- Biogen will pick up an option to license a treatment for spinal muscular atrophy from Ionis Pharmaceuticals after a Phase 3 study showed the drug improved motor function in infant patients, the Cambridge, MA-based company said Monday.
- In exchange for global rights to develop and commercialize the drug, known as nusinersen, Biogen will pay Ionis $75 million. Biogen said it planned to initiate regulatory filings for approval in "the coming months."
- Ionis has been studying nusinersen across four clinical programs, exploring the drug's effect against infantile-onset spinal muscular atrophy (SMA) as well as against later-onset SMA. Biogen will now take over all development and commercialization work for the drug.
Patients with SMA suffer from progressive muscular atrophy and weakness resulting from the loss of motor neurons in the spinal cord and lower brain stem. Severe forms of the condition, which correspond to the amount of survival motor neuron (SMN) proteins lost, can result in paralysis and difficulty breathing and swallowing.
Nusinersen is designed to increase production of SMN protein by altering the splicing of a gene closely related to the lost or defective SMN gene. Biogen only released the topline findings that treatment with nusinersen led to a statistically significant improvement in the achievement of motor milestones, compared to those patients who didn't receive treatment.
But if the Phase 3 results are enough to pass muster with regulators, nusinersen would be the first approved treatment for SMA.
Another ongoing Phase 3 trial is studying nusinersen among non-ambulatory patients with later-onset SMA, up to 12 years old at screening.
The FDA previously granted both Orphan Drug Status and Fast Track Designation to nusinersen, and the drug has received similar designations in the European Union.
Ionis is eligible for up to $150 million in additional milestones for regulatory approvals.
Nusinersen fits nicely with Biogen's focus on treatments for neurological diseases and central nervous system disorders. The good news comes on the heels of Biogen chief executive George Scangos announcing earlier this month he would step down once a successor is found.
Revenues have more than doubled under Scangos' watch, mostly from Biogen's portfolio of multiple sclerosis treatments. But the company is also at an inflection point, recently announcing a spinoff of its hemophilia business to focus more on its core neurological portfolio.
But Biogen is almost entirely reliant on sales of its MS drug for revenue, putting pressure on its R&D efforts for diversification and growth. Nusinersen is hopefully a step in the right direction.