Novartis reports Phase 3 APPLY-PNH trial met both primary endpoints

Novartis announced detailed results from the pivotal Phase 3 APPLY-PNH trial. The results showed a vast majority of patients with paroxysmal nocturnal hemoglobinuria, or PNH, who received the investigational oral monotherapy iptacopan achieved clinically meaningful increases in hemoglobin levels compared to anti-C5 therapy. The study met both primary endpoints and most secondary endpoints, with iptacopan demonstrating superiority over anti-C5 therapy in adult patients with PNH experiencing residual anemia despite prior treatment with anti-C5 therapy. The study met both primary endpoints, showing superiority for iptacopan vs. anti-C51. For the first, an estimated 82.3% of iptacopan-treated patients achieved hemoglobin-level increases of 2 g/dL or more from baseline without the need for red blood cell transfusions, compared to an estimated 2.0% of anti-C5-treated patients: an estimated 80.3% difference in favor of iptacopan. The observed number of patients achieving this primary endpoint was 51/60# for iptacopan vs. 0/35 for anti-C5. For the second primary endpoint, an estimated 68.8% of iptacopan-treated patients achieved hemoglobin levels of 12 g/dL or more without the need for blood transfusions, compared to an estimated 1.8% of anti-C5-treated patients: an estimated 67.0% difference in favor of iptacopan. The observed number of patients achieving this primary endpoint was 42/60# for iptacopan vs. 0/35 for anti-C5.