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.