Biogen Announces Positive Results from Higher Dose Spinraza Study in Spinal Muscular Atrophy Patients

DEVOTE Study Initiation:
Biogen initiated the DEVOTE study, a Phase 2/3 clinical trial, to evaluate the safety and efficacy of a higher dose of Spinraza (nusinersen) in spinal muscular atrophy (SMA) patients.

Study Design:
The DEVOTE study is a randomized, controlled, dose-escalating trial that aims to enroll 126 patients of all ages with SMA. It includes an open-label safety evaluation, a pivotal double-blind active control randomized treatment period, and an open-label treatment period.

Dosing Regimen:
Patients in the study receive two loading doses of 50 mg 14 days apart, followed by a maintenance dose of 28 mg every four months. This is compared to the current approved dosing regimen of four loading doses with 12 mg maintenance doses every four months.

Initial Findings:
Preliminary data from the DEVOTE study indicate no new safety concerns and support the continued development of a higher dose of Spinraza.

ASCEND Study:
Biogen also announced the initiation of the ASCEND study, a Phase 3b trial, to evaluate the safety and efficacy of higher doses of Spinraza in SMA patients previously treated with risdiplam (Evrysdi).

Clinical Impact:
Spinraza has been shown to improve or stabilize motor function in SMA patients, and the exploration of higher doses aims to address remaining unmet medical needs in the SMA community.