Arrowhead Pharmaceuticals, Inc. · NASDAQ:ARWR
Arrowhead Pharmaceuticals — Corporate Update (November 2025)
43 slides from Arrowhead Pharmaceuticals's corporate update, Nov 18, 2025
Slide 1: REDEMPLO (plozasiran) FDA Approval Conference Call
REDEMPLO® (plozasiran) FDA Approval Conference Call November 18, 2025
Slide 2: Welcome and Introductions
REDEMPLO® FDA Approval Conference Call November 2025 Welcome and Introductions Vince Anzalone, CFA Vice President, Finance and IR
Slide 3: 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. Any statements contained in this release except for historical information may be deemed to be forward-looking state
Slide 4: Agenda
Agenda Topic Presenter Introductions and Agenda Vince Anzalone, CFA REDEMPLO® - Arrowhead’s First FDA Approval Chris Anzalone, Ph.D. Disease Background and Label James Hamilton, M.D., MBA PALISADE Phase 3 Clinical Data Bruce Given, M.D. U.S. Commercial Launch Plan Andy Davis, MBA Key Takeaways and A
Slide 5: REDEMPLO - Arrowhead's First FDA Approval
REDEMPLO® FDA Approval Conference Call November 2025 REDEMPLO® - Arrowhead’s First FDA Approval Chris Anzalone, Ph.D. President and CEO
Slide 6: 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 7: 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 8: 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 9: 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 10: 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 11: 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 12: 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 13: Disease Background and Label
REDEMPLO® FDA Approval Conference Call November 2025 Disease Background and Label James Hamilton, M.D., MBA Chief Medical Officer and Head of R&D
Slide 14: 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 15: 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 16: 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 17: REDEMPLO Mechanism of Action Targeting APOC3
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 18: 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 19: 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 20: REDEMPLO Label – Clinical Efficacy
REDEMPLO Label – Clinical Efficacy Figure 1: Median Percent Change from Baseline in Fasting Triglycerides Over Time in Trial 1 14 CLINICAL STUDIES Table 2: Baseline (BL) and Percent Changes from Baseline in Lipid/Lipoprotein Parameters in Patients with FCS at Month 10 in Trial 1 REDEMPLO 25 mg Place
Slide 21: PALISADE Phase 3 Clinical Data
REDEMPLO® FDA Approval Conference Call November 2025 PALISADE Phase 3 Clinical Data Bruce Given, M.D. Chief Medical Scientist
Slide 22: PALISADE Phase 3 Study Enrolled Patients with FCS
PALISADE Phase 3 Study Enrolled Patients with FCS Defined Clinically or Genetically Confirmed Criteria included history of multiple TG measurements above 1000 mg/dL (at least 880 mg/dL at screening), despite standard of care; plus at least one of the following: 1. Prior genetic testing diagnostic of
Slide 23: PALISADE: Randomized Placebo-Controlled Phase 3 Study
PALISADE: Randomized Placebo-Controlled Phase 3 Study of Plozasiran in Patients with FCS Primary Endpoint: • Placebo-adjusted median Placebo (N=25) percent change in triglycerides at Month 10 Randomization SCREENING (Diet, medications, Multiplicity-controlled key and laboratory values) Plozasiran 25
Slide 24: PALISADE Baseline Characteristics
PALISADE Baseline Characteristics Plozasiran Pooled Placebo 25 mg 50 mg Characteristic (N=25) (N=26) (N=24) Mean (SD) age, years 47 (14) 48 (14) 43 (11) Female, n (%) 11 (44) 14 (54) 13 (54) Male, n (%) 14 (56) 12 (46) 11 (46) White, n (%) 19 (76) 19 (73) 17 (71) Mean (SD) BMI, kg/m2 25 (4) 26 (4) 2
Slide 25: Median TGs Reduced 80% from Baseline at Month 10
In PALISADE Median TGs Reduced 80% from Baseline at 1 month to Below Guideline Directed AP Risk Thresholds 4000 4000 Median (Q1, Q3) Levels, mg/dL Median (Q1, Q3) Levels, mg/dL 3500 3000 3000 2500 2000 2000 1500 1000 1000 500 0 0 0 1 2 3 4 5 6 7 8 9 10 11 12 0 1 2 3 4 5 6 7 8 9 10 11 12 Study Month
Slide 26: Plozasiran Significantly Reduced the Incidence of Acute Pancreatitis
Plozasiran Significantly1 Reduced the Incidence of AP† in the Phase 3 PALISADE Study* This is based on a prespecified analysis of the PALISADE study in which 25 mg plozasiran and 50 mg plozasiran were pooled. 50 mg plozasiran has not been reviewed or approved to treat any disease. Time to First Panc
Slide 27: Summary of Safety and Tolerability from PALISADE Phase 3
Summary of Safety and Tolerability from PALISADE Phase 3 Pooled Placebo Plozasiran (N=25) 25 mg (N=26) 50 mg (N=24) • A greater proportion of Adverse Reactions1 placebo-treated patients Hyperglycemia 2 (8) 5 (19) 5 (21) experienced SAEs Headache 2 (8) 3 (12) 5 (21) Nausea 2 (8) 4 (15) 3 (13) Injecti
Slide 28: U.S. Commercial Launch Plan
REDEMPLO® FDA Approval Conference Call November 2025 U.S. Commercial Launch Plan Andy Davis, MBA Senior Vice President, Global Cardiometabolic Franchise
Slide 29: FCS Population Is 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 30: 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 30
Slide 31: The REDEMPLO GO LO Campaign
The REDEMPLO GO LO Campaign Features Key Messages That Support Quality Use of Medicine For investor communication only. Not for use in product promotion 31
Slide 32: 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 32
Slide 33: 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 33
Slide 34: Rely On REDEMPLO Patient Support Program
Rely On REDEMPLO Patient Support Program For investor communication only. Not for use in product promotion 34
Slide 35: 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 36: Key Takeaways and Arrowhead Looking Forward
REDEMPLO® FDA Approval Conference Call November 2025 Key Takeaways and Arrowhead Looking Forward Chris Anzalone, Ph.D. President and CEO
Slide 37: 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 38: 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 39: 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 40: Robust Pipeline with Multiple Mid- and Late-Stage Opportunities
Robust Pipeline with Multiple Mid- and Late-Stage Opportunities *Sanofi holds product rights to plozasiran and ARO-DIMER-PA in Greater China only For investor communication only. Not for use in product promotion 40
Slide 41: 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 42: 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 42
Slide 43: Questions? Answers.
Questions? Answers. For investor communication only. Not for use in product promotion 43
Arrowhead Pharmaceuticals — Corporate Update (November 2025) on 6ix