IPTACOPAN ASH UPDATE
Iptacopan was superior to SoC for both primary endpoints; majority
of iptacopan patients achieved more normal Hb levels vs. 0 on SoC
Observed
Increase from baseline in Hb of ≥ 2 g/dL
in the absence of RBC transfusions
51/601
patients treated
with iptacopan
0/35
patients treated
with SoC
Hb ≥ 12 g/dL
in the absence of RBC transfusions
42/601
0/35
patients treated patients treated
with iptacopan
with SoC
Population
estimate
Estimated proportion² of
patients, % (95% CI)
100
90
80
70
60
10
222223220
50
82.3%
40
Difference =
80.3%
(95% CI 71.3, 87.6)
P<0.00013
2.0%
Estimated proportion² of
patients, % (95% CI)
822222220
100
68.8%
Difference =
67.0%
(95% CI 56.3, 76.9)
P<0.00013
1.8%
1. 2/62 patients in the iptacopan arm had missing data between Days 126 and 168 so were not evaluable based on observed data. 2. Marginal proportions, differences in marginal proportions and 95% Cls were calculated using a logistic
regression model using the Firth correction that adjusted for baseline covariates and accounted for missing data and the possibility of no patients meeting the primary endpoint criteria in the SoC arm; consequently, the treatment effect of
iptacopan is underestimated and the treatment effect of SoC is overestimated. Marginal proportions reflect the population average probability of a patient meeting the primary endpoint criteria. 3. P values are two-sided and unadjusted. Cl,
confidence interval
11
IPTACOPAN ASH UPDATE | DECEMBER 13, 2022
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