Sarepta Therapeutics Inc. has set the wholesale acquisition cost of the first gene transfer therapy for ambulatory patients with Duchenne muscular dystrophy at $3.2 million, making it one of the most expensive gene therapies. The company said the gross-to-net price for Elevidys (delandistrogene moxeparvovec) will be in the mid-20% range, which, suggests Mizuho Group analyst Uy Ear, would put the price at about $2.4 million. Read More
Intercept Pharmaceuticals Inc.’s second attempt to score an expanded U.S. FDA approval of its farnesoid X receptor agonist, obeticholic acid, in patients with non-alcoholic steatohepatitis (NASH) went the way of the first, with the agency issuing another complete response letter (CRL), prompting the company to drop all NASH-related investment and cut a third of its workforce. Read More
Aslan Pharmaceuticals Pte Ltd. said it inked a licensing deal with Zenyaku Kogyo Co. Ltd. for its atopic dermatitis-targeting monoclonal antibody, eblasakimab, that could reach $138.5 million in up-front and milestone payments. Read More
A recent population study in Morocco reinforced the importance of early diagnosis in handling – at best physicians can – Prader-Willi syndrome (PWS) and served, at least indirectly, to remind investors of the potentially lucrative space, where the lone drug labeled for the indication is a growth hormone. Read More
IPSirius SAS, an early stage French immuno-oncology firm, hopes to obtain a clinical trial authorization from the U.K.’s Medicines and Healthcare Regulatory Products Agency next year, to enable it to move its novel therapeutic cancer vaccine into a first-in-human trial in patients with non-small-cell lung cancer. Read More
Japan’s Pharmaceutical and Medical Devices Agency has cleared Healios K.K. and Sumitomo Pharma Co. Ltd. to begin a phase I/II study of HLCR-011, which is composed of retinal pigment epithelial (RPE) cells derived from allogeneic induced pluripotent stem cells (iPS) in patients with RPE tear. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Acasti, Bavarian, Cimeio, Nephrodi, Prime, Zucara. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Atyr, Cidara, GSK, Kite, Kymera, Mindset. Read More
