Ionis zilganersen meets primary endpoint in Alexander disease; PDUFA Sept 22, 2026

IONIS PHARMACEUTICALS INC

Primary endpoint met: gait speed stabilized vs control at Week 61 (LS mean diff 33.3%, p=0.041) in patients ≥5 years.
GMFM-88 in children 2-4 years showed 22.9-point improvement vs control (nominal p=0.034).
Key secondary endpoints (MBS, PGIS, PGIC, CGIC) consistently favored zilganersen over control.
Serious TEAEs less frequent on zilganersen (37.5%) vs control (47.1%); most AEs mild/moderate.
FDA Priority Review with PDUFA action date set for September 22, 2026.

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