Q4 2022 Results
Company overview
Financial review
2023 priorities
Appendix
and showed clinically meaningful increases in Hb levels for
treatment-naive adult patients with PNH
INNOVATION
APPOINT-PNH: Single-arm Ph3 trial in adult patients with PNH with hemolysis (LDH > 1.5x ULN)
and anemia (Hb < 10g/dL) naive to complement inhibitors
Iptacopan 200mg BID1
Duration Up to 8 weeks-
24 weeks
Time
D-58
D1
24 weeks
D168
D336
EoS
Study
period
Screening
period
Core treatment
period
Treatment extension
period
Study status
Met primary endpoint of proportion of patients achieving a
sustained increase in Hb of ≥ 2g/dL, in the absence of transfusions,
at 24 weeks
Safety profile consistent with previously reported data
Data to be presented at upcoming medical meeting
Iptacopan has the potential to be practice-changing in PNH
PNH - paroxysmal nocturnalhemoglobinuria. 1. BID twice daily.
18 Investor Relations | Q4 2022 Results
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