{"success":true,"data":{"pressRelease":{"id":"26466","rtpr_id":"nBw6Cr3RMa","ticker":"STOK","exchange":"NASDAQ","all_tickers":["STOK"],"title":"Stoke Therapeutics Announces First Quarter 2026 Financial Results and Provides Business Updates","author":"Business Wire","published_at":"2026-05-07T20:01:00.552Z","article_body":"Stoke Therapeutics Announces First Quarter 2026 Financial Results and Provides\nBusiness Updates\n\n– New 4-year longitudinal data from the Phase 1/2a open-label extension\n(OLE) studies provide additional support for the disease-modifying potential\nof zorevunersen, an investigational medicine for the treatment of Dravet\nsyndrome –\n\n– Statistically significant improvements in cognition and behavior\ndemonstrated at 1, 2, 3 and 4 years of treatment compared to OLE baseline, in\naddition to continued durability in seizure reductions –\n\n– Zorevunersen generally well tolerated, with some patients treated for more\nthan 5 years –\n\n– Enrollment of approximately 150 patients into the Phase 3 EMPEROR study\nexpected to complete in June 2026 to support a data readout in mid-2027; these\ndata are anticipated to complete the rolling U.S. NDA submission planned to\ninitiate in first quarter 2027 –\n\n– As of March 31, 2026, the Company had $411.0 million in cash, cash\nequivalents and marketable securities expected to fund operations into 2028,\nincluding $80.7 million raised through selective ATM sales in first quarter\n2026 –\n\n– Webcast and conference call for analysts and investors at 4:30PM Eastern\nTime today –\n\nStoke Therapeutics, Inc.\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.stoketherapeutics.com%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=Stoke+Therapeutics%2C+Inc.&index=1&md5=c7fef1a29617047f59c6c75898811167)\n(Nasdaq: STOK) is a biotechnology company dedicated to restoring protein\nexpression by harnessing the body’s potential with RNA medicine and has a\nlead investigational medicine, zorevunersen, in development with Biogen\n(Nasdaq: BIIB) as a first-in-class potential disease-modifying treatment for\nDravet syndrome. The Company today reported financial results for the first\nquarter ended March 31, 2026, and announced new 4-year longitudinal data from\nthe ongoing Phase 1/2a open-label extension (OLE) studies that provide\nadditional support for zorevunersen as a potential disease-modifying treatment\nfor Dravet syndrome. Statistically significant improvements were demonstrated\nin cognition and behavior at 1, 2, 3 and 4 years of treatment compared to OLE\nbaseline. Reductions in major motor seizure frequency were observed through 4\nyears of treatment in patients taking standard anti-seizure medicines (ASMs).\nZorevunersen continues to be generally well tolerated, with some patients\ntreated for more than 5 years in the Phase 1/2a and ongoing OLE studies.\n\nThe Company also announced an update on progress of the global Phase 3 EMPEROR\nstudy. Enrollment of approximately 150 patients in the U.S., UK and Japan is\nexpected to complete in June 2026 to support a data readout in mid-2027. These\ndata are anticipated to complete the rolling New Drug Application (NDA) to the\nU.S. Food and Drug Administration (FDA) planned to initiate in the first\nquarter of 2027.\n\n“These new 4-year OLE data suggest that zorevunersen may change the course\nof Dravet syndrome by providing children with durable reductions in seizures\nand the possibility of a more neurotypical development path. Together with the\nPhase 1/2a results, the ongoing extension studies offer 5 years of clinical\ndata, providing a unique opportunity to understand the long-term benefits and\nsafety of zorevunersen,” said Ian F. Smith, Chief Executive Officer and\nDirector of Stoke Therapeutics.\n\nMr. Smith continued, “We look forward to results from our pivotal Phase 3\nstudy, which is on track to complete enrollment of approximately 150 patients\nin June to support a data readout in mid-2027. As we continue to see this\nstudy through to completion, our focus turns increasingly to commercial\npreparedness and bringing zorevunersen to patients while also building our\npipeline for the future. All of this is supported by our strong financial\nposition taking us through to a potential U.S. launch in early 2028.”\n\nProgram Highlights\n\nDravet syndrome (zorevunersen)\n\n\n * New 4-year safety and efficacy data from OLE studies: Following treatment in\nthe Phase 1/2a studies 93% (75/81) patients continued treatment in one of two\nOLE studies. As of the 4-year data cutoff, 77% (58/75) patients remained in\nthese studies. The new 4-year data announced today showed that patients\ntreated with zorevunersen on top of standard anti-seizure medicines continued\nto experience durable reductions in seizures and ongoing improvements in\ncognition and behavior. Zorevunersen continues to be generally well tolerated,\nwith some patients treated for more than 5 years in the Phase 1/2a and ongoing\nOLE studies in which more than 850 doses have been administered. Elevated CSF\nprotein lab values occurred in approximately 94% of patients of which 59% have\nbeen classified as a treatment-emergent adverse event. Importantly, no serious\nor severe clinical manifestations have been associated with CSF protein\nelevations. There have been no reports of hydrocephalus.\n\n * Pivotal Phase 3 EMPEROR study progress:\n\n\n* U.S., UK and Japan: New patient entry into screening is now closed. As of\nMay\n5, 2026, approximately 130 patients had been randomized to zorevunersen or\nsham. The remaining patients required to achieve the planned enrollment of\napproximately 150 patients into this cohort are progressing through the 8-week\nscreening period. Following successful completion of screening, these patients\nwill be randomized to treatment with zorevunersen or a sham control\nadministered via lumbar puncture (LP). The final patient is expected to be\nrandomized in June 2026. These data are anticipated to be the final data\nrequired for completion of the planned rolling U.S. NDA submission.\n\n * Europe (Germany, France, Spain and Italy): 15 out of 16 sites are activated\nand screening is underway for at least 20 additional patients. Enrollment is\nexpected to complete in the third quarter of 2026.\n\n\n\n\n * Medical and scientific publications and communications continued to drive\nawareness of Dravet syndrome and the need for disease modification.\n\n\n* In April, Stoke presented data at the American Academy of Neurology (AAN)\nAnnual Meeting, the world’s largest gathering of neurologists.\n\n * In March, \nThe New England Journal of Medicine (NEJM)\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.nejm.org%2Fdoi%2Ffull%2F10.1056%2FNEJMoa2506295&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=The+New+England+Journal+of+Medicine+%28NEJM%29&index=2&md5=374864f30e59beb72ac2aa47b1e1e88a)\n\npublished zorevunersen data from the Phase 1/2a and OLE studies. An\nindependent editorial that discussed the underlying genetic cause of Dravet\nsyndrome and the disease-modifying potential of zorevunersen accompanied the\nmanuscript.\n\n\n\n\nPipeline beyond zorevunersen\n\n\n * In February, \nthe first patient was dosed\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Finvestor.stoketherapeutics.com%2Fnews-releases%2Fnews-release-details%2Fstoke-therapeutics-announces-first-patient-dosed-phase-1-study&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=the+first+patient+was+dosed&index=3&md5=d27f67614858bd4b5a311183d4e7d42a)\n\nin the Phase 1 OSPREY study of STK-002 for the treatment of Autosomal Dominant\nOptic Atrophy (ADOA), the most common inherited optic nerve disorder. To date,\ntwo patients have been dosed and recruitment is ongoing at 7 sites across the\nUK, Germany, Denmark and Austria. Dose escalation of the first four cohorts\nwill continue through 2026 and early 2027, pending safety and tolerability\nassessments.\n\n * Lead optimization is underway to identify a clinical candidate for the\ntreatment of SYNGAP1 in 2026. SYNGAP1 is a severe and rare genetic\nneurodevelopmental disease.\n\nFirst Quarter 2026 Financial Results\n\n\n * As of March 31, 2026, the Company had $411.0 million in cash, cash equivalents\nand marketable securities expected to fund operations into 2028. This includes\napproximately $80.7 million of proceeds from the sale of 2.6 million shares of\ncommon stock from selective use of the ATM facility (Controlled Equity\nOffering Sales Agreement) during the first quarter of 2026.\n\n * Revenue recognized for the three months ended March 31, 2026, was $6.2\nmillion, a decrease from $158.6 million for the same period in 2025. The\ndecrease in revenue is primarily driven by the 2025 recognition of $150.8\nmillion related to the IP license performance obligation related to the Biogen\nAgreement outside of the U.S., Canada and Mexico.\n\n * Net loss for the three months ended March 31, 2026, was $50.0 million, or\n$0.79 per share, compared to a net income of $112.9 million, or $1.90 per\ndiluted share, for the same period in 2025.\n\n * Research and development expenses for the three months ended March 31, 2026,\nwere $39.7 million, compared to $32.7 million for the same period in 2025. The\nincrease of $7.0 million was driven by an increase in activities and personnel\nexpenses to support the advancement of zorevunersen.\n\n * Sales, general and administrative expenses for the three months ended March\n31, 2026, increased to $20.0 million from $14.7 million for the same period in\n2025. The increase of $5.3 million was driven by an increase in personnel and\nlaunch readiness expenses.\n\nStoke Webcast and Conference Call for Analysts and Investors\n\nStoke management will host a webcast and conference call for analysts and\ninvestors on Thursday, May 7, 2026, at 4:30PM Eastern Time. The webcast will\nbe available on the Investors & News section of Stoke’s website at\nhttps://investor.stoketherapeutics.com/\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Finvestor.stoketherapeutics.com%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Finvestor.stoketherapeutics.com%2F&index=4&md5=b9afa1a7d17a9e60cab4dfa02acfc2ff)\n. Research analysts who plan to join the call and participate in the Q&A\nsession may register here\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fregistrations.events%2Fdirect%2FNTM426396&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=here&index=5&md5=76141f794f88a9168d2e25920c78e611)\nto receive the dial-in details and a unique PIN. All other participants are\ninvited to access the listen-only webcast by clicking here\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fedge.media-server.com%2Fmmc%2Fp%2Fdj697s4q%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=here&index=6&md5=6a547799f724985cdaf9b49f1cfcd149)\n. A replay of the webcast will be archived and available for at least 90 days\nfollowing the event.\n\nAbout Dravet Syndrome\n\nDravet syndrome is a severe developmental and epileptic encephalopathy (DEE)\ncharacterized by recurrent seizures as well as significant cognitive and\nbehavioral impairments. Most cases of Dravet are caused by mutations in one\ncopy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in\nneuronal cells in the brain. Even when treated with the best available\nanti-seizure medicines (ASMs), up to 57 percent of patients with Dravet\nsyndrome do not achieve ≥50 percent reduction in seizure frequency.\nComplications of the disease often contribute to a poor quality of life for\npatients and their caregivers. Developmental and cognitive impairments often\ninclude intellectual disability, developmental delays, movement and balance\nissues, language and speech disturbances, growth defects, sleep abnormalities,\ndisruptions of the autonomic nervous system and mood disorders. Compared with\nthe general epilepsy population, people living with Dravet syndrome have a\nhigher risk of sudden unexpected death in epilepsy, or SUDEP; up to 20 percent\nof children and adolescents with Dravet syndrome die before adulthood due to\nSUDEP, prolonged seizures, seizure-related accidents or infections( 1). Dravet\nsyndrome occurs globally and is not concentrated in a particular geographic\narea or ethnic group. Currently, it is estimated that up to 38,000 people are\nliving with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan( 2).(\n)There are no approved disease-modifying therapies for people living with\nDravet syndrome.\n\nAbout Zorevunersen\n\nZorevunersen is an investigational antisense oligonucleotide that is designed\nto treat the underlying cause of Dravet syndrome by increasing functional\nNaV1.1 protein production in brain cells from the unaffected (wild-type) copy\nof the SCN1A gene. This highly differentiated mechanism of action aims to\nreduce seizure frequency beyond what has been achieved with anti-seizure\nmedicines and to improve neurodevelopment, cognition and behavior.\nZorevunersen has demonstrated the potential for disease modification and has\nbeen granted orphan drug designation by the FDA and the EMA. The FDA has also\ngranted zorevunersen rare pediatric disease designation and Breakthrough\nTherapy Designation for the treatment of Dravet syndrome with a confirmed\nmutation not associated with gain-of-function, in the SCN1A gene. Stoke has a\nstrategic collaboration with Biogen to develop and commercialize zorevunersen\nfor Dravet syndrome. Under the collaboration, Stoke retains exclusive rights\nfor zorevunersen in the United States, Canada, and Mexico; Biogen receives\nexclusive rest of world commercialization rights. Zorevunersen is currently in\nclinical development, and its safety and efficacy have not been evaluated by\nany regulatory authority.\n\nAbout the Phase 1/2a and Open-Label Extension Studies\n\nTwo Phase 1/2a open-label, multicenter studies evaluated the effects of\nzorevunersen in patients with highly refractory Dravet syndrome ages 2 to 18\nyears (N=81). Primary endpoints were the safety profile, plasma\npharmacokinetics (PK) and exposure in cerebrospinal fluid (CSF) of single and\nmultiple doses of zorevunersen. Secondary endpoints included percentage change\nfrom baseline in major motor seizure frequency, overall clinical status (a\nmeasure of patients’ overall functioning) and quality of life. The ADMIRAL\nPhase 1/2a study included an exploratory endpoint to evaluate changes in\nneurodevelopmental status (cognition & behavior) as measured by Vineland\nAdaptive Behavior Scales, Third Edition (Vineland-3). The Phase 1/2a studies\nwere completed in November 2023. Following treatment in the Phase 1/2a\nstudies, eligible patients continued treatment with zorevunersen every four\nmonths in one of two OLEs. There was at least a 6-month gap between the last\ndose administered in the Phase 1/2a studies and the first dose administered in\nthe OLEs. The primary endpoints are the safety profile of multiple doses of\nzorevunersen. Secondary endpoints include PK parameters, percentage change\nfrom baseline in major motor seizure frequency, change in overall clinical\nstatus, and change from baseline in quality of life. Exploratory endpoints\ninclude changes in neurodevelopment status as measured by Vineland-3. The OLE\nstudies are ongoing.\n\nAbout the Phase 3 EMPEROR Study\n\nThe Phase 3 EMPEROR Study (NCT06872125) is a global, double-blind,\nsham-controlled study evaluating the efficacy, safety and tolerability of\nzorevunersen in children ages 2 to <18 with Dravet syndrome with a\nconfirmed variant in the SCN1A gene not associated with gain-of-function.\nStoke expects to complete enrollment of approximately 150 patients in the\nUnited States, United Kingdom and Japan in June 2026, with a data readout on\ntrack for mid-2027 to support the submission of a New Drug Application (NDA)\nto the FDA. At least 20 additional patients are expected to enroll in Germany,\nSpain, France and Italy, with completion of enrollment anticipated in Q3 2026.\nParticipants are randomized 1:1 to receive either zorevunersen via intrathecal\nadministration or a sham comparator for a 52-week treatment period following\nan 8-week baseline period. Following the completion of the study treatment\nperiod, eligible participants will be offered ongoing treatment with\nzorevunersen as part of an OLE study. The primary endpoint of the study is\npercent change from baseline in major motor seizure frequency at week 28 in\npatients receiving zorevunersen as compared to sham. The key secondary\nendpoints are the durability of effect on major motor seizure frequency and\nimprovements in behavior and cognition as measured by Vineland-3 subdomains,\nincluding expressive communication, receptive communication, interpersonal\nrelationships, coping skills and personal skills. Additional endpoints include\nsafety, Clinician Global Impression of Change (CGI-C), Caregiver Global\nImpression of Change (CaGI-C) and the Bayley Scales of Infant Development\n(BSID-IV). For more information, visit\nhttps://clinicaltrials.gov/study/NCT06872125\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT06872125%3Fcond%3DDravet%2520Syndrome%26viewType%3DCard%26term%3Demperor%26rank%3D1&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT06872125&index=7&md5=6e4135aa90e2f6b63a73222042973226)\n.\n\nAbout Autosomal Dominant Optic Atrophy (ADOA)\n\nADOA is the most common inherited optic nerve disorder, affecting\napproximately one in 30,000 people globally with a higher incidence of one in\n10,000 in Denmark due to a founder effect. It is a rare disease that causes\nprogressive and irreversible vision loss in both eyes starting in the first\ndecade of life. Severity can vary and the rate of vision loss can be difficult\nto predict. Approximately half of people with ADOA fail driving standards and\nup to 46% are registered as legally blind. More than 400 different\ndisease-causing OPA1 variants have been reported in people diagnosed with\nADOA. Currently there are no approved treatments for people living with ADOA.\n\nAbout STK-002\n\nSTK-002 is a proprietary antisense oligonucleotide (ASO) in clinical\ndevelopment for the treatment of ADOA. Stoke believes that STK-002 has the\npotential to be the first disease-modifying therapy for people living with\nADOA. An estimated 65% to 90% of ADOA cases are caused by variants in the OPA1\ngene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein\nexpression and disease manifestation. STK-002 is designed to upregulate OPA1\nprotein expression by leveraging the non-mutant (wild-type) copy of the OPA1\ngene to restore OPA1 protein expression with the aim to maintain or improve\nvision in people with ADOA. Stoke has generated preclinical data demonstrating\nproof-of-mechanism and proof-of-concept for STK-002. STK-002 has been granted\norphan drug designation by FDA as a potential new treatment for ADOA. A Phase\n1 study (OSPREY) of STK-002 in people with ADOA is now underway.\n\nAbout the Phase 1 OSPREY Study\n\nThe OSPREY study is a Phase 1, dose-escalating open-label study of children\nand adults ages 6 to 55 who have an established diagnosis of ADOA and have a\nconfirmed disease-causing variant in the OPA1 gene. The primary objectives for\nthe study are to assess the safety and tolerability of single ascending doses\nof STK-002, as well as to determine the exposure in blood. Secondary\nobjectives are to assess changes in visual function, ocular structure and\nquality of life after single doses of STK-002. The OSPREY study follows a\nstandard dose escalation design with participants enrolled into sequential\ncohorts receiving increasing dose levels of STK-002. Dose escalation of the\nfirst four cohorts will continue through 2026 and early 2027, pending safety\nand tolerability assessments. Data from the OSPREY study will help to inform\npotential future development of STK-002. The OSPREY study is actively\nrecruiting in the United Kingdom, Germany, Denmark and Austria. Additional\nEuropean sites are expected to activate in the coming months.\n\nFor more information on the OSPREY study, please visit:\n\n\n * https://www.ospreyclinicaltrial.com/\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.ospreyclinicaltrial.com%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Fwww.ospreyclinicaltrial.com%2F&index=8&md5=08667cc238c5df578cf4ee47bcf7d83d)\n\n * https://www.isrctn.com/ISRCTN41725621\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.isrctn.com%2FISRCTN41725621&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Fwww.isrctn.com%2FISRCTN41725621&index=9&md5=ec6fb87e856c36a7e43de862a55d679c)\n\nAbout Stoke Therapeutics\n\nStoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to\nrestoring protein expression by harnessing the body’s potential with RNA\nmedicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear\nGene Output) approach, Stoke is developing antisense oligonucleotides (ASOs)\nto selectively restore naturally-occurring protein levels. Stoke’s first\nmedicine in development, zorevunersen, has demonstrated the potential for\ndisease modification in patients with Dravet syndrome and is currently being\nevaluated in a Phase 3 study. Stoke’s initial focus are diseases of the\ncentral nervous system and the eye that are caused by a loss of ~50% of normal\nprotein levels (haploinsufficiency). Proof of concept has been demonstrated in\nother organs, tissues, and systems, supporting broad potential for Stoke’s\nproprietary approach. Stoke is headquartered in Bedford, Massachusetts. For\nmore information, visit https://www.stoketherapeutics.com/\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.stoketherapeutics.com%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Fwww.stoketherapeutics.com%2F&index=10&md5=10ebc704bd2cecf44bfb9eaaa83a686c)\n.\n\nCautionary Note Regarding Forward-Looking Statements\n\nThis press release contains forward-looking statements within the meaning of\nthe “safe harbor” provisions of the Private Securities Litigation Reform\nAct of 1995, including, but not limited to: the Company’s quarterly results\nand cash runway; its future operating results and current or future financial\nposition and liquidity; the ability of zorevunersen to treat the underlying\ncauses of Dravet syndrome and reduce seizures or show improvements in behavior\nand cognition at the indicated dosing levels or at all; the design, timing and\nresults of clinical studies, enrollment timelines, data readouts, regulatory\nsubmissions or decisions and other presentations for zorevunersen and STK-002;\nthe timing and potential outcomes of meetings with regulators regarding the\nzorevunersen program; the ability of STK-002 to treat the underlying causes of\nAutosomal Dominant Optic Atrophy (ADOA) and maintain or improve vision; our\nexpectations, plans, aspirations and goals, including those related to the\npotential of zorevunersen and our collaborations with Biogen and Acadia.\nStatements including words such as “anticipate,” “expect,” “plan,”\n“will,” or “may” and statements in the future tense are\nforward-looking statements. These forward-looking statements involve risks and\nuncertainties, as well as assumptions, which, if they prove incorrect or do\nnot fully materialize, could cause the Company’s results to differ\nmaterially from those expressed or implied by such forward-looking statements,\nincluding, but not limited to, risks and uncertainties related to: the\nCompany’s ability to advance, obtain regulatory approval for, and ultimately\ncommercialize its product candidates; that if the Company’s partners were to\nbreach or terminate their collaboration with the Company, the Company would\nnot obtain the anticipated financial or other benefits; the possibility that\nthe Company and Biogen may not be successful in their development of\nzorevunersen and that, even if successful, they may be unable to successfully\ncommercialize zorevunersen; the risk that positive results in a clinical trial\nmay not be replicated in subsequent trials or successes in early stage\nclinical trials may not be predictive of results in later stage trials; the\ndevelopment goals into 2028 and through potential U.S. launch in early 2028;\nand the other risks and uncertainties described under the heading “Risk\nFactors” in the Company’s Annual Report on Form 10-K for the year ended\nDecember 31, 2025, its quarterly reports on Form 10-Q, and the other documents\nit files with the Securities and Exchange Commission. These forward-looking\nstatements speak only as of the date of this press release, and the Company\nundertakes no obligation to revise or update any forward-looking statements to\nreflect events or circumstances after the date hereof.\n\nFinancial Tables Follow\n Stoke Therapeutics, Inc. and subsidiary \n Condensed consolidated balance sheets \n (in thousands, except share and per share amounts) \n   \n March 31,   December 31, \n   2026       2025   \n Assets \n Current assets: \n Cash and cash equivalents $ 155,659   $ 84,220   \n Marketable securities - current   187,886     200,450   \n Accounts receivable   4,451     5,936   \n Prepaid expenses   10,115     8,736   \n Interest receivable   1,690     1,969   \n Other current assets   6,880     4,389   \n Total current assets $ 366,681   $ 305,700   \n Marketable securities - long-term   67,481     106,260   \n Restricted cash - long-term   3,227     227   \n Operating lease right-of-use assets   2,493     3,101   \n Property and equipment, net   3,395     3,146   \n Total assets $ 443,277   $ 418,434   \n Liabilities and stockholders’ equity \n Current liabilities: \n Accounts payable $ 6,839   $ 4,939   \n Accrued and other current liabilities   23,901     41,035   \n Deferred revenue - current portion   10,066     11,901   \n Total current liabilities $ 40,806   $ 57,875   \n Deferred revenue - net of current portion   6,603     6,961   \n Other long term liabilities   991     1,141   \n Total long term liabilities   7,594     8,102   \n Total liabilities $ 48,400   $ 65,977   \n Stockholders’ equity \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   6     5   \n Additional paid-in capital   942,704     849,624   \n Accumulated other comprehensive (loss) income   (115 )   543   \n Accumulated deficit   (547,718 )   (497,715 ) \n Total stockholders’ equity $ 394,877   $ 352,457   \n Total liabilities and stockholders’ equity $ 443,277   $ 418,434   \n\n Stoke Therapeutics, Inc. and subsidiary \n Condensed consolidated statements of operations and comprehensive (loss) income \n (in thousands, except share and per share amounts) \n   \n Three Months Ended March 31, \n   2026     2025 \n Revenue $ 6,229   $ 158,569 \n Operating expenses: \n Research and development   39,673     32,676 \n Sales, general and administrative   19,974     14,653 \n Total operating expenses   59,647     47,329 \n (Loss) income from operations   (53,418 )   111,240 \n Other income (expense): \n Interest income (expense), net   3,397     2,889 \n Other income   18     28 \n Total other income (expense)   3,415     2,917 \n (Loss) income before income taxes $ (50,003 ) $ 114,157 \n Provision for income taxes   —     1,278 \n Net (loss) income $ (50,003 ) $ 112,879 \n Net (loss) income per share: \n Basic $ (0.79 ) $ 1.95 \n Diluted   (0.79 )   1.90 \n Weighted-average common shares outstanding: \n Basic   63,063,507     57,862,674 \n Diluted   63,063,507     59,398,600 \n Comprehensive (loss) income: \n Net (loss) income $ (50,003 ) $ 112,879 \n Other comprehensive (loss) gain: \n Unrealized (loss) gain on marketable securities   (658 )   47 \n Total other comprehensive (loss) gain $ (658 ) $ 47 \n Comprehensive (loss) income $ (50,661 ) $ 112,926 \n\n\nReferences:\n\n\n 1. Symonds, J. et al. Early childhood epilepsies: epidemiology, classification,\naetiology, and socio-economic determinants. Brain. 2021;144(9):2879-2891.\n\n 2. Based on Stoke Therapeutics’ preliminary estimates, which scaled annual\nincidence to prevalence using country-specific live birth rates over the past\n85 years and adjusted for Dravet-specific mortality. The estimate is based on\nincidence rates published by \nWu et al., Pediatrics, 2015\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fpublications.aap.org%2Fpediatrics%2Farticle-abstract%2F136%2F5%2Fe1310%2F33828%2FIncidence-of-Dravet-Syndrome-in-a-US-Population%3FredirectedFrom%3Dfulltext%3Fautologincheck%3Dredirected&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=Wu+et+al.%2C+Pediatrics%2C+2015&index=11&md5=9aabf898bbb9050a3af085889d62f4d7)\n\n.\n\n\n\nView source version on businesswire.com:\nhttps://www.businesswire.com/news/home/20260507458327/en/\n(https://www.businesswire.com/news/home/20260507458327/en/)\n\nStoke Media & Investor Contacts: \n\nSusan Willson\n\nVice President, Corporate Communications\n\nswillson@stoketherapeutics.com \n(mailto:swillson@stoketherapeutics.com) \n415-509-8202\n\nDoug Snow\n\nDirector, Communications & Investor Relations\n\nIR@stoketherapeutics.com \n(mailto:IR@stoketherapeutics.com) \n508-642-6485\n\n\nCopyright Business Wire 2026","article_body_html":"","raw_payload":{"data":{"id":"nBw6Cr3RMa","title":"Stoke Therapeutics Announces First Quarter 2026 Financial Results and Provides Business Updates","author":"Business Wire","ticker":"STOK","created":"2026-05-07T20:01:00.552Z","tickers":["STOK"],"exchange":"NASDAQ","article_body":"Stoke Therapeutics Announces First Quarter 2026 Financial Results and Provides\nBusiness Updates\n\n– New 4-year longitudinal data from the Phase 1/2a open-label extension\n(OLE) studies provide additional support for the disease-modifying potential\nof zorevunersen, an investigational medicine for the treatment of Dravet\nsyndrome –\n\n– Statistically significant improvements in cognition and behavior\ndemonstrated at 1, 2, 3 and 4 years of treatment compared to OLE baseline, in\naddition to continued durability in seizure reductions –\n\n– Zorevunersen generally well tolerated, with some patients treated for more\nthan 5 years –\n\n– Enrollment of approximately 150 patients into the Phase 3 EMPEROR study\nexpected to complete in June 2026 to support a data readout in mid-2027; these\ndata are anticipated to complete the rolling U.S. NDA submission planned to\ninitiate in first quarter 2027 –\n\n– As of March 31, 2026, the Company had $411.0 million in cash, cash\nequivalents and marketable securities expected to fund operations into 2028,\nincluding $80.7 million raised through selective ATM sales in first quarter\n2026 –\n\n– Webcast and conference call for analysts and investors at 4:30PM Eastern\nTime today –\n\nStoke Therapeutics, Inc.\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.stoketherapeutics.com%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=Stoke+Therapeutics%2C+Inc.&index=1&md5=c7fef1a29617047f59c6c75898811167)\n(Nasdaq: STOK) is a biotechnology company dedicated to restoring protein\nexpression by harnessing the body’s potential with RNA medicine and has a\nlead investigational medicine, zorevunersen, in development with Biogen\n(Nasdaq: BIIB) as a first-in-class potential disease-modifying treatment for\nDravet syndrome. The Company today reported financial results for the first\nquarter ended March 31, 2026, and announced new 4-year longitudinal data from\nthe ongoing Phase 1/2a open-label extension (OLE) studies that provide\nadditional support for zorevunersen as a potential disease-modifying treatment\nfor Dravet syndrome. Statistically significant improvements were demonstrated\nin cognition and behavior at 1, 2, 3 and 4 years of treatment compared to OLE\nbaseline. Reductions in major motor seizure frequency were observed through 4\nyears of treatment in patients taking standard anti-seizure medicines (ASMs).\nZorevunersen continues to be generally well tolerated, with some patients\ntreated for more than 5 years in the Phase 1/2a and ongoing OLE studies.\n\nThe Company also announced an update on progress of the global Phase 3 EMPEROR\nstudy. Enrollment of approximately 150 patients in the U.S., UK and Japan is\nexpected to complete in June 2026 to support a data readout in mid-2027. These\ndata are anticipated to complete the rolling New Drug Application (NDA) to the\nU.S. Food and Drug Administration (FDA) planned to initiate in the first\nquarter of 2027.\n\n“These new 4-year OLE data suggest that zorevunersen may change the course\nof Dravet syndrome by providing children with durable reductions in seizures\nand the possibility of a more neurotypical development path. Together with the\nPhase 1/2a results, the ongoing extension studies offer 5 years of clinical\ndata, providing a unique opportunity to understand the long-term benefits and\nsafety of zorevunersen,” said Ian F. Smith, Chief Executive Officer and\nDirector of Stoke Therapeutics.\n\nMr. Smith continued, “We look forward to results from our pivotal Phase 3\nstudy, which is on track to complete enrollment of approximately 150 patients\nin June to support a data readout in mid-2027. As we continue to see this\nstudy through to completion, our focus turns increasingly to commercial\npreparedness and bringing zorevunersen to patients while also building our\npipeline for the future. All of this is supported by our strong financial\nposition taking us through to a potential U.S. launch in early 2028.”\n\nProgram Highlights\n\nDravet syndrome (zorevunersen)\n\n\n * New 4-year safety and efficacy data from OLE studies: Following treatment in\nthe Phase 1/2a studies 93% (75/81) patients continued treatment in one of two\nOLE studies. As of the 4-year data cutoff, 77% (58/75) patients remained in\nthese studies. The new 4-year data announced today showed that patients\ntreated with zorevunersen on top of standard anti-seizure medicines continued\nto experience durable reductions in seizures and ongoing improvements in\ncognition and behavior. Zorevunersen continues to be generally well tolerated,\nwith some patients treated for more than 5 years in the Phase 1/2a and ongoing\nOLE studies in which more than 850 doses have been administered. Elevated CSF\nprotein lab values occurred in approximately 94% of patients of which 59% have\nbeen classified as a treatment-emergent adverse event. Importantly, no serious\nor severe clinical manifestations have been associated with CSF protein\nelevations. There have been no reports of hydrocephalus.\n\n * Pivotal Phase 3 EMPEROR study progress:\n\n\n* U.S., UK and Japan: New patient entry into screening is now closed. As of\nMay\n5, 2026, approximately 130 patients had been randomized to zorevunersen or\nsham. The remaining patients required to achieve the planned enrollment of\napproximately 150 patients into this cohort are progressing through the 8-week\nscreening period. Following successful completion of screening, these patients\nwill be randomized to treatment with zorevunersen or a sham control\nadministered via lumbar puncture (LP). The final patient is expected to be\nrandomized in June 2026. These data are anticipated to be the final data\nrequired for completion of the planned rolling U.S. NDA submission.\n\n * Europe (Germany, France, Spain and Italy): 15 out of 16 sites are activated\nand screening is underway for at least 20 additional patients. Enrollment is\nexpected to complete in the third quarter of 2026.\n\n\n\n\n * Medical and scientific publications and communications continued to drive\nawareness of Dravet syndrome and the need for disease modification.\n\n\n* In April, Stoke presented data at the American Academy of Neurology (AAN)\nAnnual Meeting, the world’s largest gathering of neurologists.\n\n * In March, \nThe New England Journal of Medicine (NEJM)\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.nejm.org%2Fdoi%2Ffull%2F10.1056%2FNEJMoa2506295&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=The+New+England+Journal+of+Medicine+%28NEJM%29&index=2&md5=374864f30e59beb72ac2aa47b1e1e88a)\n\npublished zorevunersen data from the Phase 1/2a and OLE studies. An\nindependent editorial that discussed the underlying genetic cause of Dravet\nsyndrome and the disease-modifying potential of zorevunersen accompanied the\nmanuscript.\n\n\n\n\nPipeline beyond zorevunersen\n\n\n * In February, \nthe first patient was dosed\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Finvestor.stoketherapeutics.com%2Fnews-releases%2Fnews-release-details%2Fstoke-therapeutics-announces-first-patient-dosed-phase-1-study&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=the+first+patient+was+dosed&index=3&md5=d27f67614858bd4b5a311183d4e7d42a)\n\nin the Phase 1 OSPREY study of STK-002 for the treatment of Autosomal Dominant\nOptic Atrophy (ADOA), the most common inherited optic nerve disorder. To date,\ntwo patients have been dosed and recruitment is ongoing at 7 sites across the\nUK, Germany, Denmark and Austria. Dose escalation of the first four cohorts\nwill continue through 2026 and early 2027, pending safety and tolerability\nassessments.\n\n * Lead optimization is underway to identify a clinical candidate for the\ntreatment of SYNGAP1 in 2026. SYNGAP1 is a severe and rare genetic\nneurodevelopmental disease.\n\nFirst Quarter 2026 Financial Results\n\n\n * As of March 31, 2026, the Company had $411.0 million in cash, cash equivalents\nand marketable securities expected to fund operations into 2028. This includes\napproximately $80.7 million of proceeds from the sale of 2.6 million shares of\ncommon stock from selective use of the ATM facility (Controlled Equity\nOffering Sales Agreement) during the first quarter of 2026.\n\n * Revenue recognized for the three months ended March 31, 2026, was $6.2\nmillion, a decrease from $158.6 million for the same period in 2025. The\ndecrease in revenue is primarily driven by the 2025 recognition of $150.8\nmillion related to the IP license performance obligation related to the Biogen\nAgreement outside of the U.S., Canada and Mexico.\n\n * Net loss for the three months ended March 31, 2026, was $50.0 million, or\n$0.79 per share, compared to a net income of $112.9 million, or $1.90 per\ndiluted share, for the same period in 2025.\n\n * Research and development expenses for the three months ended March 31, 2026,\nwere $39.7 million, compared to $32.7 million for the same period in 2025. The\nincrease of $7.0 million was driven by an increase in activities and personnel\nexpenses to support the advancement of zorevunersen.\n\n * Sales, general and administrative expenses for the three months ended March\n31, 2026, increased to $20.0 million from $14.7 million for the same period in\n2025. The increase of $5.3 million was driven by an increase in personnel and\nlaunch readiness expenses.\n\nStoke Webcast and Conference Call for Analysts and Investors\n\nStoke management will host a webcast and conference call for analysts and\ninvestors on Thursday, May 7, 2026, at 4:30PM Eastern Time. The webcast will\nbe available on the Investors & News section of Stoke’s website at\nhttps://investor.stoketherapeutics.com/\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Finvestor.stoketherapeutics.com%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Finvestor.stoketherapeutics.com%2F&index=4&md5=b9afa1a7d17a9e60cab4dfa02acfc2ff)\n. Research analysts who plan to join the call and participate in the Q&A\nsession may register here\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fregistrations.events%2Fdirect%2FNTM426396&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=here&index=5&md5=76141f794f88a9168d2e25920c78e611)\nto receive the dial-in details and a unique PIN. All other participants are\ninvited to access the listen-only webcast by clicking here\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fedge.media-server.com%2Fmmc%2Fp%2Fdj697s4q%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=here&index=6&md5=6a547799f724985cdaf9b49f1cfcd149)\n. A replay of the webcast will be archived and available for at least 90 days\nfollowing the event.\n\nAbout Dravet Syndrome\n\nDravet syndrome is a severe developmental and epileptic encephalopathy (DEE)\ncharacterized by recurrent seizures as well as significant cognitive and\nbehavioral impairments. Most cases of Dravet are caused by mutations in one\ncopy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in\nneuronal cells in the brain. Even when treated with the best available\nanti-seizure medicines (ASMs), up to 57 percent of patients with Dravet\nsyndrome do not achieve ≥50 percent reduction in seizure frequency.\nComplications of the disease often contribute to a poor quality of life for\npatients and their caregivers. Developmental and cognitive impairments often\ninclude intellectual disability, developmental delays, movement and balance\nissues, language and speech disturbances, growth defects, sleep abnormalities,\ndisruptions of the autonomic nervous system and mood disorders. Compared with\nthe general epilepsy population, people living with Dravet syndrome have a\nhigher risk of sudden unexpected death in epilepsy, or SUDEP; up to 20 percent\nof children and adolescents with Dravet syndrome die before adulthood due to\nSUDEP, prolonged seizures, seizure-related accidents or infections( 1). Dravet\nsyndrome occurs globally and is not concentrated in a particular geographic\narea or ethnic group. Currently, it is estimated that up to 38,000 people are\nliving with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan( 2).(\n)There are no approved disease-modifying therapies for people living with\nDravet syndrome.\n\nAbout Zorevunersen\n\nZorevunersen is an investigational antisense oligonucleotide that is designed\nto treat the underlying cause of Dravet syndrome by increasing functional\nNaV1.1 protein production in brain cells from the unaffected (wild-type) copy\nof the SCN1A gene. This highly differentiated mechanism of action aims to\nreduce seizure frequency beyond what has been achieved with anti-seizure\nmedicines and to improve neurodevelopment, cognition and behavior.\nZorevunersen has demonstrated the potential for disease modification and has\nbeen granted orphan drug designation by the FDA and the EMA. The FDA has also\ngranted zorevunersen rare pediatric disease designation and Breakthrough\nTherapy Designation for the treatment of Dravet syndrome with a confirmed\nmutation not associated with gain-of-function, in the SCN1A gene. Stoke has a\nstrategic collaboration with Biogen to develop and commercialize zorevunersen\nfor Dravet syndrome. Under the collaboration, Stoke retains exclusive rights\nfor zorevunersen in the United States, Canada, and Mexico; Biogen receives\nexclusive rest of world commercialization rights. Zorevunersen is currently in\nclinical development, and its safety and efficacy have not been evaluated by\nany regulatory authority.\n\nAbout the Phase 1/2a and Open-Label Extension Studies\n\nTwo Phase 1/2a open-label, multicenter studies evaluated the effects of\nzorevunersen in patients with highly refractory Dravet syndrome ages 2 to 18\nyears (N=81). Primary endpoints were the safety profile, plasma\npharmacokinetics (PK) and exposure in cerebrospinal fluid (CSF) of single and\nmultiple doses of zorevunersen. Secondary endpoints included percentage change\nfrom baseline in major motor seizure frequency, overall clinical status (a\nmeasure of patients’ overall functioning) and quality of life. The ADMIRAL\nPhase 1/2a study included an exploratory endpoint to evaluate changes in\nneurodevelopmental status (cognition & behavior) as measured by Vineland\nAdaptive Behavior Scales, Third Edition (Vineland-3). The Phase 1/2a studies\nwere completed in November 2023. Following treatment in the Phase 1/2a\nstudies, eligible patients continued treatment with zorevunersen every four\nmonths in one of two OLEs. There was at least a 6-month gap between the last\ndose administered in the Phase 1/2a studies and the first dose administered in\nthe OLEs. The primary endpoints are the safety profile of multiple doses of\nzorevunersen. Secondary endpoints include PK parameters, percentage change\nfrom baseline in major motor seizure frequency, change in overall clinical\nstatus, and change from baseline in quality of life. Exploratory endpoints\ninclude changes in neurodevelopment status as measured by Vineland-3. The OLE\nstudies are ongoing.\n\nAbout the Phase 3 EMPEROR Study\n\nThe Phase 3 EMPEROR Study (NCT06872125) is a global, double-blind,\nsham-controlled study evaluating the efficacy, safety and tolerability of\nzorevunersen in children ages 2 to <18 with Dravet syndrome with a\nconfirmed variant in the SCN1A gene not associated with gain-of-function.\nStoke expects to complete enrollment of approximately 150 patients in the\nUnited States, United Kingdom and Japan in June 2026, with a data readout on\ntrack for mid-2027 to support the submission of a New Drug Application (NDA)\nto the FDA. At least 20 additional patients are expected to enroll in Germany,\nSpain, France and Italy, with completion of enrollment anticipated in Q3 2026.\nParticipants are randomized 1:1 to receive either zorevunersen via intrathecal\nadministration or a sham comparator for a 52-week treatment period following\nan 8-week baseline period. Following the completion of the study treatment\nperiod, eligible participants will be offered ongoing treatment with\nzorevunersen as part of an OLE study. The primary endpoint of the study is\npercent change from baseline in major motor seizure frequency at week 28 in\npatients receiving zorevunersen as compared to sham. The key secondary\nendpoints are the durability of effect on major motor seizure frequency and\nimprovements in behavior and cognition as measured by Vineland-3 subdomains,\nincluding expressive communication, receptive communication, interpersonal\nrelationships, coping skills and personal skills. Additional endpoints include\nsafety, Clinician Global Impression of Change (CGI-C), Caregiver Global\nImpression of Change (CaGI-C) and the Bayley Scales of Infant Development\n(BSID-IV). For more information, visit\nhttps://clinicaltrials.gov/study/NCT06872125\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT06872125%3Fcond%3DDravet%2520Syndrome%26viewType%3DCard%26term%3Demperor%26rank%3D1&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT06872125&index=7&md5=6e4135aa90e2f6b63a73222042973226)\n.\n\nAbout Autosomal Dominant Optic Atrophy (ADOA)\n\nADOA is the most common inherited optic nerve disorder, affecting\napproximately one in 30,000 people globally with a higher incidence of one in\n10,000 in Denmark due to a founder effect. It is a rare disease that causes\nprogressive and irreversible vision loss in both eyes starting in the first\ndecade of life. Severity can vary and the rate of vision loss can be difficult\nto predict. Approximately half of people with ADOA fail driving standards and\nup to 46% are registered as legally blind. More than 400 different\ndisease-causing OPA1 variants have been reported in people diagnosed with\nADOA. Currently there are no approved treatments for people living with ADOA.\n\nAbout STK-002\n\nSTK-002 is a proprietary antisense oligonucleotide (ASO) in clinical\ndevelopment for the treatment of ADOA. Stoke believes that STK-002 has the\npotential to be the first disease-modifying therapy for people living with\nADOA. An estimated 65% to 90% of ADOA cases are caused by variants in the OPA1\ngene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein\nexpression and disease manifestation. STK-002 is designed to upregulate OPA1\nprotein expression by leveraging the non-mutant (wild-type) copy of the OPA1\ngene to restore OPA1 protein expression with the aim to maintain or improve\nvision in people with ADOA. Stoke has generated preclinical data demonstrating\nproof-of-mechanism and proof-of-concept for STK-002. STK-002 has been granted\norphan drug designation by FDA as a potential new treatment for ADOA. A Phase\n1 study (OSPREY) of STK-002 in people with ADOA is now underway.\n\nAbout the Phase 1 OSPREY Study\n\nThe OSPREY study is a Phase 1, dose-escalating open-label study of children\nand adults ages 6 to 55 who have an established diagnosis of ADOA and have a\nconfirmed disease-causing variant in the OPA1 gene. The primary objectives for\nthe study are to assess the safety and tolerability of single ascending doses\nof STK-002, as well as to determine the exposure in blood. Secondary\nobjectives are to assess changes in visual function, ocular structure and\nquality of life after single doses of STK-002. The OSPREY study follows a\nstandard dose escalation design with participants enrolled into sequential\ncohorts receiving increasing dose levels of STK-002. Dose escalation of the\nfirst four cohorts will continue through 2026 and early 2027, pending safety\nand tolerability assessments. Data from the OSPREY study will help to inform\npotential future development of STK-002. The OSPREY study is actively\nrecruiting in the United Kingdom, Germany, Denmark and Austria. Additional\nEuropean sites are expected to activate in the coming months.\n\nFor more information on the OSPREY study, please visit:\n\n\n * https://www.ospreyclinicaltrial.com/\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.ospreyclinicaltrial.com%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Fwww.ospreyclinicaltrial.com%2F&index=8&md5=08667cc238c5df578cf4ee47bcf7d83d)\n\n * https://www.isrctn.com/ISRCTN41725621\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.isrctn.com%2FISRCTN41725621&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Fwww.isrctn.com%2FISRCTN41725621&index=9&md5=ec6fb87e856c36a7e43de862a55d679c)\n\nAbout Stoke Therapeutics\n\nStoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to\nrestoring protein expression by harnessing the body’s potential with RNA\nmedicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear\nGene Output) approach, Stoke is developing antisense oligonucleotides (ASOs)\nto selectively restore naturally-occurring protein levels. Stoke’s first\nmedicine in development, zorevunersen, has demonstrated the potential for\ndisease modification in patients with Dravet syndrome and is currently being\nevaluated in a Phase 3 study. Stoke’s initial focus are diseases of the\ncentral nervous system and the eye that are caused by a loss of ~50% of normal\nprotein levels (haploinsufficiency). Proof of concept has been demonstrated in\nother organs, tissues, and systems, supporting broad potential for Stoke’s\nproprietary approach. Stoke is headquartered in Bedford, Massachusetts. For\nmore information, visit https://www.stoketherapeutics.com/\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.stoketherapeutics.com%2F&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=https%3A%2F%2Fwww.stoketherapeutics.com%2F&index=10&md5=10ebc704bd2cecf44bfb9eaaa83a686c)\n.\n\nCautionary Note Regarding Forward-Looking Statements\n\nThis press release contains forward-looking statements within the meaning of\nthe “safe harbor” provisions of the Private Securities Litigation Reform\nAct of 1995, including, but not limited to: the Company’s quarterly results\nand cash runway; its future operating results and current or future financial\nposition and liquidity; the ability of zorevunersen to treat the underlying\ncauses of Dravet syndrome and reduce seizures or show improvements in behavior\nand cognition at the indicated dosing levels or at all; the design, timing and\nresults of clinical studies, enrollment timelines, data readouts, regulatory\nsubmissions or decisions and other presentations for zorevunersen and STK-002;\nthe timing and potential outcomes of meetings with regulators regarding the\nzorevunersen program; the ability of STK-002 to treat the underlying causes of\nAutosomal Dominant Optic Atrophy (ADOA) and maintain or improve vision; our\nexpectations, plans, aspirations and goals, including those related to the\npotential of zorevunersen and our collaborations with Biogen and Acadia.\nStatements including words such as “anticipate,” “expect,” “plan,”\n“will,” or “may” and statements in the future tense are\nforward-looking statements. These forward-looking statements involve risks and\nuncertainties, as well as assumptions, which, if they prove incorrect or do\nnot fully materialize, could cause the Company’s results to differ\nmaterially from those expressed or implied by such forward-looking statements,\nincluding, but not limited to, risks and uncertainties related to: the\nCompany’s ability to advance, obtain regulatory approval for, and ultimately\ncommercialize its product candidates; that if the Company’s partners were to\nbreach or terminate their collaboration with the Company, the Company would\nnot obtain the anticipated financial or other benefits; the possibility that\nthe Company and Biogen may not be successful in their development of\nzorevunersen and that, even if successful, they may be unable to successfully\ncommercialize zorevunersen; the risk that positive results in a clinical trial\nmay not be replicated in subsequent trials or successes in early stage\nclinical trials may not be predictive of results in later stage trials; the\ndevelopment goals into 2028 and through potential U.S. launch in early 2028;\nand the other risks and uncertainties described under the heading “Risk\nFactors” in the Company’s Annual Report on Form 10-K for the year ended\nDecember 31, 2025, its quarterly reports on Form 10-Q, and the other documents\nit files with the Securities and Exchange Commission. These forward-looking\nstatements speak only as of the date of this press release, and the Company\nundertakes no obligation to revise or update any forward-looking statements to\nreflect events or circumstances after the date hereof.\n\nFinancial Tables Follow\n Stoke Therapeutics, Inc. and subsidiary \n Condensed consolidated balance sheets \n (in thousands, except share and per share amounts) \n   \n March 31,   December 31, \n   2026       2025   \n Assets \n Current assets: \n Cash and cash equivalents $ 155,659   $ 84,220   \n Marketable securities - current   187,886     200,450   \n Accounts receivable   4,451     5,936   \n Prepaid expenses   10,115     8,736   \n Interest receivable   1,690     1,969   \n Other current assets   6,880     4,389   \n Total current assets $ 366,681   $ 305,700   \n Marketable securities - long-term   67,481     106,260   \n Restricted cash - long-term   3,227     227   \n Operating lease right-of-use assets   2,493     3,101   \n Property and equipment, net   3,395     3,146   \n Total assets $ 443,277   $ 418,434   \n Liabilities and stockholders’ equity \n Current liabilities: \n Accounts payable $ 6,839   $ 4,939   \n Accrued and other current liabilities   23,901     41,035   \n Deferred revenue - current portion   10,066     11,901   \n Total current liabilities $ 40,806   $ 57,875   \n Deferred revenue - net of current portion   6,603     6,961   \n Other long term liabilities   991     1,141   \n Total long term liabilities   7,594     8,102   \n Total liabilities $ 48,400   $ 65,977   \n Stockholders’ equity \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   6     5   \n Additional paid-in capital   942,704     849,624   \n Accumulated other comprehensive (loss) income   (115 )   543   \n Accumulated deficit   (547,718 )   (497,715 ) \n Total stockholders’ equity $ 394,877   $ 352,457   \n Total liabilities and stockholders’ equity $ 443,277   $ 418,434   \n\n Stoke Therapeutics, Inc. and subsidiary \n Condensed consolidated statements of operations and comprehensive (loss) income \n (in thousands, except share and per share amounts) \n   \n Three Months Ended March 31, \n   2026     2025 \n Revenue $ 6,229   $ 158,569 \n Operating expenses: \n Research and development   39,673     32,676 \n Sales, general and administrative   19,974     14,653 \n Total operating expenses   59,647     47,329 \n (Loss) income from operations   (53,418 )   111,240 \n Other income (expense): \n Interest income (expense), net   3,397     2,889 \n Other income   18     28 \n Total other income (expense)   3,415     2,917 \n (Loss) income before income taxes $ (50,003 ) $ 114,157 \n Provision for income taxes   —     1,278 \n Net (loss) income $ (50,003 ) $ 112,879 \n Net (loss) income per share: \n Basic $ (0.79 ) $ 1.95 \n Diluted   (0.79 )   1.90 \n Weighted-average common shares outstanding: \n Basic   63,063,507     57,862,674 \n Diluted   63,063,507     59,398,600 \n Comprehensive (loss) income: \n Net (loss) income $ (50,003 ) $ 112,879 \n Other comprehensive (loss) gain: \n Unrealized (loss) gain on marketable securities   (658 )   47 \n Total other comprehensive (loss) gain $ (658 ) $ 47 \n Comprehensive (loss) income $ (50,661 ) $ 112,926 \n\n\nReferences:\n\n\n 1. Symonds, J. et al. Early childhood epilepsies: epidemiology, classification,\naetiology, and socio-economic determinants. Brain. 2021;144(9):2879-2891.\n\n 2. Based on Stoke Therapeutics’ preliminary estimates, which scaled annual\nincidence to prevalence using country-specific live birth rates over the past\n85 years and adjusted for Dravet-specific mortality. The estimate is based on\nincidence rates published by \nWu et al., Pediatrics, 2015\n(https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fpublications.aap.org%2Fpediatrics%2Farticle-abstract%2F136%2F5%2Fe1310%2F33828%2FIncidence-of-Dravet-Syndrome-in-a-US-Population%3FredirectedFrom%3Dfulltext%3Fautologincheck%3Dredirected&esheet=54531662&newsitemid=20260507458327&lan=en-US&anchor=Wu+et+al.%2C+Pediatrics%2C+2015&index=11&md5=9aabf898bbb9050a3af085889d62f4d7)\n\n.\n\n\n\nView source version on businesswire.com:\nhttps://www.businesswire.com/news/home/20260507458327/en/\n(https://www.businesswire.com/news/home/20260507458327/en/)\n\nStoke Media & Investor Contacts: \n\nSusan Willson\n\nVice President, Corporate Communications\n\nswillson@stoketherapeutics.com \n(mailto:swillson@stoketherapeutics.com) \n415-509-8202\n\nDoug Snow\n\nDirector, Communications & Investor Relations\n\nIR@stoketherapeutics.com \n(mailto:IR@stoketherapeutics.com) \n508-642-6485\n\n\nCopyright Business Wire 2026"},"type":"article","timestamp":"2026-05-07T20:01:01.246693599Z","server_sent_at_ms":1778184061246},"received_at":"2026-05-07T20:01:01.355Z","source_url":"https://www.businesswire.com/news/home/20260507458327/en/"},"analysis":{"id":"20711","press_release_id":"26466","analysis_json":{"industry":{"label":"Biotechnology","sector":"Health Care"},"redFlags":[],"eventType":"earnings","narrative":"Stoke Therapeutics reported Q1 2026 revenue of $6.2 million, a decrease from the prior year due to the recognition of a large one-time license fee in 2025, while maintaining a strong cash position of $411.0 million to fund operations into 2028.\n\nNew 4-year longitudinal data from the Phase 1/2a open-label extension studies showed statistically significant improvements in cognition and behavior, along with durable seizure reductions for zorevunersen.\n\nThe Phase 3 EMPEROR study is on track to complete enrollment of approximately 150 patients in June 2026, supporting a data readout in mid-2027 and a planned rolling NDA submission in Q1 2027.","sentiment":"bullish","agentHooks":{"shouldPost":true,"suggestedAngle":"4-year data confirms zorevunersen durability; Phase 3 on track for 2027 readout with cash runway secured."},"keyFigures":{"revenue":"$6.2 million","customDimensions":{"net_loss":"$50.0 million","rd_expenses":"$39.7 million","atm_proceeds":"$80.7 million","sga_expenses":"$20.0 million","cash_position":"$411.0 million","shares_sold_atm":"2.6 million"}},"quotedText":"These 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.","namedEntities":{"people":[{"name":"Ian F. 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