Apellis to file with FDA after successful kidney drug trial

Apellis Pharmaceuticals recently announced favorable results from a late-stage clinical trial investigating the efficacy of their drug, pegcetacoplan, in treating two rare kidney disease. The drug, marketed as Empaveli for a rare blood disorder, showed significant benefits for patients with C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis. Specifically, pegcetacoplan reduced urine protein levels by 68% compared to a placebo, an important marker indicating potential organ benefit.

Based on these promising results, Apellis plans to submit an approval application to the Food and Drug Administration (FDA) early next year. Additionally, their partner Sobi aims to seek approval from European regulators in 2025. Following the announcement, Apellis's stock saw an initial increase of up to 13%.

Pegcetacoplan is a complement inhibitor and is a cornerstone of Apellis's business. The drug has already received approvals for treating paroxysmal nocturnal hemoglobinuria and geographic atrophy, a condition affecting the eyes, where it is marketed as Syfovre. The eye condition indication was approved by the FDA earlier in 2023, and while initial U.S. sales were robust, they have been tempered by concerns over a rare but serious side effect. In Europe, regulators recommended against the drug's approval in June, although Apellis has sought a re-examination.

The unexpected success of pegcetacoplan in treating the kidney diseases C3G and IC-MPGN could shift the focus from its use in geographic atrophy to these other potential applications. Industry analysts highlighted that while the study in kidney indications was not highly anticipated, the results necessitate a broader discussion about the drug's potential. The 68% reduction in proteinuria reported for pegcetacoplan is notably higher than the 35% placebo-adjusted reduction achieved by Novartis's drug, iptacopan, in a similar patient population.

Analysts from Evercore ISI and Raymond James emphasized that the new data might force investors to reconsider their expectations for pegcetacoplan's market share. This reevaluation becomes particularly relevant as both pegcetacoplan and iptacopan are expected to compete in the market. Pegcetacoplan's robust performance in reducing proteinuria was consistent across various subgroups, including different disease types, age brackets, and patients with prior transplant history.

In the clinical trial, pegcetacoplan achieved a 66% reduction in urine protein levels versus placebo among the 96 participants with C3G, and a 74% reduction among the 28 participants with IC-MPGN. Secondary study endpoints also showed statistically significant improvements, and the rates of side effects and study discontinuation were comparable between the treatment and placebo groups.

Both C3G and IC-MPGN are characterized by kidney inflammation and damage. According to Apellis, approximately half of those diagnosed with these conditions experience kidney failure within five to ten years. Furthermore, among those who undergo a kidney transplant, about two-thirds see a recurrence of the disease. These disorders affect an estimated 5,000 individuals in the U.S. and 8,000 in Europe.

The promising data from Apellis's study could pave the way for new treatment options for these rare kidney diseases, potentially altering the landscape for patients and healthcare providers alike.

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