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Top-line Data From Phase 3 Trial of Ataluren in Patients with Nonsense Mutation Cystic Fibrosis Show Promising Results
Date:6/8/2012

SOUTH PLAINFIELD, N.J., June 8, 2012 /PRNewswire/ -- PTC Therapeutics, Inc. today announced the results from a Phase 3 study of ataluren, an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF). The results demonstrated positive trends in lung function as measured by FEV1 (forced expiratory volume in one second) and in the secondary outcome measure, rate of pulmonary exacerbations. Results also showed a substantial ataluren treatment effect in FEV1 and exacerbation rate in the pre-specified subpopulation of patients not receiving chronic inhaled antibiotics, representing approximately one half of the study population. These data were presented at the European Cystic Fibrosis Society Conference in Dublin, Ireland.

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"The data from this trial are promising. Ataluren has the potential to provide benefit to patients with nonsense mutation cystic fibrosis, who currently have few treatment options. Nonsense mutations do not produce any functional CFTR protein and therefore generally result in a more severe form of cystic fibrosis," stated Michael Konstan, M.D., a principal investigator at University Hospitals Rainbow Babies and Children's Hospital in Cleveland, Ohio. "Correcting a nonsense mutation in the cystic fibrosis transmembrane conductance regulator gene is a big challenge. Over 48 weeks, the decrease in FEV1 in ataluren was 50% less than the decrease on placebo, which is a substantial difference. It is also very important to note that the FEV1 and exacerbations tracked closely." 

This Phase 3 study, which was conducted across 11 countries, was a double-blind, placebo-controlled study comparing ataluren (n=116) to placebo (n=116) in nmCF patients. The primary endpoint, the relative change from baseline in %-predicted FEV1 at 48 weeks, showed a positive trend favoring atalur
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SOURCE PTC Therapeutics, Inc.
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