Novartis' Rare Blood Disorder Therapy Aces Second Late-Stage Trial

Novartis AG's NVS Phase 3 APPOINT-PNH study of iptacopan in complement-inhibitor-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH) met its primary endpoint.
Topline results showed that a significant proportion of patients treated with iptacopan (200 mg twice daily) achieved clinically meaningful hemoglobin-level increases of 2 g/dL or more from baseline without blood transfusions at 24 weeks.
In the study, the safety profile of iptacopan monotherapy was consistent with previously reported data.
Topline results for the pivotal Phase 3 APPLY-PNH study were recently announced. It met its two primary endpoints, with iptacopan demonstrating superiority over anti-C5 therapies (eculizumab or ravulizumab) in adults with PNH experiencing residual anemia despite prior anti-C5 treatment.
The study showed a statistically significant and clinically meaningful increase in the proportion of iptacopan-treated patients achieving 2 g/dL or more hemoglobin-level increases from baseline and 12 g/dL or more hemoglobin levels without the need for blood transfusions at 24 weeks, compared to anti-C5 therapies.
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