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DYNE-101

Phase 1

Myotonic Dystrophy Type 1 (DM1) | Small molecule | Other |Dyne Therapeutics, Inc.|Last Updated: May 12, 2026

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-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment116
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05481879Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1PHASE1 RECRUITING 116 — — Sep 5, 2022Jul 1, 2029May 12, 202620 United States, Australia +6
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Study Endpoints
Primary Endpoints
MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Through study completion, up to Week 217
Dose Expansion Cohorts: Change From Baseline in Myotonia as Measured by Video Hand Opening Time (vHOT)
Baseline up to Week 25
Secondary Endpoints
MAD Cohorts: Change From Baseline in Composite Alternative Splicing Index (CASI) in Skeletal Muscle Tissue
Baseline up to Week 45
MAD Cohorts: Change From Baseline in Dystrophia Myotonica Protein Kinase (DMPK) Ribonucleic Acid (RNA) Expression in Muscle Tissue
Baseline up to Week 45
MAD Cohorts: Change From Baseline in Hand Grip Relaxation Time
Baseline up to Week 121
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
MAD Cohort: Placebo-Controlled Period: DYNE-101EXPERIMENTALParticipants will be randomized to receive ascending doses of DYNE-101, once every 4 weeks (Q4W) or once every 8 weeks (Q8W) for up to 24 weeks.
MAD Cohort: Placebo-Controlled Period: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive DYNE-101 matching placebo, Q4W or Q8W for up to 24 weeks.
MAD Cohort: Treatment Period: DYNE-101EXPERIMENTALParticipants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q4W or Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q4W or Q8W for up to 24 weeks.
MAD Cohort: Long-Term Extension Period: DYNE-101EXPERIMENTALParticipants will receive DYNE-101, Q4W or Q8W for up to 168 weeks.
Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101EXPERIMENTALParticipants will receive DYNE-101, Q8W for up to 24 weeks.
Dose Expansion Cohort: Placebo-Controlled Period: PlaceboEXPERIMENTALParticipants will receive DYNE-101 matching placebo, Q8W for up to 24 weeks.
Dose Expansion Cohort: Treatment Period: DYNE-101EXPERIMENTALParticipants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.
Dose Expansion Cohort: Long-Term Extension Period: DYNE-101EXPERIMENTALParticipants will receive DYNE-101, Q8W for up to 168 weeks.
Interventions
NameTypeDescription
DYNE-101DRUGAdministered by IV infusion
PlaceboDRUGAdministered by IV infusion
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: * Diagnosis of DM1 with trinucleotide repeat size \>100. * Age of onset of DM1 muscle symptoms ≥12 years. * Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator. * Hand grip strength and ankle dorsiflexion strength...

Countries:United StatesAustraliaFranceGermanyItalyNetherlandsNew ZealandUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05481879primaryCompletionDate: changed
LOWMay 24, 2026NCT05481879studyFirstPostDate: changed