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Roche signs $500m muscle disease research deal with PTC

Swiss firm teams up with US biopharmaceutical company PTC Therapeutics and SMA Foundation to research possible treatments for spinal muscular atrophy

Roche has acquired the rights to a research programme that targets the muscle disease spinal muscular atrophy, a condition currently without an effective treatment.

The licensing deal with PTC Therapeutics will see the Swiss pharma company gain access to three preclinical compounds and several 'back-up' compounds.

In return US biopharmaceutical company PTC could receive up to $490m in upfront and milestone payments.

PTC's spinal muscular atrophy (SMA) research programme was developed in partnership with the SMA Foundation, a non-profit organisation set up to finance and encourage the development of a treatment for the condition.

Luca Santarelli, global head of Roche Neuroscience, said the relative rarity of SMA meant the candidates the partners were working on would be likely to be eligible for orphan drug status by regulatory authorities.

"Together with the involvement of the SMA Foundation, we now have the opportunity to make a significant impact in the treatment of SMA," she added.

SMA is a neuromuscular disorder that leads to muscles weakness and is caused by a missing or defective SMN1 gene. This leads to reduced levels of the survival motor neuron (SMN) protein.

PTC's compounds have so far demonstrated an ability to increase SMN levels in the nervous system in pre-clinical studies.

Development of these investigational treatments will now be overseen by Roche, PTC and the SMA Foundation.

Dinakar Singh, chairman of the SMA Foundation, said: "We are very optimistic that, by building on the pioneering efforts of PTC Therapeutics, Roche can help us realise what we have been working so hard to achieve."

PTC is now in line for an upfront payment of $30m, up to $460m in development and commercialisation milestone payments and royalties from commercial sales.

30th November 2011

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