Argenx Se · EURONEXT:ARGX
Argenx — 2020 SID Annual Meeting Virtual Conference
16 slides from Argenx's conference presentation, May 13, 2020
Slide 1: Efgartigimod in Pemphigus: Interim Phase 2 Results
2020 SID Annual Meeting Virtual Conference Prof. Dr. Matthias Goebeler Department of Dermatology, Venereology and Allergology University Hospital Würzburg, Efgartigimod in Pemphigus Würzburg, Germany Interim Phase 2 Results 13-16th May 2020
Slide 2: Disclosure
Disclosure ! Prof. Dr. Matthias Goebeler serves as a consultant for argenx and receives compensation for these services. The terms of the agreement have been reviewed and approved by the University Hospital Würzburg. 2
Slide 3: Pemphigus: Autoimmune Blistering Disease
Pemphigus: Autoimmune Blistering Disease Pemphigus is characterized by painful blisters and erosions on mucous membranes and/or skin surfaces • Pemphigus vulgaris is the most common type • Often accompanied by difficulties in swallowing and weight loss Pemphigus foliaceus • Erosions may cause severe
Slide 4: Pathogenic IgG Antibodies Play Central Role in Pemphigus
Pathogenic IgG Antibodies Play Central Role in Pemphigus Skin and mucous membrane* In Pemphigus, IgG antibodies target adhesion molecules desmoglein 1 and/or 3 (Dsg-1, Dsg-3) causing keratinocytes to separate resulting in suprabasal or subcorneal blistering of the skin and/or erosions at mucous memb
Slide 5: Goals Of New Potential Pemphigus Therapies
Goals Of New Potential Pemphigus Therapies Patients and physicians report the need for new therapies that address key unmet needs Achieve fast disease control (DC) Minimize corticosteroid use to 1 and complete remission (CR) 2 improve side effect profile • Rituximab has a relatively slow • Steroids
Slide 6: Efgartigimod Reduces IgG Antibodies By Blocking FcRn
Efgartigimod Reduces IgG Antibodies By Blocking FcRn IgG Recycling in Vascular Endothelium Efgartigimod Mechanism of Action IgG Antibody IgG Antibody FcRn FcRn Efgartigimod Roopenian et al. 2007, Nat Rev Immunol; Vaccaro et al. 2005, Nat Biotech; Ulrichts et al. 2018, J Clin Invest 6
Slide 7: Adaptive Phase 2 Proof-Of-Concept Trial
Adaptive Phase 2 Proof-Of-Concept Trial Cohort 1 Cohort 2 Cohort 3 Cohort 4 6 5 8 15 Efgartigimod dose (mg/kg) 10 25 Induction 4 Weekly infusions Weekly infusions until EoC Maintenance period (weeks) 6 8 12 Up to 34 Maintenance Dosing 2 doses at weeks 2 and 6 Every other week Discretion of investiga
Slide 8: Patient Demographics And Baseline Characteristics
Patient Demographics And Baseline Characteristics Safety Analysis Set (N=34) Efficacy Analysis Set* (N=31) Age (mean ± SD) 51.5 ± 15.3 52.4 ± 15.5 Sex (N (%)) • Male 12 (35%) 10 (32%) • Female 22 (65%) 21 (68%) Pemphigus vulgaris type 25 (74%) 23 (74%) • Mucosal-dominant 9 (36%) 9 (39%) • Mucocutane
Slide 9: Safety And Tolerability Summary
Safety And Tolerability Summary Treatment Emergent Adverse Events (TEAEs) Efgartigimod 10 mg/kg Efgartigimod 25 mg/kg Reported in ≥ 2 patients per dose group (N=19) (N=15) TEAEs (Total) 16 (84%) 12 (80%) Bronchitis 2 (11%) – Rhinitis – 2 (13%) • Favorable tolerability Nasopharyngitis – 2 (13%) profi
Slide 10: IgG Reductions Drive Fast and Deep PDAI Improvements
IgG Reductions Drive Fast and Deep PDAI Improvements IgG Reduction and PDAI Score Improvements in Responders Percentage of Baseline (%) 100 PDAI activity IgG4 Dsg-1 Dsg-3 50 0 W4 W8 W12 W16 W20 W24 W28 W32 Weekly or biweekly maintenance dosing at variable frequency Data show efgartigimod treatment p
Slide 11: Adaptive Design Yields Optimal Dosing Schedule
Adaptive Design Yields Optimal Dosing Schedule IgG/PDAI correlation Maintenance control 30 50 Cohort 1 (N = 4)* Cohort 2 (N = 5)* 40 DC 4 DC 3 PDAI activity 20 PDAI activity 30 20 10 10 0 0 0 14 28 42 56 70 84 98 112 0 14 28 42 56 70 84 98 112 126 140 Time (Days) Time (Days) CRs emerge CRs and
Slide 12: Patient Anecdote 1: Significant PDAI Improvement On Monotherapy
Patient Anecdote 1: Significant PDAI Improvement On Monotherapy; Low-dose Steroids Drive To CR Newly Diagnosed Mucocutaneous PV | 65-year old female PDAI activity score: 23 | Anti-Dsg-3: > 800 RU/mL | Anti-Dsg-1: 129 RU/mL 100 efgartigimod treatment follow-up PDAI activity Key Findings Percentage of
Slide 13: Patient Anecdote 2: Long-term, Durable CR On Minimal Therapy
Patient Anecdote 2: Long-term, Durable CR On Minimal Therapy Newly diagnosed mucosal dominant PV | 47-year old male PDAI activity score: 10.3 | Anti-Dsg-3: > 800 RU/mL | Anti-Dsg-1: 69 RU/mL Day 1 Chest blister 150 efgartigimod treatment follow-up PDAI activity Percentage of Baseline (%) 125 IgG Ant
Slide 14: Patient Anecdote 3: Ability To Taper Steroids Upon Achieving EoC
Patient Anecdote 3: Ability To Taper Steroids Upon Achieving EoC Relapsing PF (diagnosed in 2007) | 57-year old male Day 1 Day 29 Day 43 PDAI activity score: 20.3 | Anti-Dsg-1: 252 RU/mL 100 efgartigimod treatment FU PDAI activity Percentage of Baseline (%) IgG 75 Anti-Dsg-1 50 Key Findings Efgartig
Slide 15: Phase 2 Proof-Of-Concept Data Support Advancement To Phase 3
Phase 2 Proof-Of-Concept Data Support Advancement To Phase 3 Fast onset of action 90% disease control (28/31 patients) – majority after 1-2 infusions Median time to DC: 15 to 22 days (mono/combo therapy) Deep responses 70% complete clinical remission (7/10 patients) on optimized dosing* Time to CR:
Slide 16: Acknowledgements to the teams of:
Acknowledgements to the teams of: Bata-Csörgő Zsuzsanna, University of Szeged, Parodi Aurora, University Hospital San Martino Szeged, Hungary of Genova, Genova, Italy Baum Sharon, Chaim Sheba Medical Center, Remenyik Eva, University of Debrecen, Ramat Gan, Israel Debrecen, Hungary Caproni Marzia, US