Argenx Se · EURONEXT:ARGX
Argenx — Investor Presentation (May 2020)
38 slides from Argenx's investor presentation, May 2020
Slide 1: Corporate Presentation May 2020
Corporate Presentation May 2020
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: Navigating The Impact Of COVID-19
Navigating The Impact Of COVID-19 Our Ensuring alignment across our team People Supporting each other in new environment Our Adapting for our patients Implementing opportunities for home infusions Patients and telehealth visits Focused on continuity Our ADAPT readout on track for mid-2020 and BLA Bu
Slide 4: Late-stage Biotech Building Towards Commercial Success
Late-stage Biotech Building Towards Commercial Success Therapeutic franchises argenx 2021: Reaching patients Global expansion FcRn leadership MG CIDP ITP PV Late-stage pipeline Cusatuzumab strategic alliance Immunology breakthroughs Two new pipeline assets from IIP Strong balance sheet 1.3B€ in cash
Slide 5: Growing Franchises With Multiple Late-Stage Programs
Growing Franchises With Multiple Late-Stage Programs Skin Neuro- Hem/Onc muscular Kidney Kidney PV MG ITP SC Advancing to Phase 3 on positive Phase 2 data CIDP Kidney AML Severe autoimmune ARGX-117 conditions MDS GOAL OF 5 LAUNCHES IN 5 YEARS 5
Slide 6: 2020 View Of Pipeline: Poised To Have Five Phase 3 Trials Underway
2020 View Of Pipeline: Poised To Have Five Phase 3 Trials Underway Efgartigimod PV Efgartigimod SC MG (Bridging) Efgartigimod IV MG Cusatuzumab Immunology Efgartigimod SC Efgartigimod IV + AZA Innovation Program CIDP ITP (Higher-risk MDS) Hem- Onc Cusatuzumab Cusatuzumab ARGX-118 Efgartigimod IV + S
Slide 7: Deep Antibody Pipeline Of Differentiated Candidates
Deep Antibody Pipeline Of Differentiated Candidates Program Target Indication Preclinical Phase 1 Phase 2 Phase 3 Partner IV MG SC Bridging MG Efgartigimod IV ITP Initiated 4Q19 IV + SC ITP m FcRn IV ITP IV PV SC CIDP Initiated 4Q19 TBD 5th Indication Newly diag. AML (unfit) Cusatuzumab + AZA CULMIN
Slide 8: Franchises Sit In High-Value Rapid-Growth Markets
Franchises Sit In High-Value Rapid-Growth Markets Neuromuscular Hem/Onc Skin / Kidney >$5B (CAGR ~10%+) ~$7B (CAGR ~10%) MMN PV IgAN MDS MG ITP BP AMR CIDP DM MN AML Other Other ~370,000 patients > 600,000 patients IgAN: IgA nephropathy BP: Bullous pemphigoid AMR: Antibody-mediated rejection 8 argen
Slide 9: The Right Team In Place To Launch Efgartigimod
The Right Team In Place To Launch Efgartigimod COO leading commercial Commercial leaders hired across all key Field-based medical research Stepwise salesforce ramp-up organization functions liaisons in place Preparing for Significant product launch experience global launch USA JAPAN EU 9
Slide 10: Building Innovation At Every Step
Building Innovation At Every Step Preclinical / Discovery Clinical Commercial Molecule Adapt MyReal My Real Image World MG World™ ™ MG Molecule Design: Clinical Development: Commercial Approach: Immunology Innovation Thoughtful ADAPT Design Real-World Evidence Program Study 10
Slide 11: Efgartigimod: Unique Molecule Design Leads To Differentiated Profile In Phase 2
Efgartigimod: Unique Molecule Design Leads To Differentiated Profile In Phase 2 Efficacy 3/3 beachhead indications Convenience Safety Potential optionality for patients No class effect IgG Efgartigimod FcRn Antibody 11
Slide 12: Beachhead Strategy Based On Unifying Biologic Rationale
Beachhead Strategy Based On Unifying Biologic Rationale Neuromuscular Hem/Onc Skin Chronic Inflammatory Myasthenia Gravis Immune Thrombocytopenia Pemphigus Vulgaris Demyelinating Polyneuropathy Skin and mucous membrane Macrophage Complement Auto-antibodies Myelinated Nerve Basement membrane Potentia
Slide 13: MG and ITP Phase 2 Data Support Registrational Plan
MG and ITP Phase 2 Data Support Registrational Plan MG 10 mg/kg efgartigimod ITP 10 mg/kg efgartigimod American Journal of Hematology Mean platelet count (x109/L) • Reduction of total and pathogenic IgGs led to clinically meaningful improvements in disease scores (MG-ADL, QMG, QoL and Composite for
Slide 14: PV Phase 2 Data Support Registrational Plan
PV Phase 2 Data Support Registrational Plan Baseline Characteristics IgG Reduction Correlated to PDAI Score Improvement in Responders Pemphigus Vulgaris subtype PDAI activity PDAI Mucosal-dominant (N = 8) 100 lgG4 IgG4 from baseline Mucocutaneous (N = 10) baseline Cutaneous (N = 1) Dsg-1 Dsg-1 Pemph
Slide 15: Adaptive Phase 2 Trial Of Efgartigimod In PV
Adaptive Phase 2 Trial Of Efgartigimod In PV Monotherapy Treatment Corticosteroids: versus regimen: Standardization combination Dose (10mg/kg of dose and therapy with vs. 25 mg/kg) ability to taper corticosteroids and frequency 15
Slide 16: Synergy With Low-Dose Steroids In PV Phase 2
Synergy With Low-Dose Steroids In PV Phase 2 78% disease control (18/23 patients) – majority after 1-2 infusions Fast onset of action Median time to DC: 14 to 15 days (mono/combo therapy) 70% complete clinical remission (5/7 patients) on optimized dosing* Deep responses Time to CR: 2-10 weeks Mean m
Slide 17: ADAPT Trial: Built For MG Patients Based On Strengths Of Efgartigimod
ADAPT Trial: Built For MG Patients Based On Strengths Of Efgartigimod We listened to stakeholders… …and built on observed attributes of efgartigimod Phase 2 MG data: Fast onset of action Tailored Convenient • Responded within first four weeks Clinical response in 83% of patients Durable response in
Slide 18: Potential To Offer Tailored Treatment Approach In MG
Potential To Offer Tailored Treatment Approach In MG Current Standard of Care Goal of Efgartigimod Dosing Typical MG Toward minimal Taper Symptoms symptom expression Chronic dosing Patient-tailored dosing • Fast-acting steroids and slow-acting • Tailored regimen matches variability of MG immunosuppr
Slide 19: Innovative ADAPT Trial Designed To Meet Clinical Practice
Innovative ADAPT Trial Designed To Meet Clinical Practice Patient population Open-label Extension Primary endpoint readout at week 8 consistent with Retreat as needed to simulate clinical Duration of benefit measured over 26 weeks Phase 2 practice Up to 2 First treatment cycle retreatment cycles 10m
Slide 20: Potential To Disrupt Current MG Treatment Paradigm
Potential To Disrupt Current MG Treatment Paradigm Vision: Efgartigimod positioned to be used early and more broadly within existing paradigm efgartigimod Delay or Steroids IVIg Eliminate ISTs ACIs Taper Ritux Soliris Current MG Treatment Paradigm ACIs (mestinon) Steroids most common ISTs used for s
Slide 21: Addressable MG Market: Patients Who Need Therapy Beyond Steroids
Addressable MG Market: Patients Who Need Therapy Beyond Steroids 65,000 adult myasthenia gravis patients in 85% U.S. of MG patients have generalized form of disease 55,000 patients have generalized form of myasthenia gravis 36% 20,000 patients require of patients require treatment more aggressive be
Slide 22: MyRealWorld MG: Understanding Potential Value Proposition Of Efgartigimod
MyRealWorld™ MG: Understanding Potential Value Proposition Of Efgartigimod First of its kind in MG Global prospective – Patient perspective on Voice of ≥ 2000 longitudinal - diagnosis, treatment, patients - digitally observational symptom, economic and humanistic burden 22
Slide 23: ITP Phase 3 ADVANCE: Evaluating IV + SC Maintenance Dosing
ITP Phase 3 ADVANCE: Evaluating IV + SC Maintenance Dosing Trial Primary objective N=156 Durable response: * 24 weeks 10mg/kg IV (potential to adjust frequency upon response) sustained platelet count (≥50×109/L) n≥50 Patients with primary Cumulative duration of ITP with platelet * 24 weeks 10mg/kg I
Slide 24: CIDP Phase 2 ADHERE: Potential For Development Acceleration
CIDP Phase 2 ADHERE: Potential For Development Acceleration Confirm IgG Document autoantibody efficacy & safety involvement efgartigimod vs placebo Identify patients with active CIDP Treatment period Open-label Placebo-controlled Efficacy analysis based on relapse Screening Run-in period Stage A Sta
Slide 25: Cusatuzumab Strategic Alliance With Janssen
Cusatuzumab Strategic Alliance With Janssen Blocking CD70-CD27 signalling • Joint development plan focused on AML, MDS and Blocking release of soluble CD27 other heme malignancies NK • Upfront $300M + $200M equity @ 20% premium, effector up to $1.3B in milestones, double digit royalties OUS cell Cus
Slide 26: ARGX-117: Sweeping Antibody Targeting C2
ARGX-117: Sweeping Antibody Targeting C2 Unique Intervention in Showcase of Antibody Pipeline-in-a-Product Complement Cascade Engineering Capabilities Potential IgG IgM Mannose sugar Sweeping Antibody C1 MASPs MBL qrs MMN C4 Lupus Nephritis C2 CAD Classical C3 convertase Endosome GBS Ischemia CIDP C
Slide 27: Immunology Innovation Program
Immunology Innovation Program Accessing First-in-Class Targets by Collaborating with Leading Research Biologists argenx Academic Institutions & Biotechs Antibody Expertise Disease Biology Expertise Co-creating immunology solutions: building beyond each individual contribution ARGX-112 ARGX-117 ARGX-
Slide 28: Our Key Priorities In 2020
Our Key Priorities In 2020 1 Prepare for launch Execute pipeline: 2 5 registrational and 7 Phase 1-2 trials 3 Expand through Immunology Innovation Program 28
Slide 29: Thank You
Thank You visit argenx.com
Slide 30: Efgartigimod: Human IgG1 Fc Fragment With Proprietary ABDEG Mutations
Efgartigimod: Human IgG1 Fc Fragment With Proprietary ABDEGTM Mutations efgartigimod potently Leading to IgG antibodies recycle IgG elimination through FcRn… blocks FcRn… Endothelial Cell ABDEGTM Endosome MST HN Lysosome IgG Antibody FcRn IgG Antibody FcRn Efgartigimod 30
Slide 31: Efgartigimod Portfolio Comprised Of Multiple Formulations
Efgartigimod Portfolio Comprised Of Multiple Formulations Standalone Products (Built to be Interchangeable) + 10 mg/kg 1000mg fixed 10 mg/kg 360 mg fixed (2ml) IV Efgartigimod ENHANZE® Efgartigimod SC IV Efgartigimod + SC Efgartigimod Induction Maintenance MG Strategy IV infusion induction 60-minute
Slide 32: gMG Phase 2: Sustained Improvement In 75% Of Treated Patients
gMG Phase 2: Sustained Improvement In 75% Of Treated Patients 75% of efgartigimod group showed 6/12 (50%) efgartigimod group had ongoing clinically meaningful improvement in improvement at end of study MG-ADL for at least 6 consecutive weeks Duration compared to 25% of placebo group (weeks) Onset ≥
Slide 33: gMG Phase 2: Proportion Of Patients With Increasing Threshold Of Benefit
gMG Phase 2: Proportion Of Patients With Increasing Threshold Of Benefit -10 0% 0% -12 8% 0% -9 efgartigimod 0% 0% Placebo -11 efgartigimod 8% 0% Placebo Change from Baseline Change from Baseline -8 n=12 17% 0% n=12 -10 n=12 17% 0% n=11* -9 -7 25% 8% 25% 0% -8 -6 25% 17% 25% 9% -7 -5 42% 25% 42% 18%
Slide 34: ITP Phase 2: Improvement Of Platelet Counts Across Doses
ITP Phase 2: Improvement Of Platelet Counts Across Doses 46-67% of patients achieving platelet counts of ≥ 50×109/L at least two times • OLE acts as fourth cohort % of patients with an improvement of platelet counts since patients’ platelets Main Study OLE (1st treatment cycle) had to fall below 30x
Slide 35: CIDP: Therapeutic Efficacy Shown With Increasing Selectivity For IgG Reductions
CIDP: Therapeutic Efficacy Shown With Increasing Selectivity For IgG Reductions Selectivity for IgG MoA Response rate IVIg Several/undefined MoA, including increased catabolism of IgGs 50-70% PLEX Removal of humoral serum factors 33-80% Immunoadsorption (IA) with Immuno tryptophan matrix: IgG, IgM,
Slide 36: C2 At Intersection Of Classical And Lectin Pathways
C2 At Intersection Of Classical And Lectin Pathways 4 Lectin pathway implicated in disease Role still being revealed Classical Lectin Alternative IgG IgM Mannose sugar Foreign surface 5 3 C1 deficiency lupus C1 Intact alternative pathway reduced MBL C3 qrs Lectin not blocked infection risk 7 • C
Slide 37: ARGX-117: Sweeping Antibody To Eliminate Serum C2
ARGX-117: Sweeping Antibody To Eliminate Serum C2 ARGX-117 remains bound ARGX-117 binds going back into circulation to 1 4 C2 in circulation bind to new C2 because of NHance® mutations ARGX-117/C2 complex taken up by pinocytosis entering endosome; 2 3 C2 is degraded in lysosome ARGX-117 releases C2
Slide 38: 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 38