Apellis And Sobi Unveil Promising Phase 3 VALIANT Results For Pegcetacoplan

In a significant breakthrough for patients with rare kidney conditions, Apellis Pharmaceuticals, Inc. and Sobi® (STO: SOBI) have unveiled encouraging topline results from their Phase 3 VALIANT study. This clinical trial explored the efficacy of systemic pegcetacoplan in treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), both of which currently lack approved therapies.

The VALIANT study achieved its primary goal, revealing a noteworthy 68% reduction in proteinuria (log-transformed ratio of urine protein-to-creatinine) for patients receiving pegcetacoplan compared to those on a placebo, in conjunction with standard background therapy, at Week 26 (p<0.0001). These results held steady across diverse patient demographics, encompassing both adolescent and adult groups, as well as individuals with native and post-transplant kidneys.

Moreover, pegcetacoplan showcased statistical significance in key secondary outcomes, including a composite renal endpoint that assessed both proteinuria reduction and the stabilization of estimated glomerular filtration rate (eGFR). Additionally, the treatment achieved a notable 50% reduction in proteinuria from baseline levels. While histological endpoints related to kidney biopsy results indicated nominal significance, eGFR stabilization compared to placebo further underscores pegcetacoplan’s potential as a game-changer for these challenging kidney diseases.

Carla Nester, M.D. MSA, FASN, lead principal investigator for the VALIANT study and Jean E. Robillard M.D., professor of paediatric nephrology, University of Iowa Stead Family Children's Hospital, said in a statement, “As a clinician, I’m thrilled by these groundbreaking results, which show that pegcetacoplan has the potential to significantly improve the lives of patients with C3G and IC-MPGN, regardless of disease type, age, and transplant status. Currently, many patients living with these rare diseases will eventually require a kidney transplant or lifelong dialysis, so there is an urgent need for a treatment that targets the underlying cause of these diseases. These positive data are a major advance for the rare kidney disease community.”

Jeffrey Eisele, Ph.D., chief development officer, Apellis, “These results exceeded our already high expectations. Pegcetacoplan is the first investigational therapy to show such a strong reduction in proteinuria in C3G and IC-MPGN with supportive data across multiple measures of disease activity. Building on pegcetacoplan’s approval in PNH, we look forward to sharing these data with the FDA and working quickly to bring this treatment to patients with these debilitating kidney diseases.”

Lydia Abad-Franch, M.D., head of R&D, medical affairs, and chief medical officer, Sobi, "Today’s announcement further strengthens our belief in pegcetacoplan’s potential to meet the critical needs of patients with these severe and life-threatening kidney conditions. We remain committed to progressing pegcetacoplan's development and expanding its reach, driven by our steadfast mission to transform the lives of those affected by rare diseases.”

The VALIANT study highlighted the favorable safety and tolerability of pegcetacoplan, reinforcing its previously established profile. Adverse event (AE) rates, including serious AEs and those leading to the discontinuation of the study drug, were comparable between the pegcetacoplan and placebo groups. Importantly, no instances of meningitis or severe infections due to encapsulated bacteria were reported.

All participants who have completed the VALIANT study have now enrolled in the VALE long-term extension study. Apellis is preparing to submit a supplemental new drug application to the U.S. Food and Drug Administration in early 2025, while Sobi plans to file a marketing application with European Medicines Agency in the same year. Detailed findings from the study are scheduled to be presented at an upcoming medical congress.