PTC Therapeutics just selected a development candidate in its spinal muscular atrophy (SMA) collaboration with Roche and the SMA Foundation, triggering a $10 million payment to PTC from Roche.
The SMA program, which PTC had initially been developing solely in partnership with the SMA Foundation, began in 2006 with the aim of identifing and optimizing compounds that increase production of the survival motor neuron (SMN) protein, the lack of which causes SMA. Then in November 2011 Roche gained an exclusive worldwide license to the SMA program, paying PTC $30 million up front and entitling PTC to $10 million based upon the selection of a development candidate. PTC may receive up to an additional $450 million upon successful completion of other development and commercialization milestones, plus royalties on net product sales worldwide.
"We are highly encouraged by the continued progress," said Karen Chen, CSO of the SMA Foundation, in a statement. "Achieving this milestone brings us closer to developing a treatment for thousands of patients with SMA."