After obtaining $25 million from CFFT, Genzyme pays for commercialization rights and agress to milestone fees of up $337 million.



Genzyme is the second group in two days to collaborate with PTC Therapeutics to advance PTC124, which is in late-stage development for the treatment of genetic disorders due to nonsense mutations. Yesterday, the Cystic Fibrosis Foundation Therapeutics committed up to $25 million to fund a Phase II b trial of PTC124.

In this latest agreement, Genzyme will initially pay $100 million. PTC is eligible to receive up to $337 million in total milestones—$165 million in development and approval milestones, the majority of which are to be paid upon approvals in Genzyme territories, and up to $172 million in sales milestones. The sales milestone payments begin when annual net revenues reach $300 million and increase in increments through revenues of $2.4 billion.


PTC will be financially responsible for one ongoing and three additional clinical trials of PTC124. Once all four studies are completed, the companies will share R&D costs equally. PTC will commercialize the drug in the U.S. and Canada, and Genzyme will commercialize the treatment in all other countries. The firm is also eligible to receive tiered double-digit royalties from sales in Genzyme territories.


PTC124 is currently being evaluated in a Phase IIb trial for Duchenne muscular dystrophy. The Phase IIb trial in cystic fibrosis is expected to begin later this year.








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