Stoke Therapeutics, Inc. · NASDAQ:STOK

Stoke Therapeutics — Q2 2026 Earnings Presentation

21 slides from Stoke Therapeutics's earnings presentation, Aug 3, 2026

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

    Stoke Therapeutics Second Quarter 2026 Business Update Webcast for Investors & Analysts August 3, 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: Advancing Through a Period of Execution and Expansion

    Advancing Through a Period of Execution and Expansion Zorevunersen U.S. NDA Pipeline Beyond Phase 3 Progress Preparation Underway Zorevunersen • Enrollment of 162 patients • Pre-NDA meeting with FDA • Dose escalation continuing completed in June 2026 scheduled for H2 2026 following completion of sen

  5. Slide 5: Phase 3 Progress and 4-Year OLE Data

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

  6. Slide 6: Phase 3 Enrollment of Patients Completed in June 2026 Supports a Data Readout in Q3 2027

    Phase 3 Enrollment of 162 Patients Completed in June 2026 Supports a Data Readout in Q3 2027 Patient Progression Through Key Study Milestones ~145 through Week 8 • 162 patients enrolled (U.S., UK, Japan) in 10 months completion of 2x70mg loading doses or sham • Final patient randomized in June 2026

  7. Slide 7: Substantial, Durable Reductions in Seizure on Top of SOC

    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

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

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

  9. Slide 9: 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 Phase 3 EMPEROR Year 1 (n=68) Year 2 (n=60) Subdomains Year 3 (n=42) Year 4 (n=14) Datacut date: 19 Feb 2026; no patie

  10. Slide 10: 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

  11. Slide 11: Confidence in the Value of zorevunersen as a Potential Disease-Modifying Treatment for Dravet Syndrome

    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

  12. Slide 12: Expanding the Pipeline

    Expanding the Pipeline Barry Ticho, M.D., Ph.D. Chief Medical Officer © COPYRIGHT 2026 | STOKE THERAPEUTICS | 12

  13. Slide 13: Autosomal Dominant Optic Atrophy (ADOA): A Progressive Vision Disorder Caused by OPA1 Haploinsufficiency

    Autosomal Dominant Optic Atrophy (ADOA): A Progressive Vision Disorder Caused by OPA1 Haploinsufficiency 65-90% 1 out of 30,000 of cases caused by mutations in one allele Up to ~13,000 people are affected globally with a people affected in the U.S., UK, EU-4, of the OPA1 gene, most of which lead to

  14. Slide 14: Phase 1 OSPREY Study of STK-002 in Patients with ADOA

    Phase 1 OSPREY Study of STK-002 in Patients with ADOA Dosing complete in sentinel cohort Dose escalation continuing and initial data anticipated in H1 2027 Phase 1 Study Design (UK and Europe) Primary Endpoint: Safety, tolerability, and exposure of single ascending doses of STK-002 1 Intravitreal in

  15. Slide 15: Preparing for a Potential U.S. Launch

    Preparing for a Potential U.S. Launch Jason Hoitt Chief Patient Officer © COPYRIGHT 2026 | STOKE THERAPEUTICS | 15

  16. Slide 16: 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

  17. Slide 17: Concentrated U.S. Market and Established Infrastructure Provide Foundation for Early Adoption

    Concentrated U.S. Market and Established Infrastructure Provide Foundation for Early Adoption Treating Sites ~50% of identified U.S. patients are cared for by the top 50 sites “We’ve used Spinraza for years and we’ve already Existing Infrastructure established the infrastructure of recurrent 100% of

  18. Slide 18: Path to Potential U.S. Approval and Launch

    Path to Potential U.S. Approval and Launch March: Phase 1/2 and 3-Year OLE Data Published in The New England Journal of Medicine May: 4-Year Phase 1/2 OLE Data Announced June: Completion of enrollment of 162 patients into EMPEROR (U.S., UK, JP) H2 2027 H2 2026 Completion of Rolling Pre-NDA Meeting w

  19. Slide 19: Financial Summary

    Financial Summary Thomas Leggett Chief Financial Officer​ © COPYRIGHT 2026 | STOKE THERAPEUTICS | 19

  20. Slide 20: Second Quarter 2026 Financial Summary

    Second Quarter 2026 Financial Summary $420 Million Pro-Forma Cash, Cash Equivalents, & Marketable Securities based on $354.3M as of June 30, 2026 and $65.7M from a subsequent ATM sale Current financial position anticipated to fund operations through to potential U.S. commercialization of zorevunerse

  21. Slide 21: Q&A

    Q&A © COPYRIGHT 2026 | STOKE THERAPEUTICS | 21

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