Stoke Therapeutics, Inc. · NASDAQ:STOK
Stoke Therapeutics — 42nd Annual J.P. Morgan Healthcare Conference 2024
22 slides from Stoke Therapeutics's conference presentation, Jan 8, 2024
Slide 1: Stoke Therapeutics at 42nd Annual J.P. Morgan Healthcare Conference
Exhibit 99.1 Stoke Therapeutics NASDAQ: STOK Edward M. Kaye, M.D. Chief Executive Officer nd 42 Annual J.P. Morgan Healthcare Conference January 10, 2024 © Copyr ©i g Ch op t 2024 yrightS 2024 toke T Sh tok era ep T e h u e tr ic as peutic1 s
Slide 2: Disclaimer
Disclaimer 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 representation by the presenter or Stoke or any
Slide 3: © © S pe eu ut ti ic cs s
© © C Cop opyr yri ig gh ht t 2024 2024 S St tok oke e T Th he er ra ap pe eu ut ti ic cs s 3 3
Slide 4: Dravet syndrome prevalence and affected population
1 out of 16,000 babies are born with Dravet syndrome Dravet syndrome is not concentrated in a particular geographic area or ethnic group ~35,000 people affected in: United States Canada Japan Germany France United Kingdom Sources: 2018 Health Advances Report © © C Cop opyr yri ig gh ht t 2024 2024 S
Slide 5: 50% of Epilepsies are Unknown or Idiopathic; Majority Now Thought Genetic
50% of Epilepsies are Unknown or Idiopathic; Majority of These are Now Thought to be Genetic Epilepsy affects approximately 1.2 % ~85% of the US population Causes: of Dravet cases caused by a HAPLOINSUFFICIENCY • Trauma of the SCN1A gene • Tumor • Stroke Resulting in • Infection • Developmental abno
Slide 6: Our Goal: Upregulate Protein Expression to Treat Underlying Cause
Our Goal: Upregulate Protein Expression to Treat the Underlying Cause of Severe Genetic Diseases Mechanism of action for TANGO Non-productive exons control protein levels by alternative splicing pre-mRNA mRNA Full-length functional protein (productive splicing) PTC triggers degradation via NMD (non-
Slide 7: The Effects of Dravet Go Beyond "Just Seizures"
The Effects of Dravet Go Beyond “Just Seizures” Growth Time Defects Loss Intellectual Disruptions Disability & Side Effects of Autonomic Developmental Nervous Delays ~20% of children and adolescents System with Dravet syndrome die before Direct Home 1 Medical adulthood, due to SUDEP , prolonged Modi
Slide 8: STK-001 on Track to be First Disease-Modifying Treatment for Dravet Syndrome
STK-001 is on Track to be the First Disease-Modifying Treatment for Dravet Syndrome Multiple medicines available for No medicines available for Seizure management Syndrome management Available medicines used to control seizures: • Acetazolamide • Felbamate • Rufinamide • Benzodiazepines • Fenflurami
Slide 9: Data Support the Potential for Disease Modification with STK-001
Data Support the Potential for Disease Modification with STK-001 BUTTERFLY Natural History Data Data from Phase 1/2a and OLE (patients 2-18 taking standard anti-seizure medicines) Clinical Studies Safety Generally well-tolerated No meaningful improvement in seizure Substantial and sustained reductio
Slide 10: No Improvement in Convulsive Seizure Frequency Despite Standard Medicines Over 2 Years
No Improvement in Convulsive Seizure Frequency Despite Treatment with Standard Anti-Seizure Medicines Over 2 Years BUTTERFLY natural history study of 2-18 years old patients with Dravet syndrome Patients were treated with the best available anti-seizure medicines Median baseline convulsive seizure f
Slide 11: Substantial Reductions in Seizure Frequency with Initial Doses of STK-001 (70mg)
Substantial Reductions in Seizure Frequency Observed in Patients Treated with 2 or 3 Initial Doses of STK-001 (70mg) Data as of July 2023. All patients have now completed the study. End of study data anticipated 1Q 2024. 6 patients 3 patients *D1-D28: +825%, D29-D56: +626%, D57-D84: +1125%, D85-D112
Slide 12: Median Reductions in Convulsive Seizure Frequency at 3 and 6 Months
Median Reductions in Convulsive Seizure Frequency Observed Among All Patients with 2 or 3 Doses of 70mg at 3 and at 6 Months 6 Patients at 3 Months 3 Patients at 6 Months After Last Dose After Last Dose Ages 2 to 12 Years Ages 2 to 12 Years Ages 13 to 18 Years Ages 13 to 18 Years -50% -50% *28-day i
Slide 13: OLE Data: Sustained Reductions in Convulsive Seizure Frequency
OLE Data: Sustained Reductions in Convulsive Seizure Frequency Effects observed with ongoing treatment with STK-001 at 30mg, 45mg Weeks Weeks Weeks Weeks Weeks Weeks Weeks Weeks Weeks Weeks Weeks Weeks 1-4 5-8 9-12 13-16 17-20 21-24 25-28 29-32 33-36 37-40 41-44 45-48 SWALLOWTAIL: Maintenance Dose (
Slide 14: Natural History Shows Widening Gap in Cognition and Behavior
Natural History Shows Widening Gap in Cognition and Behavior Compared to Neurotypical Peers Dravet patients were taking standard anti-seizure medicines throughout the 2-years of study Source: 24-Month Analysis of BUTTERFLY: A Prospective, Observational Study to Investigate Cognition and Other Non-se
Slide 15: OLE Data: Improvements in Communication and Gross Motor Skills
OLE Data (30mg, 45mg): Substantial Improvements in Communication and Gross Motor Skills with Ongoing Treatment Improving 12.25 11.00 7.32 10.16 6.25 5.63 5.07 4.50 4.28 -1.00 -2.09 -2.30 -2.95 -3.21 -3.94 -12.58 -13.34 -13.95 Worsening BUTTERFLY (Natural History) SWALLOWTAIL (STK-001) *Mixed model r
Slide 16: OLE Data: Improvements in Executive Function
OLE Data (30mg, 45mg): Substantial Improvements in Executive Function with Ongoing Treatment Worsening 5.29 2.83 0.95 -3.99 -5.01 -6.54 Improving SWALLOWTAIL (STK-001) BUTTERFLY (Natural History) *Mixed model repeated measures with AR(1) covariance structure. Baseline covariates in BUTTERFLY matched
Slide 17: OLE Data: Improvements in Overall Condition Compared to Natural History
OLE Data (30mg, 45mg): Substantial Improvements in Overall Condition Compared to BUTTERFLY Natural History Results Consistent responses across caregiver and clinician ratings Very much worse 4.48 4.40 4.32 No change 4.24 4.23 3.98 2.80 2.68 2.50 2.23 1.95 1.53 Very much improved SWALLOWTAIL (STK-001
Slide 18: Higher Brain Exposure Leads to Greater Seizure Reduction
Higher Brain Exposure Leads to Greater Seizure Reduction PK Modeling of exposure-seizure relationship 300 R=-0.23, p=0.00000000025 200 100 0 Baseline -100 Seizure Free Average Concentration of Mean Brain Regions, excluding Thalamus © Copyright 2024 Stoke Therapeutics 18 Source: Utilization of a Phar
Slide 19: Summary of Key Clinical Data from Ongoing Studies
Summary of Key Clinical Data from Ongoing Studies Single and multiple doses of 10mg to 70mg were ü generally well-tolerated Patients treated with 2 or 3 doses of 70mg experience STK-001 Has Potential to substantial and sustained reductions in convulsive ü Address the Genetic Cause seizures Reduction
Slide 20: Q1 2024 Data Readout & Next Steps
Q1 2024 Data Readout & Next Steps Rest of Year Q1 2024 Phase 3 Preparations Key Anticipated Data qSafety, pharmacokinetic (PK) modeling, q Global regulatory interactions and cerebrospinal fluid (CSF) q Chronic toxicology data qSeizure frequency from ~20 patients who q Protocol finalization/submissio
Slide 21: 2024 Summary of Priorities
2024 Summary of Priorities Advance STK-002 for ADOA Advance STK-001 for Dravet • Initiate Phase 1 study (OSPREY) in 2024 Syndrome to Pivotal Develop & Expand Pipeline • Q1 Data Readout • Execute on collaboration with Acadia to advance 3 • Pending data, request Phase 3 neurodevelopmental programs inc
Slide 22: Q&A
Q&A © Copyright 2024 Stoke Therapeutics 22
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