Takeda Announces Positive Phase 3 Results for Soticlestat in Dravet Syndrome

Takeda today announced positive results from the Phase 3 SKYLINE trial evaluating soticlestat in patients with Dravet syndrome. The trial met its primary endpoint, demonstrating a statistically significant 36% reduction in convulsive seizure frequency compared to placebo. Soticlestat is a first-in-class cholesterol 24-hydroxylase (CH24H) inhibitor that reduces the production of 24S-hydroxycholesterol, a neurosteroid that modulates NMDA receptor activity. Dravet syndrome is a severe, treatment-resistant epilepsy that begins in infancy and is associated with significant morbidity and mortality. Takeda plans to submit regulatory applications for soticlestat based on these results.