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Sobi and Apellis Showcase Promising Efficacy of Aspaveli®

On June 6, 2025, global biopharmaceutical companies Sobi® and Apellis Pharmaceuticals, Inc. presented new findings from the open-label period of their Phase 3 VALIANT study. The investigation centered around Aspaveli® (pegcetacoplan) as a treatment for two rare and severe kidney diseases: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The data was unveiled during a late-breaking session at the esteemed European Renal Association (ERA) Congress.

The VALIANT study demonstrated a statistically significant 68% proteinuria reduction in patients treated with Aspaveli compared to those given a placebo by Week 26, with this remarkable improvement sustained through the one-year mark. Additionally, the treated patients experienced stabilization of kidney function, as evidenced by measured glomerular filtration rates (eGFR).

Dr. Fadi Fakhouri, who played a pivotal role in the study, remarked, "The one-year Phase 3 results are very compelling, confirming Aspaveli's sustained benefits across key markers of disease. Given the high risk of kidney failure, treatment efficacy is incredibly important to C3G and primary IC-MPGN patients, many of whom are in the prime of their lives. These data further underscore the potential of Aspaveli to make a meaningful difference for patients."

For patients who transitioned from placebo to Aspaveli during the open-label phase, similar benefits were reported regarding proteinuria reduction and kidney function stabilization. This suggests a robust efficacy profile that could significantly enhance patient care in this population.

Nils Kinnman, MD, PhD, Sobi's Head of Medical Affairs and Clinical Development, noted the significant implications of these results, stating, "The findings from the Phase 3 VALIANT study highlight the urgent needs of patients living with the kidney diseases C3G and primary IC-MPGN. This research exemplifies Sobi's dedication to advancing innovative therapies that can profoundly impact patients' lives."

Moreover, Peter Hillmen, M.B., Ch.B., Ph.D., Chief Medical Advisor for Rare Diseases at Apellis, emphasized the importance of the study's results, affirming, "These data reinforce the strength of the EMPAVELI efficacy and safety profile across a wide patient population suffering from C3G and primary IC-MPGN, including both adults and adolescents with their respective types of kidney disease. With an FDA decision pending this summer, we eagerly anticipate making EMPAVELI widely available for individuals living with these rare and often debilitating kidney diseases."

Throughout the congress, a total of eight presentations, including six on podium, highlighted substantial advancements in clinical research pertaining to rare kidney diseases. Notably, two abstracts from the VALIANT study were recognized among the 'Top 10' best presentations, underscoring the critical evolution within kidney disease research.

The Importance of C3 Glomerulopathy and IC-MPGN

C3 glomerulopathy and primary IC-MPGN are rare but debilitating kidney diseases that can lead to serious complications, including kidney failure. A key characteristic of these diseases is the excessive accumulation of C3, a component of the immune system, which can cause inflammation and damage to kidney tissues. Alarmingly, nearly half of the individuals diagnosed with these conditions may suffer kidney failure within just five to ten years, often necessitating a kidney transplant or lifelong dialysis. Furthermore, about 90% of kidney transplant recipients may encounter a recurrence of these diseases post-operation, which poses a significant challenge for ongoing patient care.

In the United States, approximately 5,000 individuals are affected by these conditions, with estimates suggesting up to 8,000 cases in Europe as well.

Insights on the VALIANT Study

The VALIANT study (NCT05067127) represents a groundbreaking initiative, being both randomized and placebo-controlled and involving 124 adult patients aged 12 or older diagnosed with C3G or primary IC-MPGN. Significantly, it is recognized as the largest trial conducted in these patient groups, uniquely integrating adolescent and adult participants with both native and post-transplant kidney disease. Each participant was randomized to receive either pegcetacoplan or a placebo bi-weekly for 26 weeks. Following the randomized phase, patients entered an open-label period, receiving pegcetacoplan regardless of their previous treatment. The primary endpoint focused on measuring the protein-to-creatinine ratio in urine after 26 weeks compared to baseline levels.

Pegcetacoplan's Role in Rare Diseases

Pegcetacoplan is a precision-targeted therapy aimed at moderating the over-activation of the complement cascade, a crucial aspect of the immune system implicated in the onset and progression of various serious diseases. This treatment is currently being explored for multiple rare conditions, particularly across the fields of hematology and nephrology. Pegcetacoplan has garnered approval for use against paroxysmal nocturnal hemoglobinuria (PNH) as EMPAVELI®/Aspaveli in the U.S., the European Union, and other regions globally.

Collaborative Efforts between Apellis and Sobi

Apellis and Sobi share global co-development rights for systemic pegcetacoplan. Sobi enjoys exclusive marketing rights outside the U.S., while Apellis holds full commercialization rights for the product in the United States, alongside global rights for ophthalmological applications of pegcetacoplan.

In Conclusion:
The presented findings from the Phase 3 VALIANT study signify a monumental advancement in the treatment landscape for individuals diagnosed with C3G and primary IC-MPGN. With the potential approval of EMPAVELI on the horizon, hope emerges for many affected by these challenging diseases. As research continues, the commitment of Sobi and Apellis to develop effective therapies shines brightly, promising a future where quality of life can be improved for patients grappling with rare kidney diseases.