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ARO-DM1 Infusion

Phase 1

Myotonic Dystrophy 1 | Small molecule | Other |Arrowhead Pharmaceuticals, Inc.|Last Updated: Nov 10, 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-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment78
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06138743Study of ARO-DM1 in Subjects With Type 1 Myotonic DystrophyPHASE1 RECRUITING 78 — — Mar 4, 2024Dec 1, 2026Nov 10, 202511 Australia, New Zealand +2
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Study Endpoints
Primary Endpoints
Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)
Single-dose phase (Part 1): Up to Day 90(EOS); multiple-dose phase (Part 2): Up to Day 180(EOS)
Secondary Endpoints
Pharmacokinetics (PK) of ARO-DM1: Maximum Observed Plasma Concentration (Cmax)
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
PK of ARO-DM1: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ARO-DM1 Intravenous (IV) InfusionEXPERIMENTALSingle or multiple doses of ARO-DM1 by IV infusion
Placebo by IV InfusionPLACEBO_COMPARATORSingle or multiple doses of placebo by IV infusion
ARO-DM1 Subcutaneous SC InjectionEXPERIMENTALSingle or multiple doses of ARO-DM1 by sc injection
Placebo by SC InjectionPLACEBO_COMPARATORSingle or multiple doses of placebo by sc injection
Interventions
NameTypeDescription
ARO-DM1 Intravenous (IV) InfusionDRUGARO-DM1 by intravenous (IV) infusion
Placebo Intravenous (IV) InfusionDRUG0.9% NaCl calculated volume to match active treatment by IV infusion
ARO-DM1 subcutaneous (SC) injectionDRUGARO-DM1 by subcutaneous (SC) injection(s)
Placebo Subcutaneous (SC) InjectionDRUG0.9% NaCl calculated volume to match active treatment by SC injection(s)
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: * Genetically confirmed diagnosis of DM1 * Clinician-assessed signs of DM1 including clinically apparent myotonia * Onset of DM1 symptoms occurred after the age of 12 years * Walk for at least 10 meters independently at Screening * Subjects of childbearing potential must agree t...

Countries:AustraliaNew ZealandTaiwanThailand
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06138743primaryCompletionDate: changed
LOWMay 24, 2026NCT06138743studyFirstPostDate: changed