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PTC Therapeutics Initiates Registration-Directed Phase 3 Trial of Ataluren in Cystic Fibrosis
Date:9/10/2009

rch centers in North America, Europe, and Israel. Study candidates include patients who are at least six years of age and have CF due to a nonsense mutation.

"Promising Phase 2 clinical trial data show that patients treated with ataluren can produce functional CFTR protein, resulting in improvements in chloride channel activity," said Frank Accurso, M.D., Professor of Pediatrics and Section Head of Pulmonary Medicine of the University of Colorado, Denver and a leading ataluren investigator. "With the newly initiated Phase 3 study we hope to determine if the effects of ataluren on the underlying cause of the disease can result in clinical benefit for patients with nmCF."

"As an oral therapy that may address the underlying cause of the disease, ataluren has the potential to improve the management of nmCF for patients and their physicians," said Christiane DeBoeck, M.D., Ph.D., Principal Investigator for University Hospital Leuven. "It is our hope that this long-term clinical trial of ataluren will advance our knowledge of the disease and the standard of care for nmCF patients."

"The initiation of this Phase 3 trial represents an important step forward in our efforts to develop treatments for the underlying cause of cystic fibrosis," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. "Phase 2 studies of ataluren showed encouraging potential to restore CFTR protein production and we are pleased to be supporting this study." In July 2008, Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, expanded its existing collaboration with PTC Therapeutics. PTC will receive up to $25 million from CFFT in support of the development of ataluren.

An orally administered therapy, ataluren is the first investigational new drug designed to enable the formation of a functioning protein in patients with genetic diso
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SOURCE PTC Therapeutics, Inc.
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