\nStoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body\u2019s potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. The Company today reported financial results for the second quarter ended June 30, 2026, and provided business updates including progress of the global Phase 3 EMPEROR study, preparation for a rolling New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) and the advancement of STK-002 as a potential treatment for Autosomal Dominant Optic Atrophy (ADOA).<\/p>\n
\nStoke will participate in a pre-NDA meeting with the FDA during the second half of 2026 as the Company prepares to initiate the planned rolling NDA submission for zorevunersen in the first quarter of 2027.With the June 2026 completion of enrollment of 162 patients into EMPEROR, a Phase 3 readout is anticipated in the third quarter of 2027. These data are expected to complete the rolling U.S. NDA submission in the second half of 2027. Beyond zorevunersen, dose escalation is underway in the Phase 1 OSPREY study of STK-002 in patients with ADOA, the most common inherited optic nerve disorder. Safety and efficacy data are anticipated in the first half of 2027.<\/p>\n
\n“We have made significant progress across our business this year, including the rapid enrollment of 162 patients into the Phase 3 EMPEROR study and progression of these patients through key study milestones with the first patients now approaching the end of the 52-week treatment period,\u201d said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics. \u201cWith no treatment discontinuations to date in EMPEROR, and supportive four-year safety and efficacy data from the ongoing open-label extension studies, we have confidence in the potential of zorevunersen to change the course of Dravet syndrome. We look forward to our upcoming pre-NDA meeting with the FDA to align on the overall data package content and timing, as well as the statistical analysis plan, as we prepare to initiate our rolling U.S. NDA submission in the first quarter of 2027.”<\/p>\n
\nMr. Smith continued, \u201cBeyond Dravet syndrome, we are advancing our pipeline. Dose escalation continues in our Phase 1 study of STK-002 in patients with ADOA and initial data are anticipated in the first half of 2027 to guide future clinical development. We are also expanding our early research efforts to identify new haploinsufficient disease targets, and we recently strengthened our leadership team with the addition of Tom McCauley as Chief Scientific Officer to help guide this next phase of platform expansion and translational research. In parallel we continue to enhance our commercial capabilities, enabled by our strong financial position of approximately $420 million that will take us through to a potential U.S. launch of zorevunersen by early 2028 and support the Company\u2019s next phase of growth.”<\/p>\n
Program Highlights<\/b><\/p>\n
Dravet syndrome (zorevunersen)<\/i><\/p>\n Pipeline beyond zorevunersen<\/i><\/p>\n Second quarter 2026 financial results<\/i><\/p>\n Year-to-Date 2026 Financial Results<\/b><\/p>\n Stoke Webcast and Conference Call for Analysts and Investors About Dravet Syndrome About Zorevunersen About the Phase 1\/2a and Open-Label Extension Studies About the Phase 3 EMPEROR Study About Autosomal Dominant Optic Atrophy (ADOA) About STK-002 About the Phase 1 OSPREY Study \nAll eight planned OSPREY clinical trial sites are now active in the UK, Germany, Denmark, Italy and Austria. For more information on the OSPREY study, please visit:<\/p>\n About Stoke Therapeutics Cautionary Note Regarding Forward-Looking Statements Financial Tables Follow<\/b><\/p>\n June 30,<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n December 31,<\/b><\/p>\n<\/td>\n<\/tr>\n 2026<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n110,004<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n84,220<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n182,814<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n200,450<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n7,179<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5,936<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n14,688<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n8,736<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n1,540<\/p>\n<\/td>\n 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\n$<\/p>\n<\/td>\n \n393,966<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n418,434<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n4,794<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n4,939<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n28,613<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n41,035<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n8,523<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n11,901<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n41,930<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n57,875<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n5,652<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n6,961<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n837<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,141<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n6,489<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n8,102<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n48,419<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n65,977<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n6<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n955,419<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n849,624<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(542<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n543<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(609,336<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(497,715<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n345,547<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n352,457<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n393,966<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n418,434<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n<\/table>\n Three Months Ended June 30,<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n Six Months Ended June 30,<\/b><\/p>\n<\/td>\n<\/tr>\n 2026<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2026<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n9,325<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n13,817<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n15,554<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n172,386<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n49,501<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n25,855<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n89,174<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n58,531<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n25,253<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n15,262<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n45,227<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n29,915<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n74,754<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n41,117<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n134,401<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n88,446<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(65,429<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(27,300<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(118,847<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n83,940<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n3,503<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n3,789<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n6,899<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n6,678<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n308<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n28<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n327<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n57<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n3,811<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n3,817<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n7,226<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n6,735<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(61,618<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(23,483<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(111,621<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n90,675<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u2014<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u2014<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u2014<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,278<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(61,618<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(23,483<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(111,621<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n89,397<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(0.93<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(0.40<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(1.73<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n1.54<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(0.93<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(0.40<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(1.73<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1.50<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n66,017,895<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n58,353,855<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n64,548,862<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n58,109,622<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n66,017,895<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n58,353,855<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n64,548,862<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n59,681,472<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(61,618<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(23,483<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(111,621<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n89,397<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(427<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n200<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(1,085<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n247<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(427<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n200<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(1,085<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n247<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(62,045<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(23,283<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(112,706<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n89,644<\/p>\n<\/td>\n<\/tr>\n<\/table>\n \nReferences:<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20260803082079\/en\/<\/a><\/span><\/p>\n Stoke Media & Investor Contacts:\n
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<\/b>Stoke management will host a webcast and conference call for analysts and investors on Monday, August 3, 2026, at 4:30PM Eastern Time. The webcast will be available on the Investors & News section of Stoke\u2019s website at https:\/\/investor.stoketherapeutics.com\/<\/a>. Research analysts who plan to join the call and participate in the Q&A session may register here<\/a> to receive the dial-in details and a unique PIN. All other participants are invited to access the listen-only webcast by clicking here<\/a>. A replay of the webcast will be archived and available for at least 90 days following the event.<\/p>\n
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<\/b>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<\/i> 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 \u226550 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 infections 1<\/sup>. 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 2<\/sup>.There are no approved disease-modifying therapies for people living with Dravet syndrome.<\/p>\n
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<\/b>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 unaffected (wild-type) copy of the SCN1A <\/i>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 <\/i>gene, and China\u2019s Center for Drug Evaluation has granted zorevunersen Breakthrough Therapy Designation. Stoke has a strategic collaboration with Biogen (Nasdaq: BIIB) 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. Zorevunersen is currently in clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.<\/p>\n
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<\/b>Two Phase 1\/2a open-label, multicenter studies evaluated the effects of zorevunersen in patients with highly refractory Dravet syndrome ages 2 to 18 years (N=81). Primary endpoints were the safety profile, plasma pharmacokinetics (PK) and exposure in cerebrospinal fluid (CSF) of single and multiple doses of zorevunersen. Secondary endpoints included percentage change from baseline in major motor seizure frequency, overall clinical status (a measure of patients\u2019 overall functioning) and quality of life. The ADMIRAL Phase 1\/2a study included an exploratory endpoint to evaluate changes in neurodevelopmental status (cognition & behavior) as measured by Vineland Adaptive Behavior Scales, Third Edition (Vineland-3). The Phase 1\/2a studies were completed in November 2023. Following treatment in the Phase 1\/2a studies, eligible patients continued treatment with zorevunersen every four months in one of two OLEs. There was at least a 6-month gap between the last dose administered in the Phase 1\/2a studies and the first dose administered in the OLEs. The primary endpoints are the safety profile of multiple doses of zorevunersen. Secondary endpoints include PK parameters, percentage change from baseline in major motor seizure frequency, change in overall clinical status, and change from baseline in quality of life. Exploratory endpoints include changes in neurodevelopment status as measured by Vineland-3. The OLE studies are ongoing.<\/p>\n
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<\/b>The Phase 3 EMPEROR 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 <\/i>gene not associated with gain-of-function. Stoke completed enrollment of 162 patients in the United States, United Kingdom and Japan in June 2026, and a data readout is anticipated in the third quarter of 2027 to support the submission of a rolling New Drug Application (NDA) to the FDA. Approximately 30 additional patients are expected to enroll in Germany, Spain, France and Italy, with completion of enrollment anticipated in August 2026. Participants are 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 treatment period, eligible participants will be offered ongoing treatment with zorevunersen as part of an open-label period of the 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:\/\/clinicaltrials.gov\/study\/NCT06872125<\/a>.<\/p>\n
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<\/b>ADOA is the most common inherited optic nerve disorder, affecting approximately one in 30,000 people globally with a higher incidence of one in 10,000 in Denmark due to a founder effect. It is a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life. Severity can vary and the rate of vision loss can be difficult to predict. Approximately half of people with ADOA fail driving standards and up to 46% are registered as legally blind. More than 400 different disease-causing OPA1<\/i> variants have been reported in people diagnosed with ADOA. Currently there are no approved treatments for people living with ADOA.<\/p>\n
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<\/b>STK-002 is a proprietary antisense oligonucleotide (ASO) in clinical development for the treatment of ADOA. Stoke believes that STK-002 has the potential to be the first disease-modifying therapy for people living with ADOA. An estimated 65% to 90% of ADOA cases are caused by variants in the OPA1<\/i> gene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 <\/i>gene to restore OPA1 protein expression with the aim to maintain or improve vision in people with ADOA. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002. STK-002 has been granted orphan drug designation by the FDA as a potential new treatment for ADOA. A Phase 1 study (OSPREY) of STK-002 in people with ADOA is now underway.<\/p>\n
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<\/b>The OSPREY study is a Phase 1, dose-escalating open-label study of children and adults ages 6 to 55 who have an established diagnosis of ADOA and have a confirmed disease-causing variant in the OPA1<\/i> gene. The primary objectives for the study are to assess the safety and tolerability of single ascending doses of STK-002, as well as to determine the exposure in blood. Secondary objectives are to assess changes in visual function, ocular structure and quality of life after single doses of STK-002. The OSPREY study follows a standard dose escalation design with participants enrolled into sequential cohorts receiving increasing dose levels of STK-002. Dosing of the first cohort of patients (n=3) is complete, and dosing of the second cohort is expected to begin in August 2026. Subject to ongoing safety assessments, dosing in the third and fourth cohorts are expected to follow with a readout of safety and efficacy results anticipated in the first half of 2027. Data from the OSPREY study will help to inform potential future development of STK-002.<\/p>\n\n
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<\/b>Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body\u2019s potential with RNA medicine. Using Stoke\u2019s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke\u2019s 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\u2019s 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\u2019s proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https:\/\/www.stoketherapeutics.com\/<\/a> or follow us on LinkedIn<\/a>.<\/p>\n
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<\/b>This press release contains forward-looking statements within the meaning of the \u201csafe harbor\u201d provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the Company\u2019s quarterly results and cash runway; its future operating results and current or future financial position and liquidity; 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 potential benefits, safety and efficacy of zorevunersen; the design, timing and results of clinical studies, enrollment timelines, data readouts, regulatory submissions or decisions and other presentations for zorevunersen and STK-002; the timing and potential outcomes of meetings with regulators regarding the zorevunersen program; the Company\u2019s ability to achieve an NDA submission or approval on the expedited timeframe disclosed or at all; the ability of STK-002 to treat the underlying causes of Autosomal Dominant Optic Atrophy (ADOA) and maintain or improve vision; our expectations, plans, aspirations and goals, including those related to the potential of zorevunersen and our collaborations with Biogen and Acadia. Statements including words such as \u201canticipate,\u201d \u201cexpect,\u201d \u201cplan,\u201d \u201cwill,\u201d or \u201cmay\u201d 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 the Company\u2019s results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: the Company\u2019s ability to advance, obtain regulatory approval for, and ultimately commercialize its product candidates; that if the Company\u2019s partners were to breach or terminate their collaboration with the Company, the Company would not obtain the anticipated financial or other benefits; the possibility that the Company and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; the potential that 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; the Company\u2019s ability to protect its intellectual property; the Company\u2019s ability to fund development activities and achieve development goals into 2028; and the other risks and uncertainties described under the heading \u201cRisk Factors\u201d in the Company\u2019s Annual Report on Form 10-K for the year ended December 31, 2025, 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 the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.<\/p>\n\n
\n Stoke Therapeutics, Inc.<\/b><\/td>\n<\/tr>\n \n Condensed consolidated balance sheets<\/b><\/td>\n<\/tr>\n \n (in thousands, except share and per share amounts)<\/b><\/td>\n<\/tr>\n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n \n \n \n \n \n Assets<\/b><\/td>\n \n \n \n <\/tr>\n \n Current assets:<\/td>\n \n \n \n <\/tr>\n \n Cash and cash equivalents<\/td>\n \n \n \n \n \n \n \n \n \n Marketable securities – current<\/td>\n \n \n \n \n \n \n \n \n \n Accounts receivable<\/td>\n \n \n \n \n \n \n \n \n \n Prepaid expenses<\/td>\n \n \n \n \n \n \n \n \n \n Interest receivable<\/td>\n \n \n \n \n \n \n \n \n \n Other current assets<\/td>\n \n \n \n \n \n \n \n \n \n Total current assets<\/td>\n \n \n \n \n \n \n \n \n \n Marketable securities – long-term<\/td>\n \n \n \n \n \n \n \n \n \n Restricted cash – long-term<\/td>\n \n \n \n \n \n \n \n \n \n Operating lease right-of-use assets<\/td>\n \n \n \n \n \n \n \n \n \n Property and equipment, net<\/td>\n \n \n \n \n \n \n \n \n \n Total assets<\/td>\n \n \n \n \n \n \n \n \n \n Liabilities and stockholders\u2019 equity<\/b><\/td>\n \n \n \n <\/tr>\n \n Current liabilities:<\/td>\n \n \n \n <\/tr>\n \n Accounts payable<\/td>\n \n \n \n \n \n \n \n \n \n Accrued and other current liabilities<\/td>\n \n \n \n \n \n \n \n \n \n Deferred revenue – current portion<\/td>\n \n \n \n \n \n \n \n \n \n Total current liabilities<\/td>\n \n \n \n \n \n \n \n \n \n Deferred revenue – net of current portion<\/td>\n \n \n \n \n \n \n \n \n \n Other long-term liabilities<\/td>\n \n \n \n \n \n \n \n \n \n Total long-term liabilities<\/td>\n \n \n \n \n \n \n \n \n \n Total liabilities<\/td>\n \n \n \n \n \n \n \n \n \n Stockholders\u2019 equity<\/td>\n \n \n \n <\/tr>\n \n Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 62,402,595 and 58,921,999 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively<\/td>\n \n \n \n \n \n \n \n \n \n Additional paid-in capital<\/td>\n \n \n \n \n \n \n \n \n \n Accumulated other comprehensive (loss) income<\/td>\n \n \n \n \n \n \n \n \n \n Accumulated deficit<\/td>\n \n \n \n \n \n \n \n \n \n Total stockholders\u2019 equity<\/td>\n \n \n \n \n \n \n \n \n \n Total liabilities and stockholders\u2019 equity<\/td>\n \n \n \n \n \n \n \n \n \n
\n Stoke Therapeutics, Inc.<\/b><\/td>\n<\/tr>\n \n Condensed consolidated statements of operations and comprehensive (loss) income<\/b><\/td>\n<\/tr>\n \n (in thousands, except share and per share amounts)<\/b><\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Revenue<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Operating expenses:<\/td>\n \n \n \n \n \n \n \n <\/tr>\n \n Research and development<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Sales, general and administrative<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Total operating expenses<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n (Loss) income from operations<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Other income (expense):<\/td>\n \n \n \n \n \n \n \n <\/tr>\n \n Interest income (expense), net<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Other income<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Total other income (expense)<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n (Loss) income before income taxes<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Provision for income taxes<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Net (loss) income<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Net (loss) income per share:<\/td>\n \n \n \n \n \n \n \n <\/tr>\n \n Basic<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Diluted<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Weighted-average common shares outstanding:<\/td>\n \n \n \n \n \n \n \n <\/tr>\n \n Basic<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Diluted<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Comprehensive (loss) income:<\/td>\n \n \n \n \n \n \n \n <\/tr>\n \n Net (loss) income<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Other comprehensive (loss) gain:<\/td>\n \n \n \n \n \n \n \n <\/tr>\n \n Unrealized (loss) gain on marketable securities<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Total other comprehensive (loss) gain<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n Comprehensive (loss) income<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n
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Susan Willson
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Vice President, Corporate Communications
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swillson@stoketherapeutics.com
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415-509-8202<\/p>\n
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