Biogen, Ionis: Nusinersen Meets Primary Endpoint At Interim Analysis Of Trial

(RTTNews) - Biogen Inc. (BIIB) and Ionis Pharmaceuticals, Inc. (IONS) said that nusinersen, their investigational treatment for spinal muscular atrophy or SMA, met the primary endpoint pre-specified for the interim analysis of Endear, the Phase 3 trial evaluating nusinersen in infantile-onset (consistent with Type 1) SMA.

The analysis found that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones, compared to those who did not receive treatment. Nusinersen demonstrated an acceptable safety profile in the trial.

As a result of these findings, Biogen has exercised its option to develop and commercialize nusinersen globally and paid Ionis a $75 million license fee. Biogen will initiate regulatory filings globally in the coming months.

Based on the results of the pre-specified interim analysis, the Endear study will be stopped and participants will be able to transition into the Shine open-label study in which all patients receive nusinersen. Data from the other endpoints of Endear will be analyzed when the full data set is available.

Biogen noted that it is now responsible for all nusinersen development, regulatory and commercialization activities and costs. Ionis will complete the Phase 3 studies and work with Biogen on regulatory filings.

Ionis is eligible to receive tiered royalties on any potential sales of nusineren up to a percentage in the mid-teens, in addition to up to $150 million in milestone payments based on regulatory approvals.