Slide 1: Long-Term Safety, Tolerability, and Efficacy of Efgartigimod in Patients With Generalized Myasthenia Gravis: Interim Results of the ADAPT+ S
Long-Term Safety, Tolerability, and Efficacy of Efgartigimod in Patients With Generalized Myasthenia Gravis: Interim Results of the ADAPT+ Study James F. Howard, Jr,1 Vera Bril,2 Tuan Vu,3 Chafic Karam,4 Stojan Peric,5 Jan L. De Bleecker,6 Hiroyuki Murai,7 Andreas Meisel,8 Said Beydoun,9 Mamatha Pas
Slide 2: Disclosures
Disclosures • The phase 3 ADAPT and ADAPT+ studies were funded by argenx • JFH has received research support, honoraria, and nonfinancial support from argenx JFH has also received research support from Alexion Pharmaceuticals, Cartesian Therapeutics, the Centers for Disease Control and Prevention (A
Slide 3: Efgartigimod Mechanism of Action: Blocking FcRn
Efgartigimod Mechanism of Action: Blocking FcRn • FcRn recycles IgG, extending its half-life and serum Endothelial Cell Blood Vessel concentration1 • Efgartigimod is a human IgG1 Fc fragment, a natural ligand of FcRn, engineered for Endosome increased affinity to FcRn2 Endothelium • Efgartigimod was
Slide 4: ADAPT+ Study Design
ADAPT+ Study Design ADAPT1 ADAPT+ (Placebo controlled) (Open-label efgartigimod) 2 weeks screening 26 weeks (max. 3 cycles) Up to 3 years MGFA Class II, III, IV Efgartigimod Part A (1 y) Part B (2 y) n=84 AChR-antibody positive or N=167 Efgartigimod Efgartigimod negative 1:1 MG-ADL score ≥5a N=151 P
Slide 5: Efgartigimod Demonstrated Repeatable and Sustained Improvement in Both MG-ADL and QMG Over Multiple Cycles in ADAPT+
Efgartigimod Demonstrated Repeatable and Sustained Improvement in Both MG-ADL and QMG Over Multiple Cyclesa in ADAPT+ AChR-Ab+ Population MG-ADL Total Score QMG Total Score Mean Change From Cycle Baseline by Cycle Mean Change From Cycle Baseline by Cycle (Efgartigimod + current TX) (Efgartigimod + c
Slide 6: Proportion of Patients With Increasing MG-ADL or QMG Improvement Over Multiple Cycles
Proportion of Patients With Increasing MG-ADL or QMG Improvement Over Multiple Cyclesa AChR-Ab+ Population Change in MG-ADL Total Score Change in QMG Total Score Efgartigimod (open-label) Placebo (phase 3) Efgartigimod (open-label) Placebo (phase 3) Week 3 of cycles 1-5a in ADAPT+ Week 3 of Cycle 1
Slide 7: Safety: Summary of AEs
Safety: Summary of AEs Safety Population ADAPT ADAPT+ Placebo (n=83) Efgartigimod (n=84) Efgartigimod (n=139) [34.51 PY] [34.86 PY] [138.14 PY] IR/PY % (n) IR/PY % (n) IR/PY % (n) AEs 7.83 84 (70) 7.23 77 (65) 4.06 81 (112) SAEs 0.29 8 (7) 0.11 5 (4) 0.25 15 (21) ≥1 Infusion-related reaction event 0
Slide 8: Deaths in ADAPT+: None Related to Efgartigimod per Investigator
Deaths in ADAPT+: None Related to Efgartigimod per Investigator Age, y/ Days from last Cause of Death Comorbidities/Medical History Sex dose Unknown; preexisting CV disease, autopsy Pulmonary embolism, chronic obstructive pulmonary disease, 72/F confirmed coronary artery atherosclerosis and 4 hypert
Slide 9: Distribution of Efgartigimod Complete Cycles Over Year
Distribution of Efgartigimod Complete Cycles Over 1 Year AChR-Ab+ population with ≥ 1 year of follow-up in ADAPT/ADAPT+ (N=88) 25 21 19 20 16 54.6% of patients Percent (%) 15 12 Patients received ≤5.5 cycles 10 8 7 per year 5 5 8.0% 9.1% 5.7% 13.6% 18.2% 23.9% 21.6% 0 0 1 2 3 4 5 6 7 8 9 10 Number o
Slide 10: Summary
Summary The safety profile observed during long-term treatment with efgartigimod in ADAPT+ mirrored that seen during ADAPT, even while being conducted during the COVID-19 global pandemic This analysis suggests that long-term treatment with efgartigimod is efficacious, providing consistent and repeat