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. Duchenne muscular dystrophy is characterized by rapid progression of muscle degeneration, eventually leading to loss in ambulation, paralysis, and death.
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.
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.