Biogen Idec, Isis to develop spinal muscular atrophy drug

Drug designed to address underlying genetic causes of SMA

WESTON, Mass. — Biogen Idec and Isis Pharmaceuticals will work together on a drug invented by Isis for spinal muscular atrophy, the two companies said.

According to the companies, SMA is the most common genetic cause of infant mortality and causes muscle atrophy and weakness, occurring in 1-in-10,000 births. While children with the condition appear normal at birth, symptoms can develop within a few months and significantly shorten their lifespan. The drug, ISIS-SMNRx, is designed to compensate for the underlying genetic defect that causes SMA.

Under the agreement, Biogen Idec will pay Isis $29 million upfront, plus up to $45 million in milestone payments. In addition, Biogen Idec will have the option to license the drug until the completion of the first successful phase-2/3 trial, while Isis could receive up to $225 million in license fee and regulatory milestone payments, as well as royalties on potential sales.


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