Argenx Se · EURONEXT:ARGX
Argenx — Corporate Update (February 2021)
14 slides from Argenx's corporate update, February 2021
Slide 1: Together We Discover: "GO" Decision: ADHERE Trial in CIDP
Together We Discover Reaching Patients Through Immunology Innovation “GO” Decision: ADHERE Trial in CIDP FEBRUARY 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: 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 4: CIDP: Significant Unmet Need Exists for Patients
CIDP: Significant Unmet Need Exists for Patients Diagnosis/Metrics Commonly misdiagnosed Rare, Chronic, and Diagnosis often confirmed by Progressive physicians trying therapy for 3 months and reassessing Symmetric proximal and distal weakness with sensory loss and decreased reflexes Can progress qui
Slide 5: Solid Biology Rationale for FcRn Approach in CIDP
Solid Biology Rationale for FcRn Approach in CIDP CIDP Pathophysiology Clinical Evidence Preclinical Evidence Nerve-reactive IgGs Clinical response with in CIDP patients Cellular and humoral treatments selective immunity components for IgGs Studies show pathogenic nature of patient-derived IgGs 5
Slide 6: 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 54% vs placebo 21% Immuno IA with tryptophan matrix after 24 weeks (N=117) adsorption 80-100% IA wit
Slide 7: Passive Transfer Studies Show Pathogenic Nature of Patient IgGs
Passive Transfer Studies Show Pathogenic Nature of Patient IgGs Purified IgG from CIDP patients with immunoreactivity towards myelinated nerves Purified IgG from PLEX-responsive CIDP patients Exacerbation of EAN disease model CIDP-specific reduction in nerve conduction by injection of CIDP IgG speed
Slide 8: Nerve-reactive IgGs in CIDP patients
Nerve-reactive IgGs in CIDP patients Anti-myelin sheath IgG Anti-paranodal IgG4 30-40% of patients ~10% of patients Axon Node of Ranvier Neuron Autoantibodies (unknown antigens) Node of Myelin Sheath Ranvier Paranodal proteins: eg. NF155, contactin-1 Nodal proteins: eg. NF186, gliomedin Querol et al
Slide 9: 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 10: Clear "GO" Based on Observed Activity in CIDP
Clear “GO” Based on Observed Activity in CIDP Pre-defined analysis had three potential outcomes: NO GO, REVIEW or GO based on assessment relative to predefined hurdle Proportion of patients qualifying for Stage B Responder Curve Lower 90% one-sided Clopper Pearson confidence interval GO: Responder r
Slide 11: Global Immunoglobulins Market: CIDP represents share of total IVIg
Global Immunoglobulins Market $12.8Bn 30% 28% 25% 24% 24% CIDP represents 21% 20% 24% 16% of total IVIg 15% market by volume 10% 6% and growing 5% 5% double digits 0% PID CIDP CIDP Other SID ITP MG Grifols 2019, CSL 2020 11
Slide 12: Key Conclusions
Key Conclusions • GO decision to continue ADHERE • Efgartigimod achieved 4/4 in proof-of- enrollment in support of potential concept indications; 2/2 in registration of efgartigimod in CIDP neuromuscular franchise • Decision supported by independent data • Current global IVIg sales exceeds $3B monit
Slide 13: 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 13
Slide 14: Together We Discover
Together We Discover Reaching Patients Through Immunology Innovation “GO” Decision: ADHERE Trial in CIDP FEBRUARY 2021 14