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ARO-ATXN2

Phase 1

Spinocerebellar Ataxia Type 2 | Small molecule | Neurology |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 Enrollment36
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06672445Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2PHASE1 RECRUITING 36 — — Dec 17, 2024Dec 1, 2026Feb 6, 202616 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), Day 253
Secondary Endpoints
Pharmacokinetics (PK) of ARO-ATXN2: Maximum Observed Plasma Concentration (Cmax)
Through 24 hours post-dose
PK of ARO-ATXN2: Time to Maximum Observed Plasma Concentration (Tmax)
Through 24 hours post-dose
PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Through 24 hours post-dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ARO-ATXN2EXPERIMENTALARO-ATXN2 Injection
PlaceboPLACEBO_COMPARATOR(0.9% NaCl)
Interventions
NameTypeDescription
ARO-ATXN2 InjectionDRUGsingle dose of ARO-ATXN2 by intrathecal (IT) administration
PlaceboDRUGcalculated volume to match active treatment by IT administration
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: * Non-pregnant, non-lactating * Diagnosis of symptomatic SCA2 and ≥33 CAG repeats in the ATXN2 gene based on source verifiable medical records or genetic testing at Screening * Scale of Assessment and Rating of Ataxia (SARA) score ≤14 * Subjects of childbearing potential must ag...

Countries:AustraliaCanadaFranceGermanyItalyNew ZealandSpainTaiwan
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06672445primaryCompletionDate: changed
LOWMay 24, 2026NCT06672445studyFirstPostDate: changed