Stoke Therapeutics’ treatment for Dravet syndrome — a rare, genetic epilepsy that begins in infancy — helped reduce seizures in an early-stage study.
The treatment also led to “clinically meaningful” improvements in cognition and behavior compared to a natural history study, Stoke reported Monday afternoon. The news sent Stoke’s shares $STOK up by around 95% in premarket trading.
Stoke’s goal is to tackle the underlying genetic cause of Dravet syndrome, which is the insufficient expression of a sodium channel protein. Currently, available medicines treat the seizure symptoms. Stoke’s experimental medicine, STK-001, is a short nucleotide strand that essentially “stokes” a still functional copy of the gene to make more of that sodium channel protein.
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