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PTC Therapeutics Announces Additional Positive Interim Phase 2,Results of PTC124 in Duchenne Muscular Dystrophy

al data, we plan to initiate longer-term clinical trials to evaluate the clinical benefit of PTC124 in patients with DMD."

The Phase 2 clinical trial is being conducted at three sites in the United States: Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; and the University of Utah, Salt Lake City, Utah. In the study, patients have received 28 days of PTC124 treatment at one of three dose levels. All clinical trial participants are boys with a nonsense mutation in the dystrophin gene, substantially elevated serum creatine kinase levels, and symptoms associated with DMD. The analysis presented today includes data from 26 patients with DMD who received PTC124 at the low-dose and medium-dose levels. Completion of accrual and analysis of data from a higher dose level are ongoing.

The primary endpoint of the trial has been the proportion of patients having an increase in dystrophin expression in muscle during 28 days of treatment with PTC124. Pre- and post-treatment muscle biopsies were available from all 26 patients for analysis. In vitro treatment of patient muscle cells with PTC124 showed evidence of a dose-dependent increase in dystrophin expression in all of the evaluable patients. Preliminary review of the data indicate that, at both dose levels evaluated in this analysis, approximately half of the patients demonstrated visible improvement in the staining for muscle dystrophin in vivo. Overall, four of the six, or 67 percent, of patients treated at the lower dose level and 10 of the 20, or 50 percent, of patients treated at the medium dose level demonstrated an increase in the expression of dystrophin post-treatment. Response did not appear to be dependent on type of nonsense mutation.

Additionally, statistically significant reductions in the concentrations of muscle-derived creatine kinase levels in the blood were observed during PTC124 treatment. Several parents a
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