IPTACOPAN ASH UPDATE

APPOINT-PNH study is a single-arm Ph3 trial investigating iptacopan monotherapy in treatment-naive patients with PNH Study design iptacopan 200mg BID Time Up to 8 weeks 24 weeks Duration D-58 D1 D168 24 weeks Study period Screening period Core treatment period Treatment extension period BID = twice a day EOS - End of Study ULN = upper limit of normal 21 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 D336 EoS Population (n = 40) Adult PNH patients with hemolysis (LDH > 1.5x ULN) and anemia (Hb < 10g/dL) naive to complement inhibitor therapy Primary endpoint Proportion of patients achieving a sustained increase in Hb of ≥ 2g/dL, in the absence of transfusions NOVARTIS | Reimagining Medicine

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