July 22, 2008

PTC Therapeutics, Genzyme to Collaborate on PTC124 for DMD

Genzyme Corp., a Cambridge, Mass.-based biopharmaceutical company, will collaborate with PTC Therapeutics of South Plainfield, N.J., to develop PTC124 for Duchenne muscular dystrophy (DMD), cystic fibrosis and other genetic disorders. Genzyme is the developer of Myozyme, recently approved for the treatment of Pompe disease.

“We couldn’t be happier about this development,” said Sharon Hesterlee, MDA’s vice president for Translational Research. “This is exactly what MDA’s translational research program is designed to do, to provide initial funding to innovative new biotechnology companies like PTC Therapeutics, with the hope that the technology will be brought to market in the form of a new drug.

“MDA provided PTC with an initial $1.5 million grant, enabling the company to begin developing PTC124, a medication with the potential to treat a significant portion of patients with DMD. We’re continuing to maintain a close relationship with PTC, but having Genzyme involved will lend more resources and experience to the project. During the development of Myozyme, which is now saving the lives of people with the metabolic muscle disorder Pompe disease, we worked closely and effectively with Genzyme.”

A July 17 announcement released jointly by Genzyme and PTC stated that Genzyme will make an up-front payment of $100 million to PTC, with potential additional payments later on, and will commercialize PTC124 outside the United States and Canada. The announcement said PTC will be financially responsible for one current and three additional clinical trials of PTC124 and for the commercialization of the drug in the United States and Canada.

About PTC124

PTC124 is an orally administered small molecule that targets a type of disease-causing genetic error known as a nonsense mutation, or premature stop codon. This type of error is a small DNA change that stops a cell from “reading” the gene’s full instructions for a protein and results in the formation of a shortened protein that’s less than fully functional.

About Duchenne Muscular Dystrophy

In DMD, the muscle protein dystrophin is missing in muscle cells, causing progressive degeneration of skeletal and cardiac muscles and death in early adulthood. Widespread DNA testing over the last several years has revealed that nonsense mutations are responsible for approximately 13 percent to 15 percent of cases of this disease.

About PTC124 Clinical Trials

In November, PTC announced 18 out of 38 DMD patients with known nonsense mutations who took PTC124 at three different dosage levels for one month began making full-length dystrophin.

The company’s current trial of PTC124 in DMD will analyze results from 165 boys with the disease and known nonsense mutations who are at least 5 years old and are still walking. For details, see “PTC124-Phase 2b Study of Safety and Efficacy.”