Argenx Se · EURONEXT:ARGX
Argenx — Investor Presentation (April 2022)
35 slides from Argenx's investor presentation, April 2022
Slide 1: Together We Discover: Reaching Patients Through Immunology Innovation
Together We Discover Reaching Patients Through Immunology Innovation Corporate Presentation APRIL 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 Focused on global, expanding autoimmune market 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 (comme
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: Global gMG Launch Underway
Global gMG Launch Underway VYVGART Approved December 17, 2021 United States Japan China (Zai Lab) Middle East (GenPharm) Approved Anticipated filing January 20, 2022 in mid-2022 Europe Israel (Medison) Canada Anticipated decision Anticipated filing Global in 2H 2022 in 2Q 2022 9
Slide 10: Listening to and Learning from MG Community
Listening to and Learning from MG Community 10
Slide 11: Differentiated Treatment Options for gMG Patients
Differentiated Treatment Options for gMG Patients SC efgartigimod ADAPT-NXT single injection Continuous Dosing Future Delivery Vehicles and Formulations (e.g., pre-filled syringe, Elektrofi, autoinjector) Eri, living with gMG 11 11
Slide 12: Autoimmune Pipeline
Autoimmune Pipeline Program Indication Preclinical Phase 1 Proof of Concept Registrational Commercial Update VYVGART gMG US Launch Ongoing gMG Submit BLA by end of 2022 CIDP Data 1Q23 Myositis Trial to initiate 1Q22 Pemphigus Timeline under review Bullous Pemphigoid Enrollment Ongoing Efgartigimod I
Slide 13: 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 14: ARGX-117: Sweeping Antibody Designed to Target Unique Intervention in Complement
ARGX-117: Sweeping Antibody ARGX-117 Blocks C2 Designed to Target Unique Classical Lectin IgG C1 qrs IgM Mannose sugar MAS Ps MBL Intervention in Complement C4 C2 C3 convertase C3a C3 C3b Targeting C2: C5 convertase • Leaves alternative pathway intact against infection C5a C5 • Blocks all complement
Slide 15: 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 16: 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 17: One Team, One Plan: Engineer potentially life-changing immunology
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. 17
Slide 18: 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 19: ADAPT-SC Bridging Study Design
ADAPT-SC Bridging Study Design 110 adult gMG patients Patients randomized 1:1 to receive MGFA Class II, III, IV 1000 mg SC efgartigimod or VYVGART Anti-AChR antibody positive All patients receive one (n=91) and negative (n=19) treatment cycle DESIGN MG-ADL score ≥5a (4 weekly single injections or 1-
Slide 20: Primary Endpoint Met: SC Efgartigimod Noninferior to IV
Primary Endpoint Met: SC Efgartigimod Noninferior to IV Primary endpoint of noninferiority met [p<0.0001] Mean total IgG reduction from baseline at day 29 IgG reduction (%) in all ADAPT-SC and ADAPT participants 66.4% 62.2% SC efgartigimod VYVGART ADAPT-SC AChR-Ab, acetylcholine receptor antibody; M
Slide 21: 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 22: 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 23: 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 24: 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 25: 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 26: 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 27: 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 28: 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 29: 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 30: 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 31: 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 32: 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 33: 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 33
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