Company |
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 |
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. |