Company* |
Product | Description | Indication |
Status |
| AUTOIMMUNE | ||||
Alba |
AT1001 |
Oral zonulin receptor antagonist |
Celiac disease |
Successfully completed Phase I trials and plans to move the agent forward (4/3) |
Array |
ARRY-438162 |
Small-molecule inhibitor of the protein kinase MEK |
Inflammatory |
Began Phase I trial to evaluate the drug in healthy volunteers (4/27) |
Biogen Idec |
Tysabri |
Natalizumab; humanized monoclonal antibody that inhibits adhesion molecules on immune cells |
Multiple sclerosis |
Phase III data showed significant effects on health-related quality- of- life measures, as well as on disability and function (4/7) |
Immunomedics |
Epratuzumab |
Humanized anti-CD22 antibody labeled with yttrium-90 |
Lupus |
Phase II trial demonstrated improvements in disease activity in 13 of 14 patients (4/24) |
Manhattan |
PTH (1-34) |
Topical agent based on protein that regulates the growth of skin cells |
Psoriasis |
Start of Phase IIa trial was delayed due to formulation and other issues (4/28) |
MedImmune |
MEDI-545 |
Fully human monoclonal antibody targeting interferon-alpha |
Lupus |
MedImmune began a Phase I trial to evaluate safety and tolerability in 45 patients (4/13) |
The Immune |
NeuroVax |
T-cell receptor peptidec vaccine |
Multiple sclerosis |
Open-label trial in MS patients showed restoration of FOXP3+ T cells (4/6) |
CANCER | ||||
Adherex |
Eniluracil |
Oral dihydropyrimidine dehydrogenase inhibitor |
Colorectal cancer |
Began proof-of-mechanism trial, with 5-FU, in up to 20 patients (4/26) |
Alexza |
AZ-003 |
Fentanyl delivered with inhaled Staccato system |
Acute pain |
Began Phase I trial to evaluate safety, tolerability, pharmacokinetics and to compare product to I.V. fentanyl (4/26) |
Alexza |
AZ-002 |
Alprazolam delivered with inhaled Staccato system |
Panic disorder |
Began Phase IIa trial to evaluate safety and efficacy in 36 patients (4/20) |
Alfacell Corp. |
Onconase |
Ranpirnase; cytotoxic ribonuclease |
Malignant mesothelioma |
Interim analysis from Phase IIIb trial showed a trend toward a survival advantage for drug plus doxorubicin arm vs. doxorubicin alone (4/27) |
Allos |
Efaproxyn |
Efaproxiral; small molecule designed to sensitize hypoxic areas of tumors during radiation therapy |
Brain metastases from breast cancer |
Monitors recommended continuation of Phase III ENRICH trial following an interim analysis (4/4) |
Amgen Inc. |
Panitumumab |
Fully human monoclonal antibody directed against the epidermal growth factor receptor |
Metastatic colorectal cancer |
Pivotal Phase III trial demonstrated improved progression-free survival and disease control vs.best supportive care (4/4) |
AmpliMed |
Benzimate (FB642) |
Agent believed to inhibit the growth of certain cancer cells |
Refractory cancers |
Began Phase I trial to evaluate safety and tolerability in 50 patients with advanced cancers (4/27) |
A.P. Pharma |
APF530 |
The 5HT(3) antagonist anti-nausea drug granisetron formulated with company's delivery system |
Chemotherapy- induced nausea and vomiting |
Is starting a pivotal Phase III trial to evaluate efficacy in 1,350 patients receiving chemotherapy (4/27) |
ArQule Inc. |
ARQ 501 |
Activated checkpoint therapy molecule designed to selectively kill cancer cells |
Advanced solid tumors |
Phase I trial in 64 patients demonstrated evidence of tolerability and clinical activity (4/4) |
Astex |
AT9283 |
Aurora A and B kinase inhibitor |
Cancers |
Is starting a Phase I trial to evaluate the product in hematological cancers (4/25) |
Biomira Inc. |
Stimuvax |
Liposomal vaccine encapsulating a sequence of theMUC1 cancer mucin |
Stage IIIB and IV non-small-cell lung cancer |
Phase IIb trial in 171 patients showed median survival of 30.6 months, vs. 13.3 months in the control arm (4/28) |
Chiron Corp. |
CHIR-265 |
Selective Raf kinase |
Melanoma |
Began Phase I trial to evaluate safety, tolerability, pharmacokinetics and activity in up to 60 patients (4/11) |
Cytokinetics |
SB-743921 |
Small-molecule kinesin spindle protein inhibitor |
Non-Hodgkin's lymphoma |
Began Phase I/II trial to evaluate safety, tolerability and pharmaco-kinetics; the Phase II portion is expected to include 70 patients with aggressive or indolent disease (4/17) |
Endocyte Inc.* |
EC145 |
Folate-targeted cytotoxic anticancer agent |
Cancers |
Began Phase I trial to evaluate safety, tolerability and dosing (4/20) |
EntreMed |
Panzem |
2-methoxyestradiol; naturally occurring metabolite of endogenous estrogen |
Metastatic breast cancer |
Began Phase Ib trial with paclitaxel to evaluate safety, tolerability and biological effects (4/25) |
Geron Corp. |
GRN163L |
Telomerase inhibitor |
Advanced |
Began a Phase I trial to evaluate safety, tolerability, pharmacokinetics and activity in patients with advanced cancers (4/3) |
Gloucester |
Depsipeptide (FK228) |
Histone deacetylase inhibitor |
Cutaneous T-cell lymphoma |
Monitors reviewing interim safety data from pivotal trial recommended continuation of the study without modification (4/26) |
GTx Inc. |
Acapodene |
Toremifene citrate; small- molecule non-steroidal selective estrogen receptor modulator |
Prostate cancer |
Began separate Phase IIIb trial as extension to pivotal study, open to those who have completed two years of treatment (4/21) |
Halozyme |
Chemophase |
High-dose formulation of the recombinant human hyaluronidase enzyme rHuPH20 |
Superficial bladder cancer |
Data from five patients treated with agent and mitomycin demonstrated safety (4/4); began Phase I/IIa trial with mitomycin in up to 36 patients (4/26) |
Intracel Corp.* |
OncoVAX |
Patient-specific tumor cell vaccine; immunotherapy |
Stage II colon cancer |
Reached agreement with FDA on SPA for pivotal Phase III trial to evaluate disease-free survival and other endpoints in 560 patients (4/27) |
Millennium |
Velcade(FDA-approved) |
Bortezomib; proteasome inhibitor |
Advanced follicular lymphoma |
Began Phase III trial to evaluate the drug with Rituxan in 670 patients with follicular NHL (4/18) |
NeoPharm |
IL13-PE38QQR |
Cintredekin besudotox; agent designed to deliver bacterial cytotoxic PE38 |
Malignant glioma |
Safety and tolerability weredemonstrated in a Phase I trial in 19 newly diagnosed patients (4/25) |
NeoPharm |
IL13-PE38QQR |
Cintredekin besudotox; agent designed to deliver bacterial cytotoxic PE38 |
Recurrent glioblastoma multiforme |
Encouraging survival data were presented in updated Phase I/II trials, and in asubset analysis |
Novacea Inc.* |
DN-101 |
Agent containing calcitriol, the biologically active form of vitamin D |
Advanced prostate cancer |
Began pivotal Phase III ASCENT-2 trial that will evaluate the drug with Taxotere in 900 patients (4/12) |
Oncolytics |
Reolysin |
Intratumorally delivered formulation of the human reovirus |
Advanced solid tumors |
Early data from Phase I trial in combination with radiation showed local clinical responses (4/4) |
Pro- |
Davanat |
Carbohydrate compound designed for targeted delivery |
Advanced colorectal cancer |
Preliminary data from Phase II trial with 5-FU showed a partial tumor response; company plans to move to Phase III trials (4/5) |
Provectus |
PV-10 (Provecta) |
Agent designed to be retained in tumor cells while leaving normal estrogen tissue unharmed |
Recurrent breast cancer |
Phase I trial in five patients deonstrated tolerability some evidence of efficacy was seen (4/27) |
Reata |
RTA 402 (CDDO-Me) |
Targeted therapy that modulates oxidative stress response pathways |
Cancers |
Began Phase I trial to evaluate safety, dosing and other end-points in patients with various cancers (4/7) |
Spectrum |
Satraplatin |
Oral platinum compound |
Hormone- |
Monitors in Phase III SPARC trial recommended continuation of the study, after an interim review of safety and efficacy data (4/25) |
Spectrum |
EOquin |
Synthetic prodrug activated by enzymes prevalent in hypoxic cancer cells |
Superficial bladder cancer |
Phase II marker lesion trial showed complete tumor disappearance in 30 of 46 patients (4/7) |
Sunesis |
SNS-595 |
Small-molecule cell-cycle modulator designed to induce apoptosis |
Advanced solid tumors |
Six of 21 patients in Phase I trial experienced sustained disease control lasting at least 16 weeks (4/4) |
SuperGen |
Orathecin |
Rubitecan; topoisomerase- I inhibitor in capsule formulation |
Advanced pancreatic cancer |
Phase II trial with gemcitabine as a first-line therapy in 39 patients failed to meet survival threshold for moving to Phase III (4/26) |
Vertex |
VX-680 |
Small-molecule inhibitor of Aurora kinases |
Advanced colorectal cancer |
Began Phase II trial to evaluate the agent in 20 patients (4/5) |
CARDIOVASCULAR | ||||
Actelion Ltd. |
Tracleer (FDA-approved) |
Oral dual endothelin receptor antagonist; bosentan |
Pulmonary atertial hypertension |
Began trials in patients with PAH secondary to sickle cell disease to evaluate various efficacy end-points (4/10) |
Affymax Inc.*\ |
Hematide |
Synthetic, peptide-based |
Anemia |
Began open-label trial to evaluate safety and efficacy in chronic kidey disease patients with pure red cell aplasia (4/11) |
Cangene |
WinRho SDF (FDA-approved) |
Antibody to certain types of red blood cells |
Thrombo- cytopenia |
Began trial to evaluate drug in treating thrombocytopenia in patients with hepatitis C (4/19) |
Corautus |
VEGF-2 |
Vascular endothelial growth factor-2 in the form of naked plasmid DNA |
Severe angina |
Enrollment was stopped in Phase IIb GENASIS trial after monitors saw little chance for efficacy relative to the safety risk (4/10) |
Inspire |
INS50589 Antiplatelet |
Inhibitor of the platelet P2Y12 adenosine diphosphate receptor |
For use in cardiac surgery |
Began Phase II trial to evaluate the agent in 160 patients under-going coronary artery bypass graft surgery using a cardiopulmonary bypass pump (4/26) |
Isis Pharmaceuticals |
ISIS 301012 |
Second-generation antisensene inhibitor of apoB-100 |
Cholesterol management |
Phase II trial showed reductions in apoB-100, LDL, VLDL, total cho- lesterol and triglyceride levels (4/27) |
Medicure Inc. |
MC-1 |
Naturally occurring small molecule that protects cardiomyocytes |
Heart damage |
Phase II trial in 902 patients who underwent CABG surgery met its primaryendpoint of reducing a composite of events after 30 days (4/6) |
Myogen Inc. |
Ambrisentan |
Oral endothelin receptor antagonist |
Pulmonary arterial hypertension |
Pivotal Phase III ARIES-1 trial met its primary efficacy endpoint of improved exercise capacity vs. placebo; an NDA filing is planned (4/10) |
Nuvelo Inc. |
Alfimeprase |
Recombinant enzyme that acts by directly degrading fibrin |
Acute peripheral arterial occlusion |
Began Phase III NAPA-3 trial under an FDA SPA that will evaluate the avoidance of open vascular surgery and other endpoints against placebo (4/10) |
Predix |
PRX-08066 |
Small-molecule 5-HT2B antagonist |
Hypoxia-Induced pulmonary hypertension |
Phase Ib trial in 15 conditioned adults showed a statistically significant reduction in systolic pul- monary blood pressure during exercise hypoxia (4/19) |
|
Stem Cell Therapeutics Corp. (Canada;TSE:SSS) |
NTx-265 |
Technology designed to increase the growth of innate adult stem cells |
Stroke |
Is starting a Phase IIa trial in 12 patients to evaluate safety (4/25) |
The Medicines |
Cangrelor |
Intravenous platelet inhibitor that binds directly to the P2Y12 receptor |
Anticoagulation |
Began pivotal Phase III program that will test the agent for pre- venting ischemic events in two trials that together will total 13,000 patients undergoing PCI |
CENTRAL NERVOUS SYSTEM | ||||
Acadia |
ACP-103 |
Selective 5-HT2A receptor inverse agonist |
Sleep maintenance |
Proof-of-concept study in 45 healthy volunteers demonstrated a statistically significant increase in slow wave sleep (4/19) |
Axonyx |
Phenserine |
Selective inhibitor of acetylcholinesterase |
Alzheimer's disease |
Trial data showed an increase in brain glucose metabolism and a reduction of amyloid levels in the memory and cognition areas in brains(4/20) |
DOV |
Bicifadine |
Serotonin and norepinephrine reuptake inhibitor |
Chronic low back pain |
Phase III trial in 600 patients failed to demonstrate statistically significant pain relief vs. placebo (4/24) |
Labopharm |
-- |
Once-daily formulation of the analgesic tramadol |
Pain |
Phase III trial in patients with osteoarthritis of the knee achieved statistical significance vs. placebo for the primary endpoint of pain intensity (4/12) |
Sangamo |
SB-509 |
Plasmid DNA that |
Diabetic neuropathy |
Phase I trial in 12 patients met all safety endpoints; improvements in pain and symptoms also were seen (4/6) |
Somaxon |
Silenor |
Low dose of the approved depression drug doxepin |
Insomnia |
Phase III trial demonstrated statistically significant improvements in sleep maintenance vs. placebo (4/10) |
DIABETES | ||||
Alkermes Inc. |
AIR Inhaled Insulin System |
Insulin system based on Alkermes' pulmonary drug delivery technology |
Type II diabetes |
Began Phase III trial to evaluate effectiveness in improving glucose control vs. injected pre-meal insulin in 400 patients (4/26) |
ConjuChem |
PC-DAC: Exendin-4 |
Modified exendin-4 analogue bonded to recombinant human albumin |
Type II diabetes |
Phase I/II trial demonstrated tolerability and glucose reduction supporting once-a- eek dosing (4/26) |
Generex |
Orallyn |
Oral insulin buccal spray formulation |
Type I diabetes |
Trial in young people showed the same improved metabolic control as standard therapy (4/10) |
MannKind |
Technosphere Insulin |
Dry-powder formulation of insulin delivered by company's inhaler |
Type I diabetes |
Phase II trial in 110 patients show- ed similar safety and efficacy at controlling blood sugar vs. insulinaspart (NovoLog) (4/19) |
INFECTION | ||||
Alnylam |
ALN-RSV01 |
Small interfering RNA agent designed to silence the RSV nucleocapsid "N" gene |
Respiratory syncytial virus |
Two Phase I trials totaling 101 healthy volunteers showed the agent was safe and well tolerated when administered intranasally (4/30) |
GeoVax Inc.* |
-- |
HIV DNA vaccine along with recombinant MVA vaccine |
HIV |
Began clinical trials in humans to evaluate safety and immune responses (4/14) |
Idenix |
NM283 |
Valopicitabine; oral nucleoside analogue |
Hepatitis C |
Preliminary data from Phase IIb trial showed 200 mg/day dose had comparable antiviral activity to 800-mg dose (4/29) |
Incyte Corp. |
DFC (Reverset) |
Nucleoside analogue reverse transcriptase inhibitor |
HIV |
Discontinued development due to increases in Grade 4 hyperlipasemia seen in long-term extension of Phase IIb trial (4/3) |
Inhibitex |
Veronate |
Antibody-based immune globulin |
S. aureus infections in low-birth-weight infants |
Phase III trial in 2,017 infants failed to meet its primary end-point of reducing infections (4/3) |
Tigris |
A-007 |
Intravaginal gel against HPV- induced lesions and invasive epithelial cancers |
Cervical intraepithelial neoplasia |
Began Phase II trial in 250 patients with high-grade CIN to evaluate responses and other endpoints (4/6) |
Vertex |
VX-950 |
Oral HCV protease inhibitor |
Hepatitis C |
Trial with pegylated interferon alfa-2a showed combination was well tolerated; 6 of 8 patients had HCV RNA levels below the limit of quantitation (4/29) |
MISCELLANEOUS | ||||
CombinatoRx |
CRx-170 |
Agent containing low-dose budesonide and low-dose nortriptyline |
Asthma |
Phase II trial in patients with mild allergic asthma demonstrated a statistically significant increase in breathing capacity (4/18) |
CombinatoRx |
CRx-119 |
Combination of a low dose of the steroid predni-solone, and amoxapine |
Chronic adult periodontitis |
Exploratory study failed to show a reduction in C-reactive protein or in periodontal pocket depth (4/7) |
ICOS Corp. |
Cialis (FDA-approved) |
Tadalafil; oral PDE5 inhibitor |
Erectile dysfunction |
Trial in 140 patients who had ED secondary to spinal cord injury showed improved erections vs. placebo (4/6) |
Ilypsa Inc.* |
ILY101 |
Metal-free, non-absorbed polymeric agent |
Hyper-phosphatemia |
Successfully completed Phase I trial (4/20) |
InterMune Inc. (ITMN) |
Pirfenidone |
Small-molecule p38-gamma inhibitor |
Idiopathic pulmonary fibrosis |
Began Phase III CAPACITY program, consisting of two trials that together will evaluate lung function in 580 patients (4/27) |
Neurocrine |
NBI-56418 |
Small-molecule gonadotropin-releasing hormone receptor antagonist |
Endometriosis |
Phase II trial in 76 patients demonstrated reductions in symptoms and pain vs. placebo (4/27) |
Nymox |
NX-1207 |
Investigational agent for BPH |
Benign prostatic hyperplasia |
Phase II trial will continue following review that showed no serious drug side effects (4/21) |
Othera |
OT-551 |
Eye-drop formulation of a catalytic antioxidant |
Geographic atrophy |
Began Phase II trial in 10 patients with GA, a sign of dry or atrophic age- related macular degeneration (4/5) |
PTC |
PTC124 |
Oral agent that targets nonsense mutations |
Cystic fibrosis |
Interim Phase II data in patients with CF due to a nonsense muta-ion showed improvements in CFTR chloride channel activity (4/4) |
Theravance |
159797 |
Long-acting beta agonist compound |
Asthma |
GSK began a Phase IIb trial in patients with mild to moderate asthma (4/20) |
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; FSE = Frankfurt Stock Exchange; NYSE = New York Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; SWX = Swiss Stock Exchange; TSE = Toronto Stock Exchange. | ||||
