The P-III trial (APPOINT-PNH) evaluating iptacopan (200mg, BID) in 40 adult patients. The trial met its 1EPs & showed a clinical benefit across 2EPs where 92.2% & 62.8% achieved a ≥2g/dL & ≥12 g/dL Hg-level increase from baseline without the need for RBC transfusions after 24wk.
97.6% achieved RBC transfusion independence @24wks. with no clinical breakthrough hemolysis events or MAVEs, 83.55% reduction of LDH levels with the reduction seen as early as 7 Days, 95% had LDH levels ≤1.5 times the ULN along with improved patient-reported fatigue
The results were consistent with the tolerability & safety profiles as seen in the (APPLY-PNH) trial. The P-III trial (APPLY-PNH) results were also presented which showed the superiority of iptacopan over anti-C5 therapy
Ref: Novartis | Image: Novartis
Related News:- Novartis Presents P-III Trial (APPLY-PNH) Result of Iptacopan for Paroxysmal Nocturnal Hemoglobinuria at ASH 2022
