PTC Says Primary Endpoint Not Statistically Significant In Phase 3 Trial

(RTTNews) - PTC Therapeutics, Inc. (PTCT) announced results from the Phase 3, double-blind, placebo-controlled, 48-week ACT DMD trial of Translarna (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy. The company noted that, in the overall intent-to-treat study population, the primary endpoint of change from baseline in the 6-minute walk test showed a 15 meter benefit, which was not statistically significant.

Based on this news, the company's stock declined 14 percent to $27.21 in the after-hours trading.

PTC Therapeutics stated that Translarna showed a benefit over placebo across key secondary and tertiary endpoints, including timed function tests and the North Star Ambulatory Assessment test. In addition, a pre-specified meta-analysis of the combined placebo-controlled ACT DMD and Phase 2b trials demonstrated a statistically significant benefit of Translarna across the primary and key secondary endpoints.

PTC intends to submit the results from the ACT DMD study to the EMA and to complete its rolling submission for a New Drug Application to the FDA by the end of 2015.