VALIANT Results Support Use of Pegcetacoplan in C3G, IC-MPGN Treatment

Biopharmaceutical companies Apellis and Sobi announced positive topline results from the phase 3 VALIANT study. VALIANT investigated the use of systemic pegcetacoplan in patients with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare and debilitating kidney diseases that can lead to kidney failure and have no approved treatments. Apellis and Sobi are codevelopers of systemic pegcetacoplan.

VALIANT was led by Carla Nester, MD, MSA, FASN. Participants comprised 124 patients aged 12 years or older with C3G and IC-MPGN. The study met its primary end point, demonstrating that treatment with pegcetacoplan resulted in a statistically significant and clinically meaningful 68% (P<.0001) reduction in proteinuria (log-transformed ratio of urine protein-to-creatinine ratio) compared with placebo at 26 weeks. Pegcetacoplan also demonstrated statistical significance on the key secondary end points of composite renal end point (a combination of proteinuria reduction and estimated glomerular filtration rate stabilization) and proteinuria reduction of at least 50% compared with baseline.

Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is approved in the United States and several other countries for the treatment of paroxysmal nocturnal hemoglobinuria.

Detailed data from VALIANT will be presented at an upcoming medical congress. All patients who completed the VALIANT study are now enrolled in the VALE long-term extension study.

Apellis, which has exclusive US commercialization rights for systemic pegcetacoplan, plans to submit a supplemental new drug application to the US Food and Drug Administration in early 2025. Meanwhile, Sobi, which has exclusive commercialization rights outside of the United States, will file a marketing application with the European Medicines Agency in 2025.