Argenx Se · EURONEXT:ARGX
Argenx — JP Morgan Healthcare Conference January 2020
27 slides from Argenx's conference presentation, Jan 13, 2020
Slide 1: 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 2: argenx Today: Late-Stage Biotech Building Towards Commercial Success
argenx Today: Late-Stage Biotech Building Towards Commercial Success 3 Late-Stage Pipeline PV Immunology Breakthroughs CIDP Two new pipeline assets from IAP argenx 2021: Reaching Patients Therapeutic franchises FcRn leadership ITP MG Cusatuzumab strategic alliance New Data Global expansion ADAPT ful
Slide 3: argenx 2021: Growing Franchises With Multiple Late-Stage Programs
argenx 2021: Growing Franchises With Multiple Late-Stage Programs 4 ARGX-117 Phase 1 MG Neuro-muscular CIDP SC Hem/Onc CULMINATE Ven-Cusa-Aza ITP AML 5 LAUNCHES IN 5 YEARS Advancing to Phase 3 on positive Phase 2 data Severe autoimmune conditions Kidney Skin PV Kidney GOAL OF
Slide 4: 2020 View Of Pipeline: Poised To Have Five Phase 3 Trials Underway
2020 View Of Pipeline: Poised To Have Five Phase 3 Trials Underway 5 Neuro-muscular Hem-Onc Skin PRECLINICAL PHASE 1 PHASE 2 PHASE 3 ARGX-119 ARGX-118 IAP ARGX-117 Cusatuzumab+ AZA+Ven AML Cusatuzumab High Risk MDS Cusatuzumab Platform AML Efgartigimod 5th indication Cusatuzumab +AZA Newly diagnosed
Slide 5: Building Deep Antibody Pipeline Of Differentiated Candidates
Building Deep Antibody Pipeline Of Differentiated Candidates 6 PROGRAM FIRST-IN-CLASS TARGET INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 BLA MARKETED Efgartigimod IV FcRn MG Efgartigimod SC Bridging FcRn MG Efgartigimod IV FcRn ITP Efgartigimod IV + SC FcRn ITP Efgartigimod IV FcRn ITP Efgartigim
Slide 6: Therapeutic Franchises Sit In High-Value Rapid-Growth Markets
Therapeutic Franchises Sit In High-Value Rapid-Growth Markets 7 Neuromuscular >$5B (CAGR ~10%+) Hem/Onc ~$7B (CAGR ~10%) Skin ~370,000 patients > 600,000 patients BP PV Other AMR IgAN MN Other DM BP: Bullous pemphigoid DM: Dermatomyositis IgAN: IgA nephropathy AMR: Antibody-mediated rejection MN: Me
Slide 7: The Right Team In Place To Launch Efgartigimod
The Right Team In Place To Launch Efgartigimod 8 Commercial leaders hired across all key functions Field-based medical research liaisons in place Stepwise salesforce ramp-up COO leading commercial organization Significant product launch experience Preparing for Global Launch
Slide 8: Building Differentiation Every Step Of The Way
Building Differentiation Every Step Of The Way MyReal World™MG Molecule Design: Innovative Access Program Clinical Development: Thoughtful ADAPT Design Commercial Approach: Real-world Evidence Study 9
Slide 9: Efgartigimod: Unique Molecule Design Leads To Differentiated Profile in Phase 2
Efgartigimod: Unique Molecule Design Leads To Differentiated Profile in Phase 2 efgartigimod Antibody FcRn Efficacy 3/3 beachhead indications Safety No class effect Convenience Potential optionality for patients 10
Slide 10: Beachhead Strategy Based On Unifying Biologic Rationale
Beachhead Strategy Based On Unifying Biologic Rationale 11 Myasthenia Gravis Immune Thrombocytopenia Pemphigus Vulgaris Block acetylcholine receptors Cross-link + internalize acetylcholine receptors Recruit complement Enhance platelet clearance Kill platelets Inhibit platelet production Reduce plate
Slide 11: Phase 2 Proof-Of-Concept Supports Advancement To Phase 3
Phase 2 Proof-Of-Concept Supports Advancement To Phase 3 IgG Reduction Correlates With Clinical Improvements 12 10 mg/kg efgartigimod Mean platelet count (x109/L) ITP MG 10 mg/kg efgartigimod American Journal of Hematology Reduction of total and pathogenic IgGs led to clinically meaningful improveme
Slide 12: Phase 2 Proof-of-Concept In PV Supports Advancement To Registrational Trial
Phase 2 Proof-of-Concept In PV Supports Advancement To Registrational Trial 13 * Eligible for efficacy analysis Data cut off 7 Nov 2019; 8 patients on study at time of interim analysis Data show efgartigimod treatment phases with at least biweekly dosing ; excludes IgG4 for one patient (outlier) Wee
Slide 13: Efgartigimod In Pemphigus Vulgaris: Adaptive Phase 3 Trial
Efgartigimod In Pemphigus Vulgaris: Adaptive Phase 2 Trial 14 Monotherapy versus combination therapy with corticosteroids Treatment regimen: Dose (10mg/kg vs. 25 mg/kg) and frequency Corticosteroids: Standardization of dose and ability to taper
Slide 14: Efgartigimod Synergizes With Low-Dose Steroids In Pemphigus in Phase 2
Efgartigimod Synergizes With Low-Dose Steroids In Pemphigus in Phase 2 15 Fast onset of action 78% disease control (18/23 patients) – majority after 1-2 infusions Median time to DC: 14 to 15 days (mono/combo therapy) Deep responses 70% clinical remission (5/7 patients) on optimized dosing regimen* T
Slide 15: ADAPT Trial: Built For Patients Based On Strengths Of Efgartigimod
ADAPT Trial: Built For Patients Based On Strengths Of Efgartigimod 16 Fast onset of action Responded within first four weeks Clinical response in 83% of patients Durable response in 75% of patients Sustained for at least 6 weeks Promising tolerability and built on observed attributes of efgartigimod
Slide 16: Efgartigimod Has Potential To Offer Tailored Treatment Approach In MG
17 Current Standard of Care Goal of Efgartigimod Dosing Chronic dosing TAPER Patient-tailored dosing Fast-acting steroids and slow-acting immunosuppressants Balancing symptom suppression and side effects Tailored regimen matches variability of MG Time between cycles is individualized Period of susta
Slide 17: Innovative ADAPT Design: Clinical Trial Designed To Meet Clinical Practice
Innovative ADAPT Design: Clinical Trial Designed To Meet Clinical Practice 18 gMG patients (MG-ADL>5) Stratified for AChR+ or AChR- and background therapy (n=167 total) Primary endpoint (AChR+): % responders after first treatment cycle Responder: >2 ADL points for at least 4 consecutive weeks any ti
Slide 18: Efgartigimod Has Potential To Disrupt Current MG Treatment Paradigm
Efgartigimod Has Potential To Disrupt Current MG Treatment Paradigm 19 Steroids most common add-on ISTs used for steroid sparing Later agents used for severe/refractory/crisis ACIs (mestinon) at diagnosis Vision: Efgartigimod positioned to be used early and more broadly within existing paradigm Ster
Slide 19: MyRealWorld MG: Understanding Potential Value Proposition Of Efgartigimod
MyRealWorld™ MG: Understanding Potential Value Proposition Of Efgartigimod 20 Global prospective – longitudinal - observational Voice of >2000 patients - digitally Patient perspective on diagnosis, treatment, symptom, economic and humanistic burden First of its kind in MG
Slide 20: ITP Phase 3 ADVANCE: Evaluating IV + SC Maintenance Dosing
ITP Phase 3 ADVANCE: Evaluating IV + SC Maintenance Dosing Primary objective Maintenance of response of SC compared to placebo after initial response with IV Durable response: sustained platelet count (>50×109/L) 21 Up to 12 weekly doses 10mg/kg IV > 20 weeks SC Dosing cadence to depend on response
Slide 21: CIDP Phase 2 ADHERE: Potential For Development Acceleration
CIDP Phase 2 ADHERE: Potential For Development Acceleration 22 Template Run-in period Stage A Stage B (stage A responders only) Up to 12 weeks, until clinical improvement (ECI) <13weeks Treatment period Efficacy analysis based on relapse (adjusted INCAT) Study endpoint with 88 relapse events in stag
Slide 22: Cusatuzumab Strategic Alliance With Janssen
Cusatuzumab Strategic Alliance With Janssen 23 Leukemia cell Leukemia cell CD27 CD sCD Leukemia cell Leukemia cell CD27 CD70 Cusatuzumab sCD27 NK effector cell Blocking CD70-CD27 signalling Blocking release of soluble CD27 Killing cells Joint development plan focused on AML, MDS and other heme malig
Slide 23: ARGX-117: Sweeping Antibody Targeting C2
ARGX-117: Sweeping Antibody Targeting C2 24 Sweeping Antibody Classical Lectin MMN GBS CIDP Lupus Nephritis CAD IgAN AMR Ischemia reperfusion MGUS anti-MAG MN C4GN HSCT Option exercised for C2 C5 C5-C9 (MAC) C1 qrs C4 C2 C3a C3b C5a C3 convertase C5 convertase C3 Mannose sugar MBL MASPs IgG IgM Uniq
Slide 24: Innovative Access Program: Our Strategy To Grow Our Pipeline
Innovative Access Program: Our Strategy To Grow Our Pipeline 25 Academic Institutions & Biotechs argenx Accessing First-in-Class Targets by Collaborating with Leading Research Biologists Disease Biology Expertise Texas A&M, Bern, Utrecht, Louvain, Penn, Columbia, Torino, de Duve, VIB Antibody Expert
Slide 25: Our Key Priorities In 2020
Our Key Priorities In 2020 26 1 PREPARE FOR LAUNCH 2 EXECUTE PIPELINE: 5 Registrational and 7 PHASE 1-2 TRIALS 3 EXPAND THROUGH INNOVATIVE ACCESS PROGRAM
Slide 26: JP Morgan Healthcare Conference January 2020 – San Francisco
JP Morgan Healthcare Conference January 2020 – San Francisco
Slide 27: argenx | January 2020 - San Francisco
Vv a rge n » 4 e | January 2020 - San Francisco
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