Despite mixed opinions regarding the breadth of the proposed label for Puma Biotechnology Inc.'s lead candidate, Nerlynx (neratinib), members of the FDA's Oncologic Drugs Advisory Committee ultimately voted 12-4 in favor of recommending that the agency approve the candidate as an extended adjuvant treatment for HER2-positive early stage breast cancer.
Biogen Inc. spin-off Bioverativ Inc. will pay $400 million up front plus assumed cash to acquire True North Therapeutics Inc., a privately held Ipierian Inc. spinout with an early stage FDA breakthrough-designated candidate for the rare blood disorder cold agglutinin disease (CAD).
FDA briefing documents framing a Wednesday Oncologic Drugs Advisory Committee (ODAC) review of Puma Biotechnology Inc.'s lead candidate, neratinib, highlight tolerability and trial amendment issues, but still appear supportive of the drug's efficacy as an extended adjuvant treatment for HER2-positive early stage breast cancer, analysts said. The news sent Los Angeles-based Puma's shares (NASDAQ:PBYI) up 39.2 percent in heavy trading Monday, to close at $52.60.
A surprise FDA approval allowing Vertex Pharmaceuticals Inc. to expand the label for its cystic fibrosis (CF) therapy, Kalydeco (ivacaftor), to include an additional 3 percent of the CF population with certain rare residual function mutations boosted forecast sales for the therapy and lent credence to the potential power of in vitro assays to sway the agency in certain cases.
Privately held Synlogic Inc., a company leveraging synthetic biology to genetically reengineer microbes, is going public via a reverse merger with troubled microRNA mimic pioneer Mirna Therapeutics Inc.
Disappointing interim trial data suggesting that Capricor Therapeutics Inc.'s lead candidate is unlikely to best a placebo in reducing scar size after heart attack dimmed expectations that Janssen Biotech Inc. would exercise its option to take an exclusive license on the cell therapy, sending Capricor's shares (NASDAQ:CAPR) down 62 percent to close at $1.16, near a 52-week low. The companies are still in active talks, said CEO Linda Marbán, but Capricor is already cutting its work force and reducing the scope of its operations to focus on applying the candidate, CAP-1002, to Duchenne muscular dystrophy (DMD)-associated cardiomyopathy, where recent trial data have been more encouraging.
Nantcell and Nantkwest Inc. have gained FDA permission to start enrolling a phase Ib/II trial of the Nant cancer vaccine, a step toward what Nantkwest CEO Patrick Soon-Shiong has said is a quest to develop a vaccine intended to induce immunogenic cell death and orchestrate patients' innate and adaptive immune systems.
A lack of 12-month endometrial safety data led to the FDA's rejection of an NDA filed by Therapeuticsmd Inc. for Yuvvexy, an investigational treatment of moderate to severe vaginal pain during sex. The complete response letter (CRL) for the 505(b)(2) application arrived on the heels of the regulator alerting the company to an unspecified deficiency in its application last month.
About three months after taking the wraps off a $41 million mezzanine financing, Rhythm Pharmaceuticals Inc. is kicking off a pivotal trial of its lead candidate, setmelanotide, for an ultra-rare genetic obesity disorder. The melanocortin-4 (MC4) receptor agonist will be tested in an open-label, single-arm phase III study evaluating its efficacy for the treatment of pro-opiomelanocortin (POMC) deficiency obesity, a condition estimated to affect between 100 and 500 patients in the U.S.
