Genzyme Targeting Genetic Disorders in New Pact (GENZ)
Genzyme Corporation (Nasdaq: GENZ) has formed a collaboration and development pact to go after genetic disorders. It has partnered with PTC Therapeutics, Inc. in an exclusive global collaboration to develop and commercialize PTC124. This is PTC’s novel oral therapy in late-stage development for the treatment of many genetic disorders due to nonsense mutations.
PTC will commercialize PTC124 in the United States and Canada, while Genzyme will commercialize the treatment in all other countries. Genzyme is making an up-front payment of $100 million to PTC and has committed to potential milestone and royalty payments to PTC as well in an amount that could be up to $337 million. PTC will be financially responsible for one ongoing and three additional clinical trials of PTC124.
The release notes that this is potentially applicable to hundreds of genetic diseases. It 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 this year.
Jon C. Ogg
July 17, 2008