Bellerophon Gets Clearance to Commence Mid-Stage Study of INOpulse- Bellerophon Therapeutics (BLPH) shares edged higher on Monday after the company announced that Belgian health authorities have approved the commencement of an open-label Phase 2 study evaluating INOpulse for the treatment of pulmonary hypertension in patients with chronic obstructive pulmonary disease (PH-COPD). Patient recruiting (10 total) will start this quarter with results expected by year end. BLPH shares rose as much as 35% in pre-market trading on Monday but gave up most of their gains in regular trading to close just under 2% higher.
Tobira’s Lead Product Candidate Fails in Mid-Stage Study- Tobira Therapeutics (TBRA) shares plunged 60% on Monday after the company announced that its lead product candidate cenicriviroc (CVC) failed to achieve its primary endpoint of a two-point reduction in NAFLD Activity Score in a Phase 2b clinical trial, CENTAUR, in patients with non-alcoholic steatohepatitis (NASH). The company did not though that the study showed a clinically and statistically significant improvement in fibrosis of at least one stage without worsening of NASH, a secondary endpoint, after one year of treatment (p=0.02). TBRA noted that achieving a two-point reduction in NAFLD Activity Score is not required to support a marketing application. The two endpoints needed are an improvement in fibrosis by at least one stage without worsening of NASH and complete resolution of NASH without worsening of fibrosis. TBRA plans to meet with regulators later this year to discuss the design of a Phase 3 trial.
Ocular’s Dextenza NDA Rejected- Ocular Therapeutix (OCUL) announced the receipt of a Compete Response Letter (CRL) from the FDA for its New Drug Application (NDA) seeking approval of DEXTENZA (dexamethasone insert) 0.4 mg for intracanalicular use in the treatment of ocular pain following ophthalmic surgery. The CRL cited deficiencies in manufacturing process and controls observed during an on-site inspection of the company's manufacturing facility. The company says the agency did not identify which deficiencies remain open since its last written response to regulator. No safety or efficacy issues related to DEXTENZA are involved.
Clinical Hold on Novartis’ CGF166 Lifted- Novartis (NVS) announced that the FDA has lifted its clinical hold on the company’s Phase 1/2 study evaluating CGF166 in patients with severe-to-profound hearing loss after the Data Safety and Monitoring Board recommended that the trial continue. The FDA stopped the trial in January of this year in accordance with criteria in the study's protocol that required a review of the results. Top-line data should be available in 2017.
Soligenix Obtains Additional Funding- Soligenix (SNGX) announced the receipt of $634,000 in new funding o support the development of OrbeShield (oral beclomethasone 17,21 dipropionate or BDP) as a medical countermeasure for civilian and military use to treat gastrointestinal acute radiation syndrome (GI ARS). The Biomedical Advanced Research and Development Authority (BARDA) chipped in $350K while NIH's National Institute of Allergy and Infectious Diseases (NIAID) contributed $284K. Total funding from the two groups will be as high as $33M if all contract options are exercised ($18M has been committed thus far).
PTC Therapeutics Provides Regulatory Update- PTC Therapeutics (PTCT) provided a regulatory update on its Translarna. The company said that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) informed PTCT that its renewal assessment procedure for the Translarna (ataluren) marketing authorization will not be completed by mid-year as expected, although the company believes that the current marketing authorization to remain in effect until a decision is announced by the European Commission (EC). PTCT said that is currently recruiting patients in an open-label 10-subject Phase 2 clinical trial assessing Translarna in boys aged 2 to 5 with DMD.
EPIRUS Files For Bankruptcy- EPIRUS Biopharmaceuticals (EPRS) announced that it has filed for protection under Chapter 7 of the U.S. Bankruptcy Code. In accordance with the provisions, the company's assets will be liquidated and claims paid with the proceeds.
Celgene’s Late-Stage Study of REVLIMID Fails- Celgene (CELG) announced that it will not seek a new indication for Revlimid (lenalidomide) as maintenance therapy in patients with diffuse large B-cell lymphoma (DLBCL) who responded to first-line rituximab (Roche's Rituxan) plus R-CHOP chemo. The company's decision was based on the results from a randomized Phase 3 study, REMARC, that showed a statistically valid improvement in progression-free survival in the treatment group versus placebo, but no difference in overall survival.
Viking Announces Encouraging Results for VK0214 From Preclinical Studies- Viking Therapeutics (VKTX) shares have surged more than 11% in pre-market trading today after the company announced positive preclinical results for product candidate VK0214 in a rare nervous system disorder called X-linked adrenoleukodystrophy (X-ALD), a disease caused by the deterioration of fatty covering of nerves (myelin) which reduces the ability of the nerves to relay information to the brain. It also damages the outer layer of the adrenal glands which leads to the shortage of certain hormones (adrenocortical insufficiency). It occurs in ~1 in 17,000 births. The results were generated in a proof-of-concept mouse model of X-ALD. VK0214 rapidly reduced plasma very long chain fatty acid (VLCFA) levels by more than 25% compared to controls after six weeks of treatment. VLCFAs are elevated in X-ALD sufferers due to metabolic defects.