Dyne Therapeutics, Inc. · NASDAQ:DYN
Dyne Therapeutics — Technical Presentation (September 2024)
26 slides from Dyne Therapeutics's technical presentation, Sep 3, 2024
Slide 1: Achieving the Promise of FORCE to Deliver for Patients
Exhibit 99.2 Achieving the Promise of FORCE to Deliver for Patients F O R C E DELIVER CLINICAL UPDATE | SEPTEMBER 3, 2024
Slide 2: Forward-Looking Statements & Disclaimer
Forward-Looking Statements & Disclaimer This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding Dyne’s strategy, future operations
Slide 3: Program
Program Opening remarks John Cox, President & CEO DYNE-251 DELIVER Trial in DMD Data Wildon Farwell, M.D., MPH, Chief Medical Officer Closing Remarks John Cox, President & CEO Q&A 3
Slide 4: Our Mission
OUR MISSION Life-transforming therapies for patients with serious muscle diseases 4
Slide 5: Committed to Building the World's Leading Muscle Disease Company
Committed to Building the World’s Leading Muscle Disease Company • Potential to transform the treatment paradigm for people living with DMD DELIVER • Best-in-class dystrophin resulting in unprecedented improvements in multiple functional CLINICAL 1 outcomes, including NSAA and SV95C, in multiple coh
Slide 6: Program
Program Opening remarks John Cox, President & CEO DYNE-251 DELIVER Trial in DMD Data Wildon Farwell, M.D., MPH, Chief Medical Officer Closing Remarks John Cox, President & CEO Q&A 6
Slide 7: Building a Global DMD Franchise of Transformative Therapies
Building a Global DMD Franchise of Transformative Therapies Clinical Presentation Population Overview • Mutation in the DMD gene that • Muscle weakness • ~12,000 - 15,000 (US) encodes for dystrophin • Progressive loss of function • ~ 25,000 (Europe) • Onset in first few years of life • Loss of ambul
Slide 8: Phase 1/2 Clinical Trial to Evaluate DYNE-251 in Patients with DMD
Phase 1/2 Clinical Trial to Evaluate DYNE-251 in Patients with DMD Additional Endpoints Population Primary Endpoints Stages of DELIVER • Male patients with DMD • Safety and tolerability • Pharmacokinetics • Multiple Ascending Dose with mutations amenable (MAD): 24 weeks • Change from baseline in • C
Slide 9: DELIVER Trial Design
DELIVER Trial Design Global, Randomized, Placebo-Controlled Stage Evaluating Administration of DYNE-251 in Ambulant and Non-Ambulant Male DMD Patients with Mutations Amenable to Exon 51 Skipping Therapy MAD Study Details Registrational Registrational Cohorts Initiating Dose: TBD (≥20 mg/kg, Q4W or Q
Slide 10: DELIVER Baseline Participant Characteristics: By Cohort
DELIVER Baseline Participant Characteristics: By Cohort mean (SD) or n(%) 0.7 mg/kg 1.4 mg/kg 2.8 mg/kg 5 mg/kg 10 mg/kg 20 mg/kg (N=6) (N=6) (N=6) (N=6) (N=8) (N=8) Age (years) 10.8 (2.2) 7.8 (3.3) 10.7 (2.9) 8.3 (2.8) 6.6 (2.2) 8.1 (2.4) 2 BMI (kg/m ) 19.5 (3.4) 18.6 (2.3) 22.6 (6.3) 20.9 (1.6) 18
Slide 11: DYNE-251 Safety Profile Is Favorable to Date
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function DYNE-251 Safety Profile Is Favorable to Date 1 Summary of Treatment Emergent Adverse Events (TEAEs) Most TEAEs Were Mild or Moderate in Intensity • 3 serious TEAEs potentially related to study drug in two participants Participants wit
Slide 12: DYNE-251 Drove Dose Dependent Delivery of PMO to Muscle
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function DYNE-251 Drove Dose Dependent Delivery of PMO to Muscle PMO Muscle Concentration 4,500 3,527 3,000 2,158 1,500 657 0 Baseline 6 Months Baseline 6 Months Baseline 6 Months Baseline 6 Months n=7 n=8 n=4 n=4 n=4 n=5 n=6 n=6 Placebo DYNE-
Slide 13: DYNE-251 Demonstrated Dose-Dependent Exon Skipping
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function DYNE-251 Demonstrated Dose-Dependent Exon Skipping Exon 51 Skipping 5 4 3.27 3 2 1.96 1 0.90 0.1 0.04 0.10 0.07 0.10 0 Baseline 6 Months Baseline 6 Months Baseline 6 Months Baseline 6 Months n=8 n=8 n=4 n=4 n=5 n=6 n=6 n=6 Placebo DYN
Slide 14: Higher Doses of DYNE-251 Continued to Drive Robust Dystrophin Expression
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function Higher Doses of DYNE-251 Continued to Drive Robust Dystrophin Expression DYNE-251 Showed 3.7% Unadjusted Dystrophin at 6 Months Unadjusted Dystrophin 3.15 5 2.97 4 3.71 3 3.22 2 0.28 1 0.88 0.25 0.60 0.57 0.52 0.48 0 Baseline 6 Months
Slide 15: DYNE-251 Positioned as Potentially Best-in-Class Next Generation Exon Skipper
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function DYNE-251 Positioned as a Potentially Best-in-Class Next Generation Exon Skipper, Achieving 8.7% Muscle Content Adjusted Dystrophin at 6 Months Muscle Content Adjusted Dystrophin 12 7.37 6.94 Muscle-content Dystrophin (MHC normalized)
Slide 16: Placebo Response is Consistent with Duchenne Natural History
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function 1 Placebo Response is Consistent with Duchenne Natural History DMD Patients Worsen Across a Number of Functional Measurements Over Time th NSAA Total Score Stride Velocity 95 Centile 3.5 0.4 2.0 0.2 0.5 -1.0 -0.1 -2.5 -4.0 -0.3 Baseli
Slide 17: Data from 10 mg/kg Cohort Highlights Extension of Effect
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function Data from 10 mg/kg Cohort Highlights Extension of Effect from 6 Months to 1 Year th NSAA Total Score Stride Velocity 95 Centile 3.5 0.4 2.0 0.2 0.5 -1.0 -0.1 -2.5 -4.0 -0.3 Baseline Day 85 Day 169 Day 337 Baseline Day 85 Day 169 Day 3
Slide 18: Improvements Across Multiple Functional Endpoints in Multiple Cohorts
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function Improvements Across Multiple Functional Endpoints in Multiple Cohorts Baseline Values Inform Interpretation of Data; Ongoing Exploration of Longer Timepoints th NSAA Total Score Stride Velocity 95 Centile 3.5 0.4 2.0 0.2 0.5 -1.0 -0.1
Slide 19: DYNE-251 Drove Clinically Meaningful Improvements in Stride Velocity 95 Centile
Safety Muscle Delivery Exon 51 Skipping Dystrophin by WB Function th DYNE-251 Drove Clinically Meaningful Improvements in Stride Velocity 95 Centile SV95C is a Qualified Primary Endpoint for Duchenne Trials in Europe and Leveraged Across Global Trials th Stride Velocity 95 Centile (SV95C) 0.4 • SV95
Slide 20: Opportunity to Build a Global DMD Franchise
Opportunity to Build a Global DMD Franchise: Leading with DYNE-251, Payloads Identified for Exons 53, 45, 44 Exon 51 – 13% Exon 53 – 8% Approximately Exon 45 – 8% Exon 44 – 6% Exon 50 – 4% 80% of patients Exon 52 – 4% Exon 43 – 4% have genotypes amenable Exon 55 – 2% to exon skipping Exon 8 – 2% Oth
Slide 21: Advancing DYNE-251 Towards Potentially Registrational Data Set
Advancing DYNE-251 Towards Potentially Registrational Data Set Unprecedented level of dystrophin generated, with 3.7% ✓ unadjusted and 8.7% muscle content adjusted dystrophin Improvements in multiple functional outcomes, including SV95C, an approvable endpoint in Europe, in multiple ✓ cohorts Favora
Slide 22: Program
Program Opening remarks John Cox, President & CEO DYNE-251 DELIVER Trial in DMD Data Wildon Farwell, M.D., MPH, Chief Medical Officer Closing Remarks John Cox, President & CEO Q&A 22
Slide 23: Driving Towards Potentially Transformative DM1 and DMD Therapies
Driving Towards Potentially Transformative DM1 and DMD Therapies Delivered on the Promise of FORCE: Enhanced Delivery of Therapeutics to Muscle Compelling Impact on Key Disease Biomarkers and Improvements in Multiple Functional Endpoints in Both DM1 and DMD Favorable Safety & Tolerability Profile Fu
Slide 24: Strengthening the Team to Deliver on Dyne's Next Chapter
Strengthening the Team to Deliver on Dyne’s Next Chapter Doug Kerr Johanna Friedl-Naderer Lucia Celona Chief Medical Officer Chief Commercial Officer Chief Human Resources Officer Proven Team of Biopharma Executives Prepared to Advance Multiple Programs to Market 24
Slide 25: Building the World's Leading Muscle Disease Company
Building the World’s Leading Muscle Disease Company Dynamo Culture Own Muscle Delivery & Win in DM1, DMD, FSHD Leverage FORCE 25
Slide 26: Achieving the Promise of FORCE to Deliver for Patients
Achieving the Promise of FORCE to Deliver for Patients F O R C E DELIVER CLINICAL UPDATE | SEPTEMBER 3, 2024
Dyne Therapeutics — Technical Presentation (September 2024) on 6ix