- 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.
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
|SOURCE PTC Therapeutics, Inc.|
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