Argenx Se · EURONEXT:ARGX
Argenx — Technical Presentation (May 2020)
17 slides from Argenx's technical presentation, May 26, 2020
Slide 1: ADAPT Phase 3 Topline Results: Efgartigimod in Generalized Myasthenia Gravis
26 May 2020 ADAPT Phase 3 Topline R esults Efgartigimod in Generalized Myasthenia Gravis
Slide 2: Forward-Looking Statements
Forward - Looking Statements Safe Harbor: Certain statements contained in this presentation, other than present and historical facts and conditions independently verifiable at the date hereof, may constitute forward - looking statements. Examples of such forward - looking statements include those re
Slide 3: Efgartigimod Showed Robust Benefit For Patients With gMG
Efgartigimod Showed Robust Benefit For Patients With gMG • Statistically significant and clinically meaningful improvement in MG - ADL • Fast and deep responses • Potential for individualized dosing • Safety & tolerability profile comparable to placebo Primary endpoint met Meaningful patient benefit
Slide 4: gMG is a Chronic and Debilitating Autoimmune Disease
gMG is a Chronic and Debilitating Autoimmune Disease “Severe & generalized muscle weakness” Symptoms of Generalized Myasthenia Gravis Diplopia Difficulty speaking Ptosis Difficulty swallowing Respiratory impairment Loss of motor skills Impaired mobility Extreme fatigue High need for safe & efficacio
Slide 5: IgG Auto-Antibodies Drive Myasthenia Gravis
IgG Auto - Antibodies Drive Myasthenia Gravis Efgartigimod is Designed to Eliminate Auto - Antibodies Block acetylcholine receptors ( AChR ) Crosslink + internalize ACh Rs AChR auto - antibodies in MG 1 2 Recruit complement 3 Other auto - antibodies in MG ( MuSK , LRP4, other/unknown) Block function
Slide 6: Innovative ADAPT Trial Designed With the Patient in Mind
Innovative ADAPT Trial Designed With the Patient in Mind Primary endpoint: % MG - ADL responders (≥ 2 - point improvement for at least four consecutive weeks during the first cycle in AChR - Ab+ patients) Retreatment criteria were protocol defined based on loss of clinically meaningful improvement 1
Slide 7: Baseline Characteristics
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 years (SD) 10.13 (9.0) 8
Slide 8: Favorable Tolerability Profile
Favorable Tolerability Profile 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 an AE grade ≥3 n (%) 9 (10.7) 8 (9.6) Most frequent AEs n (%) Headache 24 (28.6) 23 (27.7) Nasopharyngitis 10 (11.9) 15 (18.1) Nausea 7 (8
Slide 9: Primary Endpoint Met: Statistically Significant Improvement in Daily Living Activities
29.7% 0% 10% 20% 30% 40% 50% 60% 70% 80% Efgartigimod Placebo 67.7% N=19/64 N=44/65 MG - ADL responders ( AChR - Ab+ patients, first cycle) MG - ADL responder : ≥ 2 - point improvement for at least four consecutive weeks during the first cycle % MG - ADL responders Primary Endpoint Met: Statisticall
Slide 10: Statistically Significant Improvement in QMG Response
14.1% 0% 10% 20% 30% 40% 50% 60% 70% 80% Efgartigimod Placebo 63.1% N=9/64 N=41/65 QMG responders ( AChR - Ab+ patients, first cycle) QMG responder : ≥ 3 - point improvement for at least four consecutive weeks during the first cycle (Secondary endpoint) % QMG responders Statistically Significant and
Slide 11: 40% of Efgartigimod Patients Achieved Minimal Symptom Expression
11.1% 0% 10% 20% 30% 40% 50% 60% 70% 80% Efgartigimod Placebo 40.0% N=7/63 N=26/65 Minimal Symptom Expression ( AChR - Ab+ patients, first cycle) Minimal Symptom Expression: MG - ADL = 0 (no symptoms) or 1 % patients with MSE 40% of Efgartigimod Patients Achieved Minimal Symptom Expression Compared
Slide 12: Secondary Endpoints Support Benefit
Secondary Endpoints Support Benefit Other secondary Endpoints Measure Population Time Efgartigimod Placebo P - value Response QMG responder AChR Ab + First cycle 63.1% (41/65) 14.1% (9/64) <0.0001 Response MG - ADL responder AChR Ab + & AChR Ab - First cycle 67.9% (57/84) 37.3% (31/83) <0.0001 Durat
Slide 13: Fast Onset of Action
z Fast Onset of Action Early onset MG - ADL responders ( AChR Ab+ Efgartigimod responders, first cycle) 84.1% Early onset MG - ADL responders ( AChR Ab+ patients, first cycle) Early onset responder : Onset of MG - ADL response during the first two weeks 56.9% 25.0% 0% 10% 20% 30% 40% 50% 60% 70% 80%
Slide 14: Durable Clinical Benefit
Durable Clinical Benefit Time to qualify for retreatment ( AChR Ab+ patients, full study) 35 8 0 5 10 15 20 25 30 35 40 45 Efgartigimod Placebo Median time (Days) Time to qualify for retreatment : time from one week after last infusion until no clinically meaningful improvement Duration of response
Slide 15: Repeat Treatment Yielded Equally Strong Benefit
Repeat Treatment Yielded Equally Strong Benefit P < 0.0001 MG - ADL responder: ≥ 2 - point improvement for at least four consecutive weeks during the first cycle 67.7% 29.7% 0% 10% 20% 30% 40% 50% 60% 70% 80% Efgartigimod Placebo 70.6% 25.6% 0% 10% 20% 30% 40% 50% 60% 70% 80% Efgartigimod Placebo %
Slide 16: Expected Next Steps
Expected Next Steps Present detailed ADAPT results at upcoming meeting Submit BLA to FDA in H2 2020 Submit J - MAA with PMDA in H1 2021 1 2 3 Launch in the US in 2021, if approved 4 16
Slide 17: ADAPT Data Support MG Treatment Paradigm Shift
17 • Fast - acting steroids and slow - acting immunosuppressants • Balancing symptom suppression and side effects ADAPT Data Support MG Treatment Paradigm Shift ( 1) Subject to regulatory approval (2) Based on topline data from Adapt trial Individualized dosing Toward minimal symptom expression MG S