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IPTACOPAN ASH UPDATE

Iptacopan monotherapy was superior to SoC at reducing absolute reticulocyte count from baseline Mean absolute reticulocyte count (SD) during the 24-week randomized treatment period Mean absolute reticulocyte count, 109/L (SD) 325- 275 225- 175- 125- 75- 25- 0. ULN LLN BL Wk1 WK2 Wk4 Wk6 Wk8 Wk12 Wk16 Wk18 Wk20 Wk22 Wk24 Patients with available data Study visit Iptacopan 62 61 58 Anti-C5 SoC 35 28 27 60 32 59 31 59 31 60 33 Adjusted mean change from baseline¹ in absolute reticulocyte count (95% CI) was -115.89 (-126.49, -105.30) x 109/L for iptacopan vs +0.37 (-13.03, 13.77) x 109/L for SoC, with a difference of -116.26 (-132.17, -100.36) x 109/L (P<0.00012). 56 59 56 56 58 27 34 32 32 34 1. Between Days 126 and 168. 2. A repeated measures model, adjusting for covariates including baseline absolute reticulocyte count, was used for comparisons between the treatment arms. P value is two-sided and unadjusted. LLN = lower limit of normal ULN upper limit of normal 15 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine
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