PTC Therapeutics Announces Data from Additional Clinical Studies
June 12, 2008
PTC Therapeutics, Inc. today announced promising new data from two studies of PTC124 in cystic fibrosis. Results from an Israeli Phase 2a extension study evaluating three months of oral PTC124 treatment in adult patients with nonsense-mutation-mediated CF demonstrated statistically significant improvements in the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and a statistically significant mean [28%] decrease in the frequency of cough, one of the most prominent and burdensome CF-related symptoms.
Separately, results from a European study evaluating 14-day courses of PTC124 in pediatric patients with nonsense-mutation-mediated CF confirmed the CFTR activity observed in previous short-term studies in adult patients. PTC124's development is supported by grants from Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Foundation.
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