Argenx Se · EURONEXT:ARGX
Argenx — FY2020 Earnings Presentation
20 slides from Argenx's earnings presentation, March 2021
Slide 1: Together We Discover
Together We Discover Reaching Patients Through Immunology Innovation Full Year and Fourth Quarter 2020 Financial Results MARCH 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: 2020: Transformational Year for argenx
2020: Transformational Year for argenx MG BLA 5 Global Growing Expanded Building Accepted Efgartigimod Autoimmune Discovery The Right Team Trials Ongoing Pipeline Capabilities 3
Slide 4: 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 5: 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 • 350+ subjects or patients dosed 2 years CHRIS • Safety profile comparable Living with MG to placebo in ADAP
Slide 6: 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 7: 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 8: Chronic Inflammatory Demyelinating Polyneuropathy: 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 9: 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 10: 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 11: 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 12: 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 13: 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 14: 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 15: Full Year 2020 Financial Results
Full Year 2020 Financial Results Year Ended December 31, (in thousands of € except for shares and EPS) 2020 2019 Variance Revenue € 36,425 € 69,783 € (33,358) Other operating income 18,109 12,801 5,308 Total operating income 54,534 82,584 (28,050) Research and development expenses (325,479) (197,665
Slide 16: Listening to and Learning from MG Community
Listening to and Learning from MG Community 16
Slide 17: Efgartigimod Regulatory Update
Efgartigimod Regulatory Update United States Global Japan J-MAA expected to be filed with PMDA in first half of 2021 BLA for IV efgartigimod for treatment of EU gMG accepted for review by FDA MAA expected to be filed with EMA in second half of 2021 PDUFA date of December 17, 2021 China Zai Lab Limit
Slide 18: 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 18
Slide 19: 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 19
Slide 20: 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 20