Roche Announce Spinal Muscular Atrophy Agreement with PTC

Roche yesterday announced that they have signed a new licensing contract with PTC Therapeutics to develop treatments for a genetic muscle-weakening disease in a pact that could be worth $490 million.

The Swiss major has signed up to gain access to PTC’s spinal muscular atrophy (SMA) programme.  SMA is a genetic neuromuscular disorder that results in muscle weakness and one in every 10,000 children born is affected by it.

There are currently no effective treatments for SMA, which is the principal genetic cause of death in infants and toddlers, and caused by a missing or defective SMN1 gene, which results in lower levels of the survival motor neuron protein.

Roche noted that given the rarity of SMA, it “could be eligible for orphan status by regulatory authorities, thereby potentially reducing the time needed for a drug to reach patients”.  The compounds in PTC’s research treat the underlying origin of the disorder and demonstrate rises in SMN levels in nervous system, muscles and other tissues in SMA models.

Under the terms of the contract, Roche gains an exclusive global licence to PTC’s SMA programme, which includes three compounds currently in preclinical development, as well as potential back-up compounds.  PTC will receive a $30 million upfront fee and up to $460 million upon successful accomplishment of certain development and commercialisation milestones, plus double-digit royalties.

PTC’s programme has been developed in partnership with the SMA Foundation, which was founded in 2003 to accelerate the development of a treatment for SMA.  The development will be supervised by a joint steering committee comprised of members from the SMA Foundation, as well as Roche and PTC.

Dinakar Singh, chairman and founder of the SMA Foundation, which has given out an excess of $100 million for research, commented that “Roche can help us realise what we have been working so hard to achieve.”

Luca Santarelli, head of neuroscience at Roche, added “we found the science behind this programme very compelling, with the potential to help treat a currently incurable condition.”  He went on to claim that “this is the essence of Roche’s entire strategy, focused on solid science and high unmet clinical need, and these compounds bolster our rich pipeline in central nervous system diseases.”

PTC and Roche are old friends and signed an agreement in September 2009 to develop orally bioavailable small molecules using PTC’s Gene Expression Modulation Small-molecules technology, focusing initially on four CNS disease targets.  Roche made an upfront payment of $12 million and the potential of that deal is in the region of $1 billion.


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