Apellis Pharmaceuticals (NASDAQ:APLS) saw its shares surge 16.3% in premarket trading on Thursday following the announcement of positive topline results from the Phase 3 VALIANT study.
Conducted in collaboration with Swedish biotech company Sobi, the study evaluated the efficacy of pegcetacoplan in treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), both rare and severe kidney diseases with no approved treatments.
The VALIANT study met its primary endpoint, demonstrating a statistically significant 68% improvement in patients treated with pegcetacoplan compared to placebo.
The drug also showed significant benefits in key secondary endpoints, including a reduction in proteinuria by at least 50% and stabilization of kidney function as measured by estimated glomerular filtration rate (eGFR).
The companies said the study's results were consistent across all subgroups, including both adolescent and adult patients, and those with native or post-transplant kidneys.
"These results exceeded our already high expectations. Pegcetacoplan is the first investigational therapy to show such a strong reduction in proteinuria in C3G and IC-MPGN with supportive data across multiple measures of disease activity," said Jeffrey Eisele, Chief Development Officer at Apellis. "Building on pegcetacoplan's approval in PNH, we look forward to sharing these data with the FDA and working quickly to bring this treatment to patients with these debilitating kidney diseases."
The safety profile of pegcetacoplan was also favorable, with adverse events comparable between the treatment and placebo groups.
Following the promising results, Sobi and Apellis plan to submit data for regulatory approval in both the U.S. and the EU in early 2025. The companies aim to bring this potentially transformative treatment to market as quickly as possible, offering new hope to patients suffering from these debilitating conditions.