Argenx Se · EURONEXT:ARGX
Argenx — Investor Presentation (February 2022)
35 slides from Argenx's investor presentation, February 2022
Slide 1: Together We Discover: Corporate Presentation February 2022
Together We Discover Reaching Patients Through Immunology Innovation Corporate Presentation FEBRUARY 2022 1
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: Our Plan to Build a Leading Immunology Company
Our Plan to Build a Leading Immunology Company Global autoimmune market has surpassed $150B Committed to Reaching patients globally our Patients and their Communities Vibrant neuromuscular, hematology, skin and kidney franchises argenx 2025 Enviable Immunology Efgartigimod in 15 indications (commerc
Slide 4: VYVGART is indicated for the treatment of generalized myasthenia gravis
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 4
Slide 5: VYVGART: First FDA-Approved FcRn Antagonist
VYVGART: First FDA-Approved FcRn Antagonist Blocking FcRn Mechanism of Action: VYVGART is a human IgG1 antibody fragment Reducing IgG that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG. Restoring function IgG Antibody FcRn VYVGART FcRn, neonatal fragment cry
Slide 6: Patient Experience
GENERALIZED KIM Living with gMG MYASTHENIA GRAVIS gMG is characterized by debilitating muscle Patient weakness and fatigue. Despite taking an average of 2.3 current treatments, 61% of patients have poor well-being according to WHO-5 Index Experience have been diagnosed 1/2 with depression or anxiety
Slide 7: Phase 3 ADAPT Trial Results of Efgartigimod in gMG
Phase 3 ADAPT Trial Results of Efgartigimod in gMG 79 % 60 % Response MSE 89% Rate 84 % Responses Duration of response Onset of Action MG-ADL responders MG-ADL responders MG-ADL responders achieved MG-ADL responders experienced during first two cycles within first two weeks of treatment minimal symp
Slide 8: Challenging the gMG Treatment Paradigm
Challenging the gMG Treatment Paradigm Meeting our stakeholders where they are Empower Patients Best-in-Class Rapid HCP Enable to Demand Better Patient Adoption of Appropriate Support VYVGART Access 8
Slide 9: Infrastructure to Bring VYVGART to Physicians and Patients
Infrastructure to Bring VYVGART to Physicians and Patients * 7.7K* Neurologists treat 97% of gMG patients VYVGART Field Teams • 71 Territory Business • 10 Nurse Case Managers Managers • 13 Market Access • 10 Thought Leader Liaisons Professionals • 16 Medical Research • 10 Field Reimbursement Manager
Slide 10: Global gMG Launch Underway
Global gMG Launch Underway Japan United States Global Approved January 20, 2022 Europe Anticipated decision in 2H 2022 China (Zai Lab) Anticipated filing by mid-2022 Israel (Medison) VYVGART Approved Anticipated filing in 2Q 2022 December 17, 2021 10
Slide 11: Listening to and Learning from MG Community
Listening to and Learning from MG Community 11
Slide 12: gMG is Just the Beginning
gMG is Just the Beginning Four New Launches Anticipated By End of 2024 gMG ITP Pemphigus CIDP Myositis BP Membranous Lupus COVID-19- Sjogren’s Nephropathy Nephritis mediated Syndrome POTS Q1 2022 (SC) Q2 2022 (IV) Q4 2022 Q1 2023 Q1 2023 (SC) 70k US pts 45k US pts 500k US pts 300k US pts Registratio
Slide 13: Autoimmune Pipeline
Autoimmune Pipeline Program Indication Preclinical Phase 1 Proof of Concept Registrational Commercial Update VYVGART gMG US Launch Ongoing gMG Data 1Q22 CIDP Data 1Q23 Myositis Trial to initiate 1Q22 Pemphigus Data 4Q22 Bullous Pemphigoid Enrollment Ongoing Efgartigimod ITP Data 2Q22 ITP Data 1Q23 M
Slide 14: Global Multifranchise Autoimmune Portfolio
Sjogren’s Congenital MG Global Multifranchise ALS SMA Autoimmune Portfolio MuSK MG Long COVID-19 POTS Efgartigimod MMN Kidney DGF INDICATIONS BP MOLECULES Skin LN ARGX-117 ROW IIM China THERAPEUTIC Israel FRANCHISES Heme MN Europe CIDP Canada PV Neuro ARGX-119 GLOBAL ITP Japan MARKETS gMG US gMG: ge
Slide 15: C2 is Ideal Point of Intervention within Complement Cascade
C2 is Ideal Point of Intervention within Complement Cascade (Auto)antibodies Microorganisms Classical Lectin Alternative IgG IgM Mannose sugar Foreign surface C1 MBL C3 2 qrs Intact alternative pathway to reduce 3 C4 C2 MASPs CFB CFD infection risk Crossroad of classical & lectin pathways Manageable
Slide 16: ARGX-117 Blocks and Actively Removes C2 from Circulation
ARGX-117 Blocks and Actively Removes C2 from Circulation Features of ARGX-117 Sweeping Antibody 1 Bind & block C2 pH and Ca2+ switch 2 4 Uptake & drop off C2 Recycling of ARGX-117 Optimal No effector recycling function 3 Degradation of C2 16
Slide 17: Phase 1 Data Support Path Forward and Potential for Individualized Dosing
Phase 1 Data Support Path Forward and Potential for Individualized Dosing Schedule in Patients Favorable safety & tolerability profile supports advancing to Phase 2 patient trials Consistent PK/PD profile across IV and SC dosing that may enable infrequent dosing Phase 2 trial in multifocal motor neu
Slide 18: ARGX-119: MuSK Agonist with Broad Potential in Neuromuscular Disease
ARGX-119: MuSK Agonist with Broad Potential in Neuromuscular Disease Oury, J., Zhang, W., Leloup, N. et al. Mechanism of disease and therapeutic ARGX-119 ( ) induces AChR clustering, NMJ maturation and stabilization rescue of Dok7 congenital myasthenia. Nature 595, 404–408 (2021). https://doi.org/10
Slide 19: Pipeline Growth Driven By Immunology Innovation Program
Pipeline Growth Driven By Immunology Innovation Program CLINICAL DEVELOPMENT LEADING ANTIBODY ENGINEERING TRANSLATIONAL BIOLOGY LABS Internal Value Creation Strategy External Value Creation Strategy Staten AgoMAb FIRST IN CLASS | UNIQUE DESIGN | MULTIPLE INDICATIONS ARGX-118 Dualyx (ARGX-116) (ARGX-
Slide 20: argenx: Engineer potentially life-changing immunology therapies
One Team argenx Like the Argonauts, we’re on a bold mission: One Plan Engineer potentially Determined to Achieve the life-changing immunology Unthinkable therapies for patients. 20
Slide 21: Appendix
Appendix C O N F I D E N T I A L . F O R I N T E R N A L U S E O N L Y .
Slide 22: Immune Thrombocytopenia ADVANCE Trials: Two Run in Parallel
Immune Thrombocytopenia ADVANCE Trials: Two Run in Parallel Phase 3, multicenter, randomized, double-blind, placebo-controlled trial N=156 24 weeks 10mg/kg IV efgartigimod Primary Fixed weekly Weekly or q2w dosing adjusted Fixed weekly objective dosing according to platelet count or q2w dosing Durab
Slide 23: Chronic Inflammatory Demyelinating Polyneuropathy ADHERE Trial
Chronic Inflammatory Demyelinating Polyneuropathy ADHERE Trial Confirm IgG autoantibody Assess efficacy & safety involvement efgartigimod vs placebo Efficacy analysis Treatment period based on relapse Identify patients with active CIDP (adjusted INCAT) Open-label Placebo-controlled Screening Run-in
Slide 24: CIDP: Therapeutic Activity Shown With Increasing Selectivity For IgG Reductions
CIDP: Therapeutic Activity Shown With Increasing Selectivity For IgG Reductions Selectivity for IgG Therapeutic Response Rate ICE Trial IVIg 50-70% PLEX 33-80% Response rate active Comparable to PLEX 54% vs placebo 21% Immuno IA with tryptophan matrix after 24 weeks (N=117) adsorption 80-100% IA wit
Slide 25: Pemphigus ADDRESS Trial: Focus on Fast Onset and Steroid-sparing
Pemphigus ADDRESS Trial: Focus on Fast Onset and Steroid-sparing Primary endpoint is proportion of PV Concomitant patients achieving Screening Randomization (2x1) prednisone CRmin* within 30 weeks Pemphigus vulgaris Prednisone starting (PV) and foliaceus (PF) dose 0.5 mg/kg/day N=sample size with ab
Slide 26: Myositis: Adaptive Phase 2/3 Trial Design
Myositis: Adaptive Phase 2/3 Trial Design 180 patients Total Duration: 52 weeks Proposed Endpoints Independent 1000mg efgartigimod SC (+ SOC) Primary adjudication committee IMNM Mean Total Improvement Score (TIS) Placebo SC (+ SOC) Change (ACR/EULAR endorsed) Stratified for • IMNM 1000mg efgartigimo
Slide 27: Bullous Pemphigoid Registrational Trial Underway
Bullous Pemphigoid Registrational Trial Underway 160 patients Total Duration: 36 weeks Endpoints Bullous Pemphigoid Proposed primary endpoint Randomization (1:1) • Proportion of participants in complete Moderate-to-severe remission while on minimal OCS disease (Investigator (≤0.1mg/kg/day) for ≥8 we
Slide 28: Bullous Pemphigoid: Expanding the Skin Franchise
Bullous Pemphigoid: Expanding the Skin Franchise Autoimmune Blistering Diseases Pemphigus Pemphigoid Autoantibody Driven DSG1 and DSG3 BP180 and BP230 Convincing Rationale IVIg, PLEX, Immunoadsorption demonstrate role of IgG Unmet Patient Needs Fast-acting, tolerable therapies; ability to taper cort
Slide 29: Multifocal Motor Neuropathy Phase 2 Trial Underway
Multifocal Motor Neuropathy Phase 2 Trial Underway Patient Population Total Duration: 16 weeks Key Outcome Measures Trial to enroll Safety and tolerability approximately 45 MMN patients ARGX-117 Dose Time to IVIg re-treatment • Probable or definite Regimen 1 (N=15) MMN (per EFNS/PNS Measures of peri
Slide 30: Membranous Nephropathy (MN)
Membranous Nephropathy (MN): Most Common Cause of Nephrotic Syndrome No Current FDA-Approved Therapies PREVALENCE HALLMARK DIAGNOSIS UNMET NEED ~70,000 patients in the U.S. Thickening of the glomerular Nephrotic syndrome, No approved therapies; all capillary walls due to immune characterized by prot
Slide 31: Lupus Nephritis (LN)
Lupus Nephritis (LN): Severe Organ Manifestation of Systemic Lupus Erythematosus Need for new therapies with better tolerability profile PREVALENCE HALLMARK DIAGNOSIS UNMET NEED ~45,000 patients in the U.S. Form of glomerulonephritis LN can be presenting Oral corticosteroids and broad manifestation
Slide 32: Sjogren's Syndrome
Sjogren’s Syndrome: Systemic Autoimmune Disorder of Exocrine Glands No Current FDA-Approved Therapies PREVALENCE HALLMARK DIAGNOSIS UNMET NEED ~300,000 patients in the U.S. Inflammation of exocrine glands Delayed diagnosis, often due to IVIg treatment used to treat severe results in the development
Slide 33: 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 PREVALENCE HALLMARK DIAGNOSIS UNMET NEED ~500,000 – 1,000,000 POTS Excessive heart rate increase in an Despite well-defined diagnostic No approved thera
Slide 34: Multifocal Motor Neuropathy
Multifocal Motor Neuropathy: Serious, Debilitating Neuromuscular Disease Need for new therapies that slow progression of disease and reduce reliance on IVIg PREVALENCE HALLMARK DIAGNOSIS UNMET NEED ~13,000 patients in the U.S., UK, Slowly progressive muscle Often misdiagnosed as ALS Steroids ineffec
Slide 35: 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 PREVALENCE HALLMARK DIAGNOSIS UNMET NEED Occurs in up to 40% of kidney Ischemia reperfusion injury (IRI) is Delayed graft function