Arrowhead Pharmaceuticals, Inc. · NASDAQ:ARWR
Arrowhead Pharmaceuticals — Investor Presentation (December 2025)
38 slides from Arrowhead Pharmaceuticals's investor presentation, December 2025
Slide 1: Corporate Overview
Corporate Overview December 2025
Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results ma
Slide 3: Financial Highlights
Financial Highlights NASDAQ Global Select – Symbol ARWR Stock Common Shares Market Cash and Price Outstanding Capitalization Investments1 $56.25 ~136m ~$7.65bn ~$920m (12/1/25) (9/30/25) (9/30/25 does not include $200m Sarepta milestone and $200m Novartis upfront payment) Analyst Coverage 1. Refer t
Slide 4: Bringing RNA Interference to Patients
Bringing RNA Interference to Patients Arrowhead Pharmaceuticals is a RNAi therapeutics platform company with a broad pipeline of wholly owned and partnered candidates and achieved its first commercial launch in 2025 • REDEMPLO® (plozasiran) approved by US FDA as an adjunct to diet to reduce triglyce
Slide 5: Arrowhead’s Growth Drivers in 2026 and Beyond
Arrowhead’s Growth Drivers in 2026 and Beyond Emerging potential high value Arrowhead’s first commercial sales obesity and CNS pipelines of REDEMPLO in familial including systemic delivery chylomicronemia across blood brain barrier Phase 3 studies of plozasiran Robust pipeline and productive in seve
Slide 6: Arrowhead’s Targeted RNAi Molecule (TRiM™) Platform
Arrowhead’s Targeted RNAi Molecule (TRiM™) Platform: The Broadest and Most Versatile in the Field TRiM™ also has rules and algorithms to optimize trigger sequence and modification patterns Characterized by depth & duration of effect Activity • Ability to unlock previously undruggable targets To maxi
Slide 7: TRiM Platform Enables Delivery to Seven Cell Types
TRiMTM Platform Enables Delivery to Seven Cell Types Skeletal Cardio- Liver Lung CNS Adipose Ocular Muscle myocyte Strong Deep lung Early Early Early Preclinical Preclinical clinical clinical clinical clinical clinical Stage Stage validation validation stage stage stage (RAGE) Arrowhead is Fulfillin
Slide 8: Robust Pipeline with Near-term Commercial Opportunities
Robust Pipeline with Near-term Commercial Opportunities For investor communication only. Not for use in product promotion 8
Slide 9: REDEMPLO (plozasiran) Arrowhead’s First FDA Approval
REDEMPLO® (plozasiran) Arrowhead’s First FDA Approval
Slide 10: ARO-APOC3 to Plozasiran to REDEMPLO - Path to First Approval
ARO-APOC3 to Plozasiran to REDEMPLO - Path to First Approval First Healthy Volunteer Dosed in Phase 1 First Mixed Hyperlipidemia March 2019 Patient Dosed in MUIR-2 Phase 2 September 2021 ARO-APOC3 Nominated First SHTG Patient Dosed First FCS Patient Dosed in as Clinical Candidate in SHASTA-2 Phase 2
Slide 11: REDEMPLO Now FDA-Approved
REDEMPLO® Now FDA-Approved • REDEMPLO is the first and only siRNA/RNAi-based medicine FDA- approved to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS) • First FDA-approved medicine utilizing Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform For investor com
Slide 12: REDEMPLO Label – Indication Statement and Dosing
REDEMPLO Label – Indication Statement and Dosing -----------------------------INDICATIONS AND USAGE--------------------------- REDEMPLO is an apolipoprotein C-III (apoC-III)-directed small interfering ribonucleic acid (siRNA) indicated as an adjunct to diet to reduce triglycerides in adults with fam
Slide 13: REDEMPLO Label – No Contraindications, Warnings or Precautions
REDEMPLO Label – No Contraindications, Warnings or Precautions, and Acceptable Adverse Event Profile -------------------------------CONTRAINDICATIONS------------------------------- None. (4) -------------------------------ADVERSE REACTIONS------------------------------ Most common adverse reactions
Slide 14: REDEMPLO Positioned as Promising New Medicine for FCS
REDEMPLO Positioned as Promising New Medicine for FCS Dramatic reductions in triglycerides in patients with genetically confirmed and clinically defined FCS Reduced numerical incidence of acute pancreatitis Convenient dosing once every three months with at home self-administration Strong label with
Slide 15: Genetic/Clinical FCS Caused by Inability to Break Down Chylomicrons
Genetic/Clinical FCS Caused by Inability to Break Down Chylomicrons Overview of TG Metabolism in Patients With FCS3 FCS Is Characterized by1,2: • Extremely high fasting plasma TGs, sustained above 880 mg/dL • Marked accumulation of chylomicron particles due to impaired lipolytic clearance The buildu
Slide 16: REDEMPLO Mechanism of Action
REDEMPLO Mechanism of Action Targeting APOC3, a Key Regulator of TG and TRL Metabolism CHYLOMICRONEMIA1,2 REDEMPLO (plozasiran)2 Chylomicron Chylomicron Hepatocyte VLDL Hepatocyte VLDL TRL TRL LPL Dependent LPL Independent LPL Independent LPL Dependent Plozasiran APOC3 APOC3 APOC3 APOC3 TRL-Remnants
Slide 17: Patients With FCS Have Multiple Clinical Manifestations
Patients With FCS Have Multiple Clinical Manifestations, the Most Serious of Which Is Acute Pancreatitis Acute Pancreatitis Is the Most Serious, Severe, and Potentially Fatal Complication of FCS, Which Affects ≥50% of Patients1 The Pancreatitis Disease Continuum2 Clinical Signs and Symptoms of FCS3,
Slide 18: The FCS Lived Experience
The FCS Lived Experience Meet Scott, Living with FCS • Diagnosed after 23 years of symptoms and 25 persistent pancreatitis attacks. • Symptoms began in childhood. In his 20s, noticed eruptive xanthomas and stomach pains with no known cause. His TGs were tested, and numbers were “through the roof,” a
Slide 19: Each AP Event Associated With Significant Healthcare Burden
Each AP Event Associated With Significant Healthcare Burden Challenges around Economic Cost of Human Cost Diagnosis1 Acute Pancreatitis2,4 ▪ ~5 physicians typically ▪ Acute pancreatitis is always ▪ >$60,000 in estimated seen before diagnosis 2 a serious medical condition medical costs per episode of
Slide 20: One-REDEMPLO Pricing Model: Consistent Pricing Across Indications
One-REDEMPLO Pricing Model: Consistent Pricing Across Indications We are committed to sustainable innovation and rational pricing • Different patients should not pay different prices for this medicine based on their disease One-REDEMPLO is a pricing model that sets a single consistent product price
Slide 21: REDEMPLO (plozasiran) U.S. Commercial Launch Plan
REDEMPLO® (plozasiran) U.S. Commercial Launch Plan
Slide 22: FCS Population Estimated to be 6,500 People in the U.S.
The Genetically Confirmed and Clinically Diagnosed FCS Population Is Estimated to be 6,500 People In The U.S. SHTG ▪ TGs ≥ 500 mg/dL Severe Hypertriglyceridemia ▪ > 3 million people (SHTG) ▪ Elevated Risk of AP High-Risk SHTG High-Risk SHTG ▪ TGs ≥ 880 mg/dL or ≥ 500 mg/dL With Prior AP History ▪ ~
Slide 23: FCS Prescriber Base by Specialty
FCS Prescriber Base by Specialty ~6,500 FCS patients in the U.S. HCPs Identified Across 4 Specialties Who Primarily Treat FCS Patients LIPIDOLOGISTS ENDOCRINOLOGISTS PREVENTIVE CARDIOLOGISTS INTERNAL MEDICINE/OTHER For investor communication only. Not for use in product promotion 23
Slide 24: The REDEMPLO GO LO Campaign Key Messages
The REDEMPLO GO LO Campaign Features Key Messages That Support Quality Use of Medicine For investor communication only. Not for use in product promotion 24
Slide 25: Professional Guidelines Recommend Reducing TG Levels to <500 mg/dL
Professional Guidelines Recommend Reducing TG Levels to <500 mg/dL to Lower the Risk of Acute Pancreatitis Guideline(s) Recommendations American Association of Clinical Endocrinologists To reduce the risk of acute pancreatitis, a fibrate, prescription-grade omega-3 fatty acid, and/or (AACE) niacin s
Slide 26: In PALISADE, REDEMPLO Delivered Sustained TG Reductions
In PALISADE, REDEMPLO Delivered Sustained TG Reductions Median Level Below 500 mg/dL at Multiple Timepoints For investor communication only. Not for use in product promotion 26
Slide 27: Patient And HCP Support Ecosystem Is Now Ready
The Patient And HCP Support Ecosystem Is Now Ready To Deliver High Quality Service Rare Disease Specialist Pharmacist Field Reimbursement Navigator Patient Patient Care Support Coordinator Program For investor communication only. Not for use in product promotion 27
Slide 28: Rely On REDEMPLO Patient Support Program
Rely On REDEMPLO Patient Support Program For investor communication only. Not for use in product promotion 28
Slide 29: Feedback From Physicians, Payers, Patients, And Caregivers
Feedback From Physicians, Payers, Patients, And Caregivers Has Been Very Encouraging There is high unmet need for FCS I’m impressed by the results showing It’s remarkable how many patients patients, so a product with such similar TG reductions from baseline in reach triglyceride levels below 500 imp
Slide 30: Expansion in Cardiometabolic
Expansion in Cardiometabolic
Slide 31: Arrowhead Also Active Across TG/LDL and ASCVD/AP Spectrum
Arrowhead Also Active Across TG/LDL and ASCVD/AP Spectrum Lipid Disorders, Including FCS, sHTG, Mixed Hyperlipidemia, and HoFH, Are Characterized by a Spectrum of Elevated Levels of TGs and/or Cholesterol1-12 ANGPTL3/ APOC3 PCSK9 Incremental Mixed risk of acute Residual risk -Rare Disorder- Hyperlip
Slide 32: And We Anticipate Additional Cardiometabolic Launches
And We Anticipate Additional Cardiometabolic Launches ASCVD HOFH SHTG FCS For investor communication only. Not for use in product promotion 32
Slide 33: Obesity Overview
Obesity Overview © arrowhead pharmaceutic icale
Slide 34: Obesity Opportunities
Obesity Opportunities Obesity increases risk of many diseases Genetics and biology including diabetes, heart disease, stroke, support the Activin E ligand and more and reducing fat mass may and ALK7 receptor pathway improve patient outcomes dramatically This is not a market driven by aesthetics, rat
Slide 35: INHBE/ALK7 Pathway Regulates Energy Homeostasis in Adipose Tissue
INHBE/ALK7 Pathway Regulates Energy Homeostasis in Adipose Tissue Hepatocyte Adipocyte Effects of excessive INHBE Activin E binds ALK7 receptor ALK7 Increased Lipolysis TG Activin E Excess Circulating NEFA ALK7 caloric Increased mRNA Adipose hypertrophy & dysfunction intake INHBE Visceral adiposity
Slide 36: CNS Overview
CNS Overview © arrowhead pharmaceutic icale
Slide 37: Neurodegenerative Diseases Are an Enormous Burden Uniquely Addressable by RNA Therapeutics
Neurodegenerative Diseases Are an Enormous Burden Uniquely Addressable by RNA Therapeutics SOD1 (ALS) TDP-43 Proteinopathies Over 50 million neurodegeneration patients • Amyotrophic Lateral Sclerosis (ALS) worldwide1 and few disease modifying therapies • Fronto-temporal dementia (FTD) Tauopathies •
Slide 38: Thank You!
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