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.
Drisapersen and eteplirsen have been angling to become the first approved therapy for lethal, muscle-wasting cases of Duchenne muscular dystrophy. So when investigators pulled back the covers from a new block of data on GlaxoSmithKline's drisapersen, the analysts immediately went to work to see how it stacked up against Sarepta's eteplirsen.
With its shares fattened by speculation of a possible accelerated approval path for its closely-watched eteplirsen, an experimental treatment for Duchenne muscular dystrophy, Sarepta Therapeutics reports that it is preparing to sell up to $125 million in shares.
Shares of Sarepta were boosted this morning after the biotech announced that it reaped another round of promising results from a closely-watched Phase IIb study of its experimental treatment for Duchenne muscular dystrophy.
GlaxoSmithKline investigator John Kraus reviewed the long-awaited mid-stage results for its Duchenne muscular dystrophy drug drisapersen this afternoon, revealing that boys taking the experimental therapy benefited with a significant improvement in walking distance compared to a placebo arm.
After keeping its mid-stage data for a closely-watched and controversial drug for Duchenne muscular dystrophy under wraps, GlaxoSmithKline has pulled back part of the covers to reveal that drisapersen did in fact hit the primary endpoint on walking distance.
Sarepta Therapeutics spotlighted continued benefits of treatment with its lead drug eteplirsen today in one of the most closely watched Phase IIb studies in the biotech industry.