PTC Therapeutics' Major Milestone in SMA Collabration

PTC Therapeutics today announced the selection of a development candidate in its spinal muscular atrophy (SMA) collaboration with Roche and the SMA Foundation. The achievement of the milestone triggers a $10 million payment to PTC from Roche. 

Back in November 2011 Swiss pharmaceutical giant Roche, PTC Therapeutics and the SMA Foundation, announced a licensing agreement for PTC's SMA programme. PTC Threapeutics had by then identified three different compounds that each increased SMN protein levels and extended median survival of severe SMA mice. According to the agreement PTC was entitled to $10 million based upon the selection of a development candidate.

"We are excited about this important achievement in our SMA program", stated Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "This program exemplifies PTC's technology platform and its chemistry and biology expertise applied in an area of great unmet medical need. We are grateful for the exceptional commitment and dedication from our PTC team, as well as from our partners, Roche and the SMA Foundation. The discovery and advancement of a potential new treatment for SMA is a significant milestone for SMA patients and their families." 

Karen Chen, Chief Scientific Officer of the SMA Foundation, stated, "We are highly encouraged by the continued progress. Achieving this milestone brings us closer to developing a treatment for thousands of patients with SMA. The SMA Foundation is eager to support and propel treatments forward for patients and look forward to the continued success of our collaboration with Roche and PTC."

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