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Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights
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Collaboration broadens Biogen’s rare disease pipeline and leverages global expertise commercializing high-value, disease-modifying medicines for rare genetic diseases
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Pivotal Phase 3 EMPEROR study of zorevunersen on track to initiate in Q2 2025 with an anticipated readout in 2H 2027
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Stoke to receive $165M upfront, shared development costs and is eligible to receive up to $385M in milestones as well as royalties
CAMBRIDGE, Mass. and BEDFORD, Mass., Feb. 18, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Stoke Therapeutics, Inc. (Nasdaq: STOK) today announced a collaboration for the development and commercialization of zorevunersen, a potential first-in-class disease modifying medicine in development for the treatment of Dravet syndrome, in all territories outside the United States, Canada, and Mexico. Zorevunersen is an investigational antisense oligonucleotide (ASO) that targets the SCN1A gene, the underlying cause of most cases of Dravet syndrome. Stoke recently announced plans to initiate a global Phase 3 registrational study of zorevunersen (EMPEROR) following successful alignment with regulatory agencies in the United States, Europe, and Japan. The study is on track to initiate in the second quarter of 2025, with a pivotal data readout expected in the second half of 2027, which is anticipated to support global regulatory filings.
Significant unmet treatment needs remain for patients with Dravet syndrome, a severe, genetic developmental and epileptic encephalopathy characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. There are no approved disease-modifying therapies for Dravet syndrome, which is difficult to treat and has a poor long-term prognosis. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S., UK, EU-4 and Japan.1
“With Biogen’s deep experience in neurology and track record of success in commercializing high-value disease-modifying medicines for rare genetic diseases globally, we aim to lead the treatment of Dravet syndrome into a new era by delivering zorevunersen to all patients who could benefit,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “Additionally, this collaboration provides cash flows, that when combined with Stoke’s financial position, support the company through to mid-2028.”
“This collaboration broadens our late-stage pipeline with the addition of a Phase 3-ready disease modifying investigational medicine and allows us to leverage our rare disease product commercialization expertise and global footprint,” said Priya Singhal, M.D., M.P.H., Head of Development at Biogen. “The reductions in seizures in patients already receiving standard of care medicines, together with the improvements in multiple measures of cognition and behavior, demonstrate the potential of zorevunersen as the first disease modifying medicine that addresses the underlying cause of Dravet syndrome.”
Collaboration Terms
Stoke will continue to lead global development and retains exclusive development and commercialization rights for zorevunersen in the United States, Canada, and Mexico. Biogen receives exclusive rights to commercialize zorevunersen in the rest of the world. Upon closing of this transaction, Stoke will receive an upfront payment of $165 million. The parties will share external clinical development costs for zorevunersen (30 percent Biogen; 70 percent Stoke). Additionally, Stoke may receive up to $385 million in development and commercial milestone payments. Stoke will also be eligible to receive tiered royalties ranging from low double digits to high teens on potential net sales in the Biogen territory.
Stoke has also granted Biogen an option to license rights outside of the United States, Canada, and Mexico to certain future follow-on ASO products targeting SCN1A, in exchange for separate milestone, cost sharing, and royalty considerations.
Zorevunersen is a Potential First-in-Class Disease Modifying Medicine for Dravet Syndrome
Zorevunersen has been granted FDA Breakthrough Therapy Designation based on preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapy on clinically-significant endpoint(s). Data from Phase 1/2a and open-label extension (OLE) studies of zorevunersen showed that patients treated with zorevunersen experienced substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior, which support advancement to a global registrational Phase 3 study with potential for disease modification. Zorevunersen has been generally well tolerated across the studies.
About Dravet Syndrome
Dravet syndrome is a severe developmental and epileptic encephalopathy 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., 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.
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 expected to enter Phase 3 development in 2025. 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 the Company’s proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, 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.
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1.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.