Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

Summary

This is a Phase 3 study to assess the efficacy and safety of twice-weekly subcutaneous (SC) doses of pegcetacoplan compared to placebo in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN) on the basis of a reduction in proteinuria.

Timeline

Start

2021-11-12

Primary completion

2024-06-26

Completion

2025-01-14

Outcome

Met primary endpoint

registry analysis (superiority test); Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo; p <0.0001 NCT05067127 ↗

release “In the VALIANT study, Aspaveli demonstrated a statistically significant 68% proteinuria reduction versus placebo at Week 26, which was sustained at one year.” sobi.com ↗

release “The study met the primary endpoint, demonstrating a statistically significant and clinically meaningful 68% (p < 0.0001) proteinuria reduction” sobi.com ↗

Publications

• Vivarelli M, Ariceta G, Borovitz Y, Dixon BP, Greenbaum LA, Licht C, Mastrangelo A, Melhem N, Fujita N, van de Kar NCAJ, Wallace D, Khankin E, Wang Z, Lopez-Lazaro L, Szamosi J, Nester CM. Pegcetacoplan for Adolescents with C3 Glomerulopathy or Primary Immune Complex Membranoproliferative GN: Phase 3 VALIANT Subgroup Analysis. Clin J Am Soc Nephrol. 2026 May 14. doi: 10.2215/CJN.0000001077. Online ahead of print.
• Fakhouri F, Bomback AS, Ariceta G, Delmas Y, Dixon BP, Gale DP, Greenbaum LA, Han SH, Isbel N, Le Quintrec M, Licht C, Mastrangelo A, Mizuno M, Neves de Holanda MI, Pickering MC, Remuzzi G, Van De Kar N, Vivarelli M, Walker PD, Wallace D, Zecher D, Francois C, Deschatelets P, Li L, Wang Z, Abad-Franch L, Kinnman N, Lopez-Lazaro L, Szamosi J, Nester CM; VALIANT Trial Investigators Group. Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN. N Engl J Med. 2025 Dec 4;393(22):2210-2220. doi: 10.1056/NEJMoa2501510.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Pegcetacoplan	Peptide	540 mg	Subcutaneous
Subject	Pegcetacoplan	Peptide	648 mg	Subcutaneous
Subject	Pegcetacoplan	Peptide	810 mg	Subcutaneous
Subject	Pegcetacoplan	Peptide	1080 mg	Subcutaneous

Indications