Stoke Therapeutics, Inc. · NASDAQ:STOK
Stoke Therapeutics — Q3 2026 Earnings Presentation
25 slides from Stoke Therapeutics's earnings presentation, Oct 29, 2026
Slide 1: Stoke Therapeutics Third Quarter 2026 Business Update
Stoke Therapeutics Third Quarter 2026 Business Update Webcast for Investors & Analysts October 29, 2026 © COPYRIGHT 2026 | STOKE THERAPEUTICS | 1
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
Slide 3: Opening Remarks
Opening Remarks Ian F. Smith Chief Executive Officer & Director © COPYRIGHT 2026 | STOKE THERAPEUTICS | 3
Slide 4: Advancing Zorevunersen to Patients
Advancing Zorevunersen to Patients EMPEROR Phase 3 Progress FDA Meeting to Prepare for NDA Commercial Readiness ~110 Patients Through Week 28 Reviewed 5 Years of Clinical Data ~6,000 Patients measurement of primary endpoint from Phase 1/2 and ongoing OLE studies addressable at launch ~20 Patients Th
Slide 5: Expanding Our Research and Building Our Pipeline
Expanding Our Research and Phase 1 Study Underway of STK-002 in Patients with ADOA Building Our Rare, genetic disease Pipeline Most common optic nerve disorder Primarily caused by haploinsufficiency of OPA1 protein With an Emphasis On No approved treatments Severe genetic diseases Preclinical data s
Slide 6: Phase 3 EMPEROR Progress
Phase 3 EMPEROR Progress Barry Ticho, M.D., Ph.D. Chief Medical Officer © COPYRIGHT 2026 | STOKE THERAPEUTICS | 6
Slide 7: EMPEROR: A Global, Double-Blind, Sham Controlled Phase 3 Study
EMPEROR: A Global, Double-Blind, Sham Controlled Phase 3 Study In patients 2 to <18 years old with a confirmed SCN1A gene variant not associated with gain of function 1:1 randomization Baseline Open-label screening extension W28 W52 D1 W8 W24 Primary W40 Secondary Dose Dose Dose Endpoint Dose Endpoi
Slide 8: Patient Progression Through Key EMPEROR Timepoints
Patient Progression Through Key EMPEROR Timepoints No Discontinuations to Date Patient Progress Through Key Study Milestones 162 ~130 ~110 ~65 ~20 1:1 randomization Baseline Open-label screening extension W28 W52 D1 W8 W24 Primary W40 Secondary Dose Dose Dose Endpoint Dose Endpoints 1 2 3 4 LOADING
Slide 9: EMPEROR is Designed to Evaluate the Disease-Modifying Potential of Zorevunersen
EMPEROR is Designed to Evaluate the Disease-Modifying Potential of Zorevunersen Study Endpoints Multicomponent Assessment PRIMARY Percent change from baseline in major Four Vineland-3 Subdomains: motor seizure frequency at Week 28 • Expressive Communication • Receptive Communication KEY SECONDARY •
Slide 10: Matching Adjusted Indirect Comparison of Patient Data
Matching Adjusted Indirect Comparison of Patient Data Supported Phase 3 Selection of Dose and Vineland Subdomains Patients treated with zorevunersen Dravet syndrome natural history study patients 10.7 20 20 raw scores (95% CI) from baseline raw scores (95% CI) from baseline 8.8 8.7 LS mean change in
Slide 11: Statistically Significant Improvements in Cognition and Behavior Using a Multicomponent Assessment
Statistically Significant Improvements in Cognition and Behavior Using a Multicomponent Assessment Support Planned Phase 3 Analysis Treatment effect in Phase 1/2 and OLE patients who received cumulative doses of zorevunersen consistent with Phase 3, compared to natural history at 18 months P-value E
Slide 12: EMPEROR is Powered to Detect Statistically Significant Treatment Effects
EMPEROR is Powered to Detect Statistically Significant Treatment Effects in Primary and Key Secondary Endpoints Patient enrollment and progression with no discontinuations, combined with planned multicomponent assessment, enhance statistical power Endpoints Minimum Detectable Treatment Effect Modele
Slide 13: Advancing STK-002 for ADOA
Advancing STK-002 for ADOA Barry Ticho, M.D., Ph.D. Chief Medical Officer © COPYRIGHT 2026 | STOKE THERAPEUTICS | 13
Slide 14: Autosomal Dominant Optic Atrophy (ADOA): A Progressive Vision Disorder
Autosomal Dominant Optic Atrophy (ADOA): A Progressive Vision Disorder Primarily Caused by OPA1 Haploinsufficiency Disease and Cause Epidemiology Burden 65-90% 1 out of 25,000 up to of patients of cases caused by mutations people are affected globally 46% are registered in one copy of the OPA1 gene,
Slide 15: STK-002 Shows Increases in OPA1 Protein and Improvements in Mitochondrial Function
STK-002 Shows Statistically Significant Increases in OPA1 Protein and Improvements in Mitochondrial Function in ADOA Patient‐Derived Cells Increased OPA1 protein expression Improved mitochondrial function (maximal respiration,pmol/min/cell)2 Vehicle STK-002 40 nM Relative mitochondrial function 2.0
Slide 16: Single Intravitreal Dose of STK-002 Increased Retinal OPA1 Protein in Non-Human Primates
A Single Intravitreal Dose of STK-002 Increased Retinal OPA1 Protein by 47% at 4 Weeks in Wild-Type Non-Human Primates Versus Control STK-002 detected in RGC layer OPA1 protein in RGC layer OPA1 protein in retinal lysates P<0.05 10 P<0.05 OPA1 protein (µg/mg) 8 6 4 2 0 Vehicle 0.1mg 0.3mg 1mg 0.3mg
Slide 17: Phase 1 OSPREY Study of STK-002 in Patients with ADOA
Phase 1 OSPREY Study of STK-002 in Patients with ADOA Part A: Adults (≥18 to <55 years) Principal focus 4 ascending dose cohorts • Safety and tolerability + + Day 1 Remaining Screening & First patient in Safety review patients in Safety review Baseline cohort dosed Day 4 cohort dosed Day 29 Other me
Slide 18: OSPREY: Dose Escalation Continuing with Initial Data Expected in H1 2027
OSPREY: Dose Escalation Continuing with Initial Safety & Efficacy Data Expected in H1 2027 Cohorts 3 and 4 have the potential to reach therapeutic levels of dosing Significance Status Future May demonstrate whether Dosing complete in cohorts Inform dose selection STK-002’s ability to #1 and #2 and f
Slide 19: Preparing for a Potential U.S. Launch
Preparing for a Potential U.S. Launch Jason Hoitt Chief Patient Officer © COPYRIGHT 2026 | STOKE THERAPEUTICS | 19
Slide 20: Significant Market Opportunity
Significant Market Opportunity ~38K patients with Dravet syndrome across 7 major geographic markets Prevalence of Dravet Syndrome2 Concentrated U.S. Market 7K Japan ~6,000 patients immediately addressable at launch • <25 years old and likely in the care of a pediatric provider 16K • Insights into wh
Slide 21: Established Infrastructure at Key Sites of Care Provides Foundation for Early Adoption
Established Infrastructure at Key Sites of Care Provides Foundation for Early Adoption Treating Sites Existing Infrastructure Zorevunersen Experience ~50% 100% ~50% of identified U.S. patients are of top 50 sites have of top 50 sites have participated in cared for by the top 50 sites established int
Slide 22: Preparing to Deliver Zorevunersen
Preparing to Deliver Zorevunersen Adding Capabilities Expanding Education Collaborative Efforts Patient Services to Continuing engagement Genetic testing enable timely access with HCPs initiatives Payer Account Team Expansion into broader Medical society to educate medical directors neurology commun
Slide 23: Financial Summary
Financial Summary Thomas Leggett Chief Financial Officer © COPYRIGHT 2026 | STOKE THERAPEUTICS | 23
Slide 24: Third Quarter 2026 Financial Summary
Third Quarter 2026 Financial Summary $358.8 Million Cash, Cash Equivalents, & Marketable Securities as of September 30, 2026 Current financial position anticipated to fund operations through to early 2028 Note: Excludes $3.2 million of restricted cash. © COPYRIGHT 2026 | STOKE THERAPEUTICS | 24
Slide 25: Q&A
Q&A © COPYRIGHT 2026 | STOKE THERAPEUTICS | 25