Q4 2022 Results
SECONDARY
PRIMARY
Company overview
Financial review
2023 priorities
Appendix
Iptacopan demonstrated superiority over anti-C5 in adult PNH
patients with residual anemia despite anti-C5 treatment...
INNOVATION
APPLY-PNH: Randomized, active-comparator controlled Ph3 trial in adult PNH patients with residual anemia,
despite treatment with an intravenous anti-C5 antibody
Endpoints
Observed
Iptacopan vs. SoC
Population estimate²
Iptacopan vs. SoC
Difference
Increase from baseline in Hb of ≥ 2 g/dL
51/601 vs. 0/35
82.3% vs. 2.0%
80.3% (95% CI 71.3, 87.6) P<0.00013
in the absence of RBC transfusions
Hb ≥ 12 g/dL
42/601 vs. 0/35
68.8% vs. 1.8%
67.0% (95% CI 56.3, 76.9) P<0.00013
in the absence of RBC transfusions
Transfusion avoidance
60/62 vs. 14/35
96.4% vs. 26.1%
70.3% (95% CI 52.6, 84.9) P<0.00013
Clinical breakthrough hemolysis
2/62 vs. 6/35
Rate ratio (95% CI) of 0.10 (0.02, 0.61) means 10-fold lower
rate of annualized clinical breakthrough hemolysis
Improvements across a range of other secondary endpoints: Increasing Hb, reducing patient-reported fatigue and reticulocyte count, maintaining low LDH
17 Investor Relations | Q4 2022 Results
PNH - paroxysmal nocturnal hemoglobinuria. 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 reflect the population average
probability of a patient meeting the endpoint criteria. 3. P values are two-sided and unadjusted.
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