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PTC124 Shows Promising Activity in Cystic Fibrosis; Phase 2 Proof-of-Concept Data Published in the Lancet
Date:8/20/2008

atients were assessed in two 14-day treatment courses of oral PTC124 therapy, the first given at a lower dose and the second given at a higher dose. Results showed that at both dose levels, treatment with PTC124 was associated with statistically significant improvements (p<0.05) in CFTR-mediated chloride transport with over half of the patients entering the normal range during at least one treatment course. PTC124 induced chloride transport responses and normalization of CFTR activity across the variety of patient genotypes tested. Improvements in lung function values and body weight were also observed. PTC124 was generally well tolerated and all patients had >90 percent treatment compliance.

"This study demonstrates the potential for personalized medicine, combining selection of patients with a specific type of genetic mutation and a drug treatment that has been specifically designed to overcome that mutation," said Eitan Kerem, MD, head of pediatrics and the CF center at the Hadassah University Hospital in Mount Scopus, Jerusalem and the lead author of the study. "The publication of these ground-breaking results in the Lancet offers new hope to those patients with CF due to a nonsense mutation in the CFTR gene and establishes a path forward for evaluating the efficacy and long-term safety of PTC124."

"We are very pleased by this positive outcome from our ongoing collaboration with PTC," noted Preston Campbell, III, M.D., Executive Vice President of Medical Affairs at the Cystic Fibrosis Foundation. "The development of PTC124 fits well with our strategic goal of supporting approaches that have the potential to modify the course of CF. We are continuing to work together with PTC and the broader CF medical community to support the next steps in the evaluation of PTC124 for the clinical benefit for the treatment of nonsense-mutation CF."

"The publication of these data provides clinical proof of concept in CF for the PTC124 mechanism of action in o
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SOURCE PTC Therapeutics, Inc.
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