Roche, PTC Therapeutics Inc., (PTC) and the SMA Foundation, signed a licensing agreement for PTC's Spinal Muscular Atrophy (SMA) programme. SMA is a genetic neuromuscular disorder that causes muscle weakness. One in every 10,000 children born is affected with the disorder, which currently has no effective treatment.
PTC Therapeutics' programme has been developed in partnership with the SMA Foundation, which will remain active in the collaboration. The SMA Foundation was established in 2003 by Loren Eng and Dinakar Singh to accelerate the development of a treatment for SMA.
SMA is caused by a missing or defective SMN1 gene, which results in reduced levels of the survival motor neuron (SMN) protein. The compounds in PTC's research treat the underlying cause of the disorder and demonstrate increases in SMN levels in nervous system, muscles and other tissues in SMA models. SMA is a rare disorder and could be eligible for orphan status by regulatory authorities, thereby potentially reducing the time needed for a drug to reach patients.
Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC's SMA programme, which includes three compounds currently in preclinical development, as well as potential back-up compounds. PTC receives US$ 30 million as an upfront payment, up to US$ 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.
“This collaboration brings us one step closer to developing a treatment for a condition that has a profound effect on the lives of many thousands of children and their parents worldwide,” said Dinakar Singh, chairman of the SMA Foundation. “We are very optimistic that, by building on the pioneering efforts of PTC Therapeutics, Roche can help us realise what we have been working so hard to achieve.”
Luca Santarelli, Global Head of Roche Neuroscience, said: “We found the science behind this programme 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, PhD, president and CEO of PTC. “We are delighted that the SMA Foundation continues to be an active participant in the collaboration as we share a strong commitment to advancing this innovative potential treatment as rapidly as possible.”
Roche and PTC announced their first collaboration in September of 2009 for the development of orally bioavailable small molecules utilizing PTC's technology called Gene Expression Modulation by Small-molecules (GEMS). The SMA programme has been developed by PTC utilizing a different scientific approach than GEMS, called alternative splicing.
The Spinal Muscular Atrophy Foundation is a non-profit organization dedicated to accelerating progress towards a treatment for Spinal Muscular Atrophy through targeted funding of clinical research and novel drug development efforts.
PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered small-molecule drugs that target post-transcriptional control processes.
Roche is a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics.