Remarkable Results Show Aspaveli's Impact on Kidney Diseases

Promising Results for Aspaveli in Treating Rare Kidney Conditions

Sobi and Apellis Pharmaceuticals have recently unveiled pivotal findings from the Phase 3 VALIANT study, focusing on the effectiveness of Aspaveli (pegcetacoplan) as a treatment for rare kidney diseases such as C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). These results were presented during a late-breaking session at a prominent European congress.

Aspaveli achieved a remarkable 68% reduction in proteinuria compared to placebo after 26 weeks, with this improvement maintained for an entire year. Not only does this signify a breakthrough in treatment efficacy, but it also indicates that patients treated with Aspaveli have shown stabilization in kidney function, measured by estimated glomerular filtration rate (eGFR).

Dr. Fadi Fakhouri, a lead investigator in the study and nephrology professor, expressed that these one-year results are pivotal, confirming the lasting benefits of Aspaveli across various disease markers. The efficacy of treatment is critically important for patients battling C3G and IC-MPGN, particularly since many of these individuals are at a significant risk of kidney failure.

For patients who transitioned from placebo to Aspaveli during the study's open-label phase, similar benefits were observed, showcasing the consistency of Aspaveli's efficacy.

Dr. Nils Kinnman, Head of Medical Affairs at Sobi, emphasized the importance of these results, highlighting the urgent need for effective therapies for patients with C3G and primary IC-MPGN. This study exemplifies Sobi's dedication to developing innovative treatments that significantly enhance patient care.

Furthermore, Dr. Peter Hillmen, Chief Medical Advisor at Apellis, stated that the data bolsters the strong efficacy and safety profile of EMPAVELI, which applies to a diverse patient demographic, including both adults and adolescents with native and post-transplant kidney ailments. With an upcoming FDA decision anticipated soon, Apellis aims to expedite the availability of EMPAVELI to those affected by these severe conditions.

The results also indicated that EMPAVELI/Aspaveli was well-tolerated by patients, aligning with its established safety profile and revealing no new safety concerns.

Highlighting Significant Advances in Kidney Disease Research

The forthcoming congress will showcase eight presentations, significantly contributing to the evolving landscape of kidney disease research, including six podium talks. These presentations will delve into clinically relevant outcomes from the VALIANT study, with two selected as among the Top 10 best abstracts, indicative of their importance to ongoing research efforts.

C3 Glomerulopathy and Primary IC-MPGN Overview

C3G and primary IC-MPGN are rare and debilitating kidney conditions that can rapidly lead to kidney failure. Key disease indicators include excessive deposits of C3, resulting in kidney inflammation and damage. Alarmingly, around 50% of individuals diagnosed with these conditions may face kidney failure within five to ten years, often requiring transplant or dialysis.

Insights into the VALIANT Study

The Phase 3 VALIANT study is the largest of its kind, involving 124 participants aged 12 and older with C3G or primary IC-MPGN. Participants were randomly assigned to receive either pegcetacoplan or a placebo over a 26-week period, followed by a 26-week open-label phase for all participants. The primary efficacy endpoint focused on the urine protein-to-creatinine ratio (UPCR) changes over the study duration.

Innovative Treatment with Pegcetacoplan

Pegcetacoplan is a targeted therapy designed to modulate the complement system's excessive activation, which plays a critical role in various severe diseases. Currently, pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) as EMPAVELI/Aspaveli in numerous global markets.

Apellis and Sobi's Strategic Partnership

The collaboration between Apellis and Sobi encompasses the global co-development of pegcetacoplan. While Sobi holds exclusive commercialization rights outside of the U.S., Apellis retains these rights within the U.S. for systemic applications and the global rights for ophthalmological treatments.

About the Companies

Sobi is a pioneering biopharmaceutical entity focused on sparking transformative solutions for patients dealing with rare diseases. With approximately 1,900 dedicated professionals, their expansive reach extends across Europe, North America, and beyond. Last year's revenue was a notable SEK 26 billion, and Sobi is publicly traded on Nasdaq Stockholm as STO:SOBI.

Apellis Pharmaceuticals integrates cutting-edge science and empathy to forge impactful therapies for challenging diseases. They have introduced the first new class of complement medicine in over a decade, developing therapies targeting C3 including treatments for prevalent conditions like geographic atrophy, which significantly contributes to blindness worldwide.

Frequently Asked Questions

What are the key findings from the VALIANT study?

The study demonstrated a 68% reduction in proteinuria with Aspaveli after 26 weeks, sustained over a year, along with stable kidney function in treated patients.

What is the significance of these results for patients?

These results are crucial for patients suffering from C3G and primary IC-MPGN, indicating a promising treatment option that may help prevent kidney failure.

How does pegcetacoplan work?

Pegcetacoplan works by regulating the complement system's overactivity, potentially reducing inflammation and damage in kidney diseases.

What is the expected timeline for FDA decisions?

An FDA decision regarding EMPAVELI is anticipated soon, which could facilitate quicker access for patients awaiting treatment.

What roles do Sobi and Apellis play in this development?

Sobi and Apellis are collaborating on the development and commercialization of pegcetacoplan, each focusing on specific geographic markets.

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