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Target the Gene,
Silence the Disease

RNAi technology makes it possible to target the genes that cause disease.

Treating diseases with significant unmet needs demands a unique approach at a fast pace, that’s exactly where we thrive. Our targeted RNAi molecule, TRiM™, platform leverages a natural pathway to “silence” genes causing disease. With drug candidates in a growing number of disease areas, TRiM™ has shown it can target genes wherever they are. 

What We Do Best

Acceleration

Acceleration

Every day we can save in our development timeline is one day earlier a person may benefit from our future therapies.

Collaboration

Collaboration

We go farther when we work together to reimagine how therapeutics are developed and delivered.

Growth

Growth

We don’t let uncertainty keep us from tackling big questions and finding meaningful solutions.

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Above & Beyond

Our goal is to have 20 individual products, either partnered or wholly owned, in clinical trials or on the market by 2025. See how we’re already on the way to achieving our ambitious vision. 

Our Pipeline*

There is no shortage of needs that we can endeavor to serve. As scientific explorers, we use our discoveries to develop solutions for the people who need them most. 

Plozasiran

Severe Hypertriglyceridemia liver phase 3

Plozasiran (formerly ARO-APOC3) is designed to reduce the production of the protein Apolipoprotein-CIII (ApoC3) through the natural RNA interference (RNAi) mechanism.  ApoC3 is a protein that is produced in liver cells and inhibits the formation and clearance of various lipids and lipoproteins, including triglycerides.

The goal of treatment with plozasiran is to reduce the level of ApoC3 thereby reducing triglycerides and restoring lipids to more normal levels.

Clinical Trials

planned ID: NCT06347003

This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.

planned ID: NCT06347016

This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After Month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.

complete ID: NCT04720534

The purpose of AROAPOC3-2001 is to evaluate the efficacy and safety of ARO-APOC3 in participants with severe hypertriglyceridemia. Participants will receive 2 subcutaneous injections of ARO-APOC3.

complete ID: NCT03783377

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).

Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults with Severe Hypertriglyceridemia (SHASTA-3)

Severe Hypertriglyceridemia planned ID: NCT06347003

This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.

Inclusion Criteria

  • Established diagnosis of severe hypertriglyceridemia (SHTG) and prior documented evidence (medical history) of fasting TG levels of ≥ 500 mg/dL (≥5.65mmol/L)
  • Mean fasting TG level ≥500 mg/dL (≥5.65 mmol/L) collected at 2 separate and consecutive visits at least 7 days apart and no more than 17 days apart during the screening period
  • Fasting low density lipoprotein-cholesterol (LDL-C) ≤130 mg/dL (≤3.37 mmol/L) at screening
  • Screening HbA1C ≤8.5%
  • Willing to follow diet counseling and maintain a stable low-fat diet
  • Must be on standard of care lipid-lowering medications per local guidelines (unless documented as intolerant as determined by the Investigator)

Timeline

  • April 4, 2024

    Study First Posted

  • May 2024

    Study Start Date

  • October 2026

    Estimated Completion Date

Trial Details

Start date:

May 2024

End date:

October 2026

Participants:

405

Eligibility criteria:

18 Years or Older, All Sexes, No Healthy Volunteers

Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults with Severe Hypertriglyceridemia (SHASTA-4)

Severe Hypertriglyceridemia planned ID: NCT06347016

This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After Month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.

Inclusion Criteria

  • Established diagnosis of severe hypertriglyceridemia (SHTG) and prior documented evidence (medical history) of fasting TG levels of ≥500 mg/dL (≥5.65 mmol/L)
  • Mean fasting TG level ≥500 mg/dL (≥5.65 mmol/L) collected at 2 separate and consecutive visits at least 7 days apart and no more than 17 days apart during the screening period
  • Fasting low density lipoprotein-cholesterol (LDL-C) ≤130 mg/dL (≤3.37 mmol/L) at screening
  • Screening HbA1C ≤8.5%
  • Must be on standard of care lipid-lowering medications per local guidelines (unless documented as intolerant as determined by the Investigator)

Timeline

  • April 4, 2024

    Study First Posted

  • May 2024

    Study Start Date

  • October 2026

    Estimated Completion Date

Trial Details

Start date:

May 2024

End date:

October 2026

Participants:

300

Eligibility criteria:

18 Years or Older, All Sexes, No Healthy Volunteers

A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Severe Hypertriglyceridemia (SHASTA-2)

Severe Hypertriglyceridemia complete ID: NCT04720534

The purpose of AROAPOC3-2001 is to evaluate the efficacy and safety of ARO-APOC3 in participants with severe hypertriglyceridemia. Participants will receive 2 subcutaneous injections of ARO-APOC3.

Inclusion Criteria

  • Based on medical history, evidence of TG ≥ 500 mg/dL and ≤ 4000 mg/dL at Screening
  • Fasting TG ≥ 500 mg/dL at Screening
  • Willing to follow diet counseling per Investigator judgment based on local standard of care
  • Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
  • Willing to provide written informed consent and to comply with study requirements

Timeline

  • January 2021

    Study First Posted

  • May 2021

    Study Start Date

  • March 2023

    Actual study completion date

Trial Details

Start date:

May 2021

End date:

March 2023

Locations:

Australia, Canada, Germany, Hungary, Netherlands, New Zealand, Poland, United States

Participants:

229

Eligibility criteria:

18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers

A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome

Severe Hypertriglyceridemia complete ID: NCT03783377

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).

Inclusion Criteria

  • Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
  • Willing to provide written informed consent and to comply with study requirements
  • Normal electrocardiogram (ECG) at screening
  • Hypertriglyceridemic patients must have a history of fasting serum triglycerides of at least 300 mg/dL (3.38 mmol/L) at screening or verifiable diagnosis of FCS

Timeline

  • December 2018

    Study First Posted

  • March 2019

    Study Start Date

  • February 2021

    Actual study completion date

Trial Details

Start date:

March 2019

End date:

February 2021

Locations:

Australia, Canada, New Zealand

Participants:

112

Eligibility criteria:

18 Years to 70 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers

Zodasiran

Dyslipidemia liver phase 2

Zodasiran, formally ARO-ANG3, is designed to reduce production of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase.

ANGPTL3 inhibition has been shown to lower serum LDL, serum and liver triglyceride and has genetic validation as a novel target for cardiovascular disease.

Clinical Trials

active ID: NCT04832971

The purpose of AROANG3-2001 is to evaluate the efficacy and safety of ARO-ANG3 in participants with mixed dyslipidemia. Participants will initially receive 2 subcutaneous injections of ARO-ANG3 or placebo. Participants who complete the double-blind treatment period may opt to continue in an open-label extension during which they will receive up to 8 doses of ARO-ANG3.

complete ID: NCT03747224

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial hypercholesterolemia and severe hypertriglyceridemia.

A Double-blind, Placebo-controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-ANG3 in Adults With Mixed Dyslipidemia (ARCHES-2)

Dyslipidemia active ID: NCT04832971

The purpose of AROANG3-2001 is to evaluate the efficacy and safety of ARO-ANG3 in participants with mixed dyslipidemia. Participants will initially receive 2 subcutaneous injections of ARO-ANG3 or placebo. Participants who complete the double-blind treatment period may opt to continue in an open-label extension during which they will receive up to 8 doses of ARO-ANG3.

Inclusion Criteria

  • Based on medical history, evidence of TG ≥ 150 mg/dL but ≤ 499 mg/dL
  • Fasting levels at Screening of LDL-C ≥ 70 mg/dL OR non-HDL-C ≥ 100 mg/dL after at least 4 weeks of stable diet and stable optimal statin therapy
  • Mean fasting TG ≥ 150 mg/dL and ≤ 499 mg/dL during Screening collected at two separate and consecutive visits and at least 7 days apart and not more than 17 days apart
  • Willing to follow diet counseling and maintain a stable diet per Investigator judgment based on local standard of care
  • Participants of childbearing potential must agree to use highly-effective contraception during the study and for at least 24 weeks from last dose of study medication
  • Women of childbearing potential must have a negative pregnancy test and cannot be breastfeeding
  • Women of childbearing potential on hormonal contraceptives must be stable on the medication for ≥ 2 menstrual cycles prior to Day 1
  • Men must not donate sperm during the study and for at least 24 weeks following the last dose of study medication
  • Able and willing to provide written informed consent and to comply with study requirements

Timeline

  • April 2021

    Study First Posted

  • June 2021

    Study Start Date

  • August 2022

    Estimated study completion date

Trial Details

Start date:

June 2021

End date:

August 2022

Locations:

Australia, Canada, New Zealand, United States

Participants:

204

Eligibility criteria:

18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers

A Phase 1 Single and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-ANG3 in Adult Healthy Volunteers and in Dyslipidemic Patients

Dyslipidemia complete ID: NCT03747224

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial hypercholesterolemia and severe hypertriglyceridemia.

Inclusion Criteria

  • Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
  • Willing to provide written informed consent and to comply with study requirements
  • On a stable diet for at least 4 weeks with no plans to significantly alter diet or weight over course of study
  • Normal electrocardiogram (ECG) at Screening

Timeline

  • November 2018

    Study First Posted

  • January 2019

    Study Start Date

  • May 2021

    Acrtual study completion date

Trial Details

Start date:

January 2019

End date:

May 2021

Locations:

Australia, New Zealand

Participants:

93

Eligibility criteria:

18 Years to 70 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers

ARO-RAGE

Inflammatory Pulmonary Diseases lung phase 1

ARO-RAGE is designed to reduce production of the Receptor for Advanced Glycation End products (RAGE) as a potential treatment for various muco-obstructive and inflammatory pulmonary diseases.

Clinical Trials

recruiting ID: NCT05276570

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of ARO-RAGE in normal healthy volunteers (NHVs) and in participants with inflammatory lung disease. In Part 1 of the study, NHVs will receive a single dose of ARO-RAGE or placebo. In Part 2 of the study, adult participants with inflammatory lung disease will receive 2 doses of ARO-RAGE or placebo. Additional NHVs may be randomized to receive 1 or 2 doses of ARO-RAGE or placebo at Sponsor discretion. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

recruiting ID: NCT05533294

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-RAGE Injection in normal healthy volunteers.

A Phase 1/2a Study Evaluating the Effects of ARO-RAGE in Healthy Subjects and Patients With Inflammatory Lung Disease

Inflammatory Pulmonary Diseases recruiting ID: NCT05276570

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of ARO-RAGE in normal healthy volunteers (NHVs) and in participants with inflammatory lung disease. In Part 1 of the study, NHVs will receive a single dose of ARO-RAGE or placebo. In Part 2 of the study, adult participants with inflammatory lung disease will receive 2 doses of ARO-RAGE or placebo. Additional NHVs may be randomized to receive 1 or 2 doses of ARO-RAGE or placebo at Sponsor discretion. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

Inclusion Criteria

  • Normal pulmonary function tests at Screening (NHVs only)
  • Confirmed diagnosis of asthma based on source verifiable medical record (asthma patients only)
  • No abnormal finding of clinical relevance at Screening (other than asthma for asthma patients)
  • Stable dose of asthma controller medications for at least 4 weeks prior to Screening (asthma patients only)
  • Non-smoking
  • Able to produce an induced sputum sample at Screening
  • Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception. Males must not donate sperm during the study and for at least 12 weeks following the last dose of study drug
  • Willing to provide written informed consent and to comply with study requirements

Timeline

  • March 2022

    Study First Posted

  • June 2022

    Study Start Date

  • February 2024

    Estimated Study Completion Date

Trial Details

Start date:

June 2022

End date:

February 2024

Locations:

Australia, New Zealand

Participants:

121

Eligibility criteria:

18 Years to 65 Years, All Sexes, Accepts Healthy Volunteers

A Phase 1 Study Evaluating the Effects of ARO-RAGE Injection for Subcutaneous Administration in Healthy Subjects

Inflammatory Pulmonary Diseases recruiting ID: NCT05533294

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-RAGE Injection in normal healthy volunteers.

Inclusion Criteria

  • Normal pulmonary function tests at Screening prior to sputum induction
  • Normal 12-lead electrocardiogram (ECG) at Screening
  • Non-smoking
  • Able to produce an induced sputum sample at Screening
  • Participants of child-bearing potential (male and female) must use highly effective contraception and cannot donate sperm or eggs during the study or for at least 12 weeks following the end of the study or last dose of study drug, whichever is later. Women must have a negative pregnancy test and cannot be breastfeeding
  • Willing to provide written informed consent and to comply with study requirements

Timeline

  • September 2022

    Study First Posted

  • November 2022

    Study Start Date

  • December 2023

    Estimated study completion date

Trial Details

Start date:

November 2022

End date:

December 2023

Locations:

New Zealand

Participants:

50

Eligibility criteria:

18 Years to 55 Years (Adult), All Sexes, Accepts Healthy Volunteers

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Clinical Trials

Our clinical trials are an essential part of our research. Discover the medicines that we’re currently developing.

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Careers at Arrowhead

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