Iptacopan Reduces Proteinuria and Stabilizes Kidney Function in C3 Glomerulopathy

Introduction: C3 glomerulopathy (C3G) is a complex, chronic, ultra rare, progressive primary glomerulonephritis, resulting from alternative complement pathway overactivation, leading to kidney failure in most patients, and frequent recurrence in transplants. Iptacopan (LNP023) is an oral, proximal complement inhibitor specifically targeting factor B, that selectively inhibits the alternative complement pathway. Methods: This was a phase 2 extension study of 26 adult patients with native kidney (cohort A), or recurrent C3G (post kidney transplantation; cohort B) receiving open label iptacopan. Results: < 0.0001). In cohorts A + B combined, the median difference in C3 deposit score on renal biopsy from baseline was -7.00 (CI: -12.00 to 4.00;) at 9 to 12 months treatment with iptacopan. Conclusion: These data provide a clinical rationale for further evaluation of long-term treatment of C3G with iptacopan.