Apellis and Sobi announce pegcetacoplan meets Phase 3 primary endpoint

Apellis and Sobi announced positive topline results from the Phase 3 VALIANT study investigating systemic pegcetacoplan in patients with C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis, which are rare kidney diseases with no approved treatments. The study met the primary endpoint, demonstrating a statistically significant and clinically meaningful 68% proteinuria reduction in C3G and IC-MPGN patients treated with pegcetacoplan compared to placebo, both in addition to background therapy, at Week 26. Results were consistent across all subgroups including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneys. Pegcetacoplan also demonstrated statistical significance on the key secondary endpoints of composite renal endpoint, which combines proteinuria reduction and estimated glomerular filtration rate stabilisation, and proteinuria reduction of at least 50% compared to baseline, as well as nominal significance on the histological endpoint of reduction in C3c staining on kidney biopsy and stabilisation of kidney function as measured by eGFR compared to placebo. Sobi plans to submit a marketing application with the European Medicines Agency in 2025. Apellis also plans to submit a supplemental new drug application to the FDA in early 2025. Detailed data will be presented at an upcoming medical congress.