Privately held Neurovance Inc. struck a potential $250 million acquisition deal with Japanese pharma giant Otsuka Pharmaceutical Co. Ltd. on the basis of a single asset in attention-deficit hyperactivity disorder (ADHD).
The troubled development program for ataluren took another turn for the worse when the phase III Ataluren Confirmatory Trial (ACT CF) in nonsense mutation cystic fibrosis (nmCF) fell short across the board, missing both primary and secondary endpoints. Developer PTC Therapeutics Inc. said it was halting development of ataluren in CF.
Lexicon Pharmaceuticals Inc. plans to deploy its sales force Friday and have its first approved product, Xermelo (telotristat ethyl, previously LX-1032/LX-1606), available Monday for dispensing through its specialty pharmacies following the oral drug's approval by the FDA to treat carcinoid syndrome diarrhea in patients living with metastatic neuroendocrine tumors, or mNETs.
As the biopharma industry paused on Rare Disease Day to assess its progress and consider the plight of patients still waiting for therapeutic options, two players at opposite ends of the U.S. took the field in Duchenne muscular dystrophy (DMD). Boston-based Exonics Therapeutics Inc. is plying CRISPR/Cas9 gene editing technology with a goal of correcting the mutations that cause DMD and other neuromuscular diseases.
As the industry recognizes Rare Disease Day Tuesday, there's plenty to celebrate. The acquisition of Baxalta Inc. by Shire plc early in 2016 created a global giant with an equally large appetite for therapies to treat rare indications.
About a year after starting to work, albeit indirectly, with the vaccine platform developed by Affinivax Inc., Astellas Pharma Inc. was sufficiently impressed to come back for seconds. The companies inked a global license arrangement for Affinivax's lead vaccine candidate, targeting Streptococcus pneumoniae, that included $10 million in up-front funding.
Merck and Co. Inc. isn’t exactly ditching its hepatitis C virus (HCV) NS5B polymerase inhibitor, known as uprifosbuvir (MK-3682/3682B) – for now, at least. Enrollment continues in ongoing trials of uprifosbuvir – in development in triple- and double-combination HCV regimens, company spokeswoman Lainie Keller told BioWorld Today.
After several years incubating in the Centre for Probe Development and Commercialization (CPDC) at McMaster University in Hamilton, Ontario, Fusion Pharmaceuticals Inc. is hanging out its shingle.
Jeff Abbey, president and CEO of Argos Therapeutics Inc., needed all of three minutes to deliver the news that the company was stopping the pivotal phase III ADAPT trial of lead candidate rocapuldencel-T (roca, formerly AGS-003) for futility on the advice of the independent data monitoring committee (IDMC). Abbey did not take questions, and the company said it had nothing to add to a brief statement issued minutes before the call.
Trevena Inc. reported top-line results from its pivotal phase III APOLLO-1 and -2 efficacy studies of lead candidate oliceridine (previously TRV-130) in moderate to severe acute pain following bunionectomy and abdominoplasty (commonly known as a tummy tuck), respectively, showing that all dose regimens across both trials achieved the primary endpoint of statistically greater analgesic efficacy than placebo, as measured by responder rate. Oliceridine, branded Olinvo, also showed dose-related trends of improvements vs. morphine on multiple measures of respiratory safety and gastrointestinal tolerability in acute pain management.
