Reimagining Medicine
Company overview
Financial review
2023 priorities
Appendix
Iptacopan - provides the opportunity to redefine care across
multiple complement-driven conditions¹
INNOVATION
2021
2022 2023 2024
First-in-class, oral, selective factor B inhibitor, targeting complement system proximally via alternative pathway¹
Indication
2025 2026+ Key updates
US prevalence
Thousands
PNH
Ph3 - APPLY
Ph3-APPOINT
Superior to SoC for both primary endpoints
in patients with residual anemia despite SoC
Achieved clinically meaningful increases in
Hb levels in treatment-naive patients with PNH
<10
IgAN
Ph3 - APPLAUSE
C3G
Ph3 APPEAR
aHUS
IC-MPGN
*
9 months analysis (H2 2023) potentially provides
basis for US Subpart H filing on proteinuria reduction
Submission enabling readout H2 2023
1852
<10
Ph3 APPELHUS
Submission enabling readout in 2025
<10
Ph3
Ph3 start in H2 2023
<10
Additional ongoing early-stage (Ph2) activities in Lupus Nephritis, iAMD and Immune thrombocytopenia
PNH - paroxysmal nocturnalhemoglobinuria. IgAN - immunoglobulin A nephropathy. C3G complement 3 glomerulopathy. aHUS atypical hemolytic uremic syndrome. iAMD - intermediate age-related macular degeneration.
IC-MPGN - immune complex membranoproliferative Glomerulonephritis. *9 months readout may support US submission for accelerated approval. 1. Phase 3 studies initiated or planned; additional indications are being explored.
2. Estimated ~46-55k number of patients at high risk of progression with proteinuria > 1g/day (~25-30%).
16 Investor Relations | Q4 2022 Results
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