Abstract: INFO15-TH
Pegcetacoplan for Adolescents and Adults with C3 Glomerulopathy or Primary Immune-Complex Membranoproliferative Glomerulonephritis: Enrollment Status of the VALE Extension
Session Information
- Informational Posters - 1
November 06, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- No subcategory defined
Authors
- Khankin, Eliyahu V., Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
- Nester, Carla M., The University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States
- Ariceta Iraola, María Gema, Universitat Autonoma de Barcelona, Barcelona, CT, Spain
- Mukherjee, Anwesha, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States
- Szamosi, Johan, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- López Lázaro, Luis, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
Description
Background: C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) are rare diseases caused by C3 dysregulation that results in abnormal deposition of C3 breakdown products in the kidney, causing inflammation, kidney damage, and kidney failure. Pegcetacoplan (PEG) blocks overactivation of alternative, classical, and lectin complement pathways by binding to C3/C3b to regulate C3 cleavage and downstream activation. In the phase 3 VALIANT (NCT05067127) trial in patients with C3G or primary IC-MPGN, PEG reduced proteinuria by 68.1% (vs placebo), reduced C3c staining to 0 in 71.4% of patients, and stabilized eGFR at week 26; improvements were maintained at week 52. Here we describe the design and enrollment status of VALE (NCT05809531), a phase 3 extension study to evaluate long-term efficacy and safety of PEG in C3G and primary IC-MPGN.
Methods: Patients ≥12 years old who completed VALIANT through week 52 and had clinical benefit will continue twice-weekly doses of PEG for ~2.5 years or more (Figure). The primary endpoint is the ratio of urine protein-to-creatinine vs baseline. Secondary endpoints are changes in estimated glomerular filtration rate, proteinuria, patient-reported outcomes, and disease progression. Safety will be assessed.
Results: Of 114 patients who completed VALIANT, 113 enrolled in VALE. As of June 2025, 101 of the 113 (89%) patients who entered VALE remain in VALE. Results of this ongoing study are not yet available.
Conclusion: The ongoing VALE extension study assesses long-term efficacy and safety of PEG in a broad population of patients ≥12 years old with C3G or primary IC-MPGN.
Funding
- This study was funded by Apellis Pharmaceuticals, Inc and Sobi (Swedish Orphan Biovitrum AB).