Sobi and Apellis' Pegcetacoplan Promising in Phase 3 Rare Kidney Disease Study

Sobi and Apellis Pharmaceuticals have reported encouraging outcomes from their advanced-stage clinical trial concerning a targeted C3 therapy for patients suffering from rare kidney diseases. The phase 3 VALIANT trial has been analyzing the efficacy of pegcetacoplan in individuals diagnosed with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).

At present, there are no treatments that specifically address the root cause of these conditions. Alarmingly, about half of the patients progress to kidney failure within a decade of their diagnosis. Pegcetacoplan aims to modulate the excessive activation of the complement cascade, a component of the immune system. This drug is already approved for treating paroxysmal nocturnal haemoglobinuria under the names Empaveli and Aspaveli.

The VALIANT trial achieved its primary goal by demonstrating a 68% reduction in proteinuria (an excess of protein in the urine) in patients treated with pegcetacoplan, compared to a placebo, both supplemented by background therapy, over a span of 26 weeks. Results were consistent across adult and adolescent patients, as well as those with native and post-transplant kidneys.

Additionally, pegcetacoplan exhibited statistical significance in secondary outcomes, which included a combined measure of proteinuria reduction and stabilization of the estimated glomerular filtration rate.

Carla Nester, the lead principal investigator of the VALIANT trial from the University of Iowa Stead Family Children’s Hospital, emphasized the urgent need for a treatment targeting the underlying cause of these diseases. She noted that many affected patients eventually require a kidney transplant or lifelong dialysis. She described the data as a significant step forward for the rare kidney disease community.

All participants who have completed the VALIANT trial have now been enrolled in the VALE long-term extension study.

Sobi holds exclusive commercialization rights for systemic pegcetacoplan outside the United States, whereas Apellis retains exclusive rights within the U.S. Furthermore, Apellis has global commercial rights for the ophthalmological applications of pegcetacoplan, including the treatment of geographic atrophy.

Lydia Abad-Franch, head of research and development, medical affairs, and chief medical officer at Sobi, expressed confidence in pegcetacoplan’s potential to address the critical needs of patients with severe and life-threatening kidney conditions. She reiterated Sobi’s commitment to advancing the development of pegcetacoplan and expanding its availability.

Sobi plans to submit a marketing application for the drug to the European Medicines Agency in 2025. Meanwhile, Apellis aims to file a supplemental new drug application with the U.S. Food and Drug Administration early next year.