Company*
(Country; Symbol)

Product

Description

Indication

Status (Date)#

Ambit Biosciences Inc.

AC430

A selective Janus kinase 2 inhibitor

Autoimmune and inflammatory disease

Began a Phase I trial (12/22)

Argos Therapeutics Inc.

AGS-009

A humanized immunogliobulin G4 monmoclonal antibody that binds interferon alpha

Systemic lupus erythematosus

Started a double-blind, placebo-controlled, dose-escalation, Phase Ia trial (12/8)

Aduro BioTech

CRS-207

Based on Aduro's live-attenuated, double-deleted Listeria monocytogenes vaccine

platform

Advanced, refractory cancer

Phase I data showed it was well tolerated and had evidence for mesothelin-specific T-cell response (12/8)

Ambit Biosciences Inc.

AC480

A pan-HER inhibitor

Cancer

Began a Phase I trial (12/22)

ArQule Inc.

ARQ 736

A RAF kinase inhibitor

Cancer

Started dosing in a Phase I trial (12/7)

Enzon Pharmaceuticals Inc.

EZN-2968 and EZN-2208

An LNA-based antisense oligonucleotide

Solid malignancies, predominately involving the liver

Started Phase I studies (12/16)

ImmunoGen Inc.

IMGN901

Lorvotuzumab mertansine

Small-cell lung cancer

Started a Phase I/II trial (12/1)

ImmunoGen Inc.

T-DM1

Trastuzumab-DM1

HER2-positive metastatic breast cancer

Phase Ib/II data showed it produced a confirmed objective response to treatment in 57.1% of patients with first-line breast cancer (12/14)

Immunovaccine Inc.

DPX-0907

A DepoVax-based therapeutic cancer vaccine

Advanced breast, ovarian or prostate cancers

Phase I data showed it resulted in no dose-limiting toxicities or serious adverse events (12/16)

Intellikine Inc.

INK128

Small-molecule kinase inhibitor

Multiple myeloma

Began a Phase I trial (12/1)

Life Science Pharmaceuticals Inc. and Abbott

ABT-806

A humanized version of mAb 806 that selectively targets the epidermal growth factor receptor

Advanced solid tumors likely or known to express EGFR

Started a Phase I trial (12/15)

Jennerex Inc. and Transgene SA (France)

JX-594

An engineered oncolytic virus

Advanced metastatic, refractory colorectal cancer

Started enrolling and treating patients in a Phase Ib trial (12/17)

Mologen AG (Germany)

MGN1601

Cell-based gene therapy

Advanced renal cancer

Started a Phase I/II study (12/15)

Morphosys AG (Germany)

MOR208

Monoclonal anti-CD19 antibody

Chronic lymphocytic leukemia

Began a Phase I trial (12/3)

Oncothyreon Inc.

PX-866

A small-molecule P13K inhibitor

Colorectal cancer or squamous cell cancer of the head and neck

Enrolled the first patient in a Phase I/II trial (12/21)

Peregrine Pharmaceuticals Inc.

Bavituximab

Monoclonal antibody

Advanced hepatocellular carcinoma

Started a Phase I/II trial (12/2)

Spectrum Pharmaceuticals Inc. and Topotarget A/S (Denmark)

Belinostat

A small-molecule HDAC inhibitor

Non-small-cell lung cancer

Dosed the first patient in a Phase I/II trial (12/30)

Tekmira Pharmaceuticals Corp. (Canada)

TKM-PLK1

A polo-kinase 1 inhibitor

Advanced solid tumors

Started patient dosing in a Phase I trial; the study will enroll up to 52 patients (12/23)

Threshold Pharmaceuticals Inc.

TH-302

A hypoxia-activated prodrug

Advanced leukemias

Phase I data showed activity in multiple subjects with relapsed/refractory acute myelogenous leukemia and acute lymphoblastic leukemia; the drug also was well tolerated (12/23)

Tolerx Inc.

TRX518

A GITR-targeted antibody

Malignant melanoma

Began a Phase I trial (12/15)

Ziopharm Oncology Inc.

ZIO-201

Palifosfamide

Small-cell lung cancer

Dosed the first patient in a Phase I trial (12/30)

Acorda Therapeutics Inc.

GGF2

Glial Growth Factor 2

Heart failure

Started a Phase I trial (12/10)

Arena Pharmaceuticals Inc.

APD811

An oral candidate designed to target the prostacyclin receptor

Pulmonary arterial hypertension

Started dosing in a Phase I trial (12/13)

Isis Pharmaceuticals Inc.

ISIS-APOCIIIRx

An antisense inhibitor of apoC-III

Hypertriglycer-idemia

Began Phase I trials (12/22)

ReNeuron Group plc (UK)

ReN001

Stem cell therapy product

Stroke

Phase I testing will progress following a positive independent safety review of the first patient treated (12/23)

Clinical Data Inc.

Vilazodone

A dual-acting modulator of serotonin neurotransmission

Major depressive disorder

Two Phase I studies demonstrated that it did not prolong the QT interval and that alteration of gastric pH does not adversely affects its pharmacokinetics (12/9)

Neuraltus Pharmaceuticals Inc.

NP001

A small-molecule regulator of macrophage activation

Amyotrophic lateral sclerosis

Phase I data showed the drug was safe and well tolerated in 32 patients (12/1)

Neuraltus Pharmaceuticals Inc.

NP002

A small molecule, nicotinic receptor agonist

Dyskinesias in Parkinson's disease patients

Phase I/II data of NP002 in dyskinesias resulting from levodopa therapy showed it was generally safe and well tolerated, and was not associated with any impulsivity or withdrawal issues compared to placebo (12/6)

Orexo AB (Sweden)

OX219

A sublingual tablet formulation of buprenorphine and naloxone

Opioid dependence

Completed the initial pharmacokinetic trial, which demonstrated favorable profiles compared to comparator Suboxone (12/15)

Oxford BioMedica plc (UK)

ProSavin

Gene therapy

Parkinson's disease

Phase I/II data in nine patients showed it was safe and well tolerated at a 2x dose using an enhanced administration technique (12/23)

VistaGen Therapeutics Inc.

AV-101

An oral prodrug for neuropathic pain

Neuropathic pain

Completed the initial Phase I study, which showed it was well tolerated, had good bioavailability and did not cause any serious adverse events (12/21)

PhaseBio Pharmaceuticals Inc.

Glymera

Recombinant glucagon-like peptide-1 analogue

Diabetes II

Started a Phase I/II trial (12/2)

Chimerix Inc.

CMX157

A chemically modified version of tenofovir using the company's Phos-pholipid Intramem-brane-Microfluid- ization Conjugate technology

HIV

Phase I data demonstrated that CMS157 has a favorable safety, tolerability and drug distribution profile (12/14)

Dynavax Technologies Corp.

DV-601

A hepatitis B vaccine

Hepatitis B virus

Phase Ib data showed it was generally safe and well tolerated, and immunologic and virologic responses were achieved at all dose levels (12/27)

Dynavax Technologies Corp.

N8295

A fusion protein comprised of NP and M2e

Influenza

Phase Ia data showed that all doses were very safe and generally well tolerated (12/9)

Emergent BioSolutions Inc.

NuThrax

AV7909; a third-generation anthrax vaccine consisting of BioThrax and CPG 7909

Anthrax

Started a Phase I trial (12/28)

Idera Pharmaceuticals Inc.

IMO-2125

A Toll-like receptor 9 agonist

Hepatitis C virus

Phase I data in combination with ribavirin showed the combination was well tolerated and achieved substantial decline in virus levels at two days after the first dose of IMO-2125 and after four weeks of treatment (12/21)

Intercell AG (Austria)

IC84

Vaccine

Clostridium difficile infections

Started a Phase I trial (12/23)

Pharmasset Inc.

PSI-938 and PSI-7977

A purine and a pyrimidine nucleotide analogue

Hepatitis C virus

Started dosing in the second part of a Phase I study (12/1)

PharmAthene Inc.

Valortim

A fully human anti-toxin monoclonal antibody

Inhalation anthrax

FDA lifted the partial clinical hold, clearing the way for a Phase I trial (12/10)

Profectus Biosciences Inc.

Genevax IL-12

pDNA adjuvant

HIV

Significantly improved the vaccine-induced response rate in a Phase I study (12/15)

SEEK (UK)

FLU-v

Universal influenza vaccine

Influenza

Data showed it was safe and well tolerated and had evidence of immunogencity in a Phase I trial (12/8)

BioMimetic Therapeutics Inc.

Augment Rotator Cuff Graft

A collagen matrix hydrated with recombinant human platelet-derived growth factor BB

Large rotator cuff tears

Started enrollment in a pilot study (12/8)

Omeros Corp.

OMS201

PharmacoSurgery product candidate

Ureteral or renal stones

Was safe and well tolerated in a Phase I/II trial (12/30)

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.