Company* |
Product | Description | Indication |
Status |
| | ||||
AUTOIMMUNE | ||||
Array |
ARRY-438162 |
Small-molecule inhibitor of the protein kinase MEK |
Inflammatory diseases |
Phase I trial in 20 healthy volunteers met its primary objectives; studies in patients were being planned (10/19) |
Aspreva |
CellCept |
Mycophenolate mofetil; an immunosuppressant |
Myasthenia gravis |
80-patient trial showed no difference in efficacy between drug and prednisone vs. prednisone alone (10/11); 176-patient Phase III trial failed to meet either primary or secondary endpoints (10/26) |
Biogen Idec |
Tysabri |
Natalizumab; humanized monoclonal antibody that inhibits adhesion molecules on immune cells |
Crohn's disease |
Data showed drug maintained remission for longer than two years (10/24) |
Biogen Idec |
Tysabri (FDA-approved) |
Natalizumab; humanized monoclonal antibody that inhibits adhesion molecules on immune cells |
Multiple sclerosis |
Data from Phase III AFFIRM trial showed drug significantly reduced corticosteroid use and hospitalizations, and decreased disease activity (10/6) |
ChemoCentryx |
Traficet-EN (CCX282-B) |
Oral anti-inflammatory agent that targets the CCR9 chemokine receptor |
Crohn's disease |
Phase II trial showed a drop in disease activity and in blood levels of C-reactive protein (10/24) |
Osiris |
Prochymal |
Intravenous formulation of mesenchymal stem cells |
Crohn's disease |
Pilot Phase II study in those who didn't respond to other therapies showed a statistically significant decrease in disease activity (10/19) |
CANCER | ||||
Alfacell Corp. |
Onconase |
Ranpirnase; cytotoxic ribonuclease |
Malignant mesothelioma |
Further analysis of Phase IIIa trial showed greater median survival times for drug vs. doxorubicin (10/24) |
Allos |
Efaproxyn |
Efaproxiral; small molecule designed to sensitize hypoxic areas of tumors during radiation therapy |
Brain metastases originating from breast cancer |
Monitors recommended pivotal Phase III ENRICH trial continue to completion, following a second interim analysis (10/23) |
Antisoma plc |
AS1404 |
Small molecule derived from xanthenone acetic acid; vascular disrupting agent |
Ovarian cancer |
Phase II trial showed a response rate of 75% vs. 63% for patients receiving chemotherapy alone (10/15) |
AVEO |
AV-412 |
Next-generation, oral tyrosine kinase inhibitor of EGFR/HER2 |
Solid tumors |
Began Phase I trial to evaluate safety, tolerability and dosing in patients (10/23) |
Biogen Idec |
Zevalin (FDA-approved) |
Ibritumomab tiuxetan; monoclonal antibody targeting CD20, combined with radioisotope |
Diffuse large-B- cell lymphoma |
Began Phase III ZEAL trial to evaluate survival in patients taking Zevalin after chemotherapy regimen (10/4) |
Biovest |
BiovaxID |
Personalized vaccine; tumor antigen conjugated to KLH administered with GM-CSF |
Non-Hodgkin's follicular lymphoma |
20 of 25 indolent patients in Phase II trial developed an anti-idiotype immune response; second complete responses were significantly longer than first responses (10/19) |
Celgene |
Revlimid (FDA-approved) |
Lenalidomide; derivative of Thalomid (thalidomide) |
Advanced non- Hodgkin's lymphoma |
Seven of 22 evaluable patients in open-label Phase II trial exhibited an objective response (10/2) |
Cellgate Inc.* |
CGC-11047 |
Polyamine analogue that targets proliferating cells |
Advanced solid tumors and lymphomas |
Began Phase Ib trial to evaluate safety and pharmacokinetics with individual chemotherapeutics, in 70 patients (10/2) |
Cerus Corp. |
CRS-100 |
Attenuated strain of Listeria monocytogenes; immunotherapy |
Cancers that metastasized to the liver |
Began Phase I trial to evaluate dosing, safety and tolerability in patients (10/10) |
Cougar |
CB7630 |
Abiraterone acetate; oral inhibitor of the steroidal enzyme 17 alpha- hydroxylase/C17,20 lyase |
Advanced prostate cancer |
Eight of 12 patients in Phase I trial experienced reduced PSA levels of greater than 50%; treatment was well tolerated (10/8) |
CuraGen Corp. |
PXD101 |
Small-molecule histone deacetylase inhibitor |
Cancers |
Preliminary data from a Phase II trial and two Phase Ib/II trials, in solid tumors and multiple myeloma, showed encouraging results (10/26) |
EntreMed |
MKC-1 |
Small-molecule cell-cycle inhibitor |
Non-small-cell lung cancer |
Began Phase I/II trial to evaluate safety and dosing with pemetrexed (Alimta); Phase II portion will assess antitumor activity and progression- free survival in up to 60 patients (10/25) |
Genmab A/S |
HuMax-EGFr |
Zalutumumab; human antibody that targets the epidermal growth factor receptor |
Advanced head and neck cancer |
Began Phase I/II trial in 36 patients to evaluate first-line treatment in combination with radiotherapy and cisplatin (10/24) |
Genta Inc. |
G4460 |
Antisense agent that targets the oncogene c-myb |
Advanced hematologic cancers |
Began Phase I trial to evaluate dosing, safety, activity and down-regulation of c-myb in patients(10/13) |
Gloucester |
Romidepsin (FK228) |
Depsipeptide; histone deacetylase inhibitor |
Metastatic hormone- refractory prostate cancer |
Data from 22 patients in ongoing Phase II trial showed one partial response, two with stable disease and seven with disease stabilization(10/4) |
Hana |
Talvesta (PT-523) |
Talotrexin; nonclassical antifolate; analogue of aminopterin |
Advanced acute lymphoblastic leukemia |
Began Phase II trial to evaluate remission rates, as well as other efficacy endpoints (10/31) |
ImmunoGen |
AVE1642 |
Antibody that binds to the insulin-like growth factor 1 receptor |
Cancers |
Partner Sanofi-Aventis SA began clinical testing of the antibody (10/4) |
Innovive |
INNO-305 |
WT1 peptide therapeutic vaccine designed to stimulate both CD8 and CD4 T cells |
Hematologic malignancies and solid tumors |
Began Phase I trial to study safety, tolerability, pharmacokinetics and preliminary efficacy in WT1-expressing cancers (10/27) |
MethylGene |
MGCD0103 |
Class I-specific histone deacetylase inhibitor |
Solid tumors |
Began Phase I/II trial to evaluate the agent with Gemzar, first testing safety and dosing; the Phase II portion will assess responses in up to 60 patients (10/25) |
Northwest |
DCVax-Brain |
Dendritic cell-based immunotherapy made from patients' cells |
Glioblastoma multiforme |
Updated trial data showed 44% of drug patients were progression-free vs. 11% for historical controls; survival at two years was improved (10/30) |
Novacea Inc. |
Asentar (DN-101) |
High-dose oral formulation of calcitriol |
Cancers |
Phase I trial demonstrated safety and tolerability (10/30) |
Rexahn Corp.* |
RX-0201 |
Signal inhibitor that blocks production of the protein kinase Akt |
Cancers |
Successfully concluded Phase I trial in patients with advanced cancers (10/17) |
Vion |
Cloretazine (VNP40101M) |
Sulfonylhydrazine DNA alkylating agent |
Advanced small- cell lung cancer |
Initial data on 36 patients from single-agent Phase II trial showed a response rate of 32% (10/26) |
Ziopharm |
ZIO-101 |
Organic arsenic agent |
Solid tumors |
Data from ongoing Phase I trial showed benefit in eight of 29 patients (10/3) |
ZymoGenetics |
-- |
Interleukin-21 |
Metastatic stage IV renal cell cancer |
Began Phase I/II trial to evaluate safety and activity of the agent in combination with the approved drug Nexavar (sorafenib) (10/17) |
CARDIOVASCULAR | ||||
Amgen Inc. |
AMG 531 |
Peptibody protein designed to stimulate the thrombopoietin receptor |
Immune thrombocytopenic purpura |
Phase I/II trial demonstrated increased blood platelet counts (10/19) |
AVI |
Resten-MP |
Intravenous delivery of AVI-4126, an antisense drug targeting the c-myc transcription factor |
Cardiovascular indications |
Preliminary data from Phase II APPRAISAL trial demonstrated encouraging safety and delivery results (10/31) |
AVI |
AVI-5126 |
New generation of antisense drug |
For use in coronary artery bypass grafting |
Began pivotal trial to evaluate the product in 600 patients under-going CABG procedures (10/18) |
CardioVascular |
-- |
Formulation containing Cardio Vascu-Grow, a stimulator of angiogenesis |
Peripheral artery disease |
Began Phase I trial to evaluate safety and the growth of new blood vessels in the legs of 24 patients (10/24) |
CoGenesys |
Cardeva |
Long-acting form of B-type natriuretic peptide |
Heart failure |
Began Phase I/II trial to evaluate safety and tolerability in up to 80 stable patients with Class II or III heart failure (10/11) |
Corautus |
VEGF-2 |
Vascular endothelial growth factor in the form of naked plasmid DNA |
Severe angina |
Final data from GENASIS Phase IIb trial failed to show statistical significance in improving exercise tolerance time in any dose group (10/10) |
DeCode |
DG031 (veliflapon) |
Inhibitor of 5-lipoxygenase activating protein, or FLAP |
Prevention of heart attack |
Voluntarily suspended Phase III trial due to an unexpected formulation problem with the tablets being used in the trial (10/5) |
Metabasis |
MB07811 |
Liver-targeting thyroid hormone receptor agonist |
Hyperlipidemia |
Began Phase I trial to evaluate safety and tolerability in healthy volunteers (10/30) |
Momenta |
M118 |
Anticoagulant agent that binds to anti-thrombin III and thrombin |
Acute coronary syndromes |
Began Phase I trial in up to 36 healthy adult males to evaluate safety, tolerability and pharmaco- kinetics (10/10) |
Omrix Bio- |
-- |
Topical, human plasma- derived thrombin |
For use in general surgeries |
Pivotal Phase III trial demonstrted equivalence to bovine thrombin; a BLA filing was planned for November (10/16) |
Paion AG* |
Desmoteplase |
Genetically engineered version of a clot-dissolving protein found in the saliva of the vampire bat |
Acute ischemic stroke |
Monitors put Phase IIb/III DIAS-2 trial on hold pending further data analysis (10/25); patient enrollment was resumed with no modification of the protocol (10/28) |
Portola |
-- |
Intravenous formulation of its ADP receptor antagonist; an antiplatelet agent |
Cardiovascular indications |
Began a Phase I trial of the product (10/31) |
Renovis Inc. |
NXY-059 |
Neuroprotectant with free radical-trapping properties |
Acute ischemic stroke |
Pivotal Phase III SAINT II trial did not demonstrate a statistically significant reduction on stroke- related disability vs. placebo; secondary endpoints also were missed (10/26) |
Titan |
DITPA |
Analogue of thyroid hormone (T3) |
Congestive heart failure |
Discontinued further enrollment in Phase II trial; resources are being focused elsewhere (10/25) |
CENTRAL NERVOUS SYSTEM | ||||
Ceregene |
CERE-120 |
Gene therapy consisting of an adeno-associated virus vector carrying the gene for neurturin |
Parkinson's disease |
Phase I trial in 12 patients showed a 40% reduction in symptoms; treatment was well tolerated (10/10) |
Durect Corp. |
Memryte |
Durin implant technology with leuprolide acetate |
Alzheimer's disease |
Voyager ended Phase III trials in order to get an earlier look at potential efficacy; 600 patients had been accrued (10/19) |
Medivation |
Dimebon |
Neuroprotectant agent that's been on the market in Russia since 1983 |
Huntington's disease |
Began Phase I/IIa trial to evaluate safety, tolerability and preliminary efficacy in 75 patients (10/19) |
Neurogen |
NG2-73 |
Selective modulator of gamma aminobutyric (GABA) receptors |
Insomnia |
Began Phase II trial that will evaluate the efficacy and safety of five different dose and formulation profiles of the drug (10/30) |
NeurogesX |
Transacin (NGX-4010) |
High-concentration trans- capsaicin dermal patch |
Post-herpetic neuralgia |
Phase III trial in 420 patients demonstrated statistically significant pain reduction vs. a control patch (10/18) |
Neurologix |
-- |
Adeno-associated virus delivering gene encoding glutamic acid decarboxylase |
Parkinson's disease |
One-year data from Phase I trial in 12 patients showed statistically significant improvement from baseline; safety and tolerability also were demonstrated (10/17) |
Santhera |
SNT-MC17 |
Idebenone; small molecule optimized to facilitate the transport of electrons within mitochondria |
Friedreich's ataxia |
Phase II trial in 48 patients demonstrated safety and a trend toward improved neurological parameters (10/6) |
Somaxon |
Silenor |
Low dose of the approved depression drug doxepin |
Transient insomnia |
Phase III trial in 565 patients demonstrated statistically significant improvements in sleep onset vs. placebo, and on other sleep-related endpoints (10/23) |
Titan |
Probuphine |
Product designed to continuously deliver the approved drug buprenorphine |
Opioid dependence |
Began Phase III trial to evaluate safety and efficacy vs. placebo in 150 patients (10/25) |
TorreyPines |
Tezampanel |
AMPA/kainate receptor antagonist |
Acute migraine |
Began Phase IIb trial to evaluate pain relief and other endpoints in 300 patients (10/16) |
DIABETES | ||||
Emisphere |
-- |
Insulin delivered with company's oral eligen technology |
Type II diabetes |
Phase II trial met the company's safety and efficacy objectives (10/30) |
Sirtris |
SRT501 |
Agent that targets SIRT1, a member of the human sirtuin family of enzymes |
Type II diabetes |
Phase I trial in healthy volunteers demonstrated safety and tolerability; began Phase Ib trial in 90 patients to evaluate safety and pharmacokinetics (10/4) |
INFECTION | ||||
Advanced |
Cethromycin |
Second-generation ketolide antibiotic agent |
Acute bacterial sinusitis |
Presented data from studies that supported selection of 300-mg dose for further studies (10/13) |
Alnylam |
ALN-RSV01 |
RNAi agent designed to silence the RSV nucleo- capsid "N" gene; inhaled formulation |
Respiratory syncytial virus |
Began Phase I trial to evaluate safety, tolerability and pharmacokinetics in healthy adult volunteers (10/11) |
AVI |
AVI-4065 |
Neugene antisense compound |
Hepatitis C |
Exploratory study in 12 patients failed to show efficacy; a change in protocol was planned (10/4) |
Crucell NV |
-- |
AdVac-based tuberculosis vaccine; uses Ad35 adenovirus vector |
Tuberculosis |
Began Phase I trial to evaluate safety, tolerability and immuno-genicity in 24 healthy volunteers (10/25) |
GlobeImmune |
GI-5005 |
Targeted immunotherapy expressing a NS3-Core fusion protein |
Hepatitis C |
Phase Ib trial showed generation of cellular immune responses in 12 of 29 patients, and elicited a statistically significant improvement in alanine amino transferase levels (10/30) |
Hollis-Eden |
Neumune |
Immune-regulating hormone |
Infections and acute radiation syndrome |
Phase I trial in healthy volunteers demonstrated positive safety and hematopoietic data (10/13) |
Human |
Albuferon |
Long-acting form of interferon alpha, created with albumin fusion technology |
Hepatitis C |
Interim data from Phase IIb trial with ribavarin showed positive quality-of-life results through week 12 (10/16); Phase II data showed comparable efficacy to pegylated interferon in treatment-naïve patients (10/31) |
Idenix |
Tyzeka |
Telbivudine; oral, once-daily nucleoside analogue |
Hepatitis B |
Presented favorable data from the second year of the GLOBE study and from other clinical trials (10/27) |
Idenix |
Valopicitabine (NM283) |
Oral nucleoside analogue |
Hepatitis C |
Phase IIb trial with pegylated interferon showed favorable viral suppression at 24 weeks in treatment-naïve patients (10/27) |
MedImmune |
CAIV-T |
Trivalent vaccine; next generation of FluMist; refrigerator-stable formulation |
Influenza |
Trial in 52 children demonstrated higher antibody responses than the traditional injectable trivalent inactivated flu vaccine (10/13) |
Pharmasset |
R7128 |
Polymerase inhibitor; prodrug of the agent PSI-6130 |
Hepatitis C |
Began Phase I trial to evaluate safety and pharmacokinetics in healthy volunteers, and antiviral activity in patients (10/27) |
PowderMed |
DNA-based trivalent vaccine delivered via needle-free injection device |
Influenza |
Is starting Phase I trial to evaluate safety, tolerability and immuno-genicity (10/2) |
|
Sequella Inc.* |
SQ109 |
Inhibitor of cell wall synthesis in certain microorganisms |
Tuberculosis |
Began Phase I trial to evaluate safety and pharmacokinetics in 46 healthy volunteers (10/3) |
Starpharma |
VivaGel |
Vaginal microbicide gel |
Prevention of genital herpes |
Began Phase I trial that will evaluate safety in 60 volunteers (10/24) |
Theravance |
Telavancin |
Lipoglycopeptide; bactericidal injectable antibiotic |
Complicated skin and skin-structure infections |
Additional data from Phase III ATLAS 1 and ATLAS 2 trials demonstrated non-inferiority to vancomycin (10/10) |
Vertex |
VX-950 |
Telaprevir; oral HCV protease inhibitor |
Hepatitis C |
Phase Ib trial with pegylated interferon showed suppression of both wild-type HCV and resistant variants; also, trial showed 24 of 26 patients who took drug plus peg-FN and ribavirin had undetectable HCV RNA (10/27) |
ViroPharma |
HCV-796 |
Orally dosed hepatitis C viral polymerase inhibitor |
Hepatitis C |
Began Phase II trial to evaluate safety, tolerability, pharmacokinetics and antiviral activity when used with pegylated interferon alfa-2b plus ribavirin (10/24) |
MISCELLANEOUS | ||||
Adenosine |
Apadenoson (ATL146e) |
Selective adenosine A2A agonist; anti-inflammatory agent |
Various conditions |
Began Phase Ib trial to evaluate safety, dosing and anti-inflammatory proof of principle (10/25) |
Advanced |
Ferumoxytol |
Intravenously administered bioavailable iron |
Iron replacement in anemic chronic kidney disease |
Monitors found no safety concerns in review of data from 1,200 patients in Phase III trial (10/23) |
Amylin |
Symlin (FDA-approved) |
Pramlintide acetate; an analogue of human amylin |
Obesity |
52-week data from Phase II trial showed a 7% to 8% weight reduction vs. 1% reduction for placebo; company plans several studies testing drug with neurohormones for obesity (10/24) |
Auxilium |
AA4500 |
Injectable enzyme product |
Peyronie's disease |
89% of patients in Phase II trial who received three treatments achieved clinical success (10/25) |
Auxilium |
TestoFilm |
Testosterone replacement transmucosal film product |
Hypogonadism |
Development of the formulation, which was in Phase III comparative trials, was discontinued due to insufficient commercial viability (10/18) |
Collegium |
-- |
Agent incorporating both a sedating and nonsedating antihistamine |
Allergic rhinitis |
Began proof-of-concept study of the combination product (10/4) |
Critical |
-- |
Controlled-release formulation of zileuton (Zyflo), a 5-lipoxygenase inhibitor |
Asthma |
613-patient trial produced favorable safety and efficacy data vs. placebo and Zyflo (10/25) |
Discovery |
Surfaxin |
Engineered version of natural human lung surfactant |
Broncho- pulmonary dysplasia |
Phase II trial in 136 premature infants demonstrated safety and tolerability, and encouraging signs of efficacy (10/11) |
Dynavax |
AIC |
Toll-like receptor 9 agonist linked to ragweed allergen |
Ragweed allergy |
Pilot study showed treatment reduced allergic responses and sustained that effect more than 12 months (10/4) |
EPIX |
PRX-07034 |
Highly selective 5-HT6 receptor antagonist |
Obesity |
Began Phase I trial to evaluate safety, tolerability and pharmacokinetics in otherwise healthy obese adults; earlier Phase I trial demonstrated tolerability (10/24) |
Genaera |
Evizon (squalamine) |
Anti-angiogenesis agent; vascular endothelial growth factor inhibitor |
Wet age-related macular degeneration |
Data from Phase II trial showed no significant increase in systolic or diastolic blood pressure (10/16) |
Inspire |
-- |
Epinastine nasal spray formulation |
Seasonal allergic rhinitis |
Began Phase II program that will evaluate formulations and dosing (10/10) |
Manhattan |
Oleoylestrone |
Oral, synthetic form of Oleoylestrone |
Obesity |
Began Phase IIa trial to evaluate safety and efficacy in 24 morbidly obese male subjects (10/12) |
LAB |
LAB CGRP |
Calcitonin gene-related peptide; vasodilative agent |
Asthma |
Crossover Phase II study in 12 patients showed statistically significant broncho-protective effects vs. placebo (10/4) |
Microbia Inc.* |
Linaclotide (MD-1100) |
Peptide designed to act on guanylate cyclase-C, a receptor found on intestinal cells |
Chronic constipation |
Phase IIa trial demonstrated tolerability and safety, and improvements in bowel function (10/24) |
Nabi Bio- |
PhosLo (FDA-approved) |
Calcium acetate; phosphate binder |
Stage IV kidney disease |
EPICK study achieved its primary endpoint of superior serum phosphorus control vs. placebo; secondary endpoints also were met (10/16) |
NicOx SA |
HCT 3012 (naproxcinod) |
Nitric oxide-donating derivative of naproxen |
Osteoarthritis of the knee |
Phase III trial demonstrated statistical significance vs. placebo on all three co-primary efficacy endpoints (10/27) |
Orexigen |
Excalia |
Combination of bupropion, a dopamine and norepinephrine reuptake inhibitor, and the approved anticonvulsant zonisamide |
Obesity |
Phase IIa trial in 127 nonsmokers demonstrated increased weight loss vs. placebo (10/22) |
Regeneron |
IL-1 Trap |
Agent designed to attach to and neutralize interleukin-1 |
CIAS1-associated periodic syndromes |
Both Phase III trials met their primary endpoints of improving disease activity (10/30) |
Repligen |
RG1068 |
Synthetic human secretin |
Pancreatic disease |
Began study to evaluate the diagnostic utility of the product in MRI of the pancreas in 45 healthy vol-unteers and patients (10/30) |
PTC |
PTC124 |
Oral agent that targets nonsense mutations |
Duchenne muscular dystrophy |
Preliminary data from Phase II trial indicated increases in dystrophin and improvements in muscle enzymes (10/21) |
Tanox Inc. |
TNX-650 |
Humanized monoclonal antibody that targets interleukin-13 |
Asthma |
Began Phase I trial to evaluate safety, tolerability and pharmacokinetics in 32 healthy volunteers (10/25) |
Theravance |
TD-5108 |
Selective 5-HT4 receptor agonist |
Chronic constipation |
Began Phase II trial to evaluate safety and efficacy of a range of doses in 350 patients (10/9) |
Trine |
Crofelemer |
Agent extracted from the rain- forest plant Croton lechleri |
Diarrhea- predominant irritable bowel syndrome |
Began Phase IIb trial to evaluate efficacy and safety in 240 women with D-IBS (10/30) |
Urigen NA* |
U101 |
Intravesical formulation of FDA- approved components |
Pelvic pain of bladder origin |
Phase II trial did not meet primary endpoint of improved pain and urgency; positive trends were seen (10/30) |
Vivus Inc. |
Qnexa |
Agent incorporating low doses of the approved products phentermine and topiramate |
Obesity |
Phase II trial demonstrated a statistically significant weight loss and a reduction in waist size; lipid levels also were reduced (10/23) |
XenoPort |
XP19986 |
Transported prodrug of the generic spasticity drug R-baclofen |
Gastro- esophageal reflux disease |
Phase IIa trial demonstrated tolerbility and statistically significant reductions in reflux episodes and reflux-associated heartburn symptoms (10/22) |
| | ||||
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; ASX = Australian Stock Exchange; CSE = Copenhagen Stock Exchange; LSE = London Stock Exchange; OTC BB = Over-the-Counter Bulletin Board; TSX = Toronto Stock Exchange. | ||||
