PTC Therapeutics Announces Encouraging Additional Phase 2 Results of PTC124 in Cysti...( - New Pediatric Data Presented at ...)

- New Pediatric Data Presented at 21st North American Cystic Fibrosis

Conference Confirm Previous Findings in Adult population -

SOUTH PLAINFIELD, N.J., Oct. 5 /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 encouraging data from a Phase 2 clinical trial of PTC124 in pediatric patients with cystic fibrosis (CF) due to a nonsense mutation. These pediatric results and additional information emerging from long-term studies support the existing data from prior short- term studies in adult CF patients. These studies show that treatment with PTC124 results in statistically significant improvements in a measure of the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. These data were highlighted today in a plenary session entitled "CF Drug Development: What's New?" given by Dr. Felix Ratjen, University of Toronto Professor of Pediatrics and Respiratory Medicine Division Chief, at the 21st North American Cystic Fibrosis Conference in Anaheim, California.

(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )

Patients with CF lack the CFTR protein, a chloride channel that maintains proper hydration of epithelial cells in the lung, pancreas, and liver. PTC has completed multi-site, open-label, dose-ranging Phase 2 clinical trials in adult CF patients to determine whether PTC124 can induce production of active CFTR protein. Studies in the U.S. and Israel evaluated nasal transepithelial potential difference (TEPD) as a surrogate for CFTR protein production in adult CF patients. Across the two studies, at both PTC124 dose levels tested, TEPD assessments showed statistically significant (p<0.03) improvements of mean CFTR-dependent chloride secretion i
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