Abstract: FR-PO0836
Pegcetacoplan for 52 Weeks Maintains Proteinuria Reduction Regardless of Immunosuppressant Use or Nephrotic-Range Proteinuria at Baseline: VALIANT Subgroup Analysis
Session Information
- Glomerular Clinical Trials: From Data to Impact
November 07, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Nester, Carla M., The University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States
- Bomback, Andrew S., New York-Presbyterian/Columbia University Irving Medical Center, New York, New York, United States
- Ariceta Iraola, María Gema, Hospital Universitari Vall d'Hebron, Barcelona, CT, Spain
- Delmas, Yahsou, Bordeaux University Hospital, Bordeaux, France
- Dixon, Bradley P., University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States
- Gale, Daniel P., University College London, London, England, United Kingdom
- Greenbaum, Larry A., Emory School of Medicine, Atlanta, Georgia, United States
- Han, Seung Hyeok, Yonsei University College of Medicine, Seodaemun-gu, Seoul, Korea (the Republic of)
- Isbel, Nicole, The University of Queensland, Brisbane, Queensland, Australia
- Licht, Christoph, The Hospital for Sick Children, Toronto, Ontario, Canada
- Mastrangelo, Antonio, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardy, Italy
- Mizuno, Masashi, Nagoya Daigaku Daigakuin Igakukei Kenkyuka Igakubu, Nagoya, Aichi Prefecture, Japan
- de Holanda, Maria Izabel Neves, Hospital Federal de Bonsucesso, Ruschel Medicina, Rio de Janeiro, Brazil
- Pickering, Matthew C., Imperial College London, London, England, United Kingdom
- Remuzzi, Giuseppe, Istituto di Ricerche Farmacologiche Mario Negri, Milan, Lombardy, Italy
- Van De Kar, Nicole, Radboudumc Amalia Children's Hospital, Nijmegen, Netherlands
- Vivarelli, Marina, Ospedale Pediatrico Bambino Gesu IRCCS, Rome, Lazio, Italy
- Walker, Patrick D., Arkana Laboratories, Little Rock, Arkansas, United States
- Wallace, Dean, Royal Manchester Children's Hospital, Manchester, England, United Kingdom
- Zecher, Daniel, Regensburg University Hospital, Regensburg, Germany
- Le Quintrec, Moglie, Hopital Lapeyronie, Montpellier, Occitanie, France
- Borovitz, Yael, Schneider Children's Medical Center of Israel, Petah-Tikva, Center District, Israel
- Melhem, Nabil Z., Evelina London Children's Hospital, London, England, United Kingdom
- Fakhouri, Fadi, Universite de Lausanne, Lausanne, VD, Switzerland
Background
Pegcetacoplan (C3/C3b inhibitor) led to significant proteinuria reduction vs placebo in C3 glomerulopathy/primary immune-complex membranoproliferative glomerulonephritis in the 26-week randomized controlled period (RCP) of VALIANT (phase 3; NCT05067127). In following 26-week open-label period (OLP), all patients received pegcetacoplan. Here, we describe responses in key patient subgroups (immunosuppressant [IS] use, baseline proteinuria) during RCP and OLP.
Methods
59 of 63 pegcetacoplan-to-pegcetacoplan and 55 of 61 placebo-to-pegcetacoplan patients completed OLP. Efficacy end points included changes from baseline in proteinuria and estimated glomerular filtration rate (eGFR). Treatment-emergent adverse events were noted.
Results
Patients receiving pegcetacoplan for 52 weeks had sustained proteinuria decrease (mean [95% CI] change: week 26, –67.2% [–74.9, –57.2]; week 52, –67.2% [–75.8, –55.4]). Results were consistent with overall population regardless of IS use and baseline nephrotic proteinuria (Figure). eGFR was stable for overall pegcetacoplan-treated population (least squares mean [SE] change, mL/min/1.73 m2: week 26, –1.5 [2.2]; week 52, –3.7 [2.7]). No new safety signals were identified.
Conclusion
Proteinuria reductions in RCP were maintained regardless of baseline proteinuria and IS use, confirming pegcetacoplan’s durable and consistent efficacy. eGFR was stable. Safety was consistent with previous reports.