Sobi and Apellis Pharmaceuticals report promising results for pegcetacoplan in phase 2 NOBLE study

Sobi and Apellis Pharmaceuticals, Inc. have announced encouraging one-year outcomes from the phase 2 NOBLE study, investigating the efficacy of systemic pegcetacoplan for treating post-transplant recurrence of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). These findings were presented at the European Renal Association (ERA) Congress, held from May 23-26 in Stockholm, Sweden.

Significant Reduction in Disease Activity

The study highlighted that pegcetacoplan rapidly decreased disease activity within 12 weeks and maintained these effects over an extended period. “It is exciting to see that treatment with pegcetacoplan rapidly reduced disease activity in only 12 weeks and sustained the effects over the long term,” remarked Fadi Fakhouri, M.D., PhD, the presenting author and professor of nephrology at CHUV Lausanne, Switzerland.

Clinical Outcomes of Pegcetacoplan Treatment

Of the 11 patients treated with pegcetacoplan who had available data after one year:

– Seven patients (64%) experienced a significant reduction in C3c staining intensity by two or more orders of magnitude from baseline.

– Six patients (55%), including all three IC-MPGN patients, achieved zero C3c staining intensity, indicating complete clearance of C3c deposits.

– Seven patients (64%) also showed no inflammation as measured by the activity score of the C3G histologic index.

These results are particularly noteworthy as excessive C3c deposits are indicative of active disease, which can lead to kidney inflammation, damage, and ultimately failure. The clearance of C3c deposits and inflammation underscores pegcetacoplan’s potential to recover kidney function and prolong its efficacy.

Advancing Treatment for Rare Kidney Diseases

“The NOBLE data further indicate that pegcetacoplan is treating the underlying cause of these diseases by directly targeting C3,” explained Caroline Baumal, M.D., Chief Medical Officer at Apellis. This targeted approach is critical in treating these rare and severe conditions effectively.

Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi, emphasized the importance of addressing the root cause of post-transplant C3G and IC-MPGN. “Patients often face disease relapse, so a therapeutic approach that addresses the root cause is crucial,” she stated.

Ongoing Research and Future Prospects

Apellis and Sobi are currently conducting the Phase 3 VALIANT study, which evaluates pegcetacoplan’s effectiveness across all patients with these rare kidney diseases. The companies anticipate releasing topline results later this year.

Pegcetacoplan has been generally well-tolerated, with the majority of adverse events being mild to moderate and consistent with previously reported outcomes. It is approved under the brand names EMPAVELI/Aspaveli for treating paroxysmal nocturnal hemoglobinuria (PNH) and is under investigation for other rare diseases in hematology and nephrology.