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Argenx — Technical Presentation (December 2022)
15 slides from Argenx's technical presentation, Dec 11, 2022
Slide 1: Efficacy and Safety of Intravenous Efgartigimod in Adults With Primary ITP: ADVANCE IV Results
Efficacy and Safety of Intravenous Efgartigimod in Adults With Primary Immune Thrombocytopenia: Results of ADVANCE IV, a Phase 3, Multicenter, Double-blinded, Placebo-controlled, Randomized Clinical Trial Catherine M. Broome,1 Vickie McDonald,2 Yoshitaka Miyakawa,3 Monica Carpenedo,4 David J. Kuter,
Slide 2: Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP) • ITP is an acquired autoimmune disorder characterized by a reduction in platelet count, which can result in1-4: – Increased risk of bleeding Increase – Fatigue platelet – Decreased quality of life clearance • IgG autoantibodies, detected in most patients, targe
Slide 3: Efgartigimod Competitively Inhibits FcRn
Efgartigimod Competitively Inhibits FcRn Endothelial Cell1-4 IgG autoantibody Endosome IgG Since there is less opportunity to bind to FcRn, antibody 3 unbound IgGs, including autoantibodies, enter the lysosomal degradation pathway2,3 FcRn Fewer IgGs, including autoantibodies, are recycled 4 back int
Slide 4: ADVANCE IV (NCT04188379): Study Design
ADVANCE IV (NCT04188379): Study Design Phase 3, Multicenter, Double-blinded, Placebo-controlled, Randomized Clinical Trial Early Treatment period (24 weeks) discontinuation Chronic or N=131 visit Randomization Follow-up persistent ITP (2:1) (4 weeks) Efgartigimod 10 mg/kg IV (n=86) Placebo IV (n=45)
Slide 5: Participants Were Randomized 2:1 and Most Completed Treatment
Participants Were Randomized 2:1 and Most Completed Treatment Screened: 205 Screen failure: 74 (36.1%) Randomized: 131 Efgartigimod: 86 Placebo: 45 Completed treatment: 64 (74.4%) Completed treatment: 32 (71.1%) Discontinued treatment: 22 (25.6%) Discontinued treatment: 13 (28.9%) • Adverse event: 3
Slide 6: Baseline Characteristics Indicate Multiple Prior Therapies and Long-standing ITP
Baseline Characteristics Indicate the Majority of Participants Had Multiple Prior Therapies and Long-standing ITP Efgartigimod* (n=86) Placebo* (n=45) Age, mean, years (SD) 46.9 (16.6) 51.7 (17.9) Female, n (%) 47 (54.7) 24 (53.3) Time since diagnosis, mean, years (SD) 10.3 (12.1) 11.1 (13.1) Patien
Slide 7: Efficacy Endpoints: Primary and All Platelet-related Secondary Endpoints Were Met
Efficacy Endpoints: Primary and All Platelet-related Secondary Endpoints Were Met* Endpoint† Efgartigimod Placebo P-value Primary endpoint Proportion with sustained platelet count response, n/N (%)‡ 17/78 (21.8%) 2/40 (5.0%) 0.0316 ≥50×109/L in ≥4/6 visits during weeks 19-24, in the absence of inter
Slide 8: Early Sustained Increases in Platelet Counts
Efgartigimod Demonstrated Early Sustained Increases in Platelet Counts* Efgartigimod Placebo LS-Mean Platelet Count Change From Baseline 40 • 33 (38.4%) of efgartigimod treated participants compared to 5 (11.1%) 20 placebo reached a platelet count of 30X109 platelets at week 1 (x109/L) • Sustained p
Slide 9: Higher Responses than Placebo on IWG Response Criteria
Efgartigimod Resulted in Higher Responses than Placebo on Analysis of IWG Response Criteria, Consistent with Previous Platelet Response Results Percentage of IWG Responders* 60% 51.2% Criterion* Efgartigimod (n=86) Placebo (n=45) Difference in response 44/86 n (%) n (%) (95% CI) 50% % of Responders
Slide 10: Substantially More Weeks With Disease Control
Efgartigimod-treated Participants Experienced Substantially More Weeks With Disease Control* Extent of Disease Control (≥50×109/L): Cumulative Number of Weeks of Disease Control 20–24 10% 0% 15–19 17% 0% 10–14 28% 5% Number of Weeks 5-9 44% 12% 4 45% 15% 3 49% 15% 2 54% 22% 1 62% 30% 100 80 60 40 20
Slide 11: Sustained Platelet Count Response by Subgroup Analysis Favored Efgartigimod
Sustained Platelet Count Response by Subgroup Analysis Favored Efgartigimod* Placebo Efgartigimod All Patients OVERALL 18.9 (5.3, 30.7) Splenectomy History HISTORY OF SPLENECTOMY 18.8 (-1.9, 35.8) NO HISTORY OF SPLENECTOMY 18.9 (-1.0, 35.2) Baseline ITP Therapies ITP THERAPY AT BASELINE 16.4 (-4.0,
Slide 12: Targeted Reduction of IgG Levels
Efgartigimod Resulted in Targeted Reduction of IgG Levels* Mean % Change from Baseline in Total IgG Levels over Time*† Mean (±SE) Total lgG Percent 0 Change from Baseline -30 -60 80 77 73 74 64 55 59 68 64 60 63 65 57 62 54 62 57 60 56 53 49 53 56 51 58 45 42 45 42 36 35 39 38 34 37 34 34 31 26 31 3
Slide 13: Efgartigimod Was Well-Tolerated in Patients With ITP
Efgartigimod Was Well-Tolerated in Patients With ITP and Consistent With Other Efgartigimod Studies1-5 Efgartigimod Placebo (n=86) (n=45) Patients with event, n (%) ≥1 TEAE 80 (93.0) 43 (95.6) ≥1 serious TEAE 7 (8.1) 7 (15.6) ≥1 TEAE leading to discontinuation of study drug 4 (4.7) 1 (2.2) ≥1 treatm
Slide 14: Efgartigimod Phase 3 (ADVANCE) IV Study Conclusions
Efgartigimod Phase 3 (ADVANCE) IV Study Conclusions The benefits of targeting FcRn and lowering total IgG levels were demonstrated by clinically and statistically significant improvements in platelet counts compared with placebo Efgartigimod was well-tolerated and most adverse events were mild to mo
Slide 15: Acknowledgements
Acknowledgements • We thank the patients who participated in this study and their caregivers, the physicians and nurses who care for them, the staff at study sites, and the staff involved in data collection and analyses • This study was funded by argenx • Medical writing support was provided by Dani