Sobi and Apellis announce positive topline results from phase 3 VALIANT study of pegcetacoplan in C3G and primary IC-MPGN

Core Insights - The Phase 3 VALIANT study demonstrated a statistically significant 68% reduction in proteinuria in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) treated with pegcetacoplan compared to placebo [1][2] - Results were consistent across various subgroups, including different age groups and kidney transplant statuses [1][2] - The companies plan to submit data for regulatory approval in the US and EU in early 2025 [1][4] Study Details - The VALIANT study was a randomized, placebo-controlled, double-blinded trial involving 124 patients aged 12 and older, making it the largest trial for these rare kidney diseases [5] - The primary endpoint was the log-transformed ratio of urine protein-to-creatinine ratio at Week 26, with additional key secondary endpoints showing significant results [2][5] - All patients who completed the study have enrolled in a long-term extension study [4] Safety and Efficacy - Pegcetacoplan showed a favorable safety profile, with adverse event rates similar between treatment and placebo groups [3][5] - No cases of meningitis or serious infections attributed to encapsulated bacteria were reported [3] Market Potential - C3G and IC-MPGN are rare diseases affecting approximately 5,000 people in the US and up to 8,000 in Europe, with a significant portion of patients facing kidney failure within 5 to 10 years of diagnosis [6] - Pegcetacoplan is already approved for treating paroxysmal nocturnal hemoglobinuria (PNH) and is being investigated for other rare diseases [7] Collaboration and Commercialization - Sobi and Apellis have global co-development rights for pegcetacoplan, with Sobi holding exclusive ex-U.S. commercialization rights [8] - Apellis plans to submit a supplemental new drug application to the FDA in early 2025 [4]