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Plozasiran

Phase 3

Hypertriglyceridemia | RNA therapy | Metabolic |Arrowhead Pharmaceuticals, Inc.|Last Updated: Sep 2, 2026

Target and mechanism

Molecular targetAPOC3
Target class-Siran (Sirna)
ModalityRNA therapy

Also known as Plozasiran Injection

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment2,325

FDA Designations

BREAKTHROUGH_THERAPYPRIORITY_REVIEW

Clinical trial landscape

Plozasiran · 7 trials · 4 indications

Phase 3 7
NCT06880770Study of Plozasiran in Adults With Severe Hypertriglyceridemia at Risk of Acute PancreatitisSevere Hypertriglyceridemia
RECRUITING288 Analytics
NCT06822790Long-Term Safety and Efficacy of Plozasiran in Adults With HypertriglyceridemiaHypertriglyceridemia
RECRUITING869 Analytics
NCT06347016Study of Plozasiran in Adults With Severe HypertriglyceridemiaSevere Hypertriglyceridemia
ACTIVE NOT_RECRUITING311 Analytics
NCT06347003Study of Plozasiran (ARO-APOC3) in Adults With Severe HypertriglyceridemiaSevere Hypertriglyceridemia
ACTIVE NOT_RECRUITING446 Analytics
NCT06347133Phase 3 Study of Plozasiran in Adults With HypertriglyceridemiaHypertriglyceridemia
ACTIVE NOT_RECRUITING1,456 Analytics
NCT05902598A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia SyndromeFamilial Chylomicronemia Syndrome
COMPLETED37 Analytics
NCT05089084Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)Familial Chylomicronemia
COMPLETED75 Analytics
PHASE3RECRUITING
Study of Plozasiran in Adults With Severe Hypertriglyceridemia at Risk of Acute Pancreatitis
Severe HypertriglyceridemiaUnlock trial analytics
PHASE3RECRUITING
Long-Term Safety and Efficacy of Plozasiran in Adults With Hypertriglyceridemia
HypertriglyceridemiaUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Study of Plozasiran in Adults With Severe Hypertriglyceridemia
Severe HypertriglyceridemiaUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Study of Plozasiran (ARO-APOC3) in Adults With Severe Hypertriglyceridemia
Severe HypertriglyceridemiaUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Phase 3 Study of Plozasiran in Adults With Hypertriglyceridemia
HypertriglyceridemiaUnlock trial analytics
PHASE3COMPLETED
A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome
Familial Chylomicronemia SyndromeUnlock trial analytics
PHASE3COMPLETED
Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)
Familial ChylomicronemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Time to First Occurrence of Positively Adjudicated AP Event (Event Occurring More Than 10 Days After First Dose of Study Drug)
Randomization up to end of Double Blind Treatment Period compared to placebo (Approximate Maximum 50 Months)]
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
From first dose of study drug through Month 24
Percent change in fasting serum TG levels from baseline to Month 12 compared to placebo
Baseline, Month 12
Percent Change in Fasting Serum Triglyceride (TG) Levels from Baseline to Month 12 Compared to Placebo
Baseline, Month 12
Percent Change From Baseline in Fasting Serum Triglyceride (TG) at Month 10
Baseline to Month 10

Blood samples for lipid parameters were collected at the specified time points. Fasting serum TG at Month 10 was defined as geometric mean of 2 measurements taken during Month 10, or the other non-missing measurement if any one of the two measurements was missing during Month 10. Analysis was performed on the basis of the handling strategy for intercurrent events, missing fasting serum was imputed mainly based on the Pattern Mixture Models (PMM). Descriptive statistics were calculated based on non-imputed data.

Percent Change From Baseline at Month 10 in Fasting Triglycerides (TG)
Baseline, Month 10

Secondary Endpoints

Percent Change from Baseline in Fasting Serum Triglyceride (TG) Levels
Baseline up to end of Double Blind Treatment Period compared to placebo (Approximate Maximum 50 Months)
Proportion of Participants Who Achieve Average Fasting TG Levels of < 880 mg/dL (10 mmol/L)
From Month 3 up to end of Double Blind Treatment Period compared to placebo (Approximate Maximum 50 Months)
Proportion of Participants Who Achieve Fasting TG Levels of < 500 mg/dL (5.65 mmol/L)
From Month 3 up to end of Double Blind Treatment Period compared to placebo (Approximate Maximum 50 Months)
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Plozasiran InjectionEXPERIMENTALPlozasiran by SC injection every 3 months (Q3M) through completion of the randomized period Plozasiran by SC injection Q3M through completion of the OLE period
PlaceboEXPERIMENTALcalculated volume to match active treatment by SC injection (randomized period)
Plozasiran 25 mgEXPERIMENTALPlozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months
Plozasiran 50 mgEXPERIMENTALPlozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months
ARO-APOC3 (Plozasiran) 25 mgEXPERIMENTALRandomized Period: plozasiran 25 mg Q3M for a total of 4 doses. Open-label Period (Parts A and B): plozasiran 25 mg Q3M for a total of 8 doses.
Placebo for ARO-APOC3 (Plozasiran) 25 mgPLACEBO_COMPARATORRandomized Period: volume-matched placebo every 3 months (Q3M) for a total of 4 doses. Open-label Period (Parts A and B): plozasiran 25 mg Q3M for a total of 8 doses.
ARO-APOC3 (Plozasiran) 50 mgEXPERIMENTALRandomized Period: plozasiran 25 mg Q3M for a total of 4 doses. Open-label Period: plozasiran 50 mg (Part A), then 25 mg (Part B) Q3M for a total of 8 doses.
Placebo for ARO-APOC3 (Plozasiran) 50 mgPLACEBO_COMPARATORRandomized Period: volume-matched placebo every 3 months (Q3M) for a total of 4 doses. Open-label Period: plozasiran 50 mg (Part A), then 25 mg (Part B) Q3M for a total of 8 doses.

Interventions

NameTypeDescription
PlozasiranDRUGARO-APOC3 injection
PlaceboDRUGsterile normal saline (0.9% NaCl)
Plozasiran InjectionDRUGARO-APOC3 injection
PlacebonDRUGSubcutaneous injection
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites121

Inclusion Criteria: * Males, or nonpregnant (who do not plan to become pregnant) nonlactating females * Established diagnosis of SHTG and prior documented evidence of fasting TG levels of ≥ 880 mg/dL (≥ 10 mmol/L) * Documented evidence of at least 1 prior AP event not attributed to other etiologies...

Countries:United StatesArgentinaAustriaBrazilBulgariaCanadaChinaColombiaHungaryIsraelJordanLatviaLithuaniaMexicoOmanSaudi ArabiaSerbiaSingaporeSouth KoreaSwedenUnited Arab EmiratesUnited KingdomAustraliaBelgiumCroatiaCzechiaFranceGermanyItalyNew ZealandPolandRomaniaSlovakiaSouth AfricaSpainIrelandJapanTurkey (Türkiye)
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Recent Changes (Last 90 Days)

LOWSep 2, 2026NCT06880770lastUpdatePostDate: changed
LOWSep 2, 2026NCT06880770lastUpdatePostDate: changed
LOWSep 2, 2026NCT06880770lastUpdatePostDate: changed
LOWAug 12, 2026NCT06822790lastUpdatePostDate: changed
LOWAug 12, 2026NCT06822790lastUpdatePostDate: changed
LOWAug 6, 2026NCT06347016primaryCompletionDate: changed
LOWAug 6, 2026NCT06347016primaryCompletionDate: changed
LOWJul 24, 2026NCT06880770lastUpdatePostDate: changed
LOWJul 24, 2026NCT06880770lastUpdatePostDate: changed
LOWJul 15, 2026NCT06347003primaryCompletionDate: changed
LOWJul 15, 2026NCT06347003primaryCompletionDate: changed
LOWJul 14, 2026NCT06822790lastUpdatePostDate: changed
LOWJul 14, 2026NCT06822790lastUpdatePostDate: changed
MEDIUMJul 6, 2026NCT05089084TRIAL_REMOVED: changed
MEDIUMJul 6, 2026NCT05089084TRIAL_REMOVED: changed
MEDIUMJul 6, 2026NCT05089084TRIAL_REMOVED: changed
LOWJun 26, 2026NCT06880770lastUpdatePostDate: changed
LOWJun 26, 2026NCT06347133primaryCompletionDate: changed
LOWJun 26, 2026NCT06880770lastUpdatePostDate: changed
LOWJun 26, 2026NCT06347133primaryCompletionDate: changed

Frequently asked questions about Plozasiran

What is Plozasiran?

Plozasiran is an investigational RNA therapy being developed for hypertriglyceridemia and familial chylomicronemia syndrome. It is designed to reduce triglyceride levels in adults with these lipid disorders. Arrowhead Pharmaceuticals, Inc. (ticker ARWR) is developing the drug, which is currently in Phase 3 clinical development.

What does Plozasiran target?

Plozasiran targets APOC3, apolipoprotein C-III. It belongs to the -siran small interfering RNA (siRNA) class of RNA therapies. By targeting APOC3, the drug is designed to reduce production of this protein, which regulates triglyceride metabolism. This mechanism underlies its development for hypertriglyceridemia and familial chylomicronemia syndrome.

Who makes Plozasiran?

Plozasiran is being developed by Arrowhead Pharmaceuticals, Inc., which trades under the ticker ARWR. Arrowhead is the sponsor of the Phase 3 clinical trials evaluating the drug in hypertriglyceridemia and related lipid disorders.

What phase is Plozasiran in?

Plozasiran is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The FDA has granted it Breakthrough Therapy and Priority Review designations. Three Phase 3 trials are active, and one earlier trial has been completed.

What clinical trials is Plozasiran in?

Plozasiran is being studied in Phase 3 trials including NCT06880770 in severe hypertriglyceridemia at risk of acute pancreatitis, NCT06822790 evaluating long-term safety and efficacy in hypertriglyceridemia, NCT06347133 in hypertriglyceridemia, and NCT06347016 in severe hypertriglyceridemia. These trials are randomized, double-blind, and placebo-controlled, with enrollment totaling 2,325 participants.

Is Plozasiran the same as Plozasiran Injection?

Yes. Plozasiran Injection is another name for Plozasiran. Both refer to the same investigational RNA therapy developed by Arrowhead Pharmaceuticals, Inc. (ARWR) for hypertriglyceridemia and familial chylomicronemia syndrome. The drug is currently in Phase 3 clinical development.