Kidney Week

Abstract: TH-OR070

Pegcetacoplan for 76 Weeks Maintains Proteinuria Reduction and eGFR Stabilization for Pediatric Patients: VALIANT+VALE Subgroup Analysis

Session Information

• Glomerular Diseases: Epidemiology and Real-World Outcomes
  October 22, 2026 | Location: Room 501, Convention Center
  Abstract Time: 05:30 PM - 05:40 PM

Category: Glomerular Diseases

• 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics

Authors

• Dixon, Bradley P., University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States

Bradley P. Dixon, MD, FASN

• Greenbaum, Larry A., Emory University School of Medicine, Atlanta, Georgia, United States

Larry A. Greenbaum, MD, PhD

• Ariceta Iraola, María Gema, Hospital Universitari Vall d'Hebron, Barcelona, CT, Spain

María Gema Ariceta Iraola, MD, PhD

• Borovitz, Yael, Schneider Children's Medical Center of Israel, Petah Tikva, Center District, Israel

Yael Borovitz, MD

• Mastrangelo, Antonio, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardy, Italy

Antonio Mastrangelo

• Melhem, Nabil, Evelina London Children's Hospital, London, England, United Kingdom

Nabil Melhem

• Fujita, Naoya, Aichi Shoni Hoken Iryo Sogo Center, Obu, Aichi Prefecture, Japan

Naoya Fujita, MD, PhD

• Van De Kar, Nicole, Radboud universitair medisch centrum, Nijmegen, GE, Netherlands

Nicole Van De Kar, MD, PhD

• Wallace, Dean, Royal Manchester Children's Hospital, Manchester, England, United Kingdom

Dean Wallace, MD, MBBS

• Khankin, Eli, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States

Eli Khankin

• Mukherjee, Anwesha, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States

Anwesha Mukherjee, PhD

• Surova, Elena, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden

Elena Surova, MD, PhD

• Szamosi, Johan, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden

Johan Szamosi, MSc

• Vivarelli, Marina, Ospedale Pediatrico Bambino Gesu IRCCS, Rome, Lazio, Italy

Marina Vivarelli, MD

• Licht, Christoph, The Hospital for Sick Children, Toronto, Ontario, Canada

Christoph Licht, MD, FASN

• Nester, Carla M., The University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States

Carla M. Nester, MD, MS, FASN

Background

C3 glomerulopathy (C3G) and primary immune-complex mediated membranoproliferative glomerulonephritis (IC-MPGN) are often diagnosed in childhood. In VALIANT (phase 3; NCT05067127), adolescents (12–17 years) who received pegcetacoplan (C3/C3b inhibitor) for C3G or primary IC-MPGN for up to 1 year achieved proteinuria reduction and estimated glomerular filtration rate (eGFR) stabilization. Patients continued pegcetacoplan in the subsequent open-label extension (VALE; NCT05809531).

Methods

Fifty-five adolescents started the VALIANT study. Results from 53 adolescents who received pegcetacoplan for 76 weeks in VALIANT plus VALE were aligned by pegcetacoplan treatment duration. Efficacy endpoints 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 76 weeks had sustained proteinuria decrease (geometric mean change [95% CI] from baseline: –67.5% [–77.0, –54.1]) (Figure 1A). Results were consistent with proteinuria decreases achieved at week 26 (–68.4%) and week 52 (–69.3%) of pegcetacoplan treatment. Adolescents achieved stable eGFR with 76 weeks of pegcetacoplan (least squares mean [SE] change from baseline: +8.4 [4.43] mL/min/1.73 m²) (Figure 1B). No new safety signals were identified.

Conclusion

The clinical efficacy of 76 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 – Apellis Pharmaceuticals, Inc and Sobi (Swedish Orphan Biovitrum AB)