- 3-year data from open-label extension (OLE) studies demonstrate the potential for disease modification with durable seizure reductions and improvements in cognition and behavior on top of standard anti-seizure medicines -
- Substantial improvement in overall seizure burden and seizure free days observed with ongoing treatment -
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 presentations of new clinical data from studies of zorevunersen at the 36th International Epilepsy Congress (IEC), taking place August 30 September 3, 2025 in Lisbon, Portugal. Zorevunersen, an investigational antisense oligonucleotide, is being evaluated as a potential disease-modifying medicine for the treatment of Dravet syndrome in the global pivotal EMPEROR Phase 3 study.
"With these additional clinical data, we are developing a long-term understanding of the potential for zorevunersen to improve outcomes for patients by addressing the underlying genetic cause of Dravet syndrome," said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. "The substantial and durable reductions in seizures and improvements in cognition and behavior in patients already receiving standard anti-seizure medicines support the potential for disease modification. We look forward to sharing and discussing our latest zorevunersen data, including new analyses related to improvements in seizure free days and quality of life, with the world's leading epilepsy experts at this premier congress."
"The zorevunersen data generated to date are encouraging and support the design of the Phase 3 EMPEROR study now enrolling and dosing patients," said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. "The effects observed so far are bringing greater awareness to Dravet syndrome as a neurodevelopmental disorder while generating increasing interest in EMPEROR."
Details of the presentations at IEC are as follows:
- Title: Zorevunersen demonstrates potential as a disease-modifying therapy in patients with Dravet syndrome through durable seizure reduction and improvements in cognition, behavior, and functioning with up to 36 months of maintenance dosing in open-label extension studies
Oral Presentation Date Time: Sunday, August 31, 3:15 4:15 PM WEST/GMT +1 (10:15 11:15 AM ET)
Oral Presenter: Andreas Brunklaus, M.D., Consultant Paediatric Neurologist at the Royal Hospital for Children, Glasgow, Honorary Professor at the University of Glasgow, member of Dravet Syndrome UK's Medical Advisory Board - Title: Substantial improvements in overall seizure burden and seizure free days in patients with Dravet syndrome treated with zorevunersen: Results from Phase 1/2a and open-label extension studies
Poster Presentation Date Time: Monday, September 1, 1:45 3:15 PM WEST/ GMT +1 (8:45 10:15 AM ET)
Poster Presenter: J Helen Cross, MB ChB, Ph.D., Professor, The Prince of Wales's Chair of Childhood Epilepsy and Head of the Developmental Neuroscience Programme at University College London Great Ormond Street Institute of Child Health, Honorary Consultant in Paediatric Neurology, President of the International League Against Epilepsy
Poster Number: P351 - Title: EMPEROR Phase 3 study design: Evaluation of zorevunersen as a potential disease-modifying therapy for Dravet syndrome
Poster Presentation Date Time: Monday, September 1, 1:45 3:15 PM WEST/ GMT +1 (8:45 10:15 AM ET)
Poster Presenter: Joseph Sullivan, M.D., FAES, Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Center of Excellence at the University of California San Francisco
Poster Number: P358
Additional Company Presentation:
- Title: Emerging Concepts and Therapeutics in Dravet Syndrome: From Burden to Breakthroughs
Date Time: Tuesday, September 2, 1:55 3:05 PM WEST/ GMT +1 (8:55 10:05 ET)
Presenters: Stéphane Auvin, M.D., Ph.D., FAES, Epileptologist and Child Neurologist, Chair of the Pediatric Neurology Department at Robert Debré University Hospital Université Paris; Kelly G. Knupp, M.D., Professor, Pediatrics-Neurology, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado; and Scott Perry, M.D., Medical Director, Neurology; Co-Director, Jane and John Justin Neurosciences Center; Medical Director, Genetic Epilepsy Clinic Cook Children's Hospital
About Dravet Syndrome
Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, 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. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. 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. 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 Japan.1
About Zorevunersen
Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing 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 <18 with Dravet syndrome with a confirmed variant in the SCN1A gene not associated with gain-of-function. The trial is expected to enroll participants across the United States, Japan, United Kingdom and European Union, with participants being randomized 1:1 to receive either zorevunersen via intrathecal administration or a sham comparator for a 52-week treatment period following an 8-week baseline period. Following the completion of the study, eligible participants will be offered ongoing treatment with zorevunersen as part of an OLE study. The primary endpoint of the study is percent change from baseline in major motor seizure frequency at week 28 in patients receiving zorevunersen as compared to sham. The key secondary endpoints are the durability of effect on major motor seizure frequency and improvements in behavior and cognition as measured by Vineland-3 subdomains, including expressive communication, receptive communication, interpersonal relationships, coping skills and personal skills. Additional endpoints include safety, Clinician Global Impression of Change (CGI-C), Caregiver Global Impression of Change (CaGI-C), and the Bayley Scales of Infant Development (BSID-IV). For more information, visit https://www.emperorstudy.com/.
About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke's proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https://www.stoketherapeutics.com/.
About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.
We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media Facebook, LinkedIn, X, YouTube.
Stoke Therapeutics Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all, the timing and expected progress of clinical trials, data readouts, regulatory meetings, regulatory decisions and other presentations, and the participation of scientists associated with Stoke making presentations at IEC 2025 and the presentation of data at IEC 2025. Statements including words such as "plan," "potential," "will," "continue," "expect," or similar words and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause Stoke's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: Stokes ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Biogen were to breach or terminate the collaboration, Stoke would not obtain the anticipated financial or other benefits; the possibility that Stoke and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; Stoke's ability to protect its intellectual property; Stoke's ability to fund development activities and achieve development goals to mid-2028; and the other risks and uncertainties described under the heading "Risk Factors" in its Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and Stoke undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.
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This news release contains forward-looking statements, including, among others, relating to: the potential clinical effects of zorevunersen; the potential for zorevunersen to improve outcomes and for patients of Dravet syndrome; the potential benefits, safety and efficacy of zorevunersen and continued treatment with zorevunersen; potential regulatory discussions, submissions and approvals and the timing thereof; the treatment of Dravet syndrome; the anticipated benefits, risks and potential of Biogen's collaboration arrangements with Stoke Therapeutics; the potential of Biogen's commercial business and pipeline programs, including zorevunersen; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "assume," "believe," "contemplate," "continue," "could," "estimate," "expect," "forecast," "goal," "guidance," "hope," "intend," "may," "objective," "outlook," "plan," "possible," "potential," "predict," "project," "prospect," "should," "target," "will," "would" or the negative of these words or other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements.
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Reference:
- Based on Stoke Therapeutics' preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by Wu et al., Pediatrics, 2015.
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