Dyne Therapeutics, Inc. · NASDAQ:DYN
Dyne Therapeutics — Corporate Update (December 2025)
34 slides from Dyne Therapeutics's corporate update, Dec 8, 2025
Slide 1: Positive Topline Results from Phase 1/2 DELIVER Trial of Zeleciment Rostudirsen (DYNE-251) in DMD
Exhibit 99.2 Positive Topline Results from Phase 1/2 DELIVER Trial of Zeleciment Rostudirsen (DYNE-251) in DMD to Support Potential U.S. Accelerated Approval Ravi, living with DMD DECEMBER 8, 2025
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 Positive Topline Results from DELIVER Trial of Z-Rostudirsen Supporting Potential for U.S. Accelerated Approval Doug Kerr, M.D., Ph.D., Chief Medical Officer Closing Remarks John Cox, President & CEO Available for Q&A Erick Lucera, Chief Financial Of
Slide 4: Transforming Exon Skip Amenable DMD; Validating the FORCE Platform and Dyne Pipeline
Transforming Exon 51 Skip Amenable DMD; Validating the FORCE Platform and Dyne Pipeline Data to Support Potential Sustained Functional Validation of TM Accelerated Approval Improvement FORCE Platform • Positive results from Registrational • Sustained functional improvement • Data show that FORCE pla
Slide 5: Program
Program Opening Remarks John Cox, President & CEO Positive Topline Results from DELIVER Trial of Z-Rostudirsen Supporting Potential for U.S. Accelerated Approval Doug Kerr, M.D., Ph.D., Chief Medical Officer Closing Remarks John Cox, President & CEO Available for Q&A Erick Lucera, Chief Financial Of
Slide 6: DELIVER Clinical Update Agenda
DELIVER Clinical Update Agenda Unmet Need in DMD and Potential for Z-Rostudirsen Positive Topline Results from Registrational Expansion Cohort (REC) of DELIVER Trial to Support Planned Submission for U.S. Accelerated Approval New Positive Long-Term Results from DELIVER Trial Showed Sustained Functio
Slide 7: Exon Skip Amenable DMD: A More Severe Population with Significant Unmet Need
Exon 51 Skip Amenable DMD: A More Severe Population with Significant Unmet Need, Despite Approved Therapies Progressive Clinical Exon 51 Skip Amenable DMD – Limitations with Presentation Challenging Form of Duchenne Current Therapies • Muscle weakness and gait • Greatly reduced or absent dystrophin
Slide 8: Z-Rostudirsen Leverages FORCE Platform for Targeted Delivery to Tissues That Matter in DMD
Z-Rostudirsen Leverages FORCE Platform for Targeted Delivery to Tissues That Matter in DMD ANTIBODY Skeletal (including Proprietary Fab against TfR1 to enable targeted delivery to diaphragm) and smooth muscle and CNS muscle Cardiac muscle LINKER Connects Fab and payload PAYLOAD PMO designed to enabl
Slide 9: DELIVER Clinical Update Agenda
DELIVER Clinical Update Agenda Unmet Need in DMD and Potential for Z-Rostudirsen Positive Topline Results from Registrational Expansion Cohort (REC) of DELIVER Trial to Support Planned Submission for U.S. Accelerated Approval New Positive Long-Term Results from DELIVER Trial Showed Sustained Functio
Slide 10: DELIVER Trial Designed to Support Potential U.S. Accelerated Approval
DELIVER Trial Designed to Support Potential U.S. Accelerated Approval Trial Design Focused on U.S. Accelerated Approval Endpoints • Broad, representative patient population with DMD mutations amenable to Primary endpoints exon 51 skipping • Change from baseline in dystrophin protein levels by Wester
Slide 11: DELIVER Baseline Participant Characteristics and Function Generally Well Balanced
DELIVER Baseline Participant Characteristics and Function Generally Well Balanced Across Treated and Pooled Placebo Cohorts Z-rostudirsen 6 Placebo 20 mg/kg Q4W 10 à 20 mg/kg 20 mg/kg Q4W Mean (SD) or n (%) (MAD+REC) (REC) Q4W (MAD) (MAD) 5 N=24 N=24 N=6 N=6 Age (years) 8.2 (2.5) 7.8 (3.6) 6.8 (2.5)
Slide 12: Safety Profile of Z-Rostudirsen 20 mg/kg Q4W Remains Favorable
Safety Profile of Z-Rostudirsen 20 mg/kg Q4W Remains Favorable 1 Summary of treatment-emergent adverse events (TEAEs) Most related TEAEs were mild or moderate Placebo-Controlled (PC) Period All Study Periods Study Period (0 to 6M) (0 to ≤36M) Potentially related serious TEAEs Z-rostudirsen Z-rostudi
Slide 13: Z-Rostudirsen Achieved Statistically Significant and Robust Increase in Dystrophin at 6 Months
Z-Rostudirsen Achieved Statistically Significant and Robust Increase in Dystrophin Expression at 6 Months in REC 3 Muscle content-adjusted dystrophin (REC) p<0.0001 6 5.46 4 2 0.92 0.95 0.83 0 Baseline 6 Months Baseline 6 Months 2 n=8 n=7 n=24 n=24 Placebo Z-rostudirsen 20 mg/kg Q4W (REC) 1. Biopsie
Slide 14: REC Replicated Same 7-Fold Increase in Dystrophin Observed in MAD Cohort
REC Replicated Same 7-Fold Increase in Dystrophin Originally Observed in 20 mg/kg Q4W MAD Cohort 3 Muscle content-adjusted dystrophin (REC) p<0.0001 6 Previously presented muscle content-adjusted dystrophin from MAD 5.46 4 7-fold change from 4 7-fold 4 baseline 2 0.92 0.95 0.83 0 Baseline 6 Months B
Slide 15: REC Exceeded Fold Increase in Unadjusted Dystrophin Observed in MAD Cohort
REC Exceeded Fold Increase in Unadjusted Dystrophin Observed in 20 mg/kg Q4W MAD Cohort Unadjusted dystrophin (REC) 4 3 p<0.0001 Previously presented unadjusted dystrophin from MAD 3 2.87 2 7-fold increase from 4 6-fold 4 baseline 1 0.45 0.44 0.43 0 Baseline 6 Months Baseline 6 Months 2 n=8 n=7 n=24
Slide 16: Z-Rostudirsen Achieved ~10-Fold Higher Unadjusted Dystrophin Relative to Phase 3 Trial of SoC
Z-Rostudirsen Achieved ~10-Fold Higher Level of Unadjusted Dystrophin Relative to Level Reported in Phase 3 Trial of SoC Unadjusted dystrophin (REC) 4 3 p<0.0001 3 2.87 2 p<0.05 1 0.30 0.24 0.44 0.45 0.43 0 Baseline 6 Months Baseline 6 Months Baseline 6 Months 2 n=8 n=7 n=24 n=24 n=77 n=16 4 Placebo
Slide 17: Clinically Validated Prespecified Outcome Measures Used to Assess Functional Improvement
Clinically Validated Prespecified Outcome Measures Used to Assess Functional Improvement TTR Velocity SV95C 1 Stride Velocity 95th Centile Time to Rise Velocity Speed of rising from floor 5% fastest strides taken during 3,4 everyday living 10MWR Velocity PUL2.0 10-Meter Walk/Run Velocity Performance
Slide 18: Improvement in TTR Velocity at 6 Months Exceeded MCID Relative to Placebo
Improvement in TTR Velocity at 6 Months Exceeded MCID Relative 3 to Placebo with Nominal p<0.05 1 Time to Rise (TTR) Velocity 0.04 0.02 ∆0.04 rise/sec 0.00 2 exceeds MCID 3 (nominal p<0.05) -0.02 -0.04 BL M6 Placebo (REC+MAD) (n=18) Z-rostudirsen 20 mg/kg Q4W (REC) (n=21) 1. Ambulant participants; o
Slide 19: Improvement in 10MWR Velocity at 6 Months Relative to Baseline and Placebo
Improvement in 10MWR Velocity at 6 Months Relative to Baseline 3 and Placebo with Nominal p<0.05 10-Meter Walk/Run (10MWR) 1 Velocity 0.2 0.1 ∆0.2 m/sec 0.0 2 approaches MCID 3 (nominal p<0.05) -0.1 -0.2 BL M6 Placebo (REC+MAD) (n=18) Z-rostudirsen 20 mg/kg Q4W (REC) (n=21) 1. Ambulant participants;
Slide 20: Improvement in NSAA at 6 Months Relative to Baseline and Placebo
Improvement in NSAA at 6 Months Relative to Baseline and Placebo North Star Ambulatory Assessment 1 (NSAA) 2.5 1.5 ∆2.1 points 2 approaches MCID 0.5 3 (nominal p=0.0894) -0.5 -1.5 BL M6 Placebo (REC+MAD) (n=18) Z-rostudirsen 20 mg/kg Q4W (REC) (n=21) 1. Ambulant participants; missing values imputed.
Slide 21: Improvement in SV95C at 6 Months Relative to Baseline
Improvement in SV95C at 6 Months Relative to Baseline th Stride Velocity 95 Centile 1 (SV95C) 0.14 0.12 0.10 0.08 0.06 0.04 0.02 0.00 -0.02 BL M6 2 Placebo (REC+MAD) (n=12) Z-rostudirsen 20 mg/kg Q4W (REC) (n=20) 1. Ambulant participants; missing values imputed. 2. Placebo impacted by single partici
Slide 22: Improvement in PUL2.0 at 6 Months in Ambulant and Non-Ambulant Participants
Improvement in PUL2.0 at 6 Months in Ambulant and Non-Ambulant Participants Relative to Baseline and Placebo Performance Upper Limb v2.0 1 (PUL2.0) 1.0 0.5 0.0 -0.5 BL M6 Placebo (REC+MAD) (n=23) Z-rostudirsen 20 mg/kg Q4W (REC) (n=22) 1. Ambulant and non-ambulant participants; missing values impute
Slide 23: Preservation of Lung Function at 6 Months
Preservation of Lung Function at 6 Months Forced Vital Capacity Percent Predicted 1 (FVC%p) 6.0 4.0 2.0 0.0 -2.0 -4.0 -6.0 -8.0 -10.0 -12.0 BL M6 Placebo (REC+MAD) (n=20) Z-rostudirsen 20 mg/kg Q4W (REC) (n=15) 1. Ambulant and non-ambulant participants. BL, baseline; M6 = 169 days; M, month; sec, se
Slide 24: Z-Rostudirsen Offers a Compelling Profile for Potential Accelerated Approval
Z-Rostudirsen Offers a Compelling Profile for Potential Accelerated Approval and Addressing Unmet Need in DMD Z-Rostudirsen for Exon 51 DMD Statistically Significant and Robust Increase in Dystrophin at 6 Months 1 Favorable Safety & Tolerability Profile up to 36 Months Functional Improvement Observe
Slide 25: DELIVER Clinical Update Agenda
DELIVER Clinical Update Agenda Unmet Need in DMD and Potential for Z-Rostudirsen Positive Topline Results from Registrational Expansion Cohort (REC) of DELIVER Trial to Support Planned Submission for U.S. Accelerated Approval New Positive Long-Term Results from DELIVER Trial Showed Sustained Functio
Slide 26: Sustained Functional Improvement Compared to Baseline Across All 6 Measures up to 24 Months
Sustained Functional Improvement Compared to Baseline Across All 6 Measures up to 24 Months in a Broad Participant Population 1 1 1 18-Month CFB 18-Month CFB 24-Month CFB 2 2 20 mg/kg Q4W Pooled 10 à 20 mg/kg Q4W 10 à 20 mg/kg Q4W MAD Cohort and 20 mg/kg Q4W MAD Cohorts MAD Cohort Improvement Improv
Slide 27: DELIVER Clinical Update Agenda
DELIVER Clinical Update Agenda Unmet Need in DMD and Potential for Z-Rostudirsen Positive Topline Results from Registrational Expansion Cohort (REC) of DELIVER Trial to Support Planned Submission for U.S. Accelerated Approval New Positive Long-Term Results from DELIVER Trial Showed Sustained Functio
Slide 28: Z-Rostudirsen Data Validate Potential of the FORCE Platform
Z-Rostudirsen Data Validate Potential of the FORCE Platform FORCE Design Principles of the FORCE Platform Validation with Z-Rostudirsen DELIVER Data TfR1-mediated delivery to muscle, heart and CNS with Statistically significant and robust increase in rationally selected payload to match disease biol
Slide 29: Advancing Robust Portfolio Focused on Neuromuscular Diseases
Advancing Robust Portfolio Focused on Neuromuscular Diseases DISEASE TARGET PRECLINICAL PHASE 1/2 ESTIMATED PATIENTS Myotonic dystrophy US: ~40,000 DMPK zeleciment basivarsen (z-basivarsen, also known as DYNE-101) type 1 (DM1) EU: ~55,000 zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251
Slide 30: Program
Program Opening Remarks John Cox, President & CEO Positive Topline Results from DELIVER Trial of Z-Rostudirsen Supporting Potential for U.S. Accelerated Approval Doug Kerr, M.D., Ph.D., Chief Medical Officer Closing Remarks John Cox, President & CEO Available for Q&A Erick Lucera, Chief Financial Of
Slide 31: Executing on a Compelling Commercial Opportunity
Executing on a Compelling Commercial Opportunity Strong Value Proposition to Capital Efficient Address Unmet Need in DMD Operating Model Z-Rostudirsen for Exon 51 DMD • Leadership team in place with rare Statistically Significant and Robust neuromuscular disease expertise Increase in Dystrophin at 6
Slide 32: Transforming Exon Skip Amenable DMD; Validating the FORCE Platform and Dyne Pipeline
Transforming Exon 51 Skip Amenable DMD; Validating the FORCE Platform and Dyne Pipeline Data to Support Potential Sustained Functional Validation of TM Accelerated Approval Improvement FORCE Platform • Positive results from Registrational • Sustained functional improvement • Data show that FORCE pla
Slide 33: Transforming Dyne into a Commercial Organization as Early as 2027
Transforming Dyne into a Commercial Organization as Early as 2027 Z-rostudirsen for Exon 51 DMD Z-basivarsen for DM1 Completed enrollment of Early Complete enrollment planned for Q1 2025 Q2 2026 Registrational Expansion Cohort Registrational Expansion Cohort Positive topline results from Data planne
Slide 34: Thank You to all who participated
THANK YOU to all who participated