Arrowhead Pharmaceuticals, Inc. · NASDAQ:ARWR

Arrowhead Pharmaceuticals — Technical Presentation (2022)

11 slides from Arrowhead Pharmaceuticals's technical presentation, 2022

  1. Slide 1: ARO-APOC3 Decreases APOC3, Triglyceride, and Non-HDL-C in Severe Hypertriglyceridemia

    #AHA22 ARO-APOC3, an Investigational RNAi Therapeutic, Decreases Serum Apolipoprotein C3, Triglyceride, and Non-HDL-C Concentrations While Increasing HDL-C in Patients With Severe Hypertriglyceridemia Christie M Ballantyne, MD Baylor College of Medicine on behalf of the SHASTA-2 Study Team

  2. Slide 2: Financial Disclosure

    Financial Disclosure Presenter CM Ballantyne reports grants and/or honoraria from Abbott Diagnostic, Akcea, Althera, Amarin, Amgen, Arrowhead, AstraZeneca, Denka Seiken, Esperion, Genentech, Gilead, Illumina, Ionis, Matinas BioPharma Inc, Merck, New Amsterdam, Novartis, Novo Nordisk, Pfizer, Regener

  3. Slide 3: Role of Apolipoprotein C3-Targeted Therapies in Severe Hypertriglyceridemia

    Role of Apolipoprotein C3-Targeted Therapies in Severe Hypertriglyceridemia • Severe hypertriglyceridemia (SHTG) significantly increases risk of acute pancreatitis. • Currently, there are limited effective therapies to treat SHTG. • Apolipoprotein C3 (APOC3) regulates circulating levels of triglycer

  4. Slide 4: SHASTA-2: Double-blind, Placebo-controlled, Dose Ranging Study of ARO-APOC3

    SHASTA 2: An Ongoing Double-blind, Placebo-controlled, Dose Ranging Study of ARO-APOC3 in Subjects With SHTG Study Population: • SHTG history of TG > 500 mg/dL and ARO-APOC3 10 mg (N= 54) • fasting TG of 500 – 4,000 mg/dL during Placebo (N=18) screening period Randomization Screening/Diet Key Endpoi

  5. Slide 5: Baseline Characteristics

    Baseline Characteristics Pooled Placebo ARO-APOC3 (N=46) 10 mg (N=43) 25 mg (N=43) 50 mg (N=45) Mean (SD) age, years 56.3 (11.17) 53.9 (9.29) 56.0 (11.70) 54.8 (10.52) Female, n (%) 11 (24) 4 (9) 12 (28) 13 (29) White, n (%) 42 (91) 37 (86) 37 (86) 41 (91) Mean (SD) BMI, kg/m2 30.4 (3.8) 32.9 (5.1)

  6. Slide 6: ARO-APOC3 Demonstrates Durable Decreases in Serum APOC3 and Triglycerides

    ARO-APOC3 Demonstrates Durable Decreases in Serum APOC3 and Triglycerides at All Doses Studied APOC3 Triglycerides Mean (± SD) (APOC3, mg/dL) 60 2000 1500 50 Triglycerides mg/dL 1000 Median (Q1, Q3) 1000 40 800 30 11% 600 14% 20 73% 400 78% 10 82% 86% 200 0 87% 86% 0 0 2 4 6 8 10 12 14 16 0 2 4 6 8

  7. Slide 7: ARO-APOC3 Decreases Serum Non-HDL-C and Increases HDL-C

    ARO-APOC3 Decreases Serum Non-HDL-C and Increases HDL-C at All Doses Studied Non-HDL-C HDL-C Mean (± SD) (non-HDL-C, mg/dL) Mean (± SD) (HDL-C, mg/dL) 100 350 300 80 83% 250 3% 60 99% 200 37% 40 76% 150 45% 34% 20 4% 100 0 50 0 2 4 6 8 10 12 14 16 0 2 4 6 8 10 12 14 16 Study Week Study Week Placebo

  8. Slide 8: Changes In LDL-C and non-HDL-C at Week 16

    Changes In LDL-C and non-HDL-C at Week 16 Pooled Placebo ARO-APOC3 10 mg 25 mg 50 mg LDL-C (Ultracentrifugation) (mg/dL)* Baseline median (Q1,Q3) 54.0 (31.0, 88.0) 62.0 (29.0, 100.0) 62.5 (44.0, 92.0) 60.0 (30.0, 106.0) Median (Q1,Q3) at Week 16 72.5 (18.0, 90.0) 69.0 (38.0, 120.0) 64.5 (50.0, 126.0

  9. Slide 9: Aggregated Summary of Adverse Events

    Aggregated Summary of Adverse Events # of Subjects Reporting ≥ 1 Treatment Emergent Adverse Event (TEAE) N (%) 77/177(44%) • TEAEs reported to date reflect the TEAEs occurring in > 4 subjects N (%) underlying comorbidities and conditions of the population under study COVID-19 17 (10%) Headache 12 (7

  10. Slide 10: Interim Analysis of SHASTA-2 Suggests Favorable Changes in Triglycerides and Non-HDL-C

    Interim Analysis of SHASTA-2 Study Suggests Favorable Changes in Triglycerides and Non-HDL Cholesterol in Subjects With SHTG • Analysis was performed in SHASTA-2 once 50% of subjects reached their Week 12 visit • Interim results to date demonstrate that ARO-APOC3 durably decreases serum APOC3, TGs,

  11. Slide 11: Thank You

    THANK YOU LL fmcitsst iB Scientific #AHA22 Association. <E? Sessions

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