PTC Therapeutics Announces $25 Million Award From Cystic Fibrosis Foundation Therapeutics for Development...( - Expanded Collaboration Supports Piv...),Health

- Expanded Collaboration Supports Pivotal Phase 2b Clinical Trials of First Potential Therapy to Address an Underlying Cause of Cystic Fibrosis -

SOUTH PLAINFIELD, N.J. and BETHESDA, Md., July 16 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) and Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, today announced the expansion of an existing collaboration to support development of PTC124, the PTC's new investigational oral drug for the treatment of cystic fibrosis (CF) caused by nonsense mutations. PTC will receive up to $25 million from CFFT in support of key Phase 2b clinical trials for PTC124 in CF.

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"We are pleased to expand our collaboration with PTC Therapeutics on the development of the therapy PTC124," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. "The encouraging data from the Phase 2a 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. These exciting results lead us to believe PTC124 has the potential to be one of the first oral drugs on the market to address the basic defect in cystic fibrosis."

"We are honored both to receive this award and to continue our productive collaboration with the Foundation," stated Stuart W. Peltz, Ph.D., president and chief executive officer of PTC Therapeutics. "The significant progress that we have made with PTC124 is a result of our unique drug discovery approach as well as the extraordinary support that we have received from the physician, patient, advocacy and regulatory communities. We look forward to initiating registration-directed studies of PTC124 for nonsense-mutation- mediated CF within the next few months."

In June 2008, PTC announced encouraging new
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