Argenx Se · EURONEXT:ARGX
Argenx — Investor Presentation (September 2020)
38 slides from Argenx's investor presentation, September 2020
Slide 1: Corporate Presentation September 2020
Corporate Presentation September 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: Late-stage Biotech Building Towards Commercial Success
Late-stage Biotech Building Towards Commercial Success Neuromuscular and hematology/oncology franchises with expansion opportunities argenx 2021: Reaching patients Global expansion Positive Phase 3 ADAPT data Late-stage pipeline FcRn leadership MG CIDP ITP PV Cusatuzumab strategic alliance New pipel
Slide 4: Growing Franchises With Multiple Late-Stage Programs
Growing Franchises With Multiple Late-Stage Programs Skin Neuro- Hem/Onc muscular Kidney Kidney MG ITP SC PV Kidney CIDP Severe autoimmune AML conditions ARGX-117 MDS GOAL OF 5 LAUNCHES IN 5 YEARS 4
Slide 5: Preparing to Bring Efgartigimod to Patients in 2021
Preparing to Bring Efgartigimod to Patients in 2021 Reaching patients, physicians, payors in COVID-19 environment Supply Chain Readiness The Right Team in Place • Global manufacturing scale/flexibility • Commercial leads hired • Building inventory, 3PL selection • Field force of MRLs, TLLs, payor te
Slide 6: Deep Antibody Pipeline Of Differentiated Candidates
Deep Antibody Pipeline Of Differentiated Candidates Next Program Target Indication Preclinical Phase 1 Phase 2 Phase 3 Registration Partner Milestone Positive Topline IV MG BLA ADAPT Data Meet with FDA in SC Bridging MG Bridging 4Q 2020 Efgartigimod IV ITP Trial ongoing IV + SC ITP Ongoing discussio
Slide 7: 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 7
Slide 8: Efgartigimod: IgG1 Fc Fragment With ABDEG Mutations
Efgartigimod: IgG1 Fc Fragment With 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 8 Efgartigimod is an investigational compound, n
Slide 9: 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 Role of
Slide 10: Myasthenia Gravis: Chronic, Debilitating Autoimmune Disease
Myasthenia Gravis: Chronic, Debilitating Autoimmune Disease Symptoms of Generalized Myasthenia Gravis “Severe & generalized muscle weakness” 85% of people with MG progress* to generalized MG within 18 months Diplopia Ptosis Difficulty Difficulty swallowing speaking Up to 20% of patients experience a
Slide 11: Efgartigimod Showed Robust Benefit For Patients With gMG
Efgartigimod Showed Robust Benefit For Patients With gMG • Statistically significant and clinically meaningful Primary endpoint met improvement in MG-ADL Meaningful patient • Fast and deep responses benefit observed • Potential for individualized dosing Favorable tolerability • Safety & tolerability
Slide 12: ADAPT Data Show Significant Improvement in MG-ADL and QMG
ADAPT Data Show Statistically Significant and Clinically Meaningful Improvement in MG-ADL and QMG 12
Slide 13: ADAPT Data Show Fast, Deep and Durable Responses
ADAPT Data Show Fast, Deep and Durable Responses Minimal Symptom Expression Fast Onset of Action Durable Clinical Benefit 40% of efgartigimod patients achieved minimal Of the 44 patients who were efgartigimod Potential for individualized dosing symptom Expression compared to 11% in responders, 84.1%
Slide 14: Repeat Treatment Yielded Equally Strong Benefit
Repeat Treatment Yielded Equally Strong Benefit MG-ADL responders MG-ADL responders (AChR-Ab+ patients, first cycle) (AChR-Ab+, second cycle) 27% of P < 0.0001 36.8% of 80% P < 0.0001 Efgartigimod patients 80% Efgartigimod patients 67.7% P <who 0.0001 responded in the 70.6% not responding to 1st 70%
Slide 15: ADAPT Data Support Individualized Dosing
ADAPT Data Support Individualized Dosing Current Standard of Care Envisioned Treatment Paradigm MG Symptoms MG Symptoms Toward minimal TAPER symptom expression Fast onset of response Deep response, 40% MSE Chronic dosing Individualized dosing • Fast-acting steroids and slow-acting Well tolerated imm
Slide 16: 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 people with MG progress* to generalized MG within 18 months 55,000 patients have generalized form of myasthenia gravis 36% 20,000 patients require of patients require treatment mor
Slide 17: Immune Thrombocytopenia: Phase 3 ADVANCE Trials
Immune Thrombocytopenia: Phase 3 ADVANCE Trials Trial Primary objective N=156 Durable response: * 24 weeks 10mg/kg IV (potential to adjust frequency upon response) sustained platelet count (≥50×109/L) Discussions ongoing with FDA to bring n≥50 forward SC Cumulative duration of components of * 24 wee
Slide 18: CIDP: Phase 2 ADHERE Trial
Chronic Inflammatory Demyelinating Polyneuropathy: Phase 2 ADHERE Trial 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
Slide 19: Pemphigus Vulgaris: Adaptive Phase 2 Trial
Pemphigus Vulgaris: Adaptive Phase 2 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 invest
Slide 20: Phase 2 Data In PV Support Advancement To Phase 3
Phase 2 Data In PV 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: 2-13 weeks
Slide 21: Cusatuzumab Strategy
Cusatuzumab Strategy Newly diagnosed elderly patients with AML unfit for intensive chemotherapy Phase 2 CULMINATE Trial Phase 1b in Triple Combination Cusatuzumab + Azacitidine Trial in Japan Part 1: Dose selection Cusatuzumab continues to enroll + azacitidine 10 mg/kg 20 mg/kg + venetoclax > 100 pa
Slide 22: 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 23: 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 24: Our Key Priorities
Our Key Priorities 1 File BLA by end of year 2 Commercial preparedness 3 Execute pipeline Expand through Immunology 4 Innovation Program 24
Slide 25: Thank You
Thank You visit argenx.com
Slide 26: Innovative ADAPT Trial Designed With The Patient In Mind
Innovative ADAPT Trial Designed With The Patient In Mind Patient population Individualized treatment over 26 weeks 1st cycle Continuation or retreatment 167 (8 weeks) (up to 2 additional cycles) gMG patients (MG-ADL≥5) Stratification …….. …….. • ethnicity Efgartigimod • background therapy 10mg/kg IV
Slide 27: Phase 3 ADAPT Trial: Baseline Characteristics
Phase 3 ADAPT Trial: Baseline Characteristics Total patients AChR Ab+ patients Efgartigimod (N=84) Placebo (N=83) Efgartigimod (N=65) Placebo (N=64) Age Mean years (SD) 45.9 (14.4) 48.2 (15.0) 44.7 (15.0) 49.2 (15.5) Females n (%) 63 (75.0) 55 (66.3) 46 (70.8) 40 (62.5) Time since diagnosis Mean yea
Slide 28: Phase 3 ADAPT Trial: Favorable Tolerability Profile
Phase 3 ADAPT Trial: Favorable Tolerability Profile Most adverse events were considered mild or moderate; safety profile of Efgartigimod group was comparable to placebo Efgartigimod (N=84) Placebo (N=83) Number of patients with an Adverse Event (AE) n (%) 65 (77.4) 70 (84.3) Number of patients with
Slide 29: Phase 3 ADAPT Trial: Secondary Endpoints
Phase 3 ADAPT Trial: Secondary Endpoints Other Measure Population Time Efgartigimod Placebo P-value secondary Endpoints Response QMG responder AChR Ab + First cycle 63.1% (41/65) 14.1% (9/64) <0.0001 Response MG-ADL responder AChR Ab + & First cycle 67.9% (57/84) 37.3% (31/83) <0.0001 AChR Ab - Dura
Slide 30: Efgartigimod positioned to be used early and more broadly in gMG
Vision: Efgartigimod positioned to be used early and more broadly in gMG patients Positioning efgartigimod Delay or Steroids IVIg Eliminate ISTs ACIs Taper Ritux Soliris Current MG Treatment Paradigm ACIs (mestinon) Steroids most common ISTs used for steroid Later agents used for at diagnosis add-on
Slide 31: 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 31
Slide 32: 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 33: 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 34: PV Phase 2: IgG Reductions Drive Fast and Deep PDAI Improvements
PV Phase 2: 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
Slide 35: PV Phase 2: Adaptive Design Yields Optimal Dosing Schedule
PV Phase 2: 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 emer
Slide 36: CIDP: Therapeutic Activity Shown With Increasing Selectivity For IgG Reductions
CIDP: Therapeutic Activity 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 37: 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 37
Slide 38: Augmenting Intrinsic Therapeutic Properties of Antibodies
Augmenting Intrinsic Therapeutic Properties of Antibodies Technology Antibody Suite of Technologies Subcutaneous Delivery Technology Role V-region SIMPLE Antibody™ Platform Unlock novel and • Llama immune system delivers V-regions with high human homology complex targets • Highly diverse antibody ou