Sobi and Apellis Pharmaceuticals have shared positive new data from a late-stage study of their targeted C3 therapy in rare kidney disease patients.
The phase 3 VALIANT trial has been evaluating pegcetacoplan in over 120 patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
Results from the study’s open-label period, presented at this year’s European Renal Association Congress, showed that pegcetacoplan reduced excess protein in the urine (proteinuria) by 68% compared to placebo at week 26, with this benefit sustained at one year.
Pegcetacoplan-treated patients also continued to achieve stabilisation of kidney function, as measured by estimated glomerular filtration rate, and the drug demonstrated similar benefits in patients who switched from placebo to pegcetacoplan at the beginning of the open-label period.
C3G and primary IC-MPGN are estimated to affect 5,000 people in the US and up to 8,000 in Europe. Excessive deposits of the C3 protein are a key marker of disease activity in both conditions, and approximately 50% of patients experience kidney failure within five to ten years of diagnosis.
Pegcetacoplan is designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, and already holds approvals to treat the rare blood disorder paroxysmal nocturnal haemoglobinuria under the brand names Empaveli and Aspaveli.
Presenting author and co-lead principal investigator for VALIANT, Fadi Fakhouri, CHUV Lausanne, said: “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. This data further underscores the potential of [pegcetacoplan] to make a meaningful difference for patients.”
Pegcetacoplan has already been granted Priority Review designation by the US Food and Drug Administration for C3G and primary IC-MPGN, with a target action date of 28 July 2025. The European Medicines Agency has also validated an indication extension application for pegcetacoplan in both diseases.
Sobi has exclusive commercialisation rights for systemic pegcetacoplan outside the US, where Apellis has exclusive rights. Apellis also holds global commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.
