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Ataluren Phase 2 Data in Nonsense Mutation Cystic Fibrosis Published in the European Respiratory Journal
Date:7/1/2011

mutations are the cause of CF in about 10% of patients. Ataluren, a protein restoration therapy, is designed to overcome the nonsense mutation and enable the production of a full-length, functional CFTR protein. A genetic test can determine if a patient's disease is caused by a nonsense mutation.

"The results from this study strongly support the conduct of longer term studies to evaluate the clinical benefit of ataluren for patients with nonsense mutation cystic fibrosis," said Robert Spiegel, M.D., Chief Medical Officer of PTC Therapeutics. "A 48-week placebo-controlled Phase 3 study of ataluren in nonsense mutation cystic fibrosis is fully enrolled and we look forward to the availability of data in the first half of 2012."

ABOUT THE PHASE 2 EXTENSION STUDY

The Phase 2 clinical trial (Study 005e) was an extension of a previously conducted short-term open-label Phase 2a proof-of-concept trial (Study 005) of ataluren in adults with nmCF. Study 005e enrolled 19 patients, aged 19 years and older, who received one of two ataluren doses three times per day (morning, midday and evening) for 12 weeks (84 days). On-treatment clinic visits were conducted every 28 days, with an additional follow-up clinic visit at Day 112. The study was conducted at the Hadassah Hebrew University Hospital in Jerusalem, Israel and sponsored by PTC Therapeutics.

The study's primary endpoint was CFTR chloride channel activity as assessed by nasal transepithelial potential difference (NPD). A response was predefined as an improvement of at least -5.0 mV from baseline in total chloride transport. The results showed that ataluren induced statistically significant improvements in chloride channel activity at each dose level and for all patients combined. The aggregate mean change in total chloride transport for all patients was -5.4 mV (p<0.001).

Secondary endpoints of lung function included evaluations of forced expiratory volume in 1 second (FEV1) and
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SOURCE PTC Therapeutics, Inc.
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