Abstract: FR-PO0693
Pegcetacoplan for 52 Weeks Maintains Proteinuria Reduction and eGFR Stabilization in Pediatric Patients: Phase 3 VALIANT Subgroup Analysis
Session Information
- Pediatric Nephrology: CKD, ESKD, and Glomerular Diseases
November 07, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Pediatric Nephrology
- 1900 Pediatric Nephrology
Authors
- Greenbaum, Larry A., Emory University, Atlanta, Georgia, United States
- Nester, Carla M., University of Iowa Hospitals and Clinics, Iowa City, Iowa, United States
- Ariceta Iraola, María Gema, Hospital Universitari Vall d'Hebron, Barcelona, CT, Spain
- Dixon, Bradley P., University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States
- Borovitz, Yael, Schneider Children’s Medical Center of Israel, Petah Tikva, Israel
- Licht, Christoph, The Hospital for Sick Children, Toronto, Ontario, Canada
- Mastrangelo, Antonio, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardy, Italy
- Melhem, Nabil Z., Evelina London Children's Hospital, London, England, United Kingdom
- Fujita, Naoya, Aichi Children's Health and Medical Center, Aichi, Japan
- Van De Kar, Nicole, Radboudumc Amalia Children's Hospital, Nijmegen, Netherlands
- Wallace, Dean, Royal Manchester Children's Hospital, Manchester, England, United Kingdom
- Khankin, Eliyahu V., Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
- Mukherjee, Anwesha, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
- López Lázaro, Luis, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Szamosi, Johan, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Vivarelli, Marina, Ospedale Pediatrico Bambino Gesu IRCCS, Rome, Lazio, Italy
Background
C3 glomerulopathy (C3G) and primary immune-complex mediated membranoproliferative glomerulonephritis (IC-MPGN) are often diagnosed in childhood. In the 26-week randomized controlled period (RCP) of VALIANT (phase 3; NCT05067127), pegcetacoplan (C3/C3b inhibitor) for C3G and primary IC-MPGN led to significant proteinuria reduction vs placebo and estimated glomerular filtration rate (eGFR) stabilization for adolescents (12–17 years) and the overall population. In the following 26-week open-label period (OLP), all patients received pegcetacoplan.
Methods
28 and 27 adolescents were randomized to pegcetacoplan or placebo, respectively; 27 and 25 adolescents, respectively, entered the OLP. Efficacy end points included changes from baseline in proteinuria (measured as urine protein-to-creatinine ratio [UPCR]) and eGFR. Treatment-emergent adverse events were noted.
Results
Adolescents who received pegcetacoplan for 52 weeks had sustained proteinuria decrease (mean [95% CI] UPCR change from baseline: week 26, –73.6% [–83.1, –58.6]; week 52, –71.8% [–82.9, –53.6]) (Figure). Results for placebo-to-pegcetacoplan patients in the OLP were consistent with proteinuria decreases in the RCP for the pegcetacoplan group. Adolescents achieved stable eGFR throughout VALIANT (least squares mean [SE] change from baseline: week 26, +0.7 [3.6] mL/min/1.73 m2; week 52, +3.2 [4.1] mL/min/1.73 m2). No new safety signals were identified.
Conclusion
Clinical efficacy of 52 weeks of pegcetacoplan treatment for adolescents was consistent with the overall population of VALIANT, confirming its sustained benefit in broad patient populations.
Funding
- Commercial Support – This study was funded by Apellis Pharmaceuticals, Inc and Sobi (Swedish Orphan Biovitrum AB).