Recent Updates
Recently added Catalysts

ARO-033

Phase 1

Age-related Macular Degeneration | Small molecule | Other |Arrowhead Pharmaceuticals, Inc.|Trials Updated: Sep 15, 2026

ARO-033 development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Jun 2026

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment84

FDA Designations

No designations recorded

ARO-033 clinical trials

ARO-033 · 1 trial · 1 indication

Phase 1 1
NCT07662096A First-In-Human Study of ARO-033 in Adult ParticipantsAge-related Macular Degeneration
RECRUITING84 Analytics
PHASE1RECRUITING
A First-In-Human Study of ARO-033 in Adult Participants
Age-related Macular DegenerationUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Up to Day 337

Secondary Endpoints

Maximum Observed Plasma Concentration (Cmax) of ARO-033
SAD: Predose (0 hour) up to 48 hours postdose (Day 1 up to Day 3); MAD: Predose up to 18 hours postdose (Days 1 and 29), 24 hours postdose (Days 2 and 30), and 48 hours postdose (Days 3 and 31); AMD cohorts: Up to 4 hours postdose (Days 1 and 29)
Area Under the Plasma Concentration-time Curve From Time 0 to the Last Quantifiable Plasma Concentration (AUC0-t) of ARO-033
SAD: Predose (0 hour) up to 48 hours postdose (Day 1 up to Day 3); MAD: Predose up to 18 hours postdose (Days 1 and 29), 24 hours postdose (Days 2 and 30), and 48 hours postdose (Days 3 and 31); AMD cohorts: Up to 4 hours postdose (Days 1 and 29)
NHV Cohorts Only: Amount of ARO-033 Excreted in the Urine From Time 0 to 24 Hours After Dosing (Ae)
SAD: Predose (0 hour) up to 8 hours postdose (Day 1), and up to 24 hours postdose (Day 2); MAD: Predose (0 hour) up to 8 hours postdose (Days 1 and 29)
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
NHV Cohorts: ARO-033EXPERIMENTALParticipants will receive either a single dose of ARO-033 on Day 1 (single-ascending dose \[SAD\]) or 2 doses (multiple-ascending dose \[MAD\]) of ARO-033 administered on Day 1 and Day 29.
NHV Cohorts: PlaceboPLACEBO_COMPARATORParticipants will receive either a single dose of placebo on Day 1 (SAD) or 2 doses (MAD) of placebo administered on Day 1 and Day 29.
AMD Cohorts: ARO-033EXPERIMENTALParticipants will receive up to 5 doses of ARO-033.
AMD Cohorts: PlaceboPLACEBO_COMPARATORParticipants will receive up to 5 doses of placebo.

Interventions

NameTypeDescription
ARO-033DRUGARO-033 will be administered as a subcutaneous (SC) injection per schedule specified in the arm description.
PlaceboDRUGPlacebo matching to ARO-033 will be administered as SC injection per schedule specified in the arm description.
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: * NHV cohorts only: Adults 18 to 65 years of age who are not pregnant, not breastfeeding, and do not plan to become pregnant (or impregnate their partners) during the study and for at least 90 days following the end of the study. * AMD cohorts only: Adults ≥50 years of age w...

Countries:New Zealand
Unlock Eligibility Criteria

Recent Changes (Last 90 Days)

MEDIUMSep 16, 2026NCT07662096Enrollment: 42 → 84
MEDIUMSep 16, 2026NCT07662096Enrollment: 42 → 84
MEDIUMSep 16, 2026NCT07662096Enrollment: 42 → 84
MEDIUMSep 16, 2026NCT07662096Enrollment: 42 → 84
LOWJul 22, 2026NCT07662096Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 22, 2026NCT07662096Status: NOT_YET_RECRUITING → RECRUITING

Frequently asked questions about ARO-033

What is ARO-033?

ARO-033 is an investigational small molecule being developed by Arrowhead Pharmaceuticals, Inc. for age-related macular degeneration. It is in Phase 1 clinical development and has not been approved by the FDA. It is being studied in a first-in-human trial that is currently recruiting participants.

What is ARO-033 used for in age-related macular degeneration?

ARO-033 is being developed as a treatment for age-related macular degeneration, a condition affecting the macula that can lead to vision loss. The drug is in Phase 1 testing, so its use remains investigational. Its first-in-human study is enrolling adults, including healthy volunteers, in New Zealand.

Who makes ARO-033?

ARO-033 is being developed by Arrowhead Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ARWR. The company is the sponsor of the first-in-human study of ARO-033 in adult participants. Arrowhead is a biopharmaceutical company focused on developing medicines that treat disease by silencing specific genes.

What phase is ARO-033 in?

ARO-033 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA. The Phase 1 program consists of a single first-in-human study that is currently recruiting, with no completed trials reported to date. The study is randomized, double-blind, and placebo-controlled.

What clinical trials is ARO-033 in?

ARO-033 is being evaluated in one clinical trial, NCT07662096, titled A First-In-Human Study of ARO-033 in Adult Participants. The Phase 1 study is recruiting and plans to enroll 84 participants with age-related macular degeneration. It is randomized, double-blind, and placebo-controlled, and it is being conducted in New Zealand.