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PTC Therapeutics, Inc.

Drug Names(s): PTC124, PTC-124, Ataluren

Description: PTC124 is an orally delivered drug for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely halt the translation process, producing a shortened, non-functional protein. PTC124 allows the cellular machinery to bypass the nonsense mutation and continue the translation process, restoring the production of full-length, functional proteins. PTC124 has demonstrated the ability to restore full-length functional protein in preclinical genetic disease models harboring nonsense mutations.

Deal Structure: PTC Therapeutics and Genzyme
In July 2008, Genzyme and PTC Therapeutics announced an exclusive global collaboration to develop and commercialize PTC124.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 made 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, then the companies will share research and development costs equally. Genzyme and PTC will each bear the sales, marketing and other costs associated with commercialization of PTC124 in their respective territories.

PTC is eligible to receive up to $337 million in total milestone payments, as follows: up to $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...See full deal structure in Biomedtracker

Partners: University of Pennsylvania

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