Stoke Therapeutics, Inc. · NASDAQ:STOK

Stoke Therapeutics — Q1 2026 Earnings Presentation

20 slides from Stoke Therapeutics's earnings presentation, May 7, 2026

  1. Slide 1: Stoke Therapeutics First Quarter 2026 Business Update

    Stoke Therapeutics First Quarter 2026 Business Update Webcast for Investors & Analysts May 7, 2026 © COPYRIGHT 2026 | STOKE THERAPEUTICS | 1

  2. Slide 2: Forward-Looking Statements and Other Legal Notices

    Forward-Looking Statements and Other Legal Notices This presentation has been prepared by Stoke Therapeutics, Inc. (“Stoke” or “us”) for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or repre

  3. Slide 3: Opening Remarks

    Opening Remarks Ian F. Smith Chief Executive Officer & Director​ © COPYRIGHT 2026 | STOKE THERAPEUTICS | 3

  4. Slide 4: An Important Growth Trajectory Driven by Appreciation for zorevunersen, Phase 3 Progress

    An Important Growth Trajectory Driven by Appreciation for zorevunersen, Phase 3 Progress Stoke has transformed itself over the last year and is well positioned for our next phase of growth 2027-2028 2026 • Rolling NDA submission to initiate in 1Q 2027 2025 • Rapid enrollment of the Phase 3 EMPEROR s

  5. Slide 5: 4-Year Longitudinal Data and Phase Progress

    4-Year Longitudinal Data and Phase 3 Progress Barry Ticho, M.D., Ph.D. Chief Medical Officer © COPYRIGHT 2026 | STOKE THERAPEUTICS | 5

  6. Slide 6: Substantial, Durable Reductions in Seizure on Top of SOC Observed Through Four Years

    Substantial, Durable Reductions in Seizure on Top of SOC Observed Through Four Years of Treatment with Zorevunersen Data for all patients who continued treatment in the OLEs separated by dose received in the Ph1/2a studies Data cut date: 19 Feb 2026; 1 Month – 4 Weeks; One patient who received an in

  7. Slide 7: Median Percent Change from Ph1/2a Baseline Through Year 4 Shown in 16-Week Intervals

    Median Percent Change from Ph1/2a Baseline Through Year 4 Shown in 16-Week Intervals Data for all patients who continued treatment in the OLEs separated by dose received in the Ph1/2a studies Data cut date: 19 Feb 2026; 1 Month – 4 Weeks; One patient who received an incorrect dose of zorevunersen in

  8. Slide 8: Development in Patients with Dravet Syndrome Differs Markedly from Neurotypical Children

    Development in Patients with Dravet Syndrome Differs Markedly from that of Neurotypical Children Neurodevelopment plateaus at ~2 years of age for children living with Dravet syndrome1 Neurodevelopmental progress Comparison of developmental trajectory between neurotypical children and patients with D

  9. Slide 9: The Vineland-3 Assessment Tool is Commonly Used to Evaluate Non-Seizure Outcomes

    The Vineland-3 Assessment Tool is Commonly Used to Evaluate Non-Seizure Outcomes in Dravet Syndrome Vineland-3 Adaptive Behavior Scales* Domains COMMUNICATION SOCIALIZATION MOTOR SKILLS DAILY LIVING SKILLS Receptive: Interpersonal Gross Motor: Personal: Responds upon hearing Relationships: Moves, sc

  10. Slide 10: 4-Year Phase 1/2 OLE Data Showed Continuing Improvements in Cognition and Behavior

    4-Year Phase 1/2 OLE Data Showed Continuing Improvements in Cognition and Behavior <0.01 statistical significance achieved in 5 key subdomains for each year compared to OLE baseline Year 1 (n=68) Phase 3 EMPEROR Year 2 (n=60) Subdomains Year 3 (n=42) Year 4 (n=14) Change in raw scores (95% CI) from

  11. Slide 11: Zorevunersen Generally Well-Tolerated with Long-Term Dosing

    Zorevunersen Generally Well-Tolerated with Long-Term Dosing • 30% of patients experienced a study drug–related TEAE Phase 1/2a • Most common: CSF protein elevations (14%) and procedural vomiting (5%) studies • 22% of patients experienced a TESAE (n=81) • All were unrelated to the study drug except f

  12. Slide 12: Enrollment of ~150 Patients Expected to Complete in June 2026 to Support a Data Readout in Mid-2027

    Enrollment of ~150 Patients Expected to Complete in June 2026 to Support a Data Readout in Mid-2027 Rolling U.S. NDA submission planned to initiate in Q1 2027 ~150 Patients in the Blinded 52-week U.S., UK, Japan treatment period • New patient entry into screening now closed • Primary endpoint of cha

  13. Slide 13: Preparing for a Potential U.S. Launch

    Preparing for a Potential U.S. Launch Jason Hoitt Chief Patient Officer © COPYRIGHT 2026 | STOKE THERAPEUTICS | 13

  14. Slide 14: Significant Market Opportunity

    Significant Market Opportunity ~38K patients with Dravet syndrome across 7 major geographic markets PREVALENCE OF DRAVET SYNDROME* SIGNIFICANT NEED DESPITE AVAILABILITY OF ANTI-SEIZURE MEDICINES (ASMs) 7K Japan There are currently no disease modifying medicines approved for the treatment of Dravet s

  15. Slide 15: Concentrated U.S. Market Enables Targeted Engagement

    Concentrated U.S. Market Enables Targeted Engagement HCPs Sites of Care Patients of Dravet patients seen ~ 70% by ~1,200 HCPs of Dravet patients 16k Prevalent ~ 70% covered by 124 Sites of Care 6k Addressable at launch Vast majority of ICD10 code established in high-volume treaters 2020 will continu

  16. Slide 16: Confidence in the Value of zorevunersen as a Potential Disease-Modifying Treatment

    Confidence in the Value of zorevunersen as a Potential Disease-Modifying Treatment for Dravet Syndrome Phase 3 Progress Longitudinal Data Visibility & Credibility • Dosing regimen, endpoint selection and • 5 years of clinical data from the • Publication of zorevunersen data in powering assumptions i

  17. Slide 17: Financial Summary

    Financial Summary Thomas Leggett Chief Financial Officer​ © COPYRIGHT 2026 | STOKE THERAPEUTICS | 17

  18. Slide 18: First Quarter 2026 Financial Summary

    First Quarter 2026 Financial Summary $411.0M Cash, Cash Equivalents, & Marketable Securities as of 3/31/2026 (Includes $80.7M of proceeds from ATM sales of 2.6M shares of common stock) Current financial position anticipated to fund operations into 2028 and supports Phase 3 execution through to a pot

  19. Slide 19: Closing Remarks

    Closing Remarks Ian F. Smith Chief Executive Officer & Director​ © COPYRIGHT 2026 | STOKE THERAPEUTICS | 19

  20. Slide 20: Q&A

    Q&A © COPYRIGHT 2026 | STOKE THERAPEUTICS | 20

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