APLS - Apellis to Present Positive Phase 2 NOBLE Results of Pegcetacoplan in Post-Transplant Recurrence of Primary IC-MPGN and C3G at Kidney Week | Benzinga
- In as early as 12 weeks:
- 80% of patients treated with pegcetacoplan showed a reduction in C3c staining by one or more orders of magnitude of intensity from baseline
- 40% of patients showed zero staining intensity, indicating that C3c deposits were cleared
- Pegcetacoplan-treated patients showed improvements across key clinical measures, including proteinuria, and stable kidney function
- No approved treatment for IC-MPGN and C3G, rare diseases that often lead to kidney failure
WALTHAM, Mass., Oct. 17, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Sobi® today 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 by two or more orders of magnitude of intensity from baseline.
- Four (40%) patients showed zero staining intensity, indicating that C3c deposits were cleared.
Excessive deposition of C3 breakdown products in the kidney leads to inflammation and damage of the kidney, often causing kidney failure.
"In as early as 12 weeks, these positive results show that pegcetacoplan has the capacity to clear the deposits that are causing kidney damage and may block future damage from occurring," said Andrew Bomback, M.D., presenting author and co-director of the center for glomerular diseases at Columbia University Irving Medical Center. "People living with post-transplant C3G and IC-MPGN have high rates of disease recurrence, creating a significant burden on patients both physically and emotionally. These data are very promising, especially given there are no approved treatments currently available."
Additionally, in 12 weeks, treatment with pegcetacoplan showed a mean reduction of proteinuria (39.2% change from baseline), which is a key marker of disease progression1, 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 sC5b-9, and stabilization of kidney function as measured by estimated glomerular filtration rate (eGFR). There were no discontinuations due to treatment-emergent adverse events.
"Importantly, 40% of patients showed zero C3c staining after 12 weeks, indicating that pegcetacoplan is targeting the underlying cause of the disease," Caroline Baumal, M.D., chief medical officer, Apellis. "These results continue to strengthen our confidence in pegcetacoplan as a potential treatment for both native and post-transplant forms of these rare and serious diseases."
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