STOK 8-K
Stoke Therapeutics, Inc. (STOK)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Securities registered pursuant to Section 12(b) of the Act:
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Trading |
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 2.02 Results of Operations and Financial Condition.
On November 4, 2025, Stoke Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the quarter ended September 30, 2025.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
Exhibit Number |
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Description |
99.1 |
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104 |
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Cover Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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STOKE THERAPEUTICS, INC. |
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Date: |
November 4, 2025 |
By: |
/s/ Thomas E. Leggett |
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Thomas E. Leggett |
EX 99.1
Stoke Therapeutics Reports Third Quarter 2025 Financial Results and Provides Business Updates
– Global Phase 3 EMPEROR study patient recruitment ongoing in the U.S., UK, and Japan with more than 20 patients randomized to zorevunersen or sham; Study on track to complete enrollment in second half of 2026 –
– As of September 30, 2025, the Company had $328.6 million in cash, cash equivalents, and marketable securities, anticipated to fund operations to mid-2028 –
– Webcast and conference call for analysts and investors at 4:30PM Eastern Time today –
BEDFORD, Mass., November 4, 2025 – Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body’s 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 third quarter ended September 30, 2025 and provided business updates.
"Stoke’s progress in 2025 has the Company on an important growth trajectory. We are creating an understanding of the devastating impacts of Dravet syndrome on people’s lives and the potential of zorevunersen to improve outcomes for them through substantial seizure reductions and improvements in cognition and behavior,” said Ian F. Smith, Chief Executive Officer and Director of Stoke Therapeutics. “In just three months since the initiation of our Phase 3 EMPEROR study, we have made significant progress with more than 20 patients randomized into dosing and a steady progression of more patients entering the screening period. At the same time, presentations of new clinical data at medical congresses have provided greater understanding of zorevunersen’s disease-modifying potential that is highly differentiated from any currently available treatments. We look forward to meeting with the FDA under our Breakthrough Therapy Designation before year-end, at which time we will review the four years of safety and efficacy data from our clinical studies and discuss how we can work together to deliver zorevunersen to patients through expedited regulatory pathways.”
Mr. Smith continued, “Our strong financial position is allowing us to invest in the business, including enhancing our internal capabilities to rapidly scale in preparation for potential commercialization, while also expanding our pipeline in the areas of ADOA and SYNGAP1 as well as broadening our research activities.”
Third Quarter 2025 Business Highlights and Recent Developments
Dravet Syndrome (Zorevunersen)
Pipeline and Corporate
Third Quarter 2025 Financial Results
Year-to-Date 2025 Financial Results
Stoke Webcast and Conference Call for Analysts and Investors
Stoke management will host a webcast and conference call for analysts and investors on Tuesday, November 4, 2025, at 4:30pm Eastern Time. The webcast will be available on the Investors & News section of Stoke’s website at https://investor.stoketherapeutics.com/. Research analysts who plan to join the call and participate in the Q&A session may register here 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 replay of the webcast will be archived and available for at least 90 days following the event.
About Dravet Syndrome
Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan.1
About Zorevunersen
Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing functional NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights.
About the EMPEROR Study
The EMPEROR Phase 3 Study (NCT06872125) is a global, double-blind, sham-controlled study evaluating the efficacy, safety and tolerability of zorevunersen in children ages 2 to <18 with Dravet syndrome with a confirmed variant in the SCN1A gene not associated with
gain-of-function. Study 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. An open-label extension treatment period will allow all patients the opportunity to receive treatment with zorevunersen following the 52-week treatment period. 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), EuroQol Visual Analog Scale (EQ-VAS) and the Bayley Scales of Infant Development (BSID-IV). EMPEROR has initiated in the United States, United Kingdom, Japan and is planned for Europe. For more information on the EMPEROR study, please visit https://www.emperorstudy.com/ and https://clinicaltrials.gov/study/NCT06872125.
About Autosomal Dominant Optic Atrophy (ADOA)
ADOA is the most common inherited optic nerve disorder. 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. Roughly half of people with ADOA fail driving standards and up to 46% are registered as legally blind. More than 400 different OPA1 variants have been reported in people diagnosed with ADOA. ADOA affects approximately one in 30,000 people globally with a higher incidence in Denmark of one in 10,000 due to a founder effect. Currently there is no approved treatment for people living with ADOA.
About STK-002
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 cases are caused by variants in the OPA1 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 gene to restore OPA1 protein expression with the aim to maintain or improve vision in patients 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.
About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke’s first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3
study. Stoke’s initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke’s proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https://www.stoketherapeutics.com/.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the Company’s 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, data readouts, regulatory 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 “anticipate,” “expect,” “plan,” “will,” or “may” 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’s result 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’s ability to advance, obtain regulatory approval for, and ultimately commercialize its product candidates; that if the Company’s 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 to mid-2028; and the other risks and uncertainties described under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.
Reference:
Financial Tables Follow
Stoke Therapeutics, Inc. and subsidiary
Consolidated balance sheets
(in thousands, except share and per share amounts)
(unaudited)
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September 30, |
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December 31, |
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2025 |
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2024 |
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Assets |
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Current assets: |
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Cash and cash equivalents |
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$ |
83,394 |
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$ |
127,983 |
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Marketable securities - current |
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164,916 |
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88,916 |
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Accounts receivable |
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9,248 |
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1,668 |
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Prepaid expenses |
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7,747 |
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11,117 |
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Restricted cash - current |
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— |
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75 |
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Interest receivable |
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1,589 |
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700 |
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Other current assets |
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5,543 |
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2,297 |
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Total current assets |
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$ |
272,437 |
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$ |
232,756 |
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Marketable securities - long-term |
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80,315 |
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29,824 |
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Restricted cash - long-term |
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721 |
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721 |
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Operating lease right-of-use assets |
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3,708 |
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4,345 |
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Property and equipment, net |
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3,083 |
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3,909 |
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Total assets |
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$ |
360,264 |
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$ |
271,555 |
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Liabilities and stockholders’ equity |
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Current liabilities: |
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Accounts payable |
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$ |
5,871 |
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$ |
2,498 |
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Accrued and other current liabilities |
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30,093 |
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18,567 |
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Deferred revenue - current portion |
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5,763 |
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18,991 |
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Total current liabilities |
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$ |
41,727 |
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$ |
40,056 |
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Deferred revenue - net of current portion |
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8,558 |
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— |
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Other long term liabilities |
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1,874 |
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2,478 |
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Total long term liabilities |
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10,432 |
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2,478 |
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Total liabilities |
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$ |
52,159 |
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$ |
42,534 |
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Stockholders’ equity |
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Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 55,120,002 and 54,032,826 shares issued and outstanding as of September 30, 2025 and December 31, 2024, respectively |
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5 |
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5 |
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Additional paid-in capital |
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747,435 |
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719,997 |
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Accumulated other comprehensive income (loss) |
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446 |
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(151 |
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Accumulated deficit |
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(439,781 |
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(490,830 |
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Total stockholders’ equity |
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$ |
308,105 |
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$ |
229,021 |
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Total liabilities and stockholders’ equity |
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$ |
360,264 |
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$ |
271,555 |
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Stoke Therapeutics, Inc. and subsidiary
Consolidated statements of operations and comprehensive income (loss)
(in thousands, except share and per share amounts)
(unaudited)
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2025 |
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2024 |
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2025 |
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2024 |
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Revenue |
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$ |
10,632 |
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$ |
4,894 |
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$ |
183,018 |
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$ |
13,941 |
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Operating expenses: |
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Research and development |
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37,696 |
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22,205 |
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96,227 |
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65,710 |
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Sales, general and administrative |
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16,027 |
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12,692 |
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45,942 |
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35,950 |
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Total operating expenses |
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53,723 |
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34,897 |
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142,169 |
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101,660 |
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Income (loss) from operations |
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(43,091 |
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(30,003 |
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40,849 |
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(87,719 |
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Other income (expense): |
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Interest income (expense), net |
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3,463 |
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3,545 |
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10,141 |
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9,668 |
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Other income (expense), net |
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3 |
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28 |
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59 |
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(448 |
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Total other income (expense) |
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3,466 |
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3,573 |
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10,200 |
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9,220 |
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Income (loss) before income taxes |
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$ |
(39,625 |
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$ |
(26,430 |
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$ |
51,049 |
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$ |
(78,499 |
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Provision for income taxes |
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(1,278 |
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— |
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— |
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— |
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Net income (loss) |
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$ |
(38,347 |
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$ |
(26,430 |
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$ |
51,049 |
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$ |
(78,499 |
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Net income (loss) per share: |
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Basic |
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$ |
(0.65 |
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$ |
(0.47 |
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$ |
0.88 |
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$ |
(1.48 |
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Diluted |
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(0.65 |
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(0.47 |
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0.85 |
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(1.48 |
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Weighted-average common shares outstanding: |
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Basic |
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58,611,677 |
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56,341,074 |
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58,278,812 |
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52,991,015 |
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Diluted |
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58,611,677 |
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56,341,074 |
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60,397,681 |
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52,991,015 |
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Comprehensive income (loss): |
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Net income (loss) |
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$ |
(38,347 |
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$ |
(26,430 |
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$ |
51,049 |
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$ |
(78,499 |
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Other comprehensive gain (loss): |
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Unrealized gain on marketable securities |
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350 |
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181 |
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597 |
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190 |
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Total other comprehensive gain |
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$ |
350 |
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$ |
181 |
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$ |
597 |
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$ |
190 |
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Comprehensive income (loss) |
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$ |
(37,997 |
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$ |
(26,249 |
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$ |
51,646 |
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$ |
(78,309 |
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Stoke Media & Investor Contacts:
Susan Willson
Vice President, Corporate Communications
415-509-8202
Doug Snow
Director, Communications & Investor Relations
508-642-6485