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ARO-APOC3

Phase 2

Dyslipidemias | Small molecule | Cardiovascular |Arrowhead Pharmaceuticals, Inc.|Last Updated: May 12, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment418

FDA Designations

No designations recorded

Clinical trial landscape

ARO-APOC3 · 4 trials · 5 indications

Phase 2 3Phase 1 1
NCT05413135Study of ARO-APOC3 in Adults With DyslipidemiaDyslipidemias
COMPLETED418 Analytics
NCT04998201Study of Plozasiran (ARO-APOC3) in Adults With Mixed DyslipidemiaMixed Dyslipidemia
COMPLETED353 Analytics
NCT04720534Study to Evaluate ARO-APOC3 in Adults With Severe HypertriglyceridemiaSevere Hypertriglyceridemia
COMPLETED229 Analytics
PHASE2COMPLETED
Study of ARO-APOC3 in Adults With Dyslipidemia
DyslipidemiasUnlock trial analytics
PHASE2COMPLETED
Study of Plozasiran (ARO-APOC3) in Adults With Mixed Dyslipidemia
Mixed DyslipidemiaUnlock trial analytics
PHASE2COMPLETED
Study to Evaluate ARO-APOC3 in Adults With Severe Hypertriglyceridemia
Severe HypertriglyceridemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Through 24 months
Percent Change From Baseline at Week 24 in Fasting TG
Baseline, Week 24

Least square (LS) mean and standard error (SE) were calculated using mixed model repeated measures (MMRM) which included treatment arm, study visit, and baseline value as model terms and treatment by visit and treatment by baseline as interaction terms.

Percent Change From Baseline at Week 24 in Fasting Triglycerides (TG)
Baseline, Week 24
Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment
Up to Day 113 (+/- 3 days)

Secondary Endpoints

Change from Baseline in Fasting Triglycerides (TG) Over Time
Through 24 months
Percent Change from Baseline in Fasting TG Over Time
Through 24 months
Change from Baseline in Apolipoprotein (Apo) C-III Over Time
Through 24 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ARO-APOC3EXPERIMENTAL1 dose of ARO-APOC3 by subcutaneous (sc) injection every 3 months
ARO-APOC3 10 mg Q12WEXPERIMENTALARO-APOC3 10 milligrams (mg) subcutaneous (SC) injection on Day 1 and Week 12 (Q12W) for a total of 2 injections
ARO-APOC3 25 mg Q12WEXPERIMENTALARO-APOC3 25 mg SC injection on Day 1 and Week 12 (Q12W) for a total of 2 injections
ARO-APOC3 50 mg Q12WEXPERIMENTALARO-APOC3 50 mg SC injection on Day 1 and Week 12 (Q12W) for a total of 2 injections
ARO-APOC3 50 mg Q24WEXPERIMENTALARO-APOC3 50 mg SC injection on Day 1 and Week 24 (Q24W) for a total of 2 injections
PlaceboPLACEBO_COMPARATORVolume-matched placebo SC injection on Day 1 and Week 12 for a total of 2 injections
Placebo, Day 1 and Week 12EXPERIMENTALParticipants received a total of 2 placebo SC injections on Day 1 and Week 12 for a total of 2 injections.
ARO-APOC3 10 mgEXPERIMENTALParticipants received a total of 2 ARO-APOC3 10 mg SC injections on Day 1 and Week 12 for a total of 2 injections.
ARO-APOC3 25 mgEXPERIMENTALParticipants received a total of 2 ARO-APOC3 25 mg SC injections on Day 1 and Week 12 for a total of 2 injections.
ARO-APOC3 50 mgEXPERIMENTALParticipants received a total of 2 ARO-APOC3 50 mg SC injections on Day 1 and Week 12 for a total of 2 injections.

Interventions

NameTypeDescription
ARO-APOC3DRUGARO-APOC3 Injection
PlaceboDRUGSterile Normal Saline (0.9% NaCl)
sterile normal saline (0.9% NaCl)DRUGcalculated volume to match active treatment
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites60

Inclusion Criteria: * Adults who are nonpregnant, nonlactating and do not plan to become pregnant during the study * Able and willing to provide written informed consent * Completed the 48-week study treatment period in the parent study Exclusion Criteria: * Subject was permanently discontinued f...

Countries:United StatesAustraliaCanadaHungaryNetherlandsNew ZealandPolandGermany
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Recent Changes (Last 90 Days)

MEDIUMJun 12, 2026NCT05413135TRIAL_REMOVED: changed
MEDIUMJun 12, 2026NCT05413135TRIAL_REMOVED: changed
MEDIUMJun 12, 2026NCT05413135TRIAL_REMOVED: changed

Frequently asked questions about ARO-APOC3

What is ARO-APOC3 used for?

ARO-APOC3 is an investigational drug being studied for the treatment of hypertriglyceridemia, mixed dyslipidemia, severe hypertriglyceridemia, and dyslipidemias. It is being developed by Arrowhead Pharmaceuticals for cardiovascular conditions. The drug is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.

What does ARO-APOC3 target?

ARO-APOC3 targets apolipoprotein C3, a protein involved in triglyceride metabolism. By reducing the production of this protein, the drug aims to lower triglyceride levels in the blood. This mechanism is being investigated for its potential to treat conditions characterized by elevated triglycerides, such as hypertriglyceridemia and mixed dyslipidemia.

Who makes ARO-APOC3?

ARO-APOC3 is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker ARWR. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with various lipid disorders, including severe hypertriglyceridemia and mixed dyslipidemia.

What phase is ARO-APOC3 in?

ARO-APOC3 is in Phase 2 clinical development. Multiple Phase 2 trials have been completed, including studies in adults with severe hypertriglyceridemia, mixed dyslipidemia, and dyslipidemias. The drug is investigational and has not received FDA approval. Earlier Phase 1 studies have also been completed in healthy volunteers and patients with hypertriglyceridemia.

What clinical trials is ARO-APOC3 in?

ARO-APOC3 has been studied in several completed clinical trials. NCT04720534 evaluated the drug in adults with severe hypertriglyceridemia, NCT04998201 studied it in mixed dyslipidemia, and NCT05413135 examined its effects in dyslipidemias. An earlier Phase 1 trial, NCT03783377, included healthy volunteers and patients with hypertriglyceridemia or familial chylomicronemia syndrome.

Is ARO-APOC3 the same as plozasiran?

Yes, ARO-APOC3 is also known as plozasiran. In clinical trials, the drug has been referred to by both names. For example, NCT04998201 is titled 'Study of Plozasiran (ARO-APOC3) in Adults With Mixed Dyslipidemia.' Researchers and clinicians may use either name when referring to this investigational therapy.