Company (Location) |
Product |
Description |
Indication |
Status (Date)# |
AUTOIMMUNE | ||||
Coronado Biosciences Inc. (Burlington, Mass.) |
TSO |
Trichuris suis ova |
Crohn's disease |
Phase I data in 36 patients showed the drug to be safe and well tolerated (2/29) |
GeNeuro SA (Geneva) |
GNbAC1 |
Designed to target the envelope protein of an endogenous retrovirus |
Multiple sclerosis |
Completed its Phase I trial, which showed the drug was well tolerated (2/9) |
TxCell SA (Valbonne, France) |
Ovasave |
An antigen-specific regulatory T cell-based immunotherapy |
Crohn's disease |
Phase I/II data indicated the treatment affected the immune system specifically in patients responding to Ovasave with a decrease in blood pro-inflammatory monocytes subpopulations and with an inhibition of the immune response to Ovasave-specific antigen (2/22) |
CANCER | ||||
Aduro BioTech Inc. (Berkeley, Calif.) |
ANZ-100 and CRS-207 |
Vaccine based on a live-attenuated, double-deleted Listeria monocytogenes platform; and a further engineered version to express mesothelin |
Advanced treatment-refractory cancers |
Phase I data showed intravenous administration of ANZ-100 and CRS-207 was well tolerated and showed evidence of immune activation, induction of tumor-specific immunity and that 37% of end-stage cancer subjects treated with CRS-207 lived 15 months or longer (2/8) |
Aeterna Zentaris Inc. (Quebec City) |
AEZS-108 |
Doxorubicin LHRH-targeted conjugate compound |
Prostate cancer |
Phase I data showed the drug was well tolerated and had signs of antitumor activity (2/6) |
Athersys Inc. (Cleveland) |
MultiStem |
Cell therapy product |
Leukemia |
Phase I data in patients undergoing hematopoietic stem cell transplants showed the cell therapy product was well tolerated in both the single-infusion and repeat infusion arms (2/2) |
Bio-Path Holdings Inc. (Houston) |
BP-100-1.01 |
Lipsomal Grb-2 |
Blood cancers |
Completed treatment of the second dosage cohort in its Phase I trial, showing it was well tolerated, with no treatment-related serious adverse events reported (2/8) |
BioSante Pharmaceuticals Inc. (Lincolnshire, Ill.) |
GVAX |
Pancreas cancer vaccine |
Pancreatic cancer |
Phase Ib data demonstrated it increased the median survival of pancreatic cancer patients with previously treated, locally advanced or metastatic disease by more than 60%, from 3.3 months when treated with Yervoy to 5.5 months on ipilimumab plus the GVAX pancreas vaccine (2/28) |
Curis Inc. (Lexington, Mass.) and Debiopharm Group (Lausanne, Switzerland) |
Debio 0932 |
A heat-shock protein 90 inhibitor |
Advanced solid tumors |
Started a Phase Ib expansion study (2/17) |
Diffusion Pharmaceuticals LLC (Charlottesville, Va.) |
Trans sodium crocetinate |
First-line treatment |
Glioblastoma |
Started enrollment in a Phase I/II trial (2/14) |
Exelixis Inc. (South San Francisco) |
Cabozantinib |
An inhibitor of MET, RET and VEGFR2 |
Renal cell carcinoma |
Phase Ib data showed a high rate of durable tumor response, with a low rate of primary refractoriness to drug therapy, and a long median progression-free survival (2/6) |
Galena Biopharma Inc. (Lake Oswego, Ore.) |
Folate-binding protein vaccine |
Consists of the E39 peptide combined with the immune adjuvant, granulocyte macrophage-colony stimulating factor |
Ovarian and endometrial adenocarcinomas |
Started a Phase I/II study (2/15) |
GlobeImmune Inc. (Louisville, Colo.) |
GI-6301 |
A Targmogen candidate |
Metastatic cancers containing brachyury protein |
Started a Phase I trial (2/9) |
Hutchison MediPharma Ltd. (London) |
HMPL-504 |
Volitinib; a c-Met receptor tyrosine kinase |
Lung and gastric tumors |
Began a first-in-human Phase I trial of oncology candidate volitinib (2/23) |
Novelos Therapeutics Inc. (Madison, Wis.) |
I-124-CLR1404 |
A cancer-targeted PET imaging agent |
Advanced non-small-cell lung cancer |
Enrolled the first patient in a Phase I/II trial (2/23) |
Redhill Biopharma Ltd. (Tel Aviv, Israel) |
RHB-102 |
A once daily controlled release proprietary formulation of Ondansetron |
Cancer-related nausea and vomiting |
Started a trial to prevent nausea and vomiting in cancer patients (2/16) |
Soligenix Inc. (Princeton, N.J.) |
SGX201 |
A time-release formulation of beclomethasone 17,21-diproprionate |
To prevent acute radiation enteritis |
Phase I/II data showed oral administration was safe and well tolerated across all four dose groups, and there was evidence of a potential dose response (2/13) |
CARDIOVASCULAR | ||||
CSL Behring LLC (King of Prussia, Pa.) |
rIX-FP |
Recombinant fusion protein linking coagulation Factor IX with albumin |
Severe hemophilia |
Phase I data showed the drug was well tolerated and lasted longer in the body, due to its prolonged half-life, compared with existing Factor IX treatment options (2/3) |
Cytokinetics Inc. (South San Francisco) |
Omecamtiv mecarbil |
Oral version; a cardiac muscle myosin activator |
Acute heart failure |
Started a Phase I study (2/22) |
PhaseBio Pharmaceuticals Inc. (Malvern, Pa.) |
Vasomera |
PB1046 |
Hypertension |
Began dosing its first subjects in a Phase I trial (2/23) |
SuppreMol GmbH (Munich, Germany) |
SM101 |
A recombinant, soluble, nonglycosylated version of the Fc receptor IIb |
Primary immune thrombocytopenia |
Interim results from a Phase Ib/IIa trial showed the drug to be safe and well tolerated, with encouraging efficacy (2/15) |
CENTRAL NERVOUS SYSTEM | ||||
Alkermes plc (Dublin, Ireland) |
Vivitrol |
Naltrexone for extended-release injectable suspension |
Opioid dependence |
Started a pilot study in prisoners with a pre-incarceration history of opioid dependence (2/1) |
Nektar Therapeutics Inc. (San Francisco) |
NKTR-181 |
A mu-opioid agonist |
Pain |
Phase I data showed it was able to enter the brain slowly and produce centrally mediated opioid effects that are dose dependent and statistically meaningful (2/28) |
Oragenics Inc. (Tampa, Fla.) |
LPT3-04 |
A naturally occurring compound |
Obesity |
A 32-patient study testing LPT3-04 over a 12-week period showed a statistically and clinically significant reduction in body weight in healthy, overweight and mildly obese adult volunteers (2/15) |
Trevena Inc. (King of Prussia, Pa.) |
TRV130 |
A biased ligand that targets the mu-opioid receptor |
Postoperative pain |
Started a Phase I trial (2/14) |
INFECTION | ||||
Achillion Pharmaceuticals Inc. (New Haven, Conn.) |
ACH-1625 |
Pan-genotypic protease inhibitor |
Wild type hepatitis C virus |
Phase I data showed declines in HCV, with no rebound of the virus carrying the most common resistant mutations (2/21) |
AVI BioPharma Inc. (Bothell, Wash.) |
AVI-6002 and AVI-6003 |
Lead drug candidates |
Ebola virus and Marburg virus |
Phase I data showed positive safety and the drugs are cleared to move into randomized, double-blind, placebo-controlled, multiple-ascending-dose studies (2/10) |
Genticel SA (Toulouse, France) |
ProCervix |
Lyophilized version; papillomavirus vaccine candidate |
Human papillomavirus |
Completed patient recruitment in a Phase Ib trial (2/22) |
Tekmira Pharmaceuticals Inc. (Vancouver, British Columbia) |
TKM-Ebola |
A systemically delivered RNAi therapeutic that uses the company's lipid nanoparticle delivery technology |
Ebola virus infection |
Started patient enrollment in a Phase I trial (2/9) |
MISCELLANEOUS | ||||
Ampio Pharmaceuticals Inc. (Greenwood Village, Colo.) |
Ampion in Knee |
Nonsteroidal anti-inflammatory candidate |
Osteoarthritis |
Preliminary results of the pain score portion of its Phase I/II trial showed an overall difference of 41%, compared to 28% with a placebo vehicle control (2/1) |
BioMarin Pharmaceutical Inc. (Novato, Calif.) |
BMN-111 |
An analogue of C-type natriuretic peptide |
Achondroplasia |
Started a Phase I study (2/21) |
Halozyme Therapeutics Inc. (San Diego) |
HTI-501 |
Active recombinant human cathespin |
Moderate to severe fibrosclerotic panniculopathy |
Interim data from a Phase I study showed no serious or severe adverse events (2/2) |
Summit plc (Oxford, UK) |
SMT C1100 |
A disease-modifying drug |
Duchenne's muscular dystrophy |
Plans to conduct a Phase I trial in healthy volunteers (2/15) |
Synageva BioPharma Corp. (Lexington, Mass.) |
SBC-102 |
A recombinant form of the human LAL enzyme |
Late onset lysosomal acid lipase deficiency |
Phase I/II data showed it was well tolerated, with no serious adverse events or infusion-related reactions (2/13) |
Notes: Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue. # The date indicated refers to the BioWorld Today issue in which the news item can be found. |