Argenx Se · EURONEXT:ARGX
Argenx — J.P. Morgan Healthcare Conference 2022
30 slides from Argenx's conference presentation, Jan 10, 2022
Slide 1: Together We Discover: Reaching Patients Through Immunology Innovation
Together We Discover J.P. Morgan Healthcare Conference January 10, 2022 Reaching Patients Through Immunology Innovation
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation has been prepared by argenx se (“argenx” or the “company”) for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by the presenter or
Slide 3: VYVGART indication in generalized myasthenia gravis
3 VYVGART is indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) antibody positive Please see Important Safety Information and the full Prescribing Information for VYVGART at https://vyvgart.com
Slide 4: Challenging the gMG Treatment Paradigm
Challenging the gMG Treatment Paradigm 4 Best-in-Class Patient Support Rapid HCP Adoption of VYVGART Enable Appropriate Access Meeting our stakeholders where they are Empower Patients to Demand Better
Slide 5: Infrastructure to Bring VYVGART to Physicians and Patients
Infrastructure to Bring VYVGART to Physicians and Patients 5 Infusion Sites of Care Home Infusion Nurses Access in all 50 States VYVGART Field Teams • 71 Territory Business Managers • 10 Thought Leader Liaisons • 16 Medical Research Liaisons • 10 Nurse Case Managers • 13 Market Access Professionals
Slide 6: Global gMG Launch Underway
Global gMG Launch Underway 6 VYVGART Approved December 17, 2021 United States Global Japan Anticipated decision in 1Q 2022 Europe Anticipated decision in 2H 2022 China (Zai Lab) Anticipated filing by mid-2022 Israel (Medison) Anticipated filing in 2Q 2022
Slide 7: Our Mission is Innovation
7 Our Mission is Innovation Where critical patient need meets breakthrough science That is where we redefine immunology
Slide 8: We Innovate At Every Step
Molecule Design: Immunology Innovation Program My Real World™ MG DISCOVERY We Innovate At Every Step Clinical Development: Individualized Dosing Strategic Indication Selection Adapt DEVELOPMENT Commercial Approach: Aligning Stakeholder Interests REACHING PATIENTS Molecule Image
Slide 9: Pipeline Growth Driven By Immunology Innovation Program
ANTIBODY ENGINEERING CLINICAL DEVELOPMENT FIRST IN CLASS | UNIQUE DESIGN | MULTIPLE INDICATIONS Efgartigimod ARGX-119 ARGX-117 Internal Value Creation Strategy LEADING TRANSLATIONAL BIOLOGY LABS External Value Creation Strategy Cusatuzumab LEO (ARGX-112) Genor (ARGX-109) AbbVie (ARGX-115) ARGX-118 A
Slide 10: Efgartigimod Blocks FcRn Leading to IgG Elimination
Efgartigimod Blocks FcRn Leading to IgG Elimination IgG Antibody FcRn Efgartigimod Fc fragment uniquely modulates FcRn No observed impact on IgM, IgA or human serum albumin No observed effect on IgG production (i.e. vaccine response)
Slide 11: gMG is Just the Beginning
NEURO HEME SKIN KIDNEY Key: gMG ITP Pemphigus CIDP 11 gMG is Just the Beginning BP Myositis Four New Launches Anticipated By End of 2024 Q1 2022 (SC) Q2 2022 (IV) Q1 2023 (SC) Q4 2022 Q1 2023 New POC indications Registrational trials ongoing Membranous Nephropathy Lupus Nephritis COVID-19- mediated
Slide 12: Precision Tool Designed to Revolutionize Autoimmunity
IgG IgG IgG IgG Myositis Autoimmune Encephalitis Bullous Pemphigoid Thyroid Eye Disease Membranous Nephropathy ITP Sjogren’s Syndrome GBS AMR Pemphigus POTS CIDP Myasthenia Gravis Lupus Nephritis RA MOGAD NMO HDFN SLE wAIHA APS Anca Vasculitis Precision Tool Designed to Revolutionize Autoimmunity In
Slide 13: ARGX-117: Sweeping Antibody Targeting Complement
C5 C5-C9 (MAC) C1 qrs C4 C2 C3a C3b C5a C3 convertase C5 convertase C3 Mannose sugar MBL MAS Ps IgG IgM ARGX-117 Blocks C2 Targeting C2: • Leaves alternative pathway intact against infection • Blocks all complement effector functions Classical Lectin ARGX-117: Sweeping Antibody Designed to Target Un
Slide 14: ARGX-119: MuSK Agonist with Broad Potential in Neuromuscular Disease
ARGX-119: MuSK Agonist with Broad Potential in Neuromuscular Disease ARGX-119 ( ) induces AChR clustering, NMJ maturation and stabilization Oury, J., Zhang, W., Leloup, N. et al. Mechanism of disease and therapeutic rescue of Dok7 congenital myasthenia. Nature 595, 404–408 (2021). https://doi.org/10
Slide 15: Global Multifranchise Autoimmune Portfolio
Neuro Heme Skin Kidney Global Multifranchise Autoimmune Portfolio ROW China Europe Japan US INDICATIONS THERAPEUTIC FRANCHISES GLOBAL MARKETS Israel Canada MOLECULES Efgartigimod ARGX-117 ARGX-119 gMG ITP PV CIDP IIM BP LN MN Sjogren’s Long COVID-19 POTS MMN DGF Congenital MG ALS MuSK MG SMA gMG: ge
Slide 16: Our Plan to Build a Leading Immunology Company
Efgartigimod in 15 indications (commercial and development) Global autoimmune market has surpassed $150B Vibrant neuromuscular, hematology, skin and kidney franchises New asset each year from IIP Reaching patients globally ARGX-117 in multiple late-stage trials Our Plan to Build a Leading Immunology
Slide 17: Determined to Achieve the Unthinkable
17 Like the Argonauts, we’re on a bold mission: Engineer potentially life-changing immunology therapies for patients. argenx One Plan One Team Determined to Achieve the Unthinkable
Slide 18: Appendix
CONFIDENTIAL. FOR INTERNAL USE ONLY. Appendix
Slide 19: Immune Thrombocytopenia ADVANCE Trials
Immune Thrombocytopenia ADVANCE Trials: Two Run in Parallel 24 weeks 1000mg SC efgartigimod Durable response: sustained platelet count (≥50×109/L) in 4/6 visits between weeks 19 and 24 Phase 3, multicenter, randomized, double-blind, placebo-controlled trial 24 weeks 10mg/kg IV efgartigimod Primary o
Slide 20: CIDP ADHERE Trial
Topline data expected 1Q 2023 • Worsening of disease within 12 weeks after drug withdrawal (INCAT, I-RODS, grip strength) • Newly diagnosed/ treatment naïve skip run-in period • Confirmation of diagnosis by independent committee Chronic Inflammatory Demyelinating Polyneuropathy ADHERE Trial Treatmen
Slide 21: Pemphigus ADDRESS Trial
Topline data expected 4Q 2022 Pemphigus ADDRESS Trial: Focus on Fast Onset and Steroid-sparing 1-3 weeks Screening Efgartigimod weekly SC Placebo weekly SC Pemphigus vulgaris (PV) and foliaceus (PF) Moderate-to-Severe Disease (PDAI activity score ≥ 15) Newly Diagnosed and Relapsing Prednisone starti
Slide 22: Myositis: Adaptive Phase 2/3 Trial Design
Trial to initiate in 1Q 2022 Myositis: Adaptive Phase 2/3 Trial Design 1000mg efgartigimod SC (+ SOC) Placebo SC (+ SOC) Total Duration: 52 weeks Independent adjudication committee Stratified for • IMNM • ASyS • DM • Severity of muscle weakness (MMT) Eligibility criteria • Muscle weakness • No malig
Slide 23: Bullous Pemphigoid Registrational Trial Underway
Bullous Pemphigoid Registrational Trial Underway Total Duration: 36 weeks Bullous Pemphigoid Moderate-to-severe disease (Investigator Global Assessment Score (IGA)) Newly diagnosed and relapsing Proposed primary endpoint • Proportion of participants in complete remission while on minimal OCS (≤0.1mg
Slide 24: Multifocal Motor Neuropathy Phase 2 Trial Underway
Multifocal Motor Neuropathy Phase 2 Trial Underway Total Duration: 16 weeks Patient Population Key Outcome Measures Adults Trial to enroll approximately 45 MMN patients • Probable or definite MMN (per EFNS/PNS 2010) •Independent adjudication committee • Stable IVIg regimen • IVIg treatment dependent
Slide 25: Membranous Nephropathy: Most Common Cause of Nephrotic Syndrome
Membranous Nephropathy (MN): Most Common Cause of Nephrotic Syndrome No Current FDA-Approved Therapies ~70,000 patients in the U.S. Mean age of diagnosis is 50-60 years More predominant in men No approved therapies; all used off-label Currently used therapies include cyclophosphamide, rituximab and
Slide 26: Lupus Nephritis: Severe Organ Manifestation of SLE
Lupus Nephritis (LN): Severe Organ Manifestation of Systemic Lupus Erythematosus Need for new therapies with better tolerability profile ~45,000 patients in the U.S. 60% of adult patients with SLE develop renal symptoms Oral corticosteroids and broad immunosuppressants are current standard of care T
Slide 27: Sjogren's Syndrome: Systemic Autoimmune Disorder of Exocrine Glands
Sjogren’s Syndrome: Systemic Autoimmune Disorder of Exocrine Glands ~300,000 patients in the U.S. 9:1 female to male Men less likely to have systemic manifestations Age of typical onset: 40-50 years IVIg treatment used to treat severe cases Mild to moderate patients typically treated with secretagog
Slide 28: COVID-19 Mediated Postural Orthostatic Tachycardia Syndrome
COVID-19 Mediated Postural Orthostatic Tachycardia Syndrome: Emerging Data Linking IgG Autoimmunity to Long COVID No Current FDA-Approved Therapies ~500,000 – 1,000,000 POTS patients in the U.S. before COVID ~80% of patients are women between 15-50 years of age No approved therapies Symptomatic trea
Slide 29: Multifocal Motor Neuropathy: Serious, Debilitating Neuromuscular Disease
Multifocal Motor Neuropathy: Serious, Debilitating Neuromuscular Disease Need for new therapies that slow progression of disease and reduce reliance on IVIg ~13,000 patients in the U.S. – increasing Often underdiagnosed Predominantly men Median age 40 years Steroids ineffective First line and only a
Slide 30: Delayed Graft Function After Kidney Transplant
Delayed Graft Function After Kidney Transplant Complication Leading to Negative Graft Outcome Need for new therapies that prevent graft loss and shortens hospitalization Occurs in up to 40% of kidney transplant recipients Over 2 million people worldwide currently receive dialysis treatment or are re