Apellis and Sobi: EMPAVELI® Shows Yearlong Efficacy in Phase 3 C3G and IC-MPGN Study

Apellis Pharmaceuticals, Inc., in collaboration with Sobi®, unveiled promising results from their ongoing research into the drug EMPAVELI®, aimed at treating rare kidney conditions. At the European Renal Association Congress, findings from the Phase 3 VALIANT study were presented, showcasing the drug's efficacy in combating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). EMPAVELI demonstrated a significant reduction in proteinuria, the presence of excess protein in urine, when compared to a placebo. After 26 weeks, a 68% reduction was noted, and this benefit persisted at the one-year mark. Furthermore, EMPAVELI helped stabilize kidney function, as measured by estimated glomerular filtration rate (eGFR).

Dr. Fadi Fakhouri, a leading investigator in the study, emphasized the importance of these results, especially considering the severe nature of these diseases. Many affected individuals are at risk of kidney failure at a relatively young age. The data suggest that EMPAVELI could potentially provide a meaningful improvement in the lives of those affected by C3G and IC-MPGN. Patients initially on placebo and then switched to EMPAVELI experienced comparable benefits, further supporting the drug's effectiveness.

The study further highlighted EMPAVELI's robust safety profile, with no new concerns emerging during extended use. This assurance is vital for patients suffering from these rare kidney diseases. Dr. Peter Hillmen from Apellis expressed optimism regarding the drug's approval by regulatory bodies like the FDA, potentially allowing patients to access this treatment soon.

Dr. Nils Kinnman from Sobi underscored the urgent need for effective treatments in the realm of rare kidney diseases. The new study results reflect the commitment of both companies to advance therapies that address critical health challenges.

The congress highlighted eight presentations on rare kidney disease research, with six podium presentations focused on the VALIANT study. Two abstracts were distinguished as Top 10 ERA abstracts, showcasing the growing momentum in clinical research within this field.

C3G and primary IC-MPGN are debilitating kidney conditions characterized by excessive deposits leading to inflammation and potential kidney failure. Within five to ten years post-diagnosis, nearly half of those affected may face kidney failure, necessitating transplants or lifelong dialysis. The diseases affect thousands across the United States and Europe.

The VALIANT study is pivotal, being the largest ever conducted on these patient groups. It included diverse participants, from adolescents to adults, with both native and transplanted kidneys. The study initially employed a placebo-controlled approach, transitioning to an open-label phase where all participants received pegcetacoplan. The primary focus was on the protein-to-creatinine ratio in urine, a critical marker of kidney health.

Pegcetacoplan, the active ingredient in EMPAVELI, targets the C3 component of the complement system, a part of the immune response. It's under investigation for various rare conditions and already approved for treating paroxysmal nocturnal hemoglobinuria (PNH) in several regions, including the U.S. and European Union.

Apellis and Sobi have established a collaborative framework, sharing development responsibilities for pegcetacoplan globally, with specific commercialization rights divided geographically. Apellis retains exclusive rights in the U.S. for systemic pegcetacoplan and global commercial rights for its ophthalmological applications.

Apellis Pharmaceuticals, through its innovative approaches, strives to tackle challenging diseases by introducing novel therapies. Their commitment to addressing unmet needs in healthcare is evident through their pioneering work in complement medicine and other therapeutic areas. Meanwhile, Sobi continues its mission to transform care for those living with rare diseases, driven by breakthroughs that improve everyday life for patients worldwide.