Arrowhead Pharmaceuticals
Develops RNA interference (RNAi) therapeutics and multiple targeted delivery technologies to silence disease-causing genes. Advances programs across cardiometabolic, pulmonary, liver, neuromuscular, oncology and obesity indications, conducts global clinical trials (Phases 1–3), maintains delivery-related intellectual property, and commercializes at least one approved siRNA therapeutic in select markets.
Industries
Nr. of Employees
large (251-1000)
Arrowhead Pharmaceuticals
Patents
RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
US-12729377-B2
View DetailsRNAi agents for inhibiting expression of Beta-ENaC, compositions thereof, and methods of use
US-12655428-B2
View DetailsRNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
US-12630826-B2
View Details
RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
US-12729377-B2
View DetailsRNAi agents for inhibiting expression of Beta-ENaC, compositions thereof, and methods of use
US-12655428-B2
View DetailsRNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
US-12630826-B2
View DetailsProducts
Plozasiran (ARO-APOC3)
An siRNA therapeutic designed to reduce apolipoprotein C-III production to lower triglycerides; advanced through Phase 3 clinical development and commercialized in multiple jurisdictions for severe hypertriglyceridemia indications.
Zodasiran (ARO-ANG3)
An siRNA therapeutic targeting ANGPTL3 to reduce LDL-C and triglycerides, developed for homozygous familial hypercholesterolemia and progressed to late-stage clinical evaluation.
ARO-RAGE
An RNAi candidate designed to silence the RAGE receptor for inflammatory and muco-obstructive pulmonary diseases; evaluated in inhaled and subcutaneous clinical studies.
ARO-ALK7
An investigational RNAi therapeutic designed to silence ACVR1C (ALK7) in adipose tissue to reduce body weight and fat mass; in early clinical development for obesity.
ARC-AAT / Fazirsiran (ARO-AAT)
An RNAi therapeutic candidate developed to reduce hepatic production of mutant Z-alpha1 antitrypsin for treatment of liver disease associated with alpha-1 antitrypsin deficiency; evaluated in Phase 1/2 and later studies with biomarker and histology endpoints.
ARC-520 (HBV candidate)
An investigational siRNA regimen for chronic hepatitis B designed to reduce viral mRNA and protein expression upstream of reverse transcription; evaluated in single- and multi-dose clinical studies and in combination regimens with other antivirals.
Plozasiran (ARO-APOC3)
An siRNA therapeutic designed to reduce apolipoprotein C-III production to lower triglycerides; advanced through Phase 3 clinical development and commercialized in multiple jurisdictions for severe hypertriglyceridemia indications.
Zodasiran (ARO-ANG3)
An siRNA therapeutic targeting ANGPTL3 to reduce LDL-C and triglycerides, developed for homozygous familial hypercholesterolemia and progressed to late-stage clinical evaluation.
ARO-RAGE
An RNAi candidate designed to silence the RAGE receptor for inflammatory and muco-obstructive pulmonary diseases; evaluated in inhaled and subcutaneous clinical studies.
ARO-ALK7
An investigational RNAi therapeutic designed to silence ACVR1C (ALK7) in adipose tissue to reduce body weight and fat mass; in early clinical development for obesity.
ARC-AAT / Fazirsiran (ARO-AAT)
An RNAi therapeutic candidate developed to reduce hepatic production of mutant Z-alpha1 antitrypsin for treatment of liver disease associated with alpha-1 antitrypsin deficiency; evaluated in Phase 1/2 and later studies with biomarker and histology endpoints.
ARC-520 (HBV candidate)
An investigational siRNA regimen for chronic hepatitis B designed to reduce viral mRNA and protein expression upstream of reverse transcription; evaluated in single- and multi-dose clinical studies and in combination regimens with other antivirals.
Expertise Areas
- RNAi drug discovery and oligonucleotide chemistry
- Targeted nucleic acid delivery across multiple tissues
- Preclinical translational pharmacology (rodent and non-human primate)
- Clinical trial design and global execution (Phase 1–3)
Key Technologies
- RNA interference (RNAi) / siRNA therapeutics
- Ligand-conjugate hepatocyte targeting
- Peptide- and polymer-based delivery constructs
- Polyconjugate-style targeted delivery systems
News & Updates
Poster presentation describing an RNAi candidate targeting HIF-2α for renal cell carcinoma; materials made available following the event.
Scheduled participation in Jefferies Global Healthcare Conference and Goldman Sachs Global Healthcare Conference.
Announcement of webcast and conference call to discuss fiscal Q3 results ended June 30, 2024.
Announcement of a webcast and conference call on February 5, 2026 to discuss fiscal 2026 first quarter financial results.
Phase 3 studies met primary and prespecified secondary endpoints with substantial median triglyceride reductions and statistically significant reductions in acute pancreatitis events; plans announced for regulatory submissions.
U.S. patent notice of allowance covering peptide-based in vivo siRNA delivery architectures facilitating hepatocyte delivery.
Poster presentation describing an RNAi candidate targeting HIF-2α for renal cell carcinoma; materials made available following the event.
Scheduled participation in Jefferies Global Healthcare Conference and Goldman Sachs Global Healthcare Conference.
Announcement of webcast and conference call to discuss fiscal Q3 results ended June 30, 2024.
Announcement of a webcast and conference call on February 5, 2026 to discuss fiscal 2026 first quarter financial results.
Phase 3 studies met primary and prespecified secondary endpoints with substantial median triglyceride reductions and statistically significant reductions in acute pancreatitis events; plans announced for regulatory submissions.
U.S. patent notice of allowance covering peptide-based in vivo siRNA delivery architectures facilitating hepatocyte delivery.