Abstract: INFO09-SA
Pegcetacoplan Use in Adults and Adolescents with FSGS: Rationale and Design of a Sequential Phase 2/3 Study
Session Information
- Informational Posters - 3
October 24, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- No subcategory defined
Authors
- Barratt, Jonathan, University of Leicester, Leicester, United Kingdom
- Riella, Cristian, Biogen Inc, Cambridge, Massachusetts, United States
- Chen, Yiming, Biogen Inc, Cambridge, Massachusetts, United States
- Min, Jinny, Biogen Inc, Cambridge, Massachusetts, United States
- Melnick, Joel, Biogen Inc, Cambridge, Massachusetts, United States
Description
Focal segmental glomerulosclerosis (FSGS) is a rare, glomerular disease, often resulting in nephrotic syndrome and kidney failure. Limited targeted therapies and poor prognosis highlight an unmet medical need. Evidence of complement activation in FSGS supports the evaluation of complement inhibition therapy. This study will assess the efficacy and safety of pegcetacoplan (a C3/C3b inhibitor) added to standard of care (SOC) in primary, genetic, or undetermined FSGS.
The single-arm, open-label, phase 2 portion (N~15) started ahead of randomization for the multicenter, placebo-controlled, double-blind, phase 3 portion (N=250). Both phases include 3 periods (Figure): ≤8-week screening, 104-week open-label (pegcetacoplan; phase 2) or 1:1 randomized controlled (pegcetacoplan:placebo; phase 3) period, and 8-week follow-up (~120 weeks of subcutaneous infusions twice weekly plus SOC). Main inclusion criteria are adults (≥18 years) or adolescents (12–17 years, phase 3 only) with primary, genetic, or undetermined FSGS diagnosed by kidney biopsy or pathogenic genetic variant (phase 3 only); ≥1.5 g/day proteinuria (24-hour urine) and ≥1.5 g/g urine protein-to-creatinine ratio (≥2 first-morning urine samples); estimated glomerular filtration rate ≥25 mL/min/1.73 m2; and stable SOC for ≥12 weeks. Patients with FSGS secondary to another condition are excluded. The primary efficacy endpoint is 12-week (phase 2) or 52- and 104-week (phase 3) proteinuria reduction.
Phase 2 screening began in 2025. A potential interim analysis (≥12 patients have completed ≥8 weeks) and the primary week-12 analysis will inform the conduct of phase 3.
This study assessing the efficacy and safety of pegcetacoplan plus SOC in adults and adolescents with FSGS will inform future treatment options.
Funding
- This study was funded by Apellis Pharmaceuticals, Inc, a subsidiary of Biogen