PTC124 enters Ph II DMD trial

The USA's PTC Therapeutics has initiated a Phase II study of its drug candidate PTC124 in patients with Duchenne muscular dystrophy due to a nonsense mutation. The novel, orally-administered agent targets nonsense mutations and is being investigated initially as a treatment for DMD and cystic fibrosis, with the potential to treat a number of other genetic disorders caused by nonsense mutations.

The Phase II clinical study is enrolling patients who have DMD due to a nonsense mutation in the dystrophin gene. The primary endpoint of this multicenter, open-label, dose-ranging clinical study is assessment of muscle dystrophin expression in response to treatment.