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DYNE-251

Phase 1

Duchenne Muscular Dystrophy (DMD) | Small molecule | Neurology |Dyne Therapeutics, Inc.|Last Updated: Aug 13, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment86
FDA Designations
ORPHAN_DRUGBREAKTHROUGH_THERAPYFAST_TRACKRMATACCELERATED_APPROVAL
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05524883Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingPHASE1 ACTIVE NOT_RECRUITING 86 — — Aug 12, 2022Nov 1, 2029Aug 13, 202530 United States, Australia +7
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Through study completion, up to Week 241
Change From Baseline in Dystrophin Protein Levels in Muscle Tissue at Week 25
Baseline, Week 25
Secondary Endpoints
Change From Baseline in Muscle Tissue Exon 51 Skipping Levels at Week 25 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Baseline, Week 25
Change From Baseline in Muscle Tissue Percent Dystrophin-Positive Fiber (PDPF) at Week 25 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Baseline, Week 25
Change From Baseline in Blood Creatine Kinase (CK) Levels up to Week 241 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Baseline, up to Week 241
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Placebo-Controlled MAD Period - DYNE-251EXPERIMENTALDYNE-251 will be administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) over 24 weeks.
Placebo-Controlled MAD Period - PlaceboEXPERIMENTALPlacebo will be administered Q4W or Q8W over 24 weeks.
Open-Label and Long-Term Extension Period - DYNE-251EXPERIMENTALDYNE-251 will be administered Q4W or Q8W for up to 192 weeks after participants complete the Placebo-Controlled MAD Period of the study.
Interventions
NameTypeDescription
DYNE-251DRUGAdministered by IV infusion
PlaceboDRUGAdministered by IV infusion
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Eligibility Criteria
Age Range4 Years — 16 Years
SexMALE
Healthy VolunteersNo
Study Sites30

Inclusion Criteria: * Age 4 to 16 years inclusive, at the time of informed consent/assent. * Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping. * Upper extremity muscle group that is amenable to muscle biops...

Countries:United StatesAustraliaBelgiumCanadaIrelandItalySouth KoreaSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05524883primaryCompletionDate: changed
LOWMay 24, 2026NCT05524883studyFirstPostDate: changed