Partnering Door Opens For Sarepta With 48-Week Data In Hand

Sarepta Therapeutics’ positive data on its lead muscular dystrophy drug eteplirsen puts the company in a strong position at the partnering table and sets the stage for a neck and neck race with GSK/Prosensa to bring the first treatment for the rare disease to market.

Sarepta Therapeutics Inc. is ready to begin partnering negotiations for its lead drug eteplirsen – in development for the treatment of Duchenne muscular dystrophy – now that it has released positive 48-week data from the extension phase of a Phase IIb trial showing an unprecedented clinical improvement in patients with the rare debilitating disease.

The positive results, which demonstrate the most clinically meaningful benefit yet reported in patients treated with a mid-stage DMD drug,...