Slide 1: Interim Analysis of STK-001 for the Treatment of Dravet Syndrome
Interim Analysis of STK-001 for the Treatment of Dravet Syndrome Stoke Therapeutics November 14, 2022 Exhibit 99.2
Slide 2: Agenda
Agenda Introduction Eric Rojas, Head of Investor Relations Introductory Remarks Edward M. Kaye, M.D., Chief Executive Officer Phase 1/2a Interim Analysis Barry Ticho, M.D., Ph.D., Chief Medical Officer Closing Remarks Edward M. Kaye, M.D., Chief Executive Officer Q&A
Slide 3: Forward Looking Statements
Forward Looking Statements This presentation has been prepared by Stoke Therapeutics, Inc. (“Stoke” or “our”) 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 presen
Slide 4: Introductory Remarks
Introductory Remarks Edward M. Kaye, M.D. Chief Executive Officer
Slide 5: Dravet Syndrome: A Severe, Progressive Genetic Epilepsy
1 Sudden Unexpected Death in Epilepsy Sources: 2018 Health Advances Report; Djémié et al., Molecular Genetics & Genomic Medicine, 2016; Lagae et al., Developmental Medicine & Child Neurology, 2017; Nabbout et al., Orphanet Journal of Rare Diseases, 2013 ~35,000 Seizures are not adequately controlled
Slide 6: Non-Seizure Comorbidities Are Not Addressed by Current Therapies
Non-Seizure Comorbidities of Dravet Syndrome Are Not Addressed by Current Therapies No Approved Disease-Modifying Therapies for Dravet Syndrome Intellectual disability Developmental delays Movement and balance issues Language and speech disturbances Growth defects Sleep abnormalities Disruptions of
Slide 7: Non-Seizure Comorbidities of DS are Progressive and Measurable
* VABS = Vineland Adaptive Behavior Scales * ABC score based on Communication, Daily Living, and Socialization domains and expressed relative to normative mean of 100 Source: BUTTERFLY: An Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents wi
Slide 8: Boldly Restoring Genetic Health
Boldly Restoring Genetic Health Addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines Executing in the clinic with STK-001, the first potential disease-modifying approach for the treatment of Dravet syndrome Advancing to the clinic with STK-00
Slide 9: Interim Analysis of Phase 1/2a MONARCH and ADMIRAL Studies of STK-001
Interim Analysis of Phase 1/2a MONARCH and ADMIRAL Studies of STK-001 Barry Ticho, M.D., Ph.D. Chief Medical Officer
Slide 10: TANGO-ASO Mechanism: Nonsense Mediated Decay
Nonsense mediated decay (NMD) exon without TANGO-ASO Increased functional protein expression Nonsense mediated decay (NMD) exon with TANGO-ASO NMD: nonsense-mediated decay
Slide 11: Clinical Study Designs: MONARCH SAD, MAD and ADMIRAL MAD
Clinical Study Designs: MONARCH SAD, MAD and ADMIRAL MAD
Slide 12: Demographics for Patients Treated With >1 Dose of STK-001
Demographics for 55 Patients Treated With >1 Dose of STK-001 Enrolled Patients Have Severe Disease and are Refractory to Standard Treatments *N=45
Slide 13: Multiple Doses of STK-001 up to 45mg Well-Tolerated
Multiple Doses of STK-001 up to 45mg Well-Tolerated 27% (15/55) of patients experienced a TEAE related to study drug; All AEs related to study drug were mild or moderate 22% (12/55) of patients had a treatment-emergent SAE none related to study drug Most common TEAEs were vomiting, headache, and sei
Slide 14: Dose-Dependent Increases in Cmax and AUC24hr Observed in Plasma
Dose-Dependent Increases in Cmax and AUC24hr Observed in Plasma A greater increase was observed between 30mg and 45mg than between 20mg and 30mg Data Cut-off date: April 26, 2022
Slide 15: CSF Exposure Measurable up to Six Months Following Multiple Doses
CSF Exposure was Measurable up to Six Months Following Multiple Doses of STK-001 in MONARCH study Slight increases in CSF values were observed from 20mg to 30mg Data Cut-off date: April 27, 2022
Slide 16: Majority of Patients Reach Pharmacologically Active Brain Levels
Majority of Patients Treated with Multiple Doses (20mg or 30mg) in MONARCH Reach Pharmacologically Active Brain Levels Clinical Data for 20mg and 30mg MAD Align with Model Simulations from Patient CSF Levels Solid line: Median of the predicted concentrations, Shaded area: 95th confidence interval (2
Slide 17: 55% Median Reduction in Convulsive Seizure Frequency at 45mg
55% Median Reduction in Convulsive Seizure Frequency Observed in Patients Treated With Three Doses of STK-001 (45mg) Across the multiple dose cohorts (20mg, 30mg, 45mg), 74% (20/27) of patients experienced reductions in seizures Similar seizure reduction was observed among patients taking or not tak
Slide 18: Reductions in Convulsive Seizure Frequency Across Age Groups
Reductions in Convulsive Seizure Frequency Observed Across Age Groups Seizure reductions more evident among patients ages 2-12
Slide 19: Phase 1/2a Trials of STK-001 are Ongoing
Phase 1/2a Trials of STK-001 for Dravet Syndrome are Ongoing Design Enrollment and dosing ongoing (30mg) Enrollment and dosing ongoing (45mg) Change in seizure frequency, overall clinical status, and quality of life Parallel studies in the US & UK evaluating children and adolescents ages 2 to 18 yea
Slide 20: Early Indication of Improvements in Non-Seizure Comorbidities (BRIEF-P)
Early Indication of Improvements in Non-Seizure Comorbidities as Measured by BRIEF-P* *Behavior Rating Inventory of Executive Function–Preschool Version, an assessment of pediatric executive function
Slide 21: Conclusions and Next Steps
Conclusions and Next Steps STK-001 on track as the first potential disease-modifying treatment for Dravet syndrome Multiple doses of STK-001 up to 45mg were well-tolerated Majority of patients treated with multiple doses of STK-001 experienced a reduction in seizures The effects were more pronounced
Slide 22: Closing Remarks
Closing Remarks Edward M. Kaye, M.D. Chief Executive Officer
Slide 23: Acknowledgements
STOKE Acknowledgements ie EIS of. Esl > Kees eote | a 3-4
Slide 24: Q&A
Q&A © Copyright 2022 stoke Therapeutes | 24