Roche, PTC Therapeutics enter $490-million spinal muscular atrophy drug deal

Roche announced Tuesday a licensing deal for PTC Therapeutics' spinal muscular atrophy (SMA) programme, which could potentially be worth up to $490 million to the US biopharmaceutical company. PTC's programme has been developed in partnership with the SMA Foundation, which the drugmakers indicated will remain active in the collaboration.

Under the agreed terms, Roche will gain an exclusive global licence to the SMA programme, which includes three drugs currently in preclinical development and potential back-up compounds. In return, PTC will receive an upfront payment of $30 million from Roche, as much as $460 million in milestone payments and up to double-digit sales royalties. The companies noted that development will be overseen by a joint steering committee that will also include members from the SMA Foundation.

Luca Santarelli, Roche's worldwide head of neuroscience, remarked that there are currently no approved therapies to treat the causes of SMA, which is due to a missing or defective SMN1 gene. Santarelli explained that PTC's drug candidates activate a backup gene so it produces more of the survival motor neuron protein. He said that animal data had shown that mice that normally die in two weeks were "living 10-times as long" after being treated with PTC’s drug.

PTC’s compounds "target the cause not the symptoms" of the disease, Santarelli noted, adding that "not too many times do you get to do that." Santarelli indicated that it may be two years before a drug from the collaboration could reach clinical trials. "If we are lucky," the pace may be faster, he said.

The SMA Foundation is also working with Novartis on drugs for treating the disease, as well as with Isis Pharmaceuticals. A spokesman for Isis suggested that the company's agent may enter clinical trials in December or January.

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