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: Forward Looking Statements
Forward Looking Statements Vv argenx 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 PRE
Slide 3: Company Highlights
Company Highlights Differentiated therapeutic antibodies pioneering in severe autoimmune diseases & cancer • ARGX-113: first-in-class FcRn antagonist targeting array of IgG mediated AI diseases Novel concept in autoimmunity • • Phase 1: favorable tolerability profile; IgG reduction up to 85% Phase 2
Slide 4: Deep Pipeline In Severe Autoimmune Diseases and Cancer
Deep Pipeline In Severe Autoimmune Diseases and Cancer Wholly-Owned Product Candidates 2H18: Phase 2 interim data Formulation Phase 1/2 Phase 1/2 Complementary to ARGX-113 Partnered Product Candidates (gerilimzumab) and tiered royalties $625mm milestones & tiered royalties exclusive option to licens
Slide 5: Phase 2 Study of Efgartigimod in Patients with Generalized Myasthenia Gravis
Phase 2 Study of Efgartigimod in Patients with Generalized Myasthenia Gravis
Slide 6: Pathogenic IgGs Play Key Role in MG; Clearance by Efgartigimod
Pathogenic IgGs Play Key Role in MG Clearance by efgartigimod un.i.q.cuaueunIsmgeGedtcbmlyeaeprdaitnchacolegnmeeneich.I.ag. Gnissm Antibody FcRn Efgartigimod • • ‘ISgnGo-mwefldaikaet’eddisdeisaesease WAuatxoi-nagnatingdenwsaAnCinhgR, MuSK, LRP4
Slide 7: Pathogenic IgGs Play Key Role in MG; Clearance by Efgartigimod
Pathogenic IgGs Play Key Role in MG Clearance by efgartigimod unmet medical need... ...caused by pathogenic IgGs • • ‘Snowflake’ disease Waxing and waning • • IgG-mediated disease Auto-antigens AChR, MuSK, LRP4 unique IgG clearance mechanism Antibody FcRn Efgartigimod 8
Slide 8: Phase 2 Trial Design
Phase 2 Trial Design N=12 Primary endpoint Secondary endpoints Efficacy MG-ADL; QMG; MGC; MG-QoL PD total IgG; pathogenic IgG Immuno-genicity PK Safety & tolerability (*) >50% of the score attributed to non-ocular items 9 Clinicaltrials.gov: NCT02965573, argenx data Screening/Randomization Treatment
Slide 9: Baseline Population and Disease Characteristics
Baseline Population and Disease Characteristics Age (mean ± SD) 43.5 ± 19.3 55.3 ± 13.6 Race • • • Asian Black / African White - 1 (8.3%) 11 (91.7%) 1 (8.3%) - 11 (91.7%) American Baseline QMG score (mean ± SD) (min, median, max score) 11.8 ± 5.4 (3, 12.5, 24) 14.5 ± 6.3 (6, 14, 30) Baseline MGC sco
Slide 10: Efgartigimod Safety And Tolerability Profile
Efgartigimod Safety And Tolerability Profile 2 hour infusion enabling out-patient administration 11 argenx data
Slide 11: Lasting IgG Reduction
Lasting lgG Reduction Potent lgG reduction across isotypes argenx • Total lgG LEVELS lgGl lgG3 150 l 150 .... Vl +I 0 1-'* .... 100 100 Vl .... 100 0 LU Vl +I 0 1-"<!< 1-'* so so 50 20 20 40 Study d a y 60 80 40 St u dy d ay 60 80 0 20 40 60 80 Stu dy d a y lgG2 lgG4 150 150 100 100 w VI +I t-"' '*
Slide 12: Clinically Meaningful and Long-lasting Reduction of Efficacy Scores
argenx . Clinically Meaningful and QMG and MG-ADL scores Long-lasting Reduction of Efficacy Scores QMG MG-ADL w V) +I c "Q'l E w V) +I c 0 0 "Q'l -2 E -2 Q) c Ql Ill .".0' Ql c Ql Ill -4 .".0' -4 -0 E E -0 -6 t t t Ql Ql ti.O ti.O c c -8 -6 .".c' .z":'. 0 20 40 Study day 60 80 0 20 40 Study day 60 8
Slide 13: Robust Clinical Improvement Over Placebo Group
Robust Clinical Improvement Over Placebo Group 14 argenx data - * Missing data point of 1 patient MG-ADL change from baseline QMG change from baseline •Efgartigimod vs. placebo: increasing differentiation observed with increasing MG-ADL/QMG thresholds Day 36, 2 weeks after last dosing Day 29, 1 week
Slide 14: Total & Pathogenic IgG Reduction Correlates with Clinical Improvements
Total & Pathogenic lgG Reduction Correlates with Clinical Improvements Assessment for all efficacy scales argenx • 150 0 -2 Q.l Ill nJ 100 -4 E :::s tD 0... ..... Q.l b.O s:: 3i n OJ - 6 ·------· n < 50 nJ .s::. ·-Ill , n u 0 -8 ... ""' ....., tD , ... 0 20 40 Study day 60 80 * p < 0.05 -+ ·MG - Com
Slide 15: 75% of Treated Patients Achieved Lasting Response
75% of Treated Patients Achieved Lasting Response Patients with MG-ADL > 2 for a period of at least 6 weeks * (p = 0.0391) 75% 25% Placebo efgartigimod 16 argenx data 83% of patients treated with efgartigimod achieved a clinically meaningful response (MG-ADL 2) 75% of patients treated with efgartigi
Slide 16: Conclusions Phase 2 Trial of Efgartigimod in Patients with gMG
Conclusions Phase 2 Trial of Efgartigimod in Patients with gMG 17 Consistent and compelling safety & tolerability Fast, long-lasting and sustained benefit; clinically meaningful and statistically significant Strong correlation between IgG level reduction and disease improvement; validating focus on
Slide 17: Acknowledgements
Acknowledgements to the Teams of: Andrzej Szczudlik, Centrum Neurologii Klinicznej, Poland Maria Rosa Rottoli, Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII, Italy Angela Genge, Montreal Neurological Institute and Hospital, Canada Philip Van Damme, UZ Leuven, Belgium Renato Mantegazza, Fo
Slide 18: Efgartigimod: Pipeline-in-a-product opportunity & outlook
Efgartigimod: Pipeline-in-a-product opportunity & outlook 2018
Slide 19: ARGX-113: Pipeline-In-Product Opportunity
ARGX-113: Pipeline-In-Product Opportunity Prioritizing IgG autoantibody mediated diseases Landscape of IgG severe autoimmune diseases (selection) Epidermolysis Bullosa Acquisita Scleroderma Lupus Rheumatoid Arthritis Immune Thrombocytopenia Myasthenia Gravis Anca Vasculitis Bullous Pemphigoid Pemphi
Slide 20: Immune Thrombocytopenia Phase 2 Amended Trial Design
Immune Thrombocytopenia Phase 2 Amended Trial Design open label (re)treatment arm of 1 year (all patients) - @ 10 mg/kg Screening/Randomization Treatment Phase Follow-up Phase SoC Efgartigimod (10mg/kg) N=12 Key inclusion criteria • ITP patients with platelet levels < 30 X 109/L On a stable dose of
Slide 21: Pemphigus Vulgaris Phase 2 Adaptive Design
Pemphigus Vulgaris Phase 2 Adaptive Design Treatment Phase Follow-up Phase Maintenance 6 weeks Induction 3 weeks 8 weeks COHORT 1: N= 4 Efgartigimod (10mg/kg) Efgartigimod (10mg/kg) 4 infusions (weekly) 2 infusions (w2, w6) COHORT 2 & 3: N= 4 + 4 Efgartigimod (adjusted dose) Efgartigimod (adjusted d
Slide 22: Phase 1 Healthy Volunteer SubQ Formulation Open Label Trial Design
Phase 1 Healthy Volunteer SubQ Formulation Open Label Trial Design Screening/Randomization Treatment Phase & Follow-up Phase Single IV dose (10 mg/kg) N= 8 Inclusion criteria • • • Healthy male subjects 18-55 years old Body weight: 50 – 100 kg D57 D1 Single subQ dose (10 mg/kg) N= 8 D57 D1 2X IV dos
Slide 23: Key Upcoming Expected Milestones & Communications
Key Upcoming Expected Milestones & Communications Q1 2018 Q2 MG Ph2 Full data (AAN, April 24, LA) SubQ Ph1 HV Full data Efgartigimod AML Ph1/2 Interim data ARGX-110 Partnerships Potenial milestone(s) 2018 Q4 Q3 ITP Ph 2 Full data (ASH) MG Ph 3 Launch PV Ph2 Interim data ITP Ph2 Topline data Efgartig
Slide 24: Key Upcoming Expected Milestones & Communications
argenx’ Key Upcoming Expected Milestones & Communications 2018 aa a2 2018 Q3 a4 Egartigi [cin X Partnerships
Slide 25: Q&A