Apellis(US:APLS) Globenewswire·2025-10-20 11:00Core Insights - Apellis Pharmaceuticals announced new data from the Phase 3 VALIANT study, highlighting the sustained efficacy of EMPAVELI (pegcetacoplan) in treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) [1][4] Efficacy and Safety - EMPAVELI demonstrated durable reductions in proteinuria and stable kidney function over one year, with significant proteinuria reduction maintained at 68% compared to placebo [2][10] - The treatment showed favorable safety and tolerability, with no new safety signals reported [5] - EMPAVELI was superior to iptacopan in reducing proteinuria levels and achieving composite renal endpoints, with a greater proportion of patients achieving significant UPCR reductions [6][7] Study Design and Results - The VALIANT study was a randomized, placebo-controlled trial involving 124 patients aged 12 and older, making it the largest trial for these conditions [13] - The primary endpoint was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline [13] - Indirect treatment comparisons indicated that EMPAVELI was more effective than iptacopan in achieving proteinuria reduction and stabilization of estimated glomerular filtration rate (eGFR) [14][9] Patient Impact - Approximately one-third of patients treated with EMPAVELI achieved complete proteinuria remission, which was sustained through one year [10][9] - The treatment's efficacy was consistent across different patient groups, regardless of immunosuppressant use or baseline proteinuria levels [10][3] Market Position - EMPAVELI is the only approved treatment for C3G and primary IC-MPGN in patients aged 12 years and older, positioning it as a transformative option for patients at high risk of kidney failure [3][15] - The diseases affect an estimated 5,000 people in the U.S. and up to 8,000 in Europe, indicating a significant market opportunity for EMPAVELI [18]