Arrowhead Pharmaceuticals

What Arrowhead Pharmaceuticals does

Arrowhead Pharmaceuticals is a clinical-stage-to-commercial biotechnology company that develops RNA interference (RNAi) therapeutics using its proprietary Targeted RNAi Molecule (TRiM™) platform. The platform leverages the natural RNAi gene-silencing pathway to silence disease-causing genes and is engineered to deliver RNAi payloads subcutaneously across multiple tissue types including liver, lung, muscle, and — uniquely — the central nervous system via a proprietary blood-brain barrier crossing delivery system. The company is headquartered in Pasadena, California, employs 501-1,000 people, and trades on NASDAQ under ticker ARWR. It operates research facilities in Madison and San Diego, a manufacturing site in Verona, Wisconsin, and a European regional office in Dublin, Ireland.

The company transitioned to commercial-stage status in November 2025 when the FDA approved REDEMPLO (plozasiran) for familial chylomicronemia syndrome (FCS), a rare genetic disease affecting approximately 6,500 patients in the United States. REDEMPLO is priced at $60,000 per year, distributed exclusively through Vanscoy Rare Pharmacy, and has generated 100 prescriptions in its first 10 weeks. The company subsequently secured approvals in Canada, China, Australia, and the EU. The clinical pipeline spans 14+ programs across cardiometabolic disease (plozasiran for severe hypertriglyceridemia, zodasiran for homozygous familial hypercholesterolemia), obesity (ARO-INHBE and ARO-ALK7 with differentiated efficacy versus GLP-1 monotherapy), pulmonary conditions (ARO-RAGE), and CNS (ARO-MAPT for Alzheimer's tauopathies). ARO-DIMER-PA represents a first-in-class dual-gene RNAi therapeutic for mixed hyperlipidemia.

Arrowhead's business model combines direct product sales of REDEMPLO with substantial licensing and milestone revenue from major pharmaceutical partners. Major deals include Sarepta Therapeutics ($825 million upfront, up to $10 billion in potential milestones), Novartis ($200 million upfront, up to $2.2 billion in potential milestones), Takeda (co-development of ARO-AAT), and Madrigal Pharmaceuticals (ARO-PNPLA3 for MASH). The company has raised approximately $930 million through public equity and convertible notes offerings in 2024-2026 and secured a $500 million non-dilutive credit facility from Sixth Street. FY2025 revenue reached approximately $829 million driven by partner milestone payments, and the company achieved profitability in FY2026 Q1 with $30.8 million in net income.

Name

Arrowhead Pharmaceuticals

Legal name

Arrowhead Pharmaceuticals, Inc.

Website

http://arrowheadpharma.com/

Company type

Public

Founded year

1989

Operating status

Operating

Headcount range

501–1,000 employees

Short description

Arrowhead Pharmaceuticals is a commercial-stage biotechnology company developing RNA interference (RNAi) therapeutics via its proprietary TRiM™ platform to silence disease-causing genes across liver, lung, muscle, and CNS tissues. It markets REDEMPLO for familial chylomicronemia syndrome and licenses pipeline assets to partners including Sarepta and Novartis.

Ownership category

akta.pro rank

Product category

RNAi Biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

siRNA Therapeutics (gene silencing) (HLAAADAB)

akta.pro secondary industries

RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG), RNA Manufacturing & CMC (oligo synthesis, IVT, purification, formulation, QC) (HLAAADAF), RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ), RNA Delivery Technologies (LNPs, polymers, conjugates incl. GalNAc, peptides, exosomes) (HLAAADAD), RNA Therapeutics Safety & Immunogenicity (innate immune activation, tox, off-target) (HLAAADAI)

Keywords

• RNA interference therapeutics
• SiRNA drug development
• Cardiometabolic biopharmaceuticals
• Rare disease medicines
• Targeted gene silencing

Headquarters

HQ city

Pasadena

HQ country

United States

HQ region

North America

Offices5 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure, Supply Chain

Revenue model

1. Product Sales: Commercial revenue from approved drug REDEMPLO (plozasiran) for familial chylomicronemia syndrome, priced at $60,000 annually. The company achieved 100 prescriptions in the first 10 weeks post-launch.
2. Licensing and Milestone Payments: Major licensing agreements including Sarepta ($825M upfront, up to $10B total potential), Novartis ($200M upfront, up to $2.2B total potential), and Madrigal ($25M upfront, up to $1B total potential). Revenue recognized upon milestone achievements and upfront payments.
3. Milestone Payments: Development and regulatory milestone payments from partners including $200M from Sarepta for DM1 program progress, $200M from Novartis for Parkinson's partnership close, and $10M from Sanofi for China approval of REDEMPLO.
4. Strategic Financing: Non-dilutive $500M credit facility with Sixth Street ($400M funded at close, $100M additional available) with maturity August 7, 2031. Used to fund pipeline development and commercial readiness.

Pricing tiers

Go-to-market motion2 records

Distribution channels3 records

Marketing channels6 records

Core offering

Arrowhead Pharmaceuticals develops and commercializes RNA interference (RNAi) therapeutics using its proprietary Targeted RNAi Molecule (TRiM) platform, which silences disease-causing genes across multiple tissue types including liver, lung, muscle, and CNS. The company currently markets REDEMPLO (plozasiran), an FDA-approved siRNA medicine for familial chylomicronemia syndrome, and is advancing a pipeline of over 14 clinical-stage RNAi candidates addressing cardiometabolic, pulmonary, CNS, and rare genetic diseases.

Product overview

Arrowhead Pharmaceuticals is a clinical-stage biopharmaceutical company that develops RNAi (RNA interference) therapeutics using its proprietary TRiM (Targeted RNAi Molecule) platform. The company has transitioned from a development-stage to a commercial-stage company following FDA approval of its first drug, REDEMPLO (plozasiran), in November 2025. The portfolio includes approved and pipeline products: REDEMPLO (plozasiran) for familial chylomicronemia syndrome is the company's first approved commercial product; Plozasiran (ARO-APOC3) is in Phase 3 for severe hypertriglyceridemia; Zodasiran (ARO-ANG3) is in Phase 3 for homozygous familial hypercholesterolemia; ARO-RAGE is in Phase 2 for inflammatory pulmonary diseases; obesity candidates ARO-INHBE and ARO-ALK7 are in Phase 1/2a; ARO-MAPT for Alzheimer's disease is in Phase 1/2a; and ARO-DIMER-PA for mixed hyperlipidemia is in Phase 1/2a. The TRiM platform enables targeted gene silencing across multiple tissue types.

Differentiator

Problem solved

Functional benefit

Brands

• REDEMPLO: Brand name for plozasiran, the company's first FDA-approved siRNA medicine for familial chylomicronemia syndrome (FCS).
• TRiM

Products and services

• REDEMPLO (plozasiran) FDA-approved siRNA medicine that reduces Apolipoprotein-CIII (ApoC3) production through RNA interference to lower triglycerides in adults with familial chylomicronemia syndrome (FCS). Approved in the US, Canada, China, Australia, and the EU.
• Plozasiran (ARO-APOC3) RNAi therapeutic in Phase 3 development for severe hypertriglyceridemia (SHTG), targeting ApoC3 to reduce triglyceride production. Pipeline expansion of the plozasiran program beyond the FCS indication.
• Zodasiran (ARO-ANG3) RNAi therapeutic in Phase 3 development (YOSEMITE study) that reduces production of angiopoietin-like protein 3 (ANGPTL3), a liver-synthesized inhibitor of lipoprotein lipase, targeting cardiovascular disease and homozygous familial hypercholesterolemia.
• ARO-RAGE RNAi therapeutic in Phase 2 development targeting the Receptor for Advanced Glycation End products (RAGE) as a potential treatment for various muco-obstructive and inflammatory pulmonary diseases.
• ARO-INHBE RNAi therapeutic in Phase 1/2a development for obesity and metabolic dysfunction-associated steatohepatitis (MASH), targeting the INHBE gene for differentiated weight loss and liver fat reduction.
• ARO-ALK7 RNAi therapeutic in Phase 1/2a development for obesity, the first RNAi therapeutic to show knockdown of an adipocyte-expressed gene in humans. Targets the ALK7 gene for visceral fat reduction.
• ARO-MAPT Investigational RNAi therapeutic in Phase 1/2a development designed to treat tauopathies including Alzheimer's disease. Uses the proprietary TRiM platform with subcutaneous delivery that crosses the blood-brain barrier to target tau protein (MAPT).
• ARO-DIMER-PA First dual functional RNAi therapeutic in Phase 1/2a development for mixed hyperlipidemia, targeting both ANGPTL3 and ApoC3 simultaneously.

Quantifiable outcome

• REDEMPLO achieved 80% median triglyceride reduction from baseline in FCS patients (Phase 3 PALISADE trial)
• +5 more outcomes

Named customers1 record

Segments5 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Six partnerships are on record, tiered core and secondary.

• Madrigal PharmaceuticalscoreStrategic or Co-development Partner · 5 May 2026Licensed ARO-PNPLA3, an RNAi candidate targeting PNPLA3 I148M genetic mutation for MASH, to Madrigal Pharmaceuticals. Arrowhead received $25 million upfront with potential milestone payments totaling up to $975 million. The asset had been returned to Arrowhead after J&J rejected it. Madrigal intends to add ARO-PNPLA3 to its growing pipeline alongside FDA-approved Rezdiffra.
• TakedacoreStrategic or Co-development Partner · 10 October 2025Co-development and co-commercialization agreement for ARO-AAT (fazirsiran) for Alpha-1 Antitrypsin Deficiency-associated liver disease. Arrowhead earned $40 million milestone when first patient was treated in Phase 3 SEQUOIA study. Takeda acquired additional programs from Arrowhead.
• NovartiscoreStrategic or Co-development Partner · 2 September 2025Global license and collaboration agreement for ARO-SNCA targeting alpha-synuclein for Parkinson's disease and other synucleinopathies. Arrowhead received $200 million upfront payment with potential milestone payments up to $2 billion plus royalties. Arrowhead conducted preclinical research through CTA filing, after which Novartis assumes sole control over development, manufacturing, medical affairs, and commercialization. Deal closed in October 2025.
• Sanofi (via Visirna subsidiary)secondaryChannel Partner/ Reseller/ Distributor · 1 August 2025Arrowhead subsidiary Visirna sold rights to plozasiran (hypertriglyceridemia candidate) in Greater China to Sanofi. Sanofi also received $10 million milestone payment from Arrowhead following China's approval of REDEMPLO for FCS in January 2026.
• Sarepta TherapeuticscoreStrategic or Co-development Partner · 26 November 2024Global licensing and collaboration agreement for multiple clinical and preclinical RNAi programs targeting rare genetic diseases. Arrowhead received $825 million upfront payment with potential future payments up to $10 billion including milestones and royalties. Programs include SRP-1001 (FSHD), SRP-1003 (DM1), SRP-1005 (Huntington's), and additional candidates. Sarepta paid $500 million for rights to seven programs. Arrowhead earned $200 million milestone for DM1 program progress and $100 million milestone in July 2025.
• Roche/JanssensecondaryStrategic or Co-development Partner · 31 October 2024Janssen partnership ended in 2023 after Janssen cut its infectious disease and vaccine units. Johnson & Johnson liquidated its Arrowhead stake in 2026, exiting the relationship. Roche collaboration on hepatitis B programs continued through various arrangements.

Scale indicators7 records

Recent moves6 records

Expansion highlights8 records

Direct peers

• Alnylam Pharmaceuticals: The leading siRNA therapeutics company and the closest direct peer. Alnylam has multiple approved siRNA drugs (Onpattro, Givlaari, Oxlumo, Amvuttra, Leqvio) and a similar platform-based approach to gene silencing. Both companies compete for the same large-pharma partnership economics and target overlapping indications.
• Ionis Pharmaceuticals: Direct competitor in antisense oligonucleotide therapeutics with an approved FCS drug (Tryngolza/olezarsen) that competes head-to-head with Arrowhead's REDEMPLO. Ionis and Arrowhead target the same cardiometabolic and rare disease indications through RNA-based approaches, making them direct strategic peers.

Emerging players

• Wave Life Sciences: Clinical-stage RNA therapeutics company developing stereopure oligonucleotide therapeutics. Comparable to Arrowhead in being an RNA-focused biotech with similar pipeline-stage assets and partnership-driven business model, though smaller and earlier-stage.
• Silence Therapeutics: Clinical-stage siRNA therapeutics company developing mRNAi GOLD platform. Comparable to Arrowhead as an siRNA-focused player with cardiovascular pipeline assets (e.g., SLN360 for Lp(a)). Smaller and earlier-stage but pursues similar siRNA therapeutic strategy.
• Madrigal Pharmaceuticals: MASH-focused biopharma with FDA-approved Rezdiffra, and a current Arrowhead partner (licensed ARO-PNPLA3 in May 2026). Comparable commercial-stage liver/metabolic biotech that validates Arrowhead's MASH pipeline asset and illustrates the typical scale and profile of Arrowhead's partner counterparties.
• Viridian Therapeutics: Clinical-stage rare disease biopharma focused on FcRn inhibitors (autoimmune) and anti-IGF-1R antibodies (thyroid eye disease). Comparable commercial-stage rare-disease biotech at similar development phase and market cap tier to Arrowhead's pre-launch REDEMPLO era.

Broad incumbents

• Sarepta Therapeutics: Arrowhead's largest strategic partner ($10B potential deal), and a leading rare-disease genetic medicine company. While not a direct therapeutic competitor (Sarepta focuses on gene therapy/DMD), it is a benchmark partner of comparable scale and validates the platform's commercial appeal.
• Moderna: Broader RNA therapeutics incumbent (mRNA-based) with established platform, commercial product, and large-scale manufacturing. Comparable as a public RNA-therapeutics leader but operates with different technology (mRNA vs siRNA) and at greater scale across infectious disease and oncology.
• BioNTech: mRNA therapeutics leader with diversified pipeline in oncology and infectious disease. Comparable as a public, platform-based RNA therapeutics company with similar scientific ambitions and capital markets profile, though at much larger scale.

Others

• Royalty Pharma: Financial partner that purchased Arrowhead's olpasiran royalty interest for $250M in 2024. Listed as an ecosystem/adjacent participant that funds biopharma innovation through royalty transactions, illustrating the broader capital-market infrastructure that supports RNAi asset development.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/ArrowheadPharma
• Facebookfacebook.com/101026823293246
• LinkedInlinkedin.com/company/arrowhead-pharmaceuticals
• Instagraminstagram.com/arrowheadpharma

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Subsidiaries2 records

Funding overview

Funding rounds14 records

Investors6 records

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M&A1 record

Investments2 records

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Frequently asked questions about Arrowhead Pharmaceuticals