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Ataluren Phase 2 Data in Nonsense Mutation Cystic Fibrosis Published in the European Respiratory Journal

Fri, 07/01/2011 - 5:33am
Bio-Medicine.Org

SOUTH PLAINFIELD, N.J., July 1, 2011 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the publication of data from a Phase 2 study of ataluren, an investigational new drug, in adults with nonsense mutation cystic fibrosis (nmCF) in the European Respiratory Journal. The published three-month data showed that treatment with ataluren resulted in statistically significant improvements in chloride channel activity, CF-related cough and positive trends in lung function.

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"These encouraging data demonstrating statistically significant pharmacodynamic activity support the potential of ataluren for a significant subset of severely affected CF patients," stated Professor Michael Wilschanski, Director, Pediatric Gastroenterology, Hadassah University Hospital. "The results are important because they suggest that ataluren promotes the production of full-length, functional cystic fibrosis transmembrane conductance regulator (CFTR) protein and addresses the underlying cause of the disorder. Currently available treatments for cystic fibrosis only address symptoms, and new therapies such as ataluren are urgently needed."

Patients with CF lack adequate levels of the CFTR protein, a chloride channel necessary for the normal function of the lungs, pancreas, liver and other organs. In nmCF, an interruption in the genetic code—known as a nonsense mutation—prematurely halts the synthesis of CFTR, causing the protein to be short and non-functioning. Nonsense mutations are categorized as Class I mutations that result in little or no production of the CFTR protein. CF patients with Class I mutations typically experience more severe disease symptoms than those with lower-risk genotypes, including a greater than twofold increased risk of death(1), a higher probability of end-stage lung disease(2), and a higher prevalence of pancreatic insufficiency.(2) It is estimated that nonsense

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