Stoke Therapeutics, Inc. · NASDAQ:STOK
Stoke Therapeutics — European Paediatric Neurology Society Congress 2025
14 slides from Stoke Therapeutics's conference presentation, Jul 10, 2025
Slide 1: Zorevunersen demonstrates potential as a disease-modifying therapy in Dravet syndrome
Zorevunersen demonstrates potential as a disease-modifying therapy in patients with Dravet syndrome through durable seizure reduction and improvements in cognition, behavior, and functioning with up to 24 months of maintenance dosing in open-label extension studies European Paediatric Neurology Soci
Slide 2: Speaker disclosures
Speaker disclosures • Received honoraria for presenting at educational events and advisory boards • Consultancy work for Biocodex, GW / Jazz Pharma, Encoded Therapeutics, Servier, Stoke Therapeutics, and Zogenix/UCB © Copyright 2025 Stoke Therapeutics 2
Slide 3: DS is a severe developmental and epileptic encephalopathy with a burdensome treatment paradigm
DS is a severe developmental and epileptic encephalopathy with a burdensome and challenging treatment paradigm1 DS, Dravet syndrome; SCN1A, sodium channel protein type 1 alpha subunit; SUDEP, sudden unexpected death in epilepsy. 1. Dravet Syndrome Foundation – Voice of the Patient Report. Available
Slide 4: Zorevunersen is an investigational ASO designed to upregulate NaV1.1 expression
4 Zorevunersen is an investigational ASO designed to upregulate NaV1.1 expression by leveraging the wild-type copy of SCN1A The SCN1A gene codes for the NaV1.1 protein Dravet Syndrome Foundation – Voice of the Patient Report. Available at: https://dravetfoundation.org/wp-content/uploads/2022/05/Voic
Slide 5: Mechanism of haploinsufficiency
5 Zorevunersen is an investigational ASO designed to upregulate NaV1.1 expression by leveraging the wild-type copy of SCN1A Mechanism of haploinsufficiency DNA Mutated gene (Loss of function) Productive mutant Non-productive mRNA mutant mRNA Productive mutant mRNA Mutant mRNA degradation by NMD No N
Slide 6: ASO correction of haploinsufficiency
6 Zorevunersen is an investigational ASO designed to upregulate NaV1.1 expression by leveraging the wild-type copy of SCN1A ASO correction of haploinsufficiency DNA Mutated gene (Loss of function) ASO promotes ASO promotes NMD exon NMD exon exclusion exclusion Productive mutant Productive mutant mRN
Slide 7: Safety, pharmacokinetics, and effectiveness evaluated in Phase 1/2a and OLE studies
Safety, pharmacokinetics, and effectiveness of zorevunersen in children and adolescents were evaluated in Phase 1/2a and OLE studies ELIGIBILITY CRITERIA MONARCH SWALLOWTAIL 81 PATIENTS TREATED • 2–18 years of age and ADMIRAL and LONGWING Age at screening, years • Established DS diagnosis Phase 1/2
Slide 8: Loading and maintenance doses were generally well tolerated
Loading doses of up to 70 mg zorevunersen followed by maintenance doses up to 45 mg were generally well tolerated Drug-related TEAES • 30% of patients experienced a study drug–related TEAE Phase 1/2a • Most common — CSF protein elevations (14%) and procedural vomiting (5%) studies TESAEs (n=81) • 22
Slide 9: Durable reductions in convulsive seizure frequency with loading and maintenance doses
Durable reductions in convulsive seizure frequency with loading doses of 70 mg and maintenance doses of 45 mg zorevunersen Reductions in convulsive seizure frequency from Phase 1/2a baseline through OLE studies Phase 1/2a studies (loading doses) OLE studies (maintenance doses) Phase 1/2a data cut: D
Slide 10: Reductions in convulsive seizure frequency maintained through 2 years of OLE studies
Reductions in convulsive seizure frequency were maintained through 2 years of the OLE studies Reductions in convulsive seizure frequency from Phase 1/2a baseline OLE studies (maintenance doses) Phase 1/2a data cut: December 12, 2023 (after End of Study); OLE data cut: June 28, 2024. One patient who
Slide 11: Improvements in cognition and behavior within the first year of treatment
Improvements in cognition and behavior within the first year of treatment in Phase 1/2a and with ongoing treatment in the OLE studies Vineland-3 improvements at Month 9 of the Phase 1/2a study and Months 12 and 24 of the OLEs Phase 1/2a study (ADMIRAL) at 9 months OLE studies at 12 and 24 months Pha
Slide 12: Cognition and behavior improvements contrasted with matched natural history patients
Improvements in cognition and behavior in patients treated with zorevunersen contrasted with matched Dravet syndrome natural history patients Patients treated with zorevunersen Dravet syndrome natural history study patients Phase 1/2a data cut: December 12, 2023 (after End of Study); OLE data cut: J
Slide 13: Improvements in patient QoL observed within first months of treatment
Improvements in patient QoL were observed within the first 9 months of treatment in Phase 1/2a and continued with ongoing treatment in the OLE studies Improvements in QoL as measured with EQ-VAS Phase 1/2a studies at approximately 9 months OLE studies up to 24 months Additionally, measurable improve
Slide 14: Findings support potential of zorevunersen as a durable, disease-modifying therapy
Phase 1/2a and OLE study findings support the potential of zorevunersen to be a durable, disease-modifying therapy for Dravet syndrome • Patients treated with loading doses of 70 mg zorevunersen experienced the most substantial reductions in convulsive seizure frequency within the first 9 months of
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