Stoke and Biogen present data supporting zorevunersen's disease-modifying potential for Dravet syndrome

Stoke Therapeutics, Inc.

Propensity score weighted analysis shows statistically significant reductions in major motor seizure frequency at 6 months (70mg loading dose) vs natural history.
Improvements in cognition and behavior (Vineland-3) at 18 months with 45mg dosing; durable effects through 24 months.
EEG analysis reveals dose-dependent decrease in abnormal brain activity; reduction in EEG abnormality correlated with seizure reduction.
Safety: 81 patients, >800 doses; CSF protein elevations in 42% (Phase 1/2a) and 86% (OLE) but no clinical manifestations; 3 unrelated deaths (2 SUDEP, 1 malnutrition).
Data are consistent with endpoints in ongoing Phase 3 EMPEROR study (week 28 primary, 18-month secondary).

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