STOK (Stoke Therapeutics, Inc.) PDUFA Date | FDA Catalyst & Approval Status

Jul 27, 2026

▼ -0.8%today

Stoke Therapeutics to Host Webcast and Conference Call to Discuss Second Quarter 2026 Business and Financial Updates

Stoke Therapeutics, a biotechnology firm, is set to host a webcast and conference call on August 3, 2026, to provide business and financial updates. The company is advancing its investigational medicine, zorevunersen, aimed at treating Dravet syndrome. This event will facilitate communication with analysts and investors.

Stoke Therapeutics Appoints Thomas McCauley, Ph.D., as Chief Scientific Officer

Stoke Therapeutics has appointed Thomas McCauley, Ph.D., as its new Chief Scientific Officer. This strategic move is expected to enhance the company's research capabilities and drive innovation. McCauley's expertise may lead to advancements in Stoke's therapeutic development.

Jun 30, 2026

Stoke Therapeutics Announces Completion of Enrollment of 162 Patients Into the Phase 3 EMPEROR Study of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome

Stoke Therapeutics has successfully completed the enrollment of 162 patients in the Phase 3 EMPEROR study for Zorevunersen, aimed at treating Dravet Syndrome. This milestone reflects the company's commitment to advancing research in rare diseases. The study's progress is expected to contribute valuable insights into the efficacy of the investigational medicine.

May 29, 2026

Stoke Therapeutics to Present at Upcoming Investor Conferences in June 2026

Stoke Therapeutics, a biotechnology firm focused on RNA medicine, announced that its CEO and Chief Patient Officer will present at two investor conferences in June 2026. The presentations will highlight the company's lead investigational medicine, zorevunersen, which is in Phase 3 development for Dravet syndrome. The events will be webcasted live and archived on the company's website.

May 28, 2026

Stoke Therapeutics to Present at Upcoming Investor Conferences in June

Stoke Therapeutics is scheduled to present at several investor conferences in June. The details of the presentations and the specific conferences have not been disclosed in the article. This event may provide insights into the company's future plans and developments.

May 7, 2026

Stoke Therapeutics Announces First Quarter 2026 Financial Results and Provides Business Updates

Stoke Therapeutics has released its financial results for the first quarter of 2026, alongside updates on its business operations. The announcement provides insights into the company's financial performance and strategic direction. Further details regarding specific financial metrics and business developments were not disclosed in the article.

Apr 30, 2026

Stoke Therapeutics to Host Webcast and Conference Call to Discuss First Quarter 2026 Business and Financial Updates

Stoke Therapeutics announced a webcast and conference call scheduled for May 7, 2026, to discuss its first quarter 2026 business and financial updates. The company is focused on developing zorevunersen, a potential treatment for Dravet syndrome, which has received orphan drug and breakthrough therapy designations from the FDA. The webcast will be accessible through the company's website, and a replay will be available for 90 days.

Stoke Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4) - April 16, 2026

Stoke Therapeutics announced the grant of stock options to fourteen new employees as an inducement for their employment, effective April 15, 2026. The options, totaling 129,565 shares, have an exercise price of $35.54 and will vest over four years. The company continues to develop its lead investigational medicine, zorevunersen, for Dravet syndrome, currently in a Phase 3 study.

Apr 16, 2026

Stoke Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4)

Stoke Therapeutics has announced inducement grants in accordance with Nasdaq Listing Rule 5635(c)(4). This move is part of their strategy to comply with regulatory requirements. Further details regarding the specifics of the inducement grants were not provided in the announcement.

Apr 8, 2026

Stoke Therapeutics Appoints Clare Kahn, Ph.D., to its Board of Directors

Stoke Therapeutics has appointed Clare Kahn, Ph.D., to its Board of Directors, enhancing its leadership team as it advances the Phase 3 study of zorevunersen, a potential treatment for Dravet syndrome. Dr. Kahn brings over 30 years of experience in drug development and regulatory strategy, which will support the company's mission to deliver innovative therapies for patients. Zorevunersen aims to address the underlying causes of Dravet syndrome, a severe condition with no approved disease-modifying therapies.

Apr 8, 2026

Stoke Therapeutics to Present at the 25th Annual Needham Virtual Healthcare Conference

Stoke Therapeutics is scheduled to present at the 25th Annual Needham Virtual Healthcare Conference. The event will provide an opportunity for the company to showcase its advancements and engage with investors and stakeholders in the healthcare sector.

Stoke Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4) - March 17, 2026

Stoke Therapeutics announced the grant of stock options to six new employees as an inducement for their employment, effective March 16, 2026. The options, totaling 58,650 shares, have an exercise price equal to the stock's closing price on the grant date. Stoke's lead investigational medicine, zorevunersen, is in Phase 3 development for Dravet syndrome.

Mar 5, 2026

The New England Journal of Medicine Publishes First Data to Demonstrate the Potential for Disease Modification in Dravet Syndrome

Stoke Therapeutics and Biogen announced the publication of data in NEJM showing zorevunersen's potential for disease modification in Dravet syndrome. Results from Phase 1/2a studies indicate significant reductions in seizures and improvements in cognitive and behavioral measures. This marks a potential breakthrough in treatment as there are currently no approved disease-modifying therapies for this severe condition.

Feb 25, 2026

Stoke Therapeutics to Present at the TD Cowen 46th Annual Health Care Conference

Stoke Therapeutics announced that CEO Ian F. Smith will present at the TD Cowen 46th Annual Health Care Conference on March 3, 2026. The company focuses on RNA medicine and is developing zorevunersen, a potential treatment for Dravet syndrome. Zorevunersen has shown promise in increasing protein production and has received orphan drug and breakthrough therapy designations from the FDA.

Feb 11, 2026

Stoke Therapeutics Announces First Patient Dosed in Phase 1 Study of STK-002, a Potential Disease-Modifying Medicine for the Treatment of Autosomal Dominant Optic Atrophy (ADOA)

Stoke Therapeutics has announced the dosing of the first patient in its Phase 1 OSPREY study for STK-002, aimed at treating Autosomal Dominant Optic Atrophy (ADOA). This investigational therapy seeks to address the underlying cause of ADOA, a genetic disorder leading to vision loss. The study will assess the safety and efficacy of STK-002 in affected individuals aged 6 to 55, with results expected to inform future development.

Feb 5, 2026

Stoke Therapeutics to Present at the Guggenheim Emerging Outlook: Biotech Summit 2026

Stoke Therapeutics announced that CEO Ian F. Smith will present at the Guggenheim Emerging Outlook: Biotech Summit 2026. The company is focused on developing zorevunersen, an investigational treatment for Dravet syndrome, which has shown potential for disease modification. The presentation is scheduled for February 11, 2026, and will be available via a live webcast.

Jan 19, 2026

Stoke Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4) - January 16, 2026

Stoke Therapeutics announced the grant of stock options to nine new employees as an inducement under Nasdaq Listing Rule 5635(c)(4). The options, totaling 74,020 shares, have an exercise price of $29.28 and will vest over four years. The company is advancing its investigational medicine, zorevunersen, for Dravet syndrome, currently in Phase 3 trials.

Jan 13, 2026

Stoke Therapeutics Announces Updates to Timelines for the Completion of Enrollment and a Phase 3 Data Readout from the EMPEROR Study of Zorevunersen for the Treatment of Dravet Syndrome - Company now expects to complete

Stoke Therapeutics has updated its timelines for completing the enrollment and obtaining results from the EMPEROR Phase 3 study for zorevunersen, a treatment for Dravet syndrome. The company expects enrollment of 150 patients to be completed by the second quarter of 2026, with an anticipated data readout in mid-2027. A rolling New Drug Application (NDA) submission is planned to begin in the first half of 2027 as the company continues discussions with the FDA regarding expedited development pathways.

Stoke Therapeutics and Biogen Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AES) Annual Meeting

Stoke Therapeutics and Biogen presented promising data on zorevunersen for Dravet syndrome at the 2025 AES Annual Meeting. Long-term Phase 1/2a and open-label extension studies showed significant seizure reductions and improvements in cognitive and behavioral outcomes. A propensity score weighted analysis indicated zorevunersen's effectiveness compared to standard care. Safety data were encouraging, with manageable adverse events reported.

Dec 5, 2025

Stoke Therapeutics and Biogen Present Data that Further Support the Disease-Modifying Potential of Zorevunersen, an Investigational Medicine for the Treatment of Dravet Syndrome, at the 2025 American Epilepsy Society (AE

Stoke Therapeutics and Biogen presented encouraging data on zorevunersen at the 2025 American Epilepsy Society Annual Meeting, highlighting its potential as a disease-modifying treatment for Dravet syndrome. The Phase 1/2a and open-label extension studies demonstrated significant reductions in seizure frequency and improvements in patient cognition and quality of life. The analysis included a direct comparison to a matched cohort, supporting the benefits of zorevunersen. However, there were reports of treatment-related adverse effects, though most were not linked to the investigational medicine.

Dec 1, 2025

Stoke Therapeutics and Biogen Announce Presentations at the 2025 American Epilepsy Society Annual Meeting

Stoke Therapeutics and Biogen announced data presentations for zorevunersen at the 2025 American Epilepsy Society Annual Meeting. The investigational drug is being evaluated for treating Dravet syndrome, showing promising results from four years of clinical data. The presentations will discuss its potential effects on seizures and cognitive behavior.

Nov 4, 2025

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 random

Stoke Therapeutics reported positive advancements in its Phase 3 EMPEROR study for zorevunersen, enrolling over 20 patients across the U.S., UK, and Japan. Financial results showed a net loss of $38.3 million in Q3 2025, an increase from the previous year, though the company maintains a robust financial cushion with $328.6 million available until mid-2028. Promising two-year data indicated that zorevunersen may enhance cognition and behavior in patients with Dravet syndrome. The company remains on track for potential FDA meetings regarding expedited regulatory pathways.

Aug 12, 2025

Stoke Therapeutics Reports Second Quarter 2025 Financial Results and Provides Business Updates – First patient dosed in the Phase 3 EMPEROR study of zorevunersen in patients with Dravet syndrome – –

Stoke Therapeutics announced its second quarter 2025 financial results, highlighting the dosing of the first patient in the Phase 3 EMPEROR study for zorevunersen in Dravet syndrome. The company reported solid cash reserves of $355 million to support operations through mid-2028. New data suggested zorevunersen's effectiveness in reducing seizures and improving cognition over a three-year period. Additionally, Stoke initiated a Phase 1 study for STK-002 targeting Autosomal Dominant Optic Atrophy, indicating growth beyond its initial focus on Dravet syndrome.

Aug 11, 2025

Stoke Therapeutics and Biogen Announce First Patient Dosed in Phase 3 EMPEROR Study of Zorevunersen, a Potential Disease-Modifying Treatment for Dravet Syndrome Global, pivotal Phase 3 study will evaluate efficacy and sa

Stoke Therapeutics and Biogen have initiated the Phase 3 EMPEROR study, dosing the first patient with zorevunersen, an investigational treatment for Dravet syndrome. This pivotal study aims to evaluate the efficacy and safety of zorevunersen over a 52-week period, addressing the severe seizure and cognitive impairments associated with the condition. If successful, zorevunersen could become the first disease-modifying therapy for Dravet syndrome, providing a significant improvement in patient outcomes. The trial will include participants from the United States, United Kingdom, Japan, and Europe.

Jul 10, 2025

Stoke Therapeutics and Biogen Announce Presentation of Data from Studies of Zorevunersen, an Investigational Medicine for Dravet syndrome, at the 16th European Paediatric Neurology Society (EPNS) Congress Data from an an

Stoke Therapeutics and Biogen presented data from studies of zorevunersen, an investigational therapy for Dravet syndrome, at the 16th EPNS Congress. The analysis highlighted significant improvements in cognition and behavior at Week 68, supporting its potential as a disease-modifying treatment. This contrasts with natural history data showing limitations of current standard care. Zorevunersen aims to address the underlying causes of Dravet syndrome, offering hope for better patient outcomes.

Jun 4, 2025

Stoke Therapeutics NASDAQ: STOK June 2025 COP COPY YR RI IG GH HT T 2 20 02 25 5 | | S ST TOKE OKE T TH HER ERA AP PEU EUT TI ICS CS | | 1 Forward-Looking Statements and Other Legal Notices This presentation has been pre

Stoke Therapeutics presented its forward-looking statements regarding its product pipeline, particularly focusing on zorevunersen for Dravet syndrome. The company outlined its strategy to address genetic diseases through RNA medicine, emphasizing the potential of zorevunersen as a first-in-class treatment. However, the company also acknowledged various risks and uncertainties which could affect progress towards regulatory approval and commercialization of their products, particularly in light of global economic conditions and regulatory scrutiny.

Stoke Therapeutics Reports First Quarter 2025 Financial Results and Provides Business Updates – Regulatory alignment achieved for Phase 3 EMPEROR study of zorevunersen, a potential first-in-class disease-modifying

Stoke Therapeutics reported its first quarter 2025 financial results, highlighting significant progress in advancing zorevunersen into Phase 3 development for Dravet syndrome. Regulatory alignment has been achieved for the clinical study, bolstered by a strategic collaboration with Biogen that includes substantial upfront payment and shared development costs. As of the end of March 2025, the company held $380.3 million in cash, expected to support operations until mid-2028. Overall, Stoke is well-positioned to deliver a potentially transformative treatment for Dravet syndrome patients worldwide.

Mar 18, 2025

EX.99.1 Stoke Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Updates – Phase 3 EMPEROR study of zorevunersen, a first-in-class potential disease-modifying medicine f

Stoke Therapeutics has announced its financial results for 2024, reporting a strong cash position of $246.7 million alongside a collaboration with Biogen to commercialize zorevunersen for Dravet syndrome. The company plans to begin a Phase 3 study of zorevunersen in the second quarter of 2025, following positive regulatory alignment and data. However, the transition of CEO Dr. Edward M. Kaye to an advisory role marks a significant change in leadership as the company advances its research and development efforts.

Feb 18, 2025

▼ -9.1%on this news· ran to -22% by day 3

Biogen and Stoke Therapeutics Enter into Collaboration to Develop and Commercialize Zorevunersen for the Treatment of Dravet Syndrome, a Rare Genetic Epilepsy Associated with Refractory Seizures and Neurodevelopmental Im

Biogen and Stoke Therapeutics have announced a collaboration to develop and commercialize zorevunersen, an investigational treatment for Dravet syndrome, a severe genetic epilepsy. Stoke retains exclusive rights for zorevunersen in North America, while Biogen takes rights outside these regions. The partnership aims to enhance Biogen's rare disease portfolio and facilitate the initiation of a pivotal Phase 3 study slated for 2025. The collaboration supports Stoke financially through upfront payments and potential milestones.

Stoke Therapeutics Announces Alignment with Global Regulatory Agencies and Plans to Initiate a Phase 3 Study of Zorevunersen as Potentially the First Disease-Modifying Medicine for the Treatment of Dravet Syndrome Alignm

Stoke Therapeutics has secured alignment with regulatory agencies, including the FDA and EMA, regarding the Phase 3 EMPEROR study of zorevunersen for Dravet syndrome. The study aims to evaluate the drug's effect on seizure reduction and cognitive improvement in children aged 2 to less than 18. Zorevunersen is aimed at becoming the first disease-modifying treatment for the syndrome and has received Breakthrough Therapy designation. The Phase 3 trial is expected to commence in mid-2025.

Stoke Therapeutics Receives FDA Breakthrough Therapy Designation for Zorevunersen for the Treatment of Dravet Syndrome Supported by evidence from clinical studies that indicate that zorevunersen may demonstrate substanti

Stoke Therapeutics announced that it has received FDA Breakthrough Therapy Designation for zorevunersen, aimed at treating Dravet syndrome, a severe genetic epilepsy. Clinical studies indicate that zorevunersen could significantly reduce seizure frequency and enhance cognitive and behavioral outcomes. The company plans to initiate a global Phase 3 registrational study by the end of the year, following ongoing positive discussions with regulatory agencies. This designation is a critical step in advancing zorevunersen towards potential approval, marking hope for patients with treatment-resistant symptoms.

Nov 5, 2024

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates – Company plans to provide seizure as well as cognition and behavior data from all patients treated with initial 70mg doses

Stoke Therapeutics reported its third quarter financial results, highlighting important advancements in their development of zorevunersen for Dravet syndrome. The company plans to present detailed data on seizure, cognition, and behavior outcomes associated with zorevunersen by year-end. Discussions regarding regulatory pathways for a global Phase 3 study are ongoing, and Stoke has removed the Partial Clinical Hold previously on the drug, facilitating further progress. Despite a net loss and increasing operational expenses, the firm expressed confidence in its future plans for zorevunersen.

Aug 20, 2024

IN THE COURT OF CHANCERY OF THE STATE OF DELAWARE GERALD J. ALSID, derivatively on behalf of STOKE THERAPEUTICS, INC., Plaintiff, v. EDWARD M. KAYE, SETH L. HARRISON, ARTHUR O. TZIANABOS, ADRIAN R. KRAINER, GARRY E. MENZ

A derivative action concerning Stoke Therapeutics, Inc. has been filed in the Delaware Court of Chancery. The action involves claims of breach of fiduciary duty against the company's board members, with a proposed settlement laid out for approval. A settlement hearing is scheduled for October 28, 2024, where shareholders can express their opinions and the court will decide on the fairness of the settlement. The goal of the settlement is to resolve ongoing litigation and avoid further legal expenses.

Aug 7, 2024

STOKFDA Updates

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates – FDA removes Partial Clinical Hold as company advances toward a Phase 3 registrational study of zorevunersen (STK-001) in

Stoke Therapeutics reported its second quarter financial results and announced that the FDA has lifted the Partial Clinical Hold on zorevunersen (STK-001), allowing the progression towards a Phase 3 registrational study for treating Dravet syndrome. The company plans to update its Phase 3 study plans in late 2024, while also preparing to initiate the Phase 1 study of STK-002 for Autosomal Dominant Optic Atrophy (ADOA). Despite a reported net loss of $25.7 million for the quarter, Stoke maintains a robust financial position with $282 million in cash and equivalents.

May 6, 2024

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates – Recent data demonstrating reductions in seizure frequency and improvements in cognition and behavior support the potential

Stoke Therapeutics has reported its financial results for the first quarter of 2024, highlighting significant progress with its investigational therapy STK-001 for Dravet syndrome. Promising new data from clinical studies show reductions in seizure frequency and cognitive improvements. The company is set to meet with regulatory agencies to discuss plans for a pivotal study on STK-001, with updates anticipated later in 2024. Stoke has also strengthened its finances and expanded its management team during this period.

Stoke Therapeutics Announces Pricing of Upsized $125 Million Public Offering

Stoke Therapeutics has announced the pricing of its upsized underwritten public offering, totaling $125 million. The offering entails 5,555,557 shares of common stock priced at $13.50 per share, with an option for pre-funded warrants. The company plans to utilize the proceeds to further develop its RNA-based product candidates, including STK-001 for Dravet syndrome. The transaction is set to close around April 2, 2024, pending customary closing conditions.

Mar 25, 2024

Stoke Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results – As of

Stoke Therapeutics reported its fourth-quarter and full-year financial results for 2023, showcasing a cash position of $201.4 million expected to fund operations through 2025. Despite this strong cash runway, the company experienced a net loss of $104.7 million for the year, a slight increase compared to the previous year. Revenue generated from collaboration agreements declined, indicating potential challenges in revenue growth amidst rising operational costs.

Mar 25, 2024

Stoke Therapeutics Announces Landmark New Data That Support the Potential for STK-001 to be the First Disease-Modifying Medicine for the Treatment of Patients with Dravet Syndrome Phase 1/2a End of Study Data: 70mg doses

Stoke Therapeutics has announced significant data supporting STK-001 as a potential disease-modifying therapy for Dravet syndrome. Results from Phase 1/2a studies indicate substantial reductions in convulsive seizure frequency in patients, even in those already receiving standard anti-seizure medications. The treatment also showed durable improvements in cognitive and behavioral measures over the course of 12 months. Stoke plans to meet with regulatory agencies to discuss the design of a registrational study based on these promising findings.

Jan 10, 2024

Stoke Therapeutics NASDAQ: STOK Edward M. Kaye, M.D. Chief Executive Officer nd 42 Annual J.P. Morgan Healthcare Conference

Stoke Therapeutics presented its updates at the 42nd Annual J.P. Morgan Healthcare Conference, highlighting the progress of STK-001, a potential disease-modifying treatment for Dravet syndrome. Clinical trial data demonstrates substantial reductions in seizure frequency and improvements in cognitive and behavioral outcomes for patients. However, concerns remain about the lack of meaningful improvement in seizure frequency during initial studies and potential challenges in advancing through regulatory pathways. The company aims to continue its development efforts into 2025 to achieve its goals.

Dec 1, 2023

Stoke Therapeutics Presents Data From Multiple Studies of Children and Adolescents With Dravet Syndrome at the American Epilepsy Society (AES) 2023 Annual Meeting Data from clinical studies of STK-001 demonstrated clinic

Stoke Therapeutics presented promising data at the AES 2023 Annual Meeting regarding STK-001, a potential disease-modifying therapy for Dravet syndrome. Clinical trials demonstrated significant seizure reduction and cognitive improvements in treated pediatric patients. However, contrasting findings from a two-year natural history study indicated that conventional anti-seizure medications failed to provide meaningful benefits, highlighting the critical need for innovative treatments. The results underscore the potential of STK-001 to address the complex challenges faced by children with this condition.

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates – STK-001: Q1 2024 readout expected to include end of Phase 1/2a study results and longer-term data on the effects of repeat

Stoke Therapeutics reported its third quarter financial results, highlighting significant progress with its treatment STK-001 for Dravet syndrome. The company anticipates a readout in Q1 2024 that will provide insights into the Phase 1/2a study results and longer-term effects of repeat dosing. With a strong cash position of $214.7 million, Stoke plans to transition to a Phase 3 study design discussion with regulators in the first half of 2024. Recent studies suggest STK-001 may not only reduce seizures but also improve cognition and behavior among patients, representing a potential breakthrough in treating this severe epilepsy.

Sep 20, 2023

▼ -6.7%on this news· ran to -19% by day 3

Stoke Therapeutics Appoints Ian Smith to its Board of Directors Mr. Smith brings significant experience in the successful development and commercialization of medicines for rare genetic diseases

Stoke Therapeutics has appointed Ian Smith to its Board of Directors as of September 19, 2023. Smith brings over two decades of experience in the life sciences sector, having successfully developed various treatments for rare genetic diseases. His strategic and operational expertise is expected to be valuable as the company advances its STK-001 program for Dravet syndrome into a Phase 3 clinical trial. The appointment reflects Stoke's commitment to developing innovative therapies addressing severe health challenges.

Aug 7, 2023

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates – STK-001: Additional data from patients treated with single and multiple doses (70mg) along with data from open-label exte

Stoke Therapeutics has released its second-quarter financial results, highlighting advancements in its clinical programs, particularly for STK-001, which shows promise as a disease-modifying treatment for Dravet syndrome. The company plans to update on Phase 3 preparations for STK-001 in the first half of 2024 and has announced the initiation of a Phase 1 study for STK-002 in the UK to address Autosomal Dominant Optic Atrophy. Despite encouraging clinical data, Stoke reported a net loss of $30.7 million for the quarter and a decline in revenue from their collaboration agreement.

Stoke Therapeutics Announces Positive New Safety & Efficacy Data from Patients Treated with STK-001 in the Phase 1/2a Studies (MONARCH & ADMIRAL) and the SWALLOWTAIL Open-Label Extension (OLE) Study in Children and Adole

Stoke Therapeutics announced encouraging results from ongoing Phase 1/2a studies of STK-001 in children with Dravet syndrome, showing significant reductions in seizure frequency and improvements in cognitive and behavioral functions. The MONARCH and ADMIRAL studies involved patients aged 2 to 18, revealing substantial clinical benefits while maintaining a favorable safety profile. Notably, the data suggest potential disease modification through effective management of both seizures and cognitive issues. Additional results are expected in early 2024, indicating progress in the company’s clinical development program.

May 4, 2023

▲ +13%on this news· ran to +28% by day 3

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates – Company on-track to report new data from the ongoing Phase 1/2a clinical studies of STK-001 in children and adolescents wi

Stoke Therapeutics has reported its first quarter financial results, revealing a plan to release new data from Phase 1/2a studies of STK-001 for Dravet syndrome in mid-2023. The company has noted encouraging results with a reduction in seizure frequency in prior studies. With $254.2 million in cash available, Stoke is positioned to fund operations until late 2025. Additionally, Stoke has received approval to initiate a Phase 1/2 study of STK-002 for Autosomal Dominant Optic Atrophy, slated to begin in 2024.

Stoke Therapeutics Announces That FDA Will Allow the Administration of a Higher Single Dose of STK-001 (70mg) in the Ongoing Phase 1/2a MONARCH Study of Children and Adolescents with Dravet Syndrome Dosing anticipated to

Stoke Therapeutics announced that the FDA has permitted the administration of a higher single dose of STK-001 (70mg) in its ongoing Phase 1/2a MONARCH study targeting children and adolescents with Dravet syndrome. This cohort adds to the study's evaluation of multiple doses being tested in the ADMIRAL study. The company remains on track for upcoming data readouts, with the first patient dosing expected soon. STK-001 aims to tackle the underlying genetic cause of Dravet syndrome, a severe epilepsy condition with significant life quality challenges.

Mar 6, 2023

Stoke Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Updates - Company expects to complete Phase 1/2a studies of STK-001 in children and adolescents with Dravet syndrome in

Stoke Therapeutics has reported its fourth quarter and full year 2022 financial results, highlighting significant progress in its STK-001 development for Dravet syndrome. The company expects to finish Phase 1/2a studies in children and adolescents by 2023, aiming to initiate a Phase 3 program in 2024. Recent data have shown a reduction in seizure frequency, and the company's strong cash position extends its operational runway to the end of 2025. STK-001 is designed to address the genetic cause of Dravet syndrome and is currently undergoing clinical evaluations.

Jan 10, 2023

▲ +5.9%on this news· ran to +18% by day 3

Stoke Therapeutics NASDAQ: STOK Edward M. Kaye, M.D. Chief Executive Officer st 41 Annual J.P. Morgan Healthcare Conference

Stoke Therapeutics presented at the 41st Annual J.P. Morgan Healthcare Conference about their progress in developing treatments for severe genetic disorders such as Dravet syndrome and Autosomal Dominant Optic Atrophy. The company highlighted their innovative approach that selectively boosts protein expression, aiming to address the underlying causes of these diseases. However, they also cautioned about the risks associated with advancing clinical trials, regulatory approvals, and the potential impacts of the COVID-19 pandemic. The pipeline could offer new therapeutic options for patients who currently face unmet medical needs.

Dec 2, 2022

Stoke Therapeutics Presents Data from a Combined Interim Analysis of the Phase 1/2a MONARCH and ADMIRAL Studies of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2022 Annu

Stoke Therapeutics Presents Data from a Combined Interim Analysis of the Phase 1/2a MONARCH and ADMIRAL Studies of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2022 Annual Meeting Single and multiple doses of STK-001 up to 45m

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates - Company announces positive interim data from the ongoing Phase 1/2a clinical studies of STK-001 in children and adolescents with

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates - Company announces positive interim data from the ongoing Phase 1/2a clinical studies of STK-001 in children and adolescents with Dravet syndrome - - Single and multiple doses of STK-001 u

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates - In the fourth quarter, the Company plans to announce interim safety, pharmacokinetic (PK), and cerebrospinal fluid (CSF) drug ex

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates - In the fourth quarter, the Company plans to announce interim safety, pharmacokinetic (PK), and cerebrospinal fluid (CSF) drug exposure data from approximately 40 patients in the ongoing c

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates - 2H 2022 clinical data readout on track for patients with Dravet syndrome who received multiple doses of STK-001 (30mg) in Phase 1

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates - 2H 2022 clinical data readout on track for patients with Dravet syndrome who received multiple doses of STK-001 (30mg) in Phase 1/2a clinical studies MONARCH, ADMIRAL, and SWALLOWTAIL - -

Mar 10, 2022

Stoke Therapeutics Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Updates - Enrollment and dosing ongoing in the Phase 1/2a MONARCH and ADMIRAL studies of STK-001 in children and adoles

Stoke Therapeutics Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Updates - Enrollment and dosing ongoing in the Phase 1/2a MONARCH and ADMIRAL studies of STK-001 in children and adolescents with Dravet syndrome - - Company remains on track to

Jan 10, 2022

Stoke Therapeutics NASDAQ: STOK Edward M. Kaye, M.D. Chief Executive Officer th 40 Annual J.P. Morgan Healthcare Conference

Executive Officer th 40 Annual J.P. Morgan Healthcare Conference January 10, 2022 Copyr igh Cot p2022 yrigh t S2022 toke T Sh to er ke ap T eh ue tic ras peutics 1 Disclaimer This presentation has been prepared by Stoke Therapeutics, Inc. ("Stoke" or "our") for informational pu

Stoke Therapeutics Presents Data from the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2021 Annual Meeting Single doses of STK-001 up to 30mg

Stoke Therapeutics Presents Data from the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2021 Annual Meeting Single doses of STK-001 up to 30mg and multiple doses of 20mg were well tolerated with no sa

Nov 8, 2021

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates - Company nominates STK-002 as clinical candidate for the treatment of autosomal dominant optic atrophy (ADOA), the most common inh

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates - Company nominates STK-002 as clinical candidate for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder - - Additional data from an in

Agenda Introduction Eric Rojas, Head of Investor Relations Disease Overview, Patient Need, and STK-001 Mechanism of Action Edward M. Kaye, M.D., Chief Executive Officer Phase 1/2a MONARCH Interim Analysis Barry Ticho, M.

MONARCH study in Dravet Syndrome Interim Analysis September 21, 2021 Exhibit 99.1 Agenda Introduction Eric Rojas, Head of Investor Relations Disease Overview, Patient Need, and STK-001 Mechanism of Action Edward M. Kaye, M.D., Chief Executive Officer Phase 1/2a MONARCH Interim

Aug 10, 2021

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates - Dosing up to 30mg complete in the single ascending dose (SAD) portion in the Phase 1/2a MONARCH study of STK-001 in children and

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates - Dosing up to 30mg complete in the single ascending dose (SAD) portion in the Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome; Enrollment and dosing in

May 10, 2021

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates - Enrollment complete in single ascending dose (SAD) portion (10mg, 20mg, 30mg) of Phase 1/2a MONARCH study of STK-001 in children

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates - Enrollment complete in single ascending dose (SAD) portion (10mg, 20mg, 30mg) of Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome; Interim analysis plan

Mar 9, 2021

Stoke Therapeutics Reports Full Year 2020 Financial Results and Provides Business Updates - First patient dosed in the multiple ascending dose portion of Phase 1/2a MONARCH study of STK-001 in Dravet syndrome - - Prelimi

Stoke Therapeutics Reports Full Year 2020 Financial Results and Provides Business Updates - First patient dosed in the multiple ascending dose portion of Phase 1/2a MONARCH study of STK-001 in Dravet syndrome - - Preliminary safety and PK data from the single ascending dose por

Feb 5, 2021

Disclaimer Copyright 2021 Stoke Therapeutics This presentation has been prepared by Stoke Therapeutics, Inc. ("Stoke" or "our") for informational purposes only and not for any other purpose. Nothing contained in this pre

Stoke Therapeutics NASDAQ: STOK February 2021 Copyright 2021 Stoke Therapeutics Exhibit 99.1 Disclaimer Copyright 2021 Stoke Therapeutics This presentation has been prepared by Stoke Therapeutics, Inc. ("Stoke" or "our") for informational purposes only and not for any other pu

Nov 19, 2020

Stoke Therapeutics Announces Pricing of $97.5 Million Public Offering

Stoke Therapeutics Announces Pricing of $97.5 Million Public Offering BEDFORD, Mass., November 19, 2020 (BUSINESS WIRE) Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulat

Nov 12, 2020

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates - Company nominates OPA1 as the next preclinical target for its proprietary TANGO approach to treating the underlying cause of seve

Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates - Company nominates OPA1 as the next preclinical target for its proprietary TANGO approach to treating the underlying cause of severe - OPA1 protein deficiency is the leading cause of autos

Aug 17, 2020

Stoke Therapeutics Announces Appointment of Garry E. Menzel, Ph.D., to its Board of Directors Dr. Menzel brings significant leadership in the global healthcare sector

Stoke Therapeutics Announces Appointment of Garry E. Menzel, Ph.D., to its Board of Directors Dr. Menzel brings significant leadership in the global healthcare sector BEDFORD, Mass., August 17, 2020 Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a

Aug 10, 2020

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates - First patient dosed with STK-001 in Part A of Phase 1/2a MONARCH clinical trial for Dravet syndrome - - Company on track to iden

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates - First patient dosed with STK-001 in Part A of Phase 1/2a MONARCH clinical trial for Dravet syndrome - - Company on track to identify an additional pre-clinical candidate derived from its

Jul 9, 2020

Stoke Therapeutics Announces Publication of Data in the Journal Nature Communications That Support the Company s Proprietary Approach to Addressing Severe Genetic Diseases by Precisely Upregulating Protein Expression Sto

Stoke Therapeutics Announces Publication of Data in the Journal Nature Communications That Support the Company s Proprietary Approach to Addressing Severe Genetic Diseases by Precisely Upregulating Protein Expression antisense oligonucleotides showed dose-dependent reductions i

Jun 4, 2020

Stoke Therapeutics Appoints Julie Anne Smith to its Board of Directors Ms. Smith brings significant experience in biotech leadership and genetic diseases

Stoke Therapeutics Appoints Julie Anne Smith to its Board of Directors Ms. Smith brings significant experience in biotech leadership and genetic diseases BEDFORD, Mass., June 4, 2020 Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to trea

May 15, 2020

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates - Company on track to begin enrollment and dosing of STK-001 in Part A of Phase 1/2a "Monarch" clinical trial in children and adole

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates - Company on track to begin enrollment and dosing of STK-001 in Part A of Phase 1/2a "Monarch" clinical trial in children and adolescents with Dravet syndrome in 2H 2020 - - Research activi

May 12, 2020

Stoke Therapeutics Presents Preclinical Data That Demonstrate In-Vitro and In-Vivo Target Engagement and Protein Upregulation in OPA1 Protein Deficiency, the Underlying Cause of the Most Common Inherited Optic Nerve Diso

Stoke Therapeutics Presents Preclinical Data That Demonstrate In-Vitro and In-Vivo Target Engagement and Protein Upregulation in OPA1 Protein Deficiency, the Underlying Cause of the Most Common Inherited Optic Nerve Disorder proof-of-concept for TANGO antisense oligonucleotides

Mar 23, 2020

STOKFDA Updates

STOKE THERAPEUTICS Reports FULL YEAR 2019 financial results and provides business updateS - Company receives FDA clearance to begin dosing STK-001 in Part A of Phase 1/2a "Monarch" clinical trial in Dravet syndrome; enro

STOKE THERAPEUTICS Reports FULL YEAR 2019 financial results and provides business updateS BEDFORD, Mass.-- (BUSINESS WIRE) - March 23, 2020 -- Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases

Jan 13, 2020

Disclaimer Copyright 2020 Stoke Therapeutics, Inc. All rights reserved This presentation has been prepared by Stoke Therapeutics, Inc. ("Stoke") for informational purposes only and not for any other purpose. Nothing cont

Stoke Therapeutics Edward M. Kaye, M.D. Disclaimer Copyright 2020 Stoke construed as, a recommendation, promise or representation by the presenter or Stoke or any officer, director, employee, agent or advisor of Stoke. This presentation does not purport to be all-inclusive or t

Dec 7, 2019

Stoke Therapeutics Presents Preclinical Data From Studies of STK-001 That Showed Improvements in Survival and Reductions in Seizure Frequency in a Mouse Model of Dravet Syndrome New data from electroencephalography (EEG)

Stoke Therapeutics Presents Preclinical Data From Studies of STK-001 That Showed Improvements in Survival and Reductions in Seizure Frequency in a Mouse Model of Dravet Syndrome New data from electroencephalography (EEG) recordings showed 76% of Dravet syndrome (DS) mice treat

Nov 12, 2019

STOKE THERAPEUTICS Reports Third quarter 2019 financial results and provides business update BEDFORD, Mass.-- (BUSINESS WIRE)

STOKE THERAPEUTICS Reports Third quarter 2019 financial results and provides business update BEDFORD, Mass.-- (BUSINESS WIRE) - November 12, 2019 -- Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic di

Aug 14, 2019