\nThe Company also announced an update on progress of the global Phase 3 EMPEROR study. Enrollment of approximately 150 patients in the U.S., UK and Japan is expected to complete in June 2026 to support a data readout in mid-2027. These data are anticipated to complete the rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) planned to initiate in the first quarter of 2027.<\/p>\n
\n\u201cThese new 4-year OLE data suggest that zorevunersen may change the course of Dravet syndrome by providing children with durable reductions in seizures and the possibility of a more neurotypical development path. Together with the Phase 1\/2a results, the ongoing extension studies offer 5 years of clinical data, providing a unique opportunity to understand the long-term benefits and safety of zorevunersen,\u201d said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics.<\/p>\n
\nMr. Smith continued, \u201cWe look forward to results from our pivotal Phase 3 study, which is on track to complete enrollment of approximately 150 patients in June to support a data readout in mid-2027. As we continue to see this study through to completion, our focus turns increasingly to commercial preparedness and bringing zorevunersen to patients while also building our pipeline for the future. All of this is supported by our strong financial position taking us through to a potential U.S. launch in early 2028.\u201d<\/p>\n
Program Highlights<\/b><\/p>\n
Dravet syndrome (zorevunersen)<\/i><\/p>\n Pipeline beyond zorevunersen<\/i><\/p>\n First Quarter 2026 Financial Results<\/b><\/p>\n Stoke Webcast and Conference Call for Analysts and Investors<\/b><\/p>\n \nStoke management will host a webcast and conference call for analysts and investors on Thursday, May 7, 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 About Dravet Syndrome<\/b><\/p>\n \nDravet 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 About Zorevunersen<\/b><\/p>\n \nZorevunersen 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. 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. Zorevunersen is currently in clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.<\/p>\n About the Phase 1\/2a and Open-Label Extension Studies<\/b><\/p>\n \nTwo 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 About the Phase 3 EMPEROR Study<\/b><\/p>\n \nThe 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 expects to complete enrollment of approximately 150 patients in the United States, United Kingdom and Japan in June 2026, with a data readout on track for mid-2027 to support the submission of a New Drug Application (NDA) to the FDA. At least 20 additional patients are expected to enroll in Germany, Spain, France and Italy, with completion of enrollment anticipated in Q3 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 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:\/\/clinicaltrials.gov\/study\/NCT06872125<\/a>.<\/p>\n About Autosomal Dominant Optic Atrophy (ADOA)<\/b><\/p>\n \nADOA 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 About STK-002<\/b><\/p>\n \nSTK-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 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 About the Phase 1 OSPREY Study<\/b><\/p>\n \nThe 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. Dose escalation of the first four cohorts will continue through 2026 and early 2027, pending safety and tolerability assessments. Data from the OSPREY study will help to inform potential future development of STK-002. The OSPREY study is actively recruiting in the United Kingdom, Germany, Denmark and Austria. Additional European sites are expected to activate in the coming months.<\/p>\n \nFor more information on the OSPREY study, please visit:<\/p>\n About Stoke Therapeutics<\/b><\/p>\n \nStoke 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>.<\/p>\n Cautionary Note Regarding Forward-Looking Statements<\/b><\/p>\n \nThis 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 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 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 risk 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 development goals into 2028 and through potential U.S. launch in early 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 Financial Tables Follow<\/b><\/p>\n March 31,<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n December 31,<\/b><\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n 2026<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n155,659<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n84,220<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n187,886<\/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 \n4,451<\/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 \n10,115<\/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 \n1,690<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,969<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n6,880<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n4,389<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n366,681<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n305,700<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n67,481<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n106,260<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n3,227<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n227<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n2,493<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n3,101<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n3,395<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n3,146<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n443,277<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n418,434<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n6,839<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n4,939<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n23,901<\/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 \n10,066<\/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$<\/p>\n<\/td>\n \n40,806<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n57,875<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n6,603<\/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 \n991<\/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 \n7,594<\/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$<\/p>\n<\/td>\n \n48,400<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/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 \n942,704<\/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(115<\/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(547,718<\/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$<\/p>\n<\/td>\n \n394,877<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n352,457<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n443,277<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n418,434<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n<\/table>\n Three Months Ended March 31,<\/b><\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n 2026<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n6,229<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n158,569<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n39,673<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n32,676<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n19,974<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n14,653<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n59,647<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n47,329<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(53,418<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n111,240<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n3,397<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n2,889<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n18<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n28<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n3,415<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n2,917<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(50,003<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n114,157<\/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 \n1,278<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(50,003<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n112,879<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(0.79<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n1.95<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(0.79<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1.90<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n63,063,507<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n57,862,674<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n63,063,507<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n59,398,600<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(50,003<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n112,879<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n(658<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n47<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(658<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n47<\/p>\n<\/td>\n<\/tr>\n \n$<\/p>\n<\/td>\n \n(50,661<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n112,926<\/p>\n<\/td>\n<\/tr>\n<\/table>\n \nReferences:<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20260507458327\/en\/<\/a><\/span><\/p>\n Stoke Media & Investor Contacts: \nDoug Snow KEYWORDS:<\/b> Massachusetts United States North America<\/p>\n INDUSTRY KEYWORDS:<\/b> Science Biotechnology Research Pharmaceutical Health FDA Genetics Clinical Trials<\/p>\n\n
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\n Stoke Therapeutics, Inc. and subsidiary<\/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 \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, 62,240,347 and 58,921,999 shares issued and outstanding as of March 31, 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. and subsidiary<\/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 \u00a0<\/td>\n<\/tr>\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 Operating expenses:<\/td>\n \n \n \n <\/tr>\n \n Research and development<\/td>\n \n \n \n \n \n \n \n \n Sales, general and administrative<\/td>\n \n \n \n \n \n \n \n \n Total operating expenses<\/td>\n \n \n \n \n \n \n \n \n (Loss) income from operations<\/td>\n \n \n \n \n \n \n \n \n Other income (expense):<\/td>\n \n \n \n <\/tr>\n \n Interest income (expense), net<\/td>\n \n \n \n \n \n \n \n \n Other income<\/td>\n \n \n \n \n \n \n \n \n Total other income (expense)<\/td>\n \n \n \n \n \n \n \n \n (Loss) income before income taxes<\/td>\n \n \n \n \n \n \n \n \n Provision for income taxes<\/td>\n \n \n \n \n \n \n \n \n Net (loss) income<\/td>\n \n \n \n \n \n \n \n \n Net (loss) income per share:<\/td>\n \n \n \n <\/tr>\n \n Basic<\/td>\n \n \n \n \n \n \n \n \n Diluted<\/td>\n \n \n \n \n \n \n \n \n Weighted-average common shares outstanding:<\/td>\n \n \n \n <\/tr>\n \n Basic<\/td>\n \n \n \n \n \n \n \n \n Diluted<\/td>\n \n \n \n \n \n \n \n \n Comprehensive (loss) income:<\/td>\n \n \n \n <\/tr>\n \n Net (loss) income<\/td>\n \n \n \n \n \n \n \n \n Other comprehensive (loss) gain:<\/td>\n \n \n \n <\/tr>\n \n Unrealized (loss) gain on marketable securities<\/td>\n \n \n \n \n \n \n \n \n Total other comprehensive (loss) gain<\/td>\n \n \n \n \n \n \n \n \n Comprehensive (loss) income<\/td>\n \n \n \n \n \n \n \n \n
<\/p>\n
\n<\/b>
Susan Willson
\n
Vice President, Corporate Communications
\n
swillson@stoketherapeutics.com
\n<\/a>
415-509-8202<\/p>\n
\n
Director, Communications & Investor Relations
\n
IR@stoketherapeutics.com
\n<\/a>
508-642-6485<\/p>\n
![\"Logo\"](\"https:\/\/mms.businesswire.com\/media\/20260507458327\/en\/736143\/3\/Stoke_Logo1_-_CMYK_50percent.jpg\"){kind=logo}
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