Genzyme Corporation and PTC Therapeutics Announce Collaboration on Small Molecule for Genetic Diseases

21-Jul-2008

Genzyme Corporation and PTC Therapeutics, Inc. (PTC) announced an exclusive global collaboration to develop and commercialize PTC124, PTC's novel oral therapy in late-stage development for the treatment of genetic disorders due to nonsense mutations.

Under the terms of the agreement, PTC will commercialize PTC124 in the United States and Canada, and Genzyme will commercialize the treatment in all other countries. Genzyme will make an up-front payment of $100 million to PTC, plus potential milestone and royalty payments. PTC will be financially responsible for one ongoing and three additional clinical trials of PTC124, which is potentially applicable to hundreds of genetic diseases.

PTC124 is currently being evaluated in a phase 2b trial for Duchenne muscular dystrophy (DMD), and a phase 2b trial in cystic fibrosis (CF) is expected to begin by the end of this year. In its collaboration with PTC, Genzyme will draw on its expertise in genetic disorders and its strong regulatory, manufacturing and marketing infrastructure outside of the United States.

"Over the past two decades, Genzyme has successfully developed four therapies for patients with severe genetic diseases. PTC124 is a powerful new approach 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."

https://www.bionity.com/en/news/84934/genzyme-corporation-and-ptc-therapeutics-announce-collaboration-on-small-molecule-for-genetic-diseases.html