Professional Documents
Culture Documents
COMPENDIUM
Page 1 : explanatory notes
Page 2 : corporations
BIOTECH COMPANIES
April 2007
US BIOTECH
page I
TMC Development, The "US biotech" companies
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
-2- printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies
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
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
w/Fujisawa
AGNT ARGONAUT TECHNOLOGIES (AGNT) High speed parallel synthesis
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
-7- printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies
-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
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 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*
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
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
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
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
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
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
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
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
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
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
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 .
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
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.
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
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
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
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 (?)
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
GENE THERAPY
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 :
-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
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
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
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
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
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
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.
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
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
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
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
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
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
-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,
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
LEXG LEXICON (LEXG) -Human GeneTrap database The world's largest bank of knock-out
mice:OmniBank
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
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
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.
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
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.
Also in ovary K
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
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)
- 111 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies
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
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
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.
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
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
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
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
RXIP IPO RXi Pharmaceuticals, filed Oct 30/07 siRNA spin off CytRx
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
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
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
Caprion Pharmaceuticals* (CND) Cell organelle proteomics, dg products for CJD (MAbs
from IDXX)
- 140 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies
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
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
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 !!!!!!
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
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
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)
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
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)
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)
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 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
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
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.
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 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)
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.
- 158 - printed : 03/07/10 (d/m/y) 08:07
TMC Development, The "US biotech" companies
ZZZZ TRANSXENOGEN (Cambridge, Mass) to produce Insulin in eggs of genetically modified chicken