Apellis Pharmaceuticals announced that the New England Journal of Medicine published the Phase 3 VALIANT study results for its systemic pegcetacoplan (EMPAVELI) on December 3, 2025. The study enrolled 124 patients with C3 glomerulopathy (C3G) or primary immune‑complex membranoproliferative glomerulonephritis (IC‑MPGN) and demonstrated a 68 % reduction in proteinuria, stabilization of kidney function, and clearance of C3 deposits, confirming the drug’s efficacy in these rare complement‑driven kidney diseases.
The clinical data translate into a meaningful benefit for a population that historically has limited treatment options. By reducing proteinuria and preserving glomerular filtration, EMPAVELI addresses the primary driver of progression to end‑stage kidney disease in C3G and IC‑MPGN, potentially delaying or eliminating the need for dialysis or transplant. The FDA approval of the drug for patients aged 12 and older on July 28, 2025, coupled with the NEJM publication, positions Apellis to capture a sizable share of the estimated 5,000‑to‑8,000‑patient market in the United States and Europe.
From a business perspective, the positive results reinforce Apellis’s strategy to expand EMPAVELI beyond its original indication for paroxysmal nocturnal hemoglobinuria. The drug’s broader label, which includes adolescents and post‑transplant patients, differentiates it from competitors such as Novartis’s Fabhalta and strengthens the company’s rare‑nephrology portfolio. Revenue from EMPAVELI in the U.S. reached $27 million in Q3 2025, while SYFOVRE, the company’s geographic atrophy therapy, generated $151 million, underscoring the importance of a diversified product mix.
Financially, Apellis reported a net income of $215.7 million for Q3 2025, largely driven by a $275 million upfront payment from Sobi and a $215 million one‑time payment from the same partner. This contrasts sharply with a net loss of $57.4 million in Q3 2024, illustrating the impact of licensing deals on profitability. Total revenue for Q3 2025 rose to $458.6 million from $196.8 million in the same quarter a year earlier, a 133 % year‑over‑year increase that reflects both product sales and licensing income.
CEO Cedric Francois emphasized that the company’s “strong momentum” is built on a pipeline that includes EMPAVELI in additional rare kidney diseases such as focal segmental glomerulosclerosis and delayed graft function. He highlighted the company’s confidence in maintaining profitability through disciplined cost management while investing in high‑return verticals, signaling a balanced approach to growth and financial stewardship.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.