PTC Therapeutics, Inc. has 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.
"We are excited about this important achievement in our SMA program", stated Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics.
Peltz continued, "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."
SMA is caused by a missing or defective SMN1 gene, which results in reduced levels of the survival motor neuron (SMN) protein. It is a genetic neuromuscular disease responsible for the early death of motor neuron cells within the spinal cord leading to muscle atrophy and eventually death in the most severe form of the disease.
The oral small molecule compounds in the program target the underlying cause of the disorder by increasing SMN protein levels in the nervous system, muscles, and other tissues.
It is estimated that SMA affects approximately 10,000 to 25,000 children and adults in the United States, and that between one in 6,000 and one in 10,000 children are born with this rare disorder.
Luca Santarelli, Head of Neuroscience and Small Molecule Research at Roche commented: "The compelling science behind this project and the highly synergistic alliance between Roche, PTC Therapeutics and the SMA Foundation are the groundwork for this potential therapeutic program. We consider every step towards a treatment option in SMA of high importance for patients and families affected by this devastating and currently incurable condition."
The SMA program was initially developed by PTC Therapeutics' in partnership with the SMA Foundation. The SMA Foundation was established in 2003 to accelerate the development of a treatment for SMA.
In November 2011, Roche gained an exclusive worldwide license to PTC's SMA program. PTC received a $30 million upfront payment, is entitled to $10 million based upon the selection of a development candidate and may receive up to an additional $450 million upon successful completion of other development and commercialization milestones, plus tiered royalties on worldwide net product sales.
Development is overseen by a joint steering committee comprised of members from Roche, PTC and the SMA Foundation.
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."