IPTACOPAN ASH UPDATE

APPLY-PNH is a randomized Ph3 trial investigating iptacopan monotherapy in PNH patients with residual anemia despite SoC Study design DATA PRESENTED UP TO 24 WEEKS Iptacopan 200mg BID (n=62) Continue with iptacopan 200mg BID Anti-C5 antibody Anti-C5 antibody (n= 35) Switch to iptacopan 200mg BID Up to 8 weeks 24 weeks 24 weeks D-60 Screening period D1 D168 Randomized treatment period Treatment extension period D336 EoS PNH = Paroxysmal Nocturnal Hemoglobinuria Hb Hemoglobin RBC = Red Blood Cell BID twice a day EOS End of Study 10 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 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 NOVARTIS | Reimagining Medicine

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