Company (Location)

Product

Description

Indication

Status (Date)#

Trident Pharmaceuticals Inc. (Boston)

HF1020

A recombinant protein with immunomodulatory activity

Autoimmune diseases and allergic asthma

Started a Phase Ia study (9/20)

Acetylon Pharmaceuticals Inc. (Boston)

ACY-1215

HDAC inhibitor

Relapsed and relapsed/refractory multiple myeloma

Started patient treatments in a three-part Phase I/IIa trial (9/23)

ActoGeniX NV (Ghent, Belgium)

AG013

Oral rinsing solution

To prevent oral mucositis in head and neck cancer patients

It was well tolerated in a Phase Ib trial in patients receiving chemo-induction therapy; top-line data showed a 35% reduction in days with ulcerative oral mucositis for patients taking the drug vs. placebo (9/16)

Ariad Pharmaceuticals Inc. (Cambridge, Mass.)

AP26113

A dual inhibitor of anaplastic lymphoma kinase and epidermal growth factor receptor

Non-small-cell lung cancer

Started patient dosing in a Phase I/II trial (9/22)

Ariad Pharmaceuticals Inc. (Cambridge, Mass.)

Ponatinib

Pan-BCR-ABL inhibitor

Chronic myeloid leukemia and Philadelphia-positive acute lymphoblastic leukemia

Phase I data showed that 72% of chronic-phase CML patients achieved a major cytogenetic response, including 92% who had a T315I mutation (9/27)

Avila Therapeutics Inc. (Bedford, Mass.)

AVL-292

An oral selective covalent inhibitor of Bruton's tyrosine kinase

B-cell cancer malignancies

Completed 28 days of dosing of the first cohort of patients in its Phase Ib trial (9/15)

CytRx Corp. (Los Angeles)

INNO-206

Tumor-targeting doxorubicin conjugate

Advanced soft tissue sarcomas

CytRx is tripling the number of patients in the Phase Ib/II trial who will be treated at the Phase IIb dose (9/27)

Glycotope GmbH (Berlin)

TrasGex

A biobetter of HER-2 targeted drug Herceptin

Cancer

Enrolled the first patients in a Phase I trial (9/20)

ImmunoCellular Therapeutics Ltd. (Los Angeles)

ICT-107

A cancer vaccine

Glioblastoma multiforme

Phase I data demonstrated a three-year overall survival rate of 55% compared to a rate of 16% based on historical standard of care (9/13)

Immunomedics Inc. (Morris Plains, N.J.)

Clivatuzumab tetraxetan

Humanized monoclonal antibody

Advanced pancreatic cancer

FDA placed a partial hold on its Phase Ib/II trial due to the administration of an incorrect dose to a patient enrolled at one of the trial sites (9/26)

Jennerex Inc. (San Francisco)

JX-594

Pox virus

Metastatic tumors

Phase I data demonstrated that after intravenous administration of a single dose of the drug, JX-594 was able to specifically replicate in metastatic tumors (9/1)

Marshall Edwards Inc. (San Diego)

ME-143

An NADH oxidase inhibitor

Refractory solid tumors

Started a Phase I dose-escalation trial (9/9); first patient was dosed in the Phase I trial (9/20)

MorphoSys AG (Martinsried, Germany)

MOR202

Anti-CD38 cancer antibody

Multiple myeloma

Dosed the first patient in a Phase I/IIa trial (9/2)

MorphoSys AG (Martinsried, Germany) and Bayer AG (Leverkusen, Germany)

BAY 94-9343

HuCAL-derived antibody-drug conjugate

Cancer

Started a Phase I cancer trial (9/15)

Niiki Pharma Inc. (Hoboken, N.J.)

NKP-1339

A transferrin-targeted small molecule designed to down-regulate GRP78

Metastatic solid tumors

Phase I data showed that six of the first 24 patients enrolled exhibited antitumor activity demonstrated by disease stability and/or tumor regression for at least 12 weeks (9/21)

Pacira Pharmaceuticals Inc. (Parsippany, N.J.)

Exparel

Bupivacaine liposome extended-release injectable suspension

For use following augmentation mammoplasty using silicone implants

New data demonstrate the long-term safety of Exparel (9/27)

Spectrum Pharmaceuticals Inc. (Henderson, Nev.)

SPI-1620

A selective endothelin-B agonist

Prostate cancer and other cancers

Top-line Phase I data of SPI-1620 in combination with docetaxel showed a total of five partial responses seen in the 30 patients treated (9/20)

Sunesis Pharmaceuticals Inc. (South San Francisco)

MLN2480

Cancer candidate

Cancer

Began a Phase I trial (9/28)

Acceleron Pharma Inc. (Cambridge, Mass.)

ACE-536

A ligand trap designed to increase red blood cells and hemoglobin

Anemia

Started a Phase I trial (9/14)

Bioheart Inc. (Sunrise, Fla.)

LipiCell

Adipose-derived cells

Congestive heart failure

Is beginning a Phase I U.S. trial, called ANGEL (9/15)

PolyMedix Inc. (Radnor, Pa.)

PMX-60056

A synthetic small molecule

Bleeding during cardiology procedures

Began a Phase Ib/II trial (9/28)

ReNeuron Ltd. (Guildford, UK)

ReN001

Stem cell treatment

Stroke

An independent data safety monitoring board cleared the company to advance to a higher dose, as there were no cell-related adverse events (9/2)

SanBio Inc. (Mountain View, Calif.)

SB623

A regenerative cell therapy consisting of cells derived from genetically engineered bone marrow stromal cells

Ischemic stroke

Opened a Phase I/IIa trial (9/21); the first subject has been treated (9/29)

BioDelivery Sciences International Inc. (Raleigh, N.C.)

BEMA

Buprenorphine/naloxone formulation

Opioid dependence

Phase I data showed it delivered plasma concentrations of buprenorphine in the range necessary to treat opioid dependence with minimal exposure of naloxone (9/7)

BioLineRx Ltd. (Jerusalem)

BL-1021

An oral small molecule

Neuropathic pain

Phase Ia data showed a single administration was safe and well tolerated (9/14)

Nektar Therapeutics Inc. (San Francisco)

NKTR-181

An opioid analgesic

Chronic pain

Dosing began in a Phase I trial (9/9); Phase I data showed it produced a clear dose-dependent response in a cold pressor test in healthy subjects (9/22)

Neuralstem Inc. (Rockville, Md.)

Stem cells

Human spinal cord stem cells

Amyotrophic

lateral sclerosis

Phase I data showed that all nine patients were alive with no unresolved serious adverse reactions from the surgery (9/28)

Roche AG (Basel, Switzerland)

Unnamed molecule

Designed to target the gamma-amino butyric acid system

Cognitive and behavioral deficits associated with Down syndrome

Started a Phase I trial (9/12)

Biodel Inc. (Danbury, Conn.)

BIOD-105 and BIOD-107

Fast-acting insulin products

Diabetes

Company will not advance the products into Phase II after Phase I data showed the drugs had only "slightly" more rapid absorption and slower declines from peak compared to Humalog (9/28)

Lexicon Pharmaceuticals Inc. (The Woodlands, Texas)

LX4211

A dual inhibitor of the sodium glucose transporters 1 and 2

Diabetes

Data demonstrated a favorable safety profile and effects on multiple parameters of glycemic control and cardiovascular health in healthy subjects (9/14)

TheraVasc Inc. (Cleveland)

TV1001

Oral formulation of sodium nitrate; enteric-coated and a nonenteric-coated capsule

Diabetes

Completed a Phase I trial with data showing few adverse events and no significant increase in methemoglobin levels (9/21)

Basilea Pharmaceutica Ltd. (Basel, Switzerland)

BAL30072

Sulfactam antibiotic

Multidrug-resistant Gram-negative pathogens

Started a second Phase I trial (9/8)

Biotron Ltd. (Sydney, Australia)

BIT225

Designed to target the p7 protein

HIV

Began a Phase Ib/IIa trial (9/30)

Inovio Pharmaceuticals Inc. (Blue Bell, Pa.)

Pennvax-G

Global vaccine plus a virus vector vaccine, Modified Vaccinia Ankara-Chiang Mai Double Recombinant

HIV

Phase I data demonstrated strong immune responses and safety (9/14)

Inovio Pharmaceuticals Inc. (Blue Bell, Pa.)

Pennvax-B

Consists of SynCon immunogens targeting HIV gag, pol and env proteins from HIV subtype B

HIV

Achieved best-in-class immune responses in a Phase I study (9/15)

MicroDose Therapeutx Inc. (Monmouth Junction, N.J.)

MDT-637

Inhalable small-molecule antiviral fusion inhibitor

Respiratory syncytial virus

Dosed the first human subject in a Phase I trial (9/28)

NexBio Inc. (San Diego)

Fludase

DAS181; host-targeted drug that blocks entry of the virus into respiratory tract cells

Parainfluenza virus Type 3 infection

A bone marrow transplant patient experienced improved respiratory status within one day of initiating treatment; by day three, the patient no longer required supplemental oxygen (9/1)

Phenex Pharmaceuticals AG (Ludwigshafen, Germany)

Px-102

FXR agonist

Non-alcoholic steatohepatitis

Began a Phase I trial (9/16)

Santaris Pharma A/S (Hoersholm, Denmark)

Miravirsen

miRNA-122

Hepatitis C virus

Data showed it was well tolerated in Phase I trials (9/8)

Viamet Pharmaceuticals Inc. (Research Triangle Park, N.C.)

VT-1161

An oral, small-molecule metalloenzymes inhibitor

Fungal pathogens

Started a Phase I trial (9/15)

Acucela Inc. (Seattle) and Otsuka Pharmaceutical Co. Ltd. (Tokyo)

OPA-6566

An adenosine A2 receptor agonist

Open-angle glaucoma or ocular hypertension

Started a Phase I/II trial (9/14)

Auxilium Pharmaceuticals Inc. (Malvern, Pa.)

Xiaflex

Collegenase clostridium histolyticum

Dupuytren's contracture

Top-line pilot data showed the tolerability of two doses of the drug into palpable cords in the same hand is consistent with that of single doses as demonstrated in the pivotal Xiaflex trials (9/8)

BioSante Pharmaceuticals Inc. (Lincolnshire, Ill.)

LibiGel

Testosterone gel

Female sexual dysfunction

Completed a pharmacokinetic study, showing it increased levels of free testosterone, bioavailable testosterone and total testosterone in a study of 24 postmenopausal women (9/13)

Critical Pharmaceuticals Ltd. (Nottingham, UK)

CP024

A nasal formulation of human growth hormone

Growth hormone deficiency

Phase I data showed it was able to induce IGF-1 to the same levels as a subcutaneous injection of the marketed product (9/7)

Halozyme Therapeutics Inc. (San Diego)

HTI-501

A recombinant human proteinase known as cathepsin-L

Moderate to severe edematous fibrosclerotic panniculopathy, commonly known as cellulite

Began a Phase I/II proof-of-concept and local tolerability trial of HTI-501 in women with cellulite (9/29)

ISTA Pharmaceuticals Inc. (Irvine, Calif.)

Xibrom

Bromfenac ophthalmic solution

Neovascular age-related macular degeneration

Pilot study suggested Xibrom administered twice daily might have an additive effect when used with Lucentis in reducing retinal thickness; there was no statistically significant difference in visual acuity outcomes at month 12 between the combination group and the group dosing with Lucentis alone (9/8)

Noxxon Pharma AG (Berlin)

NOX-H94

A Spiegelmer; targets the peptide hormone hepcidin

Anemia of chronic disease

Started a Phase I trial (9/7)

Otonomy Inc. (San Diego)

OTO-104

Delivered via a single intratympanic injection

Meniere's disease

Phase Ib data suggested patients treated with OTO-104 experienced clinically meaningful reductions in vertigo frequency and improvements in tinnitus compared to placebo (9/30)

StemCells Inc. (Newark, Calif.)

HuCNS-SC

Adult neural stem cells

Chronic spinal cord injury

The first patient in its Phase I/II trial was implanted successfully with the firm's HuCNS-SC adult neural stem cells (9/26)

Synageva BioPharma Corp. (Lexington, Mass.)

SBC-102

An enzyme replacement therapy

Lysosomal acid lipase deficiency (Wolman disease)

Expanded its ongoing clinical trial to the U.S. (9/29)

Thrasos Innovation Inc. (Montreal)

THR-184

An activator of specific receptors in the bone morphogenetic protein receptor pathway

Acute kidney injury

Started a Phase I trial (9/14)

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.