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.
Sarepta Therapeutics has seen demand for its stock spike since the company revealed impressive long-term data two months ago from a mid-stage study of its therapy eteplirsen in patients with Duchenne muscular dystrophy.
Among a small group given the highest dose of eteplirsen, patients were able to outpace the placebo arm by 89.4 meters in a six-minute walk test.
Score another win for Boston in the high-stakes game of developing a thriving biotech hub. Xconomy' s Luke Timmerman has the scoop this morning that the promising Seattle-based biotech Sarepta is making the big move to Cambridge, MA.
Bothell, WA-based Sarepta's shares were up 14.29% to $14.40 per share at the close of trading on Thursday.
It took a few extra months of treatment, but a group of patients in a key Phase IIb study of Sarepta Therapeutics' (formerly AVI) Duchenne muscular dystrophy therapy eteplirsen registered much better walking distance compared to a placebo arm.