iptacopan + placebo

Iptacopan (LNP023) is a first-in-class, oral, small molecule inhibitor of complement factor B, developed by Novartis. It specifically targets the alternative complement pathway, which is believed to be excessively activated in various complement-mediated kidney diseases, including lupus nephritis. In clinical trial settings, such as the Phase 2 study NCT05268289, the designation 'iptacopan + placebo' refers to a specific intervention arm where patients receive iptacopan alongside a placebo (typically to maintain the double-blind nature of the trial against other treatment arms). Iptacopan works by binding to factor B, preventing the formation of the C3 convertase (C3bBb) and thereby inhibiting the amplification of the complement cascade that contributes to kidney inflammation and damage. While iptacopan (as Fabhalta) has received FDA approval for other indications like IgA nephropathy and PNH, it remains an investigational therapy for active lupus nephritis.

Other names

iptacopan + placebo