Stoke Therapeutics, Inc. · NASDAQ:STOK
Stoke Therapeutics — 41st Annual J.P. Morgan Healthcare Conference 2023
23 slides from Stoke Therapeutics's conference presentation, Jan 9, 2023
Slide 1: Stoke Therapeutics at 41st Annual J.P. Morgan Healthcare Conference
Exhibit 99.1 Stoke Therapeutics NASDAQ: STOK Edward M. Kaye, M.D. Chief Executive Officer st 41 Annual J.P. Morgan Healthcare Conference January 10, 2023 © Copy © Copy right 20 ri23 ght S20 toke 23 Therape Stoke Therape utics utic1 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: Our Goal: Upregulate protein expression to treat underlying cause of severe genetic diseases
OUR GOAL: Upregulate protein expression to treat the underlying cause of severe genetic diseases Stoke’s pipeline offers potential first-in-class disease modifying new medicines for diseases caused by protein insufficiency STK-001 for STK-002 for Autosomal Rett syndrome, And beyond… Dravet syndrome
Slide 4: Advantages of Stoke's Approach vs. Other Genetic Approaches
Advantages of Stoke’s Approach vs. Other Genetic Approaches Selectively boosts expression No observed unwanted Utility across small and large only in tissues where the off-target genetic effects gene targets and mutations protein is normally expressed Does not Ability to control dose level Simple an
Slide 5: Nonsense mediated decay (NMD) with and without TANGO-ASO
Nonsense mediated Nonsense mediated decay (NMD) exon decay (NMD) exon without TANGO-ASO with TANGO-ASO = 50% Increased functional functional protein expression protein © Copyright 2023 Stoke Therapeutics 5 5 NMD: nonsense-mediated decay
Slide 6: Dravet Syndrome: A Severe, Progressive Genetic Epilepsy
Dravet Syndrome: A Severe, Progressive Genetic Epilepsy 1 out of 16,000 ~35,000 85% babies are born with Dravet syndrome of cases caused by a people affected in the U.S., Canada, HAPLOINSUFFICIENCY Japan, Germany, France and the UK of the SCN1A gene of children and adolescents with Dravet Up to synd
Slide 7: Current Treatment Paradigm is Burdensome and Ineffective
Current Treatment Paradigm is Burdensome and Ineffective Most patients end up on 3 or more anti-seizure medicines (ASM) Clinician perspectives on current treatment options Dravet Syndrome Treatment “Eliminating seizures is not possible. We strive for balance Diagnosis between seizure frequency, dura
Slide 8: Our Goal: Transform the Treatment of Dravet Syndrome by Targeting the Underlying Cause
Our Goal: Transform the Treatment of Dravet Syndrome by Targeting the Underlying Cause of the Disease, Not Just the Seizures Multiple medicines available for No medicines available for Seizure management Syndrome management Available medicines used to control seizures: STK-001 • Acetazolamide• Felba
Slide 9: Voice of the Patient
Our entire life has been impacted by this diagnosis. Our family has been disrupted. Our livelihood has been impacted. “ ” Our future is unknown, and the unknown can be so consuming. – Jennifer MK., Mom of Daughter with Dravet syndrome Voice of the Patient Report Published by the Dravet Syndrome Foun
Slide 10: Dravet Syndrome is More Than "Just Seizures"
Dravet Syndrome is More Than “Just Seizures” Movement & Balance Intellectual Disability & Developmental Delays “We're disappointed when [our son’s] physical activity is limited and the short “Over time, we have seen slow, steady walk or visit that we plan with his decline in all areas, from speech,
Slide 11: Potential disease-modifying gene therapies in SCN1A-positive Dravet syndrome
Potential disease-modifying gene therapies in SCN1A-positive Dravet syndrome….include antisense oligonucleotide (STK-001)…are positioned to improve not only seizure control, but by targeting the underlying cause and restoring native “ ” gene expression, could also address the equally important comor
Slide 12: Preclinical Findings Support Disease Modifying Potential of STK-001
Preclinical Findings Support Disease Modifying Potential of STK-001 Significant reductions in premature mortality and seizure frequency in DS mice after a single dose Single dose restores Na 1.1 V p<0.0001 to near-normal levels for ✓ >3 months in DS mice PBS wild-type (n=46) STK-001 wild-type (n=27)
Slide 13: Reductions in Convulsive Seizure Frequency Observed in Patients Treated With STK-001
Reductions in Convulsive Seizure Frequency Observed in Patients Treated With STK-001 On Top of Multiple Anti-Seizure Medicines All Ages Combined by Cohort 0 -15 -20.1% (n=17) -30 -40.6% (n=4) -45 20mg 30mg 45mg -55.2% (n=6) -60 Day 29 to 140 or 168 >50% of patients were taking concomitant fenflurami
Slide 14: Reductions in Convulsive Seizure Frequency Observed Across Age Groups
Reductions in Convulsive Seizure Frequency Observed Across Age Groups Taking Multiple Doses of STK-001 74% Median seizure reduction observed in younger patients 2-12y Combined by Cohort 13 - 18/<18y Combined by Cohort 0 0 -20.1% (n=7) -25 -25 -26.8% -23.4% (n=10) (n=2) -40.6% -39.7% (n=2) (n=4) -50
Slide 15: 67% (4/6) Patients Experienced >50% Reduction in Convulsive Seizure Frequency
67% (4/6) Patients Experienced >50% Reduction in Convulsive Seizure Frequency with Three Doses of STK-001 (45mg) Reductions began after the first dose and continued with additional treatment 50 50 45 mg MAD (n=6,6,6,6,6,4) 45 mg MAD 13-18/<18y 23.8 45 mg MAD 2-12y 0 0 -26.7 -50 -50 -52.8 -57.5 -72.5
Slide 16: Reductions in Seizure Frequency Were Maintained with Ongoing STK-001 Treatment
Reductions in Seizure Frequency Were Maintained with Ongoing STK-001 Treatment Convulsive Seizure Frequency 50 25 0 -25 -50 -75 -100 Day Day Day Day Day Day Weeks Weeks Weeks Weeks Weeks Weeks Weeks Weeks 1-28 29-56 57-84 85-112 113-140 141-168 1-4 5-8 9-12 13-16 1-4 5-8 9-12 13-16 MONARCH Post Dose
Slide 17: Improvements in Non-Seizure Comorbidities Measured by the BRIEF-P
Improvements in Non-Seizure Comorbidities Measured by the BRIEF-P Indicate the Potential for Disease Modification 20 20 No STK-001 All Doses = 30mg of STK-001 10 10 0 0 -10 -10 -20 -20 -30 -30 -40 -40 Week 16 (n=8) Week 32 (n=5) Overall (n=26) ISCI (Inhibit + Emotional Control) FI (Shift + Emotional
Slide 18: Summary of Key Ph1/2a Interim Data
Summary of Key Ph1/2a Interim Data Single and multiple doses of STK-001 up to 45mg ✓ were well-tolerated 55% median reduction in convulsive seizure STK-001 Has Potential to frequency observed in patients treated with three ✓ doses of STK-001 (45mg) Address the Genetic Cause Reductions in seizure fre
Slide 19: Our Pipeline of First-in-Class Disease Modifying Potential Medicines
Our Pipeline of First-in-Class Disease Modifying Potential Medicines PROGRAM TARGET DISCOVERY & PRECLINICAL PHASE 1/2 PHASE 3 PARTNER Central Nervous System 100% Stoke Dravet Syndrome SCN1A STK-001 Global Stoke : Acadia SYNGAP1 SYNGAP1 Syndrome 50:50 Acadia Rett Syndrome MECP2 Worldwide License Acad
Slide 20: 2023 Priorities
2023 Priorities Develop & Expand Advance STK-001 for Advance STK-002 Pipeline Dravet Syndrome to Pivotal for ADOA • 45mg clinical data anticipated in • Submit CTA in the UK in the first • Expand TANGO ASOs as a first-in- mid-2023 half of 2023 to enable Phase 1/2 class disease-modifying approach star
Slide 21: Current Liquidity Anticipated to Fund Operations into 2025
Current Liquidity Anticipated to Fund Operations into 2025 $252.2M 39.4M Cash, Cash Equivalents, Common Shares Outstanding Marketable Securities, and Restricted Cash as of 9/30/2022 as of 9/30/2022 © Copy © Copyri right ght 20 2023 23 S Stoke toke Therape Therapeutic utics s 21 21
Slide 22: © Copyright 2023 Stoke Therapeutics 22
© Copyright 2023 Stoke Therapeutics 22
Slide 23: Q&A
Q&A © Copyright 2023 Stoke Therapeutics 23
Stoke Therapeutics — 41st Annual J.P. Morgan Healthcare Conference 2023 on 6ix