Fadi Fakhouri, MD, PhD, Professor of Nephrology at CHUV Lausanne, Switzerland, discusses new data on pegcetacoplan in patients with C3 glomerulopathy (C3G) and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).
C3G is a rare kidney disease characterized by damage to kidney glomeruli due to abnormal activation of the complement system. When C3 proteins get lodged in the kidney, glomeruli get injured and buildup of toxins and reduced urine production occur. This can ultimately lead to declined kidney function. Common signs and symptoms include hematuria, proteinuria, reduced glomerular filtration rate and increased creatinine levels, fatigue, and edema of the hands, feet, and ankles.
Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade. It is currently approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
VALIANT Clinical Trial
The VALIANT (NCT05067127) clinical trial is a phase 3, randomized, placebo-controlled, double-blind, multicenter study evaluating the safety and efficacy of pegcetacoplan in patients ages 12 years and older with C3G or primary IC-MPGN. A total of 124 patients were enrolled and randomized to pegcetacoplan or placebo twice weekly for 26 weeks. Following the 26-week period, an open-label phase was open to all patients. The primary endpoint was log transformed ratio of urine protein-to-creatinine (UPCR) at week 26 compared to baseline.
Recently, new data from this trial was presented at the 2025 European Renal Association (ERA) Congress. Pegcetacoplan demonstrated a statistically significant proteinuria reduction of 68% at week 26 versus placebo. This reduction was sustained at one year. Patients also achieved stabilization of kidney function measured by estimated glomerular filtration rate (eGFR). For patients that switched to pegcetacoplan from placebo during the open-label extension, similar results were seen in proteinuria reduction and kidney function stabilization. Additionally, the safety profile of pegcegtacoplan was favorable.
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To learn more about C3G and IC-MPGN and other rare kidney conditions, visit https://checkrare.com/diseases/kidney-and-urinary-diseases/