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Reimagining Medicine

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. ✓ NOVARTIS | Reimagining Medicine
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