Novartis Shares Detailed Results From Experimental Drug For Rare Blood Disorder

Novartis AG NVS announced detailed results from the pivotal Phase 3 APPLY-PNH trial.
The results showed that most of the paroxysmal nocturnal hemoglobinuria (PNH) patients receiving the investigational iptacopan achieved clinically meaningful increases in hemoglobin levels compared to anti-C5 therapy.
The study met both primary endpoints and most secondary endpoints.
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.
For the second primary endpoint, 68.8% of iptacopan-treated patients achieved hemoglobin levels of 12 g/dL or more without blood transfusions, compared to an estimated 1.8% of anti-C5-treated patients.
Iptacopan also showed superiority over anti-C5 therapy across most secondary endpoints, including change from baseline in hemoglobin levels, blood-transfusion independence, patient-reported fatigue, immature red blood cell counts, and rate of clinical BTH.
Separately, Novartis announced that the Phase 3 APPOINT-PNH trial was positive, with iptacopan providing clinically meaningful increases in hemoglobin levels in complement-inhibitor-naïve patients with PNH.
Regulatory submissions are expected in 2023.
Price Action: NVS shares are up 0.41% at $91.25 on the last check Tuesday.