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– New analyses from the ongoing open label extension (OLE) studies and findings from electroencephalogram (EEG) assessments in patients with Dravet syndrome treated with zorevunersen support the potential for disease modification –
BEDFORD, Mass. & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, and Biogen Inc. (Nasdaq: BIIB) today announced data presentations from studies of zorevunersen at the 2025 American Epilepsy Society (AES) Annual Meeting, taking place December 5-9 in Atlanta, Georgia.
Zorevunersen is an investigational antisense oligonucleotide currently being evaluated as a potential first-in-class medicine for the treatment of Dravet syndrome, a severe developmental and epileptic encephalopathy (DEE) characterized by recurrent seizures and significant cognitive and behavioral impairments. Data to be presented at AES are derived from four years of clinical data from the Phase 1/2a and ongoing OLE studies of zorevunersen and will include new propensity weighted analyses. Zorevunersen is also currently being evaluated in the global, pivotal Phase 3 EMPEROR study in children and adolescents with Dravet syndrome.
“Our four years of data showing substantial and durable effects on seizures, behavior and cognition, combined with our ability to compare directly to natural history data, allow us to more fully appreciate the disease-modifying potential of zorevunersen for the treatment of Dravet syndrome,” said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. “We look forward to discussing our latest findings with the world’s leading epilepsy experts at AES.”
“The new EEG data at AES highlight the effects of zorevunersen in decreasing abnormal brain activity that is persistently higher in patients with Dravet syndrome,” said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. “In patients treated with zorevunersen, exploratory analyses showed that a reduction in abnormal brain EEG activity was associated with an increased probability of achieving a reduction in major motor seizure frequency. These findings, in addition to the clinical data to be presented, show the potential effect of zorevunersen on the underlying disease in patients with Dravet syndrome.”
Details of the presentations at AES are as follows:
About Dravet Syndrome Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. Even when treated with the best available anti-seizure medicines (ASMs), up to 57 percent of patients with Dravet syndrome do not achieve ≥50 percent reduction in seizure frequency. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP; up to 20 percent of children and adolescents with Dravet syndrome die before adulthood due to SUDEP, prolonged seizures, seizure-related accidents or infections1. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan2. There are no approved disease-modifying therapies for people living with Dravet syndrome.
About Zorevunersen Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing functional NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.
About the EMPEROR Study The EMPEROR Phase 3 Study (NCT06872125) is a global, double-blind, sham-controlled study evaluating the efficacy, safety, and tolerability of zorevunersen in children ages 2 to
