Abstract: SA-OR048
Pegcetacoplan for 52 Weeks Results in Sustained Proteinuria Reduction to Remission (≤0.5 g/g) and Normalization (≤0.2 g/g): Phase 3 VALIANT Trial
Session Information
- Glomerular Targeted Therapies: The New Era
November 08, 2025 | Location: Room 310A, Convention Center
Abstract Time: 04:50 PM - 05:00 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
- Fakhouri, Fadi, Universite de Lausanne, Lausanne, VD, Switzerland
- Gale, Daniel P., University College London, London, England, United Kingdom
- Bomback, Andrew S., New York-Presbyterian/Columbia University Irving Medical Center, New York, New York, United States
- Licht, Christoph, The Hospital for Sick Children, Toronto, Ontario, Canada
- Remuzzi, Giuseppe, Istituto di Ricerche Farmacologiche Mario Negri, Milan, Lombardy, Italy
- Van De Kar, Nicole, Radboudumc Amalia Children's Hospital, Nijmegen, Netherlands
- Gordon, Katie, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
- Mukherjee, Anwesha, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
- Szamosi, Johan, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Quintana, Lucia, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Pickering, Matthew C., Imperial College London, London, England, United Kingdom
Background
Natural history studies report improved outcomes in C3 glomerulopathy (C3G) and primary immune complex-membranoproliferative glomerulonephritis (IC-MPGN) with proteinuria <0.88 or 1 g/g or proteinuria reduction ≥50%. In the 26-week randomized controlled period (RCP) of VALIANT (phase 3; NCT05067127), pegcetacoplan (C3/C3b inhibitor) led to significant proteinuria reduction vs placebo in C3G/primary IC-MPGN. In the following 26-week open-label period (OLP), all patients received pegcetacoplan.
Methods
59 of 63 pegcetacoplan-to-pegcetacoplan and 55 of 61 placebo-to-pegcetacoplan patients completed 52 weeks of treatment. Efficacy end points included proteinuria change from baseline.
Results
At week 26, 20 (31.8%) pegcetacoplan-to-pegcetacoplan patients achieved complete remission (≤0.5 g/g); 11 (17.5%) achieved normalization (≤0.2 g/g) (Figure). Results were maintained at week 52. At week 52, 34 (54.0%) patients achieved ≥50% proteinuria reduction. Similar proportions of placebo-to-pegcetacoplan patients achieved remission and normalization compared to the pegcetacoplan group; 25 (41.0%) achieved ≥50% reduction.
Conclusion
Proteinuria reductions to normalization/remission achieved in the RCP were maintained by patients who received pegcetacoplan for 52 weeks, confirming pegcetacoplan’s sustained efficacy. Consistent results were achieved in placebo-to-pegcetacoplan patients in the OLP.