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ARO-DUX4

Phase 1

Facio-Scapulo-Humeral Dystrophy | Small molecule | Other |Arrowhead Pharmaceuticals, Inc.|Last Updated: Feb 6, 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_CONTROLLEDDMC
Total Trials1
Total Enrollment60
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06131983Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1PHASE1 RECRUITING 60 — — Feb 22, 2024Dec 1, 2026Feb 6, 202617 Australia, Canada +6
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)
Part 1: Up to Day 90; Part 2: Up to Day 360
Secondary Endpoints
Pharmacokinetics (PK) of ARO-DUX4: Maximum Observed Plasma Concentration (Cmax)
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
PK of ARO-DUX4: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ARO-DUX4EXPERIMENTALARO-DUX4 for Injection
PlaceboPLACEBO_COMPARATOR(0.9%NaCl)
Interventions
NameTypeDescription
ARO-DUX4 for InjectionDRUGsingle or multiple doses of ARO-DUX4 by intravenous (IV) infusion
PlaceboDRUGcalculated volume to match active treatment by IV infusion
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Eligibility Criteria
Age Range16 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites17

Inclusion Criteria: * Genetically confirmed FSHD1 based on Screening evaluation or source verifiable medical record * Clinical severity score between 3 and 8 (scale, 0 to 10) * Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader * A 12-lead electrocardiog...

Countries:AustraliaCanadaGermanyItalyNetherlandsNew ZealandSpainThailand
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06131983primaryCompletionDate: changed
LOWMay 24, 2026NCT06131983studyFirstPostDate: changed