Sarepta Therapeutics Inc. has set the wholesale acquisition cost (WAC) of the first gene transfer therapy for ambulatory patients with Duchenne muscular dystrophy (DMD) at $3.2 million. The gross-to-net price for Elevidys will be in the mid-20% range, which, suggests Mizuho Group analyst Uy Ear, would put the price at about $2.4 million. The WAC is largely in line with other gene therapy pricing, Ear added, which ranges from about $2.8 million to $3.5 million, with CSL Behring LLC’s Hemgenix at the high end of the range. The U.S. FDA granted accelerated approval to the therapy June 22, making it Sarepta’s fourth approved treatment for DMD and the first to offer patients a one-time treatment option. The company stock (NASDAQ:SRPT) was down about 12% at midday to $109 per share.

Intercept quits NASH on second CRL

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 nonalcoholic steatohepatitis (NASH) went the way of the first, with the agency issuing another complete response letter, prompting the company to drop all NASH-related investment and cut a third of its workforce.

Aslan signs $138.5M licensing deal with Zenyaku for atopic dermatitis drug eblasakimab in Japan

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. Under the agreement, privately held Zenyaku gains exclusive rights to develop and commercialize eblasakimab for atopic dermatitis and all other indications in Japan. In turn, Aslan receives an all-cash, up-front payment of $12 million, and is eligible for an additional $3 million cash payment if it reaches pre-agreed conditions for its ongoing phase II TREK-AD trial. Based on TREK-AD and continuing readouts, Aslan can receive up to $29.5 million in development milestones along with $94 million in commercial milestones – totaling $123.5 million. Aslan can also exercise an option to reacquire rights to eblasakimab in Japan at any time, allowing for partnership switches down the line.

Innovative efforts crowding Prader-Willi development space

Drug developers continue to hustle in Prader-Willi syndrome (PWS), where patients afflicted with the genetic disorder have long needed a better therapy. Among players gaining notice lately in PWS are Soleno Therapeutics Inc. and Harmony Bioscience Holdings Inc., but plenty more companies own prospects that could help the condition. PWS strikes an estimated one in 10,000 to 30,000 people worldwide.

Newco news: IPSirius targets cancer stem cells with iPSC-based vaccine

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. The company, a spinout from Inserm, France’s national institute for health and medical research, and Saclay University in Paris, is exploiting an antigenic overlap between induced pluripotent stem cells (iPSCs) and cancer stem cells, by developing a cancer vaccine consisting of irradiated iPSCs. “It’s basically a delivery system for cancer targets,” chief operating officer Stephen Sullivan told BioWorld.

Healios, Sumitomo begin phase I/II trial of allogeneic iPS cells in retinal pigment epithelium tears

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. Healios provided its universal donor cells to Sumitomo under a research agreement back in 2013 to advance the development of therapies using iPS cell-derived RPE cells for age-related macular degeneration and other eye diseases.

Also in the news

Acasti, Acrivon, Altpep, Alzamend, Apogee, Aptorum, Atyr, Bavarian Nordic, Bayer, Bicycle, Biocity, Calidi, Calliditas, Cidara, Cimeio, Compugen, Disc Medicine, GSK, Horizon, Hyloris, Ideaya, Inhibikase, Inmed, Invivyd, Jasper, Kite, Kymera, Madrigal, Memo, Mindset, Nephrodi, Palleon, Parexel, Polypid, Prime Medicine, PTC, Regulus, Relief, Reviva, Roivant, Seelos, Sellas, Tvardi, Vaxxinity, Xoma, Zucara