Stoke Therapeutics, Inc. and its partner Biogen presented new long‑term data for the antisense oligonucleotide zorevunersen at the 2025 American Epilepsy Society Annual Meeting in Atlanta on December 5, 2025. The presentation highlighted durable seizure reductions, increased seizure‑free days, and sustained improvements in cognition, behavior, and quality of life for patients with Dravet syndrome.
The data come from a combined Phase 1/2a study and an open‑label extension that has treated more than 800 doses in 81 patients. Across the cohort, seizure frequency fell by a statistically significant margin at six months, and Vineland‑3 cognitive and behavioral subdomains improved meaningfully at 18 months. The safety profile remained consistent with earlier reports, with no new safety signals identified.
A key component of the analysis was a propensity‑score weighted comparison to a matched cohort from the BUTTERFLY natural history study. The weighted analysis confirmed that zorevunersen‑treated patients experienced lower major motor seizure frequency and better functional outcomes than the natural history cohort, reinforcing the disease‑modifying hypothesis that underpins the ongoing global Phase 3 EMPEROR trial.
These findings strengthen the case for regulatory approval and support the company’s strategy to advance zorevunersen toward commercialization. The partnership with Biogen brings additional expertise and resources, while the FDA’s Breakthrough Therapy Designation for zorevunersen accelerates the regulatory pathway. In a market where no disease‑modifying therapy exists for Dravet syndrome, the data position Stoke Therapeutics as a potential first‑in‑class leader and expand the company’s rare‑disease portfolio.
Dr. M. Scott Perry, Head of Neurosciences at Cook Children’s Medical Center, noted, “Open‑label data from the zorevunersen clinical studies have been highly encouraging, bringing hope and anticipation to the Dravet community. The new propensity‑score weighted analysis gives us further context for the improvements we’re seeing in patients living with this devastating disease.”
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