Novartis’ iptacopan readout sets up challenge to AZ-Alexion franchise in PNH

New head-to-head data suggest Novartis’ complement inhibitor could soon capture a share of the hematology market dominated by AstraZeneca’s blockbuster Soliris/Ultomiris franchise, although the oral therapy will need to clear more hurdles before it can fulfill Novartis’ ambitions for it to become a multibillion-dollar drug.

Novartis AG (SIX:NOVN; NYSE:NVS) said Monday that CFB inhibitor iptacopan showed superiority to C5 inhibitors Soliris eculizumab and Ultomiris ravulizumab on both co-primary endpoints in the Phase III APPLY-PNH study to treat paroxysmal nocturnal hemoglobinuria. Compared with the IV therapies at 24 weeks, Novartis’ oral therapeutic led to significantly more patients gaining 2 g/dL of hemoglobin from baseline without needing transfusions, and significantly more patients with at least 12 g/dL of hemoglobin without requiring transfusions...