Positive One-Year Data from Phase 2 Study of Pegcetacoplan in Post-Transplant Recurrence of C3G and IC-MPGN Presented as Late-Breaker at ERA Congress

• Rapid reduction in disease activity seen at 12 weeks was sustained at one year
• 55% of patients showed zero C3c staining intensity, indicating that C3c deposits were cleared
• Highlighted as late-breaking oral presentation at the European Renal Association (ERA) Congress
  

WALTHAM, Mass. and STOCKHOLM, Sweden, May 24, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi (STO:SOBI) today 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.

“It is exciting to see that treatment with pegcetacoplan rapidly reduced disease activity in only 12 weeks, and the effects were sustained over the long term,” said Fadi Fakhouri, M.D., Ph.D., presenting author and professor of nephrology at CHUV, Lausanne, Switzerland. “Post-transplant C3G and IC-MPGN patients are likely to experience disease recurrence, putting them at risk of needing another kidney transplant or dialysis. There is a huge need for a treatment that targets the cause of these diseases, and I am very encouraged by these unprecedented results.”

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.

“The NOBLE data further indicate that pegcetacoplan is treating the underlying cause of these diseases by directly targeting C3,” said Caroline Baumal, M.D., chief medical officer, Apellis. “Our ongoing Phase 3 VALIANT study evaluates the potential of pegcetacoplan in all patients with these rare kidney diseases, and we look forward to sharing the topline results later this year.”