PTC Therapeutics Announces Data from Additional Clinical Studies of PTC124 in Cystic Fibrosis Confirmin...( - Data Presented at the 31st Eur...),Health

- Data Presented at the 31st European Cystic Fibrosis Conference -

SOUTH PLAINFIELD, N.J. and PRAGUE, Czech Republic, June 12 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced promising new data from two studies of PTC124 in cystic fibrosis (CF). 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. Results from the Hadassah University Hospital in Israel were presented today by Michael Wilschanski, M.D., Director, Pediatric Gastroenterology, and by Eitan Kerem, M.D., principal investigator and head of the Department of Pediatrics and Cystic Fibrosis Center. 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. Data from the European study were presented today by Isabelle Sermet-Gaudelus, M.D., Ph.D., principal investigator at l'Hopital Necker-Enfants Malade.

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Patients with CF lack adequate levels of the CFTR protein, a chloride channel that maintains proper hydration of epithelial surfaces in the lung, pancreas, and liver. Patients with nonsense-mutation-mediated CF generally have a severe form of CF because virtually no CFTR protein is produced. Previous studies of PTC124 adult patients with CF evaluated nasal transepithelial potential difference (TEPD) as a surrogate for the presence and activity of the CFTR protein. Across the short- and long-term
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