PTC Therapeutics has snared a rich licensing deal with Genzyme for its lead development program. Genzyme has agreed to pay $100 million up front for PTC124, which is in Phase IIb for Duchenne Muscular Dystrophy. PTC124 also completed a mid-stage trial for cystic fibrosis with "promising" results. PTC retains commercialization rights in the U.S. and Canada with Genzyme taking over the rest of the world. Genzyme will pay up to $337 million in milestones.
PTC--a 2007 Fierce 15 company--has linked 2,400 genetic disorders to nonsense mutations. By targeting a specific type of genetic alteration--nonsense mutations--PTC124 bypasses the defect and leads to the restoration of a functional protein. Nonsense mutations inactivate gene function and are known to cause anywhere from five to 70 percent of the individual cases of most of these inherited diseases.
"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 CEO. "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.
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