#Sweden #Stockholm #C3G #pegcetacoplan #IC-MPGN

Positive Outcomes from VALIANT Phase 3 Study

The New England Journal of Medicine (NEJM) has published promising results from the Phase 3 VALIANT study regarding pegcetacoplan, a treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). These are uncommon yet severe kidney diseases that necessitate innovative therapeutic options. This study is significant as it shows substantial benefits across multiple disease markers, thus marking a vital milestone in nephrology.

Key Findings of the VALIANT Study

The VALIANT study monitored 124 patients aged 12 and older, who exhibited either C3G or IC-MPGN. The patients were randomized into a double-blind approach, receiving either pegcetacoplan or a placebo, administered twice weekly for a period of 26 weeks. The study focused on three primary endpoints, which included:

• - Reduction in Proteinuria: The study met its primary endpoint with pegcetacoplan-treated patients demonstrating a significant 68% reduction in proteinuria (p < 0.0001) compared to the placebo group. This reduction indicates a decreased level of protein in urine, which is crucial in assessing kidney health.

• - Stabilization of Kidney Function: Pegcetacoplan was found to stabilize kidney function, showing a +6.3 mL/min/1.73 m² improvement in estimated glomerular filtration rate (eGFR) relative to the placebo (nominal p = 0.03). This stabilization is particularly important given the high risk of progression to kidney failure in these patients.

• - Reduction of C3 Deposits: A significant majority of patients undergoing treatment with pegcetacoplan experienced a reduction in C3 staining intensity, with 71% achieving zero C3 staining, indicating a complete clearance of harmful C3 deposits (nominal p < 0.0001).

Implications of the Findings

The results from the VALIANT study are promising for those suffering from C3G and primary IC-MPGN. Dr. Fadi Fakhouri, a nephrology professor at the University of Lausanne, emphasized the study's findings on the importance of reducing proteinuria and stabilizing kidney function, particularly due to the high likelihood of kidney failure in affected patients. The advancements noted could potentially lead to the decrease in the need for dialysis or kidney transplants, providing hope for improved patient outcomes.

Lydia Abad-Franch, the Chief Medical Officer at Sobi, pointed out the consistent efficacy observed across varying patient demographics, thus enhancing the collective understanding of pegcetacoplan's potential therapeutic role. This includes efficacy demonstrated among adolescents, where these conditions are prevalent.

Next Steps for Pegcetacoplan

The data accumulated from the VALIANT study has been submitted to regulatory authorities, including the European Medicines Agency (EMA), for review and potential approval for treatments targeting these kidney afflictions. An opinion from the Committee for Medicinal Products for Human Use (CHMP) within the EU is anticipated before the year's end.

As pegcetacoplan is already approved for paroxysmal nocturnal hemoglobinuria under the brand names Aspaveli/EMPAVELI, these new findings highlight its expanded utility in the field of nephrology. The collaboration between Sobi and Apellis focuses on not only C3G and IC-MPGN but also on its applications in ophthalmological settings, indicating a broad therapeutic horizon for pegcetacoplan.

The VALIANT study stands as a pivotal moment in the quest for treatments that can effectively address rare and severe kidney diseases. Continued hope for patients rests on the promising outcomes showcased and the collaborative efforts of researchers dedicated to improving patient care in this challenging medical landscape.