IPTACOPAN ASH UPDATE

Iptacopan, a first-in-class, oral, selective factor B inhibitor, targets the complement system proximally via the alternative pathway1 Alternative pathway (Tick-over) C3 Iptacopan Factor B C3(H2O) Factor D C3 convertase Iptacopan binds to the active site of factor B, Iptacopan inhibiting the activity of C3 convertase¹ Factor B Factor B Factor D C3 C3 C3b convertase C3a C3b Proximal pathways --- Terminal pathway C5 convertase C5 C6, C7, C8, C9n C5a C5b No C3 opsonization Iptacopan controlled intra- and extravascular hemolysis in 10 patients with a sub-optimal response to eculizumab, leading to transfusion independence and an improved quality of life² MAC THE LANCET Haematology Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial Material from The Lancet Haematology is used with permission. 1. Schubart A et al. Proc Natl Acad Sci USA 2019;116:7926-31. 2. Risitano AM et al. Lancet Haematol 2021;8:e344-54. 9 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine

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