Apellis and Sobi Announce EMPAVELI® (pegcetacoplan) Showed Sustained Efficacy at One Year in Phase 3 Study for C3G and Primary IC-MPGN
- Significant 68% proteinuria reduction maintained through one year
- Demonstrated stabilization of kidney function (eGFR)
- Similar benefits shown in patients switching from placebo to EMPAVELI
- Favorable safety profile with no new safety signals
- FDA decision expected this summer with applications under review at FDA and EMA
- None.
Insights
EMPAVELI shows sustained one-year efficacy for rare kidney diseases with FDA decision expected soon, potentially expanding Apellis's market.
The Phase 3 VALIANT study results for EMPAVELI (pegcetacoplan) demonstrate impressive sustained efficacy at the one-year mark for treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) – rare kidney diseases with high risk of progression to kidney failure. The data reveals a statistically significant 68% reduction in proteinuria versus placebo at Week 26, which importantly maintained through one year of treatment. Patients also showed stabilization of kidney function as measured by estimated glomerular filtration rate (eGFR).
What's particularly notable is the consistency of benefits observed across a broad patient population, including both adults and adolescents with native and post-transplant kidney disease. Patients who switched from placebo to EMPAVELI during the open-label extension demonstrated similar magnitude of benefits, reinforcing the drug's robust treatment effect.
The safety profile remains favorable with no new safety signals identified – critical for a chronic therapy in these indications. With EMPAVELI already approved for paroxysmal nocturnal hemoglobinuria (PNH), this potential label expansion represents significant progress for these underserved patient populations with limited treatment options. The FDA decision expected this summer and ongoing EMA review represent near-term catalysts that could meaningfully expand the commercial opportunity for this complement C3 inhibitor.
- Robust proteinuria reduction and stable kidney function were maintained across a broad population of patients
- No new safety signals were observed
- New data presented at late-breaking session at the European Renal Association Congress
- Marketing applications for EMPAVELI are under review with the FDA and EMA
WALTHAM, Mass. and STOCKHOLM, June 06, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi® (STO:SOBI) today presented new data from the open-label period of the Phase 3 VALIANT study, investigating EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The data were presented as part of a late-breaking session at the European Renal Association (ERA) Congress.
In the VALIANT study, EMPAVELI demonstrated a statistically significant
“The one-year Phase 3 results are very compelling, confirming EMPAVELI’s sustained benefits across key markers of disease,” said Fadi Fakhouri, M.D., Ph.D., presenting author, co-lead principal investigator for the VALIANT study, and professor of nephrology at CHUV Lausanne, Switzerland. “Given the high risk of kidney failure, treatment efficacy is incredibly important to C3G and primary IC-MPGN patients, many of whom are in the prime of their lives. These data further underscore the potential of EMPAVELI to make a meaningful difference for patients.”
In patients who switched from placebo to EMPAVELI at the start of the open-label period, EMPAVELI demonstrated a similar magnitude of benefit in proteinuria reduction and stabilization of kidney function.
“These data reinforce the strength of the EMPAVELI efficacy and safety profile across a broad population of patients with C3G and primary IC-MPGN, including adults and adolescents with native and post-transplant kidney disease,” said Peter Hillmen, M.B., Ch.B., Ph.D., chief medical advisor, rare disease, Apellis. “With an FDA decision this summer, we look forward to bringing EMPAVELI to patients living with these rare and severe kidney diseases as quickly as possible.”
EMPAVELI showed favorable safety and tolerability, consistent with its established profile. There were no new safety signals.
“The results from the Phase 3 VALIANT study underscore the potential of EMPAVELI in addressing the urgent needs of patients living with the kidney diseases C3G and primary IC-MPGN,” said Nils Kinnman, M.D., Ph.D., head of medical affairs and clinical development, Sobi. “These studies are an example of Sobi’s commitment to advance innovative therapies that make a meaningful difference in patients' lives.”
Eight presentations highlight substantial clinical advances in rare kidney disease
A total of eight presentations, including six on podium, will be highlighted at the meeting. The presentations will showcase clinically meaningful results from the Phase 3 VALIANT study, among other data. Additionally, two abstracts were selected by congress organizers as the Top 10 best ERA abstracts. The “Top 10” are deemed significant studies underlining the growing field of clinical research in kidney disease.
About C3 Glomerulopathy (C3G) and Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately
About the VALIANT Study
The VALIANT Phase 3 study (NCT05067127) is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients with native and post-transplant kidneys. Study participants were randomized to receive pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline.
About Pegcetacoplan in Rare Diseases
Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across hematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally.
About the Apellis and Sobi Collaboration
Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.
About Apellis
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit http://apellis.com or follow us on X (Twitter) and LinkedIn.
About Sobi®
Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn.
Apellis Forward-Looking Statement
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding plans to submit applications for regulatory approval for the treatment of patients with C3G and IC-MPGN. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether systemic pegcetacoplan will receive approval for those indications from the FDA or equivalent foreign regulatory agencies when expected or at all; and any other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2025 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
Contacts:
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Media
Tracy Vineis
media@apellis.com
617.420.4839
Investors
Neil Carnahan, CFA
neil.carnahan@apellis.com
617.977.5703
Sobi
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References
1. C3 glomerulopathy. National Institute of Health, Genetics Home Reference. https://ghr.nlm.nih.gov/condition/c3-glomerulopathy#resources. Accessed November 21, 2019.
2. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474.
3. Data on file using literature consensus.
FAQ
What were the key results of EMPAVELI's Phase 3 VALIANT study for C3G and IC-MPGN?
When is the FDA expected to make a decision on EMPAVELI (APLS) for C3G and IC-MPGN?
What conditions is EMPAVELI being tested for in the VALIANT study?
What was the safety profile of EMPAVELI in the Phase 3 VALIANT trial?
How did patients who switched from placebo to EMPAVELI perform in the study?
