Genzyme Corporation and PTC Therapeutics, Inc. have 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.
Duchenne muscular dystrophy is characterized by rapid progression of muscle degeneration, eventually leading to loss in ambulation, paralysis, and death.
Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. 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. Genzyme has extensive experience with cystic fibrosis, having conducted more than six clinical trials among CF patients. In the field of DMD, Genzyme's experience with Myozyme(R) (alglucosidase alfa), for the treatment of the genetic disorder Pompe disease, will be directly applicable as patients with both diseases are treated by the same specialist physicians.