Roche, PTC Therapeutics, and the SMA Foundation, have signed a licensing agreement for PTC's Spinal Muscular Atrophy (SMA) program. SMA is a genetic neuromuscular disorder that causes muscle weakness.

PTC Therapeutics' program has been developed in partnership with the SMA Foundation, which will remain active in the collaboration.

Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC's SMA program, which includes three compounds currently in preclinical development, as well as potential back-up compounds. PTC receives 30 million as an upfront payment, up to 460 million upon successful completion of certain development and commercialization milestones, and up to double-digit royalties on commercial sales. Development will be overseen by a joint steering committee comprised of members from Roche, PTC and the SMA Foundation.

Luca Santarelli, global head of Roche neuroscience, said: "We found the science behind this program very compelling, with the potential to help treat a currently incurable condition. This is the essence of Roche's entire strategy, focused on solid science and high unmet clinical need, and these compounds bolster our rich pipeline in central nervous system diseases. As an established partner of Roche, we already have experience with PTC's scientific approach. Together with the involvement of the SMA Foundation, we now have the opportunity to make a significant impact in the treatment of SMA."

"Having been a partner with Roche for several years, we have every confidence that the combination of our own expertise and Roche's considerable capabilities in clinical development, biomarkers and diagnostics will help us maximize the potential for this programme," commented Stuart W. Peltz, Ph.D., president and CEO of PTC.