Biogen has exercised its option to develop and commercialize nusinersen following positive interim Phase III results, agreeing to advance the spinal muscular atrophy (SMA) candidate in a deal that could generate $225 million-plus for Ionis Pharmaceuticals.

Nusinersen met its primary endpoint in an interim analysis of the Phase III ENDEAR trial evaluating nusinersen in infantile-onset (consistent with Type 1) SMA, as patients treated with the candidate showed statistically significant improvement in achieving motor milestones compared to patients who did not receive treatment.

On the basis of those findings, Biogen and Ionis said, the ENDEAR study will be stopped and participants will be able to join 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, with results to be presented at future medical congresses, the companies said.

Participants enrolled in the sham-controlled arm of EMBRACE, a Phase II study that also included infantile-onset patients, will have the opportunity to receive nusinersen, Biogen and Ionis said. Other studies in the nusinersen program, including CHERISH (later-onset consistent with Type 2) and NURTURE (presymptomatic infants), will continue as planned.

“We remain committed to understanding the potential of nusinersen in the broader SMA population and will continue to focus on the rapid completion of our ongoing studies,” Alfred Sandrock, M.D., Ph.D., Biogen evp and CMO, said in a statement. “We share the community’s sense of urgency as we strive to bring the first treatment for SMA, the leading genetic cause of infant mortality, to families facing this devastating disease.”

To that end, Biogen said it is working to open a global expanded-access program (EAP) for eligible patients with infantile-onset SMA (consistent with Type 1) in coming months. Once the EAP is operational and required local approvals are in place, individual participating trial sites may start enrollment after they have transitioned ENDEAR study participants to the open-label extension study.

Biogen has paid Ionis a $75 million license fee after exercising its option to develop and commercialize nusinersen globally. Biogen is now responsible for all nusinersen development, regulatory and commercialization activities, and costs, while Ionis will complete the Phase III studies and work with Biogen on regulatory filings.

The two companies said they will also work together to transition the clinical programs that Ionis is conducting to Biogen. In addition to the license fee, Ionis could receive tiered royalties on 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.

The companies had partnered on nusinersen for SMA—one of several collaborations by the companies—since 2012, when Ionis was known as Isis Pharmaceuticals and the SMA candidate was called ISIS-SMNRX.

Nusinersen is the first antisense drug from Ionis’ neurological disease franchise to advance to regulatory review: “It illustrates the potential of our antisense technology to address severe diseases that other therapeutic modalities are unable to address adequately,” added Ionis COO B. Lynne Parshall.

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