Backing will support Phase IIb trials of PCT124.

With the expansion of an existing agreement with Cystic Fibrosis Foundation Therapeutics, PTC Therapeutics will receive up to $25 million. The funding will support Phase IIb trials of PTC124, the firm’s investigational treatment for cystic fibrosis (CF) caused by nonsense mutations.

“We are pleased to expand our collaboration with PTC Therapeutics on the development of the therapy PTC124,” says Robert J. Beall, Ph.D., president and CEO of the foundation. “The encouraging data from the Phase IIa studies indicate that PTC may become a very important drug for the treatment of CF in the 10 percent of patients who carry nonsense mutations.”

Last month, PTC reported Phase IIa data from adult and pediatric CF patients. The studies demonstrated statistically significant improvements in the primary outcome measure, nasal potential difference across a variety of age ranges, geographies, and nonsense mutation types, according to the company. In the three-month, Phase IIa extension study of adult patients, results showed a statistically significant mean decrease in the frequency of cough, one of the most prominent and burdensome CF-related symptoms.

The original agreement was inked in February 2005 and provided $1.7 million of funding to PTC Therapeutics for PTC124 development.

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