Translarna Phase 2b Data Published In Muscle & Nerve

PTC Therapeutics, Inc.

today announced the publication of data and related analysis which provide strong support for concluding that Translarna™ (ataluren) was active and showed clinically meaningful improvements in Translarna-treated patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). The data was published in the October issue of Muscle & Nerve. Translarna received marketing authorization in the European Union in August 2014 for the treatment of ambulatory nmDMD patients five years and older. The data demonstrated that from baseline to Week 48 nmDMD patients treated with Translarna (40 mg/kg/day given in three doses) had a 31.3 meter benefit in six minute walk distance (6MWD) relative to patients in the placebo group (post hoc p=0.056). Though having achieved the targeted treatment effect, the results did not achieve significance at the p