Celgene separately exercised option to work with PTC to discover small molecules against oncology targets.

Roche is paying PTC Therapeutics $12 million up front as part of an agreement to develop small molecule CNS disease drugs. PTC separately announced that Celgene exercised its option to collaborate with the company on the development of GEMS-based candidates against an oncology target. The original agreement was inked in 2007, and Celgene made a $20 million investment in the company.

Under the terms of the collaboration and licensing deal with Roche, PTC will use its GEMS (gene expression modulation by small molecule) technology, and Roche will cover research costs. The aim initially is to develop orally bioavailable  candidates against four, jointly selected CNS disease targets. PTC says that it could earn up to $239 million in research, development, regulatory, and commercialization milestones per target plus double-digit sales royalties. Roche also has an option to add four additional targets to the collaboration across different therapeutic areas.

PTC’s GEMS technology is designed to identify small molecules that modulate post-transcriptional control mechanisms. The company says that candidates target processes that act through untranslated regions of mRNA.  Lead in-house candidate include the small-molecule VEGF inhibitor, PTC299, and ataluren (PTC124), which is in development for the treatment of nonsense mutation genetic disorders.

Ataluren is currently undergoing a pivotal clinical trial in patients with nonsense mutation Duchenne and Becker muscular dystrophy. PTC said it also aims to start a second pivotal trial of ataluren in patients with nonsense mutation cystic fibrosis during 2009. The company has an exclusive collaboration with Genzyme (www .genzyme.com) for the development and commercialization of ataluren outside North America.

The FDA and the European Commission previously granted the drug orphan drug status for the treatment of DMD and cystic fibrosis due to nonsense mutations. The FDA has also granted ataluren subpart E designation for expedited development, evaluation, and marketing.

In July, PTC started a Phase II trial assessing PTC299 in adult patients with neurofibromatosis type 2 (NF2). The drug is also undergoing a Phase 1b study in advanced solid tumors and a Phase Ib/II trial in metastatic breast cancer.

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