San Diego Business Journal

Carlsbad-based Isis Pharmaceuticals Inc. has entered into an agreement with biotechnology giant Biogen Idec of Weston, Mass., to develop and commercialize an investigational drug for a rare genetic disorder known as spinal muscular atrophy, the companies said.

Isis could receive as much as $299 million plus royalties from Biogen. That includes $29 million upfront plus $45 million in development milestones. If Biogen eventually licenses the drug, it will pay as much as $225 million more in fees and payments tied to regulatory and sales milestones.

Isis in December initiated early-stage clinical trials of the drug, ISIS-SMNRx, which has already been granted orphan drug status by the Food and Drug Administration. The FDA’s orphan drug program is designed to spur development of products for rare diseases that affect fewer than 200,000 people in the U.S.

Spinal muscular atrophy, or SMA, is a neuromuscular disease characterized by muscle atrophy and weakness. It’s the most common genetic cause of death in infants and toddlers, with one child out of every 10,000 births worldwide born with the disease, according to the New York-based Spinal Muscular Atrophy Foundation.

“SMA is a heartbreaking disease — it can kill children before their second birthday and there are currently no therapies to treat the disease,” said Biogen CEO George A. Scangos.

ISIS-SMNRx is designed to compensate for the underlying genetic defect that causes SMA. Scangos said that the antisense compound has the potential to be a highly effective, first-to-market therapy for the deadly disease.

Under terms of the collaboration agreement, Isis will continue to develop the drug through midstage clinical trials, with Biogen providing advice on trial design and regulatory strategy. By the end of those trials, Biogen will have to decide wither to exercise its option to license the drug and take over global development and commercialization.

— Kelly Quigley