PTC Therapeutics, Inc (PTC) announced an exclusive research collaboration and licensing agreement with Roche for the development of orally bioavailable small molecules utilizing PTC's technology called Gene Expression Modulation by Small-molecules (GEMS). The collaboration focuses initially on four CNS disease targets to be jointly selected.

Under the terms of the agreement, Roche will make an upfront cash payment of $12 million and fund PTC's research efforts. Subject to achievement of several successive milestones, there is the potential for PTC to earn up to $239 million in research, development, regulatory and commercial milestone payments per target. PTC would also receive up to double digit royalties for all products resulting from this collaboration. Roche has the option to add four targets to the collaboration across therapeutic areas, for additional cash payments.

"We are pleased to enter into this collaboration with PTC Therapeutics. PTC's expertise in small molecules addressing RNA biology and the demonstrated productivity of their GEMS technology platform make this an attractive collaboration," said Dr Christer Nordstedt, head, CNS Discovery of Roche. "We believe that this novel and highly innovative technology will enable Roche to address important disease mechanisms that were intractable with conventional approaches. This may have profound impact on the treatment and management of several CNS disorders and therefore lead to great benefits for the patient."

"We are excited to enter into this partnership with Roche," said Stuart Peltz, president and CEO of PTC Therapeutics. "This alliance is part of our ongoing strategy to leverage the broad value of the GEMS technology beyond our core research areas. Together with Roche, we are well-positioned to pursue high-interest targets to address complex CNS disorders and bring therapeutic options for patients in need."

GEMS is PTC's novel and proprietary technology platform for the identification of small-molecules that modulate post-transcriptional control mechanisms.

PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes.