Stoke Therapeutics, Inc. · NASDAQ:STOK
Stoke Therapeutics — Investor Presentation (June 2025)
25 slides from Stoke Therapeutics's investor presentation, Jun 4, 2025
Slide 1: Stoke Therapeutics June 2025
Exhibit 99.1 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
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 represe
Slide 3: Our Goal: Restore protein expression with RNA medicine
OUR GOAL Restore protein expression by harnessing the body’s potential with RNA medicine Stoke’s pipeline offers potential first-in-class disease modifying new medicines for diseases caused by protein insufficiency STK-002 for Autosomal zorevunersen Dominant Optic Atrophy SYNGAP1 And beyond… for Dra
Slide 4: Advantages of Stoke's Approach vs. Other Genetic Approaches
ADVANTAGES OF STOKE’S APPROACH vs. OTHER GENETIC APPROACHES Selectively boosts expression No observed unwanted Utility across small and only in tissues where the protein off-target genetic effects large gene targets and is normally expressed mutations Does not alter DNA Ability to control dose level
Slide 5: Dravet Syndrome: A Severe, Progressive Genetic Epilepsy
Dravet Syndrome: A Severe, Progressive Genetic Epilepsy 2 2 ~38,000 1 OUT OF 15,600 babies are born with Dravet syndrome Patients with Dravet syndrome in of cases caused by a the U.S., Japan, Germany, France, HAPLOINSUFFICIENCY Italy, Spain and UK of the SCN1A gene of children and adolescents with U
Slide 6: Current Treatments For Dravet Aim to Reduce Seizures
Current Treatments For Dravet Aim to Reduce Seizures Leaving a Significant Gap in Treatment of the Syndrome MULTIPLE MEDICINES available for Currently NO MEDICINES available for Seizure Management Syndrome Management Bromide Cannabinoid Clobazam Diazepam Fenfluramine Levetiracetam Stiripentol Topira
Slide 7: The Effects of Dravet Go Beyond "Just Seizures"
The Effects of Dravet Go Beyond “Just Seizures” All aspects of life are affected, not only for the individual living with DS, but for their caregivers and families Seizures Stress Developmental Financial deficits burden Behavioral Work Difficulties with changes productivity Social balance, walking,
Slide 8: Natural History Data: No Meaningful Improvement in Seizure Frequency
Natural History Data: Despite Standard Anti-Seizure Medicines, No Meaningful Improvement in Convulsive Seizure Frequency Patients were treated with the Change in Convulsive Seizure Frequency best available anti-seizure medicines Median baseline convulsive seizure frequency per 28 days (95% CI), n=26
Slide 9: Clinical Data and Stakeholder Engagement Underscore Potential for Zorevunersen
Compelling Clinical Data and Key Stakeholder Engagement Underscore the Potential for Zorevunersen FDA Breakthrough Biogen Phase 3 Study Clinical Data Underway Therapy Collaboration for Substantial and durable reductions in seizure Designation Zorevunersen First sites initiated in the frequency and c
Slide 10: Initial 70mg Doses Demonstrated Substantial Reductions in Seizure Frequency
Initial 70mg Doses of Zorevunersen Demonstrated Substantial and Sustained Reductions in Convulsive Seizure Frequency on Top of Standard of Care Medicines Median percent change in convulsive seizure frequency 70 mg 1 dose from baseline in Phase 1/2a studies 70 mg 2 dose 50 70 mg 3 dose 0 ZZ -50 -100
Slide 11: Ongoing Treatment Demonstrated Durable Reductions in Seizure Frequency
Ongoing Treatment Demonstrated Substantial and Durable Reductions in Convulsive Seizure Frequency 70 mg 1 dose* Initial 2 & 3 doses of 70mg in Ph1/2 followed by 45mg 70 mg 2 dose in OLE demonstrated up to 87% reduction at Month 8 70 mg 3 dose 0 -25 ZZ -50 -75 -100 M1 M2 M3 M4 M5 M6 M7 M8 End of 45mg
Slide 12: Durable Reductions in Seizures Through Two Years of Treatment
Substantial, Durable Reductions in Seizures On Top of SOC Observed Through Two Years of Treatment with Zorevunersen Enrolled patients Data for all patients who continued treatment in the OLEs 70 mg in Ph 1/2a and ≥30 mg in OLE 0 -25 ZZ -50 -75 -100 M1 M2 M3 M4 M5 M6 M7 M8 M9 M10 M11 M12 M13 M14 M15
Slide 13: Vineland-3 Assessment Tool for Non-seizure Outcomes
The Vineland-3 Assessment Tool is Commonly Used to Evaluate Non-seizure Outcomes in Dravet Syndrome Vineland-3 Adaptive Behavior Scales* COMMUNICATION MOTOR SKILLS SOCIALIZATION DAILY LIVING SKILLS Receptive: Gross Motor: Interpersonal Personal: Responds upon hearing Moves, scoots, or crawls Relatio
Slide 14: Adaptive Functioning and Neurodevelopment Plateau in Dravet Syndrome
Adaptive Functioning and Neurodevelopment Plateau in Patients with Dravet Syndrome Widening development gap over time in patients with Dravet syndrome relative to neurotypical peers DS, Dravet syndrome; Vineland-3, Vineland Adaptive Behavior Scales, Third Edition. 1. Sullivan J et al. Poster P788 Pr
Slide 15: Improvements in Cognition and Behavior Continue Throughout the OLEs
Improvements in Cognition and Behavior Within 9 Months Continuing Improvements Throughout the OLEs Vineland-3 GSV scores in OLE build on improvements already observed in Phase 1/2 2 1 Open-label extension studies Phase 1/2a study Month 12 Month 24 LS mean change from Phase 1/2a baseline in GSV (95%C
Slide 16: Zorevunersen Generally Well-Tolerated Across Studies
Zorevunersen Generally Well-Tolerated Across Studies Phase TEAEs 1/2a • 30% of patients experienced a study drug-related TEAE To date, • Most common: CSF protein elevations (13.6%) and procedural vomiting (4.9%) studies (n=81) >700 doses of TESAEs † zorevunersen • 22% of patients experienced a TESAE
Slide 17: EMPEROR Phase 3 Study Overview
EMPEROR Phase 3 Study Overview Phase 3 design and dose regimen aligned with Ph1/2 and OLE studies Primary endpoint Key secondaries Study Design: Sham-controlled, 1:1 randomization Seizures Durability of effect on Dosing Regimen: 2x70mg + 2x45mg major motor seizure Percent change from Study Start: Ma
Slide 18: First Phase 3 Study Designed to Assess Disease Modification
First Phase 3 Study Designed to Assess Disease Modification in Dravet Syndrome Dosing Regimen of 2x70mg followed by 2x45mg based on positive data from Phase 1/2a and OLE studies 8-week baseline period 1:1 randomization 52-WEEK EFFICACY TREATMENT PERIOD D1 W8 W24 W40 Dose Dose Dose Dose 1 2 3 4 MAINT
Slide 19: Zorevunersen is Positioned to Change the Treatment of Dravet Syndrome
Zorevunersen is Positioned to Change the Treatment of Dravet Syndrome SIGNIFICANT NEED CLINICAL DATA STAKEHOLDER SUPPORT Current treatments focus on Substantial and durable reductions HCPs and caregivers have had an reducing seizure frequency. in seizures and continuing overwhelming positive reactio
Slide 20: Substantial Patient Population
Substantial Patient Population ~38K patients with Dravet syndrome across 7 major markets PREVALENCE OF SIGNIFICANT UNMET NEED DESPITE ANTI-SEIZURE MEDICINES DRAVET SYNDROME* 7K Japan No disease modifying medicines are currently available Seizures are inadequately controlled in 90% of patients 16K 1
Slide 21: Approximately 90% of HCPs See a Significant Unmet Need
Approximately 90% of HCPs See a Significant Unmet Need for Patients with Dravet Syndrome Most pressing unmet needs identified by HCPs* Percent of respondents 100% 75% 50% 25% 0% Persistent patient seizure Lack of disease modifying Limited efficacy of existing Side effects burden treatment option age
Slide 22: Strategic Collaboration with Biogen for Zorevunersen
Strategic Collaboration with Biogen to Develop and Commercialize Zorevunersen For Dravet Syndrome Collaboration leverages Biogen’s expertise commercializing high-value, disease- modifying medicines for rare genetic diseases Stoke leads global development and retains Biogen receives exclusive rest of
Slide 23: Our Pipeline of First-in-Class Disease-Modifying Potential Medicines
Our Pipeline of First-in-Class Disease-Modifying Potential Medicines DISCOVERY & PROGRAM TARGET PHASE 1/2 PHASE 3 PARTNER PRECLINICAL CENTRAL NERVOUS SYSTEM Stoke: United States, zorevunersen Dravet Syndrome SCN1A Canada, Mexico Biogen: Rest of World SYNGAP1 SYNGAP1 Stoke: Acadia 50:50 Worldwide OPH
Slide 24: Recent Achievements and Priorities Going Forward
Recent Achievements and Priorities Going Forward Advance Zorevunersen Breakthrough Therapy Designation • Drive Enrollment in Phase 3 • Drive Greater Awareness and Understanding of Potential Positive Phase 1/2a and OLE Study Data to for Disease-Modification Support and De-Risk Phase 3 Biogen Collabor
Slide 25: Copyright Stoke Therapeutics, Inc.
Copyright Stoke Therapeutics, Inc. Not for publication or distribution. © © 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 | | 25
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