PTC Therapeutics Announces Additional Positive Interim Phase 2 Results of PTC124 in Duchenne Muscular Dystrophy
BOSTON and SOUTH PLAINFIELD, N.J., May 4 -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control processes, today announced positive interim data from a Phase 2 clinical trial of PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The results from the first two cohorts of the three-cohort study show that treatment with PTC124 was associated with increases in muscle dystrophin expression and reductions in serum creatinine kinase values in at least 50 percent of evaluable patients. These data were presented today at the 59th American Academy of Neurology (AAN) Annual Meeting.