PTC Therapeutics announced results from the Phase 3 ACT DMD trial of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

ACT DMD, the largest placebo-controlled study ever conducted in patients with DMD, is a multicenter, randomized, double-blind, Phase 3 clinical trial which enrolled 228 nmDMD patients between 7–16 years. Patients were randomized to receive either Translarna 40mg/kg/day (n=114) or placebo (n=114) over 48 weeks. The primary endpoint was change from baseline in the 6-minute walk test (6MWT). Analyses of data from pre-specified subgroups, including the pre-specified subgroup of patients with baseline 6-minute walk distance (6MWD) of 300–400 meters, were also completed.

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Study results showed clinically meaningful benefits for Translarna-treated patients. In the overall intent-to-treat population, change from baseline in the 6MWT demonstrated a 15 meter benefit (P=0.213) over placebo, which was not statistically significant. A highly significant benefit of 47 meters (P=0.007) was demonstrated in the pre-specified patient population of 300–400 meters at baseline as measured by the 6MWT, which is in line with PTC’s prior experience in its Phase 2b trial and consistent with the evolving understanding of the 6MWT. Importantly, no patients in the Translarna lost ambulation (0/47) vs. 4 patients in the placebo group (4/52). In addition, a pre-specified meta-analysis of the combined ACT DMD and Phase 2b trials demonstrated a statistically significant benefit of Translarna across the primary (P=0.015) and key secondary endpoints.

The FDA previously granted Translarna Orphan Drug Status for DMD, cystic fibrosis, Mucopolysaccharidosis I (MPS 1), and aniridia. PTC intends to complete its rolling submission for a New Drug Application (NDA) to the FDA by the end of 2015.

Translarna is an investigational, oral, first-in-class protein restoration therapy.

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