Slide 1: Analysis of STK-001 for the Treatment of Dravet Syndrome
Analysis of STK-001 for the Treatment of Dravet Syndrome Stoke Therapeutics March 25, 2024 Exhibit 99.2
Slide 2: Agenda
Agenda Introduction Eric Rojas, Head of Investor Relations Introductory Remarks Edward M. Kaye, M.D., Chief Executive Officer Analysis of Phase 1/2a and Open-Label Extension (OLE) Studies of STK-001 Barry Ticho, M.D., Ph.D., Chief Medical Officer Kimberly Parkerson, M.D., Ph.D., Head of Neurology Cl
Slide 3: Forward Looking Statements
Forward Looking Statements This presentation has been prepared by Stoke Therapeutics, Inc. ("Stoke" or "we") for informational purposes only and for no other purpose. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by the presenter or
Slide 4: Introductory Remarks
Introductory Remarks Edward M. Kaye, M.D. Chief Executive Officer
Slide 5: Landmark New Data Support the Potential for STK-001 to Be the First Medicine
Phase 1/2a Study Data: 70mg doses demonstrated substantial & sustained reductions in convulsive seizure frequency of: OLE Studies (30mg, 45mg): Clinically meaningful, durable reductions in seizures and improvements in multiple measures of cognition & behavior over 12 months Reductions in seizures an
Slide 6: Our Approach: Leverage the Wild-Type SCN1A Allele
© Copyright 2024 Stoke Therapeutics Cause of Dravet Syndrome Our Approach Leverage the wild-type SCN1A allele to boost the production of full-length, fully-functional Nav1.1 protein to treat the underlying cause of Dravet syndrome of Dravet cases caused by a HAPLOINSUFFICIENCY of the SCN1A gene 50%
Slide 7: STK-001 on Track to Be the First Disease-Modifying Medicine
STK-001 is on Track to be the First Disease-Modifying Medicine to Treat the Underlying Cause of Dravet Syndrome No medicines currently available for Dravet syndrome management STK-001 The first potential disease-modifying approach to address the genetic cause of Dravet syndrome Available medicines u
Slide 8: Natural History Data: No Meaningful Improvement in Convulsive Seizure Frequency
Natural History Data: Despite Standard Anti-Seizure Medicines, No Meaningful Improvement in Convulsive Seizure Frequency Source: 24-Month Analysis of BUTTERFLY: A Prospective, Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syn
Slide 9: Natural History Data: No Improvement in Cognition and Behavior
Improving Worsening Natural History Data: Despite Best Available Anti-Seizure Medicines, No Improvement in Cognition and Behavior Note: Analysis based on data from BUTTERFLY through Month 24 analyzed with Machine Learning. Vineland-3: The Vineland Adaptive Behavior Scales, Third Edition. GSV = Growt
Slide 10: Analysis of Data from Studies of STK-001
Analysis of Data from Studies of STK-001 Barry Ticho, M.D., Ph.D. Chief Medical Officer
Slide 11: Patients Treated to Date with STK-001
81 Patients Treated to Date with STK-001 Total, n (%) N 81 Age at Screening Mean (SD) 9.9 years (5.05) Number of Concomitant Anti-Seizure Medications ≥3 69 (85%) ≥4 44 (54%) Concomitant Fenfluramine Yes 40 (49%) Baseline Convulsive Seizure Frequency per 28 days (n = 77) Median (min, max) 1
Slide 12: 70mg Doses Demonstrated the Most Substantial Reductions in Seizure Frequency
70mg Doses of STK-001 Demonstrated the Most Substantial Reductions in Seizure Frequency on Top of Standard of Care Medicines 1 Dose 2 Dose 3 Dose Indicates dosing schedule for 70mg cohorts only
Slide 13: Substantial Reductions in Seizure Frequency Sustained at 6 Months
Substantial Reductions in Seizure Frequency at 3 and Sustained at 6 Months after Last Dose with 1, 2 or 3 Doses of STK-001 (70mg) 3 Months after last dose 70mg 1 Dose 70mg 2 or 3 Dose 6 Months after last dose 3 Months after last dose 6 Months after last dose -43% -57% -85% -74% 1 dose data is from t
Slide 14: ~80% of Patients Experienced >50% Reduction in Seizures
~80% of Patients Treated with 2 or 3 Doses of STK-001 (70mg) Experienced >50% Reduction in Seizures A 50% responder rate is an important measure of efficacy Note: 3 months after last dose refers to D113 to D140 (2 dose MAD) and D141 to D168 (3 dose MAD) and 6 months after last dose refers to D197 to
Slide 15: Phase 1/2a Data Support a Potential 70mg Loading Dose Regimen
Phase 1/2a Data Support a Potential 70mg Loading Dose Regimen in a Registrational Study 85% at 3 months and 74% at 6 months post last dose ~80% of patients experienced >50% reduction in convulsive seizure frequency Data cutoff dates: Phase 1/2a Studies 12DEC2023; OLE Studies 01NOV2023 6 Month Follow
Slide 16: Durable Reductions in Seizure Frequency in OLE Studies
Durable Reductions in Seizure Frequency Observed with Continued Treatment with STK-001 in OLE Studies *End of Study = 24 Weeks After Last Dose in Phase 1/2 Study Weeks 1-4 Weeks 5-8 Weeks 9-12 Weeks 13-16 Weeks 17-20 Weeks 21-24 Weeks 25-28 Weeks 29-32 Weeks 33-36 Weeks 37-40 Weeks 41-44 Weeks 45-48
Slide 17: Analysis of Safety, Cognition and Behavior from Studies of STK-001
Analysis of Safety, Cognition and Behavior from Studies of STK-001 Kimberly Parkerson, M.D., Ph.D. Head of Neurology Clinical Development
Slide 18: Clinically Meaningful Improvements in Cognition and Behavior Over 12 Months
Improving Worsening Clinically Meaningful Improvements in Cognition and Behavior Over 12 Months with Continued Treatment with STK-001 (30mg, 45mg) Note: Analysis based on a mixed-effects model for repeated measures (MMRM). *Fine motor did not meet the threshold of clinically meaningful change. Vinel
Slide 19: Clinically Meaningful Improvements in Overall Condition Over 12 Months
Clinically Meaningful Improvements in Overall Condition Over 12 Months with Continued Treatment with STK-001 (30mg, 45mg) Consistency across clinician and caregiver assessments of improvements observed in the OLEs Note: Analysis based on a mixed-effects model for repeated measures (MMRM). Data from
Slide 20: OLE Data Support a Potential 45mg Maintenance Dosing Regimen
OLE Data Support a Potential 45mg Maintenance Dosing Regimen in a Registrational Study At the time of this analysis, 28 patients had reached one year of treatment in the OLE’s Improvements are in contrast to 2-year natural history study data that show widening gaps in cognition and behavior compared
Slide 21: Single & Multiple Doses Up To 70mg Were Generally Well-Tolerated
Single & Multiple Doses Up To 70mg Were Generally Well-Tolerated No new safety findings related to study drug Phase 1/2a Studies (n=81) OLE Studies (n=68) 30% had a TEAE related to study drug. CSF protein elevations and procedural vomiting were the most common 22% had a TESAE. These events were asse
Slide 22: Closing Remarks
Closing Remarks Edward M. Kaye, M.D. Chief Executive Officer
Slide 23: Landmark New Data Support the Potential for STK-001
Landmark New Data Support the Potential for STK-001 to be the First Medicine to Treat the Underlying Cause of Dravet Syndrome Phase 1/2a (2 and 3 doses of 70mg): Substantial & sustained reductions in seizure frequency Open-Label Extensions (30mg, 45mg): Durable reductions in seizures with dosing eve
Slide 24: Therapeutics
THERAPEUTICS
Slide 25: Stoke
STOKE THE RAG EU Tilve Ss Copyright Stoke Therapeutics, Inc. Not for publication or distribution