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.
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.
The clock is ticking for Sarepta, a multitude of biotech investors and the boys who suffer from Duchenne muscular dystrophy.
PTC Therapeutics has scored new financing to bankroll development of a compound for certain types of Duchenne muscular dystrophy and cystic fibrosis.
Shares of Sarepta--already swelled by the positive response that followed upbeat results from its early-stage study of a new therapy for Duchenne muscular dystrophy--climbed sharply this afternoon after TheStreet's biotech analyst raised alarms about a series of adverse events recorded during clinical studies of a competing therapy in late-stage development by GlaxoSmithKline.
The former Genzyme CEO and chairman has joined the biotech Prosensa as a strategic adviser, bringing his experience in building Genzyme into a world leader in rare diseases.
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.