Biogen announces new SPINRAZA data

Biogen announced new SPINRAZA data aimed at answering critical questions for the spinal muscular atrophy community. The data were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA this week in Orlando, Fla. RESPOND is an ongoing two-year, phase 4 open-label study to evaluate clinical outcomes and safety following treatment with SPINRAZA in infants and toddlers with SMA who have unmet clinical needs after treatment with Zolgensma. Interim efficacy results at six months from 29 study participants treated with SPINRAZA show: Improvements in motor function in most participants as measured by increased mean total Hammersmith Infant Neurological Examination Section 2 score from baseline; Participants with two SMN2 copies improved by a mean of over 5 points on HINE-2; All participants with three SMN2 copies improved; a mean change from baseline was not calculated due to the small number of participants; Most participants with investigator-reported suboptimal motor function at baseline improved; After a median of 230.5 days in the study, serious adverse events were reported in 13/38 participants. No serious AEs were considered related to SPINRAZA or led to study withdrawal. No new emerging safety concerns have been identified in enrolled participants who received SPINRAZA after Zolgensma. Additional interim clinical outcomes from the RESPOND study are being presented at the conference.