Biogen Idec and Isis Pharmaceuticals have entered into an exclusive, global option and partnership agreement under which they will jointly develop and commercialize ISIS-SMNRx, an antisense investigational drug that treats spinal muscular atrophy (SMA) by compensating for the underlying genetic defect that causes the disease.

The agreement awards Isis an upfront payment of $29 million, with the possibility to receive up to $45 million in milestone payments. While Isis will be responsible for global development of ISIS-SMNRx through phase II/III trials, Biogen Idec has the option to license ISIS-SMNRx until a successful phase II/III trial is completed. If Biogen Idec exercises its option, it will assume global development, regulatory, and commercialization responsibilities.

"SMA is a heartbreaking disease -- it can kill children before their second birthday and there are currently no therapies to treat the disease," said George A. Scangos, Ph.D., CEO of Biogen Idec. “Isis's antisense compound has the potential to be a highly effective, first-to-market therapy for this deadly disease.”

Isis initiated the first phase I clinic study evaluating ISIS-SMNRx in children with SMA in December 2011. Isis plans to follow this study with a phase I multiple-dose study.