IPTACOPAN ASH UPDATE
Opportunity to redefine care in multiple complement-driven
conditions
Indication
2021
2022 2023 2024
2025
2026
Market potential
PNH
Indication
Ph3 - APPLY
US prevalence
thousands
Ph3 APPOINT
Hematology
PNH
<10
IgAN
Ph3 APPLAUSE
Nephrology
Ph3 - APPEAR
Ph3 - APPELHUS
C3G
aHUS
IC-MPGN
Phase 3 studies initiated or planned; additional indications are being explored
IgAN
~46-551
C3G
<10
aHUS
<10
Ph3
IC-MPGN
<10
PNH = paroxysmal nocturnal hemoglobinuria IgAN = IgA nephropathy
* 9 months readout may support US submission for accelerated approval
C3G = C3 glomerulopathy aHUS = atypical hemolytic uremic syndrome IC-MPGN = immune complex membranoproliferative glomerulonephritis
1. Estimated number of patients at high risk of progression with proteinuria > 1g/day (-25-30%)
5
IPTACOPAN ASH UPDATE | DECEMBER 13, 2022
NOVARTIS | Reimagining MedicineView entire presentation