The clock is ticking for Sarepta Therapeutics ($SRPT), a multitude of biotech investors and the boys who suffer from Duchenne muscular dystrophy.
|CEO Chris Garabedian|
Armed with promising Phase IIb data from a small study of eteplirsen involving only 12 patients with the lethal disease, Sarepta CEO Chris Garabedian is completing one of the most closely-watched high wire acts in the industry. At a time most companies would be focused solely on organizing a pivotal late-stage study, there's intense speculation that the biotech will shoot for an accelerated approval with the data in hand. And it all comes down to their sit-down with the FDA to review mid-stage data.
The prospect of a quick march to the market with a pioneering drug like this--a Barron's article noted a potential $500 million market in the U.S. alone--has whet the appetite of investors who have bid up the little company's shares by 281% over the past year. And it's also captured the attention of drug developers looking for some fresh signs on the FDA's evolving attitude toward the data needed to prove the risk/benefit profile of a new chemical entity.
Sarepta's swelling stock price--$36.12 at yesterday's close--will either pop up or instantly deflate once Sarepta makes its move, at least according to Barron's take over the weekend. The difference between a go or no-go decision: A stock price of $50 or the $20s. A decision to go for it now would indicate that the FDA is willing to seriously consider a much smaller dataset than it would normally not take a second look at. And with regulators vowing a commitment to "breakthrough" drugs for medical needs crying out for some kind of therapy, at a time rare disease drugs are attracting growing attention from developers, the time might be right.
This is, of course, the same FDA that refused to even consider mid-stage data on Kadcyla (T-DM1), the newly approved breast cancer therapy from Genentech that went on to score an approval with stellar pivotal data. So the enthusiasm about possible encouragement from the regulators is tempered by a long track record of decisions that would suggest otherwise. And that's why a crowd has been gathering on Twitter and other social media spots to watch the spectacle.
Sarepta's Garabedian says only that it will make a decision after it sits down with the FDA by the end of this month--just days away--to look over its results and discuss the next step forward. "We have not disclosed when we plan to announce our post-FDA meeting feedback," Garabedian noted in a recent e-mail to FierceBiotech, repeating earlier guidance. "We've simply indicated it would take place after we received meeting minutes or approximately 30 days after the actual meeting."
Sarepta has been engaged in a race with pharma giant GlaxoSmithKline ($GSK), which licensed in rights for another DMD drug from privately-held Prosensa, which also once considered a mid-stage application. But the highly charged showdown has only inspired more debate about Sarepta's chances. Some analysts, including Leerink Swann's, say that in the long run the decision now won't matter so much.
But these opinions only attract more attention for the big announcement that's looming.
- here's the Reuters' story