Genzyme Corporation and PTC Therapeutics Announce Collaboration on Small Molecule for Geneti...(that holds great potential to help CF an...)

Date:7/17/2008

that holds great potential to help CF and DMD patients, and many others with a variety of devastating diseases," stated Henri A. Termeer, Genzyme's chairman and chief executive officer. "This collaboration is an excellent strategic fit for Genzyme and will be managed within the company's stated financial guidance."

"One of PTC's earliest scientific insights was that targeting nonsense mutations represented a novel approach to treating a large number of genetic disorders. The translation of that insight through the discovery and rapid development of PTC124 has been very gratifying," commented Stuart W. Peltz, Ph.D., PTC's president and chief executive officer. "This collaboration supports PTC's business strategy of establishing a fully integrated biopharmaceutical company by retaining commercial rights in the United States and Canada while engaging an experienced and capable partner to swiftly address additional markets."

PTC initiated the clinical development of PTC124 in 2004. Based on phase 2a clinical proof of concept in both DMD and CF, further development in each of these indications is being pursued in international, multicenter trials. A phase 2b trial of PTC124 in DMD is currently enrolling, and is expected to include 165 patients. A phase 2b trial of PTC124 in CF is planned to begin by the end of this year. With demonstration of clinical benefit, these two trials are expected to serve as the basis for registration of PTC124 in these indications. Further development of PTC124 will include clinical trials in multiple additional genetic disorders.

In the United States, there are an estimated 10,000 DMD patients, approximately 13 percent of which have nonsense mutations. Of the more than 30,000 U.S. patients with CF, about 10 percent have nonsense mutations. There is a significant unmet medical need for new treatments for these diseases. In addition to DMD and CF, the companies plan to explore PTC124's potential to make a difference for pa
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SOURCE Genzyme Corporation and PTC Therapeutics Inc. Copyright©2008 PR Newswire. All rights reserved

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