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Ataluren, Promising CF Drug That Addresses Underlying Defect, Enters Phase 3 Clinical Trial

September 10, 2009

PTC Therapeutics announced today the start of a Phase 3 clinical trial of an oral drug targeted at treating the underlying cause of cystic fibrosis. The potential drug, called ataluren (formally known as PTC124), aims to treat CF patients who have what is known as a "nonsense mutation."

The Phase 3 trial is a 48-week study of ataluren in people with CF age six and older. The main goal of the trial is to find out if ataluren can improve lung function in people with the disease.

“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 results, and we are pleased to be supporting this study.”

In July 2008, the CF Foundation announced it was providing up to $25 million to PTC Therapeutics to support development of ataluren. 

Nonsense mutations interrupt the production of the CFTR protein, the key protein associated with CF. They cause CFTR to be too short and non-functioning. Overall, about 10 percent of people with CF have nonsense mutations.

Sixteen care centers in the CF Foundation’s Care Center Network are participating in the study. As research centers open, details will be listed on

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