PTC data shows Translarna works in broad DMD population

PTC may finally have the dataset it needs to convince U.S. regulators of Translarna’s efficacy in DMD after a decade of trying, even as the small molecule missed the primary endpoint in the primary analysis population in Phase III testing. 

PTC Therapeutics Inc. (NASDAQ:PTCT) announced Tuesday that Translarna ataluren showed a statistically significant improvement in the six-minute walk test (6MWT) compared with placebo in an analysis of the intent-to-treat (ITT) population of the 359-patient Phase III Study 041 trial to treat Duchenne muscular dystrophy patients with nonsense mutations. In the broad ITT population, Translarna showed a 14.4-meter improvement vs. placebo (p=0.0248). ...