Q3 2023 Results
Company overview
Financial review
Conclusions
Appendix
References
INNOVATION
Iptacopan is a potential first-in-class, oral, selective factor B inhibitor,
targeting complement system proximally via alternative pathway1
Indication
2021
2022
2023
2024
2025 2026+
Key updates
PNH
Ph3 - APPLY
Ph3-APPOINT
IgA
Ph3 - APPLAUSE
*
nephropathy
C3G
Ph3 - APPEAR
aHUS
Ph3 APPELHUS
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
Clinically meaningful and highly statistically
significant proteinuria reduction at 9 months
Submission enabling readout Q4 2023
US prevalence
Thousands
<10
1852
<10
Submission enabling readout in 2025
<10
Ph3 started
<10
IC-MPGN
Ph3
Additional ongoing early-stage (Ph2) activities in Lupus Nephritis, iAMD
* 9 months readout may support US submission for accelerated approval
aHUS
PNH - paroxysmal nocturnal hemoglobinuria. IgAN - immunoglobulin A nephropathy. C3G complement 3 glomerulopathy.
IC-MPGN - immune complex membranoproliferative Glomerulonephritis. 1. Phase 3 studies initiated; additional indications are being explored.
> 1g/day (~25-30%).
19 Investor Relations | Q3 2023 Results
atypical hemolytic uremic syndrome. iAMD - intermediate age-related macular degeneration.
2. Estimated -46-55k number of US patients at high risk of progression with proteinuria
ā NOVARTIS | Reimagining MedicineView entire presentation