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.
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.
The Cambridge, MA-based biotech company plans to file an early application for the RNA drug to treat Duchenne muscular dystrophy, a rare muscle-wasting disease that robs mostly boys of their ability to walk.
Prosensa picked the right day to price its IPO. The Dutch biotech rang up the offering at $13 a share, at the top of the range, raising $78 million after bumping the number of shares on offer to 6 million. The stock will trade on Nasdaq as "RNA."
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.
Sarepta Therapeutics has kept hopes alive for gaining accelerated approval for its lead drug eteplirsen for Duchenne muscular dystrophy.
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.
For most biotechs, the sit-down with the FDA after a positive Phase II study is a fairly routine affair designed to point them down the path to a pivotal program. For Sarepta Therapeutics, though, it represents a strategic crossroad rarely seen in this industry. In one direction lies a lengthy Phase III trial, while the other path points straight to a major new market for Duchenne muscular dystrophy, a terrible affliction faced by a small group of boys.
PTC Therapeutics has scored new financing to bankroll development of a compound for certain types of Duchenne muscular dystrophy and cystic fibrosis.