Roche, PTC Therapeutics Inc. (PTC), and the SMA Foundation announced a licensing agreement for PTC’s Spinal Muscular Atrophy (SMA) program.
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.
PTC Therapeutics’ program has been developed in partnership with the SMA Foundation, which will remain active in the collaboration.
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. The disorder 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, potentially reducing the time needed for a drug to reach patients.
Roche and PTC announced their first collaboration in September 2009 for the development of orally bioavailable small molecules utilizing PTC’s technology called Gene Expression Modulation by Small-molecules (GEMS). The SMA program has been developed by PTC utilizing a different scientific approach, called alternative splicing.
Release date: Nov. 29, 2011
Source: Roche: www.roche.com
