Biogen Inc
announced new Spinraza (nusinersen) data that were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA.
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 spinal muscular atrophy (SMA) after treatment with Novartis AG's gene therapy
Zolgensma (onasemnogene abeparvovec).Interim efficacy results at six months from 29 study participants treated with Spinraza show:
Most participants demonstrated improved motor function measured by increased mean total Hammersmith Infant Neurological Examination Section 2 (HINE-2) score from baseline.
- Participants with two SMN2 copies (n=24) improved by a mean of over 5 points on HINE-2
- All participants with three SMN2 copies (n=3) improved; a mean change from baseline was not calculated due to the small number of participants.
- Most participants (25/27) with investigator-reported suboptimal motor function at baseline improved.
After a median of 230.5 days in the study, serious adverse events (AEs) were reported in 13/38 (34%) participants.
No serious AEs were considered related to Spinraza or led to study withdrawal.
William Blair writes that overall interim results suggest the added benefit of Spinraza treatment and support management commentary that sales of the $1.79 billion in FY22 franchise are stabilizing.
The analyst says Spinraza will remain a significant topline contributor for Biogen and its development partner Ionis Pharmaceuticals Inc
and bolster revenues.It says that the ongoing results from RESPOND and the risdiplam switch study to high-dose Spinraza (ASCEND) study will be critical in creating a competitive dynamic whereby Spinraza could have a path to "return to growth."
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