PTC Therapeutics and Genzyme Corporation Announce Preliminary Results from the Phase 2b Clinical Trial of Ataluren

05-Mar-2010 - USA

PTC Therapeutics, Inc. and Genzyme Corporation announced preliminary results from the Phase 2b clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD). The primary endpoint of change in 6-minute walk distance did not reach statistical significance within the 48-week duration of the study. Study results showed that ataluren was well tolerated and no clinical trial patients discontinued treatment due to an adverse event. Additional efficacy analyses are underway in patient subgroups.

“These results further demonstrate the safety profile of ataluren and support continued development,” remarked Langdon Miller, M.D., PTC’s chief medical officer. “DBMD is a progressive, debilitating, and life-threatening neuromuscular disorder. The variability of symptom onset, disease progression, and 6-minute walk distance creates challenges for clinical development. Importantly, this trial does provide a wealth of valuable data about ataluren and DBMD. Additional analyses will guide the overall clinical and regulatory path forward.”

Ataluren is also currently being investigated for use in patients with nonsense mutation cystic fibrosis in a Phase 3 study and nonsense mutation hemophilia A and B in a Phase 2a study. Ataluren’s mechanism of action offers the potential to address multiple genetic disorders with various pathophysiologies and disease manifestations.

The randomized, double-blind, placebo-controlled Phase 2b trial was designed to evaluate the safety and efficacy of 48 weeks of ataluren therapy in patients with nmDBMD. The study enrolled 174 participants at 37 sites in North America, Europe, Australia, and Israel. Participants were randomized to receive either a low dose of ataluren (10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening), a high dose of ataluren (20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening), or placebo (inactive drug in the morning, at midday, and in the evening). The primary outcome measure was the total distance walked during a 6-minute walk test, a standardized test of ambulation. Other outcome measures in the study evaluated activity at home, muscle and heart function, strength, cognitive ability, muscle integrity, and muscle dystrophin expression. Safety parameters, compliance, and ataluren blood levels were also monitored.

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