Q3 2022 Results
Company overview
Financial review
2022 priorities
Appendix
References
3
INNOVATION
APPLY-PNH demonstrated clinically meaningful superiority vs. anti-C5
Anti-C5 antibody
Up to 8 weeks
LNP023 200mg BID
(n=62)
Continue with
LNP023
200mg BID
Anti-C5 antibody
(n= 35)
Switch to LNP023
200mg BID
24 weeks
D-60
D1
Screening
period
Randomized
treatment period
24 weeks
D168
D336
EoS
Treatment
extension period
Population (n = 97)
Adult PNH patients with residual anemia (Hb
<10g/dL) on a stable regimen of anti-C5 therapy
6 months prior to randomization
Primary endpoints
Superiority for proportion of patients
achieving increase in Hb ≥2g/dL from
baseline in the absence of RBC transfusion
☑ Superiority for proportion of patients
achieving Hb ≥12g/dL in the absence of
RBC transfusion
Oral monotherapy iptacopan demonstrates clinically meaningful superiority over anti-C5 treatment in Ph3
Met 2 primary endpoints for superiority in PNH patients with residual anemia despite prior anti-C5 treatment
PNH - paroxysmal nocturnal hemoglobinuria Hb - Hemoglobin RBC Red Blood Cell BID twice a day EOS end of study
17 Investor Relations | Q3 2022 Results
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