Shots:

• The P-III trial (APPLY-PNH) evaluating iptacopan monotx. (200mg, BID) in 97 patients with PNH
• The trial met its 1EPs & 2EPs i.e., 82.3% vs 2.0% achieved Hb-level of ≥2g/dL from baseline without the need for RBC transfusions while 68.8 vs 1.8% of 12g/dL
• The therapy also showed a superiority over anti-C5 therapy in resolving extravascular hemolysis and maintaining intravascular hemolysis control & the safety profile was consistent with prior reported data with no serious inf., 57.7% had received blood transfusions in the 6mos. before randomization, 96.4% vs 26.1% were blood-transfusion free, no difference b/w treatment arms was seen for the rate of major adverse vascular events or change from baseline in LDH levels

Ref: Novartis | Image: Novartis