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PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements in Chloride Channel Function in Cystic Fibrosis Patients
Date:10/24/2008

- PTC124 Highlighted in Multiple Presentations at North American Cystic Fibrosis Conference -

SOUTH PLAINFIELD, N.J. and ORLANDO, Fla., Oct. 24 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced that results from a randomized Phase 2a European study demonstrated that treatment with the investigational drug PTC124 caused statistically significant improvements in the chloride channel function of children with cystic fibrosis (CF) caused by a particular genetic mutation, called a nonsense mutation. Results from the study were presented today by Isabelle SermetGaudelus, M.D., Ph.D., principal investigator at l'Hopital Necker-Enfants Malade. In addition, results from an Israeli Phase 2a study demonstrated statistically significant improvements in the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and a statistically significant decrease in the frequency of cough, one of the most prominent and burdensome CF-related symptoms. Data from the Israeli study were presented by Eitan Kerem, M.D., principal investigator and head of the department of pediatrics and Cystic Fibrosis Center at Hadassah University Hospital.

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

PTC124 is also being featured in a plenary presentation given by Preston Campbell, III, M.D., Executive Vice President of Medical Affairs at the Cystic Fibrosis Foundation, on promising developments in CF research.

"We are very pleased by the data continuing to emerge from the Phase 2 clinical development program for PTC124 in CF," noted Dr. Campbell. "Our alliance with PTC and the development of PTC124 are key elements of our strategic work supporting novel therapeutic approaches with the potential to modify the course of CF. We look forward to continuing our work with PTC to move PTC124 to the next stage of development, including pivotal clinic
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SOURCE PTC Therapeutics, Inc.
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