Company
(Location)

Product

Description

Indication

Status

Date

CANCER

Active Biotech AB (Lund, Sweden) and Ipsen SA (Paris)

Tasquinimod

TASQ; designed to have immunomodulatory and anti-angiogenic properties

Prostate cancer

Phase III study is fully enrolled

12/11/12

Celator Pharmaceuticals Inc. (Princeton, N.J.)

CPX-351

Cytarabine/daunorubicin liposome injection

Acute myeloid leukemia

Treated its first patient in a Phase III trial of CPX-351 compared to conventional cytarabine and daunorubicin for first-line therapy

12/27/12

Celgene International Sarl (Boudry, Switzerland)

Revlimid

Lenalidomide

Non-Hodgkin's lymphoma

Trial data showed that patients had an overall response rate of 98%, and one-year progression-free survival of 73%; a second Phase III trial resulted in a median two-year progression-free survival rate of 92% in patients with diffuse large B-cell lymphoma who received maintenance lenalidomide, compared with 83% for patients receiving lenalidomide plus Rituxan

12/13/12

GTx Inc. (Memphis, Tenn.)

Enobosarm

A selective androgen-receptor modulator

To treat muscle wasting in non-small-cell lung cancer

Attained its enrollment goal for both of its pivotal Phase III studies

12/18/12

Ligand Pharmaceuticals Inc. (San Diego)

Melphalan

Captisol-enabled, propylene glycol-free (PG-free) high-dose melphalan

Multiple
myeloma

Started a pivotal trial

12/12/12

Oncolytics Biotech Inc. (Calgary, Alberta)

Reolysin

Reovirus variant

Head and neck cancer

Early Phase III results showed it lacked survival data and slipped on part of its endpoint, but it met statistical significance (p = 0.025) in patients with metastatic tumors, proving that, when combined with carboplatin and paclitaxel against platinum-refractory, taxane-naïve tumors, the drug did better at stabilizing or shrinking tumors

12/14/12

Oncothyreon Inc. (Seattle) and Merck KgaA (Darmstadt, Germany)

L-BLP25

Stimuvax; cancer vaccine that contains a 25 amino acid sequence derived from the mucin 1 (MUC-1) antigen

Non-small-cell lung cancer

Failed to meet the primary endpoint of overall survival in a pivotal Phase III trial

12/20/12

Stemline Therapeutics Inc. (New York)

SL-401

Targets the interleukin-3 receptor

Acute myeloid leukemia and other hematologic malignancies

Data showed it was well tolerated at clinically active doses and showed single agent antitumor activity in heavily pretreated patients

12/10/12

CARDIOVASCULAR

ARCA biopharma Inc. (Broomfield, Colo.)

Gencaro

Bucindolo hydrochloride

Heart failure

Phase III data demonstrated that patients with established atrial fibrillation receiving Gencaro in the trial had improvements in heart failure clinical endpoints, and those receiving Gencaro were more likely to achieve ventricular rate control

12/13/12

Chelsea Therapeutics Inc. (Charlotte, N.C.)

Northera

Droxidopa; a catecholamine that converts directly to norepinephrine via decarboxylation

Symptomatic neurogenic orthostatic hypotension associated with Parkinson's disease

Hit the primary endpoint in a Phase III trial reducing dizziness and/or lightheadedness at the first week, but showed only a trend toward working through the fourth and eighth weeks

12/6/12

Emmaus Medical Inc. (Torrance, Calif.)

L-glutamine

Levoglutamide

Sickle cell disease

Completed patient enrollment in a Phase III trial

12/4/12

CENTRAL NERVOUS SYSTEM

AcelRx Pharmaceuticals Inc. (Redwood City, Calif.)

Sufentanil NanoTab

PCA System

Pain

Top-line Phase III data demonstrated it was noninferior (p < 0.001) to intravenous patient-controlled analgesia with morphine for the primary endpoint of Patient Global Assessment (PGA) of method of pain control over the 48-hour study period as determined by the combined percentage of patients with PGA ratings of "good" or "excellent" (78.5% vs. 66. 1%, respectively)

12/17/12

Alexza Pharmaceuticals Inc. (Mountain View, Calif.) and Grupo Ferrer Internacional SA (Barcelona)

Adasuve

Staccato loxapine

Agitation in adults with schizophrenia or bipolar disorder

The EMA gave a positive opinion on it based on two randomized, multicenter Phase III studies showing statistically significant reductions in agitation from baseline compared to placebo

12/17/12

Vanda Pharmaceuticals Inc. (Rockville, Md.)

Tasimelteon

An agonist of human melatonin receptors MT1 and MT2

Non-24-hour syndrome

Met the primary endpoint of entrainment of melatonin rhythm compared to placebo in a Phase III trial; it also showed significant improvements in sleep and wake parameters such as total sleep time, nap duration and timing of sleep

12/19/12

Vanda Pharmaceuticals Inc. (Rockville, Md.)

Fanaptum

Oral iloperidone tablets

Schizophrenia

CHMP issued a negative opinion recommending against approval

12/17/12

INFECTION

Basilea Pharmaceutica AG (Basel, Switzerland)

Isavuconazole

Antifungal agent

Infections caused by Aspergillus or other filamentous fungi

Completed recruiting patients for a Phase III pivotal trial

12/17/12

Cempra Inc. (Chapel Hill, N.C.)

CEM-101

Oral solithromycin; a first-in-class fluoroketolide antibiotic candidate

Community-acquired bacterial pneumonia

Started a double-blind, multicenter study expected to enroll about 800 patients with PORT-II to PORT-IV CABP in a head-to-head comparison of oral solithromycin at an 800-mg loading dose followed by 400 mg once daily for five days or once-daily oral moxifloxacin at 400 mg for seven days; the primary endpoint is noninferiority of early response at 72 hours

12/20/12

Durata Therapeutics Inc. (Chicago)

Dalbavancin

Intravenous doses

Acute bacterial skin and skin structure infections caused by susceptible Gram-positive bacteria

Phase III data showed that dalbavancin achieved its primary endpoint of noninferiority at 48 hours to 72 hours after initiation of therapy, as determined by the cessation of spread of the lesion, as well as the resolution of fever

12/12/12

Medivir AB (Stockholm, Sweden)

TMC435

Simeprevir; protease inhibitor

Hepatitis C virus genotype 1 infection

Three Phase III trials of simeprevir, administered once daily in combination with pegylated interferon and ribavirin, achieved sustained virologic response 12 weeks after the completion of treatment (SVR12) in 80% and 81% of patients, respectively, while it was 50% for the control group

12/21/12

The Medicines Co. (Parsippany, N.J.)

Oritavancin

Antibiotic

Acute bacterial skin and skin structure infections caused by Gram-positive bacteria, including methicillin-resistant Staphylococcus aureus

Met primary and secondary endpoints in the SOLO-1 Phase III trial; data showed that 82.3% of patients who received oritavancin met the early clinical endpoint of cessation of spread, absence of fever and no rescue antibiotics, compared to 78.9% of patients receiving vancomycin

12/21/12

Trius Therapeutics Inc. (San Diego)

TR-701

Tedizolid phosphate

Acute bacterial skin and skin structure infections

Reached its enrollment objective of 658 patients in the last of its two Phase III trials

12/11/12

MISCELLANEOUS

Allon Therapeutics Inc. (Vancouver, British Columbia)

Davunetide

A peptide believed to act as a neuro-protective agent

Progressive supranuclear palsy

Pivotal trial failed to show an effect in patients

12/19/12

Amicus Therapeutics Inc. (Cranbury, N.J.) and GlaxoSmithKline plc (London)

Amigal

Migalastat HC1

Fabry disease

Phase III trial missed its primary and secondary endpoints

12/20/12

EyeGate Pharma Inc. (Waltham, Mass.)

EGP-437

Dexamethasone phosphate ophthalmic solution

Anterior uveitis

Enrolled the last patient in a 200-subject pivotal Phase III study

12/19/12

Intercept Pharmaceuticals Inc. (New York)

OCA

Obetiocholic acid; an FXR agonist

Primary biliary cirrhosis

Completed enrollment in POISE, its pivotal Phase III study testing

12/20/12

Isotechnika Pharma Inc. (Edmonton, Alberta) and Lux Biosciences Inc. (Jersey City, N.J.)

Voclosporin

Calcineurin inhibitor

Uveitis

Phase III failed to meet its endpoint of change from baseline in vitreous haze at 12 weeks or at the time of treatment failure, if earlier

12/28/12

Theravance Inc. (South San Francisco)

TD-9855

Lead monoamine reuptake inhibitor program

Fibromyalgia

Started a Phase II study with about 375 patients

12/6/12

Trimel Pharmaceuticals Corp. (Mississauga, Ont. )

CompleoTRT

Hormone replacement

Male hypogonadism

A Phase III trial met its primary efficacy endpoints and met FDA guidelines for testosterone replacement therapy after 90 days of dosing

12/7/12


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.

SPA = Special Protocol Assessment.