Argenx Se · EURONEXT:ARGX
Argenx — Investor Presentation (October 2021)
25 slides from Argenx's investor presentation, October 2021
Slide 1: Together We Discover
Together We Discover Reaching Patients Through Immunology Innovation Corporate Presentation OCTOBER 2021 1
Slide 2: Building a Leading Immunology Company
Building a Leading Immunology Company Global launch preparations underway in U.S., Japan and EU Efgartigimod proof-of-concept in 4/4 indications with ambition to be in 15 by 2025 Committed to our Patients and their Communities Growing neuromuscular, hematology and skin commercial franchises Enviable
Slide 3: Deep Antibody Pipeline of Differentiated Candidates
Deep Antibody Pipeline of Differentiated Candidates Program Indication Preclinical Phase 1 Phase 2 Phase 3 Registration Update IV MG SC MG Data 1H 2022 IV ITP Data 1H 2022 SC ITP Efgartigimod SC PV SC CIDP SC Myositis SC Bullous Pemphigoid (BP) IV + SC MMN ARGX-117 IV + SC Kidney Diseases + AZA Newl
Slide 4: Uniquely Positioned
Zai collaboration BP Indications Myositis Uniquely Therapeutic Kidney Positioned Franchises Skin For exponential expansion ROW CIDP China Heme o efgartigimod indications PV o therapeutic franchises o global markets Global Europe Markets Neuro ITP Japan MG US 4
Slide 5: FcRn Plays a Key Role in IgG and Albumin Homeostasis
FcRn Plays a Key Role in IgG and Albumin Homeostasis Studies have shown: Endothelial Cell Circulating antibodies are taken up in the cell via pinocytosis. In the 1 endosome, IgG antibodies bind to 3 FcRn1,2 1 IgG Autoantibody Unbound IgGs enter the lysosomal 2 degradation pathway, while FcRn-bound 1
Slide 6: Efgartigimod: Broad Pipeline Opportunity
Efgartigimod: Broad Pipeline Opportunity IgG-mediated Severe Solid Biology Feasible for argenx Franchises & Indications Autoimmune Diseases Rationale Biotech Proof-of-concept in 4/4 indications; 2/2 in neuromuscular franchise Immune Lupus Myasthenia Gravis Thrombocytopenia Neuromuscular Chronic Infl
Slide 7: Efgartigimod: Data Support Favorable Benefit to Risk Ratio
Efgartigimod: Data Support Favorable Benefit to Risk Ratio 600+ subjects dosed Clinical proof-of-concept in No evidence of dose-limiting 125+ four indications toxicities in healthy volunteers or patients on efgartigimod for (MG, ITP, PV, CIDP) patients across trials over 12 months Opportunity to dos
Slide 8: Promising Value Proposition to MG Patients
Promising Value Proposition to MG Patients 79 % 60 % 89% 84 Response % MSE Rate 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 symptom expr
Slide 9: Preparing for a Successful Launch
Preparing for a Successful Launch Physician Payor Building Manufacturing Education Engagement the Team Commitment Reach the 20,000 addressable gMG patients 9
Slide 10: Efgartigimod Regulatory Update
Efgartigimod Regulatory Update United States Global Japan J-MAA for IV efgartigimod for treatment of gMG accepted for review by PMDA BLA for IV efgartigimod for treatment of gMG accepted for review by FDA EU MAA filed with EMA and validated PDUFA date of December 17, 2021 China Zai Lab to discuss po
Slide 11: Listening to and Learning from MG Community
Listening to and Learning from MG Community 11
Slide 12: Myasthenia Gravis Is Just The Beginning
MYASTHENIA GRAVIS Is Just The Beginning Myasthenia Immune Pemphigus Chronic Inflammatory Myositis Bullous Pemphigoid Gravis Thrombocytopenia Demyelinating Polyneuropathy Key: NEURO HEME SKIN KIDNEY 12
Slide 13: ITP Phase 3 ADVANCE: Two Trials Run in Parallel
ITP Phase 3 ADVANCE: Two Trials Run in Parallel Phase 3, multicenter, randomized, double-blind, placebo-controlled trial N=156 Primary objective 24 weeks 10mg/kg IV efgartigimod Fixed weekly Fixed weekly or Weekly or q2w dosing q2w dosing dosing adjusted according to platelet count Durable response:
Slide 14: CIDP: Phase 2/3 ADHERE Trial
Chronic Inflammatory Demyelinating Polyneuropathy: Phase 2/3 ADHERE Trial Assess Confirm IgG autoantibody efficacy & safety involvement efgartigimod vs placebo Treatment period Identify patients with active CIDP Open-label Placebo-controlled Efficacy analysis Stage B based on relapse Screening Run-i
Slide 15: Phase 3 Trial in Pemphigus: Focus on Fast Onset and Steroid-sparing
Phase 3 Trial in Pemphigus: Focus on Fast Onset and Steroid-sparing Concomitant Screening prednisone Randomization (2x1) Primary endpoint is proportion of PV patients achieving CRmin* within 30 Pemphigus vulgaris weeks (PV) and foliaceus (PF) • Prednisone starting dose Efgartigimod weekly SC Moderat
Slide 16: Myositis: IgG-Mediated Biology
Myositis: IgG-Mediated Biology + AChR, MuSK, LRP4 Neuromuscular Disease Myasthenia Gravis autoantibodies comprise ADAPT Data ~90% of MG patients + Chronic Inflammatory 40% anti-myelinated ADHERE Neuromuscular Disease Demyelinating peripheral nerve IgGs GO/NO-GO Polyneuropathy + Autoantibodies charac
Slide 17: 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 18: ARGX-117 is First in Class Targeting C2
ARGX-117 is First in Class Targeting C2 (Auto)antibodies Microorganisms Classical Lectin Alternative IgG IgM Mannose sugar Foreign surface C1 MBL C3 qrs C4 MASPs C2 CFB CFD C3 convertase C3b C3 C3a C3a-R Clearance by macrophages C5 convertase C5 C5a C5a-R Inflammation C5b-C9 Membrane damage MAC Subl
Slide 19: 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 19
Slide 20: 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 21: Phase 2 Multifocal Motor Neuropathy Trial Design
Phase 2 Multifocal Motor Neuropathy Trial Design Patient Population 4-10 weeks Total Duration: 16 weeks Key Outcome Measures Trial to enroll Safety and tolerability approximately 45 MMN ARGX-117 Dose patients Time to IVIg re-treatment Randomization (1:1:1) Regimen 1 (N=15) • Probable or definite MMN
Slide 22: Neuromuscular Franchise: A Company within a Company
Neuromuscular Franchise: A Company within a Company SMA MMN IIP Programs ALS MG Myositis Congenital MG CIDP ARGX-117 C2 inhibitor ARGX-119 MuSK MG SIMPLE Antibody™ aimed to boost Efgartigimod the neuromuscular junction in disease FcRn antagonist MG-SC CIDP Myositis Leveraging infrastructure across m
Slide 23: Our Pipeline Starts with our Immunology Innovation Program
Our Pipeline Starts with our Immunology Innovation Program CLINICAL DEVELOPMENT LEADING TRANSLATIONAL BIOLOGY LABS ANTIBODY ENGINEERING Internal Value Creation External Value Creation Staten AgomAb First in Class | Unique Design | Multiple Indications ARGX-118 Dualyx (ARGX-116) (ARGX-114) Genor LEO
Slide 24: Strategic Priorities
Building Tomorrow’s Immunology Company Reach gMG patients with efgartigimod Global expansion Strategic Advance clinical development in multiple Priorities Leverage IIP autoimmune indications Rooted in groundbreaking immunology research, growing through collaboration 24
Slide 25: Together We Discover
Together We Discover Reaching Patients Through Immunology Innovation 25