PTC Therapeutics Announces Additional Results from Phase 2 Study of PTC124 in Duchenne Muscular...( Data Presented at the World Musc...),Health

Data Presented at the World Muscle Society International Congress

SOUTH PLAINFIELD, N.J., Oct. 18 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs targeting post-transcriptional control mechanisms, today announced additional data from a Phase 2 clinical trial of PTC124 in patients with Duchenne muscular dystrophy (DMD) due to a nonsense mutation. The results, which include data from all three cohorts of the study, show that administration of PTC124 is associated with qualitative increases in muscle dystrophin expression and with reductions in serum creatine kinase values. These data were presented today at the World Muscle Society (WMS) International Congress in Taormina, Italy.

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Patients with DMD are boys and young men who lack dystrophin, a protein that is critical to the structural stability of muscle fibers. This Phase 2 multi-site, open-label, dose-ranging clinical trial enrolled 38 boys with loss of dystrophin due to a nonsense mutation in the dystrophin gene. Participants also had substantially elevated serum creatine kinase levels due to the disease, and symptoms associated with DMD. Boys enrolled in the trial received 28 days of PTC124 treatment at one of three dose levels, with the primary endpoint of the trial being an increase in dystrophin expression in muscle. Pre- and post-treatment muscle biopsies and blood analyses to assess muscle-derived creatine kinase were available from all 38 patients.

An in vitro analysis demonstrated PTC124-induced dystrophin expression in cultured muscle cells from all 35 (100%) of the boys with samples evaluable in this analysis. The in vivo data indicated that, across all three dose levels of PTC124, 18/38 (47%) of patients demonstrated visib
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