Roche signs up to $490 million accord with PTC Thera for SMA program; and deal with Galena

Swiss drug major Roche (ROG: SIX) has signed a licensing agreement with privately-held US firm PTC Therapeutics and the SMA Foundation for PTC’s Spinal Muscular Atrophy (SMA) program. 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.

Under the terms of the accord, 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 on 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. 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.