Biogen’s higher Spinraza dose meets the key primary endpoints in Phase II/III trial

Biogen's higher dose of Spinraza (nusinersen) for spinal muscular atrophy (SMA) has met the primary endpoint in a Phase II/III DEVOTE study. The trial showed that infants on the higher dose regimen had significant motor function improvements after six months compared to an untreated control group from the ENDEAR study, which led to the approval of the standard 12 mg dose.

Part B of the study included 75 treatment-naïve infants. The higher dose regimen involved two 50 mg doses, 14 days apart, followed by a 28 mg maintenance dose every four months, compared to the standard 12 mg regimen.

Patients on the higher dose showed a 26.19-point improvement in CHOP-INTEND motor function scores. Adverse events were consistent with Spinraza's safety profile, with fewer serious incidents in the higher dose group. Biogen plans to seek regulatory approval for this dose, with Spinraza already approved in 71 countries.