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DYNE-101

Phase 1

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment116

FDA Designations

ORPHAN_DRUGFAST_TRACKBREAKTHROUGH_THERAPYACCELERATED_APPROVAL

Clinical trial landscape

DYNE-101 · 1 trial · 1 indication

Phase 1 1
NCT05481879Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1Myotonic Dystrophy Type 1 (DM1)
RECRUITING116 Analytics
PHASE1RECRUITING
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1)
NCT05481879116
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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 to 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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Frequently asked questions about DYNE-101

What is DYNE-101 used for in Myotonic Dystrophy Type 1?

DYNE-101 is an investigational small molecule being developed for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare genetic disease. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being studied for its safety, tolerability, pharmacodynamic effects, efficacy, and pharmacokinetics in participants with DM1.

Who makes DYNE-101?

DYNE-101 is being developed by Dyne Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol DYN. The company is conducting a Phase 1 clinical trial of DYNE-101 in patients with Myotonic Dystrophy Type 1 (DM1) across multiple countries, including the United States, Australia, and several European nations.

What phase is DYNE-101 in?

DYNE-101 is in Phase 1 clinical development. It is an investigational drug for Myotonic Dystrophy Type 1 (DM1) and has not yet received FDA approval. The ongoing Phase 1 trial is actively recruiting participants and is designed to evaluate the drug's safety, tolerability, pharmacodynamic effects, efficacy, and pharmacokinetics in individuals with DM1.

What clinical trials is DYNE-101 in?

DYNE-101 is being evaluated in a single Phase 1 clinical trial registered as NCT05481879. This randomized, double-blind, placebo-controlled study is recruiting 116 participants with Myotonic Dystrophy Type 1 (DM1) aged 18 years and older. The trial is being conducted in the United States, Australia, France, Germany, Italy, Netherlands, New Zealand, and the United Kingdom.

Has DYNE-101 received FDA designations?

DYNE-101 has received several FDA designations, including Orphan Drug, Fast Track, Breakthrough Therapy, and Accelerated Approval designations. These designations are intended to expedite the development and review of the drug for Myotonic Dystrophy Type 1 (DM1). However, the drug remains investigational and is still in Phase 1 clinical trials.