Apellis Pharmaceuticals, Inc. announced positive one-year results from the Phase 2 NOBLE study investigating systemic pegcetacoplan, a targeted C3 therapy, for the treatment of post-transplant recurrence of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). These data were presented during a late-breaking oral presentation at the European Renal Association (ERA) Congress taking place May 23-26 in Stockholm, Sweden. At one year, of the 11 pegcetacoplan-treated patients with available data: Seven (64%) patients showed a reduction in C3c staining by two or more orders of magnitude of intensity from baseline.

Six (55%) patients, including the three IC-MPGN patients, showed zero C3c staining intensity, indicating that C3c deposits were cleared. Consistent with C3c staining reduction, seven patients (64%) showed zero inflammation as measured by the activity score of the C3G histologic index. Excessive C3c deposits are a marker of disease activity, which can lead to kidney inflammation, damage, and failure.

Clearance of both C3c deposits and inflammation allow the kidney to recover and prolong the function of the kidney. Additionally, treatment with pegcetacoplan resulted in sustained improvements in key measures of disease including proteinuria and continued stabilization of kidney function. Pegcetacoplan was generally well-tolerated.

The majority of adverse events were mild to moderate and consistent with previously reported results.