Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Adheron Therapeutics (San Francisco)

SDP051

Lead Cad-11 inhibitor; humanized monoclonal antibody to Cadherin-11

Rheumatoid arthritis

Phase I data showed it was safe and well tolerated, with no evidence of significant side effects

1/9/14

Redhill Biopharma Ltd. (Tel-Aviv, Israel)

RHB-104

An antibiotic

Crohn's disease

Phase I data showed it had higher bioavailability when given with food and there were no serious adverse events reported

1/7/14

CANCER

Advaxis Inc. (Princeton, N.J.)

ADXS-HPV

Immunotherapy

Human papillomavirus-associated head and neck cancer

First patient was dosed in a phase I/II study

1/22/14

Apceth GmbH (Munich, Germany)

Mesenchymal stromal cells

Genetically modified mesenchymal stromal cells harvested from a patient's own bone marrow

Advanced adenocarcinoma of the gastrointestinal tract

Phase I/II trial began to enroll patients

1/24/14

Argen-X BV (Breda, the Netherlands)

ARGX-110

Monoclonal antibody

CD70-positive cancer

Advanced it into the safety and efficacy expansion part of its phase Ib study

1/14/14

Arno Therapeutics Inc. (Flemington, N.J.)

Onapristone

An oral, anti-progestin hormone blocker

Progesterone receptor-positive tumors

Enrolled the first patient in a phase I study

1/22/14

Biolinerx Ltd. (Jerusalem)

BL-8040

Selective antagonist of chemokine receptor CXCR4

Acute myeloid leukemia and other hematological disorders

Filed the necessary regulatory submissions to commence a phase I trial

1/14/14

Epizyme Inc. (Cambridge, Mass.)

EPZ-5676

DOT1L histone methyltransferase inhibitor

Acute leukemia

Phase I data showed it hit a proof-of-concept milestone, achieving objective responses

1/8/14

E-Therapeutics plc (Oxford, UK)

ETS2101

Synthetic cannibinoid

Brain cancer

Phase I data showed that five dose-escalation steps have been completed without any drug-related serious adverse events; Patient recruitment into phase I trials of ETS2101 in brain cancer and in solid tumors was halted temporarily due to a drug supply issue

1/8/14; 1/27/14

Immune Design Inc. (Seattle)

ID-G100

Agent that includes Toll-like receptor-4 agonist glycopyranosyl lipid A and stems from the company's GLASS discovery program

Merkel cell carcinoma

The first patient was treated in a phase I trial

1/28/14

Infinity Pharmaceuticals Inc. (Cambridge, Mass.)

IPI-145

Oral inhibitor of phosphoinositide-3-kinade-delta and P13K-gamma

Relapsed/refractory T-cell lymphoma

Phase I data showed that IPI-145 was clinically active with an overall response rate of 38%, including one complete response and nine partial responses

1/27/14

Karyopharm Therapeutics Inc. (Natick, Mass.)

Selinexor

An oral selective inhibitor of nuclear export, or SINE, compound

Metastatic colorectal cancer

Phase I data showed that one patient had a partial response by standard RECIST criteria and had remained on study for eight months; 11 patients had stable disease, 10 of those for eight weeks or longer, with four of those 10 patients (11 percent) demonstrating stable disease over 25 weeks

1/23/14

Kolltan Pharmaceuticals Inc. (New Haven, Conn.)

KTN3379

Monoclonal antibody designed to block the activity of ErbB3

Cancer

Treated the first cancer patient as part of a phase I study evaluating its safety, preliminary evidence of antitumor activity and pharmacokinetics

1/22/14

Merrimack Pharmaceuticals Inc. (Cambridge, Mass.)

MM-398

A stable nanoliposomal irinotecan designed to exploit leaky tumor vasculature for enhanced drug delivery to tumors

Pediatric solid tumors

Enrolled the first patient in a phase I dose-escalating trial of MM-398 in conjunction with cyclophosphamide

1/24/14

Nektar Therapeutics Inc. (San Francisco)

NKTR-102

Etirinotecan pegol; a long-acting topoisomerase 1 inhibitor

Advanced cancer

Phase I data of NKTR-102 in combination with 5-fluorouracil showed the compound was safely combined with 5-FU/LV and showed signs of clinical benefit, including both objective responses and tumor marker reductions

1/22/14

Oncomed Pharmaceuticals Inc. (Redwood City, Calif.)

OMP-54F28

A fusion protein based on a truncated form of the Frizzled8 receptor

Pancreatic cancer

Started a multicenter phase Ib trial of OMP-54F28 (Fzd8-Fc) with nab-paclitaxel (Abraxane, Celgene Corp.) and gemcitabine

1/14/14

Oncomed Pharmaceuticals Inc. (San Francisco) and Celgene Corp. (Summit, N.J.)

Demcizumab

Humanized monoclonal antibody that inhibits delta-like ligand 4 in the Notch signaling pathway

First-line stage IV pancreatic cancer

Phase Ib data of demcizumab combined with gemcitabine and Abraxane showed it was generally well tolerated, with fatigue, hypertension, nausea and vomiting the most common drug-related toxicities; three of six evaluable patients had partial responses and two had stable disease

1/21/14

Oncomed Pharmaceuticals Inc. (San Francisco) and Glaxosmithkline plc (London)

OMP-59R5

A fully human monoclonal antibody that targets the Notch2 and Notch3 receptors

Advanced pancreatic cancer

Phase Ib data showed it was well tolerated when used with standard of care, with no dose-limiting toxicities and typical adverse events; six of 13 patients treated with OMP-59R5, gemcitabine and Abraxane achieved stable disease

1/21/14

Rexahn Pharmaceuticals Inc. (Rockville, Md.)

RX-3117

A next-generation cancer cell-specific drug

Solid tumors

Started a phase Ib trial to study the safety and efficacy of RX-3117 in cancer patients

1/9/14

Senesco Technologies Inc. (Bridgewater, N.J.)

SNS01-T

A gene regulatory approach to cancer therapy that is designed to selectively trigger apoptosis in B-cell cancers

Multiple myeloma and non-Hodgkin's B-cell lymphoma

Dosed SNS01-T in the first patient in the fourth cohort of its phase Ib/IIa study

1/10/14

Starpharma Holdings Ltd. (Melbourne, Australia)

DEP-Docetaxel

Dendrimer-enhanced docetaxel chemotherapeutic product

Solid tumors

Received the approvals to start a phase I trial with 30 patients

1/24/14

Vaxil Biotherapeutics Ltd. (Ness Ziona, Israel)

Immucin

Therapeutic vaccine

Metastatic breast cancer

Recruited the first patient for a study, VAXIL-010, that will include up to 20 patients with metastatic breast cancer displaying cancer marker MUC1, who will receive 12 injections of Immucin in combination with hormone therapy

1/29/14

CARDIOVASCULAR

Alnylam Pharmaceuticals Inc. (Cambridge, Mass.)

ALN-AT3

A subcutaneously administered RNAi therapeutic targeting antithrombin

Hemophilia and rare bleeding disorders

Started a phase I trial

1/23/14

Catalyst Pharmaceutical Partners Inc. (Coral Gables, Fla.) and Biomarin Pharmaceutical Inc. (Novato, Calif.)

Firdapse

Amifampridine phosphate

Lambert-Eaton myasthenic syndrome

The first formal human cardiac safety study met its pre-specified endpoint, demonstrating no effect on heart rate or cardiac depolarization when administered at and above therapeutic levels

1/9/14

Neuralstem Inc. (Rockville, Md.)

NSI-566

Spinal cord-derived neural stem cells

Motor deficits from ischemic stroke

The first patient was treated in the phase I/II stem cell trial at Bayi Brain Hospital in Beijing, China; it is the first trial in which Neuralstem’s spinal cord-derived stem cells are being transplanted directly into a patient’s brain for placement near the stroke lesion

1/14/14

Resverlogix Corp. (Calgary, Alberta

RVX-208

A BET inhibitor

Atherosclerosis

Major adverse cardiac event (MACE) data from two trials were combined, demonstrating that treatment with RVX-208 led to a significant reduction in MACE; RVX-208-treated-patients had less cumulative events of 6.74% vs. 15.09% (p = 0.02) in the placebo-treated group

1/16/14

CENTRAL NERVOUS SYSTEM

Collegium Pharmaceuticals Inc. (Canton, Mass.)

Oxycodone Deterx

Extended-release, abuse deterrent oxycodone microsphere-in-a-capsule product

Pain

Top-line data from a human abuse potential trial showed a significantly lower peak “drug liking” when compared with intranasal crushed immediate-release oxycodone (p < 0.0001)

1/16/14

Cortice Biosciences Inc. (New York)

TPI 287

An abeotaxane; a taxoid that binds to and stabilizes microtubules

Mild to moderate Alzheimer's disease

Enrollment began in a phase I trial

1/22/14

Cynapsus Therapeutics Inc. (Toronto)

APL-130277

Sublingual thin film strip formulation of apomorphine

Parkinson's disease

Reported positive top-line data from a healthy volunteer pilot crossover trial comparing it to a commercially available injectable formulation of apomorphine

1/14/14

Cynapsus Therapeutics Inc. (Toronto)

APL-130277

A sublingual thin film strip formulation of apomorphine

Parkinson's disease

Top-line data from a pilot study comparing APL-130277 to a commercially available injectable formulation of apomorphine indicated that APL-130277 may have advantages over an injectable product by reducing the frequency and intensity of side effects including nausea and vomiting vs. those commonly reported for a subcutaneous injectable formulation

1/15/14

Oxford Biomedica plc (Oxford, UK)

Prosavin

A lentiviral, vector-based therapy

Advanced Parkinson's disease

Phase I/II data showed a favorable safety profile and a statistically significant improvement in motor function relative to baseline at six and 12 months post-treatment

1/13/14

Stemcells Inc. (Newark, Calif.)

HuCNS-SC

Human neural stem cells

Chronic spinal cord injury

A team at the University of Calgary successfully transplanted its first subject in the phase I/II trial

1/13/14

INFECTION

Enanta Pharmaceuticals Inc. (Watertown, Mass.)

EDP-788

Bicyclolide that targets the bacterial ribosome

Methicillin-resistant Staphylococcus aureus infections

Started a phase I study

1/14/14

Geovax Labs Inc. (Atlanta)

GOVX-B11

Vaccine

HIV

Preliminary top-line results from a phase I trial investigating the ability of the vaccine to control HIV infections in patients who initiated antiretroviral drug treatment within 18 months of seroconversion showed enhanced CD8-positive T cells were elicited in eight of nine patients and enhanced CD4-positive T cell and antibody responses in four of nine participants, and there was a trend toward enhanced viral control in individuals with the best CD4-positive and CD8-positive T-cell responses

1/14/14

Medivector Inc. (Boston)

Favipiravir

Inhibits the enzyme viral RNA polymerase that functions to make copies of the viral RNA segments and to synthesize mRNAs that produce the proteins that will form new viruses

Uncomplicated influenza

Enrolled the first patient in the U.S. in one of two favipiravir phase III studies; the trial is expected to enroll more than 1,000 patients in participating clinics and practices in North, Central and South America

1/21/14

Vaxart Inc. (San Francisco)

Vaccine

Oral H1N1 seasonal influenza vaccine candidate

Influenza

Vaxart reported positive safety and immunogenicity data from a phase Ib dose-ranging study, inducing antihemagglutinin antibody responses in 88% of subjects

1/10/14

MISCELLANEOUS

Aerie Pharmaceuticals Inc. (Bedminster, N.J.)

AR-13324

Eye drops

Glaucoma

Top-line phase I data demonstrated very low systemic exposure to the drug, with blood levels at or below the limit of detection of 0.1 ng/mL at all time points; after eight days a once-daily administration reduced the average diurnal IOP to approximately 11 mmHg, representing a decrease of approximately 5 mmHg, or more than 30%

1/10/14

Galectin Therapeutics Inc. (Norcross, Ga.)

GR-MD-02

Targets galectin-3

Fatty liver disease (NASH) with advanced fibrosis

Patient enrollment was completed in the first cohort of a phase I trial

1/14/14

Oxthera AB (Stockholm)

Oxabact

An oral product composed of highly concentrated freeze-dried live bacteria, administered in capsules

Primary hyperoxaluria

Initiated a clinical study to be conducted at seven sites in three countries and will include a total of 24 patients

1/9/14

Pluristem Therapeutics Inc. (Haifa, Israel)

PLX-PAD

Placental eXpanded cells

Muscle injury

Top-line results from its phase I/II trial showed the cells were safe and that statistical significance was reached (p = 0.0067) for the primary endpoint, defined as the change in maximal voluntary isometric contraction force of the gluteal muscle at six months after total hip replacement

1/22/14

PTC Therapeutics Inc. (South Plainfield, N.J.)

Unnamed compound

Oral compound

Spinal muscular atrophy

Entered the first stage of clinical development

1/23/14

Therapeuticsmd Inc. (Boca Raton, Fla.)

TX 12-004-HR

A rapidly acting vaginal preparation capsule

Vulvar vaginal atrophy in postmenopausal women

Final pharmacokinetic results from two studies showed substantially lower systemic estradiol exposure of TX 12-004-HR compared to Vagifem

1/29/14

RESPIRATORY

Faron Pharmaceuticals Ltd. (Turku, Finland)

FPCLI001

Treatment for respiratory distress syndrome

Respiratory distress syndrome

Data showed it reduced the odds of all-cause mortality by more than 80%

1/16/14

Notes

BLA = Biologics license application; CMA = Continuous marketing application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment.

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.