Sobi and Apellis Pharmaceuticals have shared positive late-stage results for their targeted C3 therapy in rare kidney disease patients.
The phase 3 VALIANT trial has been evaluating the drug, pegcetacoplan, in patients with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
There are currently no treatments that target the underlying cause of either disease 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.
VALIANT met its primary endpoint, demonstrating a 68% proteinuria (excess of protein in the urine) reduction in patients treated with pegcetacoplan compared to placebo, both in addition to background therapy, at week 26.
Results were consistent among adult and adolescent patients, and those with native and post-transplant kidneys, the companies noted.
Pegcetacoplan also showed statistical significance on secondary endpoints including a composite measure combining proteinuria reduction and estimated glomerular filtration rate stabilisation.
VALIANT lead principal investigator Carla Nester, University of Iowa Stead Family Children’s Hospital, said: “Currently, many patients living with these rare diseases will eventually require a kidney transplant or lifelong dialysis, so there is an urgent need for a treatment that targets the underlying cause of these diseases. This positive data is a major advance for the rare kidney disease community.”
All patients who have completed the VALIANT study have now been enrolled into the VALE long-term extension study.
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.
Lydia Abad-Franch, head of research and development, medical affairs and chief medical officer at Sobi, said: “[This] announcement further strengthens our belief in pegcetacoplan’s potential to meet the critical needs of patients with these severe and life-threatening kidney conditions. We remain committed to progressing pegcetacoplan’s development and expanding its reach…”
Sobi said it plans to submit a marketing application for the drug with the European Medicines Agency in 2025, while Apellis is aiming to submit a supplemental new drug application to the US Food and Drug Administration in early next year.
