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Here are 1-3 brief analogies to describe Arrowhead Pharmaceuticals:

• Moderna for gene-silencing medicines. (Highlights its focus on advanced RNA technology for therapeutics, similar to Moderna's use of mRNA, but for 'turning off' disease-causing genes.)
• An aspiring Amgen specializing in RNA interference drugs. (Positions it as an innovative biotech company aiming for significant impact, much like established giant Amgen, but with a specific focus on RNAi technology.)

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• Fazirsiran (ARO-AAT): An investigational RNAi therapeutic in Phase 3 development for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency.
• ARO-APOC3: An investigational RNAi therapeutic in Phase 2/3 development targeting apolipoprotein C-III to reduce severe hypertriglyceridemia.
• ARO-ANG3: An investigational RNAi therapeutic in Phase 1 development designed to lower angiopoietin-like protein 3 for the treatment of dyslipidemias.
• ARO-MMP7: An investigational RNAi therapeutic in Phase 1 development to treat idiopathic pulmonary fibrosis by reducing matrix metalloproteinase-7.

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Arrowhead Pharmaceuticals (ARWR) sells primarily to other companies, specifically larger pharmaceutical companies, through licensing agreements and strategic collaborations for the development and commercialization of its drug candidates and RNAi technology.

Its major customers include:

• Johnson & Johnson (Janssen Biotech) - Symbol: JNJ
• Amgen Inc. - Symbol: AMGN
• Takeda Pharmaceutical Company Limited - Symbol: TAK

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Christopher Anzalone, Ph.D. - President, Chief Executive Officer, and Chairman of the Board

Dr. Christopher Anzalone has served as President, Chief Executive Officer, and Director of Arrowhead Pharmaceuticals since December 1, 2007, and also holds the position of Chairman of the Board. He is an entrepreneur and biotech executive. In 2005, Dr. Anzalone founded and was CEO of the Benet Group LLC, a private equity firm dedicated to creating and developing new nano-biotechnology companies from university-generated science. Prior to this, he was a partner at Galway Partners, LLC, a Washington D.C.-based private equity firm, where he was involved in sourcing, structuring, and building new business ventures. The Benet Group was acquired by Arrowhead Pharmaceuticals, Inc. on March 13, 2008. He also founded Nanotope, Inc. and was Chief Executive Officer for NanoInk, Inc.

Daniel Apel, MBA, CPA - Chief Financial Officer

Daniel Apel serves as the Chief Financial Officer of Arrowhead Pharmaceuticals. He joined Arrowhead Pharmaceuticals in 2011 and has held various finance roles, including Senior Vice President, Finance, and Chief Accounting Officer, before being appointed CFO. Prior to joining Arrowhead, he was a Senior Manager at Ernst & Young LLP.

James Hamilton, M.D., MBA - Chief Medical Officer, Head of R&D

Dr. James Hamilton is the Chief Medical Officer and Head of Research & Development at Arrowhead Pharmaceuticals. He joined Arrowhead in 2017 as Senior Vice President, Clinical Development, and was promoted to Chief Medical Officer in 2021. Before joining Arrowhead, Dr. Hamilton held leadership positions at other pharmaceutical companies, including Ultragenyx Pharmaceutical, where he served as Vice President, Clinical Development, and BioMarin Pharmaceutical Inc. as Medical Director.

Patrick O'Brien, PharmD, JD - Chief Operating Officer and General Counsel

Dr. Patrick O'Brien serves as the Chief Operating Officer and General Counsel for Arrowhead Pharmaceuticals. He has been with the company since 2006, initially as General Counsel, and his role expanded to include Chief Operating Officer. Prior to Arrowhead, he worked at Amgen Inc. for 10 years in various legal and compliance roles.

Andy Davis, MBA - SVP, Cardiovascular/Metabolic Franchise Head

Andy Davis is the Senior Vice President, Cardiovascular/Metabolic Franchise Head at Arrowhead Pharmaceuticals. He joined Arrowhead in 2018 as Vice President, Commercial Strategy, and later became the head of the cardiovascular and metabolic franchise. Before joining Arrowhead, he spent 15 years at Amgen, where he held various commercial leadership positions across multiple therapeutic areas.

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Key Risks to Arrowhead Pharmaceuticals (ARWR)

The key risks to Arrowhead Pharmaceuticals (ARWR) are primarily centered around intellectual property, regulatory and clinical success, and its financial health as it transitions towards commercialization.

1. Intellectual Property and Patent Disputes: Arrowhead Pharmaceuticals faces a significant risk due to ongoing patent infringement lawsuits with Ionis Pharmaceuticals concerning its investigational drug, plozasiran. Ionis alleges patent infringement related to plozasiran, while Arrowhead has filed its own lawsuit to invalidate the patent. This dual legal action introduces substantial uncertainty regarding the commercial prospects and market exclusivity of plozasiran, a pivotal candidate in Arrowhead's pipeline. The outcome of this high-stakes litigation could materially impact Arrowhead's future drug development and commercialization plans, affecting its ability to leverage its RNA interference (RNAi) technology.

2. Regulatory and Clinical Failure, and Pipeline Concentration: Although plozasiran received FDA approval on November 18, 2025, for familial chylomicronemia syndrome (FCS), the commercial potential for this ultra-rare disease is limited. Arrowhead's valuation remains heavily reliant on the successful clinical development and regulatory approval of its other pipeline programs, including those targeting larger metabolic populations and Alzheimer's disease, which are still in earlier stages of clinical trials. The inherent risks in the biotechnology sector, such as regulatory challenges and the high costs associated with drug development and clinical trials, mean that any negative decisions, delays, or failures in these programs could significantly impact investor confidence and the company's stock.

3. Financial Health and Execution Risk: Arrowhead is in a critical transition phase from a research-focused entity to one with commercial aspirations, a challenging endeavor for a biotech company. While the company currently boasts a strong cash position, largely attributed to collaboration deals like the $825 million Sarepta Therapeutics collaboration, it continues to incur significant Research & Development (R&D) expenses. This has led to operating losses and raises questions about long-term financial sustainability due to revenue volatility and escalating operational costs. Increasing long-term liabilities and the potential for future equity dilution to fund operations or further development also present financial risks to the business.

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The emergence and advancement of gene editing technologies (e.g., CRISPR-Cas9) for the permanent correction or inactivation of disease-causing genes. While Arrowhead Pharmaceuticals focuses on RNA interference (RNAi) to transiently silence genes, gene editing offers the potential for a one-time, permanent cure by directly altering the underlying DNA. Gene editing therapies are progressing rapidly in clinical trials, including for liver-targeted applications and diseases directly addressed by Arrowhead's pipeline (e.g., Alpha-1 Antitrypsin Deficiency (AATD) and Hepatitis B Virus (HBV)). Should these gene editing therapies prove safe, effective, and durable, they could represent a fundamentally superior therapeutic paradigm that challenges Arrowhead's chronic RNAi approach in its key therapeutic areas.

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Arrowhead Pharmaceuticals' key products and their addressable markets are as follows:

• Plozasiran (ARO-APOC3)
  • Targeted Indications: Severe Hypertriglyceridemia (SHTG), Familial Chylomicronemia Syndrome (FCS), Dyslipidemia.
  • Market Size: The market for severe hypertriglyceridemia (SHTG) alone is estimated to represent $2 billion to $3 billion in annual revenue potential globally. Sales for plozasiran, if approved, could reach approximately $707 million by 2032 across dyslipidemia, SHTG, and FCS.
  • Region: Global, with regulatory submissions in the U.S. and EMA.
• Zodasiran (ARO-ANG3)
  • Targeted Indications: Dyslipidemia, Homozygous Familial Hypercholesterolemia (HoFH), Mixed Hyperlipidemia.
  • Market Size: The ANGPTL3 inhibitor market, which includes zodasiran, in the 7MM (United States, Germany, France, Italy, Spain, United Kingdom, and Japan) is projected to increase significantly by 2034 due to the rising prevalence of cardiovascular and lipid disorders, particularly familial hypercholesterolemia. HoFH is considered a small market.
  • Region: 7MM (United States, EU4, United Kingdom, and Japan). HoFH is a rare disease affecting fewer than 200,000 people in the U.S.
• ARO-HSD (licensed to GSK)
  • Targeted Indications: Nonalcoholic steatohepatitis (NASH) and other liver diseases.
  • Market Size: NASH affects millions of people in the U.S. alone, and it represents a very large global market.
  • Region: U.S. and Global.
• ARO-C3
  • Targeted Indications: Complement-mediated diseases, including IgA nephropathy (IgAN), C3 glomerulopathy, paroxysmal nocturnal hemoglobinuria (PNH), and certain lupus populations.
  • Market Size: IgA nephropathy affects millions of people worldwide. PNH is a rare disorder.
  • Region: Global (for IgAN).
• ARO-DUX4 (licensed to Sarepta)
  • Targeted Indication: Facioscapulohumeral muscular dystrophy (FSHD).
  • Market Size: Null
  • Region: Global.
• ARO-DM1 (licensed to Sarepta)
  • Targeted Indication: Myotonic dystrophy type 1 (DM1).
  • Market Size: Null
  • Region: Global.
• ARO-AAT (partnered with Takeda)
  • Targeted Indication: Liver disease associated with alpha-1 antitrypsin deficiency (AATD).
  • Market Size: Null
  • Region: U.S. (co-commercialized), outside U.S. (Takeda commercializes).
• ARO-DIMER-PA
  • Targeted Indication: Atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia.
  • Market Size: There is a substantial unmet medical need in this large patient population.
  • Region: Not explicitly specified, but ASCVD is a global health issue.
• ARO-INHBE and ARO-ALK7
  • Targeted Indication: Obesity.
  • Market Size: Obesity is described as a large and high-value market.
  • Region: Not explicitly specified, but obesity is a global health concern.

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Arrowhead Pharmaceuticals (ARWR) is poised for future revenue growth over the next 2-3 years, driven primarily by the following factors:

1. Commercial Launch and Expansion of Plozasiran: The most immediate driver is the anticipated independent commercial launch of plozasiran, an investigational therapy for familial chylomicronemia syndrome (FCS), with a PDUFA date set for November 18, 2025. Beyond FCS, Phase 3 studies for plozasiran in severe hypertriglyceridemia (SHTG) are expected to conclude by mid-2026, opening a larger market opportunity.
2. Milestone Payments and Royalties from Sarepta Therapeutics Collaboration: Arrowhead has a significant licensing and collaboration agreement with Sarepta Therapeutics, which is a source of substantial revenue through milestone payments and future royalties on commercial sales. The company recently received a $100 million milestone payment and anticipates an additional $200 million by the end of 2025 related to the ARO-DM1 program. The total potential value of this deal, including upfront payments, equity investment, and potential milestones, exceeds $11 billion.
3. Advancement and Expansion of its RNAi Pipeline: Arrowhead is actively developing a broad pipeline of RNA interference (RNAi) therapeutics, particularly focusing on cardiometabolic and central nervous system (CNS) disorders. The company aims to have 20 clinical assets in studies by year-end, with half of these programs being partnered. Upcoming catalysts include anticipated CTA filings for ARO-MAPT and PCSK9/APOC3 dimer programs, with data readouts expected next year.
4. Growth of Recently Launched and Early Clinical Programs: Initial data from early-stage programs targeting obesity and muscle disorders, specifically ARO-INHBE and ARO-ALK7, are expected by the end of the year. Positive data from these programs could lead to further development and potential future revenue streams.
5. New Partnerships Leveraging Proprietary Technology: Arrowhead's robust discovery engine and its portfolio of wholly-owned candidates offer opportunities for forming additional strategic partnerships. These collaborations can provide non-dilutive funding through upfront payments and milestone achievements, further contributing to revenue growth.

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Share Repurchases

• In August 2025, Arrowhead Pharmaceuticals redeemed approximately $50 million worth of its common stock from Sarepta Therapeutics, placing the shares into treasury and thereby reducing outstanding shares.
• This $50 million stock repurchase was part of a $100 million milestone payment from Sarepta, with the remaining $50 million received in cash.
• The company viewed this direct buyback as an attractive opportunity to strengthen shareholder value, effectively reducing outstanding shares by about 2.3% based on the pre-transaction share count.

Share Issuance

• As part of a collaboration agreement announced in November 2024 and closed in February 2025, Sarepta Therapeutics made a $325 million equity investment in Arrowhead at $27.25 per share, representing a 35% premium.
• In fiscal year 2024, Arrowhead Pharmaceuticals raised $429.3 million through an underwritten offering of common stock.
• The number of shares outstanding for Arrowhead Pharmaceuticals increased by 12.21% in 2024 to 0.12 billion and by 1.26% in 2023 to 0.107 billion.

Inbound Investments

• In November 2024, Arrowhead Pharmaceuticals entered a global licensing and collaboration agreement with Sarepta Therapeutics, which included a $500 million upfront payment and a $325 million equity investment.
• In September 2025, Arrowhead entered an exclusive collaboration agreement with Novartis for a potential Parkinson's treatment, receiving $200 million upfront and being eligible for up to $2 billion in potential development, regulatory, and sales milestones.
• In fiscal year 2020, Arrowhead signed an agreement with Takeda to co-develop and co-commercialize ARO-AAT, involving a $300 million upfront payment and potential milestone payments of $740 million.

Capital Expenditures

• Arrowhead's capital expenditures were $141 million in fiscal year 2024 and $177 million in fiscal year 2023.
• Projected capital expenditures are $31 million for fiscal year 2025 and $32 million for fiscal year 2026.
• A collaboration agreement with Sarepta includes Arrowhead manufacturing clinical drug supply for licensed programs and commercial drug product for four clinical programs, which helps utilize existing manufacturing capacity.