK, Npv 11 2nd
Oct 16/07 FDA reviews 30 cases of pancreatitis ph III OK, Nov 25 3rd ph III efficacy BUT
Long acting (LAR) init. ph II mid 03, Aug 05 : ph II data hypoglycemias
qw OK (filing 2H09 Plant issue)
- AC162352, injectable gut hormone (L cells,
enteroendocrine cell lineage)) PYY3-36, ph I Obesity; post
prandial release by gut L cells of GLP & PYY (36 aa ). Nastech has inhaleable
AMO IPO AMSTERDAM MOLECULAR (EuroNext) AAV gene therapy
- AMT 011 : lipoproteine lipase gene for
Priced June 20/07 hyperlipoproteinelia, ph II/III, to file 1 Q/08
- AMT 020 acute intermittent porphyria, pre
- 15 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- AMT 030 : hyperoxaluria ; res
- AAV-ILGF for liver cirrhosis
AMRI ALBANY MOL. RES (AMRI) contract chemistry research -royalties for Allegra (SEPR ?) from AVE
-res agreement w/CBST (Aug 20/01)
AMRN AMARIN (AMRN) - Miraxion ( eicosapentaenoic acid) active in subsets ( < 45
30,000 US patients CAG repeats) of Huntington). 2 ph III (SPA Sept 12/05).
US init Sept 21/05, both fully enrolled. April 24/07 FAILS
Myasthenia : 15,000 cases in US (incidence) - EN 101, acq with Ester Neurosciences, NOT a siRNA,
but oral antisense to mRNA of immunogenic variant of
A-Cholinesterase, ph Ib & IIa in Myasthenia not bad
ANDS ANADYS (ANDS) - ANA 975, oral prodrug of isatoribine, small molecule JUne 2/05 part. w.Novartis $ 570m
TLR 7 agonist, ph I; ph I b for H205 in HCV. "intense
immune stimulation in animals" June 27/06 : trial halted
- ANA 380, ph II, Hep B, w/LG Korea
- agonists of TLR 7, 8 & 9
ANPI ANGIOTECH PHARM. (ANPI) -paclitaxel coated stent, phIII A. Heart Nov ??

™Boston Scientific, Cook


ANSV ANESIVA, was until June 06 transcription factor decoys
CORGENTECH (CGTK) - CGT003, adifoligide, oligo transfected to vein grafts by pressure; E2F
decoy : blocks E2F transcription factor, responsible for proliferation of
traded since Feb 12/04 smooth muscles that block arterial by-pass;
ph II CABG at 12m, 33 % reduct of 75% blockage
To merge w/AlgoRx, who has ALGRX 3268 ph III, N = 2400, CABG data 1Q05 (endpoint signif reduct of graft
ph III Powderject lidocaine.: failure March stenosis > 75%) March 30/05 Failure
ph III, N = 1400, PAD, data 2H 04 meeting end March w/FDA to discuss
27/06 regulatory pathways. Enrol compl Sept 03. Dec 6/04 failure, endpoint
graft clinical failure /amputation, but (Feb 3/05) secondary endpoint of
Dec 31/06 Cash & equiv $ 80m arterial flow significantly improved
- NFKB decoy, eczema, ph I/II to init 1H/05; pre Crohn (congr mucosal
immunol, Boston, June 27/05)
- HIF decoy : pre

- 4975 : Capsaicin PEG, sensitive nerve terminal killer,


Total Knee per year US 470,000 Capsaicin competition : NGSX
ph II already very signif,end 06, 1 single injection : FDA requests (meeting Jan 7/07) :
- Total knee replacement, N = 50 , vs placebo : pain d - safety data base 1,500 pts
14 p = 0.007 to init 1H07 :
- Bunionectomy N = 21, vs placebo, p = 0.03 --- arthroscopic shoulder ph II, N = 50
- End stage OA knee, 6 w , p = 0.03 --- Hip replacement ph II, N = 50
- Moderate OA knee, N = 55, open, ---Knee replacement higher dose ph II N = 50
- Elbow tendinitis, N = 22, vs placebo, at 4 weeks p = To init 2H/07 :
--- Knee replacement ph III,N = 450
0.026 --- Knee OA ph II, N = 200
- Interdigital neuroma, N = 58, at 4 weeks p = 0.018

- 16 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS

. FDA meeting Jan 07 requires ph II OA knee init 1H07


& arthroscopic shoulder. Ph III knee replacement
2H07

- ZINGO lidocaine spray files Nov 27/07 pediatrics (N =


1100) reduct in pain signif. PDUFA Sept 22/07; FDA
OK pediatrics Aug 17/07
ph III adults init March 27, data YE 07
ANX K ADVENTRX (ANX). IPO pulled - CoFactor (active metabolite of Leucovorin, 5-10 ASCO 06?
May 22/06 methylene tetrahydrofolate) ph III init Nov 06 in BC
(5FU/CoF vs Xeloda) & 1st line CRC (+Avastin 5FU vs
April 28/06 merges with SD Avastin/5FU/LV) endpoint PFS init June 06, ph II suggests
Pharmaceuticals (emulsion formulation better than LeucovorinCRC, 1st line
of vinorelbine, only needs bioequivalence) Avastin/5FU + Leucovorin or Cofactor (SpA), endpoint
PFS. Oct 1/07 trial IIb CRC 1st line CoF + 5FU vs
leucovorin + 5FU = signif worse side efects w VoF.
- ANX 513 paclitaxel emulsion
- ANX 514 docetaxel emulsion
AOLS C AEOLUS (AOLS) was INCARA (INCR) -liver precursor cell technol (patent issued 03/00) ?? nearly delisted
change July 16/04 -SOD programme 10150, ALS ph II acquired Renaissance (hepatic cell therapy)
many links to Interneuron - OP 2000, ultra LMWH™ulcerative colitis,phII/III w/ELN

APBI A P BIOTECH (APBI), filed oct 3 "Amersham Pharmacia Biotech"


w/MSDW A leading provider of biotech systems, products & services
Floating delayed (March 27/01)
Nycomed Amersham has 45%, PHA has
45%. Pulled April 23/02
APHT APHTON (APHT) Peptide vaccines tagged to diphteria toxin :
V 4Q04 moves to Philadelphia Gastrimmune (antigastrin)G17DT, Insegia →K, Aventis, 30 first gastric pts presented Feb 8/02 : ORR 50%,
May 2/02 : recruit CMO Pat Mooney, their -gastric 2 ph II(+5FU + cisplat) enrol compl Jan 24/02, Feb no side effects due to immunogen. ASCO 02 ORR 53%, but
Thomas Weisel analyst. 5/04 data from 1st line + 5FU cisplat median survival 8.9m. only 1/36 CR. June 12/02 : reports on N=58, ORR 48.3%.
K -pancreas stage 2, 3 or 4, ph III 1st line
APHT is responsible for dev cost, then modif letter of intent Dec 23/02 (royalty ↘, but R/D paid
w/gemcitabine(phase II survival ↗)DB N n= 383
receives royalties. for). Feb 21/03 Fast track for gastric
v/Gemzar , data 15 Feb 05 Failure on survival ( & 2nd
April 1/03 APHT says will not restructure July 18/03 : claims will file in US monotherapy for
v/placebo available Oct 30 03 : median survival 151d &
Aventis deal, and refuses to comment. placebo 83, p = 0.03 in stages II/III & III but not in stages pancreas
Aug 26/04 : files CND monotherapy , then individually). ASCO 05 failure Feb 5 Aventis Press Conf does not mention
Austral, CH in refractory pancreas.; NDA - colorectal ph II amongst 15 onco products !!!....and AVE will file
filing if Feb 05 data OK - also trial for GERD Taxotere in gastric K 2H04 (first line ph III N = 463, +

- 17 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Dec 15/04 to acq Igeneon (Austria), compl/ cisplat + 5FU, 1st line, chemo alone median survival 8.5m,
Taxotere + CT 10.2 m, signif)
March 25/05
Sept 20/04 Gastrimmune partnered w/Xoma
SASY terminated Nov 10/05 -Gonadimmune (anti LHRH) pharmacine→K,prostate
GSK
II→PSA¬ (Aug 00)
- IGN 101 vaccine (Ag is murine Mab tu Hu colon K cells)
that "mimics" EpCAM epitopes (epithelial cell adhesion Serono licensed in an anti Ep CAM monoclonal
molecule), ph III NSCLC, N = 760 (600 enrolled Dec 04). adecatumumab (Dec 7/04)
Compl. end 07. Ph II CRC
- IGN 311 Mab anti Lewis Y (comes from PDLI, SGEN
has some)
ARAY IPO ACCURAY Robotic radiosurgery

traded Feb 8/07

ARDC ARDEA RDEA 119 : MEK inhib, oral, precl : K, inflam


ARDM ARADIGM (ARDM) - Hu r Insulin (15% royalties + fees & bonus=25%) w/Novo Nordisk (building new plant April 01);
∗DEVICE : liquid is more stable and cheaper than powder; increases stake to 23% (Oct 01)
The AERx platform is a hand-held electronic bioavailability 18% (INHL : 10 %); Competition : LLY/ALKS, NKTR, Mannkind
inhaler device platform that : phase II studies ongoing, some published;
- guides the patient to breathe and Ph III init Sept 3/02 (300 pts/2Y, to end 2H04, filing 05). Events :
automatically delivers when respiration is FDA requires this + 700/6m + 500/1Y milestone for initiation of ph III (1H02)
adequate, Early jan01 : carcino concern in rats (was only rumor) ADA June 01:smokers have∨absorption
- allows dose adjustment in increments (of 1 Delays post prandial reduction : trial to stop. EASD Sept 01 : safety in asthmatics
insulin unit). March 7/06 Novo restarts !! July 06 transfer of patents ADA JUNE 02 : IIb : FEV1 OK, DL/CO ?
The device lasts 3-5 years; with disposable to NN. Data 2009.
LIQUID drug capsules it forms a - Pulmozyme, ph II; delivery in 2 min rather than in 15-30 w/ DNA,discont jan 7 for commercial reasons by
Razor/Razorblade approach - ARD 3100 :Inhaled Ciprofloxacin ph II 2007 DNA who intends to enter another agreement
∗PEOPLE : - ARD 1500 : Inhaled Remodulin, trials
CEO R. Thompson is in diabetes since 20 - Inhaled Morphine : on dec 15/00 ARDM took over w/GSK
years; he founded JNJ's LifeScan ( clinical trials due to GSK slowness (merger…). Agreement
GlucoMeter). persists. on jan 16/01 ARDM sells $5m shares to GSK, to Patents : 61
∗PIPE completed Aug 23/01 : $14.6m finance IIb (but ??) Headcount : 208
Manufacturing : filling of blisters and
- Respiratory gene therapy , PRE, w/GeneRx Alphagene adding disposable nozzles
(α 1AT) & Coxagen (for pulm Hypert.)
- 2 biotech drugs
- classical respiratory / Beta 2, steroids, antichol :
asthma, BPCO) w / Cydex, Sept 7/07

- 18 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ARIA ARIAD (ARIA) Aventis terminated Src for osteoporosis Mike Gilman, CSO, left for Biogen
K AP 22594, small molecule EPO mimetic, Pre, IND 1Q03?
patent on NFKB modulation AP 1903 + T lymphocytes transfected with fas & binder of
AP 1902 (when AP 1903 is injected, fas dimerization :
Sarcome incidence : apoptosis), GVHD, ph II
Aug 15/01:PNAS paper Src inhib for osteoporosis
soft tissue : 8,000/year US - AP 24534, Bcr-Abl kinase inhib (also active on T3151
bone : 900/year US mutant), to file IND Q4 07. Sprycel (dasatinib BMY
approved June 06, Morgan S. 2010 : $ 700M) and nilotinib
Sarcoma 5 year survival : (Novartis) are inactive. Only Ceflatonin (CXSP,to file
2008) is.
- stage 3 : 56% AP 23484 blocks VEGF activated pathways
- stage 4 : very few AP 23573 ( deforolimus) w / MRK mTOR antagonist, ph
II, K, ASCO 05 "striking benefit" ASCO 06 very exciting
ph II sarcoma CTOS Connective in sarcomas (bone & soft tissue) N = 212, ph II open,
tissue oncology society doubles PFS ( 17m vs 9 m) compared to EORTC data
base. April 6/07 FDA meeting on ph III, endpoint to be July 12/07 ($ 652) w/MRK. "many trials to be
Competition for sarcoma : (Sept 10/07)PFS, OS depends on type of translocation initiated". Torisel (WYE) approved May 30/07 RCC .
- ARQ 197, to init ph II soft tissue sarcoma complexity of caryotype). Enrol to init sept 07 (N = 650). Trials in NSCLC & Prostate. Cowen forecast $ 25/07,
- CTIC Brostacillin to init ph II Metastatic soft tissue and bone sarcoma, after favorable 1st 75/08, 375/12. RAD 001 (Novartis in Ph III)
or 2nd line therapy, vs placebo, powered to detect 33% ARIA clever trial design
increase in PFS. Init Sept 24/07; data 4Q09 (WYE
Temsirolimus approved RCC Sept 07). ASCO 07 ph II
endometrial meta, good. Other ph II : HRPC,
leukemia/lymphoma, NSCLC
ARNA ARENA PHARMACEUTICALS (ARNA) Fujisawa, LLY (term Nov 14/02), Taisho
Plan to screen the 800 GPCR of the human -T 82 : Achol enhancer, Alzheimer, ph I,may terminate
genome - G protein coupled receptors: insulin secretion, blood Competition : Meridia (Knoll), Xenical (Roche),
pressure, obesity, hippocampal memory, asthma. Acomplia (SASY)
- ADP 356 obesity, ph II, N = 352, 28d, efficacy p <
0.001but headaches & nausea; 5HT2C agonist (like
GSK/Biovitrum who stopped), ph III init Sept 06 : N =
3000
ARPN ARPIDA (SWX) - Iclaprim, diaminopyrimidine. 2 ph III, 1st finished; SSTI,
IV vs linezolid. 2nd ph III : non inf. To file end 07.
Hosp acq pneumonia : ph II
Oral form ph I
- Arpida 709 , inhaled , ph I strong lung biodistribution
AR 2474, pre (March 8/07), > topical mupirocin (was
GSK, pyocyanic acid derivative) MRSA. ICAAC Sept 07,
ph III MRSA eradication 84.7% vs 85.3% linezolid.

- 19 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ARQL K ARQULE (ARQL) Combinatorial chemistry AHP, JNJ, Bayer, PHA, PFE
Summer 03 acq Cyclis ($ 25 m) for - P 38 Map kinase inhibitors
Activated Checkpoint Therapy platform - ARQ 501, ph II, K, reactivates "checkpoints": activation
(ACTP) of E2F-1. April 2/04 lic to Roche upfront $ 15 m, total 276
Staff ↘ 31% Dec 11/02 Pancreas ph II ORR 16% w/Gemzar vs 8% Gemzar alone
Leiomyosarcome 17.5% ORR alone.
But side effect hemolytic anemia
- ARQ 171 ph I, E2F1 pathway- ARQ 761 precl
- ARQ 197, oral c-Met receptor TK inhib, ph I, lic to
Kyowa-Hakko for Asia. ASCO 07 nice ph I ORR 60%,
better than AMG 102. Ph II will be soft tissue sarcoma,
gastric, breast; also ph II in MIT (microphtalmia
transcription factor ) tumors, and pancreas K

ARRY K ARRAY BIOPHARMA (ARRY) - ARRY 886 oral MEK inhib, RAS pathway (mitogen MRK, ICOS, LLY, IMNX, TRMS, DNA
Traded since nov 17/00 activated extracellular signal regulated kinase kinase) lic to
mMelanoma PFS 1.9m w/Temozolamide, vs AZN (AZD 6244) , ph I; Dec 18/03, upfront $ 10m. Ph II Data ASCO 08
1.5 spontaneous, p = 0.012 melanoma N = 182, 1st line, vs Temodar, init Sept 06.
OS DTIC 6m, Temozol + Thallium 13m Primary PFS/OS/ORR, secondary in patients w/BRAF
mutations More at AZN R/D day Dec 7/07
Deal w/CELG Sept 24/07 $ 40 M upfront, NSCLC phII vs Alimta 2nd/3rd line
total up to 1B CRC ph II vs Xeloda 2nd/3rd line
Pancreas ph II 2nd line vs Xeloda

- ARRY 704 selective oral MEK(RAS) inhibitor, ph I init


April 07, w/AZN (AZN 8330)

- ARRY 162 ph I for RA , oral anti MEK

- ARRY 797 oral p38 MAP kinase inhib. RA. Ph II

- ARRY 520, Kinesin spindle protein inhib, ph I init May


3/07

- ARRY 380 oral ErbB2 inhib. IND filed Aug 3/07 for ph I
Herceptin resistant breast (competitor Tykerb)

- ARRY 543 dual Her2 / EGFR inhib, ph II, Breast, could present at AACR Oct 22/07 or San Antonio Dec
prostate, NSCLC, ovary, colon. Data end 07 12/07

ARWR ARROWHEAD RESEARCH Corp (ARWR) research funding company, in exchange for exclusive key investor of Calando*

- 20 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


rights
ARYX IPO ARYx Esterase destroyed drugs rather than by cytochromes

filed August 31/07 (MS) - ATI 7505, ph II, cisapride like


- ATI 5923, ph II oral anticoag
- ATI 2042, oral for atrial fibrillation
ASM (ASM/LSE) - Theragyn(Pentumomab), murine *Y MAb anti PEM lic to ABT (amended Jan 5/02 : ASM to pay dev.
(polymorphic epithelial mucin : HMF G1): costs), lic from ICRF
-ovary K ph III (one single intra-abdominal shot,
M after surg. + CT), seems to considerably lengthen survival
(there was a short interupt. of trial) ….all papers carry same name
K -gastric K, ph II ⇑results mid 02, filing 2H02. June 6/01 : FDA
- Therex (humaniz HMFG1) : breast K, phI/ II requests additional patients∏ filing not before
- Therafab : F(ab)2 of HMFG1; IV, phI: NSCLC 2004∏2005. Completion 2H04 (trial will finish when
- AngioMab : radiofragment of BC1 (that binds to total 116 deaths across both groups).
fibronectin),pilot study in Glioblastoma
- Thioplatin, K, pre
- ASA 404, DMXAA (dimethylxanthenone acetic acid), lic other vascular disrupting agents : Oxigene CA4P,
to Novartis, upstream to P38 MAP kinase, vascular AVE8062, Myriad Azixa, Nereus, MediciNova MN-
disrupting agent induces cytokines via NFKB : 029 / Angiogene Pharmaceuticals Ltd, ABT751
ASCO 05 QT & visual issues " 1st vascular disrupting agent in late stage dev"
NSCLC (all cytologies) ph II, 1st line rand w/carbo taxol Novartis Bus day Sept 12/07
ASCO 06 OS 14m vs 8.8 (HR 0.73); 2nd ph II data March
8/07 : ORR 50%; Int Lung K conf 07 OS 14.7 m ORR
37.9%
Ovary, rand 2nd line, ORR 27% vs 24%
Prostate (ASCO prostate Feb 07) + taxotere : improved
PSA response rate. ASCO 07 ditto. Oct 15/07 improves
ORR but TTP only marginally improved
April 19/07 lic to Novartis $ 890, ph III to init 2008
-Enzyme that liberates cyanide in K cell (res)
- AS 1409 fusion protein IL12 & antibody to tumor blood
vessels
ASPV ASPREVA (ASPV) priced March 4/05 Cellcept (mycophenolate) from Roche for LED ph III, K
Pac. Growth, ML vs cyclofosfamide, myasthenia (ongoing, 136 sites, to end
late 06), Lupus ph III vs ciclophosphamide : not superior
(June 27/07). Patent expiry 2009
ASTM AASTROM (ASTM) Replicell kits for auto bone marrow & osteoblasts : May 15/01 : key patent issued on "cassette" approach
V -Dendritic cells DC 1(for use as anti K vaccines) product for growing human cells
working with leading German dendritic cell in trials Germany & US, could commercialize Germany
C centers (German subsidiary Zellera AG) -osteoporosis ph I/II init oct 31
- 21 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- jaw bone fracture, ph I/II
- repair of diaphyseal fractures, report March 22/06 ph
I/II; 12 m follow up 90% very satisfactory (Oct 20/07)
- TRC (tissue repair cells) for hip osteonecrosis, ph II,
March 23/06. Ph III to init summer 07, N = 120 (20 sites),
open, carrier alone or w/cells; follow up 2 years; Oct 4/07
German Soc Stel Cell Therapy : 4 osteonecrosis patients
with good results : no hip replacement, back to work !
- April 30/07 init ph II critical limb ischemia PAD
ATIS C ADVA. TIS. SCI (ATIS) Dermagraft (from foreskins), MKT(UK) ;non-approvable Smith & Nephew (Nov 2/02 sale of Dermagraft to S
Medtronic has 7.7% by FDA in 98, refiled aug 25/00⇑ diab foot ulcer approved & N completed)
Oct 01
ATL ACTELION (ATL Zn.S), 936767 SEE EUROBIOTECH

ATLC ATLANTIC VENTURES (ATLC.OB) -synthetic analgesic cannabis


-ABI 001, myasthenia, pre,w/GZTC
- aerosol antisense to RSV
ATRX ATRIX LABS (ATRX) drug delivery systems
Aug 10/00 technol lic to PFE -Atridox,periodontal dis., MKT
-Atrigel:sustained release (1 month) Leuprolide SC,phIII, ™ less painful than IM,(competition:
Secondary filed July 25/01 filed March 27/01. OK Jan 24/02. Eligard 6 months TAP, AZN, AZA) lic to SASY for N.Amer.&
duration MediGene for EU
June 14/04 QLT merging w/Atrix - Atrigel 3 months in ph III (filed Sept 26/01)
- Atrisone (dapsone),ph III, acne, lic to Fujisawa
w/HGSI (Aug 20/01) sustained release protein

AVAN V AVANT (AVAN) -TP-10,C' inhib, sCR1 soluble Complement receptor 1 Novartis terminated transplantation deal
(cardiac surgery CABG ), ph II. Fails. Feb 02, but
Oct 01 : lic vaccine to DynPort important benefit in males . Feb 04 trial init in 300 women,
full enrol 1H05, data 3Q05
- TP 20 sialyl Lewis form of TP 10
-CETi-1,vaccine for atheroma,phII, to ↗ HDL chol ph II

-RSV vaccine,phII→AVE
- Anthrax, plague vaccine : pre
-Rotavirus vaccine,phII (Rotarix)→SBH/GSK; MKT
- oral cholera vaccine : ph II
AVGN AVIGEN (AVGN) - AAV/IX, ph I/II BAYER
-challenges to keep in mind : -genes might be more immunogenic
than proteins →facility scheduled
BAYER risk
-upscaling production.
-IM route limited by N of injection sites & post- translational June 19/01 : AVGN abandons IM route for AAV/IX
- 22 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


saturation (pre) because of Abs. AVGN will go into hepatic
Oct 25/02 : staff ¬ 28% Mark Kay's center had been temporarily halted delivery.
- Intrahepatic art.started Aug 16/01, but the issue of hepatic
carcinogenicity of AAV in mice is debated (2 publications Sept
April 4/05 stopping all AAV
5/01). Halted because vector in semen. Restarted Feb 4/02. N = 6
(ASH 02 : circulating levels). Terminated June 04
June 1/04 Coagulin B Phase I terminated
Key : Katharine High (Penn) - Park : AVI 201, gene delivery to striatum of AADC (converts
brain levodopa to dopamine), init Dec 16/04 LIC TO GENZYME

- Pain : AV-411, AV 333, IIa, PDE inhib


- Spasticity : AV 650 Tolperisone (MKT in EU, Mydocalm,
Hungary, has a history of anaphylaxis) lic from Sanochemia Jan
17/06. US ph II to init 1Q07
- AV 513 : algal fucoidan for hemophilia

AVII M AVI BIOPHARMA (AVII), was Antivirals Abgenix (human MAb to hCG), Preclinicals.
K Inc to initiate. Neugene partnership w/EXEL

Pipe May 2/03 : $ 15m -Avicine is a hCG vaccine for K –pancreas, phIII™™alone
V equal to Gemzar, assoc. improves results ™Avicine partnered with SUPG for pancreas
Sept 29/05 "exon skipping pre-RNA -colorectal,ph III since Jan 01
interference technology (ESPRIT) -Neugene,antisense to c-myc for restenosis,phII, lic to
Medtronic (May 23/01), terminated, lic to Cook March
13/06
- Antisense to XIAP genes (that block apoptosis), pre (see
Aegera )
- AVI 4126, antisense to c-myc, ph I/II,polycystic kidney
disease; in rats oral OK Jan 23/03
- AVI 4557, w/midazolam (BZD hypnotic) to modify
Cyt450, also decreases Taxol catab
- AVI 4451 antisense to androgen receptor, prostate K,
pre(Nov 17/03)
- Neugene anti P 450, ph I
-Antisense to TGF β™stem cell multiplication (feb 7/01)
-TAG6,telomere inhib :RES
- antisense to SARS coronavirus, pre
- AVI 4020 ph I/II, West Nile; croses BBB.
- antisense to H5N1, pre in-vitro good Jan 20/06
- AVI 4658 ESPRIT (Exon skipping pre-RNA interference
interference techno logy) ph I to init 1Q 07 (UK) with
morpholinioligos for Duchenne
AVNR M AVANIR (was LIDAK), (AVN) -Docosanol cream (Abreva)™herpes HSV1, FDA GSK, Yamanouchi

- 23 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


OK:june 26/02
Spinoff : Xenerex, fully Hu MAb (Hu -AVP 923 oral (Neurodex, Zenvia, dextromorphan!+
lymphocytes to SCID mice), but patent also quinidine),ph II/III→ emotional control , signif 5june
assigned to IDEC (acq from IDEC feb 01) 22/02 on pseudo bulbar sd of ALS. Ditto Nov 20/02 (ALS
sympo Melbourne),30/06 approvable. MS ph II/III init Nov
02 on PB sd.
Rolling filing PB init Dec 04, also trial in neuropathic pain.
Completed summer 05. FDA delays PDUFA to 1H/06.
New data Dec 16/05. Oct 06 "approvable", new trial May 01 : 3 cancer Ags provided by EOS
needed.
AVP-28555, MIF inhibitor for inflammation, pre,
w/Novartis
- AVP 893 : inhibits IgE synthesis, pre, decreases IL4 & 5
in BAL of mice (AVP 13358)
AVNC ADVANCIS (AVNC) Oral pulsatile delivery of ABs : tablets of multiple pellets
IPO Lehman, Pacific Growth) Aug 4/03 types, 3 in ph I/II, once a day
- Augmentin Pulsys lic to GSK, ph III to init 2005, Term
Nov 19/04 : staff reduct. 18% Oct 19/04
- Ampicillin Pulsys ph III to init Oct 05 : once daily for 7
days vs peni 4 each day/10d. June 16/05 fails
AVRX K AVALON pharma (AVRX) AVN 944 oral inhib inosine monophosphate IMP is a precursor of GTP which inhibits caspase II
dehydrogenase (lic from VRTX Feb 05) dependent apoptosis.
IPO May 3/05, Legg Mason . hem/onc, ph I Competition for 944 : Cellcept
Res :
- MYC inhibs,
- Betacatenin inhibs,
- Aurora kinase inhibs
AVXT AVAX (AVXT) -autocell therap for melanoma,M-VAX,phII→phIII to last Interim data (nov 13) : 2 year survival of 60% of 37
5 years…March 23/01 : on hold by FDA ( some non first patients. But AACR March 01 not at all
V Aug 24/00 : acq Genopoietic SA sterile batches), hold confirmed Sept 25/01;warning conclusive.
Feb 01 : CEO leaves Nov 9/01. Launch ( by Pro-Vaccine AG), produv=ced
C April 23/01:2 VPs resign by Genopoietic, CH Oct 05
K July 14/05 french trial peritoneal metastases
Dec 23/02 : C00 resigns - NSCLC stage I/II adjuvant vaccine, init Jan 06
-HSV/TK for heart transplatation immunosuppression
- Gene therapy for SCID X1
AXO M AltaRex (TSE: AXO), acq by VIRexx in -OvaRex, anti CA 125, m, ovary K, II/III, but from ascites Production discussions ongoing w : Lonza, Genesis
K 2004 rolling BLA filing 2H01 ???.. Diff. results US & CND, but Pharma (Greece), Medison (Israel)
combi signif (Jan 31/02). Lic to UTHR Lic to UTHR for US (April 22/02)
AXPH AXYS (AXPH) -Combinatorial chemistry™™™™™ ™PHA, AVE, Daichi
- 24 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


From Sequana/Arris merger (1997) -drugs : -tryptase inhib. for IBD, ph II
Equity in DPII (IPO 7/7) brings -tryptase inhib for asthma, inhaled, pre™™ ™Bayer Discont (May 24)
AXPH cash for 5 years -Cathepsin inhib for osteoporosis, pre™™™™ ™MRK
acq by Celera (June 13/01)

AXYX AXONYX (AXYX) - Phenserine, Alzheimer, ph II AChE inhib, M+ & inhibits


product β amyloid (PNAS June 14/01)
- Peptide against amyloid , Alzheimer, w/Serono,pre
- Peptide against prions, w/Serono, pre
AZN CAT Cambridge Antibody Technol. (CAT/LSE) to be acq by AZN (May 15/06) $ 1.3B
M Nasdaq : CATG SEE EUROBIOTECH
AZN M MEDIMMUNE (MEDI) -Synagis (palivizumab)→RSV MAb hz :$293/99m→427/00; 516/01; ABT (ROW)
FOR SALE April 12/07 > 12B 672/02; 849/03; 1Q04 : 422, 2Q 56, 3Q 61, 1st 9m 2005 $ 624 (US 526); June 03 : A. Acad Pediatr. tightens rules
FY 05 1.1B, 1Q06 463; FY 06 1.1B
manuf essentially by B. Ingelheim.
V Synagis : $ 3500/infant (?)
Cong Heart (30000 births/y US, 50% for Synagis)ph III ∏ AAP Oct
Dec 17/02 panel FluMist:
18/02. DB N = 1287, 4 years ∏ 47% less RSV hospitalizations. Will file
- safe & effective ages 5-49 US/EU
- 50 to 64 ; safe 10/8, effective 4/14
K Barbara Fischer (Pres Nat Vaccine Info Ctr) : NO,
-Cytogam(anti CMV) :$30m/99,36.5/00, 2Q04 : 8
-Respigam→RSV polyclonal :$4m
danger of injecting live virus. -Ethyol :$75m, 2003 : $ 100m
Approved June 17/03 : -Medi 507,siplizumab, IV/sc( hz anti CD2)→GVHD, ph II;PSO ph
II→ph III delayed (induces Abs !!), precl +in TCL xenomice
5 – 17 : 2 doses →w/GSK Cervarix. Ph III init 1Q04 (Baculo difficult
18 – 49 : 1 dose -Medi 501,2 &3(anti HPV16/18 vaccine), ph I & II™™
(FDA Nov 28/01 on surrogate endpoints) to scale up). MRK (Gardasil)in ph III (BLA filed Dec
launch Sept 03. Wye sales 4Q03 $ 15m ?
-Abegrin Vitaxin(hz MAb to alpha/nu/beta3 integrin, CRC Asco 03 05) also active on HPV 6 & 11 (men). MEDI has 4
Copromotion w/WYE, 50% revenues to each (but
COGS high); 1Q04 : $ 31, 9m 04 : $ 33
Melanoma ph II, prostate ph II; PSO & RA ph II term Aug 30/04. AACR patents on HPV VLPs (some also sublic to MRK).
06 good ph II m. Melanoma, ph III to init YE 06. Decision to stop CSL (Austr)has adjuvant lic to MRK. Adv Com May
Vitaxin + dacarbazine < dacarbazine alone
- 2nd generation Vitaxin, ph I/II, K 21/06 +
-Numax, motavizumab,respiratory MAb to RSV; 2 formulations, 1st ph III
New CEO was with MRK vaccines, and Merieux enrolled, 2nd starting (Nov 2/ 04
Fully enrol N = 6600 Dec 9/05. Filing delay (Feb 07) collab w/ Ixsys (now AMEV)
-E. coli vaccine for UTI,ph II, stop Oct 02 (poor efficacy) collab w/ Ixsys (now AMEV)
Aug 31/05 reacq EU Synagis starting June 06
- MEDI 491, B19 parvovirus vaccine, ph I
→Alkermes & AMEV
- Anti IL9→asthma,pre lic from Genaera (april 01) being improved by
AMEV
w/Vasgene (Sept 05), pre, Mab to Eph B4 and Mab to its ligand Ephrin
B4 ligand, cancer.
w/Burnham Institute res on peptides targeting EphA & EphB subfamilies
AVIRON (AVIR) of tyrosine kinases
Acq compl Jan 17/02 -FluMist, live attenuated nasal vaccine : ph III, had manuf. probl.
MKT issues : w/Medeva ; CLL vaccine unit acq by Powderject sept 8, AVIR leases
-US flu vaccine mkt is $ 200m (35% AHP, 35% facility and is manning production and building a new facility.
CLL/Medeva, 20% generic, 10% Aventis). But Has to be kept in freezer prior to administration
Aventis ww sells €200 m of flu vaccine, and is Not so easy to use : one shot from syringe in each nostril -WYE (who already sells Pasteur/Merieux type
- 25 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


looking at US. - LiquidFluMist,CAIV-T, ph III init Oct 21/04, does not need freezer, to vaccine), increased stake in April 02: 50/50 JV. Jan
-Tamiflu : sirup and prevention since nov 00. file 2Q06. Filed 3Q06. PDUFA Feb 07. Approved Jan 8/07
-Parainfluenzae III : ph II
04 WYE analyzing head to head; could terminate.
Sales 00 $ 81m, E/01 : $170m April 27/04 terminates.
-EBV : ph II (with GSK)
Price issues : -BLA filed oct 31/00,accepted for review dec 29/00, Adv Com :
-CMV : phI
-Standard shots are $10, FluMist $46 (CHIR has - Dec 15/05 lic from Biota oral anti RSV July 27/01 Efficacy OK(13/2 in adult, 8/7 in pediatric & ado),
increased Fluvirin post Powderject acq. Was $ 2 - MEDI 545 (w/Medarex), fully Hu Mab anti INF alpha. Lupus, Ph I init safety 9/6 against. Could be 1 year delay. Sept 5/01 FDA requests
!). Feb 04 : will rebate 50% 1Q06. Ph I init March 24/07 clarification on clinical & manufacturing. Berna/AVE in
Regulatory issues : - IPI 504, w/Infinity, ph I, HSP 90 inhib Switzerland is having safety issues (facial palsy) after "Nasalflu".
-FDA site inspection ? Possible approval 3Q02. AVIR files its response to FDA's "full
response letter" Jan 8/02.
-Recombination with other viral genes
-Risk in immunocompromised patients → July 02 CRL w/68 questions, 24 manuf, 44 clinical (&
- Asthma ? asthma). Answers provided Aug 27/02. FDA can respond within
2 or 6 months. PDUFA Feb 27/03 , Jan 31/03 CRL w/5
- Use of assoc. vaccines ?
questions labelling, PMS, assays) Feb 7/03 response
provided.

BAVN Bavarian Nordic MVA-BN Her 2, Breast K vaccine, ph I/II init Jan 07
BCRX K BioCryst (BCRX) - BCX 1812 : Peramivir (RWJ-270201)→Flu, oral JNJ⇑dec 26 JNJ stops trials in US, continues
neuramidinase inhib, ph III initiated feb 8/00 in ROW, Jan in Europe. Deal terminated (april
July 12/02 staff ¬ 43% 3/02 in US. Enrol could finish March 02. once a day, also
PIPE Feb 17/04 Orbimed $ 21.4m has IV formulation. Active on more subtypes than
30/01)because of insuff. activity, not safety
Sept 05 BioDefense Committee requests stockpiling.
competitors. June 25/02 failure of ph III. Poor
HHS says "not enough funds". Jan 4/06 HHS : $ 102
Feb 16/06 funding from Kleiner Perkins bioavailability, but excellent efficacy (and on H5N1).
m
"Pandemic & Biodefense fund". New IND (IV/IM) filed Nov 29/05. Fast track Jan 17/06.
IM init 2H 06, IV vs Tamiflu ph II init hosp Aug 07
ICAAC Sept 06 : ph I IV/IM very effective levels
June 15/06 Peramivir lic to Green Cross March 12/07 partn w/ SCOLR Pharma (Amex DDD)
(Korea) for oral sustained release
induces unbalance of nucleotide triphosphate pool,
June 18/07 positive data IM : bioavailability & safety.
producing apoptosis of high mitotic index cells
Sept 20/07 results from 313 patients IM NS : median time
to alleviation of symptoms 11.9h with 150 mg , 21h w/ 300.
ASH, ASCO ?
NS vs placebo. Earlier ph I used 40 mm needle, switched to
user friendly 2.6 mm. 101 parients had adequate IM
(judged on CPK) : improvement of 64.8h at 300, 44.6h at
150; Tamiflu trials improve by 1.3 days
- BCX 1777 Fodosine, IV & oral, purine nucleoside
phosphorylase inhib for T cell lymphoma, ph II, not
myelosuppressive, partnered MundiPharma/Purdue/NAPP,
Feb 3/06. Pivotals to init 3Q06. March 27/07 IV, T cell
ALL IIa : not as effective as at ASH. Also particulates in
solution. Wants to go oral for CTCL

- 26 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


BCX 4208 purine nucleoside phosphorylase inhib. for
acute graft rejection, ph I, lic to Roche Nov 30/05.($
560m). PSO init July 07
- BCX 4678, Hep C, to init ph I
BDAL Bruker Daltonics (BDAL) Mass spec to identify contaminants

BHRT IPO BioHeart Myocell, autologous muscle cells for heart failure, ph II but competitor Myosix has poor results
filed Feb 14/07
BIIB M BIOGEN IDEC ( BIIB) Avonex(β1a,CHO,IM,once a w. no skin ulcerations, 2 year →sales 00 : $ 765 M;01 : 971.6 (+28%),3Q02 $ 262 ww (+
FOR SALE Oct 15/07 study)$ 621m/99(+ 55%) 5%) of which 186 US (+2%), Rebif ww $ 139m (+ 46%) &
Avonex patent expires 2011 ???? (Bgen Orphan drug protection against Serono expires May 2003. US 18. 4Q 02 Rebif ww 172 (US 34.7); Avonex 256 (US
K only has lic. to patents of dates unclear, <Betaseron (A. Acad Neurol May 6-11/01), Serono (but
6months,DB not feasible : different aspects) ditto w/Rebif( 184). FY 02 BGEN 1034 ( + 7%, w price ↗), Rebif 549 (+
Taniguchi, Roche. Berlex has the subcut,00 : $254) May 8/01 77%). 1Q03 274 (US 189, ROW 85), Rebif 175 ww (US
McKormick patents last issued 98, lic to March 14/03 FDA warning on depression & liver 40), 2Q03 286 (US 196) Rebif 199 (US 43), 3Q298 (US
Serono & court case w/Bgen settled $ 55m March 4/04 : potential product issues (new liquid formulation 204)4Q 310 (Rebif US 55). FY 03 1.2B (Rebif 850); 1Q04
Feb 03)) prefilled syringe) pain, pH 4.8, competition 7.. 355 (US 240) (Rebif US 63, ww 259) 2Q 347 (US 227,
Serono files sBLA Oct 8/01 ,FDA OK March Rebif US 69.6 & ww 257)3Q 346 (US 224, Rebif US 77 &
8/02. 25% of Avonex sales are ex-US : ww 186). FY 04 1.4B, 2Q05 382; FY05 1.5B, 1Q06 393,
3Q06 445, FY 06 1.7 B, 2Q07 462, 3Q07 455
CHAMPS shows efficacy in early MS. -Amevive (alefacept), LFA3/IgG fusion prot (anti-
CD45RO+;binds to LT's CD2 preventing interaction w/APC's 48w Evidence (Rebif/Avonex): % pts relapse free, weeks:
CPMP OK, filed US
LFA3)→PSO,IV/IM (deep frozen) ph III:PASI 0 → 24 24→ 48* 0 → 48
April 16/02 : launches 2 stage trial : Rebif 75 82 62
75:14%IV,21%IM,(LT¬!),filed (2 pivotals w/different
- stage I : evaluate pts having received 15/21 formulations)FDA/EU Aug 6/01; launch 1Q03? Avonex 63 83 52
months of Avonex 1/w or Rebif 3/w Feb 20/03 : CPMP says NO * for the subset exacerbation free at 24 weeks
- stage II then go on following each group for Once a w 12 w course (IM or IV) costs 7 to 10000 Could infringe DNA patent
3 years on clinic and MRI CD4 must be monitored weelky & used to adjust. A. Acad Derm (Feb 22-27/02) : IM. 21% pts reach PASI 75.
Antibodies : Avonex 5%, Rebif 24% ?? Forecast 115/03, 230/04. LAUNCH. Sales 1Q03 : $ 4m, 10% have T < 250 (risk of TB, fungal, CMV etc &
reimbursement ??, 2Q03 : 7m, 3Q 12, 4Q 17; FY 40. Lymphoma) Adv Com : May 23/02 OK 8/2. CRL June
PFE w/Serono will boost Rebif from Oct 02 onwards. 1Q04 : 13; 2Q12, 3Q8, 3Q 10, FY 04 43. Sept 9/05 : plans to 14/02 requests clarif & info "on data filed". Labeling
PFE commiting 450 reps (17000 untapped general divest. FY 05 48 : selling to Astellas April 4/06, $ 60m includes weekly T cell count. Resubmission filed end
neurologists). -Adentri, Adenos. ant., natriuretic→CHF,phII,(I.V)STOP Aug 02; Class 2 answer Feb/March 03 (concerns on PMS
Manuf : N. Carolina 6x15000 l fermentors to be certified
Tysabri / Antegren /Natalizumab(Hz MAbantiα4β7 & T cell counting or overall lymphos ?). FDA OK Feb
for Amevive & Antegren 1/03
,α4β1/VLA4), IV, once a month $ 23,500/m
May 25/04 : FDA filing MS, PDUFA - MS, CV Therapeutics
25 Nov 04, OK Nov 23/04 POSITIVE MS data Jan 22/01(ph II, DB) presented Sept w/ELN; 2 Ph III in MS init. Dec 18/01. US +/-
14/01,once a month IV/6m (12m follow): new MRI lesions -88%Avonex (Sentinel N = 1200 fully enrol YE 02 to be
Crohn filed EU Sept 28/04, later in rand YE 03 to Avonex + placebo or + Antegren),
(p<0.0001 to placebo), relapse rates reduced 66% p= 0.03. But
US interim at 1Y: June 03→ early04 to look at both
prior 1 m trial relapses>placebo. New ph II, N = 213: Jan 3/03 :
June 4/04 EMEA filing MS MRI 93%, relapses 13, placebo 27. trials(MRI + flares). EU /Placebo (Affirm N = 942),
full enrol end 02, follow up 2 years.. Sentinel shows
Sales 04 (1 month) $ 3.1m
- Crohn POSITIVE ph II May 23/01(ph II,DB); 2nd ph II Jan 3/03 Antegren + Avonex better than Avonex alone on
Feb 28/05 : 2 pts w/Progressive on remissions 6mg/kg NS, 3 mg/kg signif. Ph III Enact I (850
relapse rate (p < 0.001)
enrolled), Enact II also Nov 8/04 : 1 year data Affirm clin. relapse reduced
- 27 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Multifocal leukoencephalopathy. JC 66%. 2 year disability available Feb 16/05 : risk of
- Feb 04 : RA starts disability vs placebo reduced 42%
virus destroys myelin sheaths
April 17/03 : weird letters in NEJM on Antegren
Pulled March 1/05. BLA refiled Sept - CDP 323 from UCB (Oct 3/06) oral alpha 4 integrin inhib. Ph I
27/05. Adv Com March 7/06 : OK, DATA CROHN : ENACT I (endpoint 70 pt reduct
CHMP April 28/06 OK score) missed July 24/03. ENACT II (maintenance)
April 28/06 approved EU signif Feb 04 (but relevance ? : rerandomize success
June 5/06 approved FDA (monotherapy & pts of Enact I to Antegren once a m/12m versus
restricted). Sales 2Q06 5m, FY 06 : 38m, Placebo : 44% maintain remission , & placebo 26%)
2Q07 72 (46.8 in US by Elan, 23.3 by BIIB
in EU), 3Q07 93

- 28 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


BIIB M IDEC see BIIB -Rituxan/MabThera (chim. antiCD20)→NHL→→→→ US sales booked by DNA, Roche sales in EU (R =
NHL 56000 new cases/year US. 5 year (ASCO 01 : good data for maintenance therapy) 10%). Revenues are shared profit, reimbursements,
survival 52% Aug 31/05 Filed for TNF refractory RA, PDUFA late bulk sales, royalties.1Q02 $ 80m (34% US sales),
For Rituxan ($ 16,000/year) : Feb/early March 06 (Orencia PDUFA DEc 31/05). OK 1Q03 111
-Competition : IMMU/AMGN Feb 28/06 (IDPH books 34% of US sales)
Lymphocide, ph III ASCO 03 : 21 presentations, 6 on CLL EU 2001 Roche MabThera $ 146m, 2002 E $ 450m
- GSK patent litigation :2 suits filed Not effective on RCC (# 688) ASH02 : maintenance → event free survival 23 m
against DNA. Sept 11/01 files suit - Panaclar fumaric acid ester (BG12) for Psoriasis, with, 12 without; ASCO 04 PFS 4 years 58%/34%
Fumapharm AG acq June 06, oral BG12 active in MS
against CRXA & GSK
(ph II), ph III init Jan 07 → Amer Coll Rheum N = 122 (final results N = 161
-Zevalin, ibritumomab, Y tagged mur. anti CD20,BLA filed nov 1/00 "later")Oct 29/02: ACR70 + MTX : 23%, alone 13%
;priority review,FDA letter May 3/01: (= Enbrel, Remicade). Best dose not known yet
°CLINICAL ph III : N= 113 (DB) + 57 EULAR June 18/03 : ACR 20/50/70 @ 6m = Enbrel.
IDPH, will contract out its MAb _no additional trials necessary before approval
manufacturing capacity _need to analyze incidence of infection, and interaction w/G-CSF
EULAR 04 ditto; ph III REFLEX initiated for
IDPH has 32 reps (+ DNA onco 105) _precede Y-Zevalin with Indium-Zevalin for imaging "refractory to anti-TNF" Nov 2/04 positive top line
'could show nearby vital organs IIb. April 6/05 > placebo ACR 20 w 24, only
' will require a double kit ! injected d 0 & d 15. EULAR June 05 ph IIb OK.
' phase IV requested in patients already treated with murine Ab, at
risk of HAMA.
ACR Nov 05 ACR20/50/70 > placebo; 2nd cycle
Zevalin kit :$ 17 to 28 000 : Medicare (Date ??) N = 78, data unclear. But very good in
°CMC (chemistry, manuf, controls)
reimburses from Oct 1/ 02, $ 21,911 _NDA for Yttrium to cross reference with DMF Lupus
_Y suppliers & filling partner to be inspected BMY Orencia approved Dec 27/05
30% of Rituxan pts become refractory BLA refiled July 9/01: ODAC Sept 11/01: recom. for Rituxan resistant
pts, & discussion on accelerated approval approach. Jan 7/02 (Catalytica
opportunity for Zevalin/Bexxar (and manuf issues ??), Compl. Rev. Letter Jan 08/02. OK Feb 20/02. CPMP
need to not deteriorate bone marrow, so filed Jan 01, OK Sept 23/03 (Schering AG). US launch March 27/02;
as to use 3rd line CT) Also Lymphocide. 1Q03 : 5.7, 3Q 03 4.4, FY 03 : 20; 1Q04 4.8; 3Q 5, 4Q 8, FY 04 > 50% pts grade 3 neutro or thrombopenia
April 25/02 : raises $ 675 through sale of 23
-IDEC 152(hz antiCD23)inhibits production of IgE→asthma, ph I
convertibles
Nov 29/02 President & COO files to sell AAAAI → March 03 ?, anti K synergy w/Rituxan,
-IDEC 114,(galiximab, hz anti CD 80,B7-1) →PSO, phII term. Sept 5/02;
300000 shares over 12m (has 1.5m) ph I/II in NHL, associated to Rituxan good data June 14/05 Int Conf
Malignat Lymphoma Lugano, Jan 29/07 ph III init
lic to Seikagaku (Europe + Asia)
-IDEC 131(hz anti CD 40 L)→ITP, MS,PSO,Crohn, ph II, on hold (June
02) leg thrombus in 1Crohn patient (N=200)but BGEN had halted in 99 lic to Mitsubishi (Asia)
its anti CD40L because of thrombosis.
-IDEC 151 (hz anti CD4)→RA, ph II (returned by GSK early 2000) lic to Eisai (Europe & Asia)
- HMN 214 lic from N. Shiniaku, oral stilbene prodrug, ph I, K
- anti LTBR Mab , pre (lymphotoxin B receptor) from BGEN (Feb 03)
- anti CRIPTO Mab (cell surface signaling prot, from BGEN ( Feb 03)
- IFN β gene for glioblastoma
- June 03 : new Hz anti CD20 w/DNA, res
W/PDLI : daclizumab, volociximab, fontalizumab

- 29 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


BIIB -June 01: partnership w/NsGene (subs of NeuroSearch) for
Jan 4 /07 Acq Syntonix (40 m + 80). Neublastin in neurodegener. dis
Specialist of FcRn (protects and transports -sept : jv w/Eos : anti K compounds
across epithelium), already partnered - VLA-1 inhib, pre, asthma, lic to MRK (1997)
w/Serono & BI (inhaled peptides) - IC747 LFA 1 oral inhib w/ICOS, ph I. Term June 6/03 w/Morphosys (dec 00) :MAbs to EST proteins
- Hep B vaccine lic to GSK, sublic to MRK w/ABGX (June 13/01)
- IFN α lic to SGP
- Humicade, ph III, Crohn, w/CLL (April 25/02), fails,
could be active in subgroups
- 3 early stage anti K lic to IDPH (Feb 1/03)
- anti K partnersip with Sunesis
- BAFF-R/Fc of IgG fusion protein, pre, that inhibits
overstimulation of B cells by BAFF (B activating factor of
TNF family = BLys) on BAFF-R3, RA, w/DNA
- IFN beta Adeno gene therapy hepatic metas : pre
- Oral Fumarate, PSO, Ph II
- CNF 1010 (from Conforma, acq May 3/06), ph I init
2004, geldanamycin derivative, HSP 90 inhib
- CNF 2024, oral HSP 90 inhib, ph I init 2006 (from
Cabrellis)
- Lumiliximab (Idec 152) : anti CD 23, CLL ph I/II
ASH 06, also anti IgE
- FIX Fc : F IX fused to FcRn (neonatal) to protect
against degradation, precl., from Syntonix
- Galiximab, anti CD 80, w/Rituxan, ph III, N = 700
- Lixivaptan (lic from Cardiokine, who got from WYE)
ADH receptor oal antag, ph III to init 2H07
BIME BIOMUNE SYSTEMS (BIME) neutraceuticals
BIO BioInvent AB (Sweden) BI-204, Mab anti-oxidized LDL, lic to DNA Jan 16/07,
pre, DEAL, for coronary, $ 175m. In association w/statins
prevents plaque build-up in animals
- BI 402 Mab anti factor VIII, ph I
BIORY BIORA AB (BIORY) Emdogain (periodontal surgery), MKT
BIOS BioMedicines ( BIOS)
IPO filed nov 3/00 -UBSW.
Trading April 3
BLSI BOSTON LIFE SCIENCES (BLSI) - Cethrin: r Protein spinal section, lic from BioAxone Jan
4/07, Ph I/II
-Therafectin : failed
-Altropane (imaging for Park), ph III
-Troponin→K, antiangiogenesis, ph I 3Q01
- 30 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-Inosine for optic nerve regeneration, Res (June 14)
BLT BENITEC (ASX : BLT) dsDNA
- Jan 06 : HIV IND, engineered stem cells w/lent delayed,
init City of Hope June 07
- April 18/06 : halve work force
BLUD IMMUCOR (BLUD) Blood Typing disappointing 1H sales→reorg.

Settlement w/Becton Dickinson


BMR K Bradmer Pharmaceuticals (TSX) Neuradiab (developed at DUKE): anti Tenascin (expressed Senetek has a siRNA to Tenascin for GBM
Glioblastoma median OS by 99% GBMs, not in b rain) Mab + *I, ph III to init
- surgery + RT 53w glioblastoma, N = 760 (enrol 24mm). Interim 2010
- Surg + RT + temo : 64w
- Ph II + Neuradiab 86.7w
- Recurrent 16-24w
BMRN BIOMARIN (BMRN) -iduronidase (Aldurazyme, in CHO) for Hurler(MPS I, 50/50JV Genzyme; manuf. issues seem solved, s 2nd
June 03 : $ 125 m convertibles bone, brain, liver,spleen, heart), ph III ph III enrol is completed march 4/01w 45 pts to end
30.6% owned by Glyko Biomedical Ltd BLA rolling filing init 04/02 (completed July 29/02, Aug 01(1st trial + with 10 pts).
(Toronto). Feb 7/02 : acqires Glyko accepted for filing Sept 16/02), CPMP filed 03/02. . Clinical endpoints : exercise tolerance, lung function.
GMP plant Estimate "3400 "available" patients(in rare cases early 2 year data May 15-19 (Int Congr Hum Gen):liver
April 21/04 acq Ascent Pediatrics (sells BMT has allowed retention of intelligence) size ¬24%,spleen¬82%,urine carbohydrates¬72%.
Orapred for children) , will sell Aryplase & According to Pr J.Muenzer (U.N.Carolina) enzyme will Phase III on HS pts (clinical endpoints only : 6 min
Phenoptin not cross BBB, but "holds a lot of promises for the future : walk, FEV1). Data unblinded Nov 1st 01. Press
New CEO since Nov 00: Fred Price, who a lot of chidren could have improved quality of life"He release Nov 2: 6 min p = 0.066). June 02/03 shoulder
was at AMBI also says the purity is OK, and that enzyme could be range of motion NS
Aug 14/04 Fred Price resigns associated to BMT. Dg is easy by dosage of enzyme, and a Jan 15/03 Adv Com OK (but 1 tracheostomy for
May 12/05 JJ Bienaimé CEO lot of heterogeneity exists. He knows of 500 severe cases allergy). Jan 28 CRL w/PMS (allergy monitoring),
Role of Orbimed ++ in the US. manuf inspection; launched
TKTX has a license tu US patent on the -r Hu arylsulfatase B / Aryplase/ Naglazyme , Galsulfase, 4 h infusion qW Feb 21/03 : CPMP OK
for MPS VI (Maroteaux Lamy sd., 1/200 000 births, 1,100 pts in
enzyme, but was rejected by EPO developed world), ph I/II (6pts, 24 weeks). Completed July 25/01. Sept
Jan 7/03 files for secondary $ 63.3 (UBS, 6/01 : positive results. Ph III data June 3/04, N = 39, DB, 12 min walk p =
CIBC, SGC) 0.025, age 5-29, filed Nov 29/04 (endpoint 12 min walking distance) 48
w data March 05 improvement ditto PDUFA MAY 31. Approved June
Oct 10/01 : acq IBEX and its Heparinase (already failed 1/05, post mkt trial to init in less 1 year olds w/skeletal dysplasia. CHMP
a phase III). Dev terminated Sept 22/03 OK Sept 15/05, EU OK Jan 31/06
- for MPS 4 (Morquio A) ?
PKU mkt 50,000 pts ww, 40% can respond to - PKU :
Phenoptin, i.e. 6000 pts in US; price $ 20000/Y
- Phenoptin KUVAN (tetrahydrobiopterin, sapropterin,
Lic to Serono for ex USMay 16/05; $ 257m
MPS VI mkt 1,100 pts ww, 250 identified oral) Nov 20/03 manuf agreement w/Merck KGaA.. Feb
23/04 ph I (good responders phe Ala down > 30%); lic to
Lic to Daichi for Japan Nov 04
Daïchi for Japan Nov 04. Ph II init Dec 26/04. Ph III init
April 7/05, N = 100 (pts who reduced PHE levels in Ph II),

- 31 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


filing 1H06. Data March 16/06 p < 0.0001. Open extension
confirms (Dec 19/06). Filed May 24/07
- Phenylase subcut PAL (phenyl alanine ammonia lyase),
pre, will pegylate Lic to Serono May 16/05
- 6R-BH4 (active ingredient of Phenoptin). Fails in HBP,
Feb 20/07
BMTI BIOMIMETIC PDGF/ Ca Phosphate device mkt for orthodontics, trials in Keep in mind BMP with Medtronic success &
orthopedics. Biogen/Strtker failure.
Dec 13/06 US 20 ankle fusions (14 PDGF, 6 autografto)
results similar
Jan 25/06 (Sweden) wrist fractures , N = 19, bone healing
at 6 w 100 of treated, 56 % others, but all similar at 24
weeks
Pivotals US/EU to begin
BNC BIONICHE (TSX : BNC.CN) - Cystistat (hyaluronic acid) for cystitis, lavage, MKT
- MCC (Mycobacterial cell wall complex, Mycobacterium
phlei), bladder K, ph III
BNPI IPO BIONUMERIK (BNPI) -BNP 7787 Tavocept, ph II, for neurotox of platinum
K (w/Asta), ph III in Japan
IPO filed march 6/01 (MSDW), then June - BNP 1350 , camptothecin like, ph II
9/04 UBS
BPUR BIOPURE (BPUR) Hemopure ,ph III; bovine blood for humans, BLA to be Approved South Africa April 01
filed 2H01. In dogs HbO2 dissociation problem, May 01 : Chief medical officer quits : renal safety
Equity line SG (June 01) vasoconstriction.. Aug 28/01 : transfusion sparing effect ??
Class action suits (signif, March 20/02, anesthesia sympo).
BLA filed Aug 2/02. response July 03 requesting more
data
BREL BIORELIANCE (BREL) Preclinical manuf & testing.
- To test Acambis smallpox vaccine (& fill & finish)
Was Microbiological Associates
BSLN BASILEA (SWX) - Ceftobiprole, w/JNJ, to file 2007. Clear cut data (no
mixture of MRSA and not, or of ABS), on non inferiority,
clin cure rate MRSA 91% vs 86%, 2nd ph III SSTI ok non
infer. Filing May 17/ 07, fast track, approval YE 07 ?. EU
filing June 18/07
IDSA Oct 9/07 Diabetic foot infections 86.2% success vs
Vanco + Ceftazidime 81.8%
- Isavuconazole : to start ph III
- Alitretinoin : hand dermatitis, filing 07
BSMD K BIOSPHERES (BSMD) Embolotherapy (MKT) for tumors (meningioma, uterine
myoma), vascular malformations.
- 32 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


BSTE M BioSite (BSTE) - IVDg : troponin, natriuretic peptide, CK
June 00 w/Medarex on TransPhage technol (TPT)
Leader in POC mkt(using phage display) Jan 01 LLY/MDX/BSTE alliance
March 27/07 : bid by Beckman $ 1.55 B w/LSBC to generate targets for proteomic biochips
March 02 : TPT w/AMGN
Also alliances : MOR, Diadexus, Corixa
BTA BIOTA (ASX : BTA) - Relenza GSK
- BTA 188 : antirhinovirus
May 01 : acq Numax (US)
BTH M BIOTIE (Finland : BTH1V) - Nalmefene, alcoholism, ph III
- VAP-1 Mab (vapaliximab), ch ph I failed, Hz & Hu in
R/D
- recombinant oral heparin, Sanofi terminated
BTRN BIOTRANSPLANT (BTRN) Chimeric xenotransplant systems (also for pancreas) NVS decreases support to create JV (Immerge
M BioTherapeutics, 67%NVS, 23%BTRN) who will
to acq Eligix -Patent on PERV (porcine endogenous retrovirus ) free own PERV patent & XenoMune
swine
C -XenoMune system based on anti CD2 (MEDI 507 &
BTI322), to allow xenotransplantation
-licensor of Medi 507→GVHD to MEDI
BTX BIOTIME (BTX) Hextend (starch plasma expander), MKT ABT

BVEX K BioVex*, IPO filed June 20/06 Oncovex, oncolytic virus (which also stimulates K cells to
secrete GM-CSF), ph II melanoma (N = 50), ph II H & N
N = 16 + RT + CT
CALP CALIPER TECHNOL (CALP) The Lab on a Chip company IPO Dec 99
oct 31 : wins lawsuit against Aclara
CALY CALYPTE BIOMEDICAL (CALY) urine HIV,gono & chlamydia tests 8/24 : approv. In China
CARN CARRINGTON (CARN) Nutrition & skin care
CBMX COMBIMATRIX (CBMX) Semi-conductor based array processor
Acadia has majority ownership
IPO filed (SSB) nov 22

CBST CUBIST (CBST) -Daptomycin/Cidecin/Cubicin (lipopeptide w ristocetin Aug 24/04 : FDA warning on misleading ad "active
AB nucleus acq from LLY, manuf by Dobfar/Italy;new facility on MRSA"
Dec 4/01 MRK & PFE decline partnership June 18/01): ph III (muscle tox serious at 25mg/kg), IV
very active on VAN resistant strains. Lic to Gilead for EU
CAP 1 : ceftriaxone 87%/Cidexine 79%, (jan 01) Competition : oritavancin (LLY) ph III, licensed out
better data in US, poor response in Russia July 27/00 FDA requests additional Ph III,completed July (high protein binding) to ITMN., and see THRX
500 mg vial $ 134 16/01

- 33 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


phIII efficacy (4mg/kg)on skin & soft tissue infect (march
Sept 10/02 GILD discont partnership EU 14/01), and April 01: equivalent to Vanco(16% of 2nd ph III (US) at IDSA (Oct 28/01) : SSTI, equal to
comparators) or Oxacillin(84% of comparators). Vanco / oxacilline. Patients had free access to
July 9/02 : first US case of VR staph, 2 CAP(DB/Ceftriaxone, enrol. compl.Sept 6/01, Jan 17/02 Aztreonam
treated with Bactrim !!!!. But CBST failure (efficacy 83%/85%), 2nd trial suspended. Was to file mid 02 for SSTI/CAP/VRE
makes noise. UTI ph II initated 3Q/00 : not < cipro March 7/02 : FDA says could file NDA for SSTI.
Manuf site in Italy approved by FDA : pre- For "suspected VRE", DB/Zyvox;6mg/kg, US army, data Filing Dec 23/02(pack also has UTI & some data
approval inspection completed June 19/03 2H03 ?????? from VRE bacteremia). Approval was to be
Init endocarditis (animals need 25-40 mg/kg)trial Sept simultaneous to release of VRE & endocarditis data,
Oct 3/03 : Cubicin lic to CHIR (Novartis) 5/ 02 : pts w/endocarditis & Staph bacteremia alone; or 2004. Priority review status PDUFA June 20/03.
for ex US, EU filing Dec 2/04, approved Jan open label, comp 6mg/kg to Nafcillin or Vanco. Enrol Adv Com ( CBST says FDA said no need). June 19/03
06. will end when 200 endocard. pts are enroled. Several : delayed 3 m to Sept 30/03
Sept 6/07 approved EU right side interims ? Aug 4/04 Safety Board recommends Approved Sept 12/03 "for the tmt of SSTI by
endocarditis staph strep continuation. Nov 9/04 enrol compl. N > 230 of which susceptible G+ at 4 mg/Kg/d (7-14d)". Sales 03 $ 2m,
37% MRSA, bacteriemias (51%) & right endocarditis Jan + Feb 3.3m. 1Q04 : 6.3, 2Q 13.3, 3Q 18, 4Q 21,
(23%) . Data June 28/ 05 : non inferiority to 1Q05 , FY 05 114, 2Q06 45.7 , FY 06 190, 2Q07
comparators, muscular side effects. sNDA filed for 69.5, 3Q 76.3
bacteremia w/endocarditis staph Sept 26/05 <PDUFA → IDSA (Sept 30-Oct 3/04) : positive abstracts in
March 24/06. March 27 "approvable" (label ?). May bacteremia/endocarditis & osteomyelitis (but very
29/06 FDA OK for Staph bloodstream infections & "observational") ICAAC Oct 30/04 2 cases of
right sided endocarditis resistance presented by independent clinicians
Feb 22/05 : all enrolled have completed tmt.
data mid 05
-Oral formulation init w/Emisphere (nov 6/00)
- CAB 175 IV cephalosporin, ph I terminated Feb 04 (tox)

- Oral ceftriaxone (Rocephin like) lic in Nov 27 from U.


of Utah (in GRAS carrier), ph I was to start 4Q01. Could
start 1H04 ! (go/no go bioavailability : halted April 29/04 PFE terminated in 97
BMYterminated
-Bacterial tRNA synthase inhib MRKongoing
-Bacterial tRNA synthase inhib
-Bacterial tRNA synthase inhib
June 27/01 partn w/Syrrx on bacterial targets
- 2 molecules lic (res) to MRK
CEGE M CELL GENESYS (CEGE) GVAX allo (irradiated cells transfected w/Gcsf : PSA rate of rise goes down (→ASCO 02: dose
- GVAX allo (2 K cell lines) HRPC ph II, N = 34, median dependent trend to survival).1st Ph III init 3Q04. 1
March 04 : $ 53m follow on survival 26.2 m. (taxotere = 18.9m); 2nd ph II (ASCO trial comp to Taxotere /survival, 2nd trial init July 05
V (acq Somatix in 97) prostate Feb 06) median OS > 29.1m, N = 22. April 3/07 + taxotere /Taxotere alone on OS. 1st trial to compl.
OS is now 35m enrol. 3Q06.
- 34 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


K owns 10.5 % of ABGX Also ph III w/ipilimumab (Medarex/BMY) : PSA reduct >
50%
Ceregene : 60% owned spinoff (jan 11/00) - GVAX allo pancreas ph II. Data Nov 17/05 EORTC : N
for gene therapy = 60, IIb mainly, extensive resection, then vaccine. 2 year
survival 76% (historical 40 – 50%) → w/JT (term Oct 02)
Jan 02 : $ 60m asset backed debt financing - CML ph II, init Dec 012, ongoing w/auto BMT , data
has lic to TKTI & AVE the Gene activation ASH 05, Dec 12 (enhances GVTumor). 5/19 have
patents +++ complete disappearance of bcr-abl
-Entremed
GVAX auto : -Collateral Therap
- GVAX Adeno auto NSCLC ph I/II. N=26, 12%CR, 27%
PR; w/JT(Feb 18/04 response is proportional to dose of lic to Novartis
GM-CSF) lic to Novartis
- GVAX patients irradiated K cells + "bystander GVAX":
--NSCLC ph I/II N+40 init June 01 w/JT, ongoing
--myeloma ph I/II, ongoing
-Lentiviral GT→VIII, pre, Term Jan 03
-GT with Endostatin/Angiostatin, RES
-GT with VEGF&FGF, RES
-GT IX primates/AAV™no Abs

- CG 7870 (engineered Adeno), phI/II, IV & intraprostatic,


oncolytic, K. Also combi Taxane.
- CG 7860, intratumoral, pre
- CG 0070 recurrent bladder K, Ph I/II, w/Novartis
CELG CELGENE (CELG) -thalidomide (Thalomid, patent 2018) for myeloma, ph II -lic. from Entremed; & to Pharmion & Penn for EU
(delays annonced April 01); TT2 (Total Therapy 2 trial) -FDA warning letter for off-label
C July 3/00:merger w/Signal w/pharmacogenomics, N= 600, first 120 pts to be
K unblinded. Filing was to be end 01/1H02. → 2Y delay
Oct 9/01 : Drug Royalty Corp FDA wants "survival"for "refractory" & t. to progr. ASCO 03 : 19 presentations
acquires $ 4.9 m royalty interest for early stage MM, filed Dec 03, Oct 26/04
"approvable" but wait for data ECOG E1A 100 ; Nov
ECOG has a ph III fully accrued Thalomid + 15 05 : again "approvable". CPMP filed April 02.
Annual price per patient $ 10 000. Sales 01 : $ 82m, TTM RCC signif ORR w/IL2 ∏ ASCO 02, results 1Q03
IFN
dec 02 : 140, 2003 : $ 224m, 1Q04 69, 2Q 74.6, 2Q07
Nov 14/02 acq Anthrogenesis (umbilical 117.7
cord blood banking) $ 45m MKT in Leprosy, sNDA filed for MM end 03. Appovale
letter 3Q04? could reply 1H05
May 27/03 rumor that AMGN could acq. renal K (NO PR, ASCO 01), ph II w/IL2. Ph III to start
colorectal w/Xeloda-Roche & irinothecan-PHA,
melanoma, glioma, June 30/04 HRPC + taxotere : median
- 35 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


MDS : 300,000 ww survival 28.9 m, ph open, N = 75.; ASH 04 ph III
10 to 20,000 new cases/year US, 20 to 30% myeloma + dexa : > response rate. 009 (US) : TTP 60.1w/ 20.7 p < 0.0015, ORR
are 5Q deleted - Revimid/Revlimid / lenalidomide, CC-5013 (thalidomid 51.3%/22.9% p < 0.001, CR 19.1%/3.8%; OS p<
analog, with less ?? neuropathy), ph III myeloma w/Dexa; 0.0001
MM ww 74000 new/y; prevalence ww endpoint TTP, 2nd 3rd & 4th line N = 705 (354 US, 009 + 010 (EU) TTP 53.4/20.6, ORR 47.6%/18.4% < 0.001,
200000; deaths 60000/y 351ex US 010), March 7/05 TTP (BLADE) p < 0.0015, CR 9.1%/ 1.2%; OS p< 0.03
melanoma & CHF (anti-TNF) danger++. April 28/04 "Rick Pazdur wants it" To file 4Q 05 ?
Price Revlimid : $ 4,500/month, retail melanoma halted by Data monitoring com. Adv Com Sept 14 OK for approval in transfusion
6,400 Filed April 8/05 in 5q MDS (ph II). N E J MED Feb dependent 5q deleted (10/5). But 13/2 "the data does
Competition : 9/05 ph I/II of 20/43 pts EPO refractory achieved not provide a basis for a recommended dose". On Oct
- Pharmion Vidaza azacitidine(approved transfusion independence & complete cytogenetic 3 PDUFA delayed from Oct 7 to Jan 7/05
04) response. Rand ph III ongoing in EU Approved on ph II N = 148 Dec 28/05, 1Q06 $ 32m,
- MGI/Supergen Dacogen (decitabine), placebo/5mg/10mg. Aug 05 CHMP filing for MDS 3Q 101, 2Q07 108.9
approvable MM filed Jan 06, PDUFA June 30/06 :OK Myeloma
July 3/06, -
EU OK MM 2nd line March 24/07, Commission OK
June 19
Actimid : Jan 8/03 ph II init Refractory prostate
-D-Methylphenidate(Focalin), NDA filed dec 27/00,
approvable Aug 22/01(FDA OK Nov 15/01); Ritalin LA
approvable Oct 1/01
-Serms from Signal, w/Novartis : 8490 to enter ph II 2H02,
K, another for osteoporosis
- SPC/CDC 839 w/Serono, oral anti TNF (IKK, inducible
κ kinase for NFκB) inhibitor, pre
- CC 401, JNK (c jun N terminal kinase) inhibitor, ph I
(oct 02) for inflammation.
- CC 10004, PSO, oral, ph II
- CD 1088, ph I/II, Crohn
- CC 8490, ph I/II, glioma
- Jan 21/03, w/Cytogen on Ubiquitin ligases (anti kinase)
- Placenta derived stem cells
CEPH CEPHALON (CEPH) -Provigil (sch IV/DEA)→Narcolepsy : $ 25/99;72/00m Jan 15/02 : FDA letter on "false or misleading
June 03 : $ 600m convertibles ;150/01,196/02; 290/03 (+ 40%) FY 06 727. Generics : promotion".
K 2003 → 2006 ? (orphan until 06, but sNDA filed Dec
23/02 for "daytime sleepiness"panel split Sept
Dec 3/01 : acq Lafon ($450m). This will 25/03PDUFA Oct/20 03; OK Oct 22/03; sNDAs filed by
not increase Provigil sales in France! Teva, Mylan, Ranbaxy, Barr)
trial fails in ADHD july 31/01→ stock ¬ 40%, Aug 11/06
Feb 5/02 : accounting practices FDA rejects ADHD

- 36 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


questioned (by Sterling Financials): - Nuvigil (armodafinil) filed April 4/05
SASY & Lafon deals - Gabitril (acq USfromABT, & EU from SASY) MKT,
Feb 12/03 : $ 161m takeover bid for SIRTex 1Q03 12.7m (+ 25%) 2Q 14.6. FY 06 58; Feb 25/05 black
( 13.5% premium) :Y* spheres ( 2003 sales $ box seizures lic from Kyowa Hakko, dev w/Lundbeck
10 to 12 m , very undeveloped, only used in -Actiq (oral transmucosal fentanyl),MKT($15m/00,51/01,
3 UK hospitals for Europe) 2003 forecasts 214, 1Q03 46.2m ,+ 139%) 2Q 52.7 (+85%) lic from Kyowa Hakko, dev w/TAP & Schwarz
Aug 21/03 : takeover bid for CIMA, rejected, ; FY 06 572; acq Anesta July 00 .
Sept 2/03, counterbid by AAII, finally
completed Aug 04 - Vivitrex (alcohol dependence) from ALKS. Approvable
Dec 29/05
Jan 05 acq Trisenox from CTIC -CEP-1347→PARK, ph II;Alzheimer .SAP (stress
May 13/05 : to acq Salmedix for Treanda activated protein kinase=JNK=c-JUN N terminal kinase)
(bendamustine) for NHL, ph II :*; mkt in inhibitor. Protects neurons & neurofibrils. Term May 12/05
Germany -CEP-701(trk tyr kinase inhib)→Prostate K, pancreas, ph
II, precl also in pancreas w/Gemcitabine, CML ph II. But
Dec 6/05 acq Zeneus Ltd ($ 360m), mkt Xenova failed in this field, and anti EGFR (OSIP/Roche;
Myoset (liposomal doxo), Targretin, AZN, ABGX/IMNX) and Herceptin (DNA) are close. In
Abelcet) vitro corrects FLT 3 mutations in AML (Johns
Hopkins June 3/02)
- CEP 7055, ph I, K, antiangiogenesis (VEGF kinase inhib)
- Oravescent fentanyl ph III OK May 5/05
- Treanda (bendamustine) filed for CLL Sept 20/07, ph III
nHL : ORR 75%, 2nd line, Oct 24/07
CEPH CIMA LABS (CIMA) -coating for pills (e.g. Zomig/AZN)
- oral soluble technologies, fast acting :
acq by CEPH (Aug 04) Remerin (mirtazepine, antidepressant) solTab, MKT,
w/Organon
Nulev (Hyosciamine), MKT, w/Schwarz, Irr Bowel sd
CEPO Ceptor (OTC BB: CEPO) Myodur, for Duchenne, filing IND (YE 05). Duchenne has
up-regulation of Calpain. Lepdur associates a leupeptin
analogue (calpain inhib) and a carnitine carrier molecule
CERS CERUS (CERS) -products for antisepsy of blood transfusion ("Intercept") BAXTER (owns 16.5%)
∗for platelets (amotosalen, activated by UV, cross links
C nucleic acids of pathogens) EU approved, US under review
∗for blood ph II with S-303 ?, activated by pH when RBC
are introduced into set.
-Stem cell transplants
- vaccines : listeria delivery platform. Ag : Eph A2 w/Kirin
(w/MEDI)

- 37 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


CGEN COMPUGEN (CGEN) Bioinformatics and algorithms +++
Israeli company -Software for EST databases (LEADS) with error and ⇑PFE(WLA), HGSI, ABT
alternate splicing detection
-Software (Z3) for 2D proteomic gels
-LabOnWeb.com Bayer, MediGene, Genox, GENE, AMGN, AVE
-discovery with LEADS of CXCL 16, novel HIV
receptor on LTs, Oct 5
- leading the Consortium on MAPK
CGPI COLLAGENEX (CGPI) -doxycyclin for dentists, MKT -coprom. Vioxx to dentists
CGRB. K COUGAR ( merged April 8/06 with public - CB 1089, Seocalcitol, calcitriol analogue, prostate K, ph
OB shell) II
- CB 7630 (17 α OHlase 17-20 lyase inhibitor), prostate K,
ph I
- CB 3304, Noscapine, microtubule inhib, ph I
CGXP CERAGENIX (CGXP) Tmt for cracked skin
CLGY CELLEGY (CLGY) -consumer skin care :anal fissure ointment (Anagesic, NDA withdrawn April 29/02 : headaches in 50% pats.
nitroglycerin ointment) Dec 23/04, FDA : NO
-transdermal delivery : testosterone phIII to start
CNCT CONNETICS (CNCT) -r hu Relaxin→scleroderma, ph III ⇑failure oct 9/00 BLA filing 2001 ???;part w/Flamel (jan/01)
- Luxiq (bethametasone foam) since 99
acquiring Soltec (Australia), drug delivery - Minoxidil foam (like Rogaine), in clin.
company - Velac gel (clindamycin + tretinoin) lic from Yamanouchi
(May 16/02)
Becoming a specialty dermatology company - Soriatane (retinoic acid) for PSO lic from Roche Feb
? 23/04.

COLY V COLEY ( COLY) CpG company Competition on TLR : Vaxinnate


- w/GSK : Engerix + CpG ph I/II : much higher & faster ∗part of $ 72 m deal , upfront $ 8m, for Inf dis
K Acq by PFE Nov 16/07 $ 164m response
I.P ??? - w/GSK Nov 7/02 : K vaccines with GSK genes (MAGE
-research on human cells in Dusseldorf III) , also $ 72m; Milestone Oct 25/07
- w/AVE : CpG 7279 antiallergy : enhancing Th1 response
Private placement $ 35m, Aug 25/03 to allergens rebalances the immune response away from Jan 21/03 : "on track to enter clinicals"
IgE
- potentiate Rituxan : increses expression of CD20
IPO filed (COLY) April 20/05 (ML, JP receptors on B cells
Morgan), traded Aug 10/05 $ 106 m - CpGs alone up-regulate cell surface Ags & increase CTL
proliferation.
- CpG 7909 S. Cut. Promune (to TLR9), ph I April 29/02,
Unterberg : positive echoes at ASCO 07 RCC (N= 3-6) then 24 w ph II on 12 pts. Melanoma stage 7909 partnered w/ PFE March 24/05, total $ 455,
IV ph I/II monotherapy init June 13/02. Ph II NSCLC N = JUNE 20/07 PFE terminates. Oct 30 PFE
- 38 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


75, "to improve the outcome of 1st line taxane/Pt; enrol repartners for 2nd generation; ph II 7909 ongoing
compl May 13/04, interim data ASCO 04.. Feb 3/05 1st line w/Tarceva refractory NSCLC, & ticilimumab (anti
improves RECIST more than CT alone ORR 37%/19%. CTLA4, tramelizumab, IgG2) melanoma
Active in adeno & squamous cell. Nov 28/05: 2 ph III
init stage 1st line IIIb/IV w/current standard of care
CT(taxol carbo or gemzar cisplat) vs Standard of care
alone; N = 800 x2, sites opened by PFE; Enrol compl
May 5/07, Data YE 2007; filing 1Q08. Endpoint OS
+20% (standard tmt OS 8m). June 20/07 PFE
terminates
Ph II w Avastin/ <carbo taxol 1st line NSCLC, ph II 2nd
line w Alimta/Tarceva
Ph II melanoma w/ anti CTLA4, init 1Q07
Also CTCL
- Actilon CpG 10101 ph I CTCL (40 pts, 40 nls), Hep C ph
II term. Jan 23/07
- CpG 10103 immunostimulant for inf dis & vaccines :
VaxImmune lic to Chiron Dec 26/03, lic to MRK April
12/07
- Inhaled TLR9 agonist against BioTerrorism (NIAID
grant $ 5.8m Oct 22/03)
COR CORTEX (COR) Ampakines hypothalamic AMPA agonists :
- CX 516⇑mild cognitive impairment ph IIb init April Servier (oct 16/01).N= 160, for 12m
3/02. Feb 17/04 NS
- CX 516 for ADHD w/Shire, ph II
- CX 717, ph I, Alzheimer init 2Q04 (1mg/Kg), on hold
April 3/06 animal data
competition LLY 451 395 ph II
- ORG 24448 w/ Organon schizophrenia/autis:/depression
CORT CORCEPT (CORT) C-1073, antidepressant, ph III (it is RU 486 !!!!!!!!!!)
IPO filed Dec 27/01 (CIBC) fails Aug 25/06
CPHD CEPHEID (CPHD) - Biological warfare agents detection : will start selling
suitcase sized dg machine in 03, already available to
military.
-Microfluidic genotyping
-PCR kit for CEA in lymph node biopsy (AATS,May 01)
CPRX IPO CATALYST CPP 109 based on Vigabatrin (Sanofi) gamma vinyl gaba
inhibitor of GABA transaminase, eliminates feeling of
traded Nov 9/06 pleasure associated w DA enhancing drugs. 2 trials in
Mexico. DB to init in US. But retina ??
CRA K CELERA (CRA) "to become the definitive source of genomic and related PEB
- 39 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


medical & agricultural information" -AAAS, Feb 18/00 : drosophila genome, -HUMAN
June 13/01 : acq AXYS (Arris + Sequana) GENE MAP :Science feb 16/01
DRUG DEV :
- CRA 024781, cancer, pre, histone deacetylase inhib
- Cathepsin S, w/Aventis
- Cathepsin K, odanacatib w/MRK (MRK 0822) SEpt
18/07 ASBMR, 12 m ph II data excellent, ph III init Oct
1/07, 1 a week
- Factor VII a inhib
- Tryptase asthma program.
CRGN M CURAGEN (CRGN) Genomics database (GeneScape portal) GLX
DNA
K DuPont/Pioneer
issued convertibles on Jan 20/00; James Vincent resigned BGEN
from board on Jan 19 CORR
Roche
Abgenix(MAbs to 24 CRGN targets)
Nov 7/02 staff ¬ 25% - CG 53135, FGF 20 Velafermin, for mucositis & IBD, ph Bayer +++
June 19/03 : staff ↘ 20% I, active on epithelial AND mesenchymal layers ( #
AMGN, HGSI). Dec 13/05 ph II mucositis fails; énd ph II Mitsubichi (Nov 5/02) schizophrenia targets
fails Oct 11/07
- PXD101belinostat lic from TopoTarget A/S, June 4/04,
HDAC inhib, ph II Myeloma
- CR 011 fully Hu Mab conjugate, antiglycoprot NBB,
melanoma, ph I/II
- CR 014 anti TIM1, ovary, RCC
- CR 002, anti PDGF, lupus nephritis
CRIS M CURIS (CRIS) -BMP-OP1,phIII, w/Stryker & Biogen (accepted for filing merger of Ontogeny , Reprogenesis and Creative
by FDA in July 99):Feb 1 CPMP/OK(after autograft Biomolecules (July 31/00)
CEO : changed Sept 01, Doros Platika failure),FDA negative jan 30/01(not >autograft for Dec 2/02 : BMP 7 active IV on stroke rats after 24h,
K becomes chairman pseudarthrosis)! Approved Oct/01 "when autograft not Feb 03, lic to JNJ (upfront 3.5, milestones >35)
feasible". The AHP /GI BMP-2ACS rejected by FDA for long
BMP competition is GI(AHP)/Sofamor -single chain Mabs patents (Creative Biomolecules) bone fracture but Adv panel OK spinal fusion (Jan
Danek merged w/Medtronic; for spinal - autologous cartilage (Chondrogel) for vesicoureteral 10/02
fusion. reflux and stress incontinence, ph III term Feb 15/02
Feb 15/02 : work force cut. - PPY peptide (diabetes, obesity) lic to Amylin Dec 7/02
($ 15 m deal)
-BGEN returned hedgehog prot to Ontogeny July
10/00∏July18/01 JVw/Elan for drugs that stimulate June 11/03 lic Hedgehog, with CUR-61414 (basal cell
Hedgehog pathway.Returned K) to DNA. $ 240m deal, upfront 8.5 (Cash 5, equity
- Topical antag of Hedgehog pathway w/DNA, ph I/II 3.5, & 2m/year/2 years, then milestones. Jan 25/06
partial failure. Also w/ WYE
e . July 12/06. Systemic IND filed Oct 5/06 for ph I

- 40 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


CRL Charles River Labs (CRL) Leading provider of animal research models since 50 years

CRME CARDIOME (CRME) RSD 1235 ion channel inhib, atrial fibrillation, IV ph III
OK, oral ph II
CRTQ CORTECH (CRTQ) -Bradycor : failed in TBI All activity halted
-elastase inhibitors : halted
Seeking acquirer
CRTX CRITICAL THERAPEUTICS (CRTX) - ZYFLO (zileuton), 5 lipox inhibitor, acq from ABT (not detailed since AAAAI Mar(ch 18 – 22/05
M (analysts Matt Geller, Pi 97) ATS May 20 – 25/05
CEO from SEPR, GSK, ABT (made zileuton) New manu (sNDA filed April 2/05) could launch end 05 ( in Singulair
COO from SEPR failures). Open label IIIb init March 15/05 in pts who had benefited from Competition : LT receptor antagonists : Singulair once a day; MRK
CMO from GSK Zyflo. Also orphan drug trial in Mastocytosis. manufacturer found June $ 2.6 B 2004 (rare Churg & Strauss), Accolate AZN $ 0.1 B
Res from MLNM 28/05. FDA OK Sept 29/05
4 times a day, can increase ALAT
- Retard being prepared
- Preclin : inflammation
- CTI 101 Halted (March 15/06)
- Fully human Mab anti-HMGB1 for inflammation associated tissue
damage, pre (w/MEDI)
CRXL CRUCELL - PER.C6 : human cell line, licensed to most biotech &
pharma companies. The Mabs JNJ was supposed to
revenues 2005 37.5 m € produce since 2001 seem to have vanished.
2006 : 140 m € - STAR : a technology to enhance yield of CHO X 4, being
1Q07 : 32 m € tested by DNA & MEDX, but the produced proteins would
probably need novel clinical trials….
- Berna vaccines :
- On the mkt : hep B, Hep A, influenza, typhoid
_ Being developed : the usual
- Recombinant proteins being developed : Factor V Leiden
for thrombophilia : Bof
- Plasma extracted : alpha 1 proteinase inhib lic from
Bayer, but being developed by others as recombinant & as
AAV.
CRXX IPO CombinatoRx (CRXX) filed Jan 19/05 Synergistic applications of approved drugs (NTMD model)
Pacific Growth Steroid "amplifiers" :
CRx 150 amoxapine + dipyridamole, failed June 06
Traded Nov 9/05 periodontitis
CRx 139 : prednisolone ( 2.7 mg) + SSRI (Paxil GSK), ph
II RA to be presented in Spring 07 (w MTX vs MTX alone
: it will sow that Paxil alleviates symptoms !!!
CRx 102 prednisolone + dipyridamole, to init in knee OA
CRx 119 prednisolone + amoxapine; for periodontitis.
April 10/06 misses endpoint (CRP)
- 41 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


CRx 170 budesonide/nortriptyline, April 18 positive data
lung function

Cancer :
- CRX 026 Chlorpromazine/Pentamidine, ph I ASCO 2005
harmless
CTEC CHOLESTECH (CTEC) -electronic analyzer from 1 drop of blood
- P.O.C. diagnostic screening

CTI Chemokine Therapeutics (TSE/CTI) - CTCE 9908 ph II, antag of SDF1 (stromal cell derived
CHKT OTCBB factor 1) that binds competitively to receptors (CXCR4 )
on K cells, inducing metastasis
IPO Canaccord - CTCE 0214, regained April 18/06 from PPDI, peptide
agonist of CXCR4, increases WBC, ph Ib
CTIC K CELL THERAPEUTICS (CTIC) -Lysofilline
-Trisenox (As trioxyde, NDA approv. 09/00 & EU 2002 →3 deaths published march w/QT↗ (march 9/01and
Chairman : Max Link for PML 2nd line, sales 01 : $ 6m!!!, 02 : $ 12m , 03 : $ 24, posted on FDA web site). EU approval March 12/02
04 : $ 26.4 m ; trials in myeloma. ASCO 03
Has subsidiary (Pangenex) specialized in June 10/05 sold to CEPH $ 170m → lic to Chugai (Asia) ASCO 02 ++
knockout. - Xyotax PG (r polyglutamate)-TXL (paclitaxel → ph II AACR, Nov 02
Jan 5/04 acq NovusPharma stock derivative) phIII ovary since July 02, Feb 03 data on → ASCO 03 : ph II alone in adv NSCLC 7% PR
exchange $ 236m, w/Pixantrone ph III for I/II ovary : bof,
ph III NSCLC init Oct 02 N = 840, 2nd line, data 2H05, → ASCO 04
NHL
rand, survival, comp to Taxol. Nov 20/03 dose reduced.
STELLAR II vs Taxotere 2nd line NSCLC data April 05 2nd line ASCO 06
Feb 04 : Europe hiring PharmaMar sales
personnel Stellar III 1st line, N = 400, to reach 311 deaths (June 04
: 400), endpoint 30% survival improvement, data
June 10/05 reduces workforce by 130 March 7/ 05 only non inferiority, w/carbo, comp to
Taxol;
Stellar IV ditto alone 1st line vs Vino or Gemzar in poor Sept 10/06 Xyotax lic ww to Novartis
performance status, N = 477, enrol compl Mat 04, data
mid Feb 05 Delayed
Stellar III & IV better in women (Aug 05)
To file 2H06 → 1H07 (June 1/06)
competition Abraxane APPX American Pharmaceutical
partners : nanoparticles cremophor free paclitaxel (was
ABI 007) NDA filed March 8/04. Good Data at San
Antonio03
- Dec 6/05 init PIONEER 1. NSCLC, late stage, 1st line,
poor status, female only N = 600
- PG Interferon
- 42 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- CT-2106 : polyglutamate camptothecin, ph I to start in
CRC & SCLC
- Inhibitors of LPAAT β for K (AACR, Nov 02)
- Zevalin, acq Aug 15 fromBIIB
- Brostacillin (acq w/SMI July 25 07, SMI founded by Dan
Von Hoff) minor groove binder. Targets pts w/mismatch
repair deficiencies, high glutathione, t (12:16). EORTC ph
II in 108 1st line sarcomas vs dexa. Could be enough for
filing
CTIC NOVUSPHARMA, Nuevo -BBR 2778,Pixantrone, anthracenedione (near …but cardiosafety evaluation is LVEF & not
Mercato,NOV.MI mitoxantrone), devoid of cardiotox (?), ph III w/Rituxan diastolic
traded since nov 6 compared to Rituxan alone NHL, init April 5/02, N =
President : Erich Platzer, who played a key 800, EU/US CR rates & TTP. Ph III to init N = 320, ASH 04
role in the discovery of Neupogen double refractory NHL, endpoint response, TTP, survival.
Established in 98 by a management spin-off Enrol will take 12 m. To be complete mid 05
from B.Mannheim/Roche Oncology - BBR 3464, triplatinum, not nephrotoxic, phII⇑NSCLC
(halted), ovary & gastric disappoint Oct 01
- BBR 3438, ph I, mitoxantrone derivative™gastric K
- BBR 3576, ph I, mitoxantrone derivative.

-Oral antiangiogenic,pre, w/Prolifix (UK,private)


CTRX CoTHERIX (was Exhale) CTRX - Ventavis (iloprost) nasal, PHT, lic from Schering AG;
EMEA OK Sept 03, FDA OK Jan 3/05, Orphan drug.
IPO filed March 15/04 (CIBC) 2Q05 4.1m. New device ? 1Q06 : $ 14.5m
- CTX-100 inhaled hyaluronic acid, emphysema, ph I/II,
Secondary Feb 10/05 $ 35.6 not on web site anymore
to be acq by Actelion $ 420 m Nov 20/06, - Fasudil, lic from Asaki , rho kinase inhib for PHT
compl Jan 9/07
CURN CURON MEDICAL (CURN) electron tipped catheter to treat GERD
CVBT IPO CardioVascular Biotherapeutics (CVBT) FGF for intracardiac injection, ph I/II
filed Sept 27/04
traded March 15/05
CVM K CEL-SCI (CVM) Multikine (mixture of many natural cytokines, peritumoral
injectionp, ph II
CVTX CV THERAPEUTICS (CVTX) -Ranolazine Ranexa( lic. from Syntex/Roche;patents →AHA : nov 14/00, MARISA(n=200) improves
June 14/03 PIPE $ 100m convertibles + issued 95-99), antianginal (pFOX inhib switches heart treadmill , p<0.0001)
100m credit line Acqua Wellington metab from lip to gluc, acts on mitochondria);++ treadmill A.Coll. Card (March 18/01) confirms and extends to
aug 9/00 : $ 120m equity financing line from test, ph III,→NDA filed Dec 30/02 (using SR pts with also CHF (p<0.001).
Acqua Wellington formulation). Why so late ?? → bioequiv Marisa (old Association trial (CARISA) last tmt completed Aug 8
June 7/01 : $ 100m secondary Roche supplies) Carisa ( 2 diff contract manufacturers) 01, results 4Q01. (Nov 14 AHA , but not all data,
Dec 01 : $131m secondary
- 43 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


CAVEATS : compares 750 mg bid w/1000 mg bid & placebo).
- Profitability in 2003/4 depends on 510 Is Innovex (Quintiles) the right mkt partner ? Targeting N= 823, treadmill improvement 24 sec (p=0;012).
- MKT costs could be underestimated 8000 cardiologists, only 75 reps : July 10/03 reacquires QT↗(<10 sec but signif). Time to 1 mm ST
- CHF for Ranolazine could be a mistake rights denivellation p=0.10
- QT issue in CARISA, & many pts from Also CHF ph II, data was to be4Q01 : DANGER : α-β-
east Europe JAN 13-14/03 FDA workshop → ACC : March 30/03 : 1 presentation in diabetes
on QT. New guidelines 5-10 ms OK, 10-20 Positives : remarkable study of QT . Corrects electric → AHA Nov 17-19/02 Carisa subgroup analysis
: concerns (problem is SD, not mean) anomalies linked to quinidine or cisapride. Due to late Drug Max QT Change from
Na channel blockade effect. baseline
R. seems lic in France to P. Fabre Feb 03 more data : 3 pts on high dose had QT > 500 ( 2 Placebo 425 ms - 2.2 +/- 0.9
also on placebo). 24 h IV, N = 24 → some syncopes. 750 426 2.3 +/- 1.08
Oct 26/05 CHMP filing withdrawn 1000 428 5.6 +/- 0.9
( Ranexa is 3 to 9 ms)
FDA panel Sept 15-16/03, cancelled Aug 3/03 "more
time needed to prepare adequately for the panel".
Queries from FDA on Tox (see etomoxir), Stats,
-Adentri(adenosine A1r antag) natriuretic for CHF, ph II, metab.
halted,BGEN will continue with backup (pre) Aug 10/03 : class action suit. PDUFA Oct 30/03?
Could be "approvable letter " in Oct and/or Adv Com
in Dec, or PDUFA redelayed 90 days. Nov 2/03 App.
-CVT 510(ad.A1r antag), IVantiarrythmic, ph II→phIII, letter requests another trial (bothered by unclear dose
needs additional trials ranging, constant QT elongation, also rat testicular
-CVT 3146 (ad.A2r agon for cardiac perfusion tox. Adv Com Dec 9 does not vote. CVTX may be
imaging),phIIw /Fujisawa 20% royalties, "pharmacological able to complete enrol. in 2005.
stress agent" used to be for coronary imaging, ph III to SPA OK ( ERICA )June 3/04 on N = 500 refractory to
start 2003 amlodipine (1000 mg x 2 + amlodipine/6w; endpoint
-Tangier disease gene (lic from Incyte)…to find a pill to angina frequency per week 2.8 verum vs 3.2, but SAE
increase HDL 21% vs 6% , April 18/05 p = 0.028
( Aceon, perindopril Servier, ACE inhib, comarketed 2nd SPA : "Merlin" trial for acute coronary sd non ST
w/Solvay, PDUFA extended to Sept 10/05)
elevated (data end 06 → spring 07
July 27/05 amended NDA filed, PDUFA Jan 27/06
Approved Jan 30/06. Labeling restricted to failure of
other drugs, & must be combi (Ca-, β -).
Launch 03/06 sales 2Q : $ 1.2 m.
CXSP K CHEMGENEX (CXSP, CXS/ASX) - Ceflatonin (homoharringtonine), CML ph II/III, MDS ph Interim YE 07, filing 2008
in EU : Stragen (Fr) II, AML Ph I. June 12/07 active on T3151
Merck AG on the Board (Elmar Schnee) - Quinamed (amonafide) needs genotyping to avoid side
effects, K prostate ph II, ovary breast colon ph I
CYAN CYANOTECH (CYAN) -nutritional supplements from algae

- 44 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-GFP

CYCC XCYTE (XCYT) Autocell therapy to stimulate T cells ex-vivo : K, HIV ph II NHL & MM, ASCO 05 : NS, CLL terminated,
V will refocus on HIV (May 16/05)
IPO filed (SGC) dec 26/00, withdrawn Dec
C 4/01, then refilled. Trading
March 17/06 merging with Cyclacel.
K Completed end March 06
CYCC K CYCLACEL Ltd -Seliciclib, oral CYC202, CDK inhib, purine analogue,
inhib of cdk 2 cyclin E, cdk 7 cyclin H, cdk 9 cyclin T.
Active on mantle cell cell lines (chromosomal
rd
translocation t(11, 14') (q13,q 32), NSCLC, Ph II, 3
line, ph IIb vs BSC init June 06; also
nasopharyngeal K (EBV), ph I/II
- Sapacitabine (analog of gemcitabine) , CYC 682, DNA
polymerase inhib, K, ph I; ASCO 07
- CYC-116, oral aurora kinase A, B & VEGFR2 inhib,
K,IND filed Dec13/ 06. Ph I init June 30/07
CYGN CYGNUS (CYGN) Glucowatch, FDA adv com(dec 6/99) but labeling cond sold their drug delivery bus to JNJ , to concentrate on
Aug 7/01 FDA approval of large Glucowatch ™seeking partners
manufacturing site ⇑sales starting in UK; March 22/01 FDA approval.
CYPB CYPRESS (CYPB) -Platelet extracts MKT: back to phase II, blood from Dutch
Red Cross (heat inactivated)
CEO : Jay Krantzler (McKinsey 85-89) -Prosorba columns for RA : sold to Fresenius
- Milnacipran (NA reuptake inhib) from BioMerieux P
Fabre for fibromyalgia : fails Sept 29/05; Jan 5/04
partnered w/Forest lab, 2nd ph III init Jan 06. Topline +
May 23/07
- Mirtazapine (from Organon) with another agent for sleep
apnea ph II
CYPH CYTOCLONAL THERAP. (CYPH) -Technol. to produce Taxol from a fungus BMY

-Library of antisense to 8000 human genes ???

CYTC CYTYC (CYTC) Semi automated PAP test : ThinPrep PAP test.
Acquiring DIGE (Feb 20/02), Term July
CYTK K Cytokinetics (CYTK), IPO filed Jan 28/04 - SB 715 992 ispinesib, Inhibitor of KSB (Kinesin spindle w/GSK
(G. Sachs, Pac Growth) protein) ph II : ovary, NSCLC ph II refractory not good
traded April 29/04 Sept 28/05 GSK will not move to next stage (April 1/06);
ph II 1st line ongoing, breast 2/3rd line 3PR/30.; ovary; San Antonio 05 : 2nd line mBC, N = 32, 50% grade IV
- 45 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ASCO 06 CRC ph II NS. Oct 2/06 ph II H & N fails. Nov neutropenia, 1 PR
8/06 GSK amends ( not termination, but CYTK to assume
all dev costs). ASCO 07 RCC not good. More poor data
June 30/07
- SB 7432 921, ph I, NHL, MM
- GSK 923295 pre, selected by GSK Dec 13/05, CENP
(centromere associated protein E) inhibitor. Aug 15/07 ph I
initiated.
- CK 1827452, cardiac myosin activator, ph I. IV (to
become oral). Heart failure Soc Spt 18 – 21/05. Oral init
Aug 20/06. Jan 3/07 collab w/AMGN $ 750M; ph II init
April 12/07
CYTO M CYTOGEN (CYTO) -Quadramet(Samarium)→pain metastases, MKT Schering AG (Berlex) rights regained June 16/03 by
-Dg imaging *Indium MAb -Oncoscint colorectal: MKT CYTO
-Oncoscint ovary : MKT
-Proteomics through Axcell subsidiary, WW -Prostascint : MKT
interaction mapping; on tyrosine kinases &
serine threonine kinases JV w/PGNX : The PSMA Development Company.
Term April 25/06
CYTR CYTRx (CYTR) -Flocor (was RheothRx), ph III. Lic to SynthRx for sickle
cell disease
Jan 9/07 creation of RXi Pharmaceuticals -TranzFect technology lic to MRK, used for HIV vaccine,
owned 85% by CYTR successful in Rhesus (April 2/01)
- siRNA lic from U. Mass Med School (obesity, cancer,
diabetes, ALS)
- Arimoclomol (HSP 70 co-inducer), ALS, ph II init Sept ALS more than 20 drugs in trials
05. Could activate a molecular chaperone. Active on SOD
mutant mouse w/spinal & cortical lesions. IIb to be init
4Q07, will last 18m, 400mg TID, oral
CYTX CYTORI therapeutics CYTX, (was XMPA , Specialized in Stem Cells from auto adipose tissue : device Adipose tissue : see also Stem Cell Sciences plc
C Frankfurt) to purify them : CELUTION approved can isolate and
concentrate cells in 1 hour. Uses uncultured non
San Diego company phenotyped.. Efficacy ++ in pigs for heart attacks.
Ph Iheart chronic ischemia init Jan 07, data 2008; ph
JV with Olympus CorporationTokyo Nov I/II heart attack to init 2H07
4/05 Plans : spinal disc repair
DBLT DOUBLE TWIST (DBLT) Genomics portal
was Pangea

IPO filed 9/6 (LB),withdrawn march 10/01

- 46 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


DCGN DECODE GENETICS (DCGN) Population genomics
Icelandic company -late onset Alzheimer gene Roche
Roche has 14% - schizophrenia gene (oct 20) Roche
- PAD gene (nov 14)
- gene for stroke
March 02 : acq of MediChem -Nov 3/00 : initiates large scale STR (short tandem repeat)
(highthroughput) genotyping
- w/PHA pharmacogenomics of heart disease
Staff¬ 35% Sept 30/02 - pharmacogenomics of response to steroids in asthma :
10 000 genes explored, only 20 correlate with drug
response w/Medarex & GenMab
- DG 031, inhib of FLAP (5 lipox activating protein)
that leads to LTB4. Ph III init May 4/06 to reduce
incidence MI & stroke
DDD SCOLR Pharma Trials w/ Ondansetron, Raloxifene
Parnerships with :
oral retard formularions, and enhanced - Wyeth (Dec 05) for Ibuprofen, term. March 07
bioavailability - Perrigo (PRGO), world's largest manufacturer of store-
brand OTCs
- w/ BCRX for oral peramivir (March 9/07)
DDDP K 3 Dimensional Pharmaceuticals(DDDP) -Highthroughput + Combinatorials DuPont, B.Inge.,AVEn BMY
-Products :
MEDI owns 5.9% °Oral thrombin inhibitor(3DP4815), ph I→→→→ →lic to JNJ (Centocor) jan 2/01
°Inhibs for K (RES) :
Jan 16/03 : to merge w/JNJ -of ανβ3,MDM 2, MMP, VEGF

DDXS M DIADEXUS (DDXS) Use of genomic data for dg. and MAbs
formed in 97 by SBH (21%) & Incyte (20%)
IPO (LB) filed nov 21/00, pulled 12/12/02

DGEN DELTAGEN (DGEN) -Functional genomics MRK


-Gene target validation in mammalian cells PFE ROCHE SGP
- Advanced knockout systems GLX
DIGE DIGENE (DIGE) - The only FDA approved test for HPV Roche
Being acq by CYTC (Feb 20/02), Term July - > PAP smear alone for detection of cervical K (June
½ 19/01)
DIL DIADIC (DIL)
DMI DepoMed (DMI) Drug delivery to reduce gastric intolerance (GR = gastric JNJ
retention)
- Metformin GR ph III lic to Biovail (May 30/02)
- 47 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- Ciprofloxacin GR ph II

DNA K GENENTECH (DNA) -Rituxan (since 97)anti CD20, IV, weekly: $279 ASH01 : GELA(8 cycles) results mkt (beware of
"""Roche decreases stake 65→58.9% March m/99→$424/00,$ 819/01 (+ 84%).1Q02 235 (+40%) 2Q02 : 274 ECOG(4 cycles) N = 630 accrued at ASH 02(192
9/00""" (+46%), 3Q02 293.9 (+38%),4Q02 347, 2002 : $ 1. 162B;1Q03 341of abstracts), 2nd part of ECOG MAINTENANCE (4
M Rituxan per ptn/y : $ 15, 000. 700 000 which 31 ex US sales to Roche (+38%, price +5.8% March 7) 2Q : 363 infusions every 6 months). Data 2004. 193 abstracts
potential WW pts; 50 000 new cases/y US, 5 3Q 372( 18 ex US), 4Q 413, FY 03 1,489, 1Q04 401; 2Q 424.7, 3Q at ASH 02
year survival 50% 438, 4Q 448 ; 1Q05 441 (US), 2Q05 US 450, 3Q US
452;FY 05 US 1.8B (ww 2.4B);1Q06 US 477 2Q 536, 3Q
The largest mammalian fermentation 509 4Q 560, 1Q07 535, 2Q 582; 3Q 572; RA ph IIIinit 04
facility ww : ++ for future of Rituxan for "refractory to anti-TNF"
∗Will manuf Enbrel for IMNX 1Q04 -Activase : $236m→$205/00 (competition JNJ Retaplase
H is indicated 1st line w/Taxol or 2nd line $110m. TNKase (Metalyse/Tenecteplase)Launch US feb
alone in Breast st IV 01, EU march 01. All Lytics 01 $ 197 (- 4%) w/B
Ingelheim; 2002 : $ 180 m, 03 : 185, 1Q04 46; 1Q05 54, Alkermes
. Tanox : July 10/01 : Complete response
letter requiring Labeling only adult allergic 1Q06 59
asthma -HGH : $221 m→$228/00 (competition LLY, N.Nordisk)-
All HGH 01 : $ 250 m (+ 10%); 2002 : 297; 1Q05 92,
Sept 30/04, Nature : mutation explains 1Q06 87, 2Q 94
Herceptin good results in NSCLC Nutropin depot approved (Schwarz/Europe Term.06/01),
term 06/04. April 27 & ASCO 05 : interim of 2 ph III, 1st line,
ALTU 238 HGH lic from Altus, qw, SC, Dec 06 adjuvant, "early stage BC". Adria/cyclo/Taxol +/-
Xolair approved June 20/03, NVA &
DNA to copromote in US, sales force -Herceptin (trastuzumab): $ 188 m/99→$275/00, $346/01 Herceptin : 52% decrease in recurrence = trial
(wholesaler fill, + 25%), 2002 : $ 385 m; 1Q03 93.7,+8%, stopped. Filed Feb 16/06, FDA action mid-April ?
250, sales start July 10/03 2Q 109, 3Q 108, 4Q 114, 2003 : 427, 1Q04 113.5, 3Q 126, Aug 15
4Q 126;FY04 479 1Q05 129 (US)/14 row, 2QUS 152, 3Q extended to Nov 17 Nov 16/06 OK w/ doxo cyclo
215;4Q 250; FY 05 747; 1Q06 290 2Q 320, 3Q 302, 4Q taxol
322, 1Q07 311, 2Q 329 EMEA OK May 22/06, early stage patrients Her
may 5/00 : "Dear Dr letter :potentiates cardiotox of 2+ following surgery and standard chemotherapy
Raptiva $ 17,800/y. Adv Com Sept 9/03 anthracyclines and taxol :15 deaths.Nov 8/01 alert at
unanimous on risk/benefit, 9/2 on efficacy. "Chemotherapy foundation sympo". Jan 29/01 a trial stop. Competitor Tykerb, filed Sept 18/06 & EU Oct 9
FDA says PASI 75 : 18 -37% (4 trials), 50% ph III + CT : 10000 pts, 800 centers : ongoing
relapses; 2nd course 31% efficacy in pts who To file 07 1st line meta w/ Taxotere FDA
had efficacy w/1st course. Thrombopenia, - Trastuzumab DM1, ph I, w/Immunogen, Feb 1/06
infections. -Pulmozyme : $ 111 m→$122/00, 2002 : 138, 2003 : 167;
1Q04 43; 1Q06 49, 2Q07 55
-Xolair (omalizumab) ,hz anti IgE filed June 5/00 . S cut.
(1 or 2/month) FDA/CPMP;Novartis/Tanox. $ 7000- additional platelet data filed march 01(???). Compl
10000/y. 3Q since Jul 10 : $ 6.8, FY03 25.3; 1Q04 30, 2Q Resp Letter from FDA July 10/01. Trials ongoing .

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


43.7, 3Q 54, 4Q 61, 1Q05 69, 2Q05 80.4, 3Q 81.6, 4Q 93, Refiled Dec 19/02. Advis Com May 15/03 (K X 4
FY 05 320, 4Q06 117, 1Q07 111, 2Q 120 !!!); PDUFA June 19/03
Manuf :first Vacaville (then NVS) But high dose & COGS high. NVS to file CPMP
DNA + NOV to coprom In US, Nov alone outside 1H04
Oct 27/05 CHMP approved Jan 17/06 peanut trial stopped (unsafe test)
-Raptiva Xanelim/efalizumab, hz anti CD 11a ( LFA-1 is
CD11a/CD18)→PSO, weekly, subcut, phIII (May 01: -Xoma, Serono
PASI 75improvement>placebo). Peak PASI 75 : 38% Oct 5/01 : filing delayed until summer 02 (FDA
(Enbrel 56%, ciclosporine 70%,Remicade 84%). Rebound requests human kinetics → not comparable). Also
Roche option to mkt Avastin in issues. comparability of manuf methods (XOMA/DNA).
Europe : exercised July 8/03 . Filed EU New ph III w/DNA material (Sept 17/02) > placebo, N = Delayed again April 02 (different glycosylation).
Dec 5/03, CHMP OK Oct 21/04, EU 556, data at A. A. Derm March 21/26-03 Sales 03 (20 Filed Dec 26/02 → FDA adv com Sept 9/03 PDUFA
days) $ 1.4m; 1Q04 $ 6m, 2Q 13.4, 3Q 18, 4Q 19;1Q05 Oct 27
OK Jan 14/05 "in combination with
24, 2Q05 21.3 , 3Q 20.0, 4Q 20.4, FY 05 79 2Q 22; 1Q06, FDA OK Oct 27/03
5FU/FA or FOLFIRI"UK launch
March 10/05 3Q 23, 2Q07 27 21 March 21/04 ph II PSO arthritis NS,

-hz anti VEGF A (bevacizumab, Avastin) IV every 2 W


Manu in S. Francisco, will be Vacaville Beware of Sugen/Pharmacia SU 5416 (anti VEGFR2, ph III failed
+ CT→ Colorectal : 2 trials 1st line stage 4 (+ IFL vs IFL Feb 02 in colorectal because of tox)
(1H04) & Spain alone) 1st = 900 (100 on FU/A) enrolled 2Q02 : survival
hazard ratio 20.3m > 15.6, 2nd 200 is 24 m survival. Side - ECOG 3200 ph III : Avastin/Folfox 2nd line CRC,
effect HBP. Filed Sept 29/03, PDUFA March 29/04 if N = 590.
priority review: accepted Dec 1st 03. 2nd line & adjuvant - Roche ph III N = 1600, 1st line, comparing
ongoing. Avastin/Folfox & Avastin/Oxali/Xeloda
Dec 1/03 ph II +/- 5FU, 1st line, survival NS, but $ 46,000 per tmt course
progression free survival signif 1Q04 (6w) $ 38m, 2Q 133, 3Q 183, 4Q 200; 1Q05
ECOG 3200 : Folfox+/- Avastin (Nov 29/04), 2nd line 214 (US 203), 2Q05 US 246, 3Q US 325.2 (50% in
12.5m/10.7 (but Avastin alone 2nd line < Folfox alone). CRC), 4Q 359, FY 05 1.1 B, 1Q06 398, 2Q 423, 3Q
Feb 26/04 approved 1st line metastatic CRC 435, 4Q 490, 1Q07 533, 2Q 564; 3Q 597
w/5FU based therapy Aug 4/04 Dear Dr Letter thromboembolism x 2
2nd line CRC filed Dec 18/ 05. PDUFA June May 23/06 AVANT trial to resume, early stage
20/06 : approved June 20
NSCLC (w/Xeloda) only Adeno, ph III; ECOG 4599 1st
( RCC : Onyx/Bayer & PFESutent line, N = 878, init July 01,Taxol/Carbo +/- Avastin.
endpoint modified March 8/04. 1st line data 4Q05, or →efficacy(ASCO/00), but 6% fatal bleeding NSCL
approved ) Asco 03 : Tarceva + Avastin NSCLC
ECOG June 05 : March 15/05 interim survival
12.5m/10.2m (dosing twice CRC).sBLA filed April
12/06 in combi w/Pt based therapy for 1st line
non-squamous, PDUFA Oct 11/06 OK. EU

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


OK Aug 25/07
ph II NSCLC, 2nd line, N = 120 ASCO 06, 6 m survival ASCO 02 phII: TTP↗ (147d / 41d), no survival diff
A/T 78%, A/CT 77%, CT alone 62%.
2nd ph III w/cisplat/Gemzar ASCO 07 : low dose same
efficacy as high, more safety !
SCLC : ph II w/RTx + CT N = 29 terminated after 2
traceal fistulas
ph II renal K interim (Oct 25/01)→ endpoint reached,
enrol. stopped until strategy decided. Ph III 1st line init Oct
03, CALGB 90206 will evaluate TTP, + IFN, +/- Avastin,
fully enrol. July 05; N = 700 . Same study by Roche
(AVOREN) interim Dec 11/06 PFS signif improved 10
mg/Kg. See ASCO 07 : DNA trial PFS 10.2m vs placebo
5.4 (but Sutent alone 11.8 m vs 6.2)
ECCO Nov 05 : data Avastin/Tarceva : bof ?
ph III : refractory breast K w/Xeloda, enrol compl N =
462 ,3Q01, data 14/9/02 (tough endpoint TTP↗>50%) →
misses TTP, ORR signif, 12 m survival NS. 1st line breast
stages III/IV, HER- or Herceptin treated, Taxol +/- Avastin
ECOG 2100, ph II/III, N = 722, enrol compl Feb 05.Data But 13% neurosensory (taxol 6%), Taxol not
April 15/05 doubling of PFS (11m vs 6m), 52% reduction approved 1st line Breast, Taxol alone TTP 2.9m,
in risk of disease progression or death, compared to Taxol Gemzar Taxol TTP 5.2m
alone, SABCS Dec 05 survival 28.4 m vs 25.2, Filed May
25/06 for 1st line, PDUFA Nov 22/06. DELAY FDA
requests independent review of scans, will take until
mid 07, then 6 m to approval YE 07. Feb 23/07 CHMP
OK for mBC. Aug 25/07 refiles to FDA w/Taxol 1st line
(E 2100), ODAC Dec 5/07 NO; PDUFA Feb 25/08
Pancreas :
1st line CALGB 80303, N = 602, Gemzar +/- Avastin, June
²27/06 NS
AVITA trial + Tarceva + Gemzar, DB, init July 05, last
enrol YE 06
-rFab Hz/VEGF2 (Lucentis, ranibizumab), binds to all 5
isoforms of VEGF A (SmithBarney, May June 05, Yaron → competition Visudyne (IV)
Weber) intraocular, ARMD, ph I/II promising (Oct 02), Lic ex US to Novartis
good data Aug 03, 2 ph III ongoing. Filed Jan 2/06, Lucentis competition : late stage Cand5 (Acuity),
PDUFA June 30/06 : OK; 3Q06 163, 4Q 217, 1Q07 311, Sirna 027 (RNAI/MRK)
2Q 209
- Tarceva : OSI/Roche : 774, oral anti

- 50 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


EGFR,K,phII∏ASCO 01: H&N PR 6%; NSCLC 24%).
July 25/01 : ph III NSCLC + carboplat + paclitaxel
(N=330)→ASCO 02; pancreas w/gemcitabine (N=750).
- 2C4, pertuzumab, Omnitarg: Hz Mab, interacts w/Her 2-
EGFR heterodimerization/trafficking/signalling → ASCO
03 phI, ASCO 04 : "pulled", ASCO 05 ovary (CA 125 drop
in 15% pts), breast "limited activity", HRPC (no PSA
response)
Ovary ph II complete 4Q03 (to reinitiate w/Gemzar end
04). Breast (enrol compl 3Q04; Jan 7/07 fails ??), NSCLC
(enrol compl Nov 04), HRPC ph II enrol compl 3Q04.
Go/No go 2005
ASCO 07 Pertuzumab + Herceptin not bad, envisage ph III
for 1st line
- Anti tissue factor (for coronary) IND filed 1Q03, discont
for CV Oct 8/03
Phase II/III :
- 2nd generation anti CD20
- ALTU 238 retard HGH
- anti CD40 w/Seattle Genetics
- HAE1 2nd generation anti IgE
Phase I
- ABT 263, oral anti Bcl-2, ph I
- ABT-869, anti TK-VEGFR, ph I
- 3rd generation anti CD20
- anti IFN alpha (like MEDI 545)
- apo2L/TRAIL
- apomab
- BR3-Fc
- MEK inhib
- Met Mab from Exel (XL 518)
- PARP inhib
- Systemic Hedgehog Antagonist
DNA M TANOX (TNOX) - E 25 : hu anti IgE MAb for allergic asthma & rhinitis, Novartis/DNA 10% R
BLA filed June 5/00;add. data to be filed march 01; adv
Plans $ 400 m plant in Taiwan to Com April 25-26 canceled,new data filed;CRL July 01 ATS May 24/01 : signif steroid sparing effect
manufactute MAbs (Sept 13/01) CPMP was to give opinion 2Q01(but Mut Recog with
Germany as reference???). CPMP withdrawn, resub 1H04,
June 18/02 : Approved in Australia accepted Oct 27/05
Could be dangerous if parasitosis. Also thrombopenia

- 51 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Could sell $ 10,000/Year ?⇑ enrolment halt sept 22/00(lifted nov)
- TNX 650 Mab anti IL13, NHL, ph I
Nov 10/06 acq by DNA $ 0.9 B - TNX 234, pre, dry ARMD

DENDREON (DNDN) Proprietary Ags from Prostate acid phosphatase tagged to Kirin (discont Provenge Nov 14/03), JNJ
DNDN
autologous patient's dendritic cells obtained by apheresis, and
K June 14/03 PIPE $ 26.7m reinjected ;5 centres in US, 1 in Japan: ASCO 01 ph II (11/12 pts stable 4 months)
V -Provenge, Sipuleucel-T (3 infusions in 6 w), PSA ( prostatic acid ASCO 02
They rearrange the Ags by changing position phosphatase) vaccine(+GM-CSF) →HRPC. 1st, ph III DB 9901 Gleason : histol score. 5-7 well differentiated, 8-10
of epitopes & adding a cassette visible to N =127, enrol complete Sept 01 , but failures cross over open poor differentiation.
C dendritic cells to Provenge; results on PSA not bad but not remarkable;. 1st ph Feb 23/03, Int Prostate k SYMPO / & CASE
III interim Jan 11/02 : time to progression not improved, New
anal Aug 12/02 subgroup Gleason <7signif better data. Ditto
W:LONG SURVIVAL
Major agreement w/Gambro US to use their ASCO Feb 05 Sept 18/02 : <release for Prostate K conf : TTP
clinics for cell collection provenge placebo p improved, but NS (p = 0.06)
survival 25.9 21.4 0.01 Nov 03 (Prostate K Foundation) Provenge median
CEO : Chris Henney, cofounder of IMNX. survival G < 7 28.4 22 0.0046 survival 26.3 m/placebo 19.3
Nov 02 chairman. Gold CEO TTP G < 7 0.06 Jan 12/04 : Gleason < 7 : 30.7m/22.3 placebo,
%G<6 26.8 15.6 p=0.001
Feb 26/03 : acq CVAS ($ 73m stock), %G>8 39% 44% Oct 29/04 : 3 year follow up of ph III HRPC (or
completed July 30/03 36m?), N = 127 (?), survival benefit in ALL pts
- 2nd ph III D 9902B will enroll N = 225 in 12-14m, Gleason < 7;.
SPA (special protocol assessment OK June 11/03); Feb 22/05 will
better than placebo)
Follow on Jan 27/04 : $ 130m open this trial to all Gleason scores (data end 07
- D 9902 A N = 98 HRPC, ITT "encouraging ? survival trend", Not the same population as Taxotere :
Gleason < 7 = 75% of late stage prostate K TTP NS for "all" and for "Gleason < 7". Taxotere Mitoxantrone p
ALIVE AT 36 MONTHS ). July 20/05 : anal of 3 year data NS, but better than placebo. survival 18.9 16.5 0.0094
Gleason Provenge Placebo Signif if combined w/D9901
<6 40.9% 28.6% Also ph III in andr dependent : data ASCO Feb 05 PSA doubling
7 32.1% 11.1% time signif increase
8 40.0% 12.5% BLA filed for HRPC Nov 15/06. PDUFAZ May 15/07, ODAC issue w/FDA : 2 unapproved therapies
March 07 : safety reasonable (all), substabtial evidence of
>7 27.3% 0% efficacy (13/4). Also strokes 3.9% vs 2.6%; ongoing new ph III
orphan drug status granted April 25/02
data 2010, PDUFA MAY 15/07
Jan23/06, rolling BLA to init mid year ECCO Nov 05 3 year data OS 19m vs 15.7 (log
-Mylovenge→myeloma, ph II™ 60% of pts have strong decrease
Provenge in advanced stages in Ig BLA filing 1Q 03; new ph II w/Thalomid (start Nov 5/01)
rank 0.3; Cox 0.002)
June 12/02 signif TTP in pts who have immune response,
especially IgG pts, but ASCO 02 abstract less good Rolling filing init (Clin, Pharmacol) Aug 24/06 for
- APC 8024 : breast, ovary & colorectal K, I, ant her2/neu mHRPC, essentially 9901. Completed Nov 13/06
vaccine
- acq 2 Ags (CEA & MN for kidney/colon K) from Bayer Sept
26/01
- anti HLA DR patent issued July 10/02
- Trp-p8 gene, overexpressed in K , lic to DNA Aug14/02

- 52 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- ARF (alternative reading frame) : abnormal peptides produced
by K cells used as immunogens : 100% success in mice
(April 24/03)

DNDN CORVAS (CVAS) -rNIF(neutrophil inhibitory factor from canine PFE terminates June 17/02
hookworm)UK279276, t1/2 7d. anti CD11bI→stroke, ph
rNIF manufactured by LONZA IIb (N=1000) EU enrol ended Oct 01 (>100 centres),
July 24/02 staff ↘ 40% follow up Jan 02. Results mid 02 : April 25/02 failure
(more data in June∏ no effect
Acq by DNDN $ 73m Feb 26/03 in stock -rNAP nematode anticoagulant protein (anti VII), ph
II→DVT,reduces risk 50% versus LMWH
(hist)venography. July 01 end of ph II FDA ∏delays. To Feb 5/04 lic to Nuvelo
initiate trials in unstable angina.

-oral anticoagulant, anti Xa, PRE ⇑ lic to SGP

- Hep C viral protease inhib compounds, pre⇑⇑⇑ ∏lic to SGP


- serine protease inhibs for K , RES, w/ABGX & DYAX

DORB DOR Pharmaceuticals, was ENDOREX ( - Orbec, Oral beclomethasone tmt of intestinal GvHD, fails
DOR) primary endpoint ph III ( time to treatment failure) Jan JV w/Elan
4/05, but secondary (survival) is OK.To file 4Q05. ODAC
May 9/07 : NO; FDA Oct 20/07 approvable
-OraSomes : oral liposome delivery proteins/peptides
-Oral & transmucosal drug delivery for vaccines
-Oral HGH, w/Novo Nordisk, pre
-Oral Insulin,pre
- Biodefense vaccines (ricin, botulinum)
DOVP DOV pharmaceuticals ( DOVP) - Indiplon, NBI 34060, insomnia, ph III, lic to NBIX (
Ipo filed (Lehman) Jan 29/02, completed 3.5% royalty)
April 02 ($ 65m) - Ociplanon, ph II, anxiety, w/ELN
- Bicifadine, pain, ph II, w/ELN (bunionectomy), ph III
fails April 25/06
- DOV 216303, depression ph I
- diltiazem formulation w/Biovail
- triple reuptake inhibitor

- 53 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


DPII DISCOVERY PARTNERS INTERNAT. Highthroughput screning and services
(DPII), was Irori -July 01 Part w/Maxia (partner of JNJ) who has nuclear -Customers : ABT, AMGN, AZN,BI,Bayer,
Summer 06 : to be absorbed (reverse merger) receptor targets for diabetes II BMY,JNJ, PHA,SGP,GSK,Takeda,AVE
by Infinity (INF will own 69%) - Microcide

CRA is becoming largest shareholder ( Axys


has 30%)
DRRX DURECT (DRRX) - exclusive licence for certain uses of Duros spin-off (17%) from Alza
- deliver opioids for chronic pain
-market a catheter for delivery to inner ear
- IFN α for hep C sutained release lic to BioPartners (Switzerland)
DSCO DISCOVERY LABS (DSCO) -Tyloxapol : COPD, ph II
Neonatalogy "Specialty" pharma -Surfaxin DTPC + sinopultide lucinactant (21 aa peptide 3 m delay in manuf Dec 23/02 (Akorn)
Shelf March 30/04 : $ 24m KL4 modeled from human SP-B protein) humanized Sumitomo, Taisho
June 20/03 PIPE 27.5 m surfactant US w/Quintiles (regained for warrants Nov 5/04), EU
Feb 18/05 PIPE (SGCowen), closing Feb 24 →neonatal ARDS, ph III (against Curasurf (Mkt leader w/Esteve30% royalty (Spain)
EU pig lung from Dey LP)& Survanta ABT June 03 : at least equiv to Curasurf. Feb 19/04 FDA
price of other surfactants $ 750 to 1500 per Bovine/Beractant), enrol delay from May to Oct 03 (Feb puts on "Continuous marketing application Pilot 2
infant 21/03), East Europe, S. America. Pivotal v/Exosurf (GSK Program for ARDS" div of Pulm & Allergy drugs
Animal derived surfactant sales : $ 200m but only lipid, synthetic) or Survanta (ABT bovine, US Mkt NDA filed April 14/ 04 (PDUFA Feb 13/05), filed
large unmet need leader). EU Oct 6 04. Feb 1/05 delay due to manuf
Ph II to init. neonat. resp failure (aerosol/CPAP) compliance. Re-inspection. Launch 1Q06? . Feb
Adult trials w Exosurf (lipid) and SP-C Ph II to init BP dysplasia. data Oct 11/06 death 57.8% vs 14/05 Approvable letter "highly correctable
protein have failed 65.9% manufacturing issues". Aug 15/05 FDA requests
more manuf clarifications, response for Oct 05.
Dec 28/05 acq manuf in NJ ($ 16m) →adult ARDS ph II, endpoint survival or off-respirator at Approval hoped for April 06. April 5/06
PDUFA April 5/06 d 28, with segmental lung lavage; Dec 7/04N = 78, "approvable" more specifications needed on active
Adult ARDS data 1Q06 mortality 13.8%/20%; but failure of Altana & Exosurf ingredient). Manuf delay April 25/06 until 2007.
May 6/08 work force – 34% (purely lipid), $ 2B mkt June 8/06 EU filing pulled. Meets FDA 4Q06, to
→meconium aspiration ph III refile Sept/Oct 07
-DSC-103S,vit D analogue™osteoporosis, ph I Nov 25/03 RDS related death at D 14 Surfaxin 4.7%,
Exosurf 9.6% (p = 0.001), Survanta ABT 10.5% ( p =
0.001). N = 1294 .Against Curasurf mortality d 28
11.8%/16.1%
Dec 13/04 pooled data to Survanta & Curasurf p <
0.05
DUSA DUSA (DUSA) -Levulan (ALA, no product patent protection) : Schering AG. Reimbursement issues. Term June
Competition : ™Kerastick for actinic keratosis, Mkt (3Q00) 12/02
-ALA : Medac (Germany), ESC (Israel), * bladder K , ph I/II
Photocure (Norway)filing EU * restenosis, ph I/II
-PDT : QLT, Miravant, PCYC, Scotia.
- 54 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- BLU U : non laser light source
DVAX DYNAVAX (DVAX) - Tolamba AIC( Amb a 1, ragweed Ag), ph II/III init → Stallergenes
V files (B of A)for IPO dec 7/00, withdraws Jan/Feb ,04, N = 462, fully enrolled May 19/04 (Quintiles). lic ww to UCB, returned March 19/05
April 01. Primary endpoint : change in nasal symptoms. purified Sept7/06 $ 136m precl deal w/AZN to move into
Raises $ 35m series D April 02 allergen linked to nucleotides (ISS : immunostimulatory clinic within 3 years for asthma COPD DEAL :
K sequences), for desensitization. Fails Jan 8/06 10m upfront, 17 m research funding and pmrecl
Refiles Oct 24/03 (Bear Stearns, Deutsche) - Inhaled ISS for asthma, ph I AAAAI March 9/03, ph II milestones. Rest through regulatory milestones, then
Feb 19/04 raises $ 45m (half of pricing) ongoing royalties and could copromote.
- Hep B prophylaxis Heplisav, ph II/III (ph III planned ex
March 28/06, acq Rhein AG (Ag US), Ag from Berna. June 14/05 > GSK Engerix in
manufacturer) elderlies. Ph III init June 23/05; 400 seronegative adults
aged 40 – 70, in SE Asia, vs Engerix. 2nd ph III to initiate
EU & CND for younger population.. To file 2H08
- ISS for K : ph II NHL w/Rituxan, N = 30, data end 06
- Vaccines for HIV (W/Aventis) →Triangle
- mCRC 2nd line w/ Campto erbitux, ph I init Dec 5/06
DVSA DIVERSA (DVSA) Enzyme discovery libraries for Chem, Agro, Pharm lic to IntraBiotics(released from obligations July2/01
DYAX Dyax (DYAX) A key MAb company. .Hold the Ladner patent for phage display (CAT &
Morphosys could be dependent) and for scF (CLL
M Also chromatography business could be dependent). More than 50 licensees
.alliance w/Bracco.
part w/ABGX Also have drugs in development :
part w/XTL jan 22/01 --Reltran(EPI-HNE-4),neutrophil elastase inhib (r prot in w/DebioPharm
aerosol) ∏COPD,CF; phII
--DX 88,r kallicrein inhib(IV, working on SC)→inflam;
Staff ¬ Sept 11/02 phII; JV w/GENZ, hereditary angioedema (EDEMA I & - competition : Extractive Esterasine ( Cinryze)
II, data "positive" 2Q04 but N = 48 and randomization 5/1) Baxter C1E inhib Sanquin in EU (MKT), LEV in US
Patients ww 22,000 but Bayer Trasylol did not work.Edema II open label (ph III init 1Q05 for tmt filed July 31/ 07, Nov 05 for
Price for Pharming $ 4,000/attack, 7 attacks ongoing prevention to conclude 2H07)
per year . -Jerini AG (public since Nov 1/05, Icatibant
"met FDA year end to discuss possible registration (decapeptide) antibradykinine, manuf by Bachem
pathways based on ph II". (March 18/04), rights acq Nov 7/05 for US by KOS
Edema III (N =72)SC init Dec 8/05,1st part DB (compl (acq by ABT $ 3.3 B Nov 6/06 ); IV & SC (April
Nov 20/06), then open Approval 2007 ? May 16/06 dose 5/04), ph III init Sept 8/04 in US (FAST I fails sept
ranging issue delays > 2007. Data April 12/07 OK. 20/06) & Europe (FAST II OK Sept 20/06), Orphan
Edema IV requested by FDA, to init 2Q07, approval status. Filed US Oct 8/07
YE 08 ? -Pharming Hu.r C1E inhib.in EU rand DB placebo ph
DX 88 also in CABG (Ecallantide), further only if partner. III ongoing, , good data Madrid March 15/05.
Nov 6/07 : Bayer terminates Trasylol Compassionate ongoing EU. Feb 3/06 Paul Royalties
--antiK f Hu MAbs acq with Target Quest : $ 30m for small share of royalties.
-scFv anti p21 Ras, pre -ZLB Behring ph III Berinert
- 55 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-F ab anti MUC-1, pre
- anti MUC-1 immunocytokine(IL2 conj),pre
- phage display peptide for purif. of VIII lic to AHP (May
22/01),also one for HGSI's BLyS
- DX 890 depelestat, ph II, Cystic F. & ARDS, rInhibitor
of neutrophil elastase (w/Debiopharm).
EBIO EPOCH BIOSCIENCES (EBIO), Gene modifying oligos (enhance binding to minor PEB,Aug 18/00, for Taq kits
Was MicroProbe groove)
EBSI EMERGENT (was BioPort) Anthrax vaccine, FDA approved
IPO filed Aug 16/06
ECX K EPIGENOMICS AG (ECX Frankfurt) diagostics for K based on DNA methylation
EMBX EMBREX (EMBX) AUTOMATED POULTRY VACCINATION 80% of US MKT
EMIS EMISPHERE (EMIS) -Heparin liquid: phIII initiated with Quintiles..First ph 3 enrol compl DuPont for Heparin & LMWH (July 30/00) discont
4Q01 (N=2200), 2nd to start then.Failure May 15/02 : < Lovenox
- Heparin solid, ph I
by EMIS (May 2/01)
This oral delivery is essential : the risk -Calcitonin (salmon) : phI successful. Partner Novartis exercised (Feb 00)
of respiratory side-effects should its licensing option, ph II.
-Insulin : was tested in rat model, then PHA terminated.
obviously be much less than with the Ph I good data (Sept 7/01); March 5/03 potential partner drops
respiratory route. But for the time -PTH oral with LLY, but on hold before clinicals.
being only polysaccharides and -HGH with LLY. Tested in primates.Ph I init Aug 02 Competition: buccal spray (Generex/LLY), other oral
-Oral vaccines are also being developed.
peptides have shown bioavailability (Nobex/GSK)
-Oral Axokine for Diabetes II→→→→→→→→→→→→

June 02 : staff reduction 30% - Oral Daptomycin, pre™™ Regeneron


w/CBST
ENCY ENCYSIVE (ENCY) was TEXAS -Novastan (argatroban) ,anticoagulant. FDA approved for w/ GSK
BIOTECH (TXBI) HIT june 30/00, filed dec 24/01 for PTCA(approvable SASY was for Europe, but terminated in 99
letter July 21/01)
March 21/02 : new CEO from JNJ -Sitaxsentan Thelin,oral, once a day, phII/III, PHT & -June 7/01 : 50/50 JV W/Icos endothelin antag. Feb
CHF 3/03 TXBI to regain, done April 23/03
June 2/06 CHMP OK ph II PHT
seems also active in stage II PHT 12 w random -july 11/01 : lic to SGP of oral VLA4 antag, Res
FDA June 15/07 Approvable 100/300/Placebo. Primary endpoint VO2 reached at 300. 6
min walk distance improved at 300 & 100. PHT halted
July 24/02 : Transam ↗& clotting issues… will meet
FDA. Feb 14/05 ph III STRIDE II vs/ Tracleer BID vs ATS May 05
placebo, N = 246. 100 mg > placebo (p = 0.03) on 6 min
walk distance. Filing April 05 : filed May 24/05. PDUFA BUT : inhibits warfarin catab, Tracleer increases it
March 24/06. March 27 : "approvable". June 15
accepted Compl response for review : action July 24 :
July 25 : 2nd "approvable". Refiled Nov 5/06 PDUFA
JUne 15/07
- 56 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- TBC 3711, March 23/06 2nd generation Thelin on hold

ENMD K ENTREMED (ENMD) -Endostatin prot : ph I (bad comments Boston Globe, May -
12/00), 24/26 tumors increase in 4 months (ASCO/01).
Aug 8/02 staff¬ 25% New ph I/II init July 01
- ENMD 0996 : FGF 2 vaccine
Pipe Nov 4/03 : Rodman -Angiostatin prot : ph I/II
- Panzem, oral, 2Methoxyestradiol, K, myeloma, ph II;
Dec 28/05 acq Miikana inhibits HIF (Hypoxia inducible factor & triggers apoptosis
(upregulation of DR5 (death receptor 5), & disrupts
Largest shareholder : CELG microtubules; scheduling key to efficacy.
- TFPI peptide fragments, pre, lic to Affymax (Oct 24/04),
trying to increase bioavailability w/Elan
FROM MIIKANA /
- MKC-1 (from Roche) oral cell cycle inhib to init ph II
refractory mBC 1Q06, then NSCLC
- MKC-1693 aurora kinase A inhib, pre
- MKC-1704 HDAC inhib

ENVG ENVIROGEN (ENVG) Remediation


ENZ ENZO BIOCHEM (ENZ) -antisense GT→Aids : ph I -good data CD4 still express after 8 months
V -EHT899, oral™hep B, ph II -good ph I data (oct 31) AASLD & ph II (oct 12/01)
- oral 14 aa peptide (from HLA B27) for uveitis, ph I June OK
10/05
- oral Ag for Crohn, ph I (Israel)
-sales of reagents for genomics & sequencing
ENZN ENZON (ENZN) -PegIntron (12kD)™Chronic hep C, E.U. approved May SGP( royalty 7.5%)
The one stop shop for pegylation due to 30/00; filed FDA Dec 23/99→approval jan 22/01 EASL(May 01)™HCV efficacy=Pegasys :
M relationship w/INHL & Shearwater PEGIntron +Rebetron alone NDA filed feb 6/01(priority
-May 23/01 : new CEO (A. Higgins), former rev. March 23/01)∏approved Aug 8/01,launch Oct 4/01
President of ABT pharmaceutical division. PEG-Rebetron EU approved March 27/01 -Roche Pegasys (40kD) filed FDA May 23/00(but
-Complex litigation Dig Dis Week (May 01): PEG Rebetron improves Hep C questions May 27/01∏filed Pegasys bioequivalence
SGP/ENZN/ROCHE/Shearwater (who in 35% IFN non responders. Synergy w/Lamivudine in April 15/02. Oct 17/02 Pegasys approved US Hep C;
nevertheless has "manufacturing role" in Hep B (May 02) priority review granted for combi with Copegus, Adv
ENZN PEG!);SETTLED (Aug 13/01, will ASCO 01 Efficacy of PEG IFNs not > to IFN in K, but com OK on combi Nov 14/02, FDA OK Nov 4/02. EU
share royalties) melanoma ph III ongoing Pegasys approval March21/02 & Copegus (Ribavirin
-May 22/01, INHL acq Shearwater, key PEG Jan 14/ 02 : SGP capacity for IFN plateauing. Seems like) Sept 18/02. See my ENZN file for Rebetron
competitor of ENZN overcome (Feb ½)

- 57 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- PEG secretin for cystic fibrosis IV, without PAG in ph II
Aug 21/01 : first year of profitability (Pharmagen UK)
- Abelcet (US) acq w/sales force (60) & facilities from
INF α generics : Retard SkyePharma & ELN Oct 2/02 ($ 370m), 2002 sales E $ 80 m. in Europe,
GeneMedix ( GMX/LSE, SGX) which sells but US small
GMCSF in China, could be approved - Prothecan (Peg camptothecin)ph I∏phII (July 25/01),
2007, Sicor (SCRI) is selling in East NSCLC, pancreas, gastric
Europe. - Peg-Paclitaxel (Taxol), ph I, without Cremophor.
Feb 18/03 US IFN α manuf patent issued to Term Jan 3/03
Roche ( → 2019) lic to Alexion(phII), CEGE, CAT, BMY, LLY,MOR,
- CDP 870 pegylation Baxter, Aventis, Seattle Genetics
Feb 20/03 : to merge w/NPSP. Term June Molecules from SCA ventures (wholly owned subs)™
03Profitability delayed to 2006/7 - scFv s
- PEG scFv s, w/Micromet AG (April 12/02) has linker &
fusion patents
- rhMBL (mannan binding lectin) from NatImmune A/S
for infection in deficient patients
- anti – survivin oligo, pre, lic from Santaris, July 27/06
- HIF antag for K, IND filed Jan 7/07
EPCT IPO Epicept (EPCT), filed Jan 10/05 Lidocaine patches
Wachovia, traded, acq MAXIM
EPCT Maxim Pharmaceuticals (MAXM) -Maxamine/Ceplene (histamine HCl2), increases ASCO/00 : IL2 alone™187 days survival
K to become Epicept (EPCT) expression of CD86 (CTL trigger) (AACR, March 01) + Maxamine™355 days survival. Oct 01 : 24 m
Aug 11/00 : Roche collab for Pegasys + -with IL2→phIII,melanoma,filed July 19, sept 8, priority
Maxamine hepC & K review granted Dec 13/00 ODAC negative because of follow up : p = 0.003
biometric imbalance in 305 pts DB™class action suit. June 14/03 : improved QOL in meta melanoma
April 13/04 FDA permits recovering costs Jan 20/01 : non-approvable letter from FDA, they are not
for compassionate use w/IL2 "most critically Say (Sept 22/03) will file CPMP melanoma Nov 03
content with 1 single study,new ph III ongoing, results end
ill melanoma", while ph III is completed (N 01/1Q02. 4th ph III init Jan 02,enrol + follow up 3y Nov 10/03 files EMEA data refused in US in 01 (2
= 230, enrolled Sept 03, NDA filing 1Q05. EASL (april 20/01), 72 weeks data for hep C, but only
Sept 20/05 fails to meet survival endpoint. year survival 18.2%/IL2 alone 2.7%). To withdraw.
historical comparison, no impact on stock
Filing withdrawn -AML, ph III, completed (oct 9/00) : N = 320 +IL2. Only
enrolled pts in CR. Controlled versus no tmt, 18
Oct 18/04 : halves staff AML : ASH 04
months, TT relapse > no tmt, p = 0.026 (they
Sept 6/05 : to be acq by Epicept ($ 136m) should have received maintenance tmt)
2007 : seeking EU approval remission maintenance AML

-with Ribavirin+Roferon™hep C, hi good @AASLD


-oct 13/00 : precl . activity on sarcoma
- TIP 47 : IGF II receptor binding protein, precl
- Lidopain SP ph IIIEU trial data 2Q06

- 58 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


EPIX EPIX (EPIX) MS 325 : MRI vascular enhancer (moiety binds to -Agreements with potential competition :
-Current MRI contrast mkt $ 400m (Schering albumin),ph III;enrollment by end 01; submission 2-3QO2 Mallinckrodt will manufacture, Schering will
50% w/Magnevist, Nycomed 30%) delayed until 1Q03 to include broader peripheral distribute (and EPIX has rights to SCH 555C and
-Potential mkt : Xray angiography mkt : 2m indications Gadinex 17 that Schering developed for vascular
pts/Year. MRI is less invasive. ACC March 02 : 1 of 4 ph III compares MRI w/ & wo/325 imaging)
Aug 06 : merger w/ PREDIX to angiography. w/325 ; 88% "accuracy", but p?, and - license of 2 patents to Bracco (Sept 01), upfront
"indication on tmt course" 84%. $10M
From PREDIX :
- GPCR w/GSK Jan 4/07 (upfront 30m, up to 1.2B).
Alzheimer
- S1P1 modulators w/AMGN
EPTO EPITOPE (EPTO) Oral screening tests for HIV & Nicotine sells to insurance companies
merged w/STC⇑OraSure
ERGO ERGO SCIENCE (ERGO) Bromocriptine failure in diabetes
oct 16/00 : 3 Board members resign
March 24/01 : delisted
ESPR ESPERION THERAPEUTICS (ESPR) Products licensed from PHA, Inex, etc
-The concept : statins lower LDL (bad cholesterol). ESPR
CEO (Roger Newton) codiscovered Lipitor at seeks drugs to increase HDL (good cholesterol).
WLA -The molecules : for the time being more like cholesterol
scavengers :
PIPE (July 26/01) -rApoA-1 Milano (lic from PHA)™phI. →could be IP issue w/Cidars Sinai Med Ctr (LA)
who use AAV Apo A1-Milano
Dec 22/03, PFE to acq for $ 1.3B
-proApoA-1, ph I, IV
→patent from Region Wallone expires in 2008
-LUV, ph II, IV:large unilamellar vesicles (empty
liposomes) : results 4Q01

-RLT (reverse Lipid Transport)22 aa peptide :pre


-ESP-A :small molecule HDL elevating agent,Res.
ETRX ESSENTIAL THERAPEUTICS (ETRX) -Cephalosporin, ph I , injectable JNJ
was MICROCIDE (MCDE) - oral cephalo, pre
Top management experts in ABs from SGP,
PHA, - Efflux pump inhibitors for bacterial & fungal resistance, Daichi
Acq. Athexis* ( Aug 01) that was involved in pre, potentiate Quinolones & Azithro. ICAAC 01 : 19 abstracts
Zithromax discovery - Rhinovirus protease inhibitor
- MARstem , small molecule peptide analog, bone marrow
stimulator, phI/II.

EURX IPO EURAND (Italy, Holland) Drug delivery Many partnerships


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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


founded 1969, traded May 17/07
- Zentase, EUR 1008, porcine derived pancreatic enzymes, Competition see ALTU
2 ph III completed. Rolling filing init June 12/07.
EVC EVOLUTEC Plc (EVC / AIM) - r EV 576, C5 inhibitor, pre
- r EV 131, ph II antihistaminic
- r EV 131 ph II, cataract surgery
EVSC ENDOVASC (OTC BB : EVSC) Liprostin (liposome PGE1) successful ph II in PAOD, ph
and its fully owned subsidiary Angiogenix III for 05
EXAS Exact sciences (EXAS) Genomic detection of colonic K in stools (detection of Trials at Mayo Clinic and Johns Hopkins, will initiate
IPO filed oct 27 (ML) microsatellite deletions caused by mutations in genes of blinded multicentre trial (5300 pts) 4Q01
trading since jan 31/01 mismatch repair, and loss of heterozygoty), more sensitive 11 abstracts at DDD (May 19-22/02=
Will outsource manufacturing. than occult blood or sigmoidoscopy, less invasive than CEO Myriad says "excellent test, but positive later
Can sell under "home brew" reg" colonoscopy than Myriad's Colaris, which detects
Strong IP situation predisposition to colon cancer"
PreGen 26 : available in US since June 1/01; full launch Good papers : J n. can. inst June/01,NEJM Jan
Dec 01 by LabCorp; lic to LabCorp June 27/02 3/02, but success rate of detection only 57%)
EXEG EXEGENICS (EXEG) Drug discovery:
- OASIS : patented software to design antisense
- research on mycothiol depletion to fight resistant TB

EXEL EXELIXIS (EXEL) -Functional genomics BMY ( from 2001, Dec 18/03 extended $ 100m),
K - Antisense partnership w/AVII (April 01) PHA. for Ser/Thr kinases in PTEN & Caterin
Chairman St Papadopoulos -Drug for Alzheimer : pre (lic to PHA) pathways
Sept 4/07 major collab w/Dow Agro -Insecticide($ 10 B mkt) Discont April 3/05
June 14/03 : shelf 10m shares ($ 75m) - XL 119, becatecarin topoisomerase II inhib, DEAE
Sept 8/00, acq AGTO analogue of Rebeccamycin(from BMY) , bile duct K ph
Nov 20/01, acq Genomica II, data Feb 03 Paris Conf on K , N = 20, maybe safe & → ASCO 03 : Bil K first line, N = 30 survival 8.8 m,
Oct 29/02 w/GSK upfront $ 30m, stock 14m effective. Enters ph III June 23/04 versus 5FU for 3 years, double of historical ! Bile duct K incidence US + EU
(premium x 2), dev funding $ 90m on 6 years,SPA, survival w/interims 05/06.. Not for GSK. Lic to 15 to 20 000/Y. Orphan Drug March 15/04
loan facility $ 85 m, milestones $ 220-350 :Helsinn (Ch) June 10/05 ASCO 04
collab to dev theraps in vascular, inflame &- XL 784,ph I,oral, targets cell surface Adam10
oncol using Exel drug candidates (XL 784, metalloprotease involved with growth factors, to be
647, 999, 844, 9820, 2880, 6309, 2942) developed in renal & cardiac failure. Ph II in diabetic
proteinuria
RISK : GSK returns -XL 647.ph II,inhibitor of
May 23/01 : partn. w/PDLI: targets for MAbs EGFR/Her2neu/VEGFR2/EphB4 (Ephrin B4, highly
expressed in Breast cancer), ,Aug 06 ph II NSCLC 1st line,
adeno with activating mutation or Asean init. July 26/07
GSK opts out. EXEL says data was OK (could be worried
w/Tykerb approved March 07). Interim ph II Seoul Sept
5/07 Int Assoc Lung Cancer : N = 30, ORR 60%, QT
- 60 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


elongation 81%
- XL 999, multiple Receptor TKs inhibitor., ph II,
suspended Nov 2/06 : cardiac; but not bad NSCLC &
AML : ASCO 07
- XL 9820 anti cKIT
- XL 844 anti check point kinases I
- XL 880, ph II gastric, H & N, RCC, anti Met & VEGF
R2 TK. Eval for GSK from Sept 14/07, then 90 days to
Dec 14, precl active BC, CRC, NSCLC, GBM
- XL 228 IND filed Aug 3/06, Kinase inhib of ILGFR, Src,
Abl
- XL 418, pre, anti AKT
- XL 518, anti MEK RAS pathway, IND filed Dec 20/06, XL 518 : other MEK inhibs ARRY 438162, ph I, PFE
lic to DNA Dec 22/06 PD-325901, ph II; Ardea Biosciences
- XL 281, ph I, anti RAF kinase
- XL 281, ph I, anti RAF kinase
- XL 147, anti PI3K, IND filed March 16/07
- LXR (liver X receptor) agonist, precl, lowers cholesterol,
w/BMY (Dec 6/05)
- FXR (farnesoid X receptor, nuclear hormone receptor)
drug w/WYE Dec 22/05
- XL012, oral JAK2 inhib, IND filed May 24/07
FHRX First Horizon Pharma (FHRX) A "Specialty Pharma company" with 14 marketed drugs.
Acquired on April 14 from WLA :
-Ponstel (NSAID), but how will they manufacture it
-Cognex : the Alzheimer drug nobody uses since Aricept
(PFE)
FLML FLAMEL (FLML) See EUROBIOTECH
FMVR FERMAVIR (FMVR OB) CF 1743, shingles, development candidate
owns patents for compounds varicella-zoster, CMV

FOLD IPO Amicus Therapeutics * Treatment of protein mis folding of chaperone with oral Fabry good data 42nd annual sympo study inborn
CEO Crowley's 2 children have Pompe. He small molecules (deoxynojirimycin derivatives) errors of metab. Paris. Sept 5/05
founded Novazyme - AT 1001,Amigal, migalastat, ph II init Sept 8/05, Orphan
status, Fabry. Enrol compl March 07, digestive tox Other misfolding firms : FoldRx, Reata
files for IPo May 17/06 (AMTX), withdrawn - AT 2101 (Plicera, isofagomine)Gaucher, ph II init March Pharmaceuticals
summer 06 07 (might only be for pts with high residual activity), data
Priced May 31/07 4Q07
- Pompe AT 2220 ph II init Dec 14/06, to file end 07 (?)

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


FVRL IPO FAVRILLE (FVRL) filed April 04, traded - Favid : biopsy, identify unique idiotype gene, ASCO/ASH 06 ?
K Feb 2/04 manufacture patient specific recombinant protein conj. to
Keyhole limpet hemocyanin (KLH) to vaccinate with
associating GM-CSF; NHL, ph III since July 04, enrol
compl Jan 06. Interim Nov 13/06 : failure
GCOR GENENCOR INTER (GCOR) -Biomaterials
-Genetically modified enzymes PG
Partn. w/Dow Corning : "Silicon - VP 22 technol acq from Phogen (Aug 01)
Biotechnology".
J.J. Bienaimé CEO Nov 02
GEN K GENMAB (Copenhagen) Fully human Mabs
Nasdaq GNMSF - MDX-CD 4, HuMax CD4, Zanolimumab RA, phIII
MEDX 20%; sell 5.1% on Feb 16/07 (now initiated 02. Ph I/II T cell lymphoma ASH 06; Ph III
have 11%). Same day Goldman Sachs sold CTCL w/Serono. Returned Aug 1/07
2.6 M shares HuMax-CD4™PSO, ph II,
Serono 6.7% now (2006) seems reserved for non cutaneous T cell
lymphoma (very rare). June 30/07 Serono returns
- anti IL15, pre, PSO w/IMNX/AMGN
anti IL15, ph I/II, RA w/IMNX/AMGN, Sept4/02 26%
reach ACR 70 (Enbrel : 15%)
anti IL15, pre, NHL w/IMNX/AMGN
now AMGN has put it back (AMG 714) into preclinicals
- Anti EGFR, Zalutumumab (ch, IgG1), claim activity at
0.125 mg/QW. Ph III init Sept 14/06, N = 273, 2nd line H
& N vs BSC. Primary endpoint survival (Erbitux only had
ORR, nope : since Oct 2/07 OSapproved).
1st line H & N ph III Sept 07, to potentiate RT
- Hu CD 20 ofatumumab. Dec 19/06 lic to GSK (2.1 B
upfront 102), depletes B cells more than Rituxan
Efficacy independent of levels of expression of CD 20 (so
they say). Strong action on C', Sept 7/07
ph III CLL refractory init May 06
ph III NHL init July 10 06, N = 162, refractory to
rituxan, endpoint ORR Cheson criteria (clinical, lymph
nodes, BM)
RA ph II (side-effects, see CIAO Med). Ph III in
MTX refractory, & in TNF refractory, to init 1H08 EU
- MDX 018, ph I/II glioblastoma init Sept 13/07.
IgG1 anti IL8. Will also go to COPD

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


GENR M GENAERA (GENR) -Squalamine(antiangiogen.)→NSCLK w carboplat & ASCO 03 NSCLC N = 45, ORR 23%
was MAGAININ (MAGN) Taxol, ph II N = 90, good data nov 16/01. Jan 28/03
enrol halted at N = 40 to economize. Will analyze at
Aug 11/02 staff ¬ 35% mid year for response, ph I/II ARMD. Terminated Jan
4/07
Genentech(May 2),returned dec 18∏w/MEDI
-anti IL 9→asthma, PRE (april22/01)

- Produlestan (also aminosterol),obesity, pre


- Lomucin MSI-1995,oral, blocks respiratory mucus
overproduction, ph II. Good data Oct 03.

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


GENZ A Genzyme General (GENZ) - Ceredase/Cerezyme recomb (imiglucerase) US patents 2010/2013 : $ Patents : process 2010, composition 2013
479m/99; 537/00→ Gaucher 570/01, 619/02; 03 guidance 655; 1Q03 TKTX to file IND for generic (early 04)
Fabrazyme. ADV COM Sept 26/02. 167.2/2Q 184.7, 3Q 189, 4Q 198; 1Q04 203; 2Q 209.4, 3Q 208.4, 4Q
IgE or not ?? → POSTPONED→ 219, FY 04 839, 1Q05 226, 2Q 236, 3Q 238.3, FY 05 932; 1Q06 239, 2Q sept 8/01 ph III efficacy p < 0.001, GENZ sues TKTX
254, 3Q 252, 4Q 262, FY 06 1B, 2Q07 283, 3Q 286 Genz says Replagal & Fabrazyme are "very structurally
K Jan14/03 Thyrogen :3Q03 : 11.4 m; FY 03 : 43; 2Q04 16.3, 3Q 15.4, 4Q 18, FY 04
“ near normalization of renal capillary endoth similar and funtionally equivalent"
63, FY05 78; 1Q06 23, FY 06 94, 1Q07 264, 1Q07 28.2, 3Q 26.8
is likely to predict clinically meaningful - Fabry (Pain ?)Fabrazyme agalsidase β (CHO) BLA filed june 26
Are there IgE Abs to Fabrazyme??
effect”. Ph IV (18m) required on (TKTX june 19!),CPMP OK March 28/01, w/slightly better labelling for
Replagal (TKTX),EU approval Aug 01 Trial against Replagal in Holland (not by Genz)
“prolongation to time to deterioration or
priority review granted (⇑dec 26/00 FDA requests additional data ,given
death”. Genz disagrees on design, wants April 19 /01 and initiation of ph IV on clinicals renal, heart, CNS, N =
blinded but not placebo. 82),sold in France (was ATU).1mg/kg Infusion 180 min. >95% clearance
$PDUFA April 23/03, approved April of GL3. Pain improvement same as placebo. $ 6 m/01, $ 26 m/02. 1Q03 → see BMRN (50/50 JV),Rolling BLA compl Jul 29/02.
11.8/2Q 15.4, 3Q 21.5 (6.5 in US), 4Q 32; 1Q04 38.1 (US 15.8), 2Q 49.6, CPMP filed March 2/02. 2 new ph III to init. PDUFA Jan
23 (ph III DB N = 58). ORPHAN 3Q 58 (US 21.9), 4Q 64, FY 04 209 1Q05 70, 2Q 74, 3Q 79.1, FY05 305;
If ph IV (to complete Jan 04 not +, could 1Q06 81, 2Q 89, 3Q 93, FY 359, 1Q07 101, 2Q 104.3; 3Q 105 28/03→ CRL answered, FDA to act < April 30. OK April
be removed !) - Hurler MPS1(intelligence ?), Aldurazyme 30/03, shipped May 15/03. EU OK June 11/03. FDA &
w/Biomarin,PhIII;enrollment completed march 4/01. CHO. 2Q03 : $ 1.1 EU want 100pt posr mkt trial Launched Scand,
Prices : m, 3Q 3.4, 4Q 6.7; FY 11.5; 1Q04 7.3, 2Q 9.2, 3Q 10.3, 4Q 16, FY 43,
- ceredase : 150-175 000/y 1Q05 16, 2Q 19, 3Q 20.1FY 05 76.2; 1Q06 21, 2Q 23.5 3Q 25, FY 06 Deutsc, UK, F.
- Fabrazyme : 180 000 96.8, 1Q07 28.8, 2Q 29.1
- Hectorol : 1Q06 19, 2Q 22.4, 3Q 25.5, FY 06 93, 1Q07 28, 2Q 27.3;
- Aldurazyme : 175 000 3Q 30.3
Patients : -AT III , w/GTC Biotherapeutics (mainly EU mkt $ 250m?) filed EU Jan EMEA filing Jan 26/04
Gaucher : 1/10 000 ashkenases 26/04
(3200 pts treated ww) - Myozyme(CHO from Synpacdiscont April 17/02, will use in-house JV w/Pharming, entirely GENZ (Aug 26/01)
Hurler : 1/100 000 all product) :phIII started april 2/01, ph II efficacy data on heart size
(oct 5/00, in 3 pts)+++ Competition from Novazyme*; acq Aug 7/01!!($ 137m)
Fabry : 1/40 000 caucasians Myozyme α glucosidase for Pompe, 2 studies init 1Q/2Q03. Enrol
Pompe :eastern world : 1/40 000 compl 2H03. Filing ASAP. To start observational study (n = 60), then Aug 07 Myozyme manuf issues
COGS Myozyme 20 000/200 000 placebo controlled. Also special access programs. Filed Dec20/ 04 EU
CHMP OK Jan 27/06. filed US July 29 05 PDUFA JAN30/06 delay 3
m. 3Q06 20.4, ,FY 06 59, 1Q07 37.9, 2Q 46.7; 3Q 53.6
-CAT 192 human Mab (anti TGF β) for scleroderma , ph I/II NS (Feb
Sept 22/03 : 11% of CAT 9/04), gender issue.
w/Myosix (France) ph II, myoblast
transplantation (Ph Menasche)

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS

May 4/05 acq Bone Care BCII ($ 600m),


GELTEX PRODUCTS FDA (Sept 14/01) : misleading promotion
doxercalciferol IV/Oral (Hectorol) sales
-Renagel (sevelamer, polyallylamine) : $20/99→ 55/00; →177/01(due to Pr Fournier (oct 01) : really effective doses are 12 x400/d
around $ 75 m/y. Aphios has a non toxic vit inventories).2Q02 : $ 39.5 (-4.4%) 2002 : 156; 2003 guidance 240. 1Q03 & give digestive issues. Calcifications are linked to too
D for cancer prostate. 58.8, 2Q 66, 3Q 75.5, 4Q 82; 1Q04 83.5; 2Q 87.6; 3Q 93.3, 4Q 99; FY 04 much vitamin D and not Calcium salts, and to vascular
Sept 28/05 collab w/RenaMed renal 364 1Q05 99, 2Q 101 , 3Q 106.9, FY05 417; 1Q06 119, 2Q 126.6 3Q
causes. AMGNs calcimimetic could be interesting.
epithelial cells in hollow-fiber cartridge 134.7, 1Q07 137.4; 3Q 154
. Open label ("treat to goal")ph IV, N=202, less aortic calcif. (EDTA June
26/01 then ASN Nov 01 : coronary calcif score lowered, but groups not April 18/03 Problems w Renagel/Phosphate trial, extended
comparable on initial figures). New large trial initiated but concluded to 2005.
0ct 12/01 cause results so good!!??. New trial again, mid 03. Many
agreements with dialysis clinics (Gambro Feb 4/02). Metabolic acidosis -lic to Sankyo/Parke Davis
(ASN, Oct 01). Also sold in EU–
GENZ 29 155 : MS, ph I
-Cholestagel/Welchol(cholestyramine-like) : NDA approved May 30/00
-Clost. diff. Tolevamer: ph III DB Vanco, ph III init April 2/05. Fails
July 07
- DX 88 antikallikrein (JV w/DYAX), Ph II, HAE..Discont.

GENZ M Genzyme Molecular Oncology (GZMO) -Melanoma vaccine(Ad MelanA/Mart1+gp100 : ph I/II ASCO 03 (retinal issues : hypopigmentation ?)
-aaATIII (antiangiogenesis),shape modified : PRE
Very large in-house Ag discovery program -p53 gene therapy : ph II, lic to SGP

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


V -Breast, renal & melanoma : patnt specific vaccine
Kcell/Dendritic electrofusion,phI/II (Donald Kufe licensor) ASCO 02
: breast ph I OK ASH 01, RCC init 2003
DIAGNOSTICS w/Purdue to manufacture MAbs
w/Kirin (Nov 01, ditto)

GENE THERAPY

w/Aplied Genetic Technologies Corp (May 05) AAV


- Alpha 1 AT ph I
- Pompe, pre ?
- Lebers's congenital amaurosis, pre ?

GENZ GENZYME BIOSURGERY (GZBX) -Carticel : annual around $ 20m (not on EU mkt)
-Seprafilm, Sepracoat 3Q03 : 12, FY 04 61.6, FY05 68,
FY 06 85, 1Q07 23; 3Q 26.4
-AdHIFα (hypoxia inducible factor : turns on
merger of Biomatrix,Genzyme Biosurgery & s
G.Tissue repair VEGF expression) gene therapy for myocardial
Feb 8/05 acq Verigen AG (MACE : matrix neoangiogenesis, phI A.H.A. Nov 03 limb survival rate
induced chondrocyte implantation) on sale in 67% (expected rate 50 to 65%), BOF!
EU & Australia since 1998. From Biomatrix : competition Supartz (Smith& nephew), Hyalgan
-Synvisc™O.A. knee in US & hip in EU (ph III in US) (Fournier Sanofi), Arthrease JNJ (filed) , Ostenil
,MKT in US by WYE (all rights regained Nov 5/04 (TRB Chemedica UK, bacterial fermentation),
upfront $ 121m), to reach $ 270m in 03 (for Genz : 90/02; Durolane (bacterial, 1 single injection for OA, from
2Q03 : 29.6, 3Q 29.8, 4Q 26m; 1Q04 22.4, 2Q 27.5, 3Q Q-Med Sweden), ph III Anika
24.6, 4Q 15; FY 04 88.3, 1Q05 44 2Q 59, 3Q 57.5, FY 05
219: 1Q06 53, FY 06 234, 1Q07 54
GENZ SANGSTAT (SANG) -SangCya, MKT, oral solution bioequivalent to Neoral ,
withdrawn (bioavailability¬ w/apple juice). ⇑ discont
Settlement W/NOV does not concern capsules.
New strategy announced on oct 16/00 : -Gengraf (neoral capsules )FDA approved may 16, sales by N° 1 generic (16%MKT share, $29m/01, EON has
M efforts much more on Thymoglobulin than ABT disappointing (<$50m/Y), generic (Eon) price 11%)
on Gengraf pressures. For Europe, MCA application was to be ABT
withdrawn & replaced by new formulation mid-01. Sales
$ 15M in PIPE (June 22/01) 2002 $ 37m
-THYMOGLOBULIN(rabbit) : mkt, $ 60m/01; 80/02,
Dec 29/01, files for $ 100m secondary 2Q03 : 15.4 US + (EU + ROW = 9) 4Q 03 25; 1Q04 25,
2Q 25.3 (w/Lympho) 3Q 28.3. 4Q 30, FY 04 109, 1Q05
July 30/02 : Novartis seeking court order 27, 2Q 33.6, 3Q 32.4 , FY 05 128; 1Q06 33 , FY 06 150;
to have ABT withdraw Gengraf 1Q07 39Head to head Simulect (NOV) data March 15/02 :

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


stopped as efficacy x2.5, but open.
Aug 4 03, being acq by GENZ, Aug 13/04 $ -ABX-CBL(m anti CD 147), ph IIIGVHD, lic from ABGX
600m ; TERM FEB 17/03
-ABX-RB2(f hu anti CD 147), pre, from ABGX
Partner w/THP (Therapeutic Human -RDP-58 : small oral (not absorbed) peptide TNF synth. July 12/01 part w/Synt:em for 2nd generation RDP 58
Polyclonals). Rabbitt polyclonals. Are they inhib (& inhib IL12 & γ IFN), ph II ulcerative Lic to PG April 1/04 $ 10m upfront
human or humanized (CDR grafting) colitis April 9/03 > placebo; ph II Crohn (Oct 01)
?claim to have transgenic rabbit w/human April 9/03 < placebo. Also ph II in EU. CT
polyclonals. induced diarrhea kept by GENZ
But Hematech (acq by Kirin July 25/05) has
very good technique in cows (chromatin
transfer, also used by genetic Savings for
cloned cats) knock-out Ig genes in calves
fibroblasts, knock in human genes (with
HAC human artificial chromosomes vectors).
Other Hu polyclonal see Nabi.
See also AVIDIA, with avimers, proteins
with multiple binding sites
GENZ M ILEX ONCOLOGY (ILXO) -Campath(hz antiCD52), alemtuzumab IV inf lic to Schering AG (Berlex), JV w/LeukoSite (acq by
Deficiencies in Campath file noted by FDA; TIW→CLL,BLA filed fast track Dec 99(but June adv MLNM), lic ww to Schering Feb 5/03. March 13/03
response filed June 27/00→ODAC dec 14/00 comcancelled) , EU(03/01)OK for "use in exceptional ILXO gets all non K rights →MS ph II against Rebif
: OK by 14/1, the negative vote from FDA circumstances", FDA ditto May 7/01 (5 days/y) init Jan 03( rand, open, on "disability", N =
biometrics. This is an open trial in 93 pts (PR .(sales 01 around $ 30 m; 02 : 44m; 2Q03 : 28.3; 3Q 16.1, 240, fully enrolled April 22/04.. Suspended Sept 05 3
31%, CR 2%), with high rate of side-effects 4Q 18.4, FY 03 71.7, 1Q04 21.1, 2Q04 17.2, FY 06 42.7; cases ITP. 1st ph III vs Rebif Sept 21/07
& drug related deaths. 1Q07 18 Nov 9/05 ODAC to check commitments
Acq by GENZ Dec 22/04 Ph III N = 284 CLL 1st line vs chlorambucil. Enrolled June
23/04 ASCO 2004 & AACR March
Competition : - ILX 651, ph I, tubulin active pentapeptide analog of
- Cytarabine (araC, cytosine arabinoside), Dolostatin (anti K from sea hare), ph II melanoma init Oct
neuro hemato tox, 25% CR pediatric AML 8/03 (19 sites); HRPC init June 17/04, NSCLC clofarabine 3 ph II posters ASCO 2003 (28% ORR,
- Fludarabine (Fludara Schering), 13% CR, - NMR 3, oral anti VEGF N = 39, open label)
annual sales € 150m - Clofarex IV , Clolar (clofarabine CAFdA chloro- ASH 02 : active (MD Anderson) in multirelapsed
- Cladribine (Leustatin, arabino-fluoro deoxyadenosine), , from Southern Res ALL (17) & AML (8) children : 5CR & 3 PR→Ph II
chlorodeoxyadenosine), JNJ, only ind in Inst., lic ww to Bioenvision (EU & NASDAQ BIVN) who update at ASCO 04 : N = 40 ALL (10% ORR), 29
lic US to ILXO. ph II, AML. April 2/03 : EU Orphan AML (1 CR). Active in failures of fludarabine or
Hairy Cell. Limited activity in AML (ASCO cladribine
03) Drug designation. Oct 23/03 init rolling filing for
Lic to Serono & Ivax for MS (oral): Mylinax
Clofarabine & Cladribine have refractory pediatric ALL/AMLfinalized April 1/ 04, See BIVN
mitochondrial tox then delayed to add cases up to 84 (CR in AML still 0)
- Nelarabine (Novartis), ph III : 50% CR in .,Pivotal ph II of 40 ALL, 30 AML; ORR ALL 31% ODAC Dec 1/04 (9/6 OK ALL, 14/1 NO AML),
ALL ( ASH 03) (10CR), AML 26% ( 0 CR) But 50% of responders in PDUFA Dec 30/04
- ARRANON (GSK) approved Oct 04 ALL & AML were NOT refractory. Ph II AML "older 50 to 60% febrile neutropenia

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adults" N = 25, May 20/04 : 9/14 pts with drug alone have Median overall survival ALL 8w, AML 14w
CR. US launch May 14/05. FY 05 : 12.4, FY 06 17.3; FDA OK 3rd line pediatric ALL Dec 28/04
1Q07 4 (VION is going into ph III for refractory AML)
-Eflornithine(ornit.decarb.inhib)→bladderK,phIII, fails Jan
15/04
-ILX-295501→NSCLC, ph II
GENZ K BIOENVISION (BIVN, was BIV) - Clofarex EVOLTRA clofarabine for Europe (see ILXO
for US) has ww rights from Southern Res Instit., Filed EU
Secondary $ 60m Feb 3/05 to launch Clolar Aug 21/04. Pivotal ph II init EU AML, Aug 10/04. Aug 05
in EU responded to questions : EMEA action Oct 05. Oct 13
EMEA wants more safety data. Feb 23/06 OK 3rd line
June 07 : being acq by GENZ, acq Oct 21/07 pediatric ALL. EU OK May 31/06. AML ph II non rand
filed 2007, questions end May ?
US Clolar sales 3Q06 4m
AML adult data pts unsuitable for intensive CT ORR 67%,
Europ Hemat Assoc June 6/05
Clofarabine gel PSO ph I
- Modrenal (trilostane) approved UK refractory breast K,
ph II prostate init Sept 20/04
- Gossypol (from cotton plant), ph I, prostate, bladder
- Oligon, anti-infective catheter (Ag, Pt, C impregnated),
FDA approved.
GENZ AOM ANORMED (AOM/TSE & AMEX) - AMD 3100 Mozobil Plerixafor ,small molecule inhibitor 11 abstracts ASH 05 (Dec 10 – 13)
of binding of SDF 1 (stromal derived Factor 1) to CXCR4. Phase II data ASH 04 : > 6M stem cells/Kg of patient
Aug 31 06, GENZ bids $ 380m Potentiates release of CD 34. Ph II w/G-CSF signif, ASH weight in 2 or fewer aphereses : 70% vs 20%
Sept 26 06 MLNM bids $ 515 m 2004, ph III w/GSF vs GSF alone enrol end 06. Top line
Oct 18/06 GENZ OK 580 data 2Q06 MM, NHL. Filing 2Q07 to 2H07 (Oct 23/06). COMPETITION : PGG from Biothera, ph I/II
Aug 2/07 ph III in MM & NHL doubling of HSC collected
Good SAB - AMD 3100 Mozobil IND for heart attack filed April 3/05
offering closes Dec 8/05 - AMD 070, ph II, HIV, small molecule anti CXCR4
Licensor of Fosrenol to Shire chemokine receptor, HIV
- CCR5 HIV entry inhib, precl
April 18/06 : Onyx VP CMO elected to - CXCR4 inhib, precl
Board
June 06 : why did they spend $ 7 m to change - CXCR2 : Grosß (Growth regulated oncogene). Very
the Board
active mobilizer of stem cells (even CD34-), precl, but also
GENZ (Aug 1/07) estimates peak sales at $
400M) IL8 activator

- 68 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


GERN GERON (GERN) -Telomerase inhibition GRN 163 L (13 mer oligo)→K -PHA(discont jan 23), Kyowa Hakko
treatmt ph I
C 30 % cut in workforce (June 25/02) - (w/RZYM) GRN 163 thiophosphoramidate oligo to
inhibit telomerase, binds to its RNA component, CLL, ph
CEO Thomas Okarma II init 07/05
K In K cells Telomerase is abnormally active, - TVAX ph I/IImeta prostate; w/dendritic cells (Merix
and maintains lengths of telomere even after inc.) pulsed with mRNA that codes for hTERT (protein
cell division, allowing cell to proliferate component of telomerase) antitelomerase vaccine, w/MRK
indefinitely (July 18/05) -thanks to NUCLEAR TRANSFER technique acq 99
- Oncolytic virus (w/CEGE) E2F/hTERT, pre with Rosslin Bio-Med (the firm that cloned Dolly)
-Pluripotent stem cell differentiation→skin, brain, bone,
heart, liver,etc.(lic from Wisconsin univ, who is
disappointed Nov 01). Might not elicit immune rejection
- Derivation of new hESC lines
hESC give hematopoietic cells (June 05)., also for spinal
cord injuries (to file IND end 07, cardiomyocytes,
pancreatic islet cells, osteoblasts, chondrocytes
GGEN GALAGEN (GGEN) Neutraceuticals from bovine colostrum

- 69 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


GILD GILEAD (GILD) -Ambisome : $ 129m/99;141/00;167/01; 185/02, 1Q03 41.1 2Q 51.2 acquired Nexstar
3Q 49, 4Q 54.5, 1Q04 52, 3Q 49.8; 4Q 44.4; 1Q05 54.2, 2Q 56.2., 3Q
54.7; 1Q06 54
Pure growth story less nephrotoxic than Abelcet(ELN), approved for package
insert. 91% of EU mkt, 41% of US mkt ™w/PHA
-Daunoxome < $ 5m Tamiflu sales by Roche (10%royalty 1Q04 $ 27 1Q05 17,
Became profitable 2Q02 -Vistide cidofovir (→CMV):<$5m; very high doses mice pox ! 3Q07 77.4)
-Tamiflu FDA OK 4/99; Relenza (GLX) is mkt for Tmt in US & EU(GLX files sept
CPMP rejects (end May 00) refiled 27/2/01;OK 21/3/02 18/00, but"will not proceed" nov 30/00)
Poor pipeline prevention FDA OK nov 20
Children syrup FDA OK Dec 17/00. Manuf issue at Roche (Jan Tamiflu is leader in US
02). June 24/05 seeks to get back from Roche
Dec 2/02 : to merge w/Triangle (valued at $ Aztreonam-lysine ph III inhalation SIGNIF (Dec 20/06); 2nd
364m). Compl. Jan 23/03 ph III OK May 30/07, to file 4Q 2007 Filed FDA March 21/02 & CPMP March 26/02
- P-680 epithelial Na channel inhibitor, precl, CF 6 m priority review requested
Dec 15/02 : offering of $ 300 m, 5 year
-Adefovir→Aids; FDA adv Com : no(renal tox 60-500 mg/d) Early access programs init in France (07/01) & US (04/02)
convertibles -Adefovir/ Hepsera Hep B : ph III fully enrolled (lasts 2 years),endpoint ADV com Aug 6/02 all +; Approved Sept 20/02. CPMP
liver histol.EASL (april01)reduct viral load (p<0.001)in lamivudine could OK mid 03
resistant pts. 1st ph III signif v/placebo, no kidney tox 10mg/d (June
22/01). Efficacy in e+ & e- But rebounds. IQ03 5.8, 3Q 16.4, 4Q
April 12/06 GILD invests $ 25m in CORUS,
15.8,1Q04 18.9, 3Q 29.7, 4Q 34.2; 1Q05 43, 2Q 45.8, 3Q 46.9, 4Q 51.2;
July 20/06 : to acquire Corus, inhaled 1Q07 71; 3Q 79.3. May 07 loosing MKT to BMY (Baraclude, entecavir),
antibiotics GSK (Epivir), Epicept (Tyzeka)
GLX sponsors trial in pts failing Lamivudine

-Daptomycin lic from CBST for EU ph III : returned Sept 10/02

-Tenofovir, NRTI (Viread,PMPA),one pill once a day (Zerit is 2 a day), AIDS congress Glascow Nov 02
RTI→AIDS, no renal tox.. Sales 01$13m1Q02$27m, 2Q02 $ 44.7m,3Q $
Adv Panel Oct 3/01: mitigated OK (restricted or not to
68.9.FY 225, 1Q03 107; 2Q 167, 3Q 59, 4Q 177; FY 566 (40% in EU,
where retail price will drop), 1Q04 193 (internat 77), 2Q 197 (US 109), resistant pts ??). FDA OK no restrictions Oct 27/01. EU OK
3Q193.9 (US 108.5), 4Q 198.8; 1Q05 197.8, 3Q 189.4, 4Q 182.4; 1Q06 Feb 02, Feb 03 CPMP extends indications
192, 3Q 170, 4Q 159, 2Q07 155; 3Q 149 March 29/02 : FDA warns on false claims safety : cases of
Int Congr AIDS (July 0, 2Q 209.1 (US 88.4)2) equiv to BMY Zerit in lactic acidosis. Ditto Aug 03
triple assoc. (w 3TC & Sustiva)
June 7/07 phase III + in Hep B
- TRUVADA : Viread + Emtriva , QD combi, to compete w/
Combivir (BID) (Zidovudine + Lamivudine). Ph III init Aug contrary to worries, GSK Epivir/Ziagen : BOF !
11/03. Filed US/EU March 12/ 04. FDA OK AUG 3/04, sales 3Q compar. trial Combivir : non inferior (Aug 04), ICAAC
18.2 (inventory stocking) 4Q 43; 1Q05 91.2 , 2Q05 123, 3Q Oct 30/04 > to Combivir on viral load (p = 0.038)
162.4 , 4Q 191.1 (US 149); 1Q06 249, 3Q 06 308, 4Q 337 ,1Q07
345cannibalizing Viread & Emtriva (Dec 04) 2Q07 385 (EU
199).; 3Q 409.1 EU approval Feb 22/05, launch 1Q06 Ital, Fr,
Spain, Germ.
ATRIPLA package truvad + Sustiva.JV w/BMY Dec 20/04
Integrase inhib competition : MK 518 ph III (to file
(coformulation mises bioequivalence). Aug 10/05 initializing bilayer, to
file 2006. Triple formulation bioequivalent (Jan 9/06). NDA filed April 2007), Tibotec, JNJ (protease + NNRTI), Panacos
27/06, OK July 12/06, 3Q 06 68.4, 4Q 137; 1Q07 190, 2Q 212; 3Q 241 ("maturation inhibitor)
- GS 7340 prodrug of Tenofovir, ph I, term Oct 22/04
- 70 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- LB 84451, from LG Life Sciences (Korea), caspase
inhibitor, for hepatic & pulmonary fibrosis, ph II

GILD TRIANGLE PHARM (VIRS)


-Coactinon(emivirine, NNRTI),ph III,but FDA has
Workforce cut 35% (Aug 7/01) required additional trials,TERM Jan 20/02
-Emtriva Coviracil(emtricitabine,NRTI) 1 a day, Ph III was
Shelf filed Sept 23/02 delayed (liver tox, Efficacy> Zerit (July 31/02) but ABT
Merger (Dec 5/02) w/Gilead compl Jan terminates..Filing Sept 5/02, CPMP Jan 9/03. AIDS, Hep B
16/03 : $ 464 m deal. First cash tender $ 6 for ph III.PDUFA July 3/03 Approved July 2/03; CPMP OK
all outstanding VIRS stock (price $ 4.5). July 25/03. Sales 4Q03 $4m, 1Q04 12, 2Q 16.5, 3Q 16;
Interim financing $ 50 m with convertible 1Q05 12.4, 2Q05 : 12.1, 3Q 11.7, 4Q 11.2; 1Q06 10;
notes. 3Q07 6.5
-DAPD(NRTI), ph I/II
- Amdoxovir NRTI, ph II, AIDS. Term Jan 28/04
- Clevudine, nucleoside, ph II, hep B
MYOG Myogen (MYOG) -ENOXIMONE PDE III inhib,( like Milrinone) competition PAH : ENCY, Actelion, UTHR,
- IV in EU, MKT, Perfan ICOS/LLY, PFE (Rovitia), Flolan, Cotherix
IPO raises $ 70m - Oral, 4 ph III (other trials since 20 years increase death) Selective & once a day : Thelin & Ambrisentan
Emote : weaning of Milrinone; completed Feb 9/04, Bosentan & Thelin interact with warfarin;
March 28/04 miss endpoint Myogen claims Ambrisentan NO interaction w/
European subs (only Perfan) sold Feb 3/06 to Essential endpoints TT 1st CV hospitalization, 6 min walk warfarin
Wulfing GmBH I & II (US, EU) hospital use, compl Y May 04, June 27/05
: all endpoints fail. Drug abandoned. Tracleer increases catab of Warfarin, Thelin inhibits it
Oct 2/06 to be acq by GILD (Traceer sales Empower : tolerability of β-; Feb 18 : "enrolling slowly" ? ENCY (Thelin) to file April 05
1H06 : $ 339m) : $ 2.5B - Ambrisentan , LETAIRIS, oral ph II for PAH, ATS May
23/04 : signif 6 min. 2 Ph III (186 x 2)ongoing. Feb 4/05 : May 20/05 init trial in 36 pts who discontinue
6 m delay in enrolment for ARIES I (USenrol compl Nov Tracleer or Thelin because of transaminases, Feb
10/05, data mid 06), ARIES II in Europe on schedule 13/06 correction of PAH and
Price $ 3,940/month (like Tracleer) (enrol compl end June 05). Prelim data 6 m after end enrol.
Top line data EU dec 12/05 : 6 min walk 60m>placebo
normalization of transaminases
(Thelin only 30m), no hepatotox, no interference March 7/06 lic to GSK ex US (& MYOG will
w/warfarin), top line mkt Flolan in US, but ww rights to GSK), filed
Aries I April 10/06 12 w 6 min walking test signif, but 1Q07 EMEA
time to clinical worsening NS.. Filing Dec 18 2006. Could ARIES II data ATS (May 23/06) : T to clinical
obtain priority review, to be decided before Feb 18 (could worsening signif
then launch YE 07): yes Feb 18. Approved June 15/07;
3Q07 6M, $ 177 forecasted for 2008, strong switching
from Tracleer

- 71 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- Histone deacetylase inhib to block myocyte hypertrophy,
w/Novartis, Res
- Darusentan : hypertension, ph II, oral, ( lic from ABt,
who had flushing & edema). Positive data in last line
hypertension mid-August 05. Ph III init June 5/06

GLGC GENE LOGIC (GLGC) A gene expression database (GeneExpress) company, PG,
knowing which genes are expressed in which tissues, and JT,Akzo Nobel, AHP
when. Aventis, SBHMRK
Uses Affymetrix chips to create universal database of gene SGP,UCB
expression profiles Fujisawa
GLGS GlycoGenesys (GLGS), was SafeScience CGS-100, targets galectin-3, ph I/II, K, lic from Karmanos
& Wayne
GLIA M GLIATECH (GLIA) Adcon (surgical anti-adhesion gel):$27m/99, but CSF Aug 23 : FDA finds disturbing protocol violations
Delisted Jan 14/01 leakage! Could be relaunched outside US
Merger terminated by GLFD (aug 28)
-Perceptin (anti H3)→alzheimer, ph II JNJ discont, now ADHD ph II
Being audited by National Assoc. of Security -AntiProperdin f hu MAb→coronary, stroke (remarkable Lic to ABGX ( Nov 2/01)
Dealers molecule but nobody seems to realize),pre
Files for banckrupcy May 10/02
GLPG Galapagos (GLPG AIM, Euronext) Adenoviral screening technology

JV Tibotec Crucell -Osteoarthritis, discovery , June 7/6 € 144 m deal (access


fes € 4m milestones 140)
GNBT GENEREX (GNBT) Oralin, oral insulin spray, ph I/II, w/LLY from 2000 to
2003.
Ph III to init 2H07
GNLB GENELABS (GNLB) -GL 701/ Prestara (DHEA)lic to Watson→SLE, 2 ph III Genomic activity : positional cloning on long arm of 5
(1st reduct in steroid use; 2nd improvement of bone density :genes for IL13, 4, 5, 3 & GM-CSF
Oct 24/07 w/JNJ for RA $ 1B = 12 targets, of N = 381 but only 37 with complete density measures). Adv
which kinase GT418 Com April 19/01 : no answer!!June 27/01 : not approvable.
Sept 2/02 CRL : approval contingent on addition of new ph
III on bone density. Failure Oct 5/04
Jan 16/06 will init new ph III in LE
-GL 331 (Etoposide like), ph II ????
- Hepatitis E vaccine, w/GSK, Ph II
GNMX GENOMETRIX ( GNMX) Throughput genomic services

IPO filed 3/15 (LB/CHQ) withdrawn feb


28/01

- 72 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


GNOM GENOMICA (GNOM) "Discovery Manager" : annotated genomic data base

To be acq by EXEL
GNSC GENAISSANCE PHARM (GNSC) -Have proprietary haplotype markers for over 600 genes -Haplotypes are groups of SNPs easier to correlate
-Have already correlated response to Ventoline with with drug response than SNPs.
Aug 7/02 : staff¬ 20% specific markers. -Part w/Telik : compounds active on haplotypes of E2
-J. Drews is Chairman April 25/01 : init of Mednostics trials : receptors.
-1700 patent applications on haplotypes - pts are haplotyped for 50 genes, & randomly allocated to
Lipitor, Zocor, Pravachol or Baycol. Correlation w/safety-
Creating new markets : efficacy. Results 1Q02. Enrol compl July 01
-the non responders : using haplotype - JAN 16/02 : init of trial on Mevacor
prediction in a pilot trial they went from 8% - preparing trials in asthma, diabetes, schizophrenia,
respondrs to 44% obesity, long QT sd.
-the adverse effect type responders ALSO : drug development program to market a generic +
haplotyping kit
- for clozapine
- for a statin, ph II:

Partn. w/BGEN genotyping for Amevive w/JNJ : nov 28/00; w PFE Oct 9/01; w/AZN; wBGEN
- (Feb ½)
GNSL GENOMIC SOLUTIONS (GNSL) Genomics toolbox MRK, CAT
IPO May 5 Perkin Elmer
GNT GENTIUM (GNT) - Sulfated fucans
- Defibrotide. Weak fibrinolyticfrom porcine mucus.
1,200 allo BMT in France 2005, 6,000 EU, Venous occlusive disease in BMT. THerapeutic ph III init
12,000 ww June 06 , Nov 5/07 65/80 enrolled, primary changed
w/FDA from survival 100 d to bilirub < 2 mg/dl; data
1H08, large prophylactic ph II ongoing in Europe. "Bone
marrow transplantation" May 14/07 paper Royal Marsden,
58 patients with allo: no VOD after 100 days ( litterature
20 to 30%!). June 07 positive data in MM.
July 07 Int Soc Hemost : enhances plasmin activity
GNTA K GENTA (GNTA) -G 3139 : Genasense –oblimersen
Bcl Competition : Gemin X Biotechnologies -antisense to Bcl2→K, , ph II, many centers, tested with FDA (Aug 7) : orphan drug design., fast track. Rolling
Inc. DTIC, Taxol, Taxotere,Rituxan,Camptosar, Fludara. filing was to start 1H/02). Delay(May 16/02)Init Sept
May 13/04 slashes employees 50% Ph III stage III/IV, 1st line, open, random: melanoma 03, completed Dec 8. FDA accepts + priority Feb
April 12/04 files shelf $ 150m initiated March 9/00 (enrol compl 750 Jan 03),myeloma 6/04. Action before June 8 ,Adv Com MAY 3 : 13/3
feb 01( 200 enrol compl 4Q02), CLL march 01 (enrol to NO. May 13/04 GNTA withdraws
Aventis partner (April 29/02)$ 477 : 135m compl end 1Q03). AML ph II; NSCLC init w/taxotere Nov Gnta will NOT supplement the application with
initial & near term payments (of which 75 equity if a 5/01, prostate :ph II. SCLC (Feb 02 w/carboplat + etop) complete 12 m survival data, unless requested. By
milestone is reached), 40 dev fees, 10 convertible debt,
10 lic fee, 280 cash, 65 convertible notes based on
same at AACR Nov 02 : 82% ORR, NHL (Feb 18/02) April 8 will supply 8 m safety data
- 73 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


milestones. March 17/02 advance credit line $ 40m w/Rituxan.
Royalty 25% US, 18% EU → ASH 01( but results of myeloma IIb 1H02) for
Terminated Nov 8/04
Myeloma : + thalomid + dexa init Sept 24/02 CLL (severe IL8 side effects) & AML (w/Mylotarg).
Myeloma + Velcade AACR (Oct 29/01)
PIPE $ 28 M (Nov 28/01) CML + Gleevec, init Oct 2/02
Breast w/Taxotere + doxo → ASH 02 CLL (fast track JUne 30/03)
June 11/02 : $ 5m stock repurchase ASCO 03 : w/Campath (alemtuzumab) potentiates
RCC, ph II, + α IFN
Pancreatic K, ph I/II w/Gemzar HRPC w/Taxotere ph II, N = 29, will start ph II
Manuf : Avecia EORTC
Colorectal ph II w/Eloxatin+ 5FU
Sept 10/03 Melanoma stage III & IV NSCLC w/Vinorelbine (GSK) + precl at AACR April 03
Solid T ph II w/carboplat + paclitaxel CLL July 05 rolling NDA init. 2nd line
- all pts survival 9.1m/7.9 NS w/Fludarabine + Cyclo, completed Dec 29/05.
- 480 first 10.1/8.1 p = 0.035 AACR April 05 + Iressa xenograft NSCLC interesting
- new antisense lic from Atugen AG (jan 01) PDUFA Oct 28/06 ; ODAC Sept 6/06 7/3 against,
- RECIST on all : + 11.7%, p = 0.019 endpoints complete or nodular (C but with
Sept 06 (JCO) vs dacarbazine N = 771, ph - Ganite : Gallium nitrate, NHL & K hypercal, ph I/II,
sNDA for K hypercalcemia filed April 1/03. 1Q04 : 0.4m. persistent lymphoid nodes in BM) 17% vs 7% (p =
III, ORR 13.5% vs 7.5% PFS 2.6m vs 1.6 m O.O25). But ORR 41% vs 45%, and Toxicity. July
May 10/05 Aventis terminates Promotion stopped May 10/04
05 letter of intent EU filing 1st line melanoma
Sept 19/05 CLL better ORR than placebo, w/Dacarbazine, filed Jan 3/06. Ph III melanoma
but twice as many deaths Paris Cancer Congress Jan 06. Dec 15/06 CLL
"not approvable".
Competition : Bcl2 inhibs, Geminx Biotech,
Abbott, Santaris, Ascenta
GNVC GenVec (GNVC) GENE THERAPY
K - Ad VEGF(BioBypass), ph II, coronary & PAD, AHA Nov 02 , ph II, N
= 71, signif on ST, IIb init March 1/05 (EU, Israel) w/Cordis (JNJ). PAD →WLA/PFE (discon jan 02); Jan 04 : JNJ
term Jan 03.
traded since dec 12/00
- Ad TNF w Rxinducible promoter (TNFerade) : ASCO May 02 N=22, CR 5, PR 7 All K
April 16/03 to merge w/Diacrin, completed intratumoral ph I (Nov/01 ASTRO) + Rx : 2/7 CR, ASCO May 03 pancreas N = 20, ORR = 85%
Aug 22/03 2/7/PR,pancreas ph I ASCO Jan 24/04 GI sympo "promising efficacy" Nice
TNFerade could sell at $ 25,000 (2007) Ph IIb QW/5w then Gemzar Tarceva, rand Pancreas 1st line pictures. CA-19-9 is suppressed by 70%.. DDW May
C N=150 init Aug 14/02 (+Rx + 5FU), enlarged March 16/06 17/04, N = 37, good data
to N = 330, endpoint % patients with 12m survival. Dec Sept 1/04 data in Clin Cancer Res. N = 13, 2 CR, 9
19/06 interim on 51 pats : at 12m 70.5% vs 28%. PR
Oesophageal PACT ph II/III init Nov 14/02. Maintenance AACR Oct 3/04 good data esophageal
with Gemzar/Tarceva Oct 7/04 : on hold FDA (clots in esophagus K), hold
Ph II ASCO 04 : median survival dose dependent > 340 released Feb 11/05 for pancreas, for all May 25/05
days; ASCO 03 : sarcoma N = 14, ORR = 85%
-rectal ph II
- melanoma ph II
- Soft tissue sarcoma :EORTC/NCI/AACR Nov 19-22/02,
N = 14 w/Rxtherapy, ph I/II, interim on 7 : 2CR, 3PR
__________________________________

- 74 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- Ad PEDF (pigment epithelial derived factor), ph I init
DIACRIN (DCRN) Nov 26/02. Data Amer Acad Opht Nov 03 +/-
- Adeno vector for SARS vaccine (April 25/03)
Merged (April 18/03) w/GNVC, compl Aug - Adeno vector & MATH1 gene for deafness
22/03 ______________________________________________
-porcin neurons, stroke,phI : on hold
-NeuroCell, Park, ph II (w/Genzyme)extended to 18
months (sept 15) to maximise efficacy data∏march 18/01:
results unclear
-Hepatocytes for hepatic failure ,ph I
-AutoTransplant of muscle cells to heart,phI. 3 year
follow up OK March 05 ACC. To be divested (Oct 18/05)
Dec 29/05 sold to Mytogen
GPCB GPC Biotherapeutics AG (GPCB) nd in 1st line TTP (N = 50) 5.2m/2.5m; OS 14.9/11.9
K - Satraplatin ph III, 2 line oral HRPC N = 912
endpoint TTP (from Spectrum), + Pred vs Pred alone Satraplanin lic to Pharmion for EU (Dec 05)
(Taxotere was approved 1st line). Rolling NDA (CMC) init
May 06 : data monitoring board recommends Dec 05 (was planned for 2H06). Full enrol. Dec 5/05. 2nd line median OS w/Taxane 10.7 m (ASCO Prostate
interim anal of OS Interim delayed from <end 03 to end 04/06. Final results 2005)
3Q06. Roll. filing init July 12/06. Sept 24/06 PFS 11 w, p
June 25/07 lic to Yakult (inventor of < 0.00001. Completed Feb 16/07. Asco prostate 07 11.1
irinotecan) for Japan, $ 10 m upfront w vs 9.7 (median), but at 12 m 16% satra had not
progressed vs 7% control. Free expanded accessFeb
21/07. Adv Com July 24/07, PDUFA Aug 15/07. ASCO
07 : OS not yet; is Pred the right comparator ??? ODAC
July 24 : NO. OS required. July 30 filing pulled.
Init w/Taxotere ph I Dec 05, ph II w Taxol in NSCLC, &
w/ tarceva
- 1D09C3, ph I, anti MHC class II (HLA-DR) fully human
(ASH 2004)
GPRO GenProbe (GPRO) Nuclear acid probes (hybridization) : Competitors : PCR (Roche, Becton Dickinson)
Key customer : Chiron (47% 2004 revenue) - HIV, HCV, West Nile, Chlamydia, Gonorrhea, TB Their use of amplification depends on license from
Vysis acq 2001 by ABT
GSK ID Biomedical (IDBE) - FluInsure : non living intanasal vaccine. Clinical efficacy
(July 20/04)
acquired Shire vaccine business - Fluviral, marketed in CND since 1975
- Fluviral 2nd version :
Sept 7/05 to be acq by GSK $ 1.4B CND trial vs Vaxigrip completed Jan 05
US trial to initiate end Jan 05, vs Fluzone (Aventis) IM
GSK PRAECIS (PRCS) -Abarelix/Plenaxis,prostate K, ph III,LHRH antag; Amgen (discont Sept 20/01), SASI
Lupron/TAP & Zoladex/AZN are agonists. June 12/01 : ASCO 00 : only hi data
PDUFA AUG 27/03, OK Dec 1/03 ASCO /01:only marginal differences w/Agonists
- 75 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


for "pain relief in pts who are not viable
rejected by FDA; meeting Sept 10/01→to investigate
candidates for treatment with LHRH agonists, and April 28/04 lic to Schering AG
refuse castration". Launch Jan 04. 1Q04 : $ 0.4m allergies & testosterone resurge after 3 months. Will
Dec 21/06 being acq by GSK resubmit end 02 : refiles Feb 26/03. Will file CPMP
Endometriosis ph II/III. Promotion suspended May 20/05
- Latranal, back pain, ph II. Terminated.
-Apan for Alzheimer, Ph I
- PPI 2458, ph I, Inhib of methionine aminopeptidase 2.
GSK CORIXA (CRXA) - Ribi 529 lic to AHP (Oct 30/01) will get Ribi's royalties for new Engerix (from 2002)
June 9/03 PIPE offering $ 30m May 24/02 lic to MEDX of toxins for MAbs
-Melacine(lys.from 2 hu cell lines)→melanoma,phIII -from Ribi acquisition
convertibles $ 75m
M improves survival in women with αIFN.. part : SGP
March 19/02 : breast K genes for dg lic to JNJ (BRCA
SWOG ph III (oct 30/00):dis. free surv. signif improved (p<0.005), in Approved CND (feb 16/01)
sales $ 5M by MYGN), CVSN sells Her 2FISH.
V HLA-A2 and C3 pts (60% of pts). FDA (Nov 01) wants 2nd ph III : meeting on Feb 27/02 : OK for
CEO : Steve Gillis
Melacine V nothing in A2 & C3. Nov 03 discont in US.
-her2/Neu vaccine in PLGA microspheres→K, ph I→→ →GSK (reneg Jan 30/03) CRXA pays Dev, but JV possible
K Very high expenditures: $150m/Y (Had 510 employees,
PDLI has only 60) : March 20/01 10 % staff cut. May -MPL genital herpes ph III →GSK
28/02 down to 375 MPL Egerix B to be filed → GSK
$75m line of credit from BNY (Dec 5/01) - TB vaccine, pre
Aug 13/02 : $ 45m PIPE -MUC 1 peptide vaccine : pancreatic K, ph I

BEXXAR : use of this or that RAIT for NHL will -PVAC(mycobact bov. Ag)→PSO, ph II;feb 15/01 p=0.08, but end point lic from SR Pharma (UK)
depend on variable radioresistance of pts, synergy or not too strict;march 12/01 SR Pharma reports + ph II in atopic w/Medicis (CND/USA), Zenyaki Kogyo for Japan
with ABMT, possibility of lower dose of MAb with *I, dermatitis.PhIII∏3Q01.Peak PASI 75 : 50%. New US ph III init June Trials in Brazil∏ Nov 6/02 PASI 50 p = 0.053; 75 NS
size of tumor mass (*Y, with high energy, might be 6/02 Trial init in N Zealand Nov 7/02
preferred for large tumor mass, and *I for more
disseminated masses). -High capacity for providing Ags
→to ABGX(21/03/00), MEDX(June 7/00),Purdue sept 26/00
Competition Zevalin (IDPH), also *Y Lymphocyde - Proteins that act as Ags, and could protect against acne (April 5/01),
(anti CD22,ph I/II) IMMU/AMGN,& *I Oncolym (ph genome deciphering contracted out to Genset
II/III) PPHM/Schering AG -Anergen products :Anergix RA,ph I/II (w/Organon)
Anergix Myasthenia lic WW to Beaufour (Jan 02)
CRXA owns US patents to Rx Mabs to CD 20
Oct 29/01 : Amersham for EU +++++; to file cPMP 2H03 (acq by
ASH 01 : 13 papers for Bexxar & 13 for Zevalin. Very -COULTER PRODUCTS
GE Oct 10/03)
prestigious authors for both. For B. more than 800 pts !, -Bexxar(radio-I m anti CD 20 MAb) tositumomab→NHL
&76 first line ++. 2 phase III (N = 60 + 273, ASCO 01)
BLA refiled sept 18/00; GSK,USpartner (50/50 profit sharing),
For Z issues of *Rx contaminat. of gut (Indium Jul 26/01: + CHOP trial with GSK
strong patents (Versus/Idec); oct 5/00 :priority review granted
insufficient to detect) ∗ risk for family (feces), March 19/01 FDA asks for hematol. recovery data in low grade, thyroid
-April25/00 : CLTR regains rights outside US
heart, testes. safety & comparison of manufacturers(CLTR,Lonza,BI). Chem/Pharm
NO NEED FOR INDIUM IMAGING
Bexxar manuf : B.I (like Synagis). , then in US R* filed Aug 9/01,completed Sept 7/01
March 12/02 : CRL March 25 CRXA informs FDA intent to amend
labeling/ fill & finish by MDS Nordion , in EU SWOG is running first line BEXXAR
BLA,should be May 15:diff of opinion CRXA requests dispute
Amersham. April 2/02 survey 1000 Hem/onc : most promising new agent resolution by ODAC (FDA to respond in 45 d). Safety does not
May 23/02: GSK press release says launch 02/03
seem issue. CRXA will provide info on "level of durable response"
- Clinical benefit in Rituxan refractory JCO Aug 15/02 : Bexxar & Zevalin equivalent once request is accepted. Request accepted June 27/02(and
10/3
Enrolling (Feb 03) SWOG Rituxan + CHOP vs Bexxar + CHOP withdrawn Dec 2/02. → files Oct 02 for "low
- ORR is likely to predict clinical, benefit grade NHL that does not respond or progress
in Rituxan Refractory 13/13. Feb 24/03 : European orphan Drug Designation → patent protection 10
June 30/03 Bexxar approved for Rituxan following Rituxan"; ODAC Dec 17-18/02 FDA
more years
refractory, sales init Aug 1/03 ASCO 03 5 presentations. # 2316, N = 250 follow-up median duration confirms (Dec 2/02) that "this constitutes

- 76 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Sept 22/03 recruit Head of Sales Chris Rivera complete response (30.4%) : 58.1m. Some pts now treated 9.5 years. substantiated new info addressing prior concerns";
(who was in charge of Renagel) these data include durability of response. PDUFA
Dec 14/04 Bexxar lic to GSK -prodrug of doxorubicin, Pre May 2/03→ Aug 1/03 (no new trials required)
May 2/05 being acq by GSK - INF α receptor antag : res
-April 3/00 : MEDX for fully Hu MAbs
Nov 10/03 : concentrating on TLR4 agon & antag. -May 21/01 : agreement w/Biovation (Merck KGaA) for
deimmunization
-March 02 w/Synt:em for vaccines

GTCB M GTC Biotherapeutics ( GTCB), was -AT III,ATryn, filing end 00 delayed to 2002 !, filed second ph III bleeding issue, discont Feb7/01
Genzyme Transgenics (GZTC) CPMP March 04, March 16/05 : more questions. GTW
will respond by JUly 8, Sept 15/05 time line for CHMP
May 15/01 CEO leaves, Geraghty takes over response delayed from Oct 05 to Feb 06, June 06 CHMP
interim, now Geoffrey Cox OK for cong def in surgery; 2nd ph III for FDA (N = 17)
init April 6/05. Sept 6/07 FDA grants fast track. Nov
Secondary Jan 24/05 1/06 lic to Leo for ROW (except Japan). Leo also in
DIC. Aug 6/07 ph II init by Leo in sepsis DIC, N = 200,
Other transgenic : Merrimack results 2H08
-Transgenic Remicade, PRE Centocor (JNJ )extended to 2nd MAb
-Transg Hu anti TNF alpha→RA, pre BASF/KNOLL™CAT/ABT/ELN
-Transg MAb (must be anti C'), pre Alexion
- Trans Hu Albumin as stabilizer (w/Fresenius)
- anti CD 137, K, pre, agonistic, stimulates immune
response
- MM-093 (w/ Merrimack) : r hu AFP, immunomodulator,
ph II, RA, PSO
- Transgenic anti-CD 20, w/LFB
GTOP GENITOPE (GTOP) MyVax, NHL, ph III (auto + allo vaccine + GM CSF +
IPO filed Aug 7/03 (WR Hambrechst + Punk keyhole limpet hemocyanin) July 31/06 misses endpoint
Ziegel). Traded
GVEC GENETIC VECTORS (GVEC) tests to identify DNA & RNA impurities

HALO Halozyme Therapeutics (was HTI), - Chemophase (recombinant hyaluronidase rHu PH20) to
enhance chemotherapy delivery, w/ Mitomycine,
traded on Nasdaq since May 10/07 superficial bladder K, Ph I/II init April 25/06
- r Hu PH20 associated w/ 13 Roche targets (monoclonals
?), Dec 6/06; lic to Baxter Feb 14/07 for 2 molecules
- rHu Hyaluronidase (Hylenex, Enhanze) : FDA OK Dec
05, launch 2Q07
- July 10/07 good data w/Humira (ABT)
- Sept 10/07 new deal w/ Baxter subcut extractive
immunoglobulin
- 77 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS

HBIO Harvard Bioscience (HBIO) Enabling tools for drug discovery. 10 000 products, 60
Exists since 1901!!!! countries, 1 000 page catalog.
trading since dec 7/00
HEB HEMISPHEREX (HEB) Ampligen NDA filed Oct 11/07 for chronic fatigue sd; binds to
TLR3
HEPH HOLLIS-EDEN (HEPH) Bioterrorism drugs to protect against radiation

HGSI M HUMAN GENOME SCIENCES (HGSI) Genomics :-patent applications for medical uses of more than
7400 genes .→→→→→→→→ SBH/GSK*,MRK,SGP,SASY Merck KGaG, Takeda
March 25/04 lay off 20% -collab with Compugen on cSNPs ("collection of SNP expressed "to achieve the first chromosomal map of expressed human
K genes")→→→ genes"
.-SB435495 (pyrimidone)lowers Lp-PLA2(Paf acetyl GSK(10% royalties + 20% copromotion right ∏ 30%
Interest income 01($77m)> revenues ($12m)
hydrolase), enters clinicals -feb 22/01), enters ph III in 04 profit), named by JP Garnier 480848 in Feb 03 R/D
* on june 30/01 HGSI regaineds all rights - SB462795 (cathepsin K inhib) osteoporosis, phI
on GSK genomic agreement Proteins : GSK
-KGF 2 (repifermin)→venous ulcers, mucositis, ,ph II : Dec
10/01 : no efficacy in ulcerative colitis. Interim anal. (Sept 24,03) leads to drop venous. ph II
-MPIF (myeloid progenit inhib fact)mirostipen,ph II ongoing in mucositis, fails Feb 3/04
-inhib of CCR 5 receptor (metchemokine beta3), blocks
allergic reactions.
Strong GMP manufacturing capacity, - BLyS protein : ph I, for "common variable immunodeficiency
including for MAbs disease"
- Albumin fusion products (technol acq from Principia/Aventis), SGP (oct 9/00)
like PEG, but antigenicity ??? (but no issues in the 1st 50 pts) All pts make Abs, but not neutralizing Higher discont
-new Interferon (Albuferon)⇑Hep C,phI init March 01; rate than Pegasys
Nov 4/02 : safe, longer T1/2; ph II AASLD Nov 05 can be given
- 78 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


every 2 w, but 4w inferior, Pegasys is every w. Partner 06 ?? EASL Vienna April 26-30/06 12w data
yes June 6/06 Novartis. June 7/07 ph IIb not better than
Pegays (more side effects). PH III init 1Q07 AASLD Nov 02 : works in INF α resistance
- Albutropin, ph I, HGH
- Albuleukin (IL2) ph I, Jan 8/02 Partn w/Atrix for protein delivery (Aug 01)
-owns 10% of Transgene -also PTH, INFβ , G-CSF (albugranin), EPO (albupoietin),
- Off balance sheet lease financing will have GLP (resistant to dipeptidase : Albugon GSK 716155) lic to Alliance with CAT, DYAX, MEDX, ABGX
to be recognized as liability $ 500m in 03 GSK Oct 27/04, IND filed Jan 10/06, ph I to init 06
- Convertible debt 2007 GSK opts OK July 05
- Not profitable until 2010 MONOCLONALS (phage display) →→→→→→→→
-Lymphostat (belimumab): Fully hu anti BLys (B Lymphocyte stimul): SLE fast track April 21/03, 0.25 or 10 mg/kg every 4 weeks
I, for RA (where this prot is overproduced), SLE ph I,+ April 21/03, ph II
init Sept 26/03, decreases anti DNA. RA ph II N = 283 signif April 6/05.
ww SLE 300,000 (Morgan Stanley) Oct 6/05 SLE data : only active(clinically) in pts with
anti DNA (75%). Trail is used as a drug by AMGN/IMNX, but does not work
Eular June 07 good ph II SLE due to decoy receptorsFor Japan Trailr1→ Takeda, 2 →
1st Ph III LE w/GSK init Feb 12/07, 2nd init May 30/07, Kirin
both ph III only sero +. Enrol 1 year . 2 X 810 pts (ser+) CRC ECCO 05 ph II CRC SD 30%, NHL ASH 05 : clinical
response in 3/40 pts !!
vs placebo, endpoint SELENA 12 m. On MKT 2009 ,
Competitor ZGEN
now (feb 07) filing 2010….
ACR Nov 06 in seropositive primary efficacy endpoint
46% ptts w 52, 56% w 76 JV With Vascular Genetics, now Autus (HGSI has 10%)
- LymphoRad : *I anti Blys, pre, NHL, myeloma ph I
- HGS-ETR1, anti DR4 (death receptor 4) TRAIL, mapatumumab
Hep C : see Idenix !!
IV(TNF related apoptosis inducing ligand)R1 Agonistic Mab from CAT ,
K, synergistic topotecan, ph II CRC enrol compl Feb 22/05, NHL compl
March 3/05, OK June 13/05. GSK opts OK Aug 05
- TRAILR2, I
-VEGF2 Mab, K, pre
- CCR5 Mab, AIDS,ph I (init March 05), acq from ABGX
-C2A Mab, asthma, pre
- ABthrax, Ph I, 100% survival in animals; May 04 : back burner

GENE THERAPY→→→→→→→→→→
-VEGF 2 →PAD & coronary ischemia , ph I/II (on hold, 1
death)→→→→ (Jeff Isner died )
HMSL HEMOSOL (TSE/HML;HMSL/Nasdaq) Hemolink (purified Hb from outdated bank blood) ph III, ™Dompe (south & east Europe)
filed in UK feb 01
June 7/02 : cuts 35 % of work force July 01 : files answers to queries of Health CND. Decision
end 01→ March 21/02 : refusal
April 7/03 staff total lay off Aug 14/01 : FDA requires modif ph III protocol; March
28/02 FDA allows ph II. Delays in trials enrollment
(CABG). March 13/03 enrol suspended.

- 79 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


HNAB K HANA Biosciences (HBX) - Zensana Ondansetron oral spray, filed July 06,launch 07;
GSK threat lawsuit. March 24/07 filing withdrawn
(manuf). Aug 1/07 lic to Par
- Talotrexin, Talvesta, antifolate, ph II NSCLC (2nd line),
N = 120 , ALL ph I/II; discont March 24/07; could come
back (Aug 1/07)
- IPdR, ph I, digestive K
- Marqibo (sphingosomal VCR) from Inex (March 17/06).
dec 04 ODAC : NO; new ph III ALL to init YE 07, SPA
refused (March 24/07). Aug 2/07 ph II 2nd line ALL
- Sphingosomal vinorelbine (ditto) phase II NSCLC
- Sphingosomal topotecan (ditto), pre
- Vit K3 percut lic in Oct 16/06 for anti EGFR eruptions
HYBD HYCOR BIOMEDICAL (HYBD) kits for urinalysis & allergy testing

HYSQ HYSEQ (HYSQ) -Genetic testing with proprietary arrays


-Genomic databases : cardiovasc, metabolic→→→→ CHIR, AHP, Kirin
Chairman : G. Rathmann -Downstream proteins :
Nov 11/02, merging w/Variagenics -IL1Hy1,pre, IL1Hy2,res:antiinflammation
July 26/01 : 20% reduct ow work force -DDF (CD39 L2) antithrombotic,pre
- Alfimeprase, thrombolytic, lic from AMGN
IART INTEGRA (IART), -products for neurosurgeons
Soros owns 15% - artificial skin (NaOH treated bovine collagen): MKT™ ™JNJ
IBPI IntraBiotics ( IBPI) -Ramoplanin (oral AB),ph III, suppresses intestinal VRE ; Lic from Biosearch Italia (Nuevo Mercato), also
trial in BMT to prevent febrile neutropenia events. developing it; manuf by Aventis
Was to be completed 1Q01,NDA filing delayed (march May 31/01 : nearly discontinued
April 11/01 : Deutsche Bank suspends 12/01) at least 1 year: only 104 of planned 900 pts OCT 10/01 : lic to GENE by Biosearch I.
coverage enrolled. But ph II had efficacy in 90 % of pts⇑⇑⇑⇑

Ernest Mario, CEO since April 25/02 (was -Protegrins (peptides) : aerosol CF, ph I
CEO of Alza). Steps aside Feb 03, becomes Iseganan mouth wash(mucositis) ph III
Chairman, invests dispensing error by CRO! FDA ™ fast track (jan 01) ASCO 01 : end point missed, but pain reduction in
April 26/01 : end point missed. 19% of pts
Staff ¬ 70% Oct 14/02 Oct 4/01 : ph II + p<0.01;Nov 7/01:new phIII in BMT May 6/02 : Rxtherapy trial : failure
(fully enrolled June 6/02, N=509) ; NS 27/9/02
Being Terminated : June 21/05 Feb 10/03 could launch ph II/III in prevention of ventilator -DVSA partnership modif (July 2/01)
associated pneumonia, N = 900 X 2, 1st initiated Sept 03,
450 enrolled May 5/04.. Fast track Sept 5/03. Feb 19/04
selected by FDA for Continuous Mkt Application Pilot 2
program (by anti-infectives division). 1st trial halted June
23 by Safety Com (more deaths)
- 80 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ICCC IMMUCELL (ICCC) Bovine health

ICLL INTERCELL (Vienna) vaccines HEP C many groups w POC in chimps using adeno
- HEP C therapeutic peptide vaccine (T cell response ?), ph vaccines; Transgene MVA in ph I
July 2/07 Novartis rises equity stake from II
6.1% to 16.2% for $ 270m (of which 170 - Jap. Encephalitis, ph III, w/Novartis Jap encephalirtis competition Acambis
licensing rights) - TB, ph I
ICGN ICAGEN (ICGN) oral molecules to modulate ion channels (have cloned
entire ion channel genome)
Feb 3/05 - Aug 14/07 $ 1B deal w/ PFE on pain

ICA 17043 senicapoc (W/JNJ) ph III init Feb 22/05 N =


300 ASSERT endpoint rate of crises : sickle cell disease
(active on transgenic mice red cell dehydration). Ph II
ASH 04 12 w change of Hb from baseline p < 0.001,
Orphan Drug. On hold April 4/07. JNJ terminates June
23/07
ICN ICN (ICN) To spin off " Ribapharm" (IPO filed) :
- Ribavirin (MKT by SGP)
June 11/02 : Milan Panic retires - Tiazole for CML, ph I/II
June 20/02 acq Genetronics electroporat. - Adenazole, colon K, ph I
IDDS Innov. Drug Delivery Syst. (IDDS) Ketamine nasal ph II
IPO filed (JPM) Jan 2/02 Morphine nasal ph II
IDEV INDEVUS (IDIC) was INTERNEURON -Redux Suing AHP for non disclosure!And won !!h
-Pagoclone→panic/anxiety : ph III w/PFE, returned June 10/02
-Citicoline→stroke, ph III??
- Sanctura, Trospium (lic from Madaus), hyperactive
bladder, ph III OK Sept 25/02. FDA OK Aug 04
- Valstar (valrubicin) bladder K. Approvable Aug 17/07
IDIX IDENIX, was NOVIRIO (IDIX) Telbivudine (LdT) Sebivo / Tyzeka oral, once a day, hep
B, ph III init May 03 (FDA OK Oct 06). EMEA approval
Oct 1/07 outs 1/3rd work force April 30/07. Lic to Novartis
Veltorcitabine (Val LdC), Hep B
57% Novartis NM 283, Valopicitabine, nucleoside analog, Hep C, ph
I/II, lic to Novartis (March 29/06) $ 525m. Nov 06 Valo +
Filed for IPO April 15/02 (Goldman PegIFN : 4.2 log reduction !!). July 15/07 on hold : GI side
Sachs).Withdrawn April 15/02. Refiled Dec effects
15/03 5M. Stanley, Bear Sterns) - IDX 8999 NNRT hiv PH i:ii
Chairman CEO JP Somadossi; major
investors Nomura, MPM Boston. Has $ 52m
in cash (BWT Dec 16/03).
- 81 - printed : 03/07/10 (d/m/y) 08:07
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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Refiled Dec 03. OK July 22/04

IDMI c EPIMMUNE (EPMN) was Cytel complex carbohydrates


Sept 13 : CEO resigns
K E. Loria, from Biovector SA appointed - HIV vaccine ph I/II; sold to Pharmex A/S Nov 28/05
March 15/05 : merger w/IDM, and be IDMI
(IDM Pharma Inc) compl Aug 16/05
IDMI IDM (Immuno-designed molecules)*, France -The MAK (Macrophage activated killer ) is a simple ongoing ph III with MDX 210 (partnership w/MEDX)
M Part. SASY (20 options) Jan 22/02 device for autologous immunotherapy with MAK cells. in ovary cancer; this is anti her2neu.
forthcoming IPO These are monocytes activated by γinterferon in-vitro
V oct 23/00 : MEDX increases stake to 34% - BexidemVaccine therapy for bladder K, ph II/III

Have 10 cell processing centres (1 in - MEPACT / Junovan , ph III, osteosarcoma N = 800, 4 agreement w/Epimmune Oct 14/02
C years, completed, to file 2006, IV, liposomal muramyl
Montreal)
tripeptide phosphatidylethanolamine to target Competition : ZIO 201 active metabolite ifosfamide,
May 30/07 JL Romet Lemonne leaves macrophages. NDA filed Oct 26/06 : 6 year probability of from Ziopharm Oncology
survival 77% vs 66%. ODAC May 9/07: NO 12/2; EU
filed Nov 7/06. DFS is not > control. FDA Aug 27/07 NOT
approvable

dendritic cells derived from the patient's WBC, exposed to


Ags, reinjected :
- Uvidem IDDD3 (w/SASY) melanoma, ph II (+IL13).
ASCO 07 N = 30, 1CR, 2PR, 6SD
- Collidem, ph I/II CRC
IDP K IDERA - IMO 2055 ph II, RCC, solid T Ph I w/Gemzar Carbo
- IMO 2125 Hep C, pre
Dec 11/06 w/MRK $ 455m TLR 7/8/9
June 2005 Novartis $ 136 m TLR 9 asthma,

IDXX IDEXX (IDXX) Veterinary diagnostic kits

IEX K INEX (TSE/IEX) - Onco TSE (liposomal VCR Marqibo) w/ELN, ph II/III ODAC Dec 1/04 : NO
NHL
partnership w/ALNY Jan 9/07 - TCS (transmembrane carrier system, liposomes) lic to
GSK for Camptothecin
IGEN IGEN (IGEN) The key provider of electrochemoluminescence kits Roche dg, Organon teknica, Eisai, Bayer dg.

ILGN INTERLEUKIN GENETICS (ILGN) tests for susceptibility to disease : diabetes, cardiovascular,
osteoporosis, being developed

- 82 - printed : 03/07/10 (d/m/y) 08:07


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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ILMN ILLUMINA (ILMN) Bead arrays for large scale analysis of gene variations, for
SNP genotyping
IMCL M IMCLONE (IMCL) -Cetuximab/Erbitux(C225, ch antiEGFr IGg1) IV QW Merck KGaA (8-12% royalty, coexclusive for Japan)
→K, ph III, manuf by Lonza, Merck & IMCL N =330, enrol compl May 02) →ASCO 03 (but some
CEO was Sam Waksal pancreatic +gemzar : 12% PR (N=40) control patients received Erbitux, & Feb 17/03
V Production facility in Sommerville colorectal + irinotecan(PHA) :23% response in CT head of Pharma resigns). BMY (Sept 22/01) 39%
completed end 01(3x10,000L) & 110,000 L refractory patients.N=121, open, no control; CHMPOK royalty (or 60% profit ?) & up to $ 2B deal
in a few years. Approved July 02 March 23/04, ditto FDA Indicated w/Campto in March 28/03 : Merck statement confirms has been
Jan 2/02 : FDA refuses file : irinotecan refractoriness not metastatic if refractory or intolerant to Campto alone
documented, undeclared deaths. Also stock options ?? reviewed by radiological committee
K Feb 5/02 : BMY wants to renegotiate & that S & H Oct 2/07 FDA OK for OS H&N ASCO 02 : N = 120, cisplat+/- 255, no
Waksal should resign. difference
March 7/02 : milestones reduced. Claim they will ASCO 03 : 10 posters, 1 oral BOND : ORR in CRC
provide FDA with data from EU ph III. Filing 03 ? H & N + Rx +CT(taxol,cisplat) :21%response in CT
Launch 04? st IV 11% Erbitux alone, 23% Erbitux + Saltz. (I'd
April 25/02 Merck delays CPMP filing until 1H03 refractory patients, 61 patients ph III ongoing ASCO 04 prefer> Saltz)
(colorectal & H&N). median survival 54m/ 28 m, N = 424 July 7/03 Merck file EU, H & N CHMP OK Feb
May 17/02 : Merck will not enlarge EU colorectal trial To file 2Q05, delayed to 4Q (April 13/05), June 05 23/06, EU OK April 3/06
for IMCL independent private review OK. Aug 31/5 Filed, PDUFA
May 22/02 : BMY & IMCL to run 3 "big" trials Manuf site licensed FDA June 18/04
st nd
June 12/02 : S. Waxsal arrested March 2. OK March 2/06 H& N 1 & 2 line
Feb 10/03 BMY & IMCL → limited access program CRYSTAL : 1st line CRC w/Merck Folfiri +/-
March 6/03 receives $ 60m milestone from BMY (1 year Acneiform rash in 53 %pts correlates with efficacy (but erbitux, open, rand, init May 04, N = 1000. Data
anniversary) Jan 11/07 : PFS signif sup; more at ASCO 07 (and
April 10/08 : 10K filing delayed. links with IgG1 isotype→C')
June 5/03 meeting w/FDA IMCL will refile. Fast track. Priced at $ 10,000/Course, 4 courses/year OPUs w/Folfox)
st
Approval 2H03:1H04 ??? Sales start Feb 24/04 by BMY + Merck. 1Q04 23.4 (39% Sept 4/07 Merck files EU for 1 line
Aug 14/03 file FDA w/Irinotecan CRC (Merck trial). st
royalty), 1 full quarter $ 71.4, 2Q 85.4, 3Q 114.6, 4Q 88,
Panel Dec 16/17? Priority review granted Oct 13/03 Merck 1Q06 : € 74m
(PDUFA Feb 13/04) OK Feb 12/03 & CPMP March FY 04 260; 2Q05 97.8, FY 05 413, 2Q06 176, 3Q 174.6,
23/04 FY 1.1 B ($ 652 in US), 2Q07 319ww (US 162)
Approved CH Dec 1/03 for refractory CRC - CDP 791, from UCB Celltech. Aug 16/05. F di (ab) Mab
to VEGFR-2 (KDR), ph II. Feb 5/07 IMCL opts out. ??
-cp1C11, IMC 11 21 B(antiKDR,VEGFr2)→K,ph I, ph II
RCC init Aug 25/07. ??
-Mitumomab(BEC2) anti-idiotype→SCLC,ph III (not
mentioned in 2Q earnings report).Ph II CRC fails Aug Why did they invest in Valigen (May 00) ??
29/07 & in Advanced Cell Technology ?
-IMC 18 F1anti flk(a VEGF receptor)→ph I BC, CRC
-gp75 vaccine→melanoma, RES
-melanoma vaccine hTRPx3 (2 melanoma proteins +
tyrosinase), pre
- IGF-1R , ati ILGF, IMC – A12(IgG1), ph I, ASCO 07,
ph II CRC init July 07, ph II prostate init Aug 28/07

- 83 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


IMGN M IMMUNOGEN (IMGN) -C242DM1,humaniz.MAb anti mucin cantuzumab GSK renegot. june 24/02, term Jan 24/03
+Maytansinoid (mersantine) →colorectalK, phI/II ASCO 01 : CEA decreases in 5 pts and ↗ in 4
- hu C 242 DM4
-hu901-DM1,maytan. MAb anti CD 56 →SCLC ph II,
Aug 1/03 :deal w/AVE →may 8/02, partner. BBIOY ( returned July 03 by
cervix , AML PRE→→. Ph I AACR Nov 06. 1CR in Vernalis), also GZTC
- anti CD 56 (MY-9-6 DM1), AML
- anti IGF 1 receptor for solid k, anti AKT Merkel. ASH 06 Myeloma.
pathway ?? - anti IGF 1R : K, Myeloma, AVE 1642 : in vitro very -lic (May 9)maytansinoid technol for MAbs to DNA's
active on CD 45 – plasmocytes (the worst prognosis) Herceptin, TERM June 24/02
- Trastuzumab DM1, ph I -sept 7/02 :partnership w/ ABGX
- anti CD44v6, maysantinoid, w/B/ Ingelheim, discont feb -oct 2/00 :partnership MOR
9/05 (skin toxicity ph I) -march 6/01: w/MLNM. Term Jan 26/06
- march 29/01 :w/Raven for targets & Abs.
IMMC K IMMUNICON kit for circulating tumor cells : June 1/07 lic to BMY competition Adnagen AG
- approved 2004 for Breast (w/JNJ)
- HRPC ongoing
- CRC
- w/PFe prediction of good responders to anti IGF1R Mab
(CP 751,871)
IMMT IMMTECH INTERNAT (IMMT) -dicationic antiinfectives binding to DNA minor grove
-CRP derivatives for K
IMMU K IMMUNOMEDICS (IMMU) - CEA scan : dg, MKT
M - CEA-cide, hMAb *I meta colorectal K, phII™ new peptide linker for *I
30 mn infusion versus 4h for Rituxan 85% disease free for 18 months, 58% ORR at 36m (Feb
288/02); naked Ab had quality of trial issue w/FDA ASCO 02 :Centre René Gauducheau (Nantes, Dr
30% of pts become refractory to Rituxan Kraeber-Bodere)
-LymphoCide epratuzumab( hu antiCD-22)→NHL, ph
III (since 9/00) w/Rituxan in Rituxan relapsed pts: good →lic to AMGN (20% royalties) ASH 02 ph I/II, N =
ORR Dec 9/05, RA ACR 04. Lupus ph III init June 114, active in follicular & large cell, median TTP
2/05. Ph II OK on clinical score 1pril 24/06 86.6 w. Monotherapy trial term (Jan 23/03). Nov
Sjögren ph I/II (ACR 05). Lic to UCB, May 10/06. Sept 11/03 all terminated. All rights returned April 8/04
06 FDA on hold
-*Y LymphoCide, ph I/II
IMNR IMMUNE RESPONSE CORP (IMNR) -Remune (killed, env. depleted HIV) +CT,ph III∏end 02, Agouron/Parke Davis(WLA/PFE)TERM July6/01
positive ph II data announced April 23/01, but ph II oct 31/00:legal issues on study publication
V Sept 10/02 staff ¬ endpoint missed in Spain (june 3/01). Good data in
Thailand w/TMGUE.OB (June 15/01) Trinity Medical
Group
June 11/02 : to add CpG
April 4/07 terminate HIV program

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


INCY INCYTE (INCY) DIADEXUS : JV with SBH.
acq PROTEOME (dec 00) -LifeSeq Gold : key genomic data base for EST→→ BMY,GLX,Nvrts,SGP,PFE,SBH,DNA,BGEN,MLN
-LifeExpress : adds RNA and Protein info→→ M.
30000 licenses to pharmaceutical industry!!!! -SNP in-silico data base from Oxford Glycoscience proteomics data-base
-LifeTools : bioinformatics software collab
Oct 30/01 cuts 400 jobs (out of 1100) to go LLY, ABT
into drug dev. w/DNA(Mabs to INCY MAXIA :
proteome), MEDX (Mabs to INCY targets).
Nov 02 Staff of 700 ¬ 37% - MX 5064, pre, diab, w/JNJ
.Nov 13/02 acq Maxia ( $ 28.3m) - MX 6198, pre, diab, w/JNJ COMPETITION CCR2 antags / MLNM (ph II), MRK
Early 2004 shuts down Genomics. Cuts 275 - Reverset, DFC, NRTI, Ph II. July 25/05 data : 1.4 log (ph II), ChemoCentrix.
jobs drop, Sept 28/05 FDA requires 2nd ph II. On hold April Sheddases (the ADAM family) cleave CD23 receptor
3/06 hyperlipasemia from B lymphocytes. ADAM 10 cleaves CD23, the
- INCB 3284, CCR2 inhib for inflammation, ph I/II, lic to IgE receptor. So long it stays in the receptor, the
PFE (£ 40 + 560m) for RA, CV, but keeps MS response is OK. But if it gets outside, severe allergy
- INCB 7839, sheddase inhib, ph II, K (sheddase activates occurs.
EGFR) through ADAM.

INFI K INFINITY - IPI 504 Hsp 90, Ph I, Myeloma, Gist (good data AACR Deal with MEDI Aug 25/06 $ 500m
CSO Julian Adams Prague 06), lic to MEDI/AZN
CEO Steve Holzman - IPI 609 Hedgehog pathway, pre, w/AZN
merging w/DPII, summer 06 - anti Bcl 2 res, deal w/Novartis $ 400m March 7/06
INGN K INTROGEN (INGN) -ING201 (Advexin, Advexin),Ad p 53 gene therapy ™ H Aventis returns rights (april 5/01)
traded since oct 12/00 & N K, ph III,SPA, March 04 1st ph III init (endpoint
survival), 2nd planned (endpoint TTP). Adp53 patents uncertain w/Canji/SGP
Aventis could have 23% transferred to spun NSCLC, ph II. ASCO 01 63% local response.
off Gencell prostate hi Swedish subs : Gendux
June 18/03 PIPE $ 11.5m bladder ph I
breast : good data San Antonio 2004
H & N ph II
-ING241,Ad mda 7 (melanoma differentiation associated Nov 1/06 : to file Advexin EMEA for Li-Fraumeni
gene),ph II, belongs to IL10/IL24 family
(immunodepressant)
-ING251, Ad PTEN, for glioma & colorectal, PRE
- INGN 225 mNSCLC ph II to init IQ08. Patients own
cells to stimulate anti-tumor immune response
INHX AB INHIBITEX (INHX) files for IPo March - Veronate IV (polyclonal Ab to fibronectin like elements Data presented ISSSI (11th int sympo staph infections
4/04 (Piper, Lazard) of adhesion proteins, from human donors, manuf by & staph ) Oct 24-27/04
trades July 04 NABI), ph II n = 512 completed Dec 03, signif reduct of
Staph & Candida infections in very low weight newborns. Competitors : NABI vaccine, MEDI/GSK Mab
in US 43000 very low weight neonates per Ph III init July 7/04(design agreed w/FDA). N = 2000 BSYX-A110 (anti lipoteichoic acid), ph II (from
- 85 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


year, 50% develop infection (18m). Endpoint prevention of staph infection in infants Biosynexus).
500 to 1250g, nrand v/Placebo. Secondary :
followed by Lazard, Bear Stearns, UBS. candidaFollow-up 70 days. Study to close end 05. Data
1H06 Jan 7/05 : enrolling very fast. FDA fast track (Sept
22/04). Nov 16/05 enrol compl; Nov 29/05 FDA says
approval possible for "very low weight group" even if
endpoint not met in 500 to 1250 g. Dec 5/05 : DMSB
unanimously recommends continuation Rolling filing init
Feb 14/06. Topline data April. April 6/06 : NS
- Aurexis, ph II, Hz anti staph (MSCRAMM) MAb, manuf
by Avid (subs of PPHM), to be manu by Lonza (Nov
12/04). N = 60, completed Jan 7/05. May 9/05 Top line
positive
INKB M INKINE (INKP) -Thrombospondin derivatives for K : res
-Fc receptor regulators : res
- Colirest ,oral progesterone, ph III, Inflam. B. D.
INMX INFORMAX (INMX) BioInformatic software

INO K INOVIO (INO) was GENETRONICS (GEB) →K,Bleomycin electroporation, ph II JNJ termin. July 26/01
Oct 17/06 acq Poly Gly polymers for No new data since May 99 : 33 pts in US (H & N, -communication at ICOOC (April 23/01)on H&N K,
transfection from Valentis published); 12 in France (not published); pancreatic cancer discussing but not presenting French trials (Seroussi,
S4 filed March 14/01 concept seems to have vanished. Boucicault)
Reappears Pancreas Bleo ph I March 1/05
Claim to have 190 patents (only 27 US Canaccord mentions ph III H&N and melanoma could start B.I. (June 21/01)
issued on Delphion) in Q1/Q2/01
electroporation for GT JNJ australia, Boehringer Ingelheim
Oct 29/01 : departure of COO, CFO & 20% - transluminal cath. for arterial disease w/CHIR & B. Ingel,
of staff. Sept 13/05 Merck KGaA
June 10/02 $ 4.3m PP - Prostate I/II init April 1/05 UK (Southampton)
June 20/02 electroporation Bus sold to ICN: MEDPULSER electroporation for chemo (Bleo) or DNA
NO term Aug 20/02 ph III SCC H & N, and skin (sept 06 N = 150)
ph I D genes : IL1 melanoma (Vical), IL12 (Lee Moffitt),
MRK (Her 2, CEA).
INSM K INSMED (INSM) -Somatokine( mecasermin rinfabate) subcut (ILGF + its merged with Celtrix, discussions in France,JV
competitor Tercica binding prot), filed HGH resistant growth retardation w/ELAN
March 10/05, PDUFA Jul 3/05 (Tercica filed Feb 28)
Sept 24/02 staff ¬55% diabetes, ph II (IGF alone DNA/CHIR : retinal lesions).
Approved Dec 05
- for osteoporosis : ph II
- for Steinert myotonia ph II init Jan 4/06
- 86 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-INS-1: (inositol derived)natural oral insulin sensitizer,ph
II:failure (nov 27). June 01 ADA : good data.
jan 8/02 : new analysis™better results,Sept11/02 termin.
-rH IGFBP-3/AKT Herceptin resistant Breast K, May
10/04 , sequesters circulating ILGF, ph I, ASCO 06 ?
- INSM 18 , small molecule inhib AKT, ph I/II prostate
(not on web site) ??
IOMI IOMAI Immunostimulant Ag sparing patch :
- bird flu
- traveler's diarrhea : good data (April 26/07) but ref probl
on web site. Cholera toxin adjuvant ?. May 28/07 : trial
shows vaccine made less efficient !!!
ISIS ISIS (ISIS) Vitravene (antisense to CMV)→→MKT→→→→ Ciba Vision
K New functional genomics division -Hepasense, antisense to Hep C Isis 14803, ph II Elan, rights returned Nov 6/02
(GeneTrove) (micropump inf TIW/4w)→

-ISIS104838, IV/SC, anti TNFα, ph II RA init March →JV Elan to init. oral in 01:OraSense, init April/01
Aug 22/01 : LLY has 9% 13/02; oral ph I bioavail 7%, Nov 02 (OraSense, JV
Dec 14/01 : collab w/AMGN w/ELN
-ISIS 104838, topical, ph II, PSO
LLY agreement : upfront 25 cash + 75 equity -ISIS 2302 alicaforsen (anti ICAM 1), Crohn, ph
+ 100 loan repayable after 4 years; IIIinitiated (enema) failure Dec 2/04; ph II ulcerative
milestones $ 50m, royalties & supply colitis (enema) N = 100 init April 1/03, success (Dec 2/04)
agreement, dev costs refunded. Renegotiated vs placebo & vs mesalamine.
June 5/03 on manuf & supplies. PSO : ph II : failure (Feb 25/02) but topical promising
- ISIS 301012, anti Apo B, ph II, w/ statins : lowers >
Sales forecasts of LY9 by analysts are quite 50% LDL chol, chol, TG , injectable
low. - anti PCSK9 (proprotein convertase subtilisin kexin 9)
March 3/04 $ 19.5 m BioWarfare contract. lowers LDL, lic to BMY May 9/07
→ returned Dec 26/02
- ISIS 2503 anti h-ras, ph II, pancreas data Dec 19/02 N =
March 12/04 partnership w/Alnylam 48 w/Gemzar, M survival > 6 m, breast & NSCLC enrol
compl 4Q02
- ISIS 5132 anti C-raf kinase, ovary, hi
w/LLY (Aug 22/01)
-113 715 (anti PTB1,negative modulator of insulin
ASCO 02: data on 600 pts (in LLY 4Q/01 report) :
receptor signalling )for diabetes : ph II, lic to MRK 05/01
NO, only ph II. Oct 1/02 LLY loans $ 21m to build
-ISIS3521 Affinitak (LY9),anti PKCα→NSCLC, phIII facility
open label "blinded"+ carboplat + taxol (init oct 18/00), Dec 02, UBS says “not very effective”.
fast track granted nov 8. Full enrol N=616. Jan 17/02→
data March 17/03 survival 10m/9.7 NS. 2nd ph III
N=1000 3 arms w/Gemzar + cisplat, carbo/taxol,

- 87 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


docetaxel init April 02; data 2H04?Ph II NHL
- LY 2181308 (anti-survivin), ph I, lic to LLy May 6/04
- ISIS 345749, anti STAT-3
- LY 2275796 anti initiation factor-4E, ph I, Feb 1/06
- OGX 011 w/AVE & OncoGenex, anti Clusterin (survival
gene/protein that increases when apoptosis is induced)
- ATL 1102 w/Antisense Therapeutics (Austral) anti VLA
4 integrin, ph II, MS
- anti SARS : pre
-Genetrove w/CRA, ABT, JNJ, Aventis, LLY, CHIR,
MRK, PFE, GSK
ISPH INSPIRE PHARMACEUT. (ISPH) Specialized in respiratory & eye mucosal hydration &
clearance, by modulating P2Y receptors. These are linked
to the mucosal G protein system.
Have patented UTP derivatives :
-INS 365→COPD, ph II,suspended April/01 to improve
protocol; Nov 02 : discont DNA terminates June 20/01. Kissei terminates
-INS 365,ph II→dry eyes. Fails prelim anal of ph III. w/Allergan (has a competitor)& Santen
Restarted April 3/02, signif (June 18/02),Approvable,
confirmatory underway. Feb 9/05 : failure, Dec 05 2nd
"approvable" saying need to demonstrate !
-INS 32217 (denufosol)→CF, ph II
- INS 37217 P2Y2 agonistintranasal ph I increases muco
ciliary clearance (temporarily stopped (April 02) AUG
14/02 ph II perennial rhinitis p < 0.05, ditto URTI (on
nasal drip).,ph III, N = 630, 28 days, DB, composite
endpoint, data mid 03
ISTA Ista Pharmaceuticals (ISTA) -Vitrase(hyaluronidase.)→vitreous hemorr (450 000/ -Timolol, ph III
Oct 4/02 : could be delisted Year., phIII;w/Allergan. Fails to reach endpoint of clearing - Bromfenac, topical NSAID, ph III
was "Advanced Corneal Systems" hemorrhage (March 26/02). April 10/02 FDA OK for - Caprogel, ph III, epsilon amino caproic acid
visual acuity as endpoint. Rolling filing clin end 3Q02,
May 15/02: quaterly report warns on CMC (chemistry-manuf-control) end of 3Q, validation 1Q03.
financing issues Cardinal Health will manufacture (July 9/02). Adv Com
June 12/02 plans $ 30m offering March 17/03 : no. April 7/03 "approvable letter" requests
another trial.. OK May 6/04 as "ophthalmic spreading
agent for other drugs".
-Keratase→corneal opacifications
ISV INSITE VISION (ISV) Glaucoma research & drug delivery -Ciba Vision
-Genotyping for TIGR (glaucoma) -Bausch & Lomb

- 88 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- ISV 205, anti TIGR, ph II, returned by PHA (May 01)
- ISV 401, azithromycin for bact conjunctivitis, ph III
(endpoint bacterial + clin)
- ISV 403, quinolone, lic to Bausch & Lomb
ITMN INTERMUNE (ITMN) -Actimmune : gamma interferon, MKT for chronic spinoff from Connetics
granulomatous dis.& ostopetrosis $13m/00;36/01; 105/02;
85% of sales are off label in IPF, but 1Q03 38, 2Q 33. 2004 : $ 125m, 2005 $ 107 → Eur Resp Soc Sept 15/02
declining Phase III init w/BI for resistant TB (March 01)
Interst. pulm. fibr. ph III DB N=330 completed Aug 28/02 → Amer Coll Chest Physicians Nov 3-7/02
April 03 "Patients fighting pulmonary : reduct in mortality NS (p = 0.08) except in subgroup mild Amer Thor Soc May 22/02 N=18. Press .release but
fibrosis" sue ITMN for "suppressing" a life- to moderate (p = 0.0008) perhaps by reduced not in abstracts on ++ 5 year survival ?? (weird trial)
saving drug (pirfenidone) to protect surinfection.Filing 4Q02? Fast track (Feb 02). Ph II Vienna Jan 8/03 : follow-up survival NS. Init 2H03 a 2 year
Actimmune mkt. was only N=18. Presented ATS May 05. Data 08. ph III in 600 mild to moderate IPFs. Topline 1Q08
Terminated March 6/07
Nov 10/04 Subpoena on promotion of Cryptococcus meningitis, ph II
Actimmune Other ph III anti K, ovary, w/cytostatics : halted Feb 3/06
-Duramycin (Lantibiotic, 19 aa peptide, increases Cl w/Molichem (MLCM) acq by CZVD (Cortez Ltd)
Pirfedinone would sell at 30 to 40,000 transport) CF, ph I (Johns Hopkins)
$/year; cases US/EU 200,000 - Infergen, Hep C , MKT (00 : $ 14M), active in pts lic from AMGN (June 18/01)
unresponsive to PEG IFN + riba (DDW 03), 50% of pts are
non-responders; data 2007. Sold to Valeant Nov 29/05 $
135m
- PegInfergen, pre, w/INHL (Oct 9/02) acq from Alza, Jan 01
- Amphotec (liposome amphotericine) comkt w/Alza, sold
to Three Rivers (May 25/05) NDA filing SSTI end 02 ? or 4Q03, then 1Q 04, and
- Oritavancin (lic from LLY Sept 20/01), ph III SSTI (init 2005 !!!!.I'm not sure about the protocol. Will be
Jan 01 to be completed end 02?) N = 570, ICAAC 01 = manuf by ABT. Oct ½ LLY exercises option to force
Vanco/cephalexin, but faster , ph II sepsis (was to be ITMN to purchase reduct in royalty rate. Dec 27/05
completed 1H02); high affinity for Albumin lic to Targanta
- Pirfenidone, P38 gamma inhibitor (lic in April 02 from
Marnac), ph II, , May 12/03 > prednisone on SaO2 (p =
0.014) in pulmonary fibrosis ph III ( N = 580) init April
28/06 (25% hypersensitivity reactions ?). and ph III in Marnac Inc had Pirfenidone in MS, ph II, now only
Hermansky Pudlak to initiate 1H06. Blocks TNF neurofibromatosis
receptors.. Data 09. March 20/07 135 pts added, & Tmt
increased from 60 to 72 weeks. Endpoint FEV1. Nov 26/07
bought back from Marnac
- ITMN 191 : NS3/4A protease inhib. Hep C. Lic to Roche
IVGN INVITROGEN (IVGN) sells 250 different cloning kits to more than 30 countries a low risk entry to the genomics playground (as LTEK
Acquired "Research Genetics", costs & TECH less "genomic focused",and Promega and
negatived 1Q earnings Stratagen private)

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Oct 02 : acq InforMax July 9 :merger agreementw/LTEK
July 2/03 acq Molecular Probes ($ 325m)
leader in fluorescent labels

IVPH K INNOVIVE - tamibarotene : oral retinoic acid derivative, 3rd line APL APL is due to fusion between 15 & 17 involving
(under SPA) init Aug 3/07, N = 50; endpoint 28 days CR retinoid alpha gene. Ist line ATRA, 2nd line Trisenox
- INNO 206, ph II, prodrug doxo, SCLC
- INNO 406, ph II, dual bcr-abl/ Lyn kinase inhib (in vitro
50 times > imatinib)
IZP INFLAZYME (IZP/TSX) - LSAIDS (leucocyte selective antiinflammatory drugs) Aventis Feb 99

- IPL 512602, asthma, allergic rhinitis, ph I , Aventis will Aventis Nov 02 ( $ 90m deal)
go into ph II
JI4G JERINI (Frankfurt JI4G GR) Icatibant, see Dyax. To file end 07. Top line 3Q06

JNJ PENINSULA (PPRX) - Doripenem, S 4661ph III, injectable Carbapenem, HAP,


AB VAP
CSFB, DEc 16/03, postponed March 04, - PPI-0903, cephalosporine spun out to "Cerexa"
refiled Jan 19/05, delayed Feb 10/05 :
Acquired $ 245 m April 19/05 by JNJ
JNJ SCIOS (SCIO) -Natrecor(ANF)I.V.→acute CHF,FDA turns down (but lic to Bayer
Adv com was 5+/3-;gives bradycardia). Bayer discont. GSK for EU (Jan 8/02),launch 1H04
PP of $ 125 m convertibles July 29/02 New ph III init. Oct 99, hi lmen jan 01; Adv Com May -alone (a niche mkt is possible)
25 /01:OK 10/0. FDA to decide by July 10 ( hi lment 2 negative letters JAMA end July 02
Feb 10/03 : JNJ acq for $ 2.4B (30% issues, panel concerned by t1/2>nitrites). July 10
premium on shares) cash approvable letter ∏ manuf inspection compl. July 19/01,
hi lment.. $ 380 per vial. FDA OK 8/14/01. Sales 2001
$14m, 2002E$56m, 1Q02 15.4m
-w/Medtronics assoc.w/heart failure devices AHP returns rights to Scios,
-Fiblast(rFGF)→stroke who lic to CHIR (whose FGF is bovine)
-Fblast→coronary→→→→→→→→→→→→
-SCIO 469,oral p38 kinase inhib(anti TNF,IL1 & COX2), competition : other oral p38 inhibs GSK & JNJ
ph II,RA.
KAL K CALLISTO (KAL) - Atiprimod anti PI3K / Akt & Jnk / STAT3, multiple AACR April 07
myeloma, carcinoid ph I/II
- Annamycin ph II ALL
- Degrasyn (from MD Anderson) Stat3 inhib (pre)

- 90 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


KERX K KERYX Biopharmaceut. (KERX) Regulators of signal transduction :
Jerusalem - KRX 211, pre, Jak kinase inhibitor (w/Lynx)
-KRX-101(sulodexide,mixture of heparins, oral) : diabetic
Aug 15/02 staff ¬ 45% nephropathy (reduct of proteinuria), ph II. To file for ph III
Jan 9/04 acq Access Oncology - KRX 123, prostate K, ph III
- KRX 0401 perifosine (in CND Aeterna/Zentaris): AKT other AKT inhibs : Neogenesis, Amphora,
Feb 13/04 PIPE init $ 32m inhibitor (also inhibits MAPK & JNK) oral. Ph II Breast, Immunogen, ABGX/PFE, Insmed, Exelixis (PTEN
prostate, Melanoma, pancreas, sarcoma, H & N, NSCLC, path), P. Fabre (F 50035 lic to MRK), Greewich
Breast w/Herceptin, init May 11/05, ph II leukaemia init Therapeutics (& Vioquest : Tricirbine), Rexahn
March 2/06. ASCO 06 ph I/II lung, breast; mono & combi (RXHN). Astex (anti PKB, lic to AZN July 27/05)
CT/Rx
- KRX 0402, inhibits DNA repair, ph II
- KRX 0403 spindle poison, ph I/II.
- Zerenex, ph III, iron based phosphate binder, oral
- UCN-01 (7 hydroxystaurosporine), ph II, multikinase
inhib lic from Kyowa Hakko, Oct 4/06
KOOL C THERMOGENESIS (KOOL) agents for cell therapy

KOSN K KOSAN (KOSN) -new polyketide ABs, oral, pre JNJ


trading since oct 9/00 - Epothilone D (KOS 862), phIb w Gemzar init March
4/04, non MDR sensitive Taxol analogue patent issued competitors : BMY (ph III, ASCO 06 ixabepilone,
Dec 10/03 shelf SGCowen, CIBC, Adams April 10/01 to MSKCC, exclus lic to KOSN, lic to Roche Breast ), Novartis
Harkness (Sept 24/02, term Oct 07). Upfront $ 30m. $220m deal (have another one, less toxic ??)
including milestones. Signif royalty. Ph II mono CRC,
- Drops secondary Aug 16/05 NSCLC. June 13/04 CRC halted (neurotox in pts who had
- Aug 15 : NCI amends protocols : EKG had oxali).Nov 23/04 NSCLC halted Init alone in HRPC,
prior to treatment ph II init Feb 14/05.. Oct 18/05 prostate halted, breast
ongoing HER2+. Discont Feb 27/06. Competition : IP 1504 (Infinity Pharmaceuticals ph
- KOS 1584, new Epothilone, w/Roche TERM Oct 07, ph I/II w/MEDI), highly soluble HSP 90 inhibitor,
II (R1645) Conforma
- KOS-953, tanespimycin, 17 AAG
allylaminogeldanamycin, which disrupts heat shock
protein HSP 90 binding of many oncogen client proteins
(BCR-ABL, cRAF1, ErbB2, AKT, CDK4, mutant p 53) &
thus neutralizes them, ph II melanoma , monotherapy
colon, then assoc w/Xeloda, ph I myeloma. June 15/05 : 3
EKG issues April 16/07 serial EKG on 85 pts OK; Assoc
w/ Velcade Eur Hemat Assoc June 3/05; assoc w/
Herceptin Breast
ph III MM 2nd line to init 1Q08; Sept 7/07 FDA OK on
protocol 2nd line.
- 91 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- KOS 1022, alvespimycin, HSP 90 inhib, ph I; Sept 10/07
clinical benefot in refractory Her+ breast. Preparing for 1st
line alone & 2nd line w / taxol
- KOS 1815, res, NEI (nuclear export inhibitor)
LCBM LIFECORE BIOMEDICAL (LCBM) -Titanium dental implants hydroxyapatite coated :MKt
-Hyaluronic acid :MKT (production on hold)

LEXG LEXICON (LEXG) -Human GeneTrap database The world's largest bank of knock-out
mice:OmniBank

- Switching strategy to develop proprietary drug portfolio -ABGX, BMY,AHP,TLRK,JNJ,BI


LGND LIGAND (LGND) ONTAK denileukin diflitox (DAB 389-IL2 : CTCL, mkt Royalties on Novartis's Simulect (from Seragen)
since 1999. 2002 : $ 26.6m, 2003 : 34; 1Q04 7.3
Nov 02 $ 135 m 5 year convertible offering RETINOID RECEPTOR ACTIVATORS (gels 2001:$2M)
ELN had 20% after conversion of -Panretin gel→Kaposi, MKT,CPMP OK July 17
convertibles (Dec 26/01). Selling all July -Panretin oral→breastK, ph II/III Sept 8/06 All antiK drugs sold to Eisai
3/03
-Targretin gel→CTCL, MKT, 2003 $ 4m, 1Q04 0.9
PIPE (April 17/02) $ 69m
-Targretin oral (bexarotene)→CTCL,MKT 2001 $ 14.5M,
Complex debt situation (GSK, ELN,
2003 : 10, 1Q04 : 3.5
Glycomed)
(breastK, ph II
NSCLC, good ph IIdata at ASCO, 2 ph III. 1st N = 600
4 products marketed IIIb/IV enrolled Aug 15/03. Front line + cisplat/vinorelbine
(EU), carboplat/taxol US. Survival data -> 1Q05: failure
March 28/05. 2nd line ongoing w/Gemzar, 3rd w/Carbo-
Tarceva
-Morphelan (Avinza):once a day morphine
Sept 29/04 class action suit on Avinza : sales capsule,approvable, launched July 11/02 ($ 6.2m for 3Q, w/ Elan; PDUFA March 23/02:OK March 20/02
4Q03 due to overfilling of wholesalers For $ 100 m royalty reduced from 30% to 10%
1Q03 : 6.6m, 4Q 32; 1Q04 22.4) . 3.7% Mkt share
May 03 : w/ORGANON (650 reps!!). Jan 18/06
sustained release opioids. Leaders Purdue's Oxycontin &
Organon terminates. Sept 06 sold to King $ 265 m
non branded generics
-LGD 1550→H & N K, ph II
-collab. res. w/BMY : new antialdosterone
-collab. w/GSK on PPAR agon. Diabetes 501516
-collab. w/LLY on LY 519818, PPAR agon. diab II,ph
III to init March 04 : no, delayed 2 years new FDA reg
- collab w/WYE on TSE 424 basedoxifene (SERM),
osteoporosis,phIII to file 2005
-collab w/TAP on SARMs, osteoporosis
-collab w/PFE on Lasofoxifene, osteoporosis, ph III,
NDA filed Sept 16/04

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-SB497115 w/GSK, oral small molecule thrombopietin
mimic, ph I/II (Jan 03). Ph II init Feb 05 : data nov
30/05 2nd line ITP strong efficacy
- LGD 4665, also for thrombopenia, to file IND 1H06

LIFC LIFECELL CORP (LIFC) Alloderm (from cadaver skin) : MKT


LIV Samaritan - AIDS anti –entry drug, ph III
acq Metastatin, r uteroglobin, pre, anti metastatic
On message board : a scam
LJL BIOSYSTEMS (LJLB) Highthroughput screening To merge with Molecular Devices (MDCC)
specialized in other highthroughput systems

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


LJPC LA JOLLA PHARMACEUTICALS (LJPC) - LJP 394 (Riquent) 4 twenty mer dsDNA epitopes Manuf by Avecia. ABT terminated
conjugated to triethylene glycolanti-lupus nephropathy Feb 19/03 primary endpoint missed (time to creat ↗ &
Avecia manufactures oligonucleotide, ph II/III,not promising
new ph III ( N + 730) initiated sept 21/00, reinit 3Q06, to proteinuria ↗) Follow on trial discont April 10/03.,
complete enrol. End 07. Interim March 7/07 : at 300 mg/w NDA filed Dec 03. Approvable letter Oct 15/04, more
decreases anti-DNA (soluble complexes) clinicals needed, could last 4 years.. Init Summer 04 :
…could be inducing an antibody switching like Coley).. 100/300 mg , perhaps 600 & 1200, for 12m, DB..
- LJP 1082 (4 copies of 1st domain of β2 GP1 coupled to March 15/05 FDA "unlikely" to grant accelerated
chemical moiety), I/II, thrombosis in antiphospholipid sd approval.; March 31/06 files EMEA
LLY AMEV APPLIED MOLECULAR EVOLUTION Engineered monoclonals
(AMEV) -anti ανβ3, phIIRA with MEDI (MEDI 570)
M was IXSYS
-anti CD 40 (anti graft rejection)→→→ with BMY
K
Royalties < 3% - Numax (2nd generation inhaled Synagis), clin. to start
w/MEDI
Nov 21/03 : to merge w/LLY (equiv to $ 2H01……not yet (4Q02) → Q3/03
400m). Acq completed Feb 12/04 - Anti IL 9 being optimized for MEDI
- AME 527, anti TNF, to file IND 4Q03
- AME 133, anti CD 20, to file IND 2004

LLY ICOS (ICOS) -IC 351/Cialis tadalafil(PDE5inhib)→MED, 24 hours, less JV LLY (manuf issues launch delays: 2 plants need to be
effect on PDE6 than Viagra $ 1.5B 2001 (→less blue inspected, this began April 16/02), labeling not agreed on
yet. PDUFA June 28/02:CRL April 29/02 on
vision),NDA filed June 28/01, CPMP July 12/01
acq Oct 17/06 by E. Lilly $ 2.1B confirmatory clinical & pharmacol studies, & LLY
-LeukArrest(antiCD11/18)→stroke, ph III halted----- - - GMP issues (confirmed June 28/02. Icos to file response
Completed Jan 30/07 PAFase(rPafacylhydrolase),ac. pancreatitis,ph II/IIIsepsis 2H2003 (Jan 22/ 03 in UK). CPMP OK July 26/02,
ph III endpoint 28d mortality(enrol since April 01, up to EPAR available Jan 10/03 testicular tox in animals
2500 pts,1st interim 2H02, end 2H05). Term Dec 02 w/Suntory
-Sitaxsentan, oral endothelin antag, ph II/III,CHF & PHT(
data STRIDE:. to relieve impaired exercise in PHT 3Q/02;
→50/50 JV TXBT (now ENCY); A Heart Nov 02 ??
terminated ??
- IC 485 (PDE4 inhib), ph I, COPD, ph II fails March
28/05
- RTX (from Afferon), ph II, local use vanilloid for
overactive bladder.
-IC 747, PSO, LFA1 antag oral, w/BGEN, ph II
- 23F2G, hz anti CD11/CD18( LFA1; α Lβ2, MS, Ph II, ?
- Aug 14/01: partn w/Seattle Genetics for SGN15
- IC 14 (Mab to CD 14), sepsis, ph I

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


LOR K Lorus Therapeutics LOR (CND) - GTI 2040 antisense to R2, ph II, refractory RCC N = 21, ASCO
LRP (AMEX) w/capecitabine, 21 d continuous infusion. Jan 12/04 > 50%
SD; April 12/05 N = 33 ditto; AML orphan drug status;
from merger between Lorus & Genesense breast ph II April 18/06 decrease expression molecular
Nov 2/05 : staff cut 35% target. Also ph II AML
- GTI 2051, antisense to R1 component of ribonucleotide
reductase, HRPC, ph I
- GTI 2601, anti thioreduxine, pre, w/Sumitomo 1st line + Gemzar vs Gemzar alone, then 2nd line for
- Virulizin, ph III since Nov 2001 (N = 350), pancreatic Gemzar failures (Virulizine + 5FU vs 5FU alone)
cancer , immunostimulant (bovine bile extract), increases
IL 17 E & IL 12. Data 2H05: OS 6.8m/6m. But subgroup
anal is OK for ECOG 0/1 and "meta" rather than"
advanced" Approved in Mexico for malignant melanoma.
LPTN K Lpath Anti lipid Mabs

- Sphingomab, anti sphingosine 1P, pre, antiangiogenic


- Lpathomab, anti lysophosphatides, antimetastasis, Res
LSBC M Large Scale Biol. (LSBC) -Hightroughput technologies, especially in Proteomics & -GMNI
functional genomics -BSTE, to generate Ab targets for protein biochips
June 5/02 : cuts staff - Galactosidase α , Fabry, from plants, orphan status Jan
23/03
March 30/05 : cuts staff 1/3 -Non-embryonic human stem cell research
-MAb manufacture in plants(w:Prodigene allied to
Epicyte)
- vaccine for NHL ph I
LYNX LYNX THERAPEUTICS (LYNX) Microbead techniques for analysing :
-large DNA samples :MPSS Massive parallel signature
April 18/02 cuts staff 30% sequencing→→→→→→→→→→→→→ Aventis, BASF,Hybrigenics SA, AZN
Aug 16/01 : collab. on SNPs of Diabetes -SNPs : MegaType
IIw/GCI (Genomics Collaborative Inc, a -Proteomics
tissue repository; private :Invesco,Soros) March 22/01 : collab w/UroGene SA
MAPP IPO MAPP Pharmaceuticals aerosolized drugs for asthma & migraine

priced Oct 5/07


MATK K MARTEK BIOSCIENCES (MATK) Products from microalgae : ABT (April 00) : neutraceuticals
-Long chain polyinsatutated fatty acids
-GFP

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


MATX MATRIX (MATX) -Intradose(cisplat+inject.gel)→H&NK,filed4Q00. ODAC July 31, H & N conf : did not show placebo data
Sept 10/01(improves quality of life): negative. FDA non
Oct 10/01 : 40% layoffs approvable.
-MPI 5020,radiopotentiator,phI/II
Jan 5/02 : being acq by CHIR/ Novartis --FMdC(DNA chain terminator)tezacitabine→K,ph II
- Accusite (5 FU implant) ph III discont (96)basal cell K
MAXY MAXYGEN (MAXY) Directed molecular evolution, thanks to : Novo-Nordisk, AZN, Dupont/Pioneer, PFE (sept 14)
spun out of Affymax & GSK -DNA shuffling technology
IPO dec 99 -Molecular breeding : vaccine deal w/Aventis Nov 6/01 Acq. Profund (DK) specialized in "Intelligent Protein
-MaxyScan screening Diversity"
Simba Gill, President since May 00 35 product candidates, Board links with GLX & SBH
Ernest Mario on Board (Aug 01) Maxy-Alpha, Hu αIFN 250000 x active, IND sept 20/00 : lic prot. for MS to Lundbeck
for 2006, lic to Roche, but have retained the right to
May 22/03 : IFN deal w/Roche $ 230m develop novel IFN α product candidates specifically Collab w/Shearwater, the PEG group working
tailored for infectious diseases, oncology, inflammation, w/Roche, competitors of ENZN (for INF ???)
auto-immune diseases. Sept 23/07 Roche puts ON HOLD
Maxy G-CSF ph I init Sept 06 : half life double
of Neulasta; July 2/07 init IIa w/TAC
γ IFN lic to ITMN (Sept 6/01)
Maxy VII (w/Roche, Dec 05).March 14/07
-

-Collab w/ALK-Abello (Holland) for allergy drug


MBI MICROLOGIX (MBI.TO) MBI 853 NL, prevention hosp acq. staph : suspended July
16/01
MBI 594 AN, Acne, ph II
MBI 226 (cationic peptide)gel, catheter related infection, ICAAC 02 ??
ph III July 9/02 lic to Fujisawa (20% royalty, low
Lipopedtide program acq May 02 upfront)
MBRX Metabasis (MBRX) CS 917, ph II, diabetes, neoglucogenesis inhibitor, Sankyo
halts ph II March 16/05

MCHM MACROCHEM CORP (MCHM) Transdermal/mucosal drug delivery :

-Topiglan(alprostadil)→MED, ph III fails endpoint Sept seeking partners


7/01
- EcoNail : antifungal nail lacquer, ph I

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


MCLS MediChem LifeSciences (MCLS) -Medicinal chemistry/combinatorials PFE, PHA
Inception 1987 -Partnership w/Elitra (premier provider of bacterial
traded since oct 26/00 genomic targets) to develop novel antibiotics

Acq by DeCode (March 02)


MCU MEDICURE (MCU) MC-1 (what is it), ph II, OK (Dec 05) to improve CABG Medicure International in Barbados
results, and angioplasty
Albert Friesen

MDCO The Medicines Co. (MDCO) - Angiomax (bivalirudin, lic from BGEN March 97, but Ferrer (partner for South Europe), and Nycomed
Follow-on filed March 5/03 raises $ 85m royalties to climb 10 to 21%, chemically manuf by
(MS, CIBC) UCB).Pivotal trials compare to too much Heparin. Sales 01 Europ soc Cardiol : Sept 01:HERO2 (+SK)∏ missed
PIPE (May 01, R. Stephens) : $14.2m; 2002 38.3; 1Q03 16.7, 2Q 18.8, 3Q 21.2, 4Q endpoint
recruited reps from Cor & Centocor 28.9; 1Q04 31.3, 2Q 34.4, 3Q 37.3 (EU 3.3), FY 05 150.2, Transcatheter : Sept 03 : 6 m data on Replace II : less
June 22/02 : shelf $ 32m 1Q06 32.8, 2Q 53.3, 3Q 55.7; 4Q 60.2; 1Q07 66.6 180 d mortality than Heparin + antii Gp (but NS)
FDA approved (dec 00) for unstable angina undergoing A. Heart Nov 03
PTCA,+aspirin. CPMP rejected oct 99. Refiled A Coll Card April 03 : although costs $ 335 per
T1/2 = 25 min : no need for protamin ! Aug4/03. Approved June 24/04; being reviewed for intervention & heparin $ 10, overall cost savings &
NSTEMI (Sept 07) less deaths from bleeding. June 1st 03 price increase
Risks/ victories : Ph IIIb/IV ongoing with Integrilin (Replace I),Plavix, 8.9% ($ 365)
- CPMP (approval Sept 20/04) for PCI ReoPro (Cachet), SK (Hero). TCT Sept 27/0ct 1-04 : synergy w/Plavix (especially
- New manuf process : approved by FDA REPLACE II (angioplasty) started mid 01, AHA Nov 02 1 as Plavix will be generic !); registry shows often used
May 27/03 (CHEAPER !) m data; N = 6000. Angiomax +/- antiplatelet > historical w/Cypher stent
- 4Q 04 : data from CABG (600000/Year heparin comparator. Nov 03 AHA 1 year data still good outcome 30 days
US, 1/3 off pump) . Filing 4Q04 -> Sept 05 (especially in "elderly" or "at risk". UFH or Angiomax Angiomax
?? Acute Coronary Sd : ACUITY ; Enrol compl Dec 5/05 LMWH + + antiplat alone
June 04 : FDA rejects label extension for (13,600), data ACC March 12/06 : RISK : see table. antiplat
HIT, because of CK, and bleed ? files sNDA ACC March 07 50% less bleeding than in control ischemia% 7.3 7.7 7.8
PCI :answer 05) : OK Dec 1/05 group. sNDA 3Q07 filed Aug 7/07; PDUFA non inf p 0.007 0.01
June 15/05 label extension for CABG Choose (in pts at risk of HITTS, heparin induced bleeding 5.7 5.3 3.0
"percutaneous intervention" and not only thrombopenia & thrombosis sd) & Evolution (150 on pump, 150 non inf p 0.001 0.001
PCTA) off pump) ongoing, open label. Evol.Off pump data Nov
04 : less blood loss than with Heparin (p = 0.03). Evol On
July 2/07 reacq EU rights $ 45M pump Dec 29/04 : 95% success vs 91.8% w/heparin-
protamin (success = 7 days post surgery w/ no death).
Aug 7/07 ACC recommends Angiomax for Choose data 4Q 05, sNDA in 2005
unstable angina (NSTEMI) March 5/04 publication of New Zealand trial N = Nov 8/04 PROTECT trial of Integrelin vs Angiomax :
Plavix T½ > 8h (needs liver metab, CURE 100, random, open label, off pump, same safety as Integrelin does not meet primary endpoint of coronary
trial); Cangrelor : platelet aggreg is nL 15 heparin, better graft perfusion at 90 d. flow reserve, but Integrelin improves "perfusion".
min after infusion (T1/2 10 min) -CVT-05, bacterial vaginosis, ph II: failure March 26/02

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ReoPro T1/2 12-24h. - Clevidipine/ clevelox from AZN , short acting Ca antag ACC March 6 – 9 / 05, in favor of PCI and unstable
for acute hypertension, ph III, on hold March 28/05 for angina, according to Hibernia, but less for CABG
atrial fibrillation. Filing accepted Sept 14/07, ECLIPSE
trial presented Oct 15/07
- IV antiaggregant (Plavix receptor P2Y12) Cangrelor,
from AZN, very short half life. 2 ph III init April 06 vs
Plavix. To file 2008. (ReoPro is $ 280m/year)

MDVN MEDIVATION Dimebon (dimebolin) binds cholinesterase & NMDA


receptor. Alzheimer ph II vs placebo. June 11/06 : 1 year
Sept 11/07 facility to sell $ 100 m shares data, better than Aricept. To init ph III in 2008. FDA
might allow filing before. But trials in Russia. Ph I
ongoing in US

2 MEDIGENE AG (NM 502090) Focusing on cardiovasc. & cancer, with gene therapy and w/AVE
K classical pharmacology
Board : Winnacker, Afting, who were in -Vaccine (in AAV)for melanoma, auto-cell therapy, GMP w/Schering AG
HAG top management production by AVE/Genopoietic:ph I -Genomic alliances : Affymetrix (sept 26),
- Vaccine (in chimeric virus-like particles, VLP) anti HPV Ciphergen (oct 19), Compugen (sept 13)
for cervical K : initiated in Germany. IP issues Known tox : insulin resistance, intramyocellular
-Polyphenon : drug to treat HPV, 1st ph III completed lipid accumulation, hepatic & cardiac
-Etomoxir (inhib of carnitine palmitoyl transferase hypertrophy. Terminated June 13/02
Aug 02 : cardiovasc is divested. To become (switches from fatty ac. to glucose catab),phII⇑CHF
an oncol company - Working on a gene associated with CHF (DCMAG1,
dilated cardiomyopathy…)
- Leuprogel s-cut, prostate K from Atrix for Europe (April
5/01), filed in Germany (reference state) Dec 4/01, on
MKT. - Acq.(jan 01)NeuroVir (US)⇑K Therapy vaccines
from recombinant HSV:
- G207, brain K, ph I/II (MSKCC)
-NV1020, hepatic metastases of colorectal K, ph I/II,
MSKCC.

MEDG IPO Medgenics (LSE) BIOPUMPS


Traded Dec 4/07 Biopsy sub cut to get tissue; transfect w/gene in Adeno,
reimplant :ph I/II EPO, planned HGF, hemophilia.
BIT SENSORS ???
MEDX CELLDEX filed Sept 4/04) - HCG-Vac (cancer vaccine), Ph I, CRC, pancreas, init Has acq Lorentis Ltd (program on NOTCH,
May 04 and Alteris Inc.
70% owned by Medarex - CDX 110 anti EGFRvIII peptide vaccine, glioblastoma
V
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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ph I ( Brain tumor Ctr at Duke , MD Anderson) : TTP
315d vs 124 (historical) p < 0.007; OS 21m vs 12, ph II N
= 23 enrol compl April 06

MEDX M MEDAREX (MEDX) Fully human : patents cross-licensed w/ABGX


The HuMAb-Mouse company (acq GenPharm in -MDX-CD4,→RA, ph II
1997) -MDX 010, ipilimumab anti CTLA-4, IgG1, K prostate, ASCO 02 melanoma N=17, 2PR;prostate N=7,2
May 17/07 new CEO (from Chiron, until sale)
also w/CEGE GVAX, RCC, ph I/II; ph III (SPA) init Sept PSA¬
K Howard Pien
04. N = 750, melanoma refractory stages 3 & 4 (3 arms : 010 partnered w BMY $ 530m Nov 9/04 Royalty
owns 30% of GenMab alone, w/MDX 1379, 1379 alone). Endpoint : response 20%
rate. To end 1H 2006. 2nd ph IIImonotherapy, 2nd line, init Competition PFE/AMGN/ABGX Ticilimumab
acq. 34% of IDM (oct 24/00) April 1/06. enrol compl Jan 4/07 To file 4Q/07-1Q/08. 1st IgG2(royalty 10%)
line init June 19/06, N = 500 +/- dacarbazine, endpoint Anti CTLA-4 side effects ?
PFS
-Anti CD 137 (BMS-66513), ph I init Nov 15/05
- MDX 066 (anti toxin A) and MDX 1388 (anti B), Sept w/IMNX
6/06 init of ph II Clostridium difficile associated diarrhea.
cGMP manufacturing :
Single dose randomized vs placebo. N = 200. Data end
Utrecht 07/1Q08
San Jose (CA) -MDX44 f hu anti IL8 Fab, very high affinity, PSO, res +activated Monocytes w/IDM: ph II ASCO:good data
Annandale & Greenwich (NJ) (w/BSTE); but look at ABGX!! outside of cell therap : Scil AG
- Golimumab, CNTO 148 , anti TNF (successor of $ 3B w/Merck KGaG
Aug 15/02 : delaying manuf build-up Remicade) (once a month SC ! Humira is QW. To file end
08. 3-5 % royalty, w/JNJ; ACR 4Q07
Partnership w/PFE Sept 20/04 - MDX IL15, ph I/II, RA
- MDX 060 (antiCD30), lymphoma, ph II
- MDX 070, anti PSMA, cross lic. w/MLNM
- Ticilimumab (CP-675,206),anti CTLA4, IgG2, ph III,
melanoma, w/PFE
- CNTO-1275 anti IL12/IL 23, 3-5 % royalty JNJ, PSO REGENERON : Targeted genomics
- 19D12 : anti IGF-R EOS : high throughput research of Ag targets
Bispecific : Kirin (transchromomouse)& crossbreed of both mice.
-MDX-210 (anti her2/Neu & FcγRI) MEDI : Numax
Ovary , ph III, -Epigen (Human carcinoma Ag), -OGS to develop
Prostate, ph II ,PSA¬ Abs
-MDX-447 (anti EGFr)→K, ph I/II→→→→→→→→ -LLY (nov 6), -GMNI, -Athersys, JNJ (Centocor),
-MDX-220 (anti TAG72)→K, ph I/II,colon&prostate Corixa
Humanized : - BIOSITE : phage display, + LLY:Transphage
-MDX-33(anti FcgammaRI)→autoimmuni., ph II w/AVE -Novo Nordisk, -Novartis, -Aventis, -Seattle
-MDX-22, BM purge/AML, ph II Genetics (feb 01)
Immunotoxin bound : ricin -Immusol (inverse genomics), feb/01
- 99 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-MDX-44, →PSO, ph I/II -SGP March 29/01, INCY (Oct 18/01)
-MDX-RA→anti epith. regrowth post corneal surg.
Vaccines :
- MDX 1379 : 2 gp100 melanoma Ags.

MIPI IPO Molecular Insight, priced Feb 2/07 - Onalta, edotreotide, radiolabeled somatostatin, ph II, lic
from Novartis
MITI C MicroMet was CancerVax ( CNVX) - Canvaxin (3 allogenic whole cell lines vaccines), ph III Partnered w/Serono Dec 16/04 50/50 US, exclusive
From John Wayne Cancer Institute (Santa melanoma ( N = 1100 stage III, 660 stage IV), New enrol ROW
Monica) ????? on hold (characterization of product) May 2/02. Reinit
V April 15/03 (feb 04 enrol 870 stage III, 349 stage IV). Trial stopped Oct 04
Files for IPO Aug 15/03 (Lehman) : 72m Enrol stage III, N = 1118 completed Sept 20/04, survival
trading since Oct 30/03 interim 1Q05delayed (Jan 05) to 3Q (all data delayed
M from end 05 to mid 06) for st III, 06 for st IV (April
K Jan 9/06 to merge w Micromet AG (closed 6/05 stage IV discont). Oct 4/05 st III discont
May 3/06) : Dec 9/02 Melanoma stage III 5 year data (N = 2600)
- MT 201 fully Hu Mab anti Ep CAM survival w/vaccine 56.4 m, versus 31.9 (p = 0.0001)
expressed by cancer stem cells Fast track. Safety Committee OK on interim March 1/04
(adecatumumab epithelial cell adhesion Colon to start
molecule), partnered w/Serono Dec 7/07. Ph - March 14/04 : T-oligos lic from Semaco, telomere
II Prostate, mBK (w/Taxotere). Fails Dec homologue oligos, K
4/06 but endpoint too tough (good dose - Monoclonals : from Cellmatrix, D93 (anti cleaved
response) collagen, antiangiogenic)
- EGF vaccine lic from Cuba. ASCO 04. After 1st line in
NSCLC, rand to BSC or vaccine. Survival (p < 0.05) 17.33
m / 10.27 m. Co dev w/YM Bioscience
- MT 203, anti GM-CSF for RA, w/Nycomed. Ti init clin
2008

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


MLNM MILLENIUM (MLNM). N = 2150, Genomics $ 245m/01, $ 193/02. Decreasing
-obesity, diabetes II, -Cancer, -Atherosclerosis, -CNS,, Roche;ABT (term Aug 15/03)w Meridia from BASF
M Dec 02 Staff ↘ June 6/03 : to cut 600 -Inflam.resp.dis. -Fungal infect, LLY
Dec 5/02 : changes in senior execs; -Bacterial infec, LLY AHP (K channel protein) AZN PFE WYE
Merge w/CORR Feb 12/02 -Inflammation- Thrombospondin mutations™coronary dis.™™™ Bayer (expired Oct 03 )
Humanized MAbs from LeukoSite (merger oct 14/00) : AVE (term at 3 years April 22/03)
K Until 2006 EPS to be disappointing because
-Campath(anti CD52)→CLL),EU OK march 29/01 "under exceptional
of genomic revenues ↘ circumstances, after failure of other agents". FDA ditto (May 01). Sales
3Q01 : $11m →lic to Schering AG(Berlex), & JV LeukoSite/ILXO
" Indicated ( May 03) for MM pts who --LDP-01(antiintegrin)→stroke, hi, w/Xoma, terminated Oct 10/03 sold back to ILXO (Oct 29/01), royalties from 05, fees before
have received at least 2 prior therapies, -LDP-02(to a4b7 integrin r)→ulcerative col, ph II, w/XOMA/DNA
9/16/02 primary end point NS (activity index), but remission signif;
with disease progression" March 05 - MLN 591 *Mab to PSMA, ph I, prostate K, MLN 2704
approved 2nd line DDW 03 remission rate ↗ Oct 8/03 : not to move forward
w/maytansinoid DM1 selected by CDER March 04 for MAP2, term
→Genentech, BZL Biologics for DM1 (ph I)
Jan 26/06
w/Paion GmBh (March 15/02)
Myeloma 15000 new/y US, 11000 deaths ww Other small molecules :
-LDP-519(proteasome inhib)→stroke, I Proteasomes activate bcl-2, NFκB, CDKs
Prevalence 80,000
Price $ 20,000/year
-Velcade LDP(PS)-341 Bortezomib dipeptide boronic acid analog
→ ASH 01(myeloma CREST). ASCO 02.only 78 pts
(proteasome inhib), I.V.→refractory myeloma, ph II enrol compl(N=75
Manuf. cost ??? expanded to 200). CLL ph II, NHL,pancreas (w/gemzar) colon (w/5FU or presented, prot M ¬ >90% in 20% pts .Ph III(
Sales init May 20/03: 2Q : $ 7.9m, irinotecan, halted Jan 04)ph I , Breast (w/Taxotere)ph I,carcinoids ph II, APEX) init June 12/02 N=700 (95 sites), refractory,
ovary,NSCLC ph II 2nd line ASCO 04, RCC ph II.Neuropathy gr III : open label comp Dexa, 9 months enrol (fully enrol
3Q 23 ; FY 59.6; 1Q04 29.6, 1Q05 3%
45, FY 06 220?5 (+ 15%), 2Q07 -LDP-977(small mol. inhib of Leukotrienes)→asthma,ph I,oral, lic to Oct 03), powered for 30% ↗, TTP. Dec 15/03 control
Taisho (on hold, liver tox) arm stopped because of success at interim Fast track
62.6 -Molecules from LeukoSite/Morphosys partnership granted (June 5/02).DATA ASCO 04. May 18/04
ASCO 03 > 20 abstracts -anti CCR3, asthma, pre, w/Roche
-anti CCR 1,inflam, pre, w/Aventis Apex survival signif.
Onco sales force : 75
Research : melanocortin - MLNM 4760, MCR 4 antag, carboxypeptidase inh ph I, obesity ASH 02 complete ph II SUMMIT : 202 pts single
- XR 11612, oral dual topoisomerase inhib, pre, K, from Xenova Dec 01
arm), : M prot ↘ 50% 27%pts, ↘ 100% 10%; M
-Integrilin/eptifibatide(antigpIIb/IIIa) : now leader in Rx growth
COR THERAPEUTICS (CORR) (but the acq ,overtaking Aggrastat & ReoPro .Lowering of death post stenting 35% survival 16.4 m (hist 6m)ph II filed Jan 22/03;
was in convertible notes to be redeemed (p<0.001). 2001 : $ 231 m. 2002 : $ 303, 1Q03 89 2Q 92(50 to MLNM, PDUFA July 22/03 Approved May 13, 03, filed EU
April 29/03 → $ 637m cash, could be 1Q04 48) . Could benefit from low price in post stenting package. CABG Feb 4/03. Medicare OK Sept 26/03; EMEA OK
delayed 1 year) . NO, paid April 29/03
trial (Cabernet) to start 2Q03; STEMI (w/PTCA init Nov 02) JAN 22/04. VISTA 1st line MM w/ M & P, N = 682.
-Cromafiban (oral) : hi Could be $B MKT. On hold High signif OS, PFS. Data ASH 07
CV Sales force : 100 -Oral Xa inhib, pre⇑DVT, ph I init Dec 23/02
- CT 53518 (MLN 518) small molecule FLT3 receptor TK antag, AML,
SGP (50/50 profit sharing), 160 coprom revenue
July 1/03 : EU Velcade to JNJ (just after ph I started June 02 (in vitro active on cells with FLT3 mutation). Fast 2002
track June 5/02. ∏ ASH 03??; competition CEPH 701, NOV PKC CRUSADE : N=60000 high risk heart patnts, until 2004,
they received the CPMP questions). Low
upfront shows concern, CPMP could wait for ph III 412, SU 5614 (PHA/PFE) compares assoc Lovenox to w/high mol weight heparins.
results for milestones. Upfront 15m + 40% of joint dev -MLN0415 (w/ Sanofi ), oral, IKK inhibitor, decreases NF kappa B But (March 18/02) AHA/ACC new guidelines recommends
costs (to 2005, could be $ 500m), milestones $ 500 (125 activation mainly anti gp2b/3a in UA w/PTCA, rather than UA
Myeloma, 330 other K, 65 if EU sales reach a certain alone.A. Heart Nov 17/20 02 (data on first 25, 000)
level), R 20 to 25%.

June 23/04, forEU, switches Integrelin to


GSK. July 25/05 US rights to SGP

- 101 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


MNKD MANNKIND (MNKD) inhaled dry powder Insulin, ph III

MNTA MOMENTA - Generic M Enoxaparin, a NDA filed Aug 05. Review will
exceed 24 months; rejected BY fda Nov 6/07 : risk of
immunogenicity
- M118 next generation heparin, ph II
MOGN K GUILFORD PHARMACEUTICALS Gliadel : BCNU wafer→recurrent glioblast.,MKT (00 : $ reacq. (oct 24/00) from Aventis for equity.w/Orion
Merger aborted GLIA (aug 28/00) 16m, 01 : $ 14m, 03 : 19). ODAC Dec 4/01 OK(8/5) for
first line, but chairman says placebo!Non Approvable
US Gliadel sales force 45 March 20/02; Sept 20/02 some new data filed. FDA OK
Feb 26/03
Acq (July 21/05) by MGI Pharma : $ 177 -Paclimer :taxol wafer→ovary K, IND ph I/II Amgen discont. (Sept 19/01)
m -NIL-A,Neuroimmunophilin ligand (oral) : Park, ph I→II
(Aug 8). July 26/01 : NS !!
- Aquavan (propofol prodrug) : (anesthetic), NDA to be
filed YE 07
- Naaladase inhib, ph II : diabetic neuropathy
- Dipeptidase inhib w/PFE, pre, May 8/03
MOGN MGI PHARMA (MOGN) -Palonosetron, ALOXI CT antiemetic, NDA filed Nov02
Approved July 25/03. Sales 2004 : $ 159.3, 1Q06 63, ASCO 03 . N = 1800/palonosetron success 81% /
Dec 2/02 : PIPE $ 21m 3Q07 111 ondansetron 68.6%.. But Generics & new Merck drug.
-Salagen tablets for chronic dry mouth after H&N radiation
Sept 1/04 acq Zycos & Aesgen -DIDRONEL, MKT,
-Irofulven, ph III, liver & pancreatic cancer, term April
17/02 (< 5FU), ph II ongoing NSCLC, ovary
- MG 98 antisense to DNA methyltransferase, ph II, H&N
and renal, AML ph I parner : MethylGene discontinues Dec 15/06
- Dacogen lic from SUPG Sept 1/04, approvable Sept 5/05.
EMEA filing withdrawn Nov 15/05. FDA OK May 3/06,
lic to JNJ July 6/06 ex US
- ZYC 101a vaccine for cervical intraepithelial neoplasma,
better than tTransgene (Eurogyn 2006, April 25/06)
- Saforis, acq w/Aesgen, for oral mucositis : "approvable"
Oct 13/06
- AKR 501 thrombopoietin mimetic (c-Mpl agonist), ph II,
oral (GSK has Promacta in ph III.

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


MORP MORPHOSYS (NM, 663200) Technology platform for 2nd generation MAbs that can be PHA 97→99 (dis)
produced in E. coli : B. Mannheim 95→97 (dis)
Jürgen Drews on the Board -HuCAL : user friendly human combinatorial antibody DuPont :98
M library, with 2X 109 Ab variable domain genes,phage CHIR : 98
Value ++ if they become a drug Company display (non exclusive licence from Dyax),patent litigation LeukoSite (MLNM,10 targets) :98
They have synthesized the key genes of Abs, with CAT : Aug 20 preliminary ruling in favour of Genome Pharma Corp AG : 99
keep them in phages, and can express them in MOR. Ongoing litigation on Winter II Bayer (10 Targets) : 99
E. coli : time to production faster than for - CysDisplay : improves screening (Abs are recovered B.I. expanded Nov 13/06
transgenic mice from phages by chemical cleavage rather than elution) Roche (MAb for Alzheimer), MLNM, GPC Biotech,
Planning US listing. -TRIM : triage techn. excl. lic. Johns Hopkins EOS,
-scFv : non exclus lic from SCA ventures (Enzon) ProChon Biotech(Israel):MAb to HGFr™K
Dec 01 : Schering acq 10% - F(ab) : lic from Biosite oct 00 : alliance w/IMGN :hu MAbs for K
Dec 3/07 big expansion of Novartis - MOR 101 (anti ICAM1/CD54) for burns, pre dec 00 : BGEN: MAbs against EST proteins
agreement, for 10 years, potential $ 1B - MOR 102, PSO, pre jan 01 : Centocor/JNJ partnership (30 targets)
- MOR 201, blood borne K, pre 2003 PFE until 2008, expanded Dec 20/06 until
2011
MDH MEDICURE (CND) - MC 1,, cardioprotective for CABG
- Aggrastat : for ACS
MRK ROSETTA Inpharmatics (RSTA) Bioinformatics and Yeast models IMNX
acq by MRK (May 11/01)
MRVT MIRAVANT (MRVT) SnET2 photodynamic therapy ph III , ARMD, term Jan PHA
15/02
MS BIOMS (TSX : MS.CN) MBP 8298, synthetic myelin basic peptide (17
aa)molecular replicate of the site of attack, ph II/III SPMS
(secondary progressive MS, and not RRMS)
Maestro 01, Ph II/III CND/EU, DB, TTP, 24 m trial. 2nd
interim 2008, full anal 2009.
US ph III N = 500 init June 07, completion expected 2010
Also a trial in RRMS in EU
MSHL K Marshall Edwards Inc (MSHL) - Phenoxodiol (lic from Novogen, Australia), oral, ph III
init Aug 07, 2nd line squamous cell K cervix/vagina; small
subs of Australia Novogen isoflavone, blocks sphingosine kinase & AKT (prevents
phosphorylation of XIAP)
IPO Dec 18/03, trading since Dec 18/03) - Phenoxodiol IV ph II, leukaemia

Also in ovary K

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


MVIR MEDIVIR (Sweden) - Lipsovir, ph III, herpes
- Hep C protease inhib ph II w/Tibotec
- Valomaciclovir, ph III
- Cathepsin K, osteoarthritis

MYG K MethylGene (MYG : TO) -MG 98, w/British Biotech & MGI Pharma, ph I, DNA
methyltransferase inhib
Don Corcoran -MGCD 0103, orally active histone deacetylase enzyme, lic to Taih o, Oct 21/03 ASH 05
ph I.>? Partn. w/Pharmion Jan 31/06
-Small molecule inhibitors of VEGF R family and also
c-Met, Tie-2, Ron- kinases
- Sirtuin antagonists (histone deacylase inhibs), pre,
w/PHRM
MYGN K MYRIAD (MYGN) Genomic by population studies (Utah, Quebec) :
BRCA 1 & 2
65 reps selling to oncologists of predictive CHD1 (coronary heart disease)
tests BRCA, Colaris, Melaris (for melanoma) Gene predisposing to Diabetes I
since Sept 27/01 MMAC1 (glioma gene)
MMSC1 (prostate K gene)
-MPC7869 Flurizan(flurbiprofene stereoisomer !),
prostate K,ph II, acq. from Encore, ph II/III init Dec 01 (to
last 2y)JAN 8/07 fails; also in Alzheimer , called SALA
selective amyloid beta lowering agent (lowers amyloid
beta). 2 Ph III.US fully enrolled (N = 1687) top line YE 07,
18 m Tmt; global to complete enrol 1H08 Slight efficacy,
side-effects
-MPC 1203, antithrombotic protein, pre.
- MPC 0920 oral antithrombin, precl
- MPI 176716, pre, apoptosis activator, prostate K, NHL
- MPC 2130 apoptosis inducer, ph I to init 1H07
- MPC 6827 Azixa, from Epicept who got from Maxim,
anti tubulin polymerizing agent, "vascular disrupting" ph
II init March 8/07 ,glioblastoma w/oxaliplat vs oxaliplat
alone. AACR 07 data in vitro induce surge !! Aug 16 init
3rd ph III in brain metas of NSCLC, w/temozolamide

NABI NABI (NABI) -StaphVAX (types 5 & 8, 85% of cases), ph III: sept Supplies plasma to Baxter
18/03 fails to meet 12 month endpoint, but signif at 10
V Nov 4/04 filing Phoslo EU (Mut Recog) months (40%). 2nd ph III fully enrolled Aug 17/04 in ICAAC 05
(with also CARE study) ESRD N = 3600, enrol. in less than 1 year. Data
- 104 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


AB (primary endpoint 8 m) Oct/Nov 05. Fails Nov 1/05
Dec 20/05 closes EU operations Filed EU Dec 21/04 (w/1st ph III). Withdrawn Nov 1/05
Could be due to contract manufacturer (changed to
Cambrex, add Ags 336 & PS-1; will reinitiate (March
22/06)
Jan 5/05 : init of study in prophylaxis of orthopaedic
surgery infections. N = 120, Data 3Q05. End point Ab
levels, BLA filing 4Q05, Oct 3/05 95% pts reach good
Ab level.
Other target : cardiac valve protheses.
- next generation staphvax ( 5, 8 & 336)
-Human extractive immunoglobulins :
-NABI-HB, MKT →prevents Hep B
-WinRho SDF (anti D)→ITP
-Autoplex T→anti inhibitor for Hemophilia A
- Altastaph Ph II failure Nov 7/04. Stopped Nov 1/05
- Civacir : hu polyclonal Ab for Hep C in liver transplants
acq Phoslo (Ca acetate) : DB > Renagel Nov 03, Kidney
Internat May 04 > Renagel (CARE)
June 04 init Phoslo + Lipitor to prevent calcify in ESRD.
Data end 05.
- Antithymoglobulin, acq from Fresenius April 4/06
NBIX NEUROCRINE BIOSCIENCES (NBIX) -CRFr antag 30775→anxiodepression, ph II J & J discont (liver enz ∨)
-CRF antag NBI34041,,anxiety/depression & Irr BS (July lic to GSK Oct 29/01; $15m milestone 4Q01
-Shelf filing (Nov 29/01) 24/01)
- Secondary Nov 01 : $ 163M -NBI5788APL (peptide from MBP) →MS, ph II Novartis
Indiplon lic to PFE Dec 19/02, upfront $ (APL = altered peptide ligand) terminated March 8/06
100m, milestones → $ 300m (clin trial -NBI 3001(IL4 fusion toxin)→glioma, ph II data
But (Oct 14/02) Taisho abandons all but Japan
success, filing & approvals). Dev funding; $ Dec26/02, N = 32, survival ↗
acq Northwest Neurol., MCR specialist.
175 m credit line post launch; PFE will -NBI 6024(APL)→Diabetes I, ph II, w/Taisho
develop NBIX sales force (200) for Indiplon -MCR4 agonist™™™™™™ Lic from AHP 98 (Ambien patent 2005) then from
+ Zoloft -Indiplon NBI 34060 Indiplon,hypnotic,8ph III init Nov
DOVP, latest patent∏ 2020 !
16/01(non BZD GABA agon). 2 formulations (bed-time
IR : capsules Permanent news flow : 1st ph III data Nov 14/02 on IR
MR & middle of night IR but out of the system by
MR : tablets : p < 0.0001. 2nd IR ph III + April 22/03 (3 IR ph III to
morning; filing 1Q04 a bit delayed N=2200. Launch 1H05.
come, & 3 MR; middle ?)
Ph III MR 15 mg, N = 229. Data released Sept 9/04. Filed
April 11/05 : Sanofi extended release June 23/04 Sanofi files Ambien CR (patent 2007), and
IR Oct 19/ 04, MR filed Nov 22/04
Ambien "approvable". SEPR has refiled June 16/04 -> PDUFA Dec 15/04.
Dec 21/04 : FDA refuse IR (navigation issue)
approved Jan 12/05, but mkt delays due to schedule
Jan 11/05 will also resubmit MR : IR end of 1Q/2Q, MR
IV issues.
June 25/06 PFE terminates 2Q? IR capsules refiled April 14/05. MR Tablets refilled
Sept 6/05 Sanofi Ambien CR approved
- 105 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


May 30/05. PDUFAs capsule May 15/06, tablets March Others : LLY, AHP, Taisho
27/06. ADV COM May 15/06 for both : IR approvable,
MR not approvable : 2 years delay for MR ?

- Oral GnRH antag, ph I

NBY IPO NovaBay antibiotics


NEOL M NEOPHARM (NEOL) -Technologies licensed out :
LEP(liposomal cardiolipine encapsulated paclitaxel), ph →PHA, fails, NEOL could reinitiate
June 04 Chairman & CEO resign II/III →PHA, fails ; NEOL sues PHA (formulation!) April
K LED(lipos. encaps. doxorubicine), Ph II/III 21/02; SEC investigation May 11/04, not relevant
Sept 6/04 : Kapoor (largest shareholder files -Developments :
to fire the Board) LE-AON antisense to c-raf in lip., ph I/II May 04 Amer Ass Neurol surgeons : side effects,
IL 13 ( only K cells have IL 13 receptors) chimeric long term depends on cath position. 5 ongoing
(pseomonas toxin) prot.cintredekin besudotox→glioma trials
2nd line, ph I/II (MD Anderson + CBER) median survival CMA Continuous marketing application Pilot 2
March 16/05 CEO (Greg Young ) resigns 52w. Follow-up Eur Ass Neurooncol Sept 18/06 : best program by Div of Oncology
results in patients with optimaly placed catheters. Ph III
Nov 1/05 new CEO G. Herrera March 8/04 against Gliadel (PRECISE) , continuous April 8/04 manuf by Akzo Nobel
intratumoral infusion. Orphan. To last 18m.N = 300; Prim
forecasted price of Cintredekin : $40,000 endpoint 50% > Gliadel (historic = 28 w), interim at 100 AACR April 03
deaths (reached Nov 21/05, end Dec, review com recom ASCO 03
continue). Interim 160 deaths 2Q06Filing end 06 ? or
earlier : enrol 250 Sept 27/05. Enrol compl April 06.
Could fail (June 22 : cath positioning). Dec 11/06 fails;
survival 36.4 m vs 35.3. March 29/07 FDA requires new
trial
June 11/07 to init nebulized in pulmonary fibrosis
- TR 38 : targets EGFR + pseudomonas, tox, ph I
Mesothelin scFv MAb(+ PE38 toxin)→mesothelioma,
H&N ph I/II
Liposomal SN 38, (active metab of Campto whose
patent expires 2007,$800m/y), ph I/II. Failure
CRC April 2/07
NEUSF NEUROSEARCH (DK) - ACR 16, end of ph II, Huntington
- NS 2359 depression ph II GSK
Nov 16/06 expands GSK alliance - NS 2359 ADHD ph II GSK
- Tesofensine obesity ph IIb, March 7/07 partner delayed
(is it like Nomifensine hepatotox ??). Data Sept 17/07 dose
dependent signif weight loss
NEXL V NEXELL (NEXL), was VIMRx -Cell therapy equipment Baxter owns 20 %
- 106 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


C -acq K Ags from EPMN(march 01) for dendritic cell -Bought CellPro in 98
vaccination
K

NFLD NORTHFIELD LABS (NFLD) Polyheme : pyridoxylated outdated Hb shelflife 12 m, ph


III; Have infused up to 20 Units per patient !
Feb 7/05 Secondary ( $ 67 m) to build plant. In trauma 75% survival rate ( historical comparator 20%). Oct 01 : Amer Coll Surg
BLA filed Aug 29/01. Nov 19/01 : FDA issues "refusal to
file letter". New ph III (endpoint 30 d survival) authorized
March 6/03. N = 720 tmt starts at site of injury. July 04
gets continue nod from monitoring committee. April 12/04
ditto. 3rd interim (250 enrolled). Next at 500. Enrol to be
compl end 05. Dec 20/06 fails. May 23/07 FAILS
NGEN NANOGEN (NGEN) Genetic testing with nanochips Becton Dickinson, Elan, Aventis,Hitachi
NGPI NEOGENESIS (NGPI)
IPO filed Nov 19/01, withdrawn Feb 11/02
NGSX IPO NeurogesX - Transacin (NGX 4010),, capsaicin patch ( 1 application see ANSV
MS, Pacific Growth, Lazard gives 3 months efficacy). 3 ph III (1 fails, 2 good), post
herpes neuralgia, to file EU 2007, US 2008. Int congr
Traded since end April 07 neuropathic pain June 8/07. Reduct in pain 29% vs 20% p
= 0.091.signif data herpetic neuralgie (but takes 2 h to
apply)
Also liquid formulation precl
NKTR Jan 03 : NEKTAR (NKTR), was Inhale - Hu rHuInsulin (Exubera), ph III (since Aug 99), diab I ™PFE ( production of insulin by AVE), end 05
(INHL) & II. Aventis sells to PFE
Jan 07 new CEO from RNAI Already 48 months efficacy and safety(FEV1 & DLCO). copromotion PFE/AVE(3/7 sales share,INHL 20%R)
∗PROPRIETARY DRY POWDERS : In type II Hb A1c 9.7%™7.4%, 90 % patients want to stay May : ATS : less LRTI than in controls !
- PulmoSol : big molecules on "inhaled " rather than return to oral. June 01 : ADA better HbA1c with bedtime Ultralente
- PulmoSphere : small molecules Filing ww 1H03, later, launch 1H04 ( 05….) open label than 2 regular insulins/24h, but more Abs
∗MULTIPLE DEVICES : 1000 ptnt safety trial started May 02 (for 2/3 years) June 02 ADA 28% pts have Abs (4% with sub cut),
- "Inhance" : for insulin 1 case of fibrosis in 1000 pts is not a concern. signif decrease in DL/CO
- "Solo" disposable According to Pr Attali (Aug 30/01) who coordinates
- MDI : for PulmoSphere French trials & has seen the data, this 1 case could be
∗ACQUISITIONS / eventually related, but 3 others have other causes. He PFE dec 18/01 analyst meeting : FEV1 ¬ 1%. July
- Bid for Quadrant ($60m) overtaken by ELN compares activity to that of Humalog (LLY) 25/02 seems ditto in injectable
in Oct 00. July 21/01 : loses infringement Filed EMEA March 04, CHMP OK Oct 14/05, FDA ADA June 07 : 3 years trial "small lung function
case for trehalose stabilisation against accepted for filing March 2/05. Could reach mkt 2006 median decline, reverts on cessation"
Quadrant) EU, 2007 US. FDA panel OK Sept 9/05 (7/2). PDUFA
-Bid (dec 21, 00; $200m, compl. feb 01) for late Oct., delayed to 1Q06. FDA OK JAN 06 (launch
Bradford Particle Design (supercritical fluid mid 06). On MKT May 06. GP launch delayed until Jan
- 107 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


technology ensures longer shelf life). 07. SALES ??
Partners : AZN,BMY, GSK, LLY, PFE PFE analyst conf end Nov 06 : working on
∗Key People : new generation device; Exubera terminated
J. Patton, R. Platz , world experts in Biogen
respiratory protein delivery.
Oct 18/07
-INF β, ph I , MS, suspended Jan 24/02 AVE-Behring. Competition : Bayer (IV,MKT) and
VP regulatory spent 15 years at FDA.
∗May 22/01 : acq Shearwater (operates PPL/Bayer (ph II)
-AAT, ph I aerosol, cong. emphysema,completed April/01;
roughly at breakeven), will split royalties on
ph III delayed. Launch 05
CDP 870 w/ENZN
June 18 /02 Aventis R/D day too LLY (discont Oct 24/01)
enthusiastic→short -Forteo (PTH), ph I , osteoporosis
Patents : 31
Headcount : 339
Dec 17/02 : staff ↘ 10% - Calcitonin (salmon), ph I, osteoporosis
Manufacturing : final steps at
- Tobramycin, ph I, DPI (lung delivery x 3.5, speed X 10),
INHL
w/CHIR
- Amikacin, w/Bayer , Aug 6/07 ph II
-Albuterol, pre, asthma.
SHEARWATER :
#technology for Pegasys,milestones +R
#role in manuf of PegIntron, R
#PEG-hGHr antag (Somavert)for acromegaly,
w/PHA/PFE, BLA OK March 25/03, also lowers ILGF,
milestones + R
# AMGN's Neulasta , milestones + R
# New mol w/JNJ oct 10/01, milestones +R
NMPS MATRITECH (NMPS) Nuclear matrix prot test : better than PAP
NOVC K NOVACEA (NOVC) - DN 101, Asentar, ph III, oral calcitriol formulation, qw could improve safety of Taxotere
IPO filed Feb 13/06 HRPC, + Taxotere QW (ph II N = 250, had shown 49%
listed May 10/06 improvement OS vs Taxotere Q3W alone, p < 0.04), N =
Lead Bear Stearns & Credit Suisse, also 900, init April 14/06, endpoint OS. May 30/07 lic to SGP
Pacific Growth $ 440m; Nov 5/07 HALTED!
CMO & VP Dev were at Genentech Oct 11/07 Pancreas + Gemzar +/- Tarceva
- Oral Vinorelbine ph III to init late 06 (Breast, NSCLC),
lic for US from P. Fabre,
- AQ4N, banoxantrone topoisomerase II inhib prodrug, lic
from Kudos Ltd, to init ph I/II 2H/06 glioblastoma
NPRO NAPRO BIOTHERAPEUTICS (NPRO) specialized in extraction of paclitaxel bulk from yew trees -sells to Faulding
-since July 99, contract with ABT
-complex affair with IVAX
NPSP NPS Pharmaceuticals (NPSP) April 21/04 for EU : Nycomed files EMEA March 05,

- 108 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


merged w/Allelix (dec 99) -recombinant hPTH( 84aa) Preos SC daily→ osteoporosis, OK Feb 06
ph III(N = 2600), 12m interim 2Q03, dosing to last 18 m
Oct 25/02 shelf raises $ 95.8m, MS, ML, (conclusion Sept 03);. Aug 19/02 12m data in rats no →AZN discontinued (LLY's Forteo is 34aa fragment
SSB sarcoma (but 24 m study). NIH trial compares to Fosamax. (e. coli)of PTH,SC daily, $ 7,300/y; induces
Jan 20/02 to merge w/ENZN (MS, SGC). To file 2H 04??? Delayed (feb 8/05) to 1H05, ACR osteosarcoma in rats at 15 m (black box), FDA
Closing June 03, stock exchange (26% Oct22/04 ph III 21% hypercalcemias > 107. March approv. Nov 02, CPMP OK Dec 02; launched , sales $
premium to ENZN shares) : NPS will own 10/06, FDA "approvable" (hypercalcemia). Feb 06 25m/03, 1Q06 87m.. OK for spine, but radial
53%. Term June 6/03 CHMP says OK ! May 3/06 FDA requests new trial demineralistion. April 03 Public Citizen warns
(June 12/06 53% staff reduction) against Forteo.11% hypercalcemias
Sept 22/03 bad data on assoc w/Fosamax Oct 21/02 : commercial lots will be manuf by BI. Bulk by
Synco, F & F by Vetter
- ALX 0600, Gattex, teduglutide (GLP2 analogue) ph II,
lic to Nycomed, short bowel sd., April 6/04 ph III
init; Oct 11/07 misses primary
-AMG 073 Cinacalcet, w/AMGN , calcimimetic, but (A. AMGN filed Sept 9/03, approved March 04
Soc N Oct 01) vomiting, resp infect, pain, 10% royalty
-NMDA antag from spider venom (NPS 1506)
→stroke, ph I
→head trauma, pre
-Metabotropic glutamate rec. program w/AZN (03/01)
NRGN NEUROGEN (NRGN) -NGD91 3(GABA modul)→anxiolytic, ph I PFE (disappointing, Dec 20/01), will drop
-NGD97 1(GABA modul)→alzheimer, ph II PFE, failure Nov 12/02, will drop
March 20/04 $ 100m PIPE -NGD96 3(GABA modul)→insomnia,ph I March 11/02 PFE
Oct 3/02 staff ¬ 10% -Melanocortin recept antag→obesity, ph I Nov 13/06 PFE
-NGD98 1(CRFr blocker)→depression, pre, w/AVE (Dec
20/01), returned Sept 16/04
- NGD 2000-1 : C5a receptor antag, ph II. RA primary
endpoint CRP (fails June 16/04); asthma.(fails Jan
04)
- NG2-73 insomnia, ph II, Aug 2/07 confusing data
- NGD-8243 ph II Lic to MRK, vanilloid receptor antag for
pain
- Aplindore, ph II to start. Park. DA partial agonist (D2)
NRGX Neurologix (OTCBB : NRGX) - XIAP (X linked inhibitor of apoptosis) in AAV, active in
animal model of Huntington (Nov 18/05)
- Gene therapy w/GAD (glutamic acid decarboxylase)
AAV, ph I published June 22/07, N = 12; Good.
PARKINSON
NRLS Neuralstem, OTCBB

- 109 - printed : 03/07/10 (d/m/y) 08:07


TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


NRM NEUROCHEM (TSE:NRM, - Alzhemed, ph III, 3 amino 1 propane sulfonic acid, fails Lundbeck discont Oct 12/01
NRMX Nasdaq Aug 27/07
Bellini was Chairman - Alzheimer vaccine
- Alzheimer dg , w/Nycomed Amersham
US IPO Aug 20/03 w/UBS (NRMX) - Fibrillex, Kiacta ( 1-3 propane disulfonate) ph II/III ( n =
183) to end Dec 04, Orphan, “ amyloidosis, lic to Centocor
Dec 22/04. Certainly the most clear cut possibility for a
success. April 19/05 misses endpoint (kidney function p +
0.06 and death). Aug 22/05 rolling filing initiated,
completed April 18/06, PDUFA Aug 13/06 :
"approvable" request more efficacy data and manuf.
New filing , PDUFA July 16/07 "approvable" again
NSTK NASTECH (NSTK) - calcitonin nasal spray , aNDA "not approvable" July 06
- morphine nasal ph II
- PTH ( 1 – 34)nasal ph II; ph III to init 06 N = 600 – 800,
Nov 8/06 lic from City of Hope rights to vs LLy Forteo (non-inferiority) on bone mineral density on
"Dicer substrate RNA interfrence IP and a single bone site. Lic to PG Feb 1/06. Terminated Nov
technology" 5 rights exclusive. 7/07
- Peptide PYY ph I, nasal, anti hunger, obesity. March 2/06
: MRK drops.
- siRNA anti TNF from Alnylam , pre
- siRNA anti H5N1 : G 00101, pre
NTEC NEOSE (NTEC) ∗Therapeutics ("GlycoTherapeutics"), w/ BioGenerics
AG
Incorporated 1990 GlycoPeg / NE 180 EPO in baculo, IND 3Q04, on hold
Public 1996 FDA July 05, ph II Jan 07 Switzerland
GlycoPeg/G-CSF in baculo IND 3Q04, delayed, partnered
w/ Biogenerix AG (Ratiopharm)
GlycoPeg VII, VIII, IX w/NN; precl
-Carbohydrate identical to cell mb site for H. pylori :
discont in ph II, 1997
-Carbohydrate NE 1530 for otitis media : discont in ph II,
1997
-Collab w/Bracco for imaging : discont
-Collab w/Neuronyx since June 00 on glycolipids for Park.
(res)
-Collab w/Novazyme for lysosomal storage disease,
restructured in March 01 (NTEC is no longer in JV)
-Manuf GMK ganglioside conjugate vaccine for BMY.
BMY terminated May 15/01 & reassigned to Progenics.
ECOG stopped but Ph III ongoing w/EORTC (too many -partners : LLY, BGEN, WYE (Dec 01) for rPSGL-Ig

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


centers, still recruiting, 7 years follow up) (P selectin glycoprot ligand, linked to Fc), ph II,
∗Neutraceuticals ("GlycoActives") coronary reperfusion sd : terminated May 10/02,
Carbohydrate bulking agents for McNeil (JNJ) & Wyeth failure ph II§
(AHP) infant feed formulas. Construction of pilot plant is
slow.
∗Enzymatic carbohydrate modifications
("GlycoAdvance") acquired in 99 from Cytel : technology
to remove Galactose, & to increase sialylation. Not
impressive, could at best generate low contractual
revenues. Could apply to plant MAbs & EPO. Applied to
TP 10 from Avant
NTGC NETGENICS (NTGC) bioinformatics
IPO filed 3/13/00 (CHQ)
NTI Neurobiological Technologies Arvin, ph III, stroke

NTMD NITROMED (NTMD) BiDil filed Dec 23/04, PDUFA June 23/05
April 13/05 Frank Douglas appointed to ADV COM JUNE 16 OK 8/8 (but lupus side
Board effects)
Jan 6/06 Manuel Worcel leaves
FDA OK June 23/05 : heart failure in blacks.
March 21/06 : CEO & CFO resign Launch July 15/05. Weekly Rx Aug 12 : 250;
sales 3Q05 : $ 1.1m
NUVO NUVELO (NUVO) - Alfimeprase (w/AMGN, who opts not to continue on Nov recombinant fibrolase (from Southern Copperhead
1/04) ph II for acute periph arterial occlusion & catheter Snake venom) , direct fibrin lytic enzyme, not a
traded since March 3/04 occlusion. Ph III PAO (NAPA II)init April 18/05, N = 700, Plasminogen activator
endpoint : avoidance of surgery in patients slated for Lic to Bayer Jan 5/06 ($ 385m) & 40% of dev costs
R & R 05 : presented by CFO who says surgery. NAPA III SPA n = 300 identical structure, Sept
"anticoagulants" for "thrombolytics". 05 init ph III central venous cath. occlusion. Jan 31/06 lic
to Bayer ($ 385 m). Dec 11/06, fails. June 27/07 Bayer
terminates
- r NAPc2, ph II, DVT, acq from DNDN, and ph II a for
acute coronary sd , ANTHEM, reinitiated May 04 (TIMI
32), data 1H 05. Jan 5/07, 2nd line m CRC, ph II. March
27/07 1st line CRC w/Folfox Avastin
- ARC 183 aptamer direct thrombin inhibitor, ph I, for
CABg, t ½ 2 min (no reversal agent needed). Terminated
Sept 22/05
- NU 206 (R Spondin, Wnt agonist), w/Kirin, for gut
epithelium regeneration
NVAX NOVAVAX (NVAX) -cream transdermal deliver. Estros(phIII) & Andros(phI)
-anti HPV vaccine (with rVLP)
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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


(VLP=Virus like particles)
- anti avian flu vaccine, pre, VLP
NVLT K NOVELOS (OTC BB NVLT) NOV 002, ph III, NSCLC, w/chemotherapy, oxidized
glutathione based chemoprotectant (MKT in Russia)
US ph III w/Taxol & Carbo 1st line NSCLC init June 06, N
= 850
NVS CHIRON (CHIR) - Pulminiq (inhaled ciclosporin) for lung transplant. Ph III (have divested Dg to Bayer Sept 98)
NVS owns 42%. N = 26, primary endpoint survival NS; FDA questions →incredible $100m shipping to UK!(one time event)
V Nov to acq (Sept 1/05 , $ 4.5 B blinding ! Adv Com June 6/05 : 8/8 Schering AG/Berlex
Oct 31 /05 $ 5.1B, closed April 19/06 -Vaccines : $344 m/00, 357/02, 687/03. Fluvirin UK ban
K Jan 8/02 : acq MATX (for Tezacitabine) $ Oct 6/04
61m -Betaseron : $99 m direct + royalties. May 5/01 Amer
Acad Neurol 12 months MRI for new lesions : Searle
Oct 3/03 : acq Cubicin for ex-US Betaseron 15%, Avonex 30%
-Proleukin : $112 m, 03 : 115, 2004 129m Proleukin competition : human expressed IL2, pre,
-TFPI / Tifacogin→sepsis, ph III, N = 2000, Nov 21/01 fail Apollo Life Sciences, Australia
to reduce 28 day mortality( but the 225 subset with CAP
& not receiving Heparin benefited); Xigris reduces from
31% to 25%. 18 May 04 reinitiates ph III in severe CAP,
Acq. (Aug 15/00)PGNS for $700m N = 2100, primary endpoint 28d mortality :Captivate;
data 4Q06. Dec 19/05 Indep. Com says "continue". →→→ presented ACC 1H00, is bovine,hu rFGF was
Sold Gene therapy manuf facility to CEGE -FGF2 (antiapoptosis)→coronary dis.,ph II lic by Scios in late 99……
ACC : fails to meet end-point
Acq Powderject May 19/03 $ 880m, which -VIII gene ther.,ph I, retro, IV RZYM
has contract with UK for Bavarian Nordic -antiangiogenic ribozyme (Angiozyme)→K :I/II
small pox vaccine - TOBI, patented formulation for nebulizer Tobramycin:
$90m/01, 147/02, 172/03, 2Q04 : 51
marketed in US & UK(EU mut. rec Aug 7/00).
-PA-824, resistant TB, ph I lic to TB Alliance (Feb 02)
- Tezacitabine (fluorodeoxycytidine), K, ph II, was HMR,
failure oesophageal K March 20/04
- HBV-MF 59, hep B, ph II, halted Oct 02
- Melanocortin 4 R, res, lic to GSK (Jan 8/03), obesity.
- CHIR 258, ph I, pan anti kinases VEGF, PDGF, c-Kit,
Flt3
- CHIR 265 RAF kinase inhib, melanoma, ph I (init April
11/06)
NWBT V Northwest Biotherapeutics (OTC BB - Dendritic cell vaccine (auto + BCG)to PSMA (DCVax
NWBT) prostate), ph I/II (AACR 02 : N=33, 1 a month x 4 ∏ 55%
C IPO filed Aug 14/01(Unterberg,Tobin) ORR; ph III started (N=495) filing 2004…enrol suspended
Traded since Dec 17/01 Oct 12/02 (financial reasons). Restarting Feb 05.
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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


K - DC Vax Brain ph II in US since Dec 06, to conclude end
08. ATU in Switzerland
- Dendritic cell approach to lung (IND), renal,
- Immature dendritic cell (+ auto tumoral peptides)for
glioma (DCVaxBrain) ph I (AACR 02 : 30% progression
rate at 1 year, hist Comp 85%),ph II to start N= 90
- HuRx Prostate : fully Hu Mab to prostate, ph II w/Medarex
- DC-Vax direct : autologous dendritic cells, pre
- patent on CXCR4 gene (activated by Hypoxia ) in K
cells, that migrate looking for ligand (CXCL12 (especially
secreted in bone & lung) precl (Feb 06) lung metas
reduced 75%
NWRN IPO NEWRON ( SWX) - Safinamide (lic to Serono/Merck), Park, , DA uptake MKT leader Mirapex (DA agonist) $ 300 m/year
Chairman Rolf Stahel inhib, Na channel blocker, MAO-A inhib Teva/Lundbeck MAO inhib to reach 140 m In 2010.
Lehman/MS 1st ph III good data June 19/06, 2nd ph III init Nov 27/06. Many generics
Traded Dec 11/06 Data 3Q/07 : Aug 22/07 efficacy endpoint not met
Park :>1 M US patients - Ralfinamide, pain, ph II. Neuropathic pain vs placebo.
July 2/07 NS reduct in severity of pain; retinal anomalies
in animal tox
NXXI AMBI (AMBI) Nutrition products (Lite Bites)
change to NUTRITION 21 (NXXI)

OGXI IPO ONCOGENEX antisense


K filed Dec 13/06, withdrawn March 7/07 - OGX-011 antisurvivin / clusterin w/ISIS, 5 ph II
RBC ongoing; prostate, NSCLC, Breast
- OGX 427 anti HSP 27, IND for 2007
- OGX 225 decreases product of ILGF binding proteins,
pre
OMRI IPO OMRIX Human plama derived Thrombin Evithron; approved Aug
priced April 21/06 28/07
ONC Oncolytics (CND) Reolysin : squamous cell skin K, ph I/II intratumoral + RT
: PR, Oct 1/07
ONCOB OncoMethylome Sciences (Belgium) - PCR technol to measure DNA methylation competition Epigenomics
- Urine based prostate cancer test
ONTY ONCOTHYREON was BIOMIRA (BIOM) -Theratope(STn-KLH) :vaccine for Breast K,PhIII, fast w/Merck KGaA (returned)
V track design. by FDA on May 8; CA 125¬
May 4/01 : partnership w/Merck KGaA (enrollment 900 pts ended April 2/01). Interim anal
K worth $ 150m for Theratope & BLP25 2H01(might miss endpoint)delayed (oct 22/01)to 3Q02
(except in Israel:Megapharm). Discont. TTP (Sept 02) misses endpoint,; final report mid-03on
survival: misses, Dec 8/03 subset misses TTP
Staff ¬ Oct 02 - Theratope meta colorectal ph II enrol compl Oct 9 02, N
- 113 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


= 20. Data mid 03.
Henney CEO since Aug 06. -anti NHL vaccine ™Biovector SA :Terminated by BIOM April 2/01
Oct 30/06 acq ProIX -Stimuvax, BLP25 vacc (25 aa of Muc1 in lip.)ph II→ W/Merck KGaA
prost., NSCLC rand. open 2nd line, stage IIIb/IV, N = 171.
Median survival treated 17.7m > control 13.3. IIIb
subgroup median survival > 23m (Dec 3/04) But ASCO
05 p = 0. 09; May 1/06, anal IIb OS 30.6m vs 13.3. Jan
2/07 new ph III in stage III stable after at least 2 cycles of
Pt/Rx + Best standard of care. Wrap up YE 08. PLacebo
control, endpoint survival. N = 1300 (w/Merck AG Jan 06)
- PX12, Ph II, thioreduxin inhib (refract pancreas K)
- PX 478 Hif 1 α inhib, pre
- PX 866 PI-3 kinase inhib, pre
- PX 316 AKT inhib, pre
ONXX ONYX (ONXX) -Onyx 015,modif. Adeno kills p53 deleted K cells, milestone from WLA/PFE (May 23/00)
K oct 12 : pricing of secondary $15 ph III H & N initiated June 6 Aug 7/01 : reacq H & N rights from PFE. Sept 16/02
to be manuf by XOMA (delays ++) PFE terminates. Dec 18/03 lic to Sangamo
Aug 14/00 : Leonard Post becomes CSO ph II→50% shrinkage
liver metastases : ph I/II low dose : 155d survival, high Aug 2: res agreement w/EPTTCO Ltd, a prodrug
Oct 30/01 : staff cut 30% dose 342 days (ASCO/01) specialist (015 could be prodrug vehicle)
Feb 28/02 : resources reallocated to BAY pancreatic K : ph II. All terminated Jan 27/03
429006 -Sorafenib, Nexavar, Raf kinase inhib & VEGFR2 & Bayer 50/50 (but Bayer responsible for clin dev)
PDGFRβ (BAY 439006, Nexavar, ORAL bis aryl urea), ASCO 03 : very preliminary N = 118. See AACR
July 14/03 secondary $ 80m ph II, hepatocarcinoma, colon, melanoma Oct 02 (in patients T cells, decreases ERK
Feb 3/04 shelf (filed Dec 13) raises 156 .RCC pivotal ph III 2nd line with SPA init Oct 27/03.( Ph II phosphorylation).
Price could be $ 25,000/year interim had 44% ORR). 100 sites (US, CND, France), N =
Sales 3Q06 $ 45m, FY 06 153.8, 3Q07 104 900, survival, fully enrolled end 04, 12 m follow up. Only Might file ph II end 04 ?????NO (Oct 26/04) Ph II
ECOG 0 & 1. March 22/05 interim : longer PFS, filed July ESMO Nov 04, N = 502, 202 w/RCC, 65 stabilized
PFE SU 11248, Sutent ph III, halted for 11/05 for advanced RCC, PDUFA JAN 11/06, approved by BAY, rand to placebo/verum : better absence of
efficacy Feb 05, success in GIST, PDUFA Dec 20/05, PRICE $ 4,333/month, but ph III will progression w/Verum.
Feb 06. Ph III RCC data end 07 continue until survival endpoint, but unblinded May 05 &
all pts crossed to drug. ASCO May 16/05 PFS 24w/12w, Sutent (PFE) filed Aug 10 for 2nd line GIST and
PR 2% (Sutent 40%). ECCO Nov 3/05 : interim anal at RCC. EU filing 3Q05. US OK Jan 27/06, CHMP OK
220 deaths. p = 0.018 which is NS.: anal is planned at April 28/06, July 27 "conditional approval". Full
540 deaths and requires p < 0.0005 EU filing Sept 12/05, approval Jan 8/07. Feb 8/07 approved 1st line
CHMP OK April 28/06, EU ph III 2nd line RCC to init. Others : PFE Axitinib (ph II ASCO 07), GSK
RCC 1st line w/IFN ph III data YE 05 NS Pazopanib (ASCO 07) PR 27% in RCC
-NSCLC : EORTC Nov 17/05 serious side effects
Ph III w Carbo/Taxol init Feb 16/06, N = 900 (squamous
& adeno). Data 2008

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


. HCC .Advanced hepatocarcinoma 1st line, ph III init
HCC : 19,000 new cases/Y US, 32,000 EU March 8/05, enrol compl June 06, survival; April 18/06
EMEA grants Orphan designation. Data YE 07, Feb 11/07
stopped at interim N = 602 (in ph II OS was 9.2m). ASCO
07 10.7m vs 7.9 p = 0.0006. Filing being prepared.
.Melanoma ph III init (?)end March 05, N = 800, 1st line,
w/ carbo-Taxol, stages III/IV, powered for 30% survival
increase (9m to 12 m). May 13/05 init ph III 2nd line w/
carboTaxol, N = 270, PFS NS Dec 4/06. Dec 2/06 fails;
ongoing with DTIC
-Agent killing Rb deficient cells, pre, IND 4Q01?
- Small molecule inhib of cell cycle kinase enzyme, w/PFE
OPHM ORAPHARMA (OPHM) Mynocyclin in proprietary periodontal biodegradable
microspheres delivery system, ph III

OPTR IPO macrocyclic AB PHARMACEUTICALS - Difimicin, OPT 80 (RNA polymerase inhib) oral, 2 ph III Competition : oral Vanco (Viropharma), Genz,
filed Nov 11/06. Traded Feb 9/07 (init March 24 & May 1/07, N = 664 x 2) Clostridium Oscient (ramoplanin), Replidyne (precl), Targanta
Piper Jaffray, R & R difficile associated diarrhea (reacq 1Q07 from Par Pharma (oritavancin)
PAR 101)..Remarkable in vitro data. Clin data 1H/08,
PIPE Oct 25/07 $ 35.9M Filing 2H 08. Ph II usually no or low systemic absorption.
- Plurifloxacin, ph III, traveller's diarrhea
ORCH ORCHID BIOSC. (ORCH) SNP scoring : Genetic Bit Analysis Bayer, MLNM, PEB, BMY, LLY, AZN
IPO, traded since May 5 -creating portfolios of "medically interesting SNPs"
-screening populations receiving best selling drugs
- RxScreen mouth swab to detect genetic risk of sensitivity
to drug
Also Agro mkt correlating crop growth & SNPs, hence
hybridization rather than GMO
OREX IPO OREXIGEN - Contrave, to init ph III (Naltrexone + Zyban), N = 1500,
IPO filed Dec 20/06 filing 2009
ML - Excalia, NDA expected 2010 : Zyban + Zonisamide,
Traded end April 07 which already has 2 deaths by Stevens Johnson
ORG ORGANOGENESYS (ORG) Apligraf(grown from foreskins), MKT in US →AVENTIS
C for wound healing;
FDA june approval for diabetic foot ulcers
ORPH Orphan Medical (ORPH) Xyrem (oxybate;gamma hydroxybutirate "date-rape
drug"), ph II, narcolepsy/cataplexy; Adv Com cancelled
(march 4/01). July 3/01 approvable letter w/safety
concerns

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ORTC ORTEC (ORTC) Wound dressing with bovine collagen
OSCI AB OSCIENT (OSCI) , was GENOME -microbial & fungal genomics(PathoGenome database) Aventis, Bayer, SGP∏ ICAAC ?
THERAPEUTICS CORP (GENE) -Asthma genomics SGP
-Osteoporosis genomics AHP
March 7/02 : sale of $ 15 m convertibles to -Gastrointest. genomics AZN
accelerate Ramoplanin. -Ion channel genomics ICAgen

Sept 26/02 : staff cut 17% - Ramoplanin; ph III, sachet oral prevention of VRE sepsis. Lic (oct 01) from Biosearch Italia after failure of
33% of 950 pts enrolled. Filing 2004 : delayed (slow IBPI.
Nov 18/03 : to merge w/Genesoft (with FDA enrol.). July 04 : enrol closed at N = 650 ICAAC 01 : 1 in vitro poster
approved Factive, Gemifloxacin) Capsules bioequiv Dec 10/02;
Feb 20/03, init ph II C. versus Vanco, difficile
Feb 9/04 Follow on 88m diarrhea, N = 87, data Aug 11/04 : response 71%,
Vanco 78% Fast track Feb 18/04; was supposed to file
2005……..
- Factive approved ( Sept 9/04)for CAP & Acute
exacerbations of chronic bronchitis caused bt S.
pneumoniae, H. Influenza, H. para, Moraxella
OSIP K OSI (OSIP), was Oncogene Science CCP 754(H ras farnesylation inhib)→bladder K,ph I/II acq. certain assets from Cadus, hence links with
Sir M. Richmond, GLX, elected to Board CP 663427 (ras farnesyl inhib) K, pre Solvay
CCP 547632, anti VEGFr(Flk1 TK),ph I
Bob Ingram (ex GSK) chairman Jan 7/03 CP-724714, oral anti HER2 tyr kinase inhib , ph I, w/PFE PFE(returned)→ AACR (April 6-10/02). Term Dec 12/02
Issue of $ 200 m convertibles (Jan 29/02), $ OSI 774 (erlotinib),Tarceva oral EGFR inhib & decreases N of
40 m repurchased Aug 29/02 EGFRvIII that dimerize. Ph II alone in refractory NSCLC(C+PR 24%) PFE
Sept 3/03 issued $ 135m convertibles -ph III refractory (2nd & 3rd line)BR21 , 150 mg/d controlled (against
"best supportive care", survival endpoint (Iressa refractory had median
AZN filed (Dec 28/01) a ph II on refractory →DNA/Roche; PFE(term. because of FTC :WLA), in
survival 6 m), ↗ from 330 to 731 pts (Sept 25/02, now powered for 33%
NSCLC, with IRESSA gefitinib, US will share commercial costs & profit w/DNA. In
difference rather than 50%) →enrol init Oct 2001 & compl Jan 30/ 03
transformed into rolling. data 2Q/04, approval 2H04? (fast track Sept 24/02). EU royalties low 20ies.
Reviewed at Sept 24/02 ODAC : Rolling filing init Jan 21/04 (clin data April 26/04, > BSC for survival, ASCO 03 : -glioblastoma ph I +/-
- 11/3 "a 10% response rate is reasonable to TTP, ORR, but BSC is < Taxotere !!; data at ASCO, 6.7m/4.7, p <
nd
0.001 NDA completed Aug 2 , PDUFA Jan 30, 2005.FDA OKNov temozolomide (Iressa also in glioma)
predict clin. benefit"
18/04 $ 2,026 "locally advanced or metastatic NSCLC after failure of w/ABC2 clinical network : ph II init Aug
- 9/5 "symptom improvement cannot be at least one prior CT. But only 10% of patients have reduction of tumor 9/03 (Iressa Jan 8/04 glioblastoma fails)
adequately evaluated without a control size (BWFW Dec 27/04) - bronchioalveolar K ph II
arm".(there was no control group & end Sales 4Q04 : $ 13.3. 1Q05 47.6, 2Q 70.2, 3Q 73.2, 4Q 84, - Tarceva + Avastin NSCLC
points are T. response and symptoms). FY 05 275, 1Q06 93, 2Q 103, 3Q 100, 4Q 109, 1Q07 102 - Metastatic colorectal ph II
Also too many adenocar. Eligibility ? 25% (US) 91 (ROW); 2Q07 ww 212; 3Q Roche 126.9 US 101
rashes. Aug 26/04 : filing in refractory to EU. June 27/05 CHMP OK "when
PDUFA : Feb 5/03 delayed (Jan 9/03) to prescribing Tarceva factors associated with prolonged survival Needs lower dose than Iressa
May 5/03 : approved ($1,872 /m; 22,000/y) should be taken into account. EU OK Sept 21/05. EPAR Nov 3/05 Carbo/pacli mean survival alone 9.5 m
- New ph II dose escalation refractory NSCLC init Nov 10/03
for advanced or metastatic NSCLC resistant - New ph II init March 19/04, 1st line, open, rand, versus carbo/taxol, N = Gemzar/cisplat alone mean surv 8.7
to Pt or taxotere (non controlled ORR trial 102, poor performance score April 10/03 : for Tribute, Tarceva to be
Ideal 1 & 2), survival trial ongoing (data - Sept 13/04 init open label expanded access to BR21 discont in case of "progression". Could be

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


2005), acne : 25% -NSCLC naïve 150mg/placebo endpoint survival (bar at 33% >) : linked to Taxotere subst to Taxol increasing
US(TRIBUTE) ph III init Jul 01 enrol compl Aug 02(N=1000, +
lung risk
Iressa has no dose effect (500mg<250 in ph carboplat & paclitaxel)fast track May 15/02 results Oct 1/03(TTP ↗),
EU(TALENT) ph III init Nov 20/01enrol compl Sept 02 N =
II), ph III is less powered than Tarceva (3 1200(+gemcitabin e/cisplat) ;completion end 02, results Oct1/03,
arms), which has much better →failure to meet 25% ↗ survival Pancreas Gemzar alone mean survival 23w
bioavailability. pancreas first line ph III init Oct 01,enrol compl Jan 28/03 → ASCO 02 (ovary K) : nl efficacy & tox
(N=800,w/gemzar, downsized to569)data Sept 20/04, end point survival More Iressa(filed CPMP Feb 11/03) :approval ??
GILD oncol acq ($ 130 m cash + 40 in 6.4m/5.9 p = 0.025, filed April 2005. No difference in ORR, 1 year interst. Pneumonia Japan (125 cases, 39 deaths)
survival 24% vs 17%. Filed May 2/05, ODAC Sept 13 reviewer critical
stock), dec 01 but OK 10/3, PDUFA Nov 2 : approved Nov 2/05, JULY 30/06 CHMP
-ESMO Oct 02 : Intact 1 (non US, N = 1093, gemcitabine + cisplat,
CLPA Feb 10/02 : to acq. OK JUne 11/03 NO
no diff survival 250/500/CT alone); Intact 2 ( US, N = 1023,
carboplat + paclitaxel). Feb 10/03 AZN files EU same data as FDA
March 12/03 acq from SRA US/K - ovary (PR 10%), (IDEAL 1 & 2). 2002 sales japan $ 62m
Novantrone for $ 55m (sales US 02 $ 80m, - H&N→ASCO 01, 6%PR, 80% rashes. ISEL data Dec 17/ 04 failure, N = 1692, vs placebo. But (Dec
55 in K) ph II,
-NX-211, acq from GILD (Nov 01)who acq from GSK, 27/04), BWFW, Rick Pazdur suggests exploratory
liposomal lurtotecan (topoisomerase I inhib), SCLC, ovary trial in mutated patients. Jan 3/05 strong OSIP insider
Dec 21/04 class action suit on misleading (N = 80, comp to Topotecan).Data 2H03 sales announced, Jan 4/05 AZN withdraws EU filing.
survival statements (BWT Dec 21/04) - OSI 7904 L (liposomal thymidylate synthase inhib, was ODAC March 4/05 to discuss. march 07 : 100
Aug 22/05 : to merge w/EYET. Closed Nov GW 1843) ph I tissue samles to ViroLogic for mutation search.
4/05 $ 935m -OSI 7836, Gemzar like, ph I, nucleoside analogue (in Data June 05. April 19/05 NCI halts an Iressa trial
vitro > Gemzar). "consolidation in stage III NSCLC after 1st line chemo/Rx"
- Gelclair : oral gel for mucositis (from CELG) June 17/05 Iressa label change
- OSI 906 ph I, oral inhibitor of ILGF1-R pathway
- PSN 9301 DPP inhib , ph I, diabetes
Oct 2/07 partnershi w/Aveo for EMT (c-MET ?°
OSIP Eyetech ( EYET) Macugen intraocular (Pegaptanib Na),aptamer lic. from PFE Dec 18/02, upfront 100m, 195 m milestones on
Gilead ($ 32m, April 2000); anti VEGF, ph III, ARMD submissions & approval, 450 m milestones upon
Filed for IPO Sept 15/03 (EYET), ML, intravitreal every 6 weeks reaching certain sales levels. Pfe funds Dev.
MSDW. Jan 30/04 : $ 136m. Nov 03 Amer Acad Opht : fails to show > Visudyne Filing June 17/04, Adv Com August 27. Sept 20/04
Nov 8/05 : Lucentis (DNA) > Visudyne update provided to FDA; PDUFA DEC 17 OK Dec
20/04 EU approval slated Jan 06
OSIP CELL PATHWAYS (CLPA) -Aptosyn/exisulind (oral cGMP PDinhib):famil. -lawsuit against CLPA (March 13/01) for false
polyps:NDA filed Aug 99⇑ rejected sept 25/01 statements
Being acquired (Feb 10/03) $ 32m by OSIP _assoc w/Gemzar
(share exchange, 58% premium) _assoc w/Xeloda -LLY
_assoc w/Taxotere,NSCLC N = 600,ph III init 03/01& -Roche
suspended Oct 5/01, resuming Nov enrol compl March -Aventis
6/03; endpoint is survival ↗;prostate ph II
- CP 461(2nd generation Aptosyn), ph II, CLL, RCC
-Apoptosis inducers
-Nilandron(acq from AVE july 5), MKT ($14m/Y)

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS

OSIR C OSIRIS * Adult banked allo mesenchymal stem cells (from Bone verum placebo p
Marrow) for IV : arrythmia 9% 37% 0.025
files for IPO May 12/06 - Prochymal, ph III, GVHD, Crohn overall 42% 11% 0.027
- Provacel (IV) ph I, Myocard Infarct w/ Boston Scientific, improval 6
traded since Aug 06 completed N = 53, April 5/06, data ACC March 07 months
July 26/07 partnership w/GENZ for Acute - Chondrogen (adult mesenchymal stem cells), ph II init eject fr 25% no
radiation sd". To focus first on Prochymal April 2/05 for meniscus repair; Feb 13/07 no efficacy. Nov improval
27/07 : 1 year data, vs hyaluronic acid, knee, signif. FEV1 17 points 6 points < 0.05
- Osteocal, bone matrix for grafts, MKT since July 05
- Trial for Crohn

OSTE C OSTEOTECH (OSTE) World's largest processor of allograft bone tissue : 500 acq. 90% of OST Developpement (Clermont Ferrand)
recipients every day :
Grafton, MKT in US as gel and plasticine for surgeons 30/9/02 : voluntary retrieval of some tissues.

OSTX OSTEX (OSTX) Urine test for bone resorption (cross-linked telopeptides)
OXB K OXFORD BIOMEDICA TROVAX poxvirus (MVA) delivers gene for 5T4 (highly w/Sanofi
expressed in RCC) ph III TRIST init (Jan 10/07). ASCO
07 N = 48/53. Alone or wIL2 or w/IFN : 2CR, 3PR, 19SD
lic to Sanofi $ 690 m, CRC ph III to initiate
OXGN K OXIGENE (OXGN) -Combretastatin(slightly modified Distilbene, which is BMY(dec 99). Terminated Oct 25/01
mkt since 1945 (antiangiogenic)™K,phI/II (3 trials, all
with severe neurol side-effects-ataxia-and one with 3
JV w/PPHM (what happened to CEO ?), responses/14pts); AACR posters only in vitro. Dec 5/05 ph
ARCUS, for vascular targeting agents Ib w/Avastin to initiate. Ovary ph II. mNSCLC to start
(Dec 05) in UK w/rx + CT
June 10/03 : PIPE $ 15m -Sensamide (metoclopropamide/Primperan)ADPRT inhib,
radiosensitizer, ph II glioma∏disappointing BMY terminated Oct 01 (cardiac tox ??)
Jan 14/04 PIPE (R & R) 22.3m - Combretastatin A4 prodrug ph II thyroid K w/doxo
cisplat Rx. Fast track, orphan. Disrupts tubulin of
endothelial cells (like ZD 6126). To enter I/II in combi for
H& N, Lung, prostate, ARMD
-Neusensamide (new formulation) ph II/III NSCLC
∏disppointing (1998)
-Declopramide (new name for Sensamide), NFκB inhib,
ph II ongoing
- Cordycepine, ALL, halted in 99 for toxicity.

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


OXIS OXIS (OXIS), Nasdaq & LNM -Dg Kits for Free Radicals
-Glutathion peroxydase mimetic→Ulc.Colitis,ph II
PANC PANACOS (PANC) PA 457 , bevirimat, AIDS, Ph II once a day pill (not
formulated yet), IIb to init 2Q06 HIV maturation inhib
(blocks conversion of capsid precursor p 25 to p 24. Dec
20/06 poor bioavailability
PARD M PONIARD was NeoRx Corp (NERX) -STR →myeloma, phIII suspended nov 7: TTP/HUS
(STR=skeletal targeted radiotherapy, with Holmium), hold
Jerry McMahon, PhD, CEO, key to discovery lifted April 24/03, to reinit 1Q04 at lower doses.. Term
K of Sutent when at Sugen May 9/05
-Pretarget™(avidin conj MAb then Y/Biotin
SCLC is 20% of lung cancers NHL, ph I
Lung,prostate?
- NX 473/ AMD 473/ Picoplatin IV, acq from Anormed
(had been returned by AZN) April 04, Ph II init 05 SCLC
vs Topotecan, N = 75 (2/1), 2nd line. Trial to last 12 to
18months. April 25/06 expanded. Nov 06 median survival
26.7 w (historical 13-14, GSK; 16-20 NCCN)
SPA Jan 3/07, ph III, 2nd line N = 400 vs BSC, primary
OS, filing 09, lic to Pharmion. Init May 1/07
*HRPC, ph I
*CRC ph I ASCO 07, FOLPI : much less neurotoxic
- Kinase inhibitors (w/Scripps Florida)
PCOP PHARMACOPEIA (PCOP) -Combinatorial chemistry, with proprietary bar code
The leading profitable technology platform -Modeling, simulation, rational drug design, lead
company optimization
-In vitro pharmacology
- In house molecules :
- DARA dual acting angiotensin & endothelin receptor JAK 3 competition : PFE CP-690,550 RA ph II
antag, precl
anti JAK 3 immunosuppressant, res, lic to WYE Jan
4/07
Merger w/EOS term Jan 17/02 anti integrin ARMD, res
adenosine A2a receptor modulator (Parkinson ), res
CCR1 receptor modulator (MS, RA)
anti CXCR2 COPD, ph I
anti p 38, RA, Ph I, w/BMY
PCYC PHARMACYCLICS (PCYC) uses texaphyrins + Me for photodynamic therapy :
-Xcytrin(Gadolinium texaph.)→brain metas, ph III non
Cost per pt. : $ 10,000; blinded(N= 428, but 28 open) ASTRO. Nov 7/01 : protocol shown.
US : 170 000 brain meta cases/y (fully enrolled 03/ 01,Dec 13/01 : fails to meet primary ASTRO 02 : Oct 6-9/02: 12m data on NSCLC meta :
- 119 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


endpoints.Jan 8/02 improves time to neurol progression in TT neurol P 7.4m/not reached (p = 0.048); CNS death
pts w/NSCLC metas; all groups 3.8m/4.3m (p = 0.018); ¬ 51.5%/32.5% (p = 0.02°
NSCLC 3.7m/5.5m (p = 0.025). FDA April 02 meeting :
Competition : requested to conduct new specific ph III (init Jan 7/03)
- Dusa's Levulan SPA (special protocol assessment) SMART N=550, time
- Miravent's Purlytin to neurol progression (powered for 1 m improvement). ⇑ASCO 01 : ph II glioma median survival 17.3m
- QLT's Photofrin Filing 2005/6. Enrol compl March 14/05, Dec 19/05 : fails.
FILED DEC 22/06. Feb 21/07 "refusal-to-file" letter. April
4/07 "filing over protest"
→Glioma ph II enrol compl Aug 2/ 01
→ NSCLC ph I (w/Taxotere)ph II init Sept 05, 2nd
line, RECIST
ASH 04 : poster N = 13, 3 might respond ??
NHL w/Rituxan + Zevalin init Nov 03
trials started w/Taxotere, doxo, & H &
N(5FU/cis), also RCC
- single agent (no Rx) CLL, Int conf hematol
Alcon Labs (returned Oct 17/01)
malignancies March 11/04, interim, partial efficacy.
nov 8/04 precin stabilisation of plaques
-Lutrin(Lutetium texaph.)→breast K, ph II;rights returned
from Nycomed (May 01)
-Optrin→ARMD, ph II
-Antrin→PAD, ph II, coronary ph I Transcath Conf Sept
02
From Celera (April 06)
- HDAC inhib, ph I, K
- HDAC 8 selective inhibitor, K, precl
- PCI 24781 HDAC inhib & downregulates RAD 51 (like
SUPG MP-470)
- VIIa inhib, pre
PDGM PARADIGM (PDGM) Agro functional genomics PHA (from Monsanto), Bayer(herbicides)
IPO may 5
PDLI M PROTEIN DESIGN LABS (PDLI) CDR grafting , better known as "humanization", is the I.P. of PDL (Queen →hence royalties 3% (1 quarter late)worth 6-
patent, 1996). Agreement on Queen/Cabilly Sept 98- Sept 03 : renewed
FOR SALE Oct 2/07 12$/share from Zenapax (Roche 10.5%),Herceptin
Aug 29/07 : rebecoming develop stage (DNA), Synagis (MEDI/ABT), Mylotarg, Xolair,
company -Zenapax/daclizumab (anti CD25) once a month for PSO (maintenance Xanelim, MDX 210, Humicade, Avastin, LUCENTIS,
-All revenues are from licenses & interest of remission,ph II endpoint is time to recurrence), ulcerative colitis (ph II anti IL5 GSK, 870, LDP 02, Antegren, Medi 507,
data 1Q04), MS w BIIB (I/II), Diabetes( I/II), RA,asthma(II)data 1Q04 ,
-Clever business model leveraging patents long term safety since years
5G1.1
through licenses & services. -M 195,Zamyl (hz anti CD 33)→AML, ph III (N=193) March 20/02 discont. for PSO maintenance. For non
ORR 43% (> Mylotarg). Lic (March 18/03 to Actinium for Act 225 & Bi graft indics PDL got 60% on sales & free goods. Sept 30/03 PDLI
Manufacturing : commercial facility being 213 in AML (ph II MSKCC, 30 already on Bi) acquires all rights for non graft. Sept 16/04 new Roche deal
Nov 1/05 subcut w/Roche for graft maintenance Asthma. Stop Aug 31/06. Nov 24/06 Roche terminates all Zenapax
built. Clin. trial facility exists in Minnessota.
March 12/07 MS ph II OK w/BIIB Nice study in ulcerative colitis, N=10, but ph II N =
- 120 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-hz anti IL4 pascalizumab (SB240683)asthma, ph I/II™march 01 159 DB fails May 17/04 ( b ut Simulect works ?); Asthma AAAAI
ROYALTIES growth (18 m delays on prior AAAA:+ data; ph II init Oct 9/01 DB R on FEV1, pts without steroids or March 19/04 : OK
long acting β+). Failure May 7/03. Term. ASH 01, does not meet endpoint(CR at 70d).Filing 2H03 or before,
forecasts, but great safety) : -1D10, Remitogen, hz anti HLA-DR, NHL, ph II no liver tox (> Mylotarg, N=104, 0RR 31%) ∏ ASCO 02(May 20,
03 :with launch of Xolair Korn, April 18/02 : "we may not continue in NHL, but perhaps in another Failure : TERMINATED)
04 : w/Antegren(++), Avastin ., Xanelim K". Term Jan 7/03 lic from GSK, AAAAI, 03/03
(4Q03) ASH 01: ph I, 4 PR/7pts. Ph II ongoing (1PR on 25
-hz anti L selectin,Trauma, ph II, w/Scil, Ib PSO first pts, angioedema,)
May 2/02 : Korn ↗ Chairman, Levitt
-hz antiγ IFNfontolizumab, HuZaf, Crohn, ph I/II, PSO
(Head R/D) resigns. New CEO Mark
(init June 02)
McDade (worked w/Max Link at BM,
cofounder Corixa, to become Chairman. Dig Dis Week May 21/02 (but in prior data low dose
-Nuvion (visilizumab), hz anti CD3, ster resistant GVHD, is < placebo) again placebo works as well, or nearly
To resign end 07
ph IIb N=80 ∏ % 180d survivors (open) since March 02, DDW again 2004, March 23/04 : fails again in Crohn
Jan 03 : acq EOS PSO(discont June21/01), Infl BD
Perhaps also for diabetes (then Zenapax) Should work (cf Ortho )
Nuvion discont Aug 30/07
July 8/03 : to offer up to $ 200m in Ulc. Colitis ph I DDW May 03; but → lymphopenia w
convertibles due 2023 in PIPE (already TH2 cytokine switch (IL6, INF α, INF γ), could file
has $ 150m convert. due Feb 07) -Hz anti IL12∏autoimmune disease (MS), ph I (Nl volunteers) init June on ph II 2004, w/ph III starting late 04 & ongoing
26/02, back to precl Jan 8/03 until 2005. DDW May 15-20/04 on 27 pts open ph
Aug 15/03 : DNA claims "Queen " does - M 200 volociximab w/BIIB, ch, : anti-α 5 β 1integrin
(antiangiogenesis), K, ph I, 66% SD but neutralizing Abs & humanization
I, "provocative results" interim Sept 28/04 N = 30,
not apply to Xolair. could decrease specificity. Ph II RCC init Jan 12/05, melanoma, pancreas. 3 doses, not very clear.. Aug 04 : 2 cases
Data 2Q06 w/DLTCompetition Sangstat/PG, Otzuka (OPC
June 30/04 : Max Link elected Chairman -Ostavir →Hep B "fully human", ph II 6535), remicade (N E J Med Dec 05)
- Ularitide (from ESP), natriuretic peptide (like Natrecor see Scios/JNJ £
375 m in 2004, but creat increase at all doses in March 05 metaanal of
Jan 25/05 acq ESP Pharma $ 475 m : 1269 pts) Ph II SIRIUS EU, PCWP improved , but failure ph II in tmt of
hospital branded generics, IV Nifedipine, acute renal failure. For sale Aug 30/07
busulfex, acebutolol - Terlipressin (synthetic ADH w/ triglycyl Lys) for esophageal varices &
hepatorenal sd

PDLI EOS (private) Repository of Hu cancer samples (genomic screening MEDX, MOR, BGEN, AVE
w/AFFX), seeking targets for MAbs
M Was to merge w/PCOP, term Jan 02, acq
by PDLI Jan 03
PEB P E BIOSYSTEMS (PEB) PEB has 4 operating units : The PE corp. has 2 operating groups PEB & CRA
-Applied Biosystems :material for genomics PEB sales : $1.2 B.
THEY MAKE EQUIPMENT FOR ALL -Perseptive Biosystems : cosumables for mol. hi .
THE GENOMIC COMPANIES -PE informatics : software INVESTING IN PEB IS LIKE INVESTING INTO A
-Tropix : chemoluminescence GENOMIC FUND !
PERL PERLEGEN (PERL) "analyze millions of unique genetic variations in clinical PFE, MRK, GSK, AZN, JNJ
IPO filed April 06 (Lehman, Deutsche Bank) trial participants"
Chairman is CEO of Affymetrix
Fruchart on Board
- 121 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


PFE VICURON (MICU) was VERSICOR Antibiotics and antifungals : Competition: Micafungin Fujisawa (filed April
AB (VERS) -V-echinocandin/anidulafungin, IV antifungal, phIII (in 30/02, delayed 18 m after meeting w/FDA approved
vitro > Cancidas/ caspofungin ( also an March 17/05 for Oesophageal AND prophylaxis
SAB from BMY/LLY/MRK echinocandin)/MRK approv. Jan 01 sales 2004 $ 430m:. during HSCT)also echinocandin, filed > Diflucan ( oral
Filing April 29/03 ( oesophageal candidiasis 50mg), fluconazole PFE, 2002 $ 1.1B). Diflucan has HSCT indication, but
not Cancidas
PIPE April 12/02 $ 44.9m launch 2-3 Q04? NO, Jan 26/04 FDA requests 90 d
more (to May 25/04). May 24 : approvable. New strategy Sept 21/04 : for esophageal will
July 30/02 : acq Biosearch Italia Feb 7/04 : sepsis, DB, N = 256 100 mg/d IV vs 400mg complete 100 mg ongoing pivotal and file 2Q05; for
March 18/03 N= 600 oesophageal fluconazole (Diflucan PFE). Response rate at end of tmt sepsis will file 3Q05
candidiasis, IV anidulafungin efficacy= oral 75.6%/60.2%; 6 w follow-up demonstrates non-inferiority
diflucan, but 14 d relapse rate 3 times 55.9/44.1%
PHA/PFE; competition AZN 2563, ph I
higher -2nd generation oxazolidine VEC3583, PRE∏phI, lic to
PHA (who has Linezolid/ZYVOX MKT april 00)
July 12/03 : secondary $ 23 m -Antibacterial Deformylase & MUR pathway inhibitor, lic to Novartis, ™ ICAAC 02
Feb 13/04 $ 200 m shelf PRE, lic to Novartis
-V-glycopeptide BI 397(dalbavancin), Vancomycin like, lic. from Biosearch Italia (NO US comp of matter
akin to Teicoplanin. Once a week IV, ph II SSTIcompl. IP)
June 16/05 to be acq by PFE May 21/02(ph III init Dec 16/02, SSTI : 2 X N = 550, ICAAC 02 for ph II late breaker Sept 13; N = 62,
comp to Zyvox/Cephalexin , enrol compl May 11/04). open clinical success 94% (standard tmt 72%) &
Aug 12/04 Conf Call : 3 SSTI ph III, endpoints clin resp. bact 72% (standard 64%).
N = 565 versus Cefazolin 89.1/89.1% Full data ECMID Prague, May 1-4 2004
N = 854, complicated, vs Zyvox, 88.9/91.2 Filed EU Dec 03
N = 156, MRSA, vs Vanco 89.9/86.7. Will file.
Bacteremia init March 10/02 N=180, open, catheter
infections, QD/QW. Completion 1Q03. Data Jan 12/04 :
21 days after end of tmt response rate (clin & bact)
87% for Dalba, 50% for Vanco. Filing Dec 21/04 SSTI.
Priority review ? 6 m to PDUFA. May 11/05 PDUFA
delayed to Sept 21/05
PGNX M PROGENICS (PGNX) -Cancer : ganglioside conjugate vaccines BMY (terminated May 15/01)
GMK (GM2 Ag + Immunogenic protein
KLH)→melanoma, ph III. interim anal (may 26/00):placebo>verum
V - Methylnatrexone IV, SCph II for post –op ileus: active
Jan 05 ROCHE
-AIDS PRO 542 (fusion protein IgG2/CD4, anti CD 4, ph
K I/II, synergistic w/T20 (Trimeris)
-Pro 140, anti CCR5 MAb ,hz, AIDS, pre, high activity
in mice, ph I
-Fully Hu MAb anti PSMA, JV w/CYTO (who regains
rights April 25/06), collab w/ABGX, ph I

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


PHRM K PHARMION (PHRM) - Thalidomide in Europe, withdrawn May 04, resubmitted
Price Vidaza $ 32,000 per patient to EMEA 2005. Seeking Compassionate 1Q04 : 12.6m, 3Q
Cancer Consultants survey March 1/05 17.8, FY 06 77 (under review, May 07)
preferred frontline : - Vidaza , azacytidine (acq from PHA) , demethylating
agent for MDS.23% patients CR or PR NDA filed Dec ASH 04 poster on induction in AML
Vidaza 49% 29/03, accepted Feb 23/04, priority : 2 ph II BSC +/-
Thalomid 16% Vidaza. FDA OK May 20/04 .Sales 3Q04 20.4, 3Q06
Revlimid 12% 36m; FY 06 142, 2Q07 32.5, 3Q 33.3 CHMP filed Sept
Dacogen 5% 04, withdrawn Nov 17/05. To refile late 07. Ph III vs BSC
Also Zanestra / tipifanib (JNJ) PDUFA 2H05 OS 24.4 m/ 15 m (OS for Dacogen will be mid 08)
- Amrubicin, acq from Cabrellas who got from Sumitomo)
Nov 19/07 CELG to acq $ 2.9B lack of cumulative tox. Ph II SCLC, ph III init Oct 17/07
2nd line w/topotecan, NSCLC, Breast , filing 2010 ( HDAC competition : MRK Zolinza (SAHA) approved
- Satraplatin for EU : lic from GPC Biotech, Dec 20/05, Oct 06 for CTCL.
filed EU June 26/07 . But OS ??
- MGLD 0103 HDAC inhib ph I/II, w/Methylgene
POTP K Point Therapeutics (POTP) -Talabostat, oral, inhibits FAP (fibroblast activating ASCO 06 : CLL, CRC, melanoma
protein), a tumor stroma member of the DPP
March 13/07 Nasdaq delisting risk (dipeptidyl peptidase) family of serine threonine kinases
NSCLC IIIb/IV ph II durable CRs; PFS 2nd line 4.9m, 3rd
line 2.7m. Ph III 2nd & 3rd line x2 one w/Taxotere vs Tax
Oct 10/07 merges w/Dara alone, other w/Alimta vs Alim alone. Endpoint PFS ( N =
800) data 4Q07 but w/Taxotere behind schedule (because
Alimta/Tarceva is gaining mkt share, 2nd line; also
melanoma, refractory CLL (w/Rituxan), pancreas
(w/Gemzar) data 2nd line : Jan 12/07 N = 60, interim 21 :
48% survival > 6m (primary endpoint). Final data mid 07.
Why did'nt they wait for ASCO-GI ? why did a Board
member resign ? March 1/07 : 51 evaluable, endpoint not
reached. May 21/07 trial halted
CLL ph II ASH 05 2nd/3rd line 21% PR, 50% SD
melanoma ph II
- PT 630 diabetes type 2
POZN POZEN (POZN) Treatments of migraine :
-MT 100, oral metoclopropamide + naproxen, ph III, Jan
traded since oct 11/01 26/01 : FDA asks for carcinotox (rats expected 2003, p53
knock out mice OK now). Could file UK (Mut Recog)
4Q03. NDA filed Dec 17/02. June 2/04 Non approvable
-MT 300, injectable DHE, 2 ph III, 1st one meets endpoint
(July 22/02), 2nd meets primary Oct 4/02 (sustained relief
24h)

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- Trexima MT 400 (triptan + NSAI), acute tmt; ph II signif,
lic to GSKfor US $ 160m deal June 03. April 21/05 ph III
OK, to file, seeks EU partner. June 10/06 "approvable",
need for more trials
PN 400, combi esomeprazole/naproxen, NDA planned
2009- MT 500, 5HT antag,prevention, ph I
PHST PHARSIGHT (PHST) Computer assisted trial designs AZN, SBH, JNJ, LLY
PPDI PHARMACEUT. PROD. DEV. (PPDI) CRO also in PHARMACOGENOMICS :
-Genomic Banking
-Genotyping
-Polymorphism discovery
- Oral Dipeptidyl-peptidase IV inhibitors (ph I & II), lic to
Takeda July 14/05
PPHM M PEREGRINE (PPHM) -Oncolym, LYM, (m I*anti HLA-Dr10β)→NHL,ph II/III lic to Schering AG,retuned June 11/01
was TECHNICLONE (TCLN) -Cotara(*I ch MAb anti necrotic core of tumors)→glioma,
to rename : Peregrine ph II, fast track granted Oct 10/01, ph III planned
K
for IQ02, OK for FDA Feb 4/03. Waits for partner
Jan 12/01 : CEO takes leave of absence - bavituximab (antiphospholipid)Tarvacin, ph I K, HCV JV Oxigene ("Arcus") ended March 4/02
partn w/Scotia
PRTG PROTARGA (PRTG) Targaceuticals (drug + docosahexaenoic acid release in
IPO filed (UBSW) Dec 12/01, target) :
Pulled Oct 3/02 - Taxapresin : paclitaxel + docosa, metastatic melanoma,
Files for Bankruptcy 2003. Assets ph III to start 1H03
disseminated

PRW K PRO-PHARMACEUTICALS (PRW) Davanat, polysaccharide derived from galactomannan, ph ASCO


II, 3rd 4th line CRC, bile duct carcinoma ph II

PTCT K PTC Therapeutics * molecules for exon skipping

10% of the 30,000 US people w/CF have - PTC 124 oral, Feb 22/05, funds from CF Foundation
non-sense mutations To init ph II Duchenne & CF YE 05. ph II initiated Dec
IPO filed April 3/06 (PTCT) M. Stanley, 23/04 for Duchenne & CF (with nonsense mutations 15%,
Pacific Growth 10% of cases). April 4/06 improves nasal transepithelial
Postponed April 20/07 potendial difference. Nov 6/06: Confirmation
Oct 23/06 increases dystrophin in muscle biopsies. Nov
Jan 8/07 partnership w/PFE 141 M (probably 5/06 normalizes blood neutrophil count
PTC 299) - other drugs for hemophilia, neurofibromatosis, retinitis
pigmentosa, LSD.
Sept 13/07 CELG invests $ 20M into K - PTC 199, antiangiogenic, precl, lic to Bausch & Lomb

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


targets - March 06 lic small molecules candidate inhibiting Her C
IRES to SGP $ 200m : deal
- PTC 299 K, ph Ia, init March 12/06, oral, anti VEGF
production; successful, ph I/II to init (4Q07) in Breast
- June 7/06 Spinal Muscular Atrophy research
PTIE PAIN THERAPEUTICS (PTIE) Develop associations of opiates & antagonists to enhance
investors : Bill Gates, Mr Robertson (from R. safety Oxydrex ph III fails Nov 22/05
Stephens) - PTI 901, ph III, IBS, fails Dec 12/05
PTD PEPTECH (Australia) - PMX 53 : anti C5a receptor for ARMD (ph I completed) Royalties for antiTNF until 2010

earned 30% of Domantis sale to GSK


PTIL Protherix (LSE) Polyclonal anti TNF for sepsis, w/AZN. Nov 06 FDA &
EMEA request 2nd ph IIb
PTN PALATIN (PTN) - Peptidomimetic small molecules, melanocortin agon.
-NeutroSpec (was LeuTech), Tc tagged Mab anti CD 15 :
PIPE $ 11.5m Nov 02 radiodg of leukocyte collections, manuf data requested
PIPE $ 19 m, April 03 (sept 29/00) by FDA before approval for "equivocal
appendicitis, osteomyelitis". FDA approved July 6/04.
Sales halted Dec 19/05 : 2 deaths
- LeuTech for anthrax detection : trial with US army (Nov
01)
- Melanocortin obesity program lic to AZN Jan 31/07 $ Aug 12/04 lic to King (being acq by Mylan , $ 250 m
310m (upfront 20)
-Bremelanotide, PT141,7aa analog of α MSH, MED, ph II,
intranasal, melanocortin MR3 & 4 agon., ph III to init
1Q07. Aug 3/06 arousal post menop doubled
March 05 synergy w/Viagra
Feb 8/05 ph II data in women.
Ph III was to init end 06, but Flushing & nausea (mild).
Delayed (Aug 30/07) by FDA. Sept 10/07 King terminates
QLTI QLT PHOTOTHERAPEUTICS (QLTI) ∗Photodynamic therapies :
K -Photofrin QLT→K(NSCLC , esophagus), $10 mWW,to
Visudyne launch April 14/00. be sold ww to Axcan (May 00)
SASY sell in US SASY
QLTI becomes profitable ! Beaufour Ibsen terminated June 99∏ Axcan
-Visudyne IV (verteporfin) →ARMD, revenues for QLT $ COHR & Zeiss are the key laser sellers : used in 1500
Nov 23/02 : staff ¬ 18% 129m/01, 1Q03 USD 83 m., 1Q05 $ 125, 2005 : centres
485 Competition : Alcon (Anecortave, steroid, head to
June 14/04 to merge w/Atrix (Eligard for CPMP approval April 12/00, FDA April 14/00, Feb 6/01 head 2 year trial since Jan 02), Eyetech, anti
Prostate K, $ 89m for 2005) FDA expands indication (pathologic myopia), VEGF (DNA)
Novartis Ophtalmics 50/50 JV(total sales 01 US $
- 125 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


CPMP approves "occult" AMD 223m; 02 : 275 to 300))

∗Signal transduct inhibs : acq of Kinetek, June11/01

∗XR 9576(tariquidar, sort of MDR inhib), lic in from


Xenova (Aug 14/01),ph II, NSCLC, breast (MD ASCO 03 : Breast (Hortobagyi)
Anderson). 2 phase III init June 27/02 NSCLC US
(w/Paclitaxel) EU (w/Vinorelbine). Feb 21/03 enrol
suspended 3 m for safety evaluation.
Pivotal halted May 13/03. Chemorefractory breast MD
Anderson continues
* Synthetic retinoid for Leber, pre, lic from Retinagenix
(April 5/06) upfront $ 1.5 m Deal
QSC QUESTCOR PHARM (QSC) -Ethanol→sclerosis of oesophageal varices, MKT merger of Ribogene & Cypros
-Inuline→renal tests, MKT
-Migrasta(propranolol nasal spray), in dev
-Ceresine→TBI, ph I/II
QURK IPO Quark Biotech siRNA
Filed 03/07 JP Morgan, CIBC
- RTP 801i-14, for AMD, inhibits RTP 801, ph I, lic to
nPFE
- AKI i-5, inhibits p 53, ph I, for systemic delivery to
prevent acute renal failure

RDYN REPLIDYNE REP 3123, precl, inhic C. difficile


- Faropenem, ph III, bronchitis
- REP 8839 SSTI, MRSA ph I

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


REGN M REGENERON (REGN) -Axokine(2nd gener CNTF)→ALS PG discont
reduces appetite, seems to activate HSV (cold sores), ph II Gives cough & nausea
+ in obesity, but effect maintained after arrest. PhIII Data April 03 on 2500 pts : neutralizing Abs in 70%
K PG now (aug 3) owns 18% (injectable once a day)enrolled (Jan 02)4 other phase III pts (less efficacy). Weight loss signif, but
ongoing (N = 4000). Side-effect Guillain Barré, Bell. BLA small.
Aventis deal Sept 8/03 : upfront $ 80m + 45 filing 2H04. Launch ?? 2H05 Price $ 3000/year
stock. Early milestone 25. Other milestones -Axokine oral →obesity
on "approvals" for 8 indications US & EU : -Axokine Pegylated, w/Shearwater INHL, ph I
360. Ave funds dev (if profitable, REGN will -BDNF→ALS →with EMISPHERE
refund 50%). Ave will build manuf facility, 1st subcut trial failed
will share 50/50 promotion & profits 2nd underway →Amgen
intrathecal 18 months DB random, enrol.ended Nov 99
- IL1 trap ribonacept : ph II ongoing RA (data 3Q03). Sept intrathecal has been halted by FDA
Feb 16/06 Acomplia not approvable for 05 discont in RA, continued in periodic dis.
smoking, and "approvable" for obesity Blocks IL1 on cell surface. W/NVS (April 2/03) : upfront $
21 m cash + 48 equity; 275 m milestones if product
Feb 5/07 : Ab platform lic to AZN approved. Ph III costs shared. Pre ph III paid. Oct 7/03 :
ACR20 ph II target not reached June 10/05 : ph I/II active
in CIAS1 (cold induced autoinflammatory sd 1) periodic
Nov 29/07 Sanofi deal $ 1B (from 4 to 19% sd . Top line YE 06 : Oct 30 good ph III data , rol. filing
ownership) : completed June 8/0707; PDUFA Nov 07 delayed (Nov
- Ab to IL6R, ph I, RA : but Actemra BLA 3/07) until Feb 29/08 ASCO 2006 : ph I, N = 27, ovary, CRC, kidney
filed by Roche Nov 25/07, and Notch very - IL4/13 trap, asthma, ph I
hypothetical - VEGF trap , aflibercept (extracell domain of VEGFR 1 Dec 22/05 deal extends to Japan. SASY funds dev.
- anti DLL4 (delta like ligand) to start & 2 fused to Fc of IgG1) SC/ IV ph I, solid K & NHL, Sanofi guidance (Feb 23/06) : filing 2007/2008
clinicals 2008 - some good data in NSCLC. NCI, lic to AVENTIS Sept
8/03. Jan 10/06 Sanofi confirms license.;
- Ph I/II w/ FOLFOX init May 13/06;
- Dec 05 : more than 16 studies, & ovary, malignant ascites
- ph II : ovary (interim ph II N = 200 ASCO 07, 3rd or
more line, 8% PR, 77% SD, CA-125 reduct > 50% in 13%
pts. Avastin trial ASCO 07 gave 16% PR, but bowel
perforations), malignant peritoneal ascites, adeno
NSCLC 2ND/3rd line (ph II ASCO 07, adenoK resistant to
Pt & erlotinib); ph II breast suspended Nov 9/07
To init 5 ph III early 07 (Sanofi presentation Feb 13/07)
- 1st line HRPC + Taxotere : init Aug 23/07, N = 1200,
primary : OS
- 1st line Pancreas + Gemzar; N = 650 to init YE 07
- 1st line gastric + Taxotere
- 2nd line NSCLC + Taxotere: init Aug 23/07 ; see Sept
- 127 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


R/D day: N = 900, should have init late Sept 07
- 2nd line CRC + Folfiri; N = 1,200, to init YE 07. Ph II
suspended Nov 30/07
1st filing 2008
on mkt 2009
- ARMD ph I not w/SASY Good data ARVO May 1/06, 8
abstracts, systemic; local init 3Q06
phase III ARMD to init 2H07 (vs lucentis, to try to show
same efficacy q3m than q1m), final ph II data early 07
Lic to Bayer Oct 18/06, upfront 75 m, cost sharing,
milestones 245m.
March 27/07 good ph II interim on "retinal thickness".
Ph III vs Lucentis int Aug 3/07, N = 1200. Primary
endpoint 1 year Lucentis q/4w vs Trap q/4w, vs Trap q/8w
Targeted genomics, functionomics & designer protein →partnership with MEDAREX (MAb), 13march
therapeutics 00
REX K VIREX (REX), was Altarex Ovarex (oregovomab), murine mAb, ph III, ovary K;
failure Nov 5/07
RGEN M REPLIGEN (RGEN) - Protein A (for protein purification)
-rPF4 seems to have been discarded patent issued Feb 00;
-Secretin (synthetic : Secreflo)→autism, in ph II (daughter BMY data at ACR Oct 04 Abatacept Orencia(T cell
of CEO is autistic). Ph III init Feb 15/02. Data 1Q03 costimulation modulator). Patients refractory to anti-
April4/01: 136 pts, DB, did not meet primary endpoint, but TNF, vs placebo, 6 m, N = 258
did statistically improve signs ACR20 ACR 50 ACR 70
-CTLA4-Ig→BMT from mismatches, ph II (BMY is in ph CTLA4-Ig 50.4% 20.3% 10.2%
III with another CTLA4-Ig in RA; patents issues) placebo 19.5 3.8 1.5
-TAU (triacetyluridine)ph I, tmt of mitochondrial acidosis p < 0.001 <0.001 <0.03

Orencia sold since Feb 06. 1H06 $ 23m

RIGL K RIGEL (RIGL) Post-genomic combinatorial chemistry CEGE, JNJ, NOV, PFE, NBIX

filed(w/MSDW) 9/18/00 - R 112Small molecule IgE receptor inhib (blocks Syk AAAAI, March 05
Trading since Nov 28/00 kinase), asthma, ph II. Dec 1/05 fails
secondary Feb 20/04 : $ 57m - R 406 oral : mastocyte IgG receptor inhib, ph I, RA..
- Drugs active on Ubiquitin ligases : K (lic to MRK Nov
15/04)
- R 763 : oral & IV aurora kinase inhib,. Oct 25/05 lic to see also VRTX, Supergen, SNSS, AZN, BI, PFE,
Serono $ 125m Ph I init July 12/07 Cyclacel, Entremed
- R 788, syk kinase inhib, ph II initiated Sept 06, RA
- JAK 2 inhib, pre
- 128 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- JAK 3 kinase inhib, R 348, pre, RA
- Polo like kinase inhib, pre
- AXL kinase inhib, pre

RNAI SIRNA Therapeutics ( RNAI) April 17,03 -Angiozyme(anti VEGF R1),ph I/II∏phII fails (April CHIR
was RIBOZYME PHARM. (RZYM) 29/02) in Breast (Mono), ongoing ph II colorectal ( Saltz
Oct 30/06 MRK to acq $ 1.1B irinotecan + 5FU + leucovorine). Data June 10/02 first 40 ASCO 03
Aug 11/02 : staff ¬ 25% pts at 12w, only 12.5% have disease progression
CEO Howard Robin (was Christophersen). J (historical. comp 25%) OCT 30/06 MRK to acq : $ 1.1B
Curnock Cook chairman. -Heptazyme(anti Hep C virus), ph I completed sept 12,
Feb 17/02 : $ 48 m PIPE (Sprout, Venrock, . Term April 29/02 (animals go blind)
Oxford Bioscience) for siRNA ( 50 patents, -Herzyme(anti EGFR)→breast K, ph I, Aug 30/01
30 issued) - HepBzyme (anti hep B), pre. Term Aug 16/02 ELN (term)
April 3/06 US patent on nucleic acids - Telomerase inhib GRN 163 (w Geron)
targeting IKK (NFKB) - siRNA s for Huntington : res, w/Targeted (Jan 05)
Sept 29/05 $ 250m deal w/Allergan for - siRNA-027 w/ALLERGAN, targets VEGF R1 for -acq Innovir (that had been acq by Vimrx, now
ophtalmics (upfront 5m, milestones 245, ARMD, ph I , data Aug 10/06 not bad Nexell,for oligos anti Hep B and APL)
res funding) DEAL - siRNA HCV, IND to file Q406 -Functional genomics with Roche & Schering AG
April 3/06 $ 700m w GSK respiratory - siRNA for permanent hair removal (knocks out the
DEAL "hairless" transcription factor
RNA RIBAPHARM (RNA) - Ribavirin, MKT by SGP, hep C ; royalties 155/00, Competition :
IPO filed 6/15/01 (UBS), priced April 12/02 139/01 ¬ - a form of ribavirin developed by Roche (Copegus)
- Levovirin, ph I, lic to Roche, - US Ribavirin generics : Teva, Geneva.
Spin off from ICN - Viramidine ph I
- IL 12, anti K, colon
Considerable Ribavirin patent litigation
issues.
RNVS RENOVIS (RNVS) Cerovive, ph III efficacy in stroke, p = 0.038, filing 2007 lic to AZN
after 2nd ph III, delayed, hemorrhage : failure March 14/06
small molecule inhib. of vanilloid receptor VR1 for pain
(Sept 05 $ 187 m research deal w/PFE)
ROSG IPO Rosetta Informatics micro RNA

RPRX V REPROS Therapeutics, was ZONAGEN -VASOMAX (oral phentolamine), NDA filed SGP, could get UK approval in 02 : CSM says no,
(ZONA) Clinical hold by FDA (new 2 year rat data filed 04/01, application withdrawn (April 29/02)
brown fat prolif in mice))
Oct 31/02 : merging w US subsidiary of - Contraceptive vaccines suspended Sept 26/01
Lavipharm (large Greek profitable pharma) - Praellex : antiprogesterone ph II fibroids, endometriosis
- Androxol : pituitary androgen agonist

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


RTIX REGENERATION TECHNOL. (RTIX) Allografts from cadaveric donors (BioCleanse system)

RXIP IPO RXi Pharmaceuticals, filed Oct 30/07 siRNA spin off CytRx

RXHN REXAHN (RXHN) - RX- 0201, Akt inhibitor, ph I, antisense


- RX 0047, HIF inhibitor, pre
SANTARIS A/S micro RNA

SBIO SYNBIOTICS (SBIO) Veterinary diagnostics

SCLN K SCICLONE (SCLN) Zadaxin : thymosine™hep B & C, MKT in 3rd world


ph III fails dec 14/05
Dec 22/06 w/SigmaTau, w/DTIC – IFN, N = 386, ph II,
Melanoma. Longer median survival. 5 arms
+ DTIC : 10.2 m vs 6.6m
+ DTIC + IFN 19.9m
SEPR SEPRACOR (SEPR) -levocetirizine (for Zyrtec), ph III, approv. in Germany (jan UCB (for Europe)
July 30/02 : VP/RD resigns 16, 01) & 4 other EU countries
The stereoisomer company -R-fluoxetine (for Prozac), ph II ? LLY(term oct19/00),
-norcisaprid (for Propulsid), ph II ? JNJ(term April 23/01)
March 25/04 expanding sales force from 450 -norastemizole/Soltara/Tecastemizole (for Hismanal), NDA filed march 12/01;Not approvable March
to 1250 filed, discont Dec 03 7/02. QT∨, phospholipidosis,
-descarbloratadine Clarinex (for Claritin),MKT cardiomyopathy
The future rested on Soltara, but
Hismanal had serious QT issues : are they -Xopenex, levalbuterol, MKT,approved 2002; 2004 sales $ SGP3% royalty (CPMP OK, sept 21, 00)FDA
absent with Soltara ??? : answer Nov 16- 320m. March 14/05 metered dose inhaler approved, 2Q07 OK(Dec 20/01). No interaction with macrolides.
21 ACAAI: no QTc > 500 ms, and CPMP $ 105 ABT
limits to 450! Also AUC up -Allegra (for Seldane), MKT Aventis (3% royalties), Albany Molecular
w/Erythromycine -R-formoterol (for Foradil Novartis), ph III,
-S-oxybutinin SR(for Ditropan AZA),ph III,; 2nd ph III to
Nov 7/01 : PP of $ 400 m convertible start 2H04, ? Liquid form & tablet will be filed N=436, DB,
subordinates -S-Zopiclone(Lunesta, Estorra), for insomnia, ph III,, but Placebo, filing Feb 2 Q03., approvable March 1/04
How will they survive Claritin generics( Dec t1/2 = 5-7 h. Bad taste. Filed Jan 03, PDUFA Nov 30/03 , Resubmitted June 16. Class II (6m)PDUFA
02) ? 2006 $ 566.8 M2Q07 $ 143. EU filed 3Q07, lic to GSK Dec15/04 FDA OK Dec 15/04 : 6 m efficacy/safety.
Nov 02 : class action suit on Soltara Sept 11/07 Delay on availability due to schedule IV issues.
Cash 2Q07 : 861 March 1/04 : approvable letter Available April 7/05 for long term tmt (under
- S-Amlodipine, ph II (less edema) ? schedule IV)
SFPH- Sheffield Pharmaceuticals - Coprexa (tetrathiomolybdate), ph II, Wilson
OTC
merging (Nov 06) w/ Pipex

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


SGEN M SEATTLE GENETICS (SGEN) - SGN 10, BR96 scFv, pseudomonas toxin linked, ovary w/Aventis
K,with Taxotere, ph I;ASCO 01:problems of vascular
EPS not positive until 2007 leakage. Term Nov 7/02.
K - SGN 15, BR96 ch, doxo linked, ph II, with Taxotere, w/Aventis, w/ICOS
IPO filed nov 20 ( JPM),priced $7:DNA acq breast(term Nov 7/02)/prostate/colon K/NSCLC. ASCO 01 ASCO 03 : HRPC w/ or wo/ Taxotere, NSCLC :
$2m, MEDX acq $2m in PIPE : serious digestive side-effects; ovary w/Gemzar init Aug progression free survival 15.3w (taxotere alone
02 group 7.5w), without GI side effects → August 03
Non-exclusive single chain licenses from World conf on lung cancer : survival Tax alone
ENZN & Creative Biomolecules (CRIS) 5.1m, assoc 11.4 m
- SGN 14 , naked anti CD40 (competition IDPH, AMEV),
Created by ex BMY employees, with license pre DNA discont Oct 9/02
from BMY to the BR96 Abs (anti Lewis y, -SGN 30 (anti CD30, activated T cells), ph I init March
also present in gut) improved by AMEV. 21/02, Hodgkin & NHL; ph II CTCL
Competition Hz anti Lewiw lic by PDLI to - SGN 17/19 ADEPT (antibody directed enzyme prodrug → ASH 03
Igeneon AG 'Austria) therapy): sFv against p97(melanoma Ag), fused to β
lactamase, then pt receives cephalosporin derivative of
July 2/03 : PIPE $ 41m Melphalan
- SGN 40 : Hz anti CD 40, lic to DNA Jan 7/07, 800m
(upfront 60m); see Idec 131 & AMEV/LLY ph I & II MM,
NHL, CLL; ASH 06 good ph I data NHL June 01:lic toxins & linkers to EOS
- Auristatin, proprietary cytotoxic that can be linked to Abs. f hu partnership w/Medarex (feb 7/01)

SGMO SANGAMO (SGMO) Engineered zinc finger (ZF) transcription factors : AZN, Bayer, DuPont,, DNA, GENSET, GLX, Roche
nucleases (cut DNA where you wish) IMNX, JNJ, MLNM, Merck KGaA, PFE, PNU, PNG,
Schering AG, SKB, WL
-Aug 17 :method to improve expression of EPO w/MDRX : MAbs to GPCRs
-Method to overexpress GPCR
- Z F + gene for X-SCID, res
- Z.F. to disrupt CCR 5 R, to init 2006
- VEGF / Z.F. disrupted gene acq from Edwards
Lifesciences, ph II, gene therapy peripheral
neuropathy and ischemia
SGXP K SGX (SGXP) was Structural Genomix Cytidine analogue, ph II/III, 3rd line AML fails Aug 29/06
IPO
Traded Feb 1/06 - SGX 393, bcr/abl inhib w/Novartis, IND 1H08
- SGX 523, anti c-MET, to file IND 1Q08
Good team - JAK 2 inhib, res, polycythemia
- Ras inhib, res
SHM SHEFFIELD MEDICAL TECHNOL (SHM) re-fillable MDI (lic from Siemens) Dev & Mkt agreement w/Zambon

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


SHP SHIRE -GA-EPO (Dynepo), human cell line, filed FDA/CPMP Aventis
TRANSKARYOTIC THERAPIES (TKTX) Aug 3/00, additional process validation requested from patent litig AMGN (May 7/02 complex hearing,US
AVE by FDA (nov 24/00). Dec 12/01 : OK CPMP. decision expected 2H03) & UK, no launch before
Really human (glycosylation pattern !!!)EPO AMGN EU patents expire 04 completion ;costs assumed by AVE. Reacq by TKTx
Gene activation patent is licensed from Aug 3/04 : manuf by Lonza 1Q04;Sept 14/04 Aventis licenses to TKT onco data
CEGE available 1H05, if US patent litigation completed
Oct 18/04 : TKT loses in US, Oct 21 UK court
-Replagal agalsidase α,Fabry (TKTX says 5000 pts revokes AMGN patent ! Shire to launch EU 1H06
CFO tells me (Nov 01) : Replagal is ww),BLA (human cell line∏better glycosylation) filed
manufactured by a bunch of contract June 19/00,CPMP+ march01,giving slightly better
manufacturers labelling than to Fabrazyme, EU approval Aug 01
Adv Com Sept 27/02 efficacy (p< 0.0001)Jan4/01:FDA requests more data (did -Sumitomo, waiting for approval 2H04
TKTX answer ?? : NO, Dan Geffken, CFO Oct 15/01)
POSTPONED→Jan 13/14-03 : 15/0 May 01 (int congr hum genetics) : signif reduct cardiac Price 0.2 mg Replagal = 1mg Fabrazyme
failure to provide evidence of mass; 100% clearance of GL3, infusion 40 min (0.2 = $ 165,000 /patient/year
efficacy mg/kg)
June 5/01 : signif reduct in pain, (uninterpretable Registry : 150 treated EU pts
methodol. says FDA Sep 26/02)& improvement in EKG
March 19/03 : staff↘ 25% & GFR. Dec 2/02 : no statistical ∨ GFR Selling l EU 02 : $ 47.5; 1Q03 12, 2Q 14.4, 3Q 15.2,
April 4/03 : CFO Dan Geffkin resigns Sept 25/01 : signif improvement in cerebral blood flow 4Q 15.2. 2004 : $ 77m (4Q 22.5). New manuf process
Hunter : 2000 pts WW -ex vivo Gene therapy VIII, ph I (transfected auto- to be approved in EU
- Since Dec 03, President of Dr Reddy US on fibroblasts). June 01 : NEJM
Board. -G.I. (AHP).June 5/01:signif increase 4/6pts
-Renovating Alewife manuf facility in US (to -GA II, ph I, returned by AVE dec 8/00
restart 2H04 for I2S & GCB)
- Iduronate sulfatase Elaprase I2S, Elarsulfatase ; Hunter
April 25/05 to be acq by Shire, OK July sd (MPS II) , ph I/II, ph III, 12m , N = 96, init Sept
27/05 19/03,completed April 3/04, filing FDA Nov 23/05 ,
PDUFA May 25/06, extended on May 16 to Aug 25 , W/GENZ in Japan (30% royalty)
Hunter : around 2000 pts ww approved July 25/06; EMEA Dec 1/05, approval Jan
11/07 Top line ph III June 05 : June 20/05 top line data p =
0.005 (composite forced vital capacity + 6 min walk)
- GA-GCB : Gaucher, ph I/II fully enrolled April 20/05.
Data 2H05, on mkt in 2008
- LSD-CNS : Hunter & San Filippo (MPSIII)
- GAL-6S : Morquio, res
- GA-CSF, ph I (terminated by Aventis) seeking partner
- GA- FSH, pre, seeking partner
- Juvista (lic from Renovo June 20/07), r TGF beta 3, ph II,
to improve/prevent scarring

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


SHR COLLATERAL THERAPEUTICS (CLTX) AdFGF4(Genvascor)→PAD, phI Partn. with Schering AG(who is decreasing support)

Being acq by Schering AG (March 20/02) ™present. A.Coll Card march 20/01, improvement in
-AdFGF4 (Generx)→ischemic heart, ph II/III ™™→→→ treadmill but not signif. But at 8 weeks perfusion
defect reduced versus placebo.
-FGF/VEGF gene (GeneCor), PRE, w/CEGE
- Adenyl cyclase gene (Corgenic),PRE, CHF,w/TGEN
SIGA SIGA small pox therapy competitor Chimerix is in ph I since June 28/06 :
web site not updated SIGA-246, oral cysteine proteinase inhibitor. Ph I safey CMX 001)
Lead investor (Eliot Rose Asset Management OK. POC monkeys before or 12 days after inoculation Oct
(Providence) sold 71% of his holdings oct 18/ 06
31/06 March 19/07 child with eczema vaccinatorum improved
SIBI K SiBiono * Gendicine, adeno p 53 for H & N K. Approved,
CHINA commercialized since 2004. See "Human Gene Therapy"
Sept 05
SIRT IPO SIRTRIS Pharma SRT 501 : resveratrol, ph Ib, appetite control
- Activation of SIRT1 : protects animals against
calorie restriction diets activate SIR-1 neurodegenerative disease (June 26/07)
(increases NAD synthesis) - New product tested on ageing mice (CRADA) Sept 6/07
SLN K SILENCE THERAPEUTICS (LSE) siRNAs
- for respiratory, lic to AZN, July 6/07, $ 400
was SR Pharma that merged w/Atugen AG - for eye, lic to PFE, ph I
(antisense) - cancer, precl : pancreas & NSCLC to init 2008
SLXP SALIX (SLXP) gastrointestinal diseases

SMDX IPO SALMEDIX (SMDX), - SDX 105, Bendamustine : Treanda marketed in


K filed Sept 15/04 Germany; ROW Ph II NHL
Pacific Growth - SDX 101 R-etodolac, CLL ph I
- SDX 102,n L Alanosine, Ph II, NSCLC
SNAP SYNAPTIC PHARM CORP (SNAP) - melanocortin receptors
- strong IP in GPCR (165 issued patents
Being acq (Nov 12/02) by Lundbeck $ 121 m -alpha 1antag→BPH, ph II MRK terminated 2 March 00
cash -serotonin compounds→depression, ph I LLY
SNMX SENOMYX (SNMX)
filed (DB) Feb 21/01

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


SNSS IPO SUNESIS (SNSS), cdk inhibs in K BIIB (kinase program for K), JNJ, MRK (Alzheimer)
filed Dec 23/04 (Lehman, SG Cowen), - SNS 595 (lic from Dainippon) naphtyridine analog, IV :
refiled Dec 23/04 Lehman, Needham cell cycle modulator inhibits CDK 2,3 & 9; ph II, SCLC ASH 06
(ECCO 07), AML (but mucositis, ASH); ph II NSCLC 2nd
K traded since SEpt 27/05 line init Jan 4/06 terminated May 07. Ovary 2nd line AACR
Oct 07
- Raf kinase inhib (w/BIIB), pre
- SNS 032 cdk 2/7/9 inhib (lic from BMY) ph I/II 4Q05,
ph I to repeat 1Q07 B cell malignancies
- SN 314, aurora kinase inhib, ph I Sept 07
SNTA IPO Synta Pharmaceuticals - STA 4783 (bis thiobenzoyl hydrazide induces HSP 70 competition for Apilimod anti IL12 / IL 23 Mabs :
since inception raised $ 200m expression on K cells and activates NK), ph IIb meta CNTO-1275 ph III pso (NEJM Feb 8/07),
Lazard, Lehman Bear melanoma, DB +/- TaXol PFS 3.68m vs 1.84 (N = 81, p = ABT 874 (Pso, MS, Crohn)
K Team from MLNM, VRTX, BIIB 0.03) ORR 15% vs 3.6% ph III init Nov 12/2007, Taxol +
Pipeline acquired from Pricipia/Shionogi STA vs Taxol, endpoint PFS. Results 2H08. Catalysts
Postponed Jan 05, refiled Nov 22/06 EORTC Oct 22-26, Nov 1-4 Int Melanoma Conf New York
PRICED Feb 6/07 - STA 9090 : HSP 90 inhib, pre
- oral inhib of IL12/IL23 Apilimod (chemically different
mMelanoma OS : 6-9 m from imiquimod), ph II, RA

Oct 10/07 w/GSK $ 1.1B


SNTS SANTARUS ZEGERID, MTT, omeprazole + bicar in capsules or
powder. Indications : - short term tmt of active duod ulcer
- short term tmt of active gastric ulcer
- GERD
- erosive esophagitis
claim to be superior to Nexium or Protonix for "nocturnal
acid breakthrough".
SNUS K SONUS PHARMACEUTICALS ( SNUS) -EchoGen, ultrasound contrast agent, approved Europe, sold to Nycomed Amersham (Aug 6/01)
FDA filing withdrawn (oct 13/00)
-QW 4737, to assess myocardial perfusion, ph I
acquiring Synt:em Nov 5/04, $ 30m -QW 8184, paclitaxel emulsion formulation oil in water
w/Vit E, Tocosol, ph II init, April 5/02., fast track bladder
(Oct 03), lic to Schering AG Oct 18/05. Ph III metastatic
breast init July 8/06, weekly, compared to Taxol, but
Abraxane's response rate is double of Taxol.Enrol compl
Nov 16/06Data 1H07. Filing 2H07 ? Failure Sept 24/07
SNVT SAVIENT (SNVT) was Bio-Technology -Oxandrin (anabol. steroid) : $25m (Mkt w/ABT-Ross) Contract fees from Teva decreased in 99
General Corp (BTGC) -Bio Tropin (HGH) : $17m March 23/05 divests to Ferring its Israel
-OxSodrol (r hu SOD) →ARDS, in dev manufacturing facilities
Class action suit 2002 -r hu anti Xa, PRE
- 134 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- Prosapeptide analgesic ph II, terminated March 05
Changes (June 23/03) to SAVIENT ( SNVT) - Nuflexa : hyaluronic acid for joints (Ferring to MKT in
EU)
- Uricase ph III
SP Specialty Labs (NYSE : SP) 3000 different "esoteric " diagnostics performed.
Founded 1975
IPO filed 9/12 (ML), trading since dec 8
SPA SR Pharma (SPA/LES)/ Atugen - SRL 172 (M.Vaccae) : NSCLC, ph III, fails; booster of
Hep B vaccine to start
-BCG immunotherapies
- PVAC, partnered w/CRXA
ATUGEN AG *
Potential IPO Antisense, lic to Genta (jan 01):GeneBloc molecules for w/G.O.T. Therapeutics GmbH for lipid delivery
-CEO Rudi Neirinckz "knock-down" drug discovery
-VP Bus Dev : Zisi Fotev Roche, Bayer, BI, AZN

SPPI SPECTRUM Therapeutics (SPPI) was Hypoxanthine analogues :


K NEOTHERAPEUTICS (NEOT) -AIT 082(Neotrofin)→Alzheimer, ph IIb,halted, but PET
promising ™higher dose ?,III init feb 01 at high dose alone
(april10), hi lment 6 months completed March 15/02,
June 18/02 could be delisted April 29/02 fails to meet 12w endpoint
K ∏Spinal cord injury, hi
Aug 23/02 restructuring - Parkinson : interim ph II (May
30/02)efficacy 500mg<250 ∏ Halted
- elsamitrucin, topoisomerase I & II inhib, ph II
-AIT 034→Dementia, IND filed Sept 26/01
- AIT 202, obesity, pre.
- Satraplatin,oral , lic from BMY, prostate K; July 02 pre
ph III meeting w/FDA postponed, init Sept 29/03. Ph III Lic to GPC Biotech, Oct 1/02
nd
2 line HRPC expanded to EU. Licence fee $ 22m.
ASCO 03 1st line TTP 5.2 m/2.5
Fully enrol Dec 05 (N = 912). Interim efficacy end 06.
Rolling filing FDA by GPC Dec 15/05 5CPC)
NSCLC + taxol, 1st line, ph III init Dec 9/05
- RenaZorb, lic in from Altair (Jan 31/05), close to Renagel
- SPI 153 ozarelix w/Aeterna for EU, ph II, HRPC, LHRH
antag
- Lucanthone, ph II, K , radiation sensitizer
- Eoquin, apaziquone, ph II, intravesical, K, bladder;
frontline w/transurethral resection 2 ph III (March 07) to

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


init. Data 2009/2010
SQNM SEQUENOM (SQNM) "MassArray", industrial scale SNP analysis : collab w/FDA -Clients : Genzyme, NIH, NCI,GSK, AHP, GNSC,
acq GMNI (May 29/01, completed Sept & Aventis for quality control of live vaccines and GSK
M 20/01 RNA viruses (Oct 25/01) -Partners : Gemini genomics plc (population
The leading provider of instrumentation for genomics database) Incyte, Methexis
SNP genotyping Only 400 of the 40000 genes are involved in "very Genomics (Belgium)
frequent diseases" : this is what SQNM is looking for Hitachi, GSK (diabetes II)
2 landmark patents receive notice of Gemini :
allowance Jan 10 Population (and twin) genomics
-Patent for dg of HBP predisposition Gemini :
-exploring genes of depression & anxiety -sept 6/00 : patent genes for "poor responders"
CYP 2D6 & CYP 2C19 (hyperactive cyt P 450), but
AFFX is marketing a chip for this ?
-sept 14 : w/LSBC isolate 70 proteins concerned with
osteoporosis, OA, diab, cardio-vasc.
-GenMab
SSB STRESSGEN (TSE : SSB) Hsp 7, ph II, respiratory papillomatosis, recomb vaccine,
Ph II in children; endpoint is "intervals between surgery",
phII/ III to init, delayed (April 20/06); ph I/II high grade
cervical dysplasia to init 2Q06
STEM STEM CELLS (STEM) was now focusing on stem cells -lic its device to Neurotech S.A
Cytotherapeutics -use of PBSC as hepatocytes (nov 1) -2 key patents issued (june 01) on harvest of neuronal
C Sept 24/02 staff ¬ 25% -use of human fetal cells to differentiate into neurons stem cells, & separation of hepatic & pancreatic stem
July 05 lic in c-myc ERTAM adult human stem (repopulation of mouse brain OK), liver, pancreas, cells.. Strong adult neurol stem cell patent estate
cell immortalization technology from delayed Sept 25/02 (stroke, Huntington, Parkinson). ReNeuron's cells can
ReNeuron, with "removable trigger" (Myc is - July 29/04 : neural stem cells surviving in brain of differentiate into Tyr-hydroxylase expressing neurons.
a transcription factor) immunosuppressed stroke rats.
- HuCNS-SC (human neural stem cells), ph I to init
(March 9/06) in Batten (neuronal ceroid lipofuscinosis)
STEM UK Stem Cell Sciences plc (STEM : AIM) Nov 15/05 : license hMADS technology (human
multipotent adipose derived cells) from Inserm
Established in Melbourne, headquartered in Nice Sophia Antipolis (Dani & Ailhaud), for :
C Edinburgh, with Japanese subsidiary (SCD - Duchenne
KK, which is a JV with Sosei). - Cardiac
Expanding operations to US and Cambridge preclinical to initiate 2006
UK

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


SUPG M SUPERGEN (SUPG) -Nipent→hairy cell L, MKT, sold to Mayne Ltd June
22/06 $ 34m (sales 05 : $ 15m)
-ExtraMItomycin : branded generic, FDA OK Nov 18/02 ABT expressed concerns in Aug 00 on trial
-Rubitecan orathecin (campto derived oral* management
topoisomerase I inhib)→pancreas K, ph III(June 12/01, 1st ABT terminates March 02
*others are injectable : Campto (AVE, PHA), line, N = 1000/gemzar, too many crossovers , →ASCO 03 (ph III data) primary endpoint (survival)
Hycamtin (SBH) Filed 2 other ph III (409, 400 pts) in Refractory. In one not met
survival not hit, but TTP & ORR OK : Rol. NDA init Dec
April 25/03 class action suit on misleading 30/02 CMC, precl Feb 7/03), completed Jan 27/04. FDA
MitoExtra statements action Nov 04, PDUFA extended (Nov 29) to Feb 26/05.
EMEA filed July 1/04. Jan 4/05 FDA says does not
June 25/03 : PIPE $ 21.25 in convertibles support, SUPG withdraws. Jan 20/06 withdraws patent issued 3 feb 00
EMEA lic from PP
PIPE March 5/04 : $ 34m Ph II + Gemzar 1st line pancreas fails April 26/05
→ ASH 03
Sept 18 : good efficacy in CML
→ASH 03
- MP 470, c-MET and Rad 51 inhibitor, pre : the only
one in this category, could potentiate
chemotherapy & Radiotherapy
-Decitabine, DACOGEN IV (DNAmethyl transferase
inhib),myelodysplasia ph III versus BSC, May 12/03 fast
track. CEO says survival benefit (PHRM Vidaza does not) lic to MOGN Sept 1/04
Feb 13/04 interim : time to death or transformation to
AML improved. reanalysis April 1/04 logrank p = 0.198, Oct 1/04 filed EU by EuroGen , SUPG's EU
Wilcoxon p = 0.042. Rolling filing init May 31/04 (CMC) subsidiary
Completed Nov 2/04 (will get milestone from MOGN)
ph II init + Gleevec June 14/03 CML
ph I/II init JUne 30/03 w Carboplat, K
Sept 5/05 : FDA approvable, additional anal of transfusion
data. OK May 3/06 N = 170, ORR 17% vs 9% p < 0.001.
Royalties from MOGN 20% up to sales $ 50m, then +
2.5% per 50m. Peak 30% DEAL
-Water soluble Taxol→→→→→→→→→→→→precl
-MAb for vascular targeting→K, pre
-AVICINE (from AVI) hcG vaccine ph II, ph III soon
- aurora kinase drugs pre, from MONTIGEN
-VEGF targeting (w/PPHM)

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


SYBB SYNSORB BIOTECH (SYBB) -Synsorb Pk : carbohydrate to inactivate and treat E coli O Takeda for Japan
157:H7 toxiinfect; ph III (fast track) May 25/01 provides CND govmt with emergency
Dec 12/01 : staff trimmed 50% given within 2 days, decreases HUS 59%, comp. to plac., supplies
discont dec 14/00
Complex litigation/merger with Scout Synsorb Cd( Clostridium difficile)→pseudomembranous
Capital (May 02) colitis, ph II.: Recurrence rates : -placebo : 41.7%;-verum
21.9%. Terminated Dec 12/01
TARG IPO TARGANTA Oritavancin, lic Dec 05 from ITMN who had licensed from
filed May 07 LLY Sept 01. Phlebitis at injection sites.
Traded Oct 10/07 To file NDA 1Q08, seeks non inferiority delta ph III 15%
(FDA usually requires 10%)
TBIO TRANSGENOMIC (TBIO) -WAVE system for SNP scoring MRK, GSK, LLY, NIH, CNRS, GeneOdyssée SA
-automated systems for nucleic acid analysis
TELK K TELIK (TELK) -TLK 286 Telcyta,nitrogen mustard Canfosfamide, (might ASCO 02 : refractory ovary ph II ORR 15%;
be activated by non K cells) K prodrug, ph II,activated byrefractory NSCLC no ORR
CEO Wick, ex Lederle, Dana Farber GST P1-1 (Glutathion transferase). Jan 02 : enrol compl inMicroscopic hematuria 10% of pts
CMO Gail Brown, oncologisy from Harvard CRC N = 73 (CEA ↘), breast init.April 02 , NSCLC N = AACR Nov 19-22/02 in irinotecan failures TTP 2.9m,
51, ovary K N = 36. Oxaliplat 1.6m, 5FU/LV 2.7m
was "Terrapin" mNSCLC 1 line ph IIb (AACR 07) N = 129. First patients ASCO 03 refractory NSCLC, ovary, breast
st

receive Carbo Taxol & Telcyta. 77% have objective


Secondary filed Sept 12/02 ∏ $ 74.4 Sept response or stable. Half go on with Telcyta, half do not.
30/02. w/Telcyta OS 14.2m, without 8.4m, p = 0.0003. But
overall OS median 9.6 m. This only demonstrates that
Secondary filed Nov 03 raises $ 130 m maintenance treatment can be useful.
(UBS, Lehman, Bear Stern, Needham,
Lazard, Fortis). ASSIST I Ovary ph III init March 27/03, 3rd line, N = 440,
Jan 28/05 secondary $ 91.6 m random , fast track, vs Doxil or Hycamtin. Accrual 15m, ASCO 05 : frontline data ASSIST II
endpoint survival. Dec 30/04 fully accrued; powered to
detect 28.6% delta. ASCO 03 ORR 18% bur only N = 11
(DOXIL FDA ORR 13.8%, Hycamtin 21%)
. Data 4Q05/1Q06 : Dec 26/06 FAILURE. ASCO 07 OS
8.5m vs control 13.6 March 13/06 : ODAC rejects Gemzar/carbo in 2nd
line ovary on PFS
ASSIST II FDA OK Sept 26/02 for ph III for NSCLC,
Fast track granted Dec 2/03, 2nd /3rd line, N = 520, to be
compl. end 1Q05, vs Iressa, endpoint survival. Data
4Q05/1Q06
ASCO 05 : interim data ph I/II 1st line w/cisplat : 36%
ORR; also Telcyta/carbo/taxol 62% ORR
ASSIST III, 3nd line ovary w/carbo, vs Doxil alone, init

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Dec 29/04, N = 244, endpoint ORR ( ph II ASCO 04 ORR
63% , & Doxil ORR 2/3rd line FDA 19.7%). Data release
end 05 ? NO : data 1Q06 : but ORR is worse than PFS ,
and data could be read Carbo > Doxil !!
June 07 FDA on hold, Oct 15/07 hold removed
-TLK 199, Telintra, myelostimulant, I/II, MDS, init April
23/02. ASH 06
-Insulin receptor kinase modulator, TLK16998,pre, oral
→ADA (June 01)
TEVA SICOR (SCRI) was Gensia Sicor Generics for hospitals (injectable) : and IFN α2b
Acq Biotechnica, Vilnius (Lithuania) July 01. (Reaferon) registered in 16 countries
FDA inspected.

Acq by TEVA
TGEN TARGETED GENETICS (TGEN) -AAV-CFTR, ph I/II, ph II data June 5/03 : trend in FEV1 Celltech (term Dec 2/02), discont March 16/05
-AAV-E1A(anti her2/Neu)→K H&N, ph II (on hold until ASCO 02 ovary 6/7 pts have reduct of CA 125
Aug 10/01 acquired Genovo (owned by partner found)
BGEN). It's good to be near BGEN, but -AAV-VIII(B domain off), pre;clin for 2003, resp delivery ™WYE (NOV 9/00). Term Dec 17/02
Genovo was involved in questioned gene ?
therapy trials -AAV-HIV, tg AAC 09 ph I (Bel, D, India), IM; Ph II S. International AIDS vaccine initiative; WW around 30
Staff ¬ 25% Aug 5/02 Africa init Nov 05. N = 78 x 2 + 16 Zambia, data end 07. . preventive vaccines in trials
Staff ↘ 50% Dec 17/02 But (ph I) is there an immune response in humans ?. Elan
-Emerald, collab for 2nd gener →→→→→→→→→→ Collateral Therap
Class action suit Jan 27/03 -AAV-ad cyclase gene→CHF, pre→→→→→→→→
- AAV SERCA 2 alpha gene (Mydicar) coronary infusin
for heart failure, w/Celladon, ph I
- AAV-IX, res
- tg AAC 94, pre, AAV/TNFR-Fc, efficacy on RA in
animals. Ph I, safe and effective (sustained improvement in
7/9 pts 8 w after tmt, ph I/II N = 40 data mid 06, amended
March 16/06 to N = 120. Interim June 07 : 33% pts high Others Huntington : Trophos (Marseille) : ALS,
dose get 2 points reduct in swelling at 12 w. 0 w/ placebo. Spinal Muscular Atrophy
Full data mid 08
- Jan 11/05 partnership w/RNAI (Sirna) on AAV delivery
of silencing sRNAs for Huntington
- AAV-ornithine transcarbamylase July 29 patient dies
liver failure (should not have been enrolled)
- AAV + RPE 65 gene ph I/II intra-ocular

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


THLD K THRESHOLD (THLD) - Glufosfamide (ifosfamide + glucose), ph III, 2nd line ASCO 06 ??
pancreatic K w/Gemzar, fails July 17/06; anal Feb 27/07
filed April 2004 CIBC, traded March 05 fails again on OS; BUT
Sept 27/07 in
diabetics taking sulfonylureas OS 13.7
m vs 2.4 m
Jan 31/07 init ph II Pt resistant ovary K, SCLC (halted Oct
11/07
April 9/07 soft tissue sarcoma
- TH 070, ph III, BPH, N = 500, results early 4Q06; liver
tox May 12/06
- 2 deoxyglucose, ph I, solid K
THRX THERAVANCE (THRX), was "Advanced -Telavancin,IV, glycopeptide ph II : efficacy ++ GSK has option to lic. all their products
Medicine" (ACCMID, Prague, May 11/04). Lic to Astellas
AB (Tokyo)Nov 8/05 ($ 221m) competitors : PFE , Zeven (dalbavancin) from
files for IPO June 14/04, traded since Oct 04 - ATTAIN : Hosp acq pneumonia staph/pneumo vs Vicuron/ Biosearch Italia/ versicor : "approvable" July
20% owned by GSK; had a call to acq 50%, vanco, ph III, last patient enrolled June 21/07 20/06, still under FDA review (Jan 4/07).
did not (July 2/07) - SSTI, ph III, N + 1867. Aug 23/06 : non inf to Basilea /JNJ has a nice molecule (ceftobiprole) filed
(Lehman, ML, Perseus) Vanco (microbial eradication 89.9 vs 85.4 p = 0.053). 2007.
Filed Dec 9/06, but is "non inferiority "sufficient"?
Significant side –effects : QT, hypoK, FDA action YE 07, PDUFA Oct 20/07; APPROVABLE
nausea, insomnia Ph II data July 10/07 clinically slightly > to Vanco
Filed EMEA April 07
- Long acting beta agonist (1Q05 lic by GSK), ph II good
data June 27/05
- Long acting muscarinic antag (lic by GSK 1Q05) .
Competitor of Atrovent BI, Spiriva PFE/BI) TD 5742,
GSK 656 398, ph I June 13/05
- TD 1792, AB, Astellas opts out Sept 7/07
TLCR IPO TALECRIS - Gamunex (IVIG) : $ 162m 1Q07, for ITP, CLL, BMT
filed (MS) July 30/07 (but CSL Behring will launch IVIG in US 1Q08
- alpha 1 AT
- Albumin
- VII + X
TLN K Thallion, was Ecopia Biosciences (TSE/EIA) Microbial genomics initially June 6/07 partnet w/ ABT Ab targets in lung cancer
- ECO 4601, ph I/II, anti MAP kinase (RAS pathway),
Jan 5/07 to merge w/Caprion crosses BBB, completed Aug 22/09
- CAP-232, ph II melanoma, acts on glycolytic pathways.

Caprion Pharmaceuticals* (CND) Cell organelle proteomics, dg products for CJD (MAbs
from IDXX)
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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Topo K A/S - Savene Totect , MKT, topo II inhib for extravasation. To the 1st HDAC inhib (MRK SAHA, Vorinostat,
launch US 2H 07. Approved Sept 7/07Sales 1H07 DKK Zolinza) was approved for CTCL Oct 6/06
April 11/07 acq Apoxis : 8.3M
- APO 866 , inhib of nicotinamide - PDX 101, belinostat, HDAC inhib, oral & IV , 18 trials PDX 101 time to response 1 m vs Zolinza 2 m
psosphoribosyltransferase, ph II melanoma & ongoing, ph II, CRC, CTCL, peripheral T cell lymphoma,
CTCL MM 2nd 3rd line w/Velcade after Velcade alone N = 35, Competition : MRK on MKT, Bayer MS 275 ph II,
- APO 010, recombinant modified fas ligand, data YE 07; w Curagen. ASCO 07 ph I/II in advanced K : Methylgene/Pharmion MGCD 103 ph II, LBH 589
ph I very safe, and HCC 1st line ph I/II. Ph III to init 2008. Novartis
AML ph I/II init Aug 28/07. Oct 07 Curagen increases
expenditure. Oct 25/07 AACR positive data ph II ovary
alone or w/carbo-taxol
- Avugane, HDAC inhib, ph II, acne
- Baseca, HDAC inhib, ph II, basal cell carcinoma, lic to
LEO, PSO
- Topotect to protect from side effects of Topo II inhibs
- Zemab, anti Her 2 with Pseudomonas toxin, ph I
- Oct 4/07 w/Lundbeck : siramesine (sigma 2 receptor
ligand) , caspase independent apoptosis inducer, to go
into ph II (ph I anxiety, safe)
TPPH K TAPESTRY (TPPH), was napro - TPI 287, 3rd generation taxane, ph I. 2 posters ASCO 07.
Biotherapeutics Poor efficacy, no neutropenia
- Quassinoids, precl

TRBN K TRUBION Monoclonal like molecules with only F(ab) and Fc : anti CD 20 lic to WYe Jan 3/06 $ 800m, upfront 40m
- TRU 015 anti CD20, ph II, RA data 2H07 (enrol compl DEAL
traded Oct 18/06 (TRBN), Morgan Stanley, Jan 17/07, so end of trial July 17/07, data Sept 12/07 24 w oct 5/06 partenr w/DYAX
Pacific Growth, Lazard ACR 20/50/70 : 61%, 13%, 4% vs placebo 33/9/2). Single
dose ) B cell depletion similar & efficacy similar to
Rituxan, perhaps less activation of C' (less intolerance ?),
SLE
- TRU 016 anti CD 37, pre, NHL, CLL
TRCA TERCICA (TRCA) Increlex (r Hu ILGF1) filed Feb 28/05 for children licensed from DNA
resistant to HGH. Priority review, PDUFA Aug 3/05. To
US MKT 30,000 prevalence of short stature file in EU. Approved Aug 31/05
w Nl or high HGH (6,000 have low ILGF) Ph III in severe IGF1 deficiency showed statistical efficacy
Competitor Insmed : March 7/07 wins patent on 8 years.
fight vs Insmed Phase IIIb ongoing in less severe cases. Also hopes for
obesity, osteoporosis

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


TRGA TREGA BIOSCIENCES (TRGA) -Combinatorial chemistry(iDEA, ADME simulation in- Chugaï, IMNX, ONO, ROCHE
silico)
Acquired by Lion Biosciences AG. on March -Melanocortin receptors: Novartis
12/01 MC-4r (obesity, diabetes)→→→→→→→ ONO
MC-1r (pain, asthma)→→→→→→→→→
-HP228→pain, ph II
TRGNY K TRANSGENE (TRGNY/Nasdaq;LNM -Ad-CFTR, ph I. Terminated BioMerieux Alliance is the holding company of
6274) -Vero-IL2→melanoma, ph II, cellular vector will be TRGNY & BioMerieux (which sells reagents
Founded in 1979. Since then has burnt more replaced by adeno for in vitro dg)
than € 200m -Ad-IL2→K, ph I Partnership with SGP (who has lic-in TRGNY's Ad
-pox vector,Muc1 vaccine→breast K, ph II. N=31, 2 pts for its p53)
Transgene has less partnerships and more have tumor reduct > 50% HGS has 10% (giving TRGNY access to data base &
employees (197) than any other gene therapy - MVA (modified virus Ankara) + Muc 1 + IL2, to start ph right to 10 genes)
company II : Breast, prostate, NSCLC ,w cisplat & Navelbine
-plasmid with minidystrophin (™Duchenne), ph I initiated Partnership w/ Mirus Corp for IV musculotrope
sept 00 delivery of minidystrophin ( but Univ of Pittsburg
Dec 20/00 : BioMerieux merges w/P.Fabre. (PNAS nov 28/00) has AAV + dystrophin, active in
BMPF has 70% of T., Mr A Merieux & Mr P - TG 4001, therapeutic HPV vaccine for highgrade animals)
Fabre jointly own 52.8% of T. Demerger intraepithelial neoplasia : clears HPV 16 (Nov
June 02, TRGNY stays w/BioMerieux. 13/06). Ph III to init Q3 07, now (April 07) YE
07 or 08, lic to Roche € 218m
New CEO : Gilles Bélanger (all his career - TG 4040 (MVA + HCV) therapeutic vaccine for HCV,
w/Merieux). Then (July 02) JF Carmier ph I/II init Jan 13/07
New issue offering for €80m : April 23/01, - TG 4010, IIb, NSCLC, MVA MUC1 – IL12
raise 63m (May 25/01) from Merieux, PF, - TG 1042 : CTCL, ph II, adeno IFN gamma
AFM.

CV to belicensed out, using HGSI genes :


CTFG2 for ischemia & TIMP4 for restenosis.
TRGT IPO TARGACEPT (TRGT), filed Feb 4/05 Neuropharmacology
Pacific Growth
- Inversine (mecamylamine), MKT, HBP
posponed Feb 28/05, refiled March 11/06 - Isopronicline, ph II, Alzheimer (targets NNRs, neuronal
TRADED APRIL 12/06 nicotinic receptors)
- TC 2696, Pain, ph II
TRIBY TRINITY BIOTECH (TRIBY) -gold particle tagged MAb for invitro dg :
Hep B, H. pylori, Chlamydia, HIV
-Saliva test for HIV antibodies

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TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


TRMS TRIMERIS (TRMS) Peptides that inhibit(bind gp 41) viral fusion to cells.
PIPE completed May 15/01 -T20 Fuzeon(36aa) enfuvirtide→AIDS subcut 90mg BID ROCHE; FDA fast track, manuf of commercial
PIPE for $ 41m filed Jan 26/02 or minipump ph II viral load ¬69-97% in non-responders supplies initiated (Boulder, Co),validation
Secondary $ 111m, Aug 26/02, netted $ 102 to other drugs. 2 unblinded ph III (TORO 1US,2 EU)in end 02/1Q03, to produce 3-5 tons/year. But
(Sept 26/02) for dev & commercialization association with optimal (genotyping + phenotyping) 106 stages. Roche PR Aug 2/02 : cannot
costs of Fuzeon antiviral CT, fully enrol. Aug 6/01, last patient to reach 6 exceed present capacity in the short term.
EPS + in 03 : 50% of operating profit in m. endpoint March 02. Launch 03. Fast track.
N.America (will Roche do all the MKT ? Early Access program opens Aug 19/02 : is it 1st step to KEY AIDS DATES :
not clear in 10K: TRMS will pay $ 25m in rationing ? - ICAAC01 : ph I/II pediatrics efficacy + safety
2003), 10-12% royalty in Europe TORO 1 (N=491) viral load ¬ 1.7 log (control O.763 log, - Retrovirus conf : Feb 24-28/ 02 : viral load reduct –
Peak sales $ 800 m at 10, 000/Pt with 25% p<0.0001) a bit different from retrovirus conf 2.62 logs/background alone – 1.87)
penetration of those who failed 2 combis. TORO 2 (N=504) data 17 May 02: viral load 24w ¬ Results : World Aids conf July12 02
Could be much more.Dec 02 : price raised to
1.65log (1.43 at Barcelona), control ¬ 0.65 (p< 0.0001). ICAAC : full data Toro 2
$ 16,500→20000 (Feb 25/03) 48 w data 4Q02
Filed FDA Sept 16/02 priority review granted (OK March In the western world 500,000 pts are treated
BUT : July 2/02 at Seville resistance workshop Visible 14/ 03)., CPMP filed Sept 20/02, OK March 20/03 for AIDS. 80,000 could be treated by TRMS,
Genetics detects mutations linked to T20 intense 25,000 were forecasted for year end 2003, now
selection pressure. This could compromise use in non 2Q/03 US $ 4.3m, ex US 0.87, 3Q 13.1 (US 10.7) , 2003 $
NRTI resistant patients. 35m, 2004 $ 135 only 12,500 ??, perhaps only 7,500 ( Dec 19/02,
COULD SUPPLY EXCEED DEMAND ?? guidance May 12/03 : 8 to 10,000); could be
March 20/03 : will be distributed in US by Chronimed - end 05 sNDA planned for needle free Bioject delivery
(CHMD) who provide no guidance (April 22/03) even
enough for 37000 pts in 2004 (→ 2005). July
for 2004 ! 15/03 Roche analyst meeting : manuf facility
Mid 05 the super protease from -T1249→AIDS, subcut, ph I/II, can be given once a day. to be expanded (WHEN??) for Fuzeon & 1249
Boehringer Tipranavir could be approved, On hold Jan 6/04. Halted
and it's association w/Fuzeon could be - June 14/05 TRMS alliance with Chembridge for "new COGS around 33%
excellent (CROI, Feb 05) peptide".
-RSV
-Influenza
TRPS TRIPOS (TRPS) Leading provider of Biotech software Lion, NN, Arena, BMY, Merck KGaA

TTP M TITAN (TTP) -Zomaril(iloperidone)→schizo, ph III;filing delayed (July Novartis, lic from Aventis in 97
01)→ end 02, approval ?? 04. July 22/02 Novartis
Schering AG
C compares to Geodon : QT↗. Dec 05 NVS licenses to
Vanda
-Spheramine→Park, ph I, encaps. hu DA retinal cells
-CeaVac-(antiidiotype MAb)→K colorect, ph II→phIII
(June 19/01)Dec 11/02 endpoint missed, NSCLC (+
TriAb)
- TriGem (antiidiotype vaccine), seems very effective in
neuroblastoma (ASCO 01), ditto melanoma

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-Rb gene therapy, pre
- Pivanex, ph II, NSCLC, histone deacetylase inhib.

TWTI THIRD WAVE TECHNOLOGY (TWTI) SNP scoring with "Invader"


IPO filed 7/31 (LB), traded since Feb 12/01

UBDE C US Biodefense (OTC BB : UBDE) - liver cell progenitors


forms subsidiary "Stem cell research institute - human neural crest stem cells
of california" - drug resistant hemopoietic stem cells
- human oral keratinocytes (for hemophilia, diabetes)
- non marrow stem cells for cardiac regeneration
UCB Celltech Chiroscience/Medeva acq by UCB
M (CCH/LSE;CLL/Nasdaq)

UCOR UROCOR (UCOR) Services for urologists (dg, trials, specimen banking)
UGNE UNIGENE (UGNE,OTCBB) Oral PTH, pre, osteoporosis Lic to GSK (April 15/02)
Competition LLY Forteo (ph III)
NPS ph III
EMIS oral, pre.
URGP URIGEN was VALENTIS (VLTS) PLASMIDS IN LIPIDS merger of Megabios & GeneMedicine (03/99)
Oct 9/06 to merge w/Urigen -CFTR, ph II GLX
Aug 17/01 : w/Flemington, sublingual insulin -IL12→H & N K, ph I/II Roche
spray (res). -BRCA 1→ovary K,pre LLY
Jan 17/02 : closing the GeneMedicine -IL2 IV, NSCLC, ph I
facilities - IL2 local H&N, w/Roche, Terminated dec 3/01
- VEGF 165, CAD, PAD ph II (Finland)
Staff ¬ Oct 9/02 - Del 1(development regulated endothelial locus 1), ph I/II
: PAD, ischemic heart. Sept 29/04 misses endpoint
July 10/06 : to sell ? - VLTS 934 : non ionic poloxamer (anti-inflammatory) ph
IIb. Jan 11/06, N = 148 vs placebo. Important competitor : Cilostazol (Pletal)
improvement in exercise tolerance, fails July 10/06
UTHR M UNITED THERAPEUTICS (UTHR) -Beraprost ,oral prostacyclin derivative→PVD, PHT -Toray, Japan
K 2nd ph IIIenrol.compl feb 26/01 (there had been a first
one by Aventis….)but is Aventis still interested) Oct 15/01 : ph III PVD failure
Price for Remodulin $ 50,000/year CLAIMED THEY Would GET EUROPEAN APPROVAL
IN Q1/00 !!!!!!

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


-UT-15 (Uniprost/Remodulin, treprostinil), subcut
Total Remodulin pts in US around 700 prostacyclin analogue for PHT ,that could be the successor
of Flolan IV(epoprostenol). –EU filing through France (feb Amer Heart ( McLaughlin)
5/01). Rejected by AFSSAPS SEpt 23/03. Approved France Approvable letter Feb 11/02 Improves dyspnea, but
Feb 23/05 patent extension until 2014 March 8/05 not distance in 6 min walk (Tracleer does). Approved
-Aug 14/00: incomplete filing to FDA,oct 16 complete class II to IV May 24/02 (Flolan & Tracleer are III &
Told by FDA "no need for Adv. Com on feb 9" ??. April IV). Ph IV requested to show that Flolan patients can
June 6/05 : sildenafil approved (Revatio) 11/01 FDA adds 3 months delay Adv Com Aug 9/01 : be switched. Aug 21/03 :ph IV timeline increased
20 mg tid OK6/3. FDA OK May 22/02
sales 03 $ 49, 1Q04 12.6; 2Q 16.2, 3Q 18.4, 2Q05 28.5;
June 13/06 lic oral formulation of 1Q07 38.2; 2Q 49.4; 3Q 57
Remodulin from Supernus - Feb 2/04 files for IV with only Bioequivalence data
(PDUFA Nov 30) FDA could want to see data from
Sept 4/07 deal w/ARDM : WHY ?? Viveta ongoing open label, UTHR could file it Oct . FDA OK IV
troo bulky ???? Nov 24/04 "for those not able to tolerate a subcut"-
Inhalable Remodulin (VIVETA) initiated (but
(Jan 9/07 Actelion acq Cotherix for CoTherix ?? Ventavis (Iloprost from Shering AG,
Ventavis) approved in EU, and US DEC 29/04, but UTHR
inhaler very bulky. However VIVETA trial TRIUMPH
positive data Nov 1/07

- Ovarex orgovomab IV murine anti CA 125 (ascites), ph


III, lic from AltaRex for US (April 22/02). Oct 15/04
AltaRexx acq by ViRexx.. 2nd ph III enrol compl June
20/06. N = 177, data Nov 5/ 2007: FAILURE. Prior trial
not positive
VBI K VIVENTIA (VBI TSX) - Proxinium, ph II, cytotoxic Mab to EpCAM, H & N K
- Vicinium

VGIN VISIBLE GENETICS (VGIN) Leader in HIV genotyping, using DNA sequencing, rather GLX
than probes such as those of Affymetrix, Nanogen
Being acq by Bayer ($ 61.4 m cash) July or Innogenetics
24/02
VGNX VARIAGENICS (VGNX) Pharmacogenomics : streamlining development by Covance, Quintiles
Nov 11/02 : merging w/Hyseq eliminating (with SNPs & haplotypes) non responders.
VIAC ViaCell (VIAC) -Umbilical cord stem cells : system to collect and preserve Aug 30/05 publication on human umbilical cord stem
IPO filed Jan 30/02 (UBS, CSFB), pulled Jan baby's cord blood stem cells (Viacord). cells, piglets improve ejection fraction after infarction
C 5/04, refiled April 04, traded Jan 23/05 ACC March 06 : dose dependent response in animal
myocardial I nfarction. June 13/06 lic to JNJ/Centocor

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


equity held by AMGN, GENZ, GSK - CB 001, ph I, cord blood technology w/AMGN, data end
06, K
Goal "true off the shelf stem cell product" - SCF, Flt3 ligand, non exclusive ww license from AMGN
for ex-vivo cell therapy ( Jan 2/04)
- Viacyte : system to preserve and store human eggs
- w/GENZ, partnership to create an islet stem cell
transplant product for diabetes
VIAP VIA was CORAUTUS (VEGF) - Gutted adenos, w/Centocor (May 8/02) BAXTER
was GENSTAR (GNT), was -Maximum : VIII in adenovirus derived vector : ph I (June
Urogene(UROG) 18/01), suspended for coag & hepatic issues. Restarted
April 3/03, Staff ↘ Sept 25/01; 8 months after the patient still has 1% VIII
(Bob Sobol) - MAX AD Prostate (IL3)
- Lentivirus as vectors for HIV genes
- VEGF 2 from Vascular Genetics : IIb init 2H03. Catheter
merged w/Vascular Genetics → AUTUS → delivery of VEGF for severe angina; ph II suspended
CORAUTUS Feb 6/03 March 14/06 (pericardial effusion)
Feb 8/07 merger w/ VIA (oral leukotriene - VIA 2291 (from ABT) 5 lipox inhib; carotid
synthesis inhib for carotid endarterectomy) endarterectomy, ACS.
- Library of PDE inhibs from Neuro3d (Strasbourg)

VICL K VICAL (VICL) - HGS (hepatic growth factor) w/plasmid lic to AnGes MG strategic alliance HGS (feb 23/00),and Vascular
Inc (Osaka) who partnered w/Daichi, ph III (cf Genetics (private, owned by HGS),on
"GENES IN A BOTTLE" VICL web site)peripheral heart dis & ischemic VEGF2
heart . June 18/07 strong data ph III PAD.
Preparing to file in Japan

-Allovectin(HLA-B7), ph III→melanoma, completed June


02, data 2H02. Term Sept 19/02 for low dose.
CEO quits (April 00) Now high dose ph II → ASCO 04
SPA (Feb 10/05) for high dose vs dacarbazine or
temozolomide, ph III, N = 375, end point ORR at MRK (influenza, HBV, HCV, HIV init Jan 7/00); Aug
24 weeks. To init 1Q07 w/AnGes 21/03 influenza, HPV & HSV returned. MRK gets 3
new K vaccines.
→H & N,ph II (august 01 H&N conf only 10%
Aventis (PMSV)
of patients show response)
JNJ (Centocor) for K
-Leuvectin(IL2),ph II,Terminated (april 01) renal K
failure. Ph I direct intratumor, then poration
Nked DNA vaccines→→→→→→→→→→→→→
- Anthrax effective pre
- Flu vaccine very positive data H5N1 in mice & ferrets,
May 2/06

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


VION K VION PHARMACEUTICALS (VION) -Promycin (alkylating)→K, ph III, fails interim anal. Boehringer Ingelheim

-Triapine (aminopyridine carboxyaldehyde


CSO Terrena Doyle was in charge of Taxol thiosemicarbazone, an iron chelator), K, (inhibitor of other RR inhibs are Hydroxyurea, fludarabine,
10 years gemcitabine
June 2/02 staff cut from 50 to 35 ribonucleotide reductase) + Gemzar, AACR Nov 02 →
Synergy, ph II init May 03 pancreas , NSCLC : Sept 04 not ASCO 04 ?
Feb 10/04 PIPE R & Renshaw > to Gemzar alone With Sloan Kettering for metastasis imaging
Feb 6/04 trial in prostate & H&N w/Triapine alone
Claim they have the rights on 3TC. terminated.
Interference case with Biochem
Pharma since Nov 29/99 -TAPET : use of genetically engineered Salmonella to
deliver CT or * to K,ph I
- VNP 40101M (cloretazine, sulfonylhydrazine) ph II, ph I/II Int symp and expert workshop on leukaemia &
glioma Fast track 2nd line elderley AML, ph III(SPA OK lymphoma, Amsterdam, 16-19 March 05 :<30% ORR
Feb 10/05) initiated March 28/05 : Ara C +/- cloretazine, N elderly AML
=420, accrual 30m. Endpoint CR & CRp (powered for ASH 05
50% delta), interim at 210 pts; data bof Oct 26/06. AML
terminated May 23/07
VIR K VIRexx Ovarex, lic to UTHR

VLGC VIROLOGIC (VLGC) - PhenoSense HIV,MKT, to measure clinically signif drug


Pipe Nov 02 $ 7m resitance (adapted to fusion inhibitors)
PFE acq (Nov 02) 5% - March 05 : w/ AZN and w/DNA tests for mutations in
Staff Nov 02 ¬ ; N = 40 tumor and skin (dimerization of receptors)
June 2/04 : to merge w/Aclara
VPHM VIROPHARMA (VPHM) -PLECONARIL (antipicornavirus) SASY for Europe (renegotiated), lic to AVE (Sept 01)
.viral meningitis, filing 2Q00™™™™™™™™™ Term Aug 2/02
De Rosen CEO (Aug 22/01) .common cold: VRI (viral respiratory infection) 2096 pts, results +, but not remarkable (April11/01)
65% had picornavirus. Duration of running nose 6.2
days/7.7 (p<0.001).Filed Aug 1/01 Negative view in Cholesterol increase ??
Barron's (May 01).Not approvable letter June 5/02. Lic to Adv Com March 19/02 15/0 against(menstrual
SGP Aug 04 anomalies, arrythmias)
-Hep C VP50406 : ph II. Terminated
-VP 14637⇑RSV, ph I (oct 10) discont. Sept 24/01 w/AHP
(animal CNS tox)
- Maribavir, ph II, CMV in allo BMT. Data end 1Q06
- HCV 796 for hep C, ph Ib. Safety : discont Aug 12/07
- Vancocin (oral vanco not reabsorbed) lic from LLY (Oct
22/04; $ 116m), for Clostridium difficile diarrhea
(LLY sales 2003 : $ 28). Will price 20 x 250 mg

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


capsules at $ 446. Sales for 9m 05 : $ 92m. March
17/06 generic threat
- Feb 27/06 lic in non toxicogenic strains of c. difficile, to
treat and prevent CDAD
VQPH VioQuest (VQPH OB) -VOD 002 API- 2, tricyclic nucleoside inhibitor of Akt
merging with Greenwich May 05 (and test for Akt), ph I/II at Moffit & MD Anderson
Meichel Weiser, from Paramount, on Board -VOD 001, Na Stibogluconate (was for leishmaniosis),
tyrosine phosphatase inhib, ph II (Taussig)
VRA VIRAGEN (VRA) IFN α natural human leukocyte derived : high dose (9
MIU/d) provide esperimental protection for SARS

VRML VERMILION was CIPHERGEN (CIPH) Chips for proteomics (SELDI) : surface enhanced laser ALKS/AMGN/AZN/BI/CAT/CNTBY/CBMI/GENE/
desorption/ionization chips GLX/NN/WLA/AVE/SGP/SBH/Roche
Biosepra (Cergy Pontoise) sorbents for high scale
separation of proteins
VRTX K VERTEX (VRTX) -Agenerase (amprenavir)protease inhib, mkt GSK (CPMP OK june 30/00). Royalties negligible: $
- Lexiva fos-amprenavis, VX175 (908), less pills,filing 17m 2004
Staff 980 , then 848. June 11/03 ↘ 10% US/EU Dec 02 → FDA OK Oct 20/03. July 27/03 GSK
Aurora acq (July 01) trial misses endpoint (non inferior to Kaletra). EU OK July 7/02 AIDS meeting : 2 ph III data NDA & CPMP
March 23/04 filed Dec 23/02
Secondary June 05 : $ 153m - VX-385, , anti HIV protease, ph II, w/GSK
Aventis R/D day June 18/02 too enthusiastic -VX 740 Pralnacasan, oral, IL18 & ICE inhib (IL1 conv
on 740 enz)→RA, ph II April 25/02 : ACR 20/12w p=0.07.
Schering AG - VX 765 proprietary ICE inhib ph I to initiate 2Q03 AVE upfront $ 20m. Full dev funding. Milestones $
NVS -Oral IMPD inhib like Ribavirin : 62m RA, others for OA; launch 2006,ACR Oct 25-
IMPD : inosine monophosphate -Hep C, merimepodip VX 497, ph II 29/02 : subgroup anal signif at high dose (1200 mg/d
dehydrogenase -PSO, ph II, VX 148, ph II ; ACR 20 @ 6m = 44%, but Enbrel 65%!). OA ph II
-Timcodar (neurophilin),diab neuropath, ph II init Jan 03. Nov 11/03 AVE halts (animal liver tox) ,
-VX 710, MDR & MRP antag, ph II waits for 12 m tox (in > 12m)
-Kinase inhibs→→→→→→
- VX 950, telaprevir Protease inhib Hep C, oral liquid, to
Hep C : 10,000 deaths /year in US (33% of become tablet, ph Ib init 2H03, June 14/04, lic Asia to COMPETITION :
all liver transplants). INF α + ribavirin gives Mitsubishi. May 10/05 : 14 d reduct in viral (HCV ITMN is only preclinical, Idenix is ph I/II (April
12/07 53% naïve pts reach 48 weeks undetectable).,
38% sustained viral response . genotype 1, 70 to 75% of US pts) load 4 logs
aasld Nov 2-6/07 also SGP protease inhib SCH 7, Anadys, GILD,
(IFN α leads to 1-2 log decrease) (see also Avalon AVN- Roche 1626 (bone marrow tox), Wyeth HCV 796,
944, ph I, Sept 05).Ib combi to init YE 05 Ph II 2006, ph Coley Actilon, HGSI Albuferon, Pharmasset (VRUS)

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


III 1H08 (filing 2010/11). 28d trial init w/ IFN & w/Roche
RibavirinDec 5/05. Feb 6.06 drops to undetectable ! July
10/06 lic ex US to JNJ.
Oct 27/06 : active on Hep C wild and resistant Nov 2-6/07 two large ph II : PROVE I (US) & II
Jan 07 init of PROVE III in hep C refractory to IFN (EU) AASLD : SVR (sutained virological response)
-VX745,oral p38 MAP kinase inhib→RA, ph II, low 60% for Prove I, but Prove 2 79% undetectable at
terminated, CNS animal tox 12 w, 70% in P. I.
- VX 702, 2 generation oral P 38 MAPKinase inhib ph I Side effects rash (54% vs 31% IFN Riba ), nausea
nd

init June 12/02, RA ph II init June 10/05; March 9/06 : (50% vs 31%) in PROVE II at AASLD Nov 07.
12w ACR 20 40% (placebo 30%). Ph II in acute coronary. Discont rate Prove I 18% verum vs 3% control
- VX 409 Na channel modulator, pre, lic to GSK Dec
13/05 $ 405m (upfront 20)
- VX 322 w/NVA, anti Flt3 & cKIT
- VX-680 (MK 0457), small molecule inhib of Aurora
kinase 2, FLT3, JAK2 , BCR-ABL (active on Aurora competition : Rigel, Merck Serono, MLNM,
T3151mutation). K,. Interferes w/cell cycle (centrosome); Cyclacel, Sunesis
competition see Rigel), lic to MRK. Ph I good. Dec 7/05 3
phase II ongoing. Pivotal ph II init Dec 21/06in CML &
ALL w T3151 mutation (MRK could file on this). N
=270. Recruit suspended Nov 20/07 (QT elongation)
- May24/ 04 : discovery of small molecules active on
CFTR, see CFF conf June 5/04

VX 680, lic to MRK June 22/04 ($ 350m)


VRTX AURORA BIOSCIENCES (ABSC) Ultra High Throughput Screening AHP, Becton Dickinson,, BMY, LLY, Roche, DNA,
GSK, MRK, PFE, PNU, WLA,Organon
Acqcq by VRTX (July 01) GenomeScreen (but a collaborator left on March 4
VSGN VASOGEN (VSV, now VSGN) - Celacade : to init ph III 3Q/03 "stress a sample of patients Lic to Ferrer for EU. To launch 2H07 as a device.
blood 20 min, reinject → IL10 ↗, cardiovasc antiinflam../
PIPE July 1/03 : $ 37.5m CHF, PAD. Device , patient's blood is treated in VSGN
unit, then reinjected IM. Aug 30/05 Safety Comm; closes
trial in PAD. March 13/06 endpoint missed
- VP 025 , pre, neuroinflammation
VVUS VIVUS (VVUS) - MUSE , local PDE5 inhib, MKT
- Topical Alprostadil, ph III, female sexual arousal (ph II
success p < 0.02)
- Testosterone spray , ph II, N = 261
- Evamist , ph III init Dec 04, low dose estrogen spray,
HRT

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


VXGN VAXGEN (VXGN) -anti HIVpreventive vaccine in phase III:r gp 120 from spinoff from Genentech
CEO resigned Dec 00 HIV 1MN & HIV 1 GNE8. Safe after 23000 injections,
V Short 11% of float efficacy (interim, final nov 02)could be reviewed Nov
01→ inconclusive?? (contains SQ 21). Feb 27/02, NIH
JV w/South Korean partners in Incheon : decides not to test w/Aventis vaccine (because AVE data
Celltrion Inc. To manuf for BMS abatacept bad). Feb 25/03 failure to reduce infection , but effective in
& belatacept black subset. Failed 2003 in Thailand
- Dec 16/02 smallpox vaccine from Kaketsuken (LC-16,
mkt in Japan). IND filing early 03, grown in rabbit kidney
cells)
- Anthrax vaccine (rPA 102), Nov 5/04, $ 878m BioShield
contract for 75 m doses (BioPort to provide 5 m !!). Nov
5/06 probl w FDA. Gov contract canceled Sept 14/07
VYSI VYSIS (VYSI) Genetic diagnostic products : Her2/neu test (ODAC + Dec part w/ABT (April 5/01)
First profitable quarter 5/01) & many mutation dg kits
Being acq by ABT (tender Oct 24/01) "Genomic Disease Management company"
CEO: John Bishop
WL6 K WILEX BIOTECHNOLOGY, German -WX-G 250 Rencarex anti CAIX (carbonic anhydrase IX) exclusive lic.from Centocor
GR Ag, non labelled, ph I (Mayo Clinic), since ASCO
01; RCC ph III ongoing 2007 in non-metastatic April 8/02 CPMP orphan drug designation for both
pts, after total or partial nephrectomy, enrol
420/856 April 3/07. Data 2H08 Nov 14/07 Int Sympo on CA-IX
-*I chimeric WX-G 250, ph II, ⇑renal K, at Ludwig
Institute (New York);orphan drug (June 01)
- WX-UKI, ph I, K, small molecule active on urokinase
activator system
XGEN XENOGEN (XGEN) Luciferase tool-box for biologics & genomics
Initial funding from SR-One, Pictet, etc.. Proprietary in-vivo biphotonic imaging (from glowing
fungi to light producing mice)
XNPT XENOPORT (XNPT) - XPI 3512, Gabapentin prodrug, ph II, diab neuropathy,
restles leggs. Lic Feb 8/07 to GSK (XNPT could
copromote Requip); lic to Astellas for Asia Dec 05. April
25/07 improvement vs placebo, but no data vs Requip –
GSK, patent expires Dec 07) or Mirapex (B.I.)
- XPI 986 generic bacofen (Gaba agonist) for gastric reflux

XNVA K XENOVA (XNVA) -XR 5000(topoisomerase I & II inhib),side-effects in phII June 1: colorectal discont, breast& ovary ongoing,
results for 4Q
merged with Cantab -XR 9576 :MDR inhib, ph II, ph III to start 4Q if partner
found : QLT (AUG 14/01)

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


XOMA M XOMA (XOMA) -The BIG Neuprex failure→→→→→→→ →lic to BAXTER (24 JAN/00). XOMA regains July
… Crohn ph II init July 27/01 8/03
K - Xanelim anti CD 11 MAb→PSO, ph III (25% of US
operating profit, in EU mid single digit royalties). April →w/DNA: 6 months delay annonced Oct 5/01, more
13/03, 90 days after approval will have to repay DNA's delays announced April 8/02 on manuf diff
loan of around $ 60m (offset against royalties). Must also w/DNA
share mkt costs in US. RA trial terminated May 12/03
-to manufacture ONYX 015 : suspended
- w/MLNM : LDP 01
- W/MLNM CAB 2, recomb anti C4 fusion prot for
reperfusion injury
- Aug 17/04 : fails Ph III BPI in acne
- HCD 122, fully human anti CD 40 myeloma ph I
w/CHIR Novartis (see also IDPH, AMEV, SGEN), CLL,
MM
XZL SELECT THERAPEUTICS (XZL) - Cytomatrix : technology for expansion of stem cells -Part. w/ Haemonetics (HAE)
C without cytokines. Ph I/II BMT to start Dana
Farber mid 01 for auto & cord - JV w/ Cytomatrix LLC : Cell Science Therapeutics
- In Researsh ditto : pancreas, liver, brain (CST)
- Activation of Ag presentation to dendritic cells by
linkage to verotoxin
-w/Crucell (June 26/01), PERC6 ,who will
manufacture gene therapy product.
YMI K YM Biosciences (CND) TSX/AIM : YMB, - Tesmilifene (DPPE Partner : CIM (Centro de Immunologia, Cuba)
AMEX YMI diethylphenylmethylphenoxyethanamine, tamoxifene
IPO filed March 02. Trading since June derivative), ph III, histamine antag, chemotherapy
12/02 potentiator for K, prostate ph II w/doxo, breast meta 1st line had something with PHA, but PFE ???
Raised $ 9.7m ph III w/BMY (term. 99) w/doxo <vs doxo alone OS
Monoclonal manufactured in Cuba by CIM signif, but TTP NS ASCO 05 doxo + tesmilifene survival
(cannot be sold in US) 29.7m / 12.2 in unplanned subgroup anal
CSO left Nov 02 Temilifene + Taxotere N = 39 w/Sanofi, data end 07
. To reinitiate (FDA OK March 03) w/epi + cyclo (SPA,
Atlas could merge it ? (but that was special protocol assessment) Breast Meta. Terminated Jan
Novus…CTIC) 30/07
- Theracim hR3, nimotuzumab (anti extracellular epitope,
April 14/06, acq Eximias : like all the others !), EGFR hz MAb, ph II( CND, Cuba),
- Thymitaq, thymidilate synthase inhib H&N. NSCLC (UK, CND) ph II enroled. Glioma ph II
(from Agouron), failed in HCC 35% ORR, partner Oncoscience (Germany) EU ph III
- Orataq, oral formulation underway April 06 pontine + Rx, ph I/II init March
No intention to develop, bought for cash 23/06. Lic to Sankyo for Japan
and US site -*Y Theracin in Italy, ph I/II, glioma
- 151 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies

FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- anti EGFR vaccine, ph II, NSCLC, see CNVX, Returned
to CIM Oct 8/02
- Norelin (GnRH vaccine), ph I/II, prostate K, reduces
testosterone.a
YRK YORK (YRK, London)

ZGEN ZYMOGENETICS (ZGEN) - Discoverer of. Novolin and. Novoseven (which is fully Oct 00 w/Medarex
retained by NN, no royalties) Sept 01 w/Serono, for 2 preclinical candidates.
Established 1981. Was NN until Nov 00, -IL 20 (inflam), pre
then 62%. Now NN has 32.7% (2006 Proxy) - IL 21 (K), ph I/II, melanoma, RCC,NHL, CLL lic to NN;
CATALYSTS for 1H07 : studies planned 07 w/Rituxan, Sutent, Nexavar
- EU partner for rHu Thrombin - Il 28, 29, 31, res
- 2H07 : clear evidence of efficacy of - Atacicept (TACI-Ig) fusion prot linking extracellular part Competition BIIb & DNA have a BAFF-Ig fusion
Atacicept of TACI to Fc of Ig to BLISS & APRIL, lic to protein
. Serono/Merck (2001). CLL ongoing
RA ph Ib TNF refractory enrol complete
RA Ph II init Dec 20/06 rand, biw. Primary : ACR 20 at 26
w.
SLE ph II/III to init mid 07 (direct HGSI competitor). Oct
4/07 FDA SPA OK, N = 200 lupus nephritis, will do 2nd
study in SLE per se
- rHu XIII,pre (but Behring & SASY/IDM), ph I (Feb
4/03), lic from Aventis/Behring
- PDGF lic to JNJ (Regranex) & to BioMimetic (for
periodontal disease, May 29/02) pivotal ongoing competition : plasma derived human thrombin
- rHu Thrombin (manuf by ABT), ph II, surgical Omrix OMRI lic to Ehicon (JNJ), ph III equiv
bleeding (liver resection, spine surgery). Ph III (compared activity to bovine, BLA filed Nov 6/06, approved Aug
to bovine IIa from King, sales $ 240m in 2004) init Oct 28/07
27/05, N = 417, filed Dec 18/ 06 (10 m review : PDUFA Thermogenesis device (trial) to produce thrombin
Oct 18 Aug 28/07 extended to Jan 17); ph III data Sept5/ from patient blood samples.
06 : hemostasis identical, Antibodies 1.5% vs 22%, p <
0.0001. Spray formulation, FDA says (March 7/07) no
need for additional trial.
June 19/07 lic to Bayer ex US, and B. will help US
marketing & sales for 3 years
Nov 2/07 King files "citizen petition against ZGEN !!
- Pegylated IFN lambda (IL 29): to init ph I, 1H07 (more
tolerable than IFN alpha)

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ZIOP K ZIOPHARM (OTC BB : ZIOP) - ZIO-101, ph II, organic As for advanced Myeloma, MDS,
HCC
- ZIO-201, I/II, isophosphoramide mustard lysine :
sarcoma (combined formulation of ifosfamide &
cyclophosphamide)
- ZIO-301, indibulin (binds tubulin), ph I
ZZZZ ACUITY* siRNA

- Cand5, anti VEGF, ph I ARMD, ph II diabetic macular


edema, full enrol May 1/06
- N-chlorotaurine, lic April 06 , anti-infective
- anti Syk kinase, from Zabecor, for ocular inflammation
(May 06)

ZZZZ K ADHEREX ADH-1 limb infusion for N-cadherin positive melanoma


ZZZZ K AEGERA* (CND) AEG 35169 antisense to XIAP (x linked inhibitor of other XIAP : TetraLogic, AVII, DNA, NRGX
apoptosis protein) ph I
AEG 35156, 2nd generation anti XIAP, ph II, AML w/
AraC & Idarubicin; Also ph I/II NSCLC, Pancreac, BC
ZZZZ AEOLUS * AEOL 101150 for ALS ph I
ZZZZ K AFFYMAX * - Hematide : peptide EPO mimetic, ph II Lic to Takeda for Japan (Feb 13/06)
- G-CSF agonist peptide
- TRAIL-R2 against K
ZZZZ K AGENDIA BV marker test for resistance to Herceptin
ZZZZ K ALETHIA (CND) anti – clusterin Mabs, interact with EMt (c-MET)
ZZZZ K AMBIT KINOME SCAN screens activity on over 300 kinases FLT 3 competition MLN 518, relatively specific but
- AC 220, FLT3 inhib, w/ Cephalon. not that potent; PKC 412 Novartis hits a lot of other
kinases
ZZZZ K ANGIOGENICS, Public but not traded developed around Id genes & proteins that halt K
neoangiogenesis : control dev of VEGF receptors
ZZZZ K ANTISENSE PHARMA GmbH - AP 12000 (antisense to TGF beta 2), glioma IIb (ASCO
07 > temozolomide), CRC I/II, Melanoma I/II
ZZZZ ARCHEMIX anti von Willebrand ph I ACS, ph IIa init Nov 13/07; N =
300, compared to ReoPro
ZZZZ ARGENES (Tokyo) ARG 198 anti Fas IgM Mab (Fas = CD 95), RA
ZZZZ c ARTERIOCYTE * - Adult BM stem cells, auto, ischemia : PAD ph I
- Allo BM stem cells, precl
ZZZZ ARTISAN ART-123, recombinant trombomodulin (lic from ASAHI)
for DIC in sepsis
ZZZZ K ASTEX* - AT7519 cell cycle inhib, ph I, w Novartis
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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


- AKT inhib, res w AZN
ZZZZ K AUREON Techniques to identify patients who could benefit from Iressa w/AZN
ZZZZ K AVEO pharmaceuticals - AV 412 (from Mitsubichi), ph I, oral anti RTK EGFR & Her2 competition Tykerb
- AV 951 ph I anti VEGFR
Oct 2/07 partnership w/OSIP - AV 299, pre, anti HGF (hepatocyte growth factor)/c-Met
pathway, lic to SGP April 4/07 $ 477m

ZZZZ K BIPAR* BSI201, ph 1, PARP (poly adenylribose polymerase) inhib.


ZZZZ K BIOTICA - anti HSP 90 : lead optimization
- mTOR inhib, precl, lic to Wyeth $ 195 m DEAL Upfront,
milestones, res support, royalties. The companies willw MTOR competition : Isotechnika, Ariad
ork together on a discovery program. Wyeth will handle
dev & commercialization of selected candidates
ZZZZ K BIOVAX * oncolytic virus, ph II
ZZZZ BIOXELL (Italy) ph II non bacterial prostatitis
funded by Roche, BB Biotech, etc ph II (BXL 628, vitamin D 3 analogue) in BPH
ZZZZ CALANDO* siRNA
key investor : ARWR delivery systems based on cyclodextrins
ZZZZ CELLADON * Mydicar enhances SERCA2a, which regulates myocardial Partnership w/Targeted
contractility. Percut AAV delivery to heart
Kris Zsebo
ZZZZ C CELLERANT* Allo progenitor hematopoietic stem cells that do not need
to be matched
ZZZZ CELLOMICS IPO filed 3/3 (PVS) The neologism after "Proteomics" : virtual cell concept - Carl Zeiss
posponed dec 21,withdrawn Oct 31/01 - Aclara
CFO comes from Oncormed

ZZZZ CEREGENE* gene therapy AAV for Alzheimer (NGF, ph I), ALS, Park
CEO Jeff Ostrove (GDNF & Neurturin)
SAB Fred Gage, Inder Verma - CERE 120, AAV Neurturin (boosts Levodopa
transformation into DA, more for late stage PARK than the
Avigen product which is for late stage), Park, ph II, N = 51
follow up 12 m April 07 : Reduct of Park symptoms in
36% of Pts (p < 0.001). April 07 init comparartive ph II vs
placebo.
June 21/07 : lic ex US to GENZ

ZZZZ COMBIMATRIX going into siRNAs


IPO filed nov 22/00 (SSB), pulled

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS

ZZZZ COPERNICUS* Non viral inhaled gene transfer :


- CF : pre
- IX : pre
ZZZZ CORUS (CSHP) filed Oct 19/04( Corus 1030 asthma : inhaled lidocaine, lic from mayo April 12/06 GILD invests $ 25M
ML, Pacific Growth) PULLED Clinic, ph II
Corus 1020 inhaled Aztreonam for Cystic Fibrosis, ph II
(could be restricted to associations, e;g; Tobi)
ZZZZ C Cryocell Internat.* Umbilical cord stem cells
ZZZZ DICERNA siRNA company (26 to 30 bp). Alnylam & Sirna only
protected to 25

ZZZZ Drug Abuse Science


withdrawn oct 15/01

ZZZZ DNA Sciences (DNAS) Genomic drug & dg discovery part w/Amersham Pharm. Biotech
IPO filed (jan 8/01) by LB
Withdrawn 21/7/01
ZZZZ EDISON pharma Mitochondria : Leber, Friedreich
ZZZZ ELITRA (ELIT)* -Bacterial genomic drug targets, novel ABs & antifungals -oct 12/00, acq Mycota (CND) : candida genomics
Intend to mkt their drugs, in the US at least. - Purchased the PathoSeq business from Incyte -partner : LG Chem (Korea)
IPO filed 9/5 (CHQ),withdrawn (nov 21/00) - partn w/DCGN July 2/02
Looking for genes common to many bacteria, and
Harry Hixson chairman June 26/03 absent from humans - Oct 26/01 : will provide AB targets to MRK
ZZZZ ExSar EXR 101 ph I, Tay Sachs
ZZZZ FAUST pharmaceuticals SA, Stasbourg FP 001 ALS, Ph II, antiglutamatergic
Board Gilly
CEO ex CEO Guilford
Investors Sofinnova, Auriga, etc
ZZZZ K FeRx * MTC (magnetic targeted carriers). Proprietary magnetic
field applicator.
Experienced management - MTC Dox, ph II/III, primary hepatoma, intraarterial (ph Orphan drug, fast track (Aug 27/02), regained rights
II was in China). AACR Nov 02 21 lesions , 1CR, 2PR from ELN Sept 27/02
- MTC + Mitomycin intraarterial refractory NSCLC, init
Jan 04

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ZZZZ FIBROGEN* - FG 2216 oral prolyl hydroxylase inhib, degrades HIF,
activates EPO production, May 1/06, Astellas 765 m deal

ZZZZ K FivePrime Therapeutics - FTP 039, modified growth factor receptor that targets a
protein overexpressed in K, pre
Gail Maderis - FTP 038, diabetes II, pre
- RA, w/BI
ZZZZ FoldRx * Protein mis folding company
- Parkinson : alpha synuclein program to prevent
misfolding
- FAC (familial amyloid cardiomyopathy) mutations
induce heart failure in afro-americans above 60; Fx-1006
stabilizes transerythrin (TTR) to prevent it from misfolding
ZZZZ Gentra Systems* nucleic acid purification technology

Founded in 1988 Specialists of automation. Claim to have the world's first


fully automated system.
sales 2000 : $ 5.2m
67 employees
16 patent applications
expected sales 2005 : $ 135m

ZZZZ HYPERION urea cycle disorders


ZZZZ K ILYPSA* - ILY 101 phosphate binder, lic to Astellas, ph I
- ILY 102, Na binder
- ILY 103, C. difficile toxin binder
- ILY 105, K binder
ZZZZ K INTARCIA files Feb 7/04? pulled - Omega IFN in Alza Duros (3 – 6 months) : Hep C ph II
CSFB, Lazard, Pac. Growth - Atamestane , aromatase inhib , 2 ph III + Toremifene vs
Letrozole (but on TTP, and mainly eastern Europe)
ZZZZ K INTRACEL Oncovax, autologous colon K vaccine, approved in
Holland & Switzerland, ph III

ZZZZ K INTRADIGM siRNA for systemic. In nanoparticles w ligand , and


nucleic acid carrier.
- ICS 283 K pre
ZZZZ KEMIA p38 MAP kinase inhib, allosteric, ph I, RA, Aug 06

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ZZZZ K METASTATIX CXCR4 inhibitors in K and AIDS

ZZZZ MIRUS CORP * non-viral DNA delivery systems (novel cationic polymer). ⇑ TRGNY, for Duchenne
Claim to be able to reach muscle delivery by IV
route (but use tourniquet)
ZZZZ MITOKOR*(MITO) The Mitochondrion company Partners : PFE, WYE, CHIR
Very strong management team.
(Alta, Domain, SR-One) -Res in Alzheimer, Parkinson, osteoarthritis, Diabetes II
IPO filed (RBC) March 12/02, withdrawn (mutations in mitochondrial ATP synthase & tRNA lysine
March 25/03 genes
25 issued patents - MITO 4509, ph I, non feminizing E2, Park
- non feminizing E2, ph III, w/WYE, Alzheimer
- Neurostat (E2), stroke, pre, mitochondrial stabilizer
- MITO 3108, obesity, pre
ZZZZ MOMENTA M118, anticoagulant, ph II, IV
ZZZZ C Nereus* - NPI 2358, pre vascular disrupting agent
- NPI 0052, pre, proteasome inhibitor
- NFKB modulator
ZZZZ C Neurotech* (USA) NT 501, cell therapy for retinitis pigmentosa
ZZZZ NeuTec (AIM, UK) AuroGraf,,recopmbinant anti staph antibody acq by Novartis, June 7/06
ZZZZ K NOVELIS anti MDM 2
ZZZZ OCTAGEN Corp * Second generation F VIII for hemophilia
ZZZZ K Oncolytics Biotech Reolysin , oncolytic reovirus, because K cells with
(ONC/TSE;ONCY/Nasdaq) activated ras pathway are incapable of blocking reoviruses;
prosta ph I init April 02, glioma ph I/II init July 02. June
20/06 intratumoral + RT
ZZZZ OPTHERION recombinant Complement factor H (mutated in 50% AMD
patients) , also CV disease & RA
ZZZZ K PEPLIN Ltd (Australia) PEP 005, topical , basal cell carcinoma gel. 2 applications
clear 71% of tumors
ZZZZ C PharmaStem * (was Biocyte) Umbilical cord stem cells
ZZZZ K Plexxikon - PLX 4032 IND filed Oct 1/06 melanoma DEAL Roche :
upfront 40, 6 m research 2 y, 660 m milestones . Small
molecule anti B-raf V600E
- Servier $ 100 m collab Sept 30/06 to discover non-
peptidic renin inhibitor.

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ZZZZ PHYTON * long acting GH antagonist

ZZZZ PROTALIS Glucocerebrosidase, Gaucher, from plant cell cultures, ph


III
ZZZZ K RECEPTOR BIOLOGIX * Dimercept, pre, Mab to the 4 HER receptors
CEO Michael Shepard, co-discoverer of
Herceptin
ZZZZ REGADO Biosciences RB 006 aptamer anti IX, good anticoagulant

ZZZZ RENAMED Device for acute renal failure ph II 67% survival at day 28
vs 39 conventional dialysis, ph III to init mid 07
ZZZZ Santhera AG SNT – MC17, Idebenone, ph III Friedreich ataxia (20,000
patients US & EU). Also in Duchenne & Leber
ZZZZ Senesco * Inhibitor of eucaryotic init. factor 5 A, for sepsis

ZZZZ K SENETEK Plc siRNA to Tenascin C Glioblastoma

ZZZZ SR Pharma Plc ATU 027 reconstituted lyophilized si RNA

ZZZZ Symphogen A/S r Hu polyclonal antibodies

ZZZZ Targegen * - TG 100115, ph I/II, anti P13 kinase, Myocardial


infarction
- TG 100801, pre, multitarget kinase inhibitor. Would act
on VEGF mechanism for AMD. Non-invasive : eye-drops

ZZZZ Therapeutic Human Polyclonals Hz recombinant rabbit polyclonals. Competition : Symphogen A/S, Kirin (by acq of
Developing humanized Thymoglobulin Humatech in 2005, cow based humanized
acq by ROCHE April 2/07 $ 56 m polyclonals)

ZZZZ THERION, * Pox virus based vaccines

H&Q, Pacific Horizon, Sofinov -Alvac CEA/B7.1, colorectal K gene therapy vaccine hi ™Aventis-Pasteur
-Gene Therap. vaccine for melanoma & NSCLC
Funding difficulties - Panvac-VF (anti CEA & MUC 1 w immunostimulants
B7.1, ICAM 1, LFA3) metastatic pancreas 2nd line init
for sla June 06 3Q04, 2nd line; survival vs/BSC; N = 250, + GM-CSF.
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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


Fails June 28/06. Median survival 2nd line is 3 months,
whatever the drug used
- Prostvac VF, Ph II

ZZZZ K TRACON TRC 102, anti DNA repair , ph I w/Temodar

ZZZZ TRANSXENOGEN (Cambridge, Mass) to produce Insulin in eggs of genetically modified chicken

ZZZZ TROPHOS ( Marseille) TRO 19622, Spinal Muscular Atrophy, ph Ib Oct 07

ZZZZ K VasGene * - Veglin (VEGF antisense, to A, C & D), ph II, no sign of


hypertension. RCC, CRC, NSCLC.
- Mab to Eph B4 TK receptor, and to Ephrin B4 ligand,
pre, w/MEDI
ZZZZ VasoGenix * Calcitonin Gene Related Peptide for cardiology

ZZZZ VASTOX plc small molecule to up-regulate utrophin (similar to


dystrophine), res, Duchenne

ZZZZ XENCOR * Engineers Fc fragment domains of Mabs (Medarex also)

ZZZZ XENON GENETICS* (CND) "Extreme Genetics"

ZZZZ ZELOS * CND PTH analogue

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FIRM (TICKER)/ Specific comments ACTIVITIES PARTNERS/UPCOMING NEWS


ZZZZ ZYSTOR * attach peptide tag to LSD enzymes, targeting receptors on
cell surface, avoiding M6P pathway (PNAS
March 04). Technique lic from Symbiontics
POMPE

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