ARROWHEAD PHARMACEUTICALS, INC.

Arrowhead Pharmaceuticals, Inc.

Overview

Arrowhead develops medicines that silence disease-causing genes using RNA interference (RNAi). The company uses a broad library of RNA chemistries and delivery methods to trigger RNAi and achieve rapid, deep, and durable knockdown of target genes. Arrowhead maintains a discovery pipeline capable of generating new clinical candidates each year and has approximately 20 Arrowhead-discovered drug candidates in clinical trials, including wholly owned and partnered programs.

Core platform

• TRiM (Targeted RNAi Molecule) platform for tissue-targeted delivery.
• Features include ligand-mediated targeting, modular linker chemistries, pharmacokinetic (PK) enhancements, and sequence-specific stabilization chemistries.
• Supports delivery of siRNA to multiple tissues beyond the liver and can silence two genes simultaneously in a single RNAi molecule (dual functional dimer technology).

Selected pipeline programs and status

• Plozasiran (ARO-APOC3) — Severe Hypertriglyceridemia (sHTG) / Familial Chylomicronemia Syndrome (FCS)
  • Phase 3 SHASTA-3 and SHASTA-4 completed with deep triglyceride (TG) reductions; prespecified secondary endpoints met.
  • SHASTA-3: median TG reduction 79% at Month 12.
  • SHASTA-4: median TG reduction 81% at Month 12.
  • Pooled analysis showed reduced acute pancreatitis events, with greater absolute benefit at higher acute pancreatitis risk.
  • sNDA submitted to the U.S. FDA; potential U.S. launch targeted 2Q 2027, subject to regulatory review and approval.
• Zodasiran (ARO-ANG3) — Homozygous Familial Hypercholesterolemia (HoFH) and related dyslipidemias
  • Phase 3 YOSEMITE trial ongoing; expected to complete mid-2027 for eligibility in adolescents and adults.
• ARO-DIMER-PA — Mixed Hyperlipidemia
  • Dual silencing of PCSK9 and APOC3; Phase 1/2a ongoing with interim results showing deep and durable silencing of both targets.
• ARO-INHBE and ARO-ALK7 — Obesity
  • Phase 1/2a trials ongoing.
• ARO-RAGE — Inflammatory pulmonary disease
  • Phase 2 trial ongoing.
• ARO-MAPT — Alzheimer’s disease and tauopathies
  • Phase 1/2a trial; CNS-delivery capability demonstrated in preclinical and early data.
• ARO-C3 and ARO-CFB — Complement-mediated renal diseases
  • Phase 1/2a trials completed for both.
• Several programs are licensed to or in collaboration with partners, including Amgen, Takeda, GSK, Sarepta, Madrigal, Novartis, Visirna, and Sanofi; Arrowhead retains rights or receives milestones/royalties per agreements.

Approved product and commercialization

• REDEMPLO (plozasiran)
  • First FDA-approved RNAi therapeutic, approved as an adjunct to diet to reduce triglycerides in adults with FCS.
  • Marketing authorization in the U.S., EU, Canada, Australia, China, and the U.K.
  • Company building a commercial organization to support REDEMPLO and to scale for a potential sHTG indication.
  • Commercial model includes reliance on specialty pharmacy and a third-party distributor network for a substantial portion of sales; some programs rely on partners for commercialization and distribution.

Intellectual property

• Approximately 710 issued patents worldwide (about 464 directed to RNAi triggers and 165 directed to targeting groups/targeting compounds) and roughly 1,022 patent applications across 113 patent families.
• Patents cover TRiM delivery technologies and RNAi trigger designs, with families addressing CNS, adipose, blood-brain barrier shuttle, cardiomyocyte, and muscle delivery platforms, plus linkers, PK modifiers, and GalNAc/αvβ6 targeting groups.
• Patent expiry estimates exist for multiple target areas (e.g., APOC3, ANGPTL3, DUX4, HTT), subject to issuance and maintenance.
• Arrowhead licenses third-party technologies; license terms may require ongoing payments and licensors may retain certain rights that can affect development.

Facilities and personnel

• Employees: As of September 30, 2026, 1,059 full-time employees in the United States, plus 2 in Switzerland and 1 in New Zealand (total 1,062).
• Facilities:
  • Pasadena, California — ~98,000 sq ft (Corporate Headquarters)
  • Madison, Wisconsin — ~116,000 sq ft (Research Facility)
  • San Diego, California — ~144,000 sq ft (Research and Office Facility)
  • Verona, Wisconsin — drug manufacturing facility (~160,000 sq ft) and analytical/lab facility (~140,000 sq ft) in the Verona Technology Park
• Capabilities: In-house R&D labs, cell culture, animal models, translational biomarker development, in-house PK/PD analytics, iPSC/animal facilities, and a GMP oligonucleotide manufacturing facility at Verona designed to produce GMP drug substance and finished drug product with scalable capacity.

Financial highlights

• Cash and investments: As of September 30, 2026, cash and cash equivalents, restricted cash, and available-for-sale securities totaled approximately $1,193.0 million.
• Debt and liquidity:
  • November 2, 2026 refinancing: amended financing with a $400.0 million senior secured term loan facility due November 2, 2031 (Credit Facility).
  • Prior indebtedness: $700.0 million aggregate principal amount of 0.00% Convertible Senior Notes due 2032 outstanding.
  • Post-refinancing total indebtedness outstanding: $400.0 million under the Credit Facility and $700.0 million under the convertible notes.
  • The Credit Facility bears interest at SOFR + 5.50% (with a floor) or base rate + 4.50% (with a floor).
• Revenue and profitability: The company has a history of net losses and limited product revenue to date; it continues to derive revenue from collaborations, milestones, and other non-product sources.
• Convertible notes: Outstanding convertible notes include potential cash repay/repurchase obligations on a fundamental change or at maturity; note-related covenants and repurchase risks are disclosed.

Subsidiaries and corporate structure

• Arrowhead Madison Inc.
• Arrowhead Australia Pty Ltd
• Arrowhead Pharmaceuticals NZ Limited
• Arrowhead Pharmaceuticals Ireland Limited
• Arrowhead Pharmaceuticals Switzerland GmbH
• Arrowhead Pharmaceuticals Canada Inc.
• Visirna Therapeutics Inc. (Visirna)

Regulatory and compliance

• Subject to U.S. federal and state regulatory regimes (FDA, FDCA, PHSA) for drug approval, manufacturing, advertising, and safety, and to international regulatory regimes (EMA/CHMP, MHRA, NMPA, etc.).
• Data privacy obligations include HIPAA in the U.S. and GDPR/UK GDPR in the EEA/UK; data transfer mechanisms and cross-border data flows are a regulatory consideration.
• Faces IP enforcement and competition risks and is party to licenses with third parties; Ionis Pharmaceuticals alleged patent infringement in 2025.
• Exposed to evolving pricing and reimbursement regimes in the U.S. and abroad, including potential impacts from U.S. reforms and international pricing benchmarks.

Key takeaways

• Arrowhead focuses on RNAi therapeutics and the TRiM delivery platform to expand tissue targeting beyond the liver.
• The company has around 20 pipeline candidates in various clinical stages and one approved product, REDEMPLO, for FCS, with a regulatory pathway underway for a potential sHTG indication.
• Arrowhead maintains a substantial IP portfolio and multiple partnerships with major pharma and biotech companies to advance programs and fund development.
• As of 9/30/2026, Arrowhead held approximately $1.19 billion in cash and investments, carries $1.10 billion of outstanding indebtedness after refinancing, and continues to operate with net losses and limited product revenue to date.