Under the terms of the agreement, PTC will commercialize PTC124 in the US 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 IIb trial for Duchenne muscular dystrophy (DMD), and a Phase IIb trial in cystic fibrosis (CF) is expected to begin by the end of 2008.
Once these four studies are completed, the companies will share R&D costs equally. Genzyme and PTC will each bear the sales, marketing and other costs associated with commercialization of PTC124 in their respective territories.