Duchenne muscular dystrophy
After the markets closed last night, Prosensa issued an upbeat self-assessment of its 48-week extension data from a Phase II study of its lead drug drisapersen for Duchenne muscular dystrophy.
Patient groups are a potent force in the Duchenne muscular dystrophy field, where parents are known to take a vocal role in pushing regulators to help biotechs advance new therapies for the lethal genetic condition. Now one of those parents, J.P. Morgan vet Ilan Ganot, has reportedly been raising some serious cash to do deals on early-stage therapies that can be rushed through clinical development.
Shares of PTC Therapeutics took a hit this morning, dropping about 20% after the European Medicines Agency threw its considerable weight against the biotech's application to gain a conditional approval for its Duchenne muscular dystrophy drug ataluren.
Prosensa's Duchenne muscular dystrophy treatment endured a Phase III setback that led partner GlaxoSmithKline to head for the exit, but the Dutch biotech is back with a new analysis of drisapersen's data that could spell a path forward, sending its shares up 25%.
Sarepta's up-and-down quest to get eteplirsen in the hands of patients with Duchenne muscular dystrophy is looking a little sunnier thanks to more promising data and an optimistic reading of some FDA tea leaves, news that sent the biotech's shares up 35% on Thursday.
GlaxoSmithKline has shut the door on its hopes in Duchenne muscular dystrophy, handing back rare-disease drug drisapersen to Prosensa, its former partner.
Shares of Sarepta plunged 64% by the end of the day Tuesday, wiping out hundreds of millions of dollars of market valuation in a brutal reaction to the company's admission that the FDA had closed the door to an early approval of eteplirsen, its promising mid-stage therapy for Duchenne muscular dystrophy
In what has been one of the most hotly contested issues among biotech investors this year, many had bet big that the FDA would push through an accelerated approval for eteplirsen based on some very promising results from a mid-stage study. The study, though, only included a dozen boys, making the prospect of an early approval problematic at best.
John Kraus, GSK's medical leader for drisapersen, unveiled more data from the failed study on the exon-skipping technology, which fell short not only on its primary endpoint for improved walking distances in a 6-minute test but also flunked out on three separate secondary endpoints, with the outcomes looking almost identical in each case with a placebo.
GlaxoSmithKline and Prosensa today conceded defeat in the Phase III study for drisapersen, one of two closely-watched therapies for Duchenne muscular dystrophy which had been vying for the lead in the field. Investigators reported that the therapy failed to significantly improve walking distance in patients.