Argenx SE · EURONEXT:ARGX
Argenx — JP Morgan Healthcare Conference 2021
24 slides from Argenx's conference presentation, January 2021
Slide 1: Together We Discover
Together We Discover Reaching Patients Through Immunology Innovation JP Morgan Healthcare Conference JANUARY 2021 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
Slide 3: Reaching People Living with MG in 2021
Reaching People Living with MG in 2021 MORGAN, Living with MG 3
Slide 4: 2020: Transformational Year for argenx
2020: Transformational Year for argenx MG BLA 5 Global Growing Expanded Building Submitted Efgartigimod Autoimmune Discovery The Right Team Trials Ongoing Pipeline Capabilities 4
Slide 5: 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 5
Slide 6: Reaching Patients Worldwide
Reaching Patients Worldwide + Expand Accelerate Mutual Passion patients we reach + enrollment into global bringing innovative number of indications registrational trials therapies to patients 6
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: 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 9: Efgartigimod: First-in-Class FcRn Antagonist
Efgartigimod: First-in-Class FcRn Antagonist ERI Living with MG • Proof-of-concept in three indications (MG, ITP, PV) Patients • IV and SC injection in development on drug for • 350+ subjects or patients more than dosed 2 years • Safety profile comparable CHRIS to placebo in ADAPT trial Living with
Slide 10: 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 11: 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 12: 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 13: 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 13
Slide 14: Listening to and Learning from MG Community
Listening to and Learning from MG Community 14
Slide 15: 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 15
Slide 16: 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 16
Slide 17: 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 18: 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 19: 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 20: 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 20
Slide 21: 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 21
Slide 22: Together We Discover
Together We Discover Reaching Patients Through Immunology Innovation JP Morgan Healthcare Conference JANUARY 2021 22
Slide 23: ADAPT Data Showed Fast, Deep and Durable Responses
ADAPT Data Showed Fast, Deep and Durable Responses 40% of Efgartigimod Patients Minimal Symptom Durable Clinical Efgartigimod Demonstrated Significant Achieved Minimal Symptom Expression Expression Potential for Individualized Benefit Magnitude Of Benefit Compared to 11% in Minimal Symptom Expressio
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 53% vs placebo 21% Immuno IA with tryptophan matrix after 24 weeks (N=117) adsorption 80-100% IA wit