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Isis, Biogen Strike Potential $299M Deal For Rare Spine Disorder Treatment

Isis Pharmaceuticals has found a new partner in Biogen Idec that’s betting a large amount of money on a new therapy for a rare spinal disorder in newborns.

Weston, MA-based Biogen Idec (NASDAQ: BIIB) said today that it has agreed to work with Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS) on an experimental antisense drug for spinal muscular atrophy in a collaboration that could be worth as much as $299 million. Isis will get $29 million in upfront cash, plus another $45 million from Biogen for development before it may choose to get a license. If Biogen buys a license to the Isis drug candidate, then Isis could get another $225 million in a license fee and milestone payments, plus a double-digit percentage royalty of product sales, if there are any.

Spinal muscular atrophy is a genetic disease that affects an estimated one out of every 10,000 newborns, and causes severe muscle atrophy and weakness. The disease is caused by a faulty version of a gene called survival motor neuron 1 (SMN1) which doesn’t produce enough of the SMN1 protein needed to maintain muscle strength. Spinal muscular atrophy has various forms that cause disability, and in the most severe cases, death in infants. The Isis drug, which comes as an injection into the spine and recently entered clinical trials, is designed to help produce more of the functional SMN1 protein.

Spinal muscular atrophy “is a heartbreaking disease—it can kill children before their 2nd birthday and there are currently no therapies to treat the disease,” said George Scangos, Biogen’s CEO, in a statement. “It is exactly the kind of disease and program that we are focused on at Biogen Idec. The unmet need could not be any greater, the program fits with our mission to bring innovative therapies to patients with serious neurologic diseases, and Isis’ antisense compound has the potential to be a highly effective, first-to-market therapy for this deadly disease.”

Isis, whose business is built around doing scientific work on antisense drugs and then finding partners to finish development, said Biogen fits the bill for this condition because of its expertise in neurology.

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