Company* |
Product | Description |
Indication | Status |
| | ||||
AUTOIMMUNE | ||||
Enzo Biochem |
Alequel |
A complex of autologous colon-derived antigens administered orally |
Crohn's disease |
Phase II trial in 32 patients showed clinical responses in 67% of the drug group vs. 43% in the placebo group (9/29) |
Genelabs |
Prestara |
Prasterone; synthetic |
Lupus |
Published data from trial GL95-02 in 381 women showing a higher percentage of responders in the drug group vs. placebo (9/9) |
Isotechnika |
ISA247 |
Immunosuppressive agent |
Psoriasis |
Interim data from trial of trans-ISA247 showed no ECG changes or QTc signals seen as compared to placebo (9/9) |
Protein Design |
Nuvion (visilizumab) |
Humanized antibody directed at CD3 antigen on activated T cells |
Ulcerative colitis |
Interim data from the Phase I portion of a Phase I/II trial will be used to pick doses going forward (9/29) |
CANCER | ||||
Ariad |
AP23573 |
Small molecule designed to inhibit cell-signaling protein mTOR |
Bone and soft- tissue sarcomas |
Began Phase II single-agent trial to evaluate clinical benefit and pharmacogenomics in up to 175 patients (9/30) |
Ariad |
AP23573 |
Small molecule designed to inhibit cell-signaling protein mTOR |
Advanced cancers |
Phase I data on 49 patients showed tumor regression in nine patients and disease stabilization in another 15 patients (9/30) |
ArQule Inc. |
ARQ 501 |
Activated Checkpoint Therapy molecule designed to selectively kill cancer cells |
Advanced solid tumors |
Interim Phase I data showed a dose- proportional response and some disease stabilization; a trial is being planned in combination with Taxotere (9/29) |
Bioenvision |
Modrenal (trilostane) |
Drug designed to increase estrogen binding to ER beta and decrease binding to ER alpha |
Prostate cancer |
Began Phase II trial in up to 43 androgen-independent patients who have rising prostate-specific antigen levels (9/23) |
Cyclacel Group plc* (UK) |
CYC682 |
Small-molecule nucleo-side analogue; prodrug of CNDAC |
Solid tumors and lymphoma |
Began third Phase I trial, a Phase Ib in the U.S. to test the safety of escalating doses in patients (9/7) |
Epimmune |
EP-2101 |
Vaccine with nine CTL epitopes from four tumor- associated antigens |
Non-small-cell lung cancer and colorectal cancer |
Reported positive Phase I data in both indications and a move into a Phase II trial to test survival and safety in 84 late- stage NSCLC patients (9/28) |
Favrille Inc.* |
FavId |
Vaccine designed to stimulate immune responses |
Follicular B-cell non- Hodgkin's lymphoma |
Began Phase III trial under FDA SPA that will assess time to disease progression (9/22) |
Genentech |
Tarceva |
Erlotinib HCl; small-molecule HER1/EGFR inhibitor |
Pancreatic cancer |
Phase III trial in 569 patients showed a statistically significant 23.5% survival benefit for drug plus gemcitabine vs. gemcitabine alone (9/20) |
Genentech |
Tarceva |
Erlotinib HCl; small-molecule HER1/EGFR inhibitor |
Non-small-cell lung cancer |
Began Phase IIIb ACT trial to evaluate survival and responses in patients with second- and third-line disease who previously have received chemotherapy (9/10) |
Genmab A/S |
HuMax-CD4 |
Human antibody that targets the CD4 receptor on T lymphocytes |
Cutaneous T-cell lymphoma |
Phase II data on 38 patients showed an average response duration of more than 6.6 months, based on an analysis of responding mycosis fungoides (9/1) |
GenVec |
TNFerade |
Adenovector with TNF-alpha gene and a radiation- responsive promoter |
Soft-tissue sarcoma |
Published Phase I data showed 11 of 13 patients demonstrated objective tumor responses; drug with radiation was well tolerated with no dose-limiting toxicity (9/10) |
GPC Biotech |
Satraplatin |
Oral platinum compound |
Non-small-cell lung cancer |
Began Phase I/II trial in up to 30 patients in combination with radiation therapy (9/1) |
Human Genome |
HGS-ETR1 |
Agonistic human monoclonal antibody to TRAIL Receptor 1 |
Advanced solid tumors/non-Hodgkin's lymphoma |
Phase I trial demonstrated safety and tolerability in both indications, and supported a move into Phase II trials (9/29) |
Human |
HGS-ETR2 |
Agonistic human monoclonal antibody to TRAIL Receptor 2 |
Advanced solid tumors |
Initial results of Phase I trial demonstrated safety and tolerability, and supported continued dose escalation (9/29) |
Immuno- |
Uvidem |
Vaccine consisting of mature dendritic cells loaded with lysates of tumor cell lines |
Melanoma |
Began Phase II trial in 37 patients with Stage III or IV disease that is progressing (9/21) |
Insmed Inc. |
rhIGFBP-3 |
Recombinant human insulin-like growth factor binding protein-3 |
Cancers |
Began Phase I trial to evaluate safety, tolerability and pharmaco-kinetics of an intravenous dose in 30 patients (9/8) |
Keryx Bio- |
KRX-0401 (perifosine) |
Oral AKT inhibitor |
Advanced soft-tissue sarcoma |
Single-agent Phase II trial in 23 patients showed one partial response, and two patients remained progression free at six months (9/30) |
Lorus |
GTI-2040 |
Antisense oligonucleotide complementary to the R2 component of ribonucleotide reductase |
Metastatic renal-cell carcinoma |
Phase II trial in combination with capecitabine showed disease stabilization in more than half the patients taking recommended Phase II dose (9/30) |
Maxim |
Ceplene |
Histamine dihydrochloride in combination with interleukin-2 |
Advanced malignant melanoma |
Phase III trial in 230 patients with liver metastases failed to show an improvement in survival vs. IL-2 alone (9/20) |
Medarex Inc. |
MDX-010 and MDX-1379 |
Fully human anti-CTLA-4 antibody; gp100 melanoma peptide vaccine |
Metastatic melanoma |
Began Phase III trial to test the products alone and in combination in 750 patients under an FDA SPA (9/28) |
MediGene AG |
NV1020 |
Oncolytic herpes simplex virus vector |
Liver metastases from colorectal cancer |
Began Phase I/II trial in about 30 patients to test safety, tolerability and efficacy, as well as synergies with chemotherapy (9/1) |
Novacea Inc.* |
AQ4N |
Hypoxic cell-activated antitumor therapy |
Advanced malignancies |
Began Phase I trial to determine the safety and tolerance of multiple weekly intravenous doses (9/23) |
Onxy |
BAY 43-9006 |
RAF kinase and VEGF inhibitor |
Advanced liver cancer |
Phase II single-agent trial in 137 patients showed 43% experienced stable disease for at least four months and another 9% experienced tumor shrinkage (9/29) |
Peregrine |
Tarvacin |
Chimeric monoclonal antibody that binds to a phospholipid, phosphatidylserine |
Advanced solid tumors |
Filed IND for Phase I trial to test safety, tolerability, dosing and pharmacokinetics in 28 patients (9/22) |
Pharmacyclics |
Xcytrin |
Motexafin gadolinium; small molecule from the texaphyrin class designed to induce apoptosis |
Non-Hodgkin's B-cell lymphomas |
Presented data from Phase I trial showing all three refractory patients treated with drug, Rituxan and Zevalin achieved complete remission at one month (9/20) |
PowderMed |
-- |
Vaccine that uses DNA encoding for the NY-ESO-1 tumor-specific antigen with PowderMed's delivery technology |
Non-small-cell lung cancer |
Company and the Ludwig Institute for Cancer Research began a Phase I trial in up to 18 patients (9/17) |
Protein Design |
M200 |
Anti-alpha5beta1 integrin chimeric monoclonal antibody |
Solid tumors |
Phase I trial in refractory disease showed 10 of 15 evaluable patients had stable disease (9/29) |
Rexahn Corp.* |
RX-0201 |
Signal inhibitor that blocks the protein kinase Akt |
Advanced cancers |
Began Phase I trial to evaluate safety and pharmacokinetics, as well as responses (summer 2004) |
Salmedix Inc.* |
SDX-102 |
L-alanosine |
Brain tumors |
A National Cancer Institute consortium began a Phase I/II trial in patients with previously treated tumors (9/30) |
Seattle |
SGN-30 |
Monoclonal antibody that targets CD30+ hematologic malignancies |
Primary cutaneous anaplastic large-cell lymphoma |
Began Phase II trial designed to evaluate the antitumor activity and tolerability in up to 40 relapsed or resistant patients (9/23) |
Threshold Pharmaceuticals |
Glufosfamide |
Small molecule targeting abnormal glucose metabolism |
Metastatic pancreatic cancer |
Received FDA SPA for and began Phase III trial to test drug in 306 refractory patients in combination with best supportive care (9/13) |
Vion |
Cloretazine (VNP40101M) |
Sulfonyl hydrazine DNA alkylating agent |
Acute myeloid leukemia |
Plans a pivotal Phase III trial comparing drug and Ara-C to Ara-C alone in patients with AML in first relapse (9/23) |
Wilex AG* |
WX-671 |
Oral, second-generation serine protease inhibitor targeting the uPA system |
Solid tumors |
Began Phase I trial to evaluate bioavailability, pharmacokinetics and safety in 12 healthy male volunteers (9/20) |
CARDIOVASCULAR | ||||
AtheroGenics |
AGI-1067 |
Oral agent designed to inhibit the production of VCAM-1 and other molecules in the inflammatory process |
Atherosclerosis |
Phase IIb CART-2 trial showed the drug reduced plaque volume by a statistically significant 3.8% vs. standard care (9/27) |
Avant Immuno- |
TP-10 |
Soluble complement inhibitor |
Cardiac surgery |
Published data from 564-patient Phase II trial showed a significant decrease in mortality and acute myocardial infarction in male patients; however, lack of benefit in female patients led to failure to meet primary endpoint (9/20) |
Cardiome |
RSD1235 |
Oral formulation of drug designed to selectively block ion channels in the heart |
Atrial fibrillation |
Began Phase I trial to test two controlled- release formulations compared to an immediate-release formulation (9/24) |
Cardiome |
Oxypurinol |
Xanthine oxidase inhibitor that is the active metabolite of allopurinol |
Congestive heart failure |
Two physician-sponsored trials showed a statistically significant improvement in the left ventricle ejection fraction (9/13) |
Cel-Sci Corp. |
Multikine |
Mixture of human cytokines designed to stimulate an immune response |
Elevated cholesterol |
Meta-analysis of studies showed reduced cholesterol levels in 120 patients with head and neck cancer, with no liver toxicity (9/9) |
Corautus |
VEGF-2 |
Vascular endothelial growth factor-2; designed to promote angiogenesis |
Severe |
Began Phase IIb GENASIS trial to test safety and efficacy in up to 404 patients with Class III or IV angina (9/8) |
ImaRx |
SonoLysis |
Therapy that uses ultrasound to cavitate gas-filled microbubbles to dissolve blood clots |
Deep-vein thrombosis |
Began Phase I/II trial to test safety and potential efficacy (9/28) |
KAI |
KAI-9803 |
Protein kinase C inhibitor; targets the delta-PKC isozyme |
Acute myocardial infarction |
Began Phase I/II trial to evaluate safety and efficacy in 150 patients undergoing urgent angioplasty (9/28) |
NicOx SA |
HCT 3012 (formerly AZD3582) |
Nitric oxide-releasing compound from company's CINOD class |
Hypertension |
Began trial in 50 patients to assess blood pressure levels for drug group vs. those taking rofecoxib (9/8) |
Nuvelo Inc. |
Alfimeprase |
Modified fibrolase delivered via catheter; thrombolytic agent |
Peripheral arterial occlusion |
Phase II NAPA-1 trial in 113 patients showed thrombolysis rates of up to 76% and restoration of arterial flow of up to 60% (9/30) |
Scios Inc. |
Natrecor (FDA-approved) |
Nesiritide; recombinant B-type natriuretic peptide |
Congestive heart failure |
Published FUSION I trial results of 210 patients with advanced decompensated disease, showing benefits on mortality and rehospitalization (9/1); observational study showed significantly reduced in-hospital mortality and length of stay compared to inotropic agents in acutely decompensated heart failure patients (9/13) |
Synthetic Blood |
Oxycyte |
Perfluorocarbon-based blood substitute and therapeutic oxygen carrier |
Blood substitute |
Plans to begin a Phase II trial in orthopedic surgery patients; the study will focus on preventing tissue hypoxia (9/7) |
Valentis Inc. |
Deltavasc |
5% solution of PINC polymer mixed with the Del-1 gene |
Peripheral arterial disease |
Phase II trial in 100 patients failed to demonstrate statistical significance in improving exercise tolerance after 90 days; the PINC polymer-only control was more active than expected (9/29) |
Vasogen Inc. |
Celacade |
Immune-modulation therapy |
Chronic heart failure |
Published results of trial in 73 patients showed a significant reduction in the risk of death and all-cause hospitalization for drug vs. placebo when added to standard care (9/15) |
CENTRAL NERVOUS SYSTEM | ||||
Aeolus |
AEOL 10150 |
Catalytic antioxidant designed to destroy oxygen-derived free radicals |
Amyotrophic lateral sclerosis |
Began Phase I trial in ALS patients to evaluate the safety, tolerability and pharmacokinetics (9/7) |
Ceregene Inc.* |
CERE-110 |
In vivo delivery of nerve growth factor genes through an adeno- associated viral vector system |
Neuro-degenerative diseases |
Began Phase I trial that will evaluate safety and efficacy (9/21) |
DOV |
Bicifadine |
Non-narcotic analgesic |
Lower back pain |
Began pivotal Phase III trial under an FDA SPA that will study the efficacy and tolerability of three doses in 600 patients (9/28) |
DOV |
Bicifadine |
Non-narcotic analgesic |
Pain |
Began first of four pivotal trials of the drug; the trial will test safety and efficacy in 480 patients with acute pain following bunionectomy surgery (9/24) |
Guilford |
Aquavan injection |
Water-soluble prodrug of propofol |
Sedation |
Began Phase III trial comparing Aquavan to midazolam HCl in up to 267 patients undergoing colonoscopy procedures (9/20) |
Labopharm Inc. |
-- |
Once-daily formulation of the analgesic tramadol |
Pain |
Plans another Phase III trial to support its NDA; the trial, MDT3- 005, is expected to begin in 4Q:04 (9/22) |
Metaphore |
M40403 |
Small molecule that mimics the activity of superoxide dismutase |
Pain in cancer patients |
Began Phase II trial in 24 patients to evaluate safety and analgesic activity of the drug combined with opioids (9/29) |
Neurocrine |
Indiplon |
Modified-release form of a non- enzodiazapine agent that targets GABA-A receptor |
Insomnia |
Phase III trial in 229 elderly patients with chronic insomnia showed a statistically significant improvement on the primary end-point of total sleep time, as well as secondary endpoints; NDA filing is planned for October (9/9) |
NovaDel |
-- |
Lingual spray version of alprazolam, a drug sold by Pfizer Inc. as Xanax |
Anxiety |
Began a pilot pharmacokinetic feasibility study in humans (9/23) |
Orphan |
Xyrem (FDA-approved) |
Sodium oxybate oral solution |
Excessive daytime sleepiness |
Phase IIIb trial in narcoleptic patients comparing drug to modafinil (Provigil) showed both drug arms and combination drug arm were statistically significant vs. placebo (9/14) |
Predix |
PRX-00023 |
Serotonin 1A (5HT1A) receptor agonist |
Neuropsychiatric disorders |
Phase I trial in healthy volunteers showed induction of surrogate biomarkers and was well tolerated (9/14) |
Repligen Corp. |
RG1068 |
Synthetic human secretin |
Obsessive- compulsive disorder |
Began a Phase I trial in up to 16 patients to evaluate safety, tolerability and potential impact on the symptoms of OCD (9/24) |
INFECTION | ||||
Advancis |
Amoxicillin Pulsys |
Amoxicillin formulation delivered with once-daily pulsatile Pulsys technology |
Pharyngitis from Group A streptococcal infections |
Began Phase III non-inferiority trial comparing drug to penicillin in eradicating bacteria (9/23) |
aRigen Inc.* |
WAP 8294A2 |
Depsipeptide antibiotic |
S. aureus infections |
Began Phase I trial to assess the safety, tolerability and pharmacokinetics of an intravenous infusion (9/21) |
Hemispherx |
Alferon N |
Purified mixture of natural alpha interferons |
West Nile virus |
Patient treatment began in a trial of the product (9/7) |
Medarex Inc. |
CDA-1 (MDX-066) |
Fully human antibody believed to target and neutralize the effects of Toxin A |
Clostridium difficile- associated diarrhea |
Began Phase I trial to test safety and drug concentrations in up to 30 healthy volunteers (9/29) |
StressGen |
HspE7 |
Fusion product consisting of heat- shock protein fused to HPV antigen |
Internal genital warts |
Broadened program to include Phase II trial to evaluate responses in 30 patients (9/9) |
The Immune |
Remune |
Immune-based therapy comprised of a whole-killed virus and an adjuvant |
HIV |
Published data from 35 patients showed increased CD4+ and CD8+ T-cell lymphocyte activity (9/28) |
VaxGen Inc. |
LC16m8 |
Attenuated smallpox vaccine candidate |
Smallpox |
Plans to begin Phase I/II trial comparing product to Dryvax, and test safety and immunogenicity in 150 volunteers (9/7) |
Vical Inc. |
--
|
Immunotherapeutic vaccine incorporating genes encoding three CMV proteins |
Cytomegalo-virus |
Began Phase I trial to evaluate safety and immunogenicity in up to 40 healthy subjects (9/29) |
MISCELLANEOUS | ||||
Adolor Corp. |
Entereg |
Peripherally acting mu opioid receptor antagonist |
Postoperative ileus |
Published data from Phase III trial (14CL313) showed accelerated gastrointestinal recovery in those undergoing laparotomy for bowel resection or radical hysterectomy (9/23) |
BioSante |
LibiGel |
Transdermal testosterone gel |
Female sexual dysfunction |
Phase II trial in 46 surgically menopausal women showed a statistically significant increase in satisfying sexual events vs. baseline and placebo (9/22) |
Cellegy |
Tostrelle |
Testosterone gel |
Female sexual dysfunction |
Second interim analysis in Phase II trial in postmenopausal women showed testosterone production and an increase in satisfying sexual events; enrollment stopped in preparation of Phase III trials (9/8) |
ConjuChem |
DAC:GRF |
Chemically modified form of growth hormone-releasing factor |
Growth hormone deficiency |
Began Phase I trial in up to 30 healthy volunteers to assess safety and tolerability, as well as pharmacokinetics (9/21) |
Depomed |
Furosemide GR |
Controlled-release formulation of the diuretic furosemide |
Edema |
Phase II trial in 30 patients with congestive heart failure showed comparable activity to an immediate-release formulation (9/23) |
DOR BioPharma |
orBec |
Oral formulation of beclomethasone dipropionate; a corticosteroid |
Graft-vs.-host disease |
Said it completed a pivotal Phase III trial with 129 patients; data were not released (9/15) |
Genaera Corp. |
Squalamine |
Anti-angiogenesis agent; inhibits various growth factor signaling |
Wet age- related macular degeneration |
Began Phase II trial to evaluate safety and efficacy in combination with initial Visudyne treatment in 45 patients (9/28) |
Genzyme |
Fabrazyme (FDA-approved) |
Agalsidase beta; enzyme-replacement therapy |
Fabry's disease |
Phase IV trial in 82 patients showed a substantially lower rate of events that mark the advance of the disease (9/9) |
InKine |
INKP-102 |
New-generation sodium phosphate purgative tablet |
Bowel cleansing prior to colonoscopy |
Began Phase III trial under FDA SPA comparing drug to company's marketed product, Visicol (9/9) |
Jerini AG* |
Icatibant |
Bradykinin B2 receptor antagonist |
Hereditary angioedema |
Began pivotal FAST 1 trial in the U.S. and Canada to test the subcutaneous formulation (9/8) |
LAB |
-- |
29-amino-acid peptide analogue of growth hormone-releasing hormone |
Growth hormone deficiencies |
Phase I/II trial in 10 healthy volunteers showed increased levels of GH, as well as demonstrating safety (9/30) |
Nabi Bio-pharmaceuticals |
NicVAX |
Nicotine conjugate vaccine |
Nicotine |
Phase II trial in 68 smokers showed a 33% quit rate in the highest-dose drug group vs. 9% in the placebo group (9/28) |
Nymox |
NX-1207 |
Investigational drug for BPH |
Benign prostatic hyperplasia |
Phase II trial in 20 nonresponding men showed statistically significant symptomatic improvement at both 30 days and after a one-year follow up (9/21) |
RegeneRx Bio- |
Thymosin beta-4 |
Naturally occurring peptide with multiple mechanisms of action |
Chronic pressure ulcers |
Filed IND amendment for a Phase II trial that will test several topically applied doses (9/22) |
Sirna |
Sirna-027 |
Chemically modified short interfering RNA targeting vascular endothelial growth factor receptor-1 |
Wet age-related macular degeneration |
Filed IND for Phase I trial to assess safety, tolerability and biological activity following a single intra-vitreal injection (9/7) |
Valera |
-- |
Implant using Hydron delivery platform to provide 12-month continuous administration of histrelin |
Central precocious puberty |
Began pivotal Phase III trial to test the company's delivery of the synthetic GnRH agonist (9/8) |
Vivus |
Alista |
Topical formulation of alprostadil |
Female sexual arousal disorder |
Began Phase III trial at 40 sites to test the product in women aged 21 to 60 who have undergone a hysterectomy (9/7) |
Zonagen Inc. |
Androxal |
Oral antagonist designed to induce production of endogenous testosterone |
Hypogonadism |
Presented additional data from positive Phase I/II trial, showing a normal daily rhythm of circulating testosterone (9/2) |
Notes: | ||||
* Privately held | ||||
BLA = biologics license application; FDA = Food and Drug Administration; IND = investigational new drug application; | ||||
NDA = new drug application; SPA = special protocol assessment. | ||||
Unless otherwise noted, the stock symbols listed for public companies are on the Nasdaq market. | ||||
AMEX = American Stock Exchange; CSE = Copenhagen Stock Exchange; FSE = Frankfurt Stock Exchange; NYSE = New York Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; TSE = Toronto Stock Exchange. | ||||
