Dyne Therapeutics, Inc. · NASDAQ:DYN
Dyne Therapeutics — Corporate Update (June 2025)
29 slides from Dyne Therapeutics's corporate update, Jun 17, 2025
Slide 1: Advancing Functional Improvement in DM1: DYNE-101 U.S. Accelerated Approval and Clinical Update
Advancing Functional Improvement in DM1 DYNE-101 U.S. Accelerated Approval and Clinical Update JUNE 17, 2025 Exhibit 99.2
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 Closing Remarks John Cox, President & CEO Updated Plan for U.S. Accelerated Approval and New Clinical Data for DYNE-101 Doug Kerr, M.D., Ph.D., Chief Medical Officer Opening Remarks John Cox, President & CEO Available for Q&A Erick Lucera, Chief Financial Officer
Slide 4: Today's Update
Today’s Update 1. Safety data as of April 23, 2025; vHOT = video hand opening time; MAD = multiple ascending dose; LTE = long-term extension. Breakthrough Therapy Designation Accelerated Approval Pathway with vHOT New Long-Term Data from ACHIEVE Trial FDA granted Breakthrough Therapy Designation for
Slide 5: Program
Program Closing Remarks John Cox, President & CEO Updated Plan for U.S. Accelerated Approval and New Clinical Data for DYNE-101 Doug Kerr, M.D., Ph.D., Chief Medical Officer Opening Remarks John Cox, President & CEO Available for Q&A Erick Lucera, Chief Financial Officer
Slide 6: DM1 is a Devastating Neuromuscular Splicing Disorder
Restore normal RNA splicing to achieve functional improvement for those living with DM1 Functional Improvement via Splicing Correction in Nucleus OUR APPROACH Mutation in the DMPK gene leads to mis-splicing of multiple genes Onset at any point, depending on DM1 phenotype Life expectancy of 45 - 60 y
Slide 7: DYNE-101 Addressing the Central Pathobiology of DM1
DYNE-101 Addressing the Central Pathobiology of DM1 to Enable Broad Functional Improvement1 Muscle Strength: Quantitative Muscle Testing Functional Assessments: 10-Meter Walk / Run; 5 Times Sit to Stand Patient Reported Outcomes: Myotonic Dystrophy Health Index (MDHI) Robust and widespread delivery
Slide 8: Revised Registrational Expansion Cohort to Support Potential U.S. Accelerated Approval
Revised Registrational Expansion Cohort to Support Potential U.S. Accelerated Approval ACHIEVE MAD cohorts complete; all cohorts now at 6.8 mg/kg Q8W in long-term / open label extension ACHIEVE Registrational Expansion Cohort currently enrolling Primary endpoint: Change from baseline in middle finge
Slide 9: New Data Support the Potential for Accelerated Approval of DYNE-101
New Data Support the Potential for Accelerated Approval of DYNE-101 with vHOT as Primary Endpoint Intermediate clinical endpoint DYNE-101 Outcomes DYNE-101 Safety Data A clinical measure with the potential to detect a drug effect earlier than other clinically meaningful endpoints and which is consid
Slide 10: New Data Support the Potential for Accelerated Approval of DYNE-101
New Data Support the Potential for Accelerated Approval of DYNE-101 with vHOT as Primary Endpoint Intermediate clinical endpoint DYNE-101 Outcomes DYNE-101 Safety Data Data support improvement in vHOT as early indicator of clinical benefit with DYNE-101 Sustained improvements increase confidence in
Slide 11: Data Support vHOT Improvement as an Early Indicator of Clinical Benefit
Data Support vHOT Improvement as an Early Indicator of Clinical Benefit with DYNE-101 vHOT Change from Baseline at 6 Months Placebo Month 6 (N=14) 6.8 mg/kg Q8W Month 12 (N=6) Improvement With DYNE-101, all participants at highest 3 doses who improved in 6M vHOT improved in 12M strength, 5xSTS, and/
Slide 12: New Data Support the Potential for Accelerated Approval of DYNE-101
A clinical measure with the potential to detect a drug effect earlier than other clinically meaningful endpoints and which is considered reasonably likely to predict clinical benefit New Data Support the Potential for Accelerated Approval of DYNE-101 with vHOT as Primary Endpoint Intermediate clinic
Slide 13: Robust and Sustained vHOT Improvement
Robust and Sustained vHOT Improvement at 6 and 12 Months vHOT Middle Finger Improvement 1. vHOT Middle Finger (sec) is the average of all myotonia trials for an individual participant in ACHIEVE; vHOT = video hand opening time; SEM = standard error of the mean; BL = baseline. 3 months = 85 days; 6 m
Slide 14: Strength Continues to Improve from Month to Month
Strength Continues to Improve from Month 6 to Month 12 Quantitative Muscle Testing (QMT) Total Score Improvement SEM = standard error of the mean; BL = baseline; %p = percent predicted; 3 months = 85 days; 6 months = 169 days; 12 months = 337 days.
Slide 15: Robust Benefit Across Multiple Timed Function Tests
Robust Benefit Across Multiple Timed Function Tests Sustained at 6 and 12 Months 5 Times Sit to Stand Test Improvement SEM = standard error of the mean; BL = baseline; 3 months = 85 days; 6 months = 169 days; 12 months = 337 days. 10-Meter Walk/Run Test
Slide 16: MDHI Total Score Improvement
Myotonic Dystrophy Health Index (MDHI) Total Score Improvement Deep Improvement in Patient Reported Outcome Sustained at 6 and 12 Months SEM = standard error of the mean; BL = baseline; 3 months = 85 days; 6 months = 169 days; 12 months = 337 days.
Slide 17: Sustained Improvement in CNS-Related MDHI Subscales
Cognitive Impairment Communication Emotional Issues Sleep Pain Fatigue Improvement Subscale Change from Baseline (Mean +/- SEM) SEM = standard error of the mean; BL = baseline; Patient-reported outcomes (PRO) including Myotonic Dystrophy Health Index (MDHI) collected at baseline, 6 months (169 days)
Slide 18: New Data Support the Potential for Accelerated Approval of DYNE-101
A clinical measure with the potential to detect a drug effect earlier than other clinically meaningful endpoints and which is considered reasonably likely to predict clinical benefit New Data Support the Potential for Accelerated Approval of DYNE-101 with vHOT as Primary Endpoint Intermediate clinic
Slide 19: Most TEAEs Were Mild or Moderate in Intensity
TEAE Category Participants with ≥1 TEAE – n (%) 1.8 mg/kg Q4W+Rec. N=16 3.4 mg/kg Q4W+Rec. N=16 3.4 mg/kg Q8W N=8 5.4 mg/kg Q8W N=8 6.8 mg/kg Q8W N=8 Overall (N=56) Any TEAE 16 (100%) 16 (100%) 8 (100%) 8 (100%) 8 (100%) 56 (100%) Any related TEAE 9 (56%) 10 (63%) 3 (38%) 6 (75%) 6 (75%) 34 (61%) An
Slide 20: vHOT is the Primary Endpoint to Support Potential Accelerated Approval
vHOT is the Primary Endpoint to Support Potential Accelerated Approval of DYNE-101 Myotonia is an early and common sign of DM1 Data support vHOT as early indicator of clinical benefit with DYNE-101 DYNE-101 has demonstrated robust improvement in vHOT as early as 3 months Myotonia First symptom1 38.3
Slide 21: Comprehensive Development Program for DYNE-101 in DM1
Demonstrated Proof of Concept in MAD and LTE Comprehensive Development Program for DYNE-101 in DM1 Registrational Expansion Cohort to Support U.S. Accelerated Approval Planned Phase 3 Study ü Selected registrational dose of 6.8 mg/kg Q8W Data support vHOT improvement as early indicator of clinical b
Slide 22: Program
Program Updated Plan for U.S. Accelerated Approval and New Clinical Data for DYNE-101 Doug Kerr, M.D., Ph.D., Chief Medical Officer Opening Remarks John Cox, President & CEO Available for Q&A Erick Lucera, Chief Financial Officer Closing Remarks John Cox, President & CEO
Slide 23: Summary
Summary 1. Safety data as of April 23, 2025; vHOT = video hand opening time; MAD = multiple ascending dose; LTE = long-term extension. Breakthrough Therapy Designation Accelerated Approval Pathway with vHOT New Long-Term Data from ACHIEVE Trial FDA granted Breakthrough Therapy Designation for DYNE-1
Slide 24: Building Momentum Toward Two Potential Launches in 2027
Building Momentum Toward Two Potential Launches in 2027 DYNE-101 for DM1 DYNE-251 for Exon 51 DMD Q4 2025 Complete enrollment planned for Registrational Expansion Cohort Q1 2025 Completed enrollment of Registrational Expansion Cohort Mid-2026 Data planned for Registrational Expansion Cohort Late 202
Slide 25: Aiming to Deliver Functional Improvement for People Living with Neuromuscular Diseases
Aiming to Deliver Functional Improvement for People Living with Neuromuscular Diseases Two clinical programs in registrational cohorts for DM1 and DMD following positive proof-of-concept data NEAR-TERM VALUE DRIVERS STRONG FINANCIAL POSITION LATE-STAGE PIPELINE Key data readouts in 2025 & 2026 poten
Slide 26: Q&A
THERAPEUTICS Q&A
Slide 27: Appendix
THERAPEUTICS Appendix
Slide 28: Consistent Splicing Correction Maintained Through Month 11
Consistent Splicing Correction Maintained Through Month 11 Splicing Improvement Median of multiple aliquots (revised approach) Single aliquot (original approach) New Data Note: One post-baseline sample in 3.4 mg/kg Q4W and one baseline sample in 6.8 mg/kg treatment groups not included within splicin
Slide 29: ACHIEVE Baseline Participant Characteristics: By Treatment
ACHIEVE Baseline Participant Characteristics: By Treatment Mean (SD) Placebo (N=14) 1.8 mg/kg Q4W (N=6) 3.4 mg/kg Q4W (N=6) 5.4 mg/kg Q8W (N=6) 6.8 mg/kg Q8W (N=6) Age (years) 32.6 (9.6) 37.0 (10.5) 31.2 (4.4) 40.2 (6.5) 37.2 (9.7) BMI (kg/m2) 24.4 (4.7) 21.6 (5.8) 21.1 (1.8) 21.4 (2.5) 23.4 (5.6) C