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ARO-ANG3

Phase 3

Mixed Dyslipidemia | Small molecule | Cardiovascular |Arrowhead Pharmaceuticals, Inc.|Trials Updated: Oct 6, 2026

ARO-ANG3 development status

Highest phase Phase 3 (NCT07037771)
Phase scored for ARWRPhase 2
Registered trials 4 across 1 sponsor since Nov 2018

ARO-ANG3 target and mechanism

ModalitySmall molecule

Also known as zodasiran Injection, Zodasiran

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials4
Total Enrollment377

FDA Designations

No designations recorded

ARO-ANG3 clinical trials

ARO-ANG3 · 4 trials · 5 indications

Phase 3 2Phase 2 1Phase 1 1
NCT07473843Study of Zodasiran in Adolescent Participants With Homozygous Familial HypercholesterolemiaHomozygous Familial Hypercholesterolemia
RECRUITING10 Analytics
NCT07037771A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)Homozygous Familial Hypercholesterolemia
ACTIVE NOT_RECRUITING70 Analytics
PHASE3RECRUITING
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Homozygous Familial HypercholesterolemiaUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
Homozygous Familial HypercholesterolemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Percent Change from Baseline to Month 12 in Fasting LDL-C
Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)
Baseline, Month 12
Percent Change From Baseline in Fasting TG at Week 24
Baseline, Week 24
Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment
Up to 113 (+/- 3 days) post-dose

Secondary Endpoints

Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB)
Baseline, Month 12
Change from Baseline to Month 12 in Fasting ApoB
Baseline, Month 12
Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C)
Baseline, Month 12
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ZodasiranEXPERIMENTAL -
PlaceboPLACEBO_COMPARATORCalculated volume to match active treatment by sc injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period)
ARO-ANG3 50 mgEXPERIMENTALTwo doses of ARO-ANG3 by subcutaneous (sc) injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period.
ARO-ANG3 100 mgEXPERIMENTALTwo doses of ARO-ANG3 bysc injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period.
ARO-ANG3 200 mgEXPERIMENTALTwo doses of ARO-ANG3 by sc injection at Day 1 and Week 12 during double-blind treatment period. Up to 8 doses of ARO-ANG3 by sc injection during the open-label extension period.
ARO-ANG3EXPERIMENTAL -

Interventions

NameTypeDescription
ZodasiranDRUGBy sc injection
zodasiran InjectionDRUGARO-ANG3 Injection
PlaceboDRUGsterile normal saline (0.9% NaCl)
ARO-ANG3DRUGARO-ANG3 Injection
sterile normal saline (0.9% NaCl)DRUGcalculated volume to match active treatment
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Eligibility Criteria

Age Range12 Years to 17 Years
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Adolescents 12 to \<18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study * Body weight ≥35 kilograms (kg) at screening * HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening gen...

Countries:United StatesDenmarkAustraliaAustriaBelgiumBrazilCanadaCzechiaFranceGeorgiaGermanyIsraelJapanNew ZealandSaudi ArabiaSouth AfricaSpainSwedenTurkey (Türkiye)
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Recent Changes (Last 90 Days)

LOWOct 6, 2026NCT07473843Status: NOT_YET_RECRUITING → RECRUITING
LOWOct 6, 2026NCT07473843Status: NOT_YET_RECRUITING → RECRUITING
LOWSep 30, 2026NCT07473843Enrollment: 12 → 10
LOWSep 30, 2026NCT07473843Enrollment: 12 → 10
MEDIUMAug 12, 2026NCT07037771Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 12, 2026NCT07037771Status: RECRUITING → ACTIVE_NOT_RECRUITING

Frequently asked questions about ARO-ANG3

What is ARO-ANG3 used for?

ARO-ANG3 is an investigational drug being developed for the treatment of dyslipidemias, including mixed dyslipidemia, familial hypercholesterolemia, and hypertriglyceridemia. It is designed to address abnormal levels of lipids in the blood, which are risk factors for cardiovascular disease.

Who makes ARO-ANG3?

ARO-ANG3 is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ARWR. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy.

What phase is ARO-ANG3 in?

ARO-ANG3 is currently in clinical development. It has completed a Phase 1 trial and a Phase 2 trial. The drug is not yet approved by regulatory authorities and remains investigational, meaning it is still being studied for safety and effectiveness.

What clinical trials is ARO-ANG3 in?

ARO-ANG3 has been studied in two completed clinical trials. The first, NCT03747224, was a Phase 1 study in healthy volunteers and patients with dyslipidemias, familial hypercholesterolemia, or hypertriglyceridemia. The second, NCT04832971, was a Phase 2 study in adults with mixed dyslipidemia.

Is ARO-ANG3 the same as any other drug?

ARO-ANG3 is the primary name for this investigational drug. No alternative names have been reported for this asset. It is distinct from other drugs in development and is identified by its unique code, ARO-ANG3.