Sobi has announced the publication of results from the phase 3 VALIANT study, investigating pegcetacoplan for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), in The New England Journal of Medicine (NEJM).
“Pegcetacoplan has demonstrated substantial and clinically meaningful effects across all three key markers of disease: proteinuria reduction, estimated glomerular filtration rate (eGFR) stabilisation, and clearance of C3 deposits. Given the high risk of kidney failure, the need for dialysis and kidney transplant, these data are a welcome advance for patients with C3G and primary IC-MPGN,” said Fadi Fakhouri (University of Lausanne, Lausanne, Switzerland), lead author of the manuscript.
The data published in NEJM highlights the positive phase 3 VALIANT results at week 26, which were consistent across patients with C3G and primary IC-MPGN, adolescents and adults, and native and post-transplant kidney disease.
The study met its primary endpoint, demonstrating a statistically significant 68% (p<0.0001) proteinuria reduction in pegcetacoplan-treated patients compared to placebo. Pegcetacoplan-treated patients also achieved stabilisation of kidney function, with a difference of +6.3mL/min/1.73m2 compared to placebo (nominal p=0.03) as measured by eGFR.
A majority of pegcetacoplan-treated patients achieved a reduction in C3 staining intensity (nominal p<0.0001) with 71% of pegcetacoplan-treated patients achieved 0 C3 staining intensity, demonstrating complete clearance of C3 deposits. Pegcetacoplan showed favourable safety and tolerability in the VALIANT study, consistent with its established profile.
Lydia Abad-Franch, head of Research, Development, and Medical Affairs (RDMA) and chief medical officer at Sobi, said: “The phase 3 VALIANT data show the potential of pegcetacoplan for patients with C3G or primary IC-MPGN with consistent efficacy in both diseases and across patient groups, including adolescents and young adults where the conditions are most prevalent. We submitted the indication extension to the European Medicines Agency at the beginning of the year and hope to offer this new option to patients in Europe soon.”
Results from the VALIANT study at one year were recently presented at the European Renal Association (ERA) congress (4–7 June, Vienna, Austria) and at the American Society of Nephrology (ASN) Kidney Week 2025 (6–9 November, Houston, USA), showing sustained improvements in key markers of disease as well as favourable safety and tolerability.
An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected before year-end in the European Union (EU).
