PTC Therapeutics in up to $1 billion discovery collaboration with Roche

New Jersey, USA-based PTC Therapeutics has entered an exclusive research collaboration and licensing agreement with Swiss drug major 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 central nervous system 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, which in total could earn the firm up to $1 billion. 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 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, chief executive 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."

As well as this latest deal with Roche, PCT already has a number of collaborations in place with some major pharmaceutical companies, such as global giant Pfizer (a GEMS deal), Schering-Plough (hepatitis C) and Genzyme Corp. The latter deal involves ataluren, which is in a clinical trial for patients with nonsense mutation Duchenne and Becker muscular dystrophy.

PCT also announced that another partner, Celgene, has exercised its option to collaborate on advancing an oncology target addressed through the application of GEMS. In September 2007, Celgene made a $20 million equity investment in PTC.