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ARO-AAT

Phase 2

Alpha 1-Antitrypsin Deficiency | Small molecule | Other |Arrowhead Pharmaceuticals, Inc.|Last Updated: Dec 24, 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 Trials2
Total Enrollment61
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03946449Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)PHASE2 COMPLETED 16 — — Oct 31, 2019Dec 14, 2023Oct 15, 20254 Austria, Germany +1
NCT03362242Study of ARO-AAT in Normal Adult VolunteersPHASE1 COMPLETED 45 — — Mar 12, 2018Mar 21, 2020Dec 24, 20251 New Zealand
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Study Endpoints
Primary Endpoints
Percent Change From Baseline in Total Liver Z-AAT, Insoluble Liver-ZAAT, and Soluble Liver Z-AAT at Week 24: Cohorts 1/1b
Baseline, Week 24
Percent Change From Baseline in Total Liver Z-AAT, Insoluble Liver-ZAAT, and Soluble Liver Z-AAT at Week 48: Cohort 2
Baseline, Week 48
Number of Participants With Adverse Events (AEs) Possibly or Probably Related to Treatment
Part A (single-ascending dose [SAD] phase): up to 29 (+/- 2) days post-dose; Part B (multiple-ascending dose [MAD] phase): up to 113 (+/- 2) days post-dose
Secondary Endpoints
Percent Change From Baseline in Serum Z-AAT Over Time: Cohorts 1/1b
Baseline, Weeks 2, 4, 6, 16, 24
Percent Change From Baseline in Serum Z-AAT Over Time: Cohort 2
Baseline, Weeks 2, 4, 6, 16, 22, 28, 34, 40, 48
Alanine Aminotransferase (ALT) Values Over Time: Cohorts 1/1b
Baseline (Day 1), Day 2, Week 2, Week 4, Week 4 (24-48h post dose), Week 6, Week 16, Week 16 (24/48h post dose), Week 24
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ARO-AAT 100 mg Cohort 1bEXPERIMENTALPrimary Study Period (6-12 months): 100 mg dose of subcutaneous ARO-AAT for a minimum of 3 doses, with 2 optional treatment extension periods. Treatment Extension I (12 months): 100 mg dose of subcutaneous AROAAT every 12 weeks (Q12W). Treatment Extension II (up to 24 Months): 100 mg dose of subcutaneous ARO-AAT Q12W.
ARO-AAT 200 mg Cohort 1EXPERIMENTALPrimary Study Period (6-12 months): 200 mg dose of subcutaneous ARO-AAT for a minimum of 3 doses, with 2 optional treatment extension periods. Treatment Extension I (12 months): 200 mg dose of subcutaneous AROAAT Q12W. Treatment Extension II (up to 24 Months): 200 mg dose of subcutaneous ARO-AAT Q12W.
ARO-AAT 200 mg Cohort 2EXPERIMENTALPrimary Study Period (6-12 months): 200 mg dose of subcutaneous ARO-AAT for a minimum of 5 doses, with optional treatment extension periods. Treatment Extension I (12 months): 200 mg dose of subcutaneous AROAAT Q12W. Treatment Extension II (up to 24 Months): 200 mg dose of subcutaneous ARO-AAT Q12W.
ARO-AATACTIVE_COMPARATOR -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ARO-AATDRUGsolution for subcutaneous injection
ARO-AAT InjectionDRUGSingle or multiple doses of ARO-AAT by subcutaneous (sc) injections
Sterile Normal Saline (0.9% NaCl)OTHERCalculated volume to match active comparator
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Diagnosis of AATD * Liver biopsy indicating Metavir F1-F3 liver fibrosis based on local pathology read. * Women of childbearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use contraception * Willing to provide written inform...

Countries:AustriaGermanyUnited KingdomNew Zealand
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