Stoke Therapeutics, Inc. · NASDAQ:STOK
Stoke Therapeutics — Q2 2025 Earnings Presentation
29 slides from Stoke Therapeutics's earnings presentation, Aug 12, 2025
Slide 1: Second Quarter 2025 Business Update Webcast for Investors & Analysts
Stoke Therapeutics Second Quarter 2025 Business Update Webcast for Investors & Analysts August 12, 2025
Slide 2: Agenda
Long-Term 36-Month OLE Data Kimberly Parkerson, M.D., Ph.D., SVP, Head of Neurology Clinical Development Q&A Agenda CEO Opening Remarks Ian F. Smith, Interim Chief Executive Officer & Director Pipeline Update: ADOA Preclinical Data and Development Plan Barry Ticho, M.D., Ph.D., Chief Medical Officer
Slide 3: 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 4: Opening Remarks
Opening Remarks Ian F. Smith Interim Chief Executive Officer & Director
Slide 5: Stoke is Positioned to Deliver in Dravet and Beyond
Stoke is Positioned to Deliver in Dravet and Beyond Execution Clinical Data Platform Expansion Strength to Deliver Clear path to value creation supported by clinical progress, financial strength, and investment in core capabilities Global Phase 3 Underway First patient dosed in EMPEROR study Robust
Slide 6: Phase 3 Design and Progress
Phase 3 Design and Progress Barry Ticho, M.D., Ph.D. Chief Medical Officer
Slide 7: Current Treatments for Dravet Aim to Reduce Seizures But Do Not Treat Cognitive and Behavioral Challenges
Current Treatments for Dravet Aim to Reduce Seizures But Do Not Treat the Cognitive and Behavioral Challenges Bromide Clobazam Fenfluramine Stiripentol Valproate Cannabinoid Diazepam Levetiracetam Topiramate Zonisamide ? ASM, antiseizure medication; DS, Dravet syndrome; SOC, standard of care. 1. Lag
Slide 8: Initial 70mg Doses of Zorevunersen Demonstrated Substantial and Sustained Reductions in Convulsive Seizure Frequency
Initial 70mg Doses of Zorevunersen Demonstrated Substantial and Sustained Reductions in Convulsive Seizure Frequency on Top of Standard of Care Medicines Laux, L et al. Zorevunersen (STK-001) demonstrates potential for disease modification including reductions in seizures and improvements in cogniti
Slide 9: Ongoing Zorevunersen Treatment Demonstrated Durable Reductions in Major Motor Seizure Frequency
Ongoing Zorevunersen Treatment Demonstrated Substantial and Durable Reductions in Major Motor Seizure Frequency OLE data cut 30 May 2025. 1 patient in the 70 mg 1 dose group who experienced <4 seizures during Phase 1/2 baseline period was excluded. Patients were not included in 6M after last Ph1/2a
Slide 10: Substantial Improvements in Cognition and Behavior Support Phase 3 Design
Substantial Improvements in Cognition and Behavior with Zorevunersen Treatment Support Phase 3 Design Dravet syndrome natural history study patients Patients treated with zorevunersen Phase 1/2a data cut: December 12, 2023 (after End of Study); OLE data cut: June 28, 2024. Mixed-effects models for r
Slide 11: Phase 3 Study Designed to Assess Disease Modification in Dravet Syndrome
First Phase 3 Study Designed to Assess Disease Modification in Dravet Syndrome 1:1 randomization 52-WEEK EFFICACY TREATMENT PERIOD LOADING DOSES (sham or 70mg zorevunersen) MAINTENANCE DOSES (sham or 45mg zorevunersen) 8-week baseline period Dose 1 D1 Dose 4 W40 Dose 2 W8 Dose 3 W24 Dosing regimen o
Slide 12: EMPEROR Phase 3 Study Overview
Primary endpoint Seizures Percent change from baseline in major motor seizure frequency in patients receiving zorevunersen as compared with sham at Week 28 Key secondaries Durability of effect on major motor seizure frequency measured at Week 52 Improvements in behavior & cognition measured by Vinel
Slide 13: EMPEROR Study Supported by Data; First Patient Dosed and Significant Demand
EMPEROR Study Supported by Data; First Patient Dosed and Significant Demand to Enter Trial Large Data Set Informed Phase 3 Study Design Significant & Growing Demand Natural history reflects patient need Ph1/2 data established loading doses OLE data support maintenance regimen Improvements in cogniti
Slide 14: Potential for Disease Modification
Potential for Disease Modification Kimberly Parkerson, M.D., Ph.D. Head of Neurology Clinical Development
Slide 15: Durable Reductions in Seizures Observed Through Three Years of Treatment
Substantial, Durable Reductions in Seizures on Top of SOC Observed Through Three Years of Treatment with Zorevunersen OLE data cut 30 May 2025. One patient who received an incorrect dose of zorevunersen in Phase 1/2a, 3 patients who experienced less than the minimum number of convulsive seizures dur
Slide 16: The Effects of Dravet Go Beyond "Just Seizures"
The Effects of Dravet Go Beyond “Just Seizures” DS, Dravet syndrome. Voice of the patient report. Externally-Led Patient-Focused Drug Development Meeting on Dravet Syndrome. Available at: https://dravetfoundation.org/dsf-funded-research/externally-led-pfdd-meeting/. Accessed March 2025. Difficulties
Slide 17: Development in Dravet Patients Differs Markedly from Neurotypical Children
Development in Patients with Dravet Syndrome Differs Markedly from that of Neurotypical Children Comparison of developmental trajectory between neurotypical children and patients with Dravet syndrome Graph provided for illustrative purposes only. Sullivan et al Natural history of children and adoles
Slide 18: The Vineland-3 Assessment Tool Evaluates Non-Seizure Outcomes in Dravet Syndrome
The Vineland-3 Assessment Tool is Commonly Used to Evaluate Non‑Seizure Outcomes in Dravet Syndrome COMMUNICATION Receptive: Responds upon hearing name called Expressive: Says “Dada”, “Mama”, or caregiver name Written: Writes alphabet letters using correct orientation MOTOR SKILLS Gross Motor: Mov
Slide 19: 36-Month OLE Data: Continuing Improvements in Cognition and Behavior
36 Month Data from Ongoing OLE Studies of Zorevunersen: Continuing Improvements in Cognition and Behavior OLE data cut: 30 May 2025. Mixed-effects model for repeated measures constructed using available data from enrolled patients in OLE studies. One patient who received incorrect dose in Ph1/2a stu
Slide 20: Zorevunersen Generally Well-Tolerated with Long-Term Dosing
Zorevunersen Generally Well-Tolerated with Long-Term Dosing Phase 1/2a data cut: December 12, 2023 (after End of Study); OLE data cut: May 30, 2025. *≥1 CSF protein value >50 mg/dL. Percentage based on 72/75 patients who had ≥1 post-baseline CSF protein value in the OLE studies, of whom 62/72 (86.1%
Slide 21: Pipeline Update: ADOA Preclinical Data and Development Plan
Pipeline Update Autosomal Dominant Optic Atrophy (ADOA) Preclinical Data and Development Plan
Slide 22: Autosomal Dominant Optic Atrophy: A Progressive Vision Disorder Caused by OPA1 Haploinsufficiency
Autosomal Dominant Optic Atrophy (ADOA): A Progressive Vision Disorder Caused by OPA1 Haploinsufficiency Sources: Kjer. Acta Ophthalmologica Scandinavica. 1996; Yu-Wai-Man P et al. Ophthalmology, 2010; Yu-Wai-Man P, Chinnery PF. Ophthalmology, 2013; Physician and Payer Interviews; P. Amati-Bonneau P
Slide 23: Compelling Preclinical Findings Support Further Development of STK-002
Compelling Preclinical Findings Support Further Development of STK-002 Summary of Key Preclinical Data Increased OPA1 protein and ATP-linked respiration in ADOA patient fibroblasts Dose-dependent increases in OPA1 protein expression detected in mouse and rabbit retinas Well tolerated for up to 29 da
Slide 24: New Evidence of Early Efficacy and Tolerability of STK-002 in ADOA NHP Model
New Evidence of Early Efficacy and Tolerability of STK-002 in ADOA Non-Human Primate Model Continued improvement in retinal function (PhNR¥) PhNR as measured by ERG PhNR amplitude increased over baseline Number of eyes (6 control, 6 treated eyes) Continued improvement or stabilization in mitochondri
Slide 25: Phase 1 Study in ADOA Patients Now Underway in UK
Phase 1 Study in ADOA Patients Now Underway in UK Phase 1 Study (OSPREY) Design (UK and Europe) 1 2 3 4 Intravitreal injection in one eye 48-week duration Patients ages ≥18 to <55y (n=21) Single ascending doses (0.1, 0.3, 0.5, & 0.7 mg/eye) Primary Endpoint: Safety, tolerability, and exposure of sin
Slide 26: Financial Summary
Financial Summary Tommy Leggett Chief Financial Officer
Slide 27: Closing Remarks
Closing Remarks Ian F. Smith Interim Chief Executive Officer & Director
Slide 28: Stoke is Positioned to Deliver in Dravet and Beyond
Stoke is Positioned to Deliver in Dravet and Beyond Execution Clinical Data Platform Expansion Strength to Deliver Clear path to value creation supported by clinical progress, financial strength, and investment in core capabilities Global Phase 3 Underway First patient dosed in EMPEROR study Robust
Slide 29: Stoke Therapeutics
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