Business
Biogen and Stoke Therapeutics Announce First Patient Dosed in Phase 3 EMPEROR Study of Zorevunersen, a Potential Disease-Modifying Treatment for Dravet Syndrome
– Global, pivotal Phase 3 study will evaluate efficacy and safety of zorevunersen compared to sham over a 52-week treatment period – – Dravet syndrome is a
About this update from Biogen Inc.
[{"type":"text","content":"– Global, pivotal Phase 3 study will evaluate efficacy and safety of zorevunersen compared to sham over a 52-week treatment period – – Dravet syndrome is a rare genetic disease characterized by refractory seizures and neurodevelopmental impairments, with no currently approved medicines that address the underlying cause of the disease – CAMBRIDGE, Mass. and BEDFORD, Mass., Aug. 11, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today announced that the first patient has been dosed in the global Phase 3 EMPEROR study of zorevunersen for the treatment of Dravet syndrome. Zorevunersen, an investigational antisense oligonucleotide, has the potential to be the first disease-modifying treatment for Dravet syndrome. “Our Phase 1/2 and open-label extension studies have provided a large dataset to support our understanding of zorevunersen and guide the EMPEROR study design, including dosing, duration and selection and powering of the endpoints,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “Given the severity of this disease and the limitations of current treatments, the substantial and durable reductions in seizures and continuing improvements in cognition and behavior support our belief that zorevunersen may improve outcomes for patients with Dravet syndrome.” “The initiation of the EMPEROR study is a critical milestone in zorevunersen's development,” said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. “Despite treatment with available anti-seizure medicines, no approved medications currently address the underlying cognitive and behavioral aspects of this rare, genetic disease. Together with Stoke, we look forward to working in collaboration with the hope of bringing forward zorevunersen as the first disease-modifying treatment option, if approved, for Dravet syndrome.” EMPEROR Pivotal Phase 3 Design Summary Anticipated Enrollment: Patients with Dravet syndrome between the ages of 2 and up to 18 years of age with a confirmed variant in the SCN1A gene not associated with gain of function.Duration: Following an 8-week baseline period, participants will be randomized 1:1 to receive either zorevunersen or sham for...