PTC's Translarna submission ongoing despite Phase III miss

PTC Therapeutics Inc. (NASDAQ:PTCT) gained $3.26 (10%) to $33 in after-hours trading Thursday after it reported data from the Phase III ACT DMD study of Translarna ataluren to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). The company said Translarna missed the study's primary endpoint, but said the "totality of clinical data" support Translarna's clinical benefit, and it plans to complete submission of a rolling NDA by YE15.

PTC said patients in the study's intent-to-treat population receiving Translarna showed a 15-meter benefit in a six minute walk test (6MWT), the trial's primary endpoint. The results were not statistically significant (p=0.213). The 228-patient study evaluated patients after 48 weeks. ...