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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

oncomitant treatment with systemic aminoglycosides.

Patients with CF lack adequate levels of the CFTR protein, a chloride channel necessary for normal function of the lung, 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 other genotypes, including a shorter life span, a higher probability of end-stage lung disease, and a higher prevalence of pancreatic insufficiency. Ataluren, a protein restoration therapy, is designed to overcome the nonsense mutation and enable the production of a full-length, functional CFTR protein. A simple genetic test can determine if a patient's disease is caused by a nonsense mutation.

"PTC has a long-standing commitment to discovering and developing new treatments for rare, life-threatening disorders such as cystic fibrosis. We are very encouraged by the data from our trial, which was a long-term placebo-controlled study of a CFTR corrector targeting nonsense mutations," stated Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, Inc. "The results in the patients not on inhaled aminoglycosides are particularly promising. Substantial differences between ataluren and placebo in mean relative change in %-predicted FEV1 and pulmonary exacerbation rate were demonstrated and the fact that both endpoints improved is a strong indicator of ataluren's activity."

About the Phase 3 TrialThe primary objective of the double-blind, placebo-controlled study was to determine whether ataluren can improve lung function relative to placebo in patients with nonsense mutation cystic fibrosis, as measured by forced expiratory volume in 1 second (FE
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SOURCE PTC Therapeutics, Inc.
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