Q1 2023 Results
Company overview
Financial review
Conclusions
Appendix
References
INNOVATION
Iptacopan APPOINT-PNH data showed clinically meaningful
increases in Hb levels for treatment-naive adult patients with PNH
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
D168
24 weeks
Study
period
Screening
period
Core treatment
period
Treatment extension
period
D336
EoS
Study status
Met primary endpoint of proportion of patients
achieving a sustained increase in Hb of ā„ 2g/dL,
in the absence of transfusions, at 24 weeks
Safety profile consistent with previously reported data
Data to be presented at EBMT April 26, 2023
Hb hemoglobin. LDH lactate dehydrogenase.
ULN upper limit of normal.
PNH - paroxysmal nocturnal hemoglobinuria. EBMT - European society for blood and marrow transplantation.
1. BID twice daily.
9 Investor Relations | Q1 2023 Results
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