Sobi and Apellis Pharmaceuticals, Inc. announced that positive results from the Phase 2 NOBLE study investigating pegcetacoplan for the treatment of post-transplant recurrence of primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G) will be presented at the American Society of Nephrology (ASN) Kidney Week Annual Meeting. The results showed the potential for a treatment effect in both IC-MPGN and C3G patients treated with pegcetacoplan. At Week 12, of the 10 patients (IC-MPGN: n=2; C3G: n=8) treated with pegcetacoplan: Eight (80%) patients showed a reduction in C3c staining (reflective of damage-causing deposits) by one or more orders of magnitude of intensity from baseline. Five (50%) patients showed a reduction in C3c staining bytwo or more orders of magnitude of intensity from baseline.

Four (40%) patients showed zero staining intensity, indicating that C3 deposits were cleared. Excessive deposition of C3 breakdown products in the kidney lead to inflammation and damage of the kidney, often causing kidney failure. Additionally, in 12 weeks pegcetacoplan showed a mean reduction of proteinuria (39.2% change from baseline), which is a key marker of disease progression, in a subgroup of patients with high baseline levels (=1g per day).

Other biomarkers also improved, including an increase in mean serum C3, reduction in mean serum C5b-9 and stabilisation of kidney function, as measured by estimated glomerular filtration rate (eGFR). There were no discontinuations due to treatment-emergent adverse events. The Phase 3 VALIANT study investigating pegcetacoplan in adolescent and adult patients with native and post-transplant recurrence of IC-MPGN and C3G is ongoing, with top-line results expected in 2024. Data from the Phase 2 DISCOVERY study investigating pegcetacoplan in patients with C3G was recently published in KI Reports.

Development for pegcetacoplan for IC-MPGN and C3G is being led by Apellis.