Argenx Se · EURONEXT:ARGX
Argenx — Investor Presentation (June 2021)
27 slides from Argenx's investor presentation, June 2021
Slide 1: Together We Discover
Together We Discover Reaching Patients Through Immunology Innovation Corporate Presentation JUNE 2021 1
Slide 2: Achieving 'argenx 2021' Vision
Achieving ‘argenx 2021’ Vision Commercial franchises argenx 2021: Reaching patients Global expansion FcRn leadership, 4/4 POC MG CIDP ITP PV Late-stage pipeline ARGX-117 pipeline-in-a-product opportunity MMN Cusatuzumab strategic alliance Immunology breakthroughs Immunology Innovation Program Strong
Slide 3: Demonstrated Execution Across Business
Demonstrated Execution Across Business MG BLA 5 Global Growing Expanded Building Accepted Efgartigimod Autoimmune Discovery The Right Team Trials Ongoing Pipeline Capabilities 3
Slide 4: Uniquely Positioned
Zai collaboration 6th Indications Kidney 5th Uniquely Therapeutic 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 Biology is Foundational to the Immune System
FcRn Biology is Foundational to the Immune System FcRn recycles IgG antibodies extending their Efgartigimod Blocks FcRn leading abundancy to IgG elimination Endothelial Cell • Human IgG1 Fc fragment uniquely modulates FcRn, preserving characteristic pH dependent binding of endogenous IgG Endosome •
Slide 6: Efgartigimod: Broad Pipeline Opportunity
Efgartigimod: Broad Pipeline Opportunity Landscape of Solid Biology Feasible for argenx Franchises & Indications IgG-mediated Severe Rationale: Biotech: Efgartigimod to date achieved proof-of-concept in 4/4 Predominantly Orphan indication, indications; 2/2 in neuromuscular franchise Autoimmune Disea
Slide 7: Deep Antibody Pipeline of Differentiated Candidates
Deep Antibody Pipeline of Differentiated Candidates Program Indication Preclinical Phase 1 Phase 2 Phase 3 Registration Partner IV MG SC MG IV ITP SC ITP Efgartigimod SC PV SC CIDP SC Fifth Indication SC Sixth Indication + AZA Newly diag. AML (unfit) CULMINATE Janssen Cusatuzumab + AZA + VEN Newly d
Slide 8: Efgartigimod: First-in-Class FcRn Antagonist
Efgartigimod: First-in-Class FcRn Antagonist ERI Living with MG • Proof-of-concept in four indications (MG, ITP, PV, CIDP) Patients • IV and SC injection in on drug for development more than • 400+ subjects or patients dosed 2 years CHRIS • Safety profile comparable Living with MG to placebo in ADAP
Slide 9: Patient Experience
MYASTHENIA GRAVIS “A person with MG on a good Patient day operates at 70%. On bad days, you can get 10% out of Experience your battery.” 1/2 2.6 of Patients Years have been diagnosed Symptoms can vary with depression from patient to patient, day to mean time from or anxiety in day, or even throughou
Slide 10: Promising Value Proposition to MG Patients
Promising Value Proposition to MG Patients 78 % 60 % OVER Response Rate 84 % Deep Responses 50 % Supportive of Fast Onset Individualized of Action Dosing MG-ADL responders during first two MG-ADL responders within first two MG-ADL responders achieved minimal Patients likely cycles weeks of treatment
Slide 11: SC Bridging Strategy Leverages Correlation Between Pharmacodynamic and Clinical Effect
SC Bridging Strategy Leverages Correlation Between Pharmacodynamic and Clinical Effect Established association of total IgG and MG-ADL Bridging study (n=50) underway to support following efgartigimod treatment registration of SC efgartigimod • Study designed to demonstrate non- inferiority of PD eff
Slide 12: 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 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: Efgartigimod Phase 3 Trial in Pemphigus
Efgartigimod Phase 3 Trial in Pemphigus - Focus on Potential to Drive 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 do
Slide 15: 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 15
Slide 16: 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 expected to be filed with EMA in second half of 2021 PDUFA date of December 17, 2021 Chi
Slide 17: Listening to and Learning from MG Community
Listening to and Learning from MG Community 17
Slide 18: Myasthenia Gravis Is Just The Beginning
MYASTHENIA GRAVIS Is Just The Beginning Myasthenia Immune Pemphigus Chronic Inflammatory Fifth Sixth Gravis Thrombocytopenia Demyelinating Indication Indication Polyneuropathy Key: NEURO HEME SKIN KIDNEY 18
Slide 19: Expanding Reach Within Immune System With ARGX-117
Expanding Reach Within Immune ARGX-117 System With ARGX-117 Innate Immune System Efgartigimod Epithelial barriers Adaptive Immune System Antibodies B lymphocytes (e.g. IgG) Phagocytes Dendritic cells T lymphocytes Effector T cells Complement NK cells 19
Slide 20: ARGX-117: Broad Opportunity By Targeting C2
ARGX-117: Broad Opportunity By Targeting C2 Unique Phase 1 Healthy Phase 2 Intervention Volunteer Data Indications IgG IgM Mannose sugar Expected Mid-2021 C1 MAS MBL qrs Ps C4 C2 Multifocal Motor Neuropathy C3 convertase SC and IV C3a C3 C3b Formulations Kidney Indications C5 convertase C5a C5 C5-C9
Slide 21: ARGX-117: Potential Dosing Optionality
ARGX-117: Potential Dosing Optionality Pharmacokinetics Pharmacodynamics Option exercised for C2 Half-life ARGX-117: 2-3 weeks Blocking for 2 months after repeat dosing Cynomolgus monkey data C2 levels cynomolgus monkey = 4x human 21
Slide 22: Feasibility: Orphan Potential & Economically Viable Indication
Feasibility: Orphan Potential & Economically Viable Indication Unmet need for new therapies that slow down progression of disease and reduce reliance on IVIg MMN Multifocal Motor Prevalence Diagnosis / Treatment Neuropathy Metrics “ALS patient that didn’t die” ~13,000 patients in the US Anti-GM1 IgM
Slide 23: Cusatuzumab Strategy
Cusatuzumab Strategy Newly diagnosed elderly AML patients who are unfit for intensive chemotherapy Phase 2 CULMINATE Trial Phase 1b ELEVATE Trial in Triple Cusatuzumab + Azacitidine 20 mg/kg Combination Go-forward dose selected cusatuzumab + CR CRc azacitidine CR Rates Endosome n=14 n=21 + venetocla
Slide 24: Immunology Innovation Program (IIP)
Core Strategy To Grow Our Pipeline Immunology Innovation Program (IIP) DISCOVERY Optimizing the collision of great minds BIOLOGY TECHNOLOGY Collaborators WHOLLY-OWNED PIPELINE ASSETS COMPANY OUT-LICENSING OR Efgartigimod, CREATION COLLABORATIONS 117, 118, 119 Dualyx Genor (109) Staten (116) Leo (112
Slide 25: Leading Antibody Discovery and Engineering Toolkit
Leading Antibody V-Region Capabilities SIMPLE Discovery and AntibodyTM Platform Engineering Toolkit Fc Engineering to Optimize Antibodies NHANCE® DHS Mutations SC Dosing Optionality ENHANZE® Technology ACT-Ig® POTELLIGENT® SMART-Ig® ABDEGTM 25
Slide 26: 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 26
Slide 27: Together We Discover
Together We Discover Reaching Patients Through Immunology Innovation 27