Stoke Therapeutics at Guggenheim: dravet data build launch case

Stoke Therapeutics presented its case at the Guggenheim Healthcare Innovation Conference on November 10, 2026, highlighting the potential of its lead Dravet syndrome medicine to become a disease-modifying therapy. The company showcased durable clinical gains, with an 80% seizure reduction over four years in open-label extension data and improved cognitive and behavioral endpoints using a basket-style MC4 Vineland score.

Stoke's Phase III EMPEROR program is ahead of schedule with 162 patients enrolled, and top-line data are expected in Q3 2027. The company estimates a U.S. Dravet opportunity of about 16,000 patients, with around 6,000 immediately addressable at launch. Stoke's Dravet syndrome program targets the underlying cause of the disease by increasing protein production from the healthy allele, rather than just suppressing symptoms.

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