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Roche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)
Date:11/29/2011

BASEL, Switzerland, SOUTH PLAINFIELD, N.J., and NEW YORK, Nov. 29, 2011 /PRNewswire/ -- Roche (SIX: RO, ROG; OTCQX: RHHBY), PTC Therapeutics, Inc. (PTC) and the SMA Foundation, announced today a licensing agreement for PTC's Spinal Muscular Atrophy (SMA) programme. 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.

PTC Therapeutics' programme 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.

Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC's SMA programme, which includes three compounds currently in preclinical development, as well as potential back-up compounds. PTC receives USD30 million as an upfront payment, up to USD460 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.

"This collaboration brings us one step closer to developing a treatment for a condition that has a profound effect on the lives of many thousands of children and their parents worldwide," said Dinakar Singh, Chairman of the SMA Foundation.
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SOURCE PTC Therapeutics, Inc.
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