Apellis and Sobi’s Aspaveli Application Validated by EMA for Rare Kidney Diseases
Apellis and Sobi’s Aspaveli application gains crucial validation, aiming to tackle severe kidney conditions.
Overview of Aspaveli’s Regulatory Progress
Apellis Pharmaceuticals and Sobi announced that the European Medicines Agency has validated the extension application for Aspaveli (pegcetacoplan) to treat C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). These are both serious rare kidney diseases that currently lack approved treatment options. This validation is a follow-up to the positive findings from the Phase 3 VALIANT study, which reported significant reductions in proteinuria and stabilized kidney function in patients using pegcetacoplan. Previously authorized for another condition, the drug is also under review in the U.S., with a forecasted launch as early as late 2025. The partnership seeks to meet an urgent need for effective therapies in this patient group.
Positives to Consider
- The validation from the EMA marks a critical advancement for expanding the treatment applications of Aspaveli® (pegcetacoplan) for rare kidney conditions.
- Results from the Phase 3 VALIANT study revealed a notable 68% reduction in proteinuria among pegcetacoplan patients, confirming its effectiveness.
- The urgent need for treatments for C3G and IC-MPGN, as highlighted in the release, positions the companies as influential players in a critical healthcare sector.
- If approved by the EMA, the move could lead to commercial opportunities in Europe, bolstering the company’s capabilities beyond its previous approvals for other conditions, like paroxysmal nocturnal hemoglobinuria.
Challenges Ahead
- Despite EMA validation, the absence of existing treatments for C3G and IC-MPGN underscores high unmet medical needs, raising concerns about the industry’s ability to handle these complexities.
- The release contains cautionary statements about the unpredictability of regulatory approvals and commercial success, highlighting potential risks in future outcomes for pegcetacoplan.
- The partnership with Sobi assigns exclusive commercialization rights outside the U.S. to Sobi, potentially limiting revenue streams for Apellis in those markets.
Frequently Asked Questions
What is the EMA’s stance on Aspaveli®?
The EMA has validated an application for Aspaveli® to extend its indications to treat C3 glomerulopathy and IC-MPGN.
Can you explain C3G and IC-MPGN?
C3G and IC-MPGN are rare, serious kidney diseases that can progress to kidney failure, necessitating dialysis or transplantation.
How does pegcetacoplan perform in clinical trials?
The Phase 3 VALIANT study demonstrated that pegcetacoplan led to a significant 68% reduction in proteinuria compared to placebo.
When might pegcetacoplan be available in the U.S.?
If approved, pegcetacoplan could potentially launch in the U.S. for C3G and IC-MPGN by the second half of 2025.
Who are the partners involved with pegcetacoplan?
Apellis Pharmaceuticals and Sobi have partnered for the global co-development and commercialization of pegcetacoplan.
Disclaimer: This is an AI-generated summary from a GlobeNewswire press release. There may be inaccuracies in this summary. For the complete release, click here.
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Full Release
WALTHAM, Mass. and STOCKHOLM, Sweden, Feb. 20, 2025 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi
®
(STO:SOBI) today announced the European Medicines Agency (EMA) has validated an indication extension application for Aspaveli
®
(pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), both of which are rare and life-threatening kidney diseases without approved treatments.
Sobi and Apellis Move Closer to Treatment Approval for Rare Kidney Diseases
Significant Study Results Provide Hope for Patients in Europe and Beyond
Kidney conditions can often lead to severe complications, such as kidney failure, necessitating lifelong treatment options like kidney transplants or dialysis. Dr. Lydia Abad-Franch, Sobi’s head of R&D and chief medical officer, emphasized that the absence of approved treatments makes their recent milestone even more significant for patients facing these rare kidney diseases.
The positive momentum comes from the Phase 3 VALIANT study where pegcetacoplan was evaluated. This study achieved its primary goal, showing a remarkable 68% reduction in proteinuria in patients treated with pegcetacoplan versus those receiving a placebo, with a strong statistical significance (p<0.0001) at Week 26. Notably, the effectiveness was consistent regardless of disease type, age, or transplant status.
Patients treated with pegcetacoplan also showed stabilization in kidney function, marked by improvements in their estimated glomerular filtration rate (nominal p=0.03). Furthermore, a significant number of those patients experienced a decrease in C3c staining intensity, indicating a positive biological response (nominal p<0.0001). The treatment maintained a favorable safety profile, aligning with existing data.
Dr. Jeffrey Eisele, chief development officer at Apellis, expressed the critical need for approved therapies for C3 Glomerulopathy (C3G) and Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) that extend kidney function. The validation from the European Medicines Agency brings them closer to providing a solution for patients in Europe. They are also working toward potential U.S. approval of pegcetacoplan for these conditions, targeting the second half of 2025.
Previously, the European Commission authorized Aspaveli in 2021, aimed at treating paroxysmal nocturnal hemoglobinuria.
Detailing the VALIANT Study
The VALIANT Phase 3 study (
NCT05067127
) involved a randomized, placebo-controlled, double-blinded design across multiple centers, assessing pegcetacoplan’s efficacy and safety in 124 patients aged 12 and older diagnosed with C3G or primary IC-MPGN. It stands as the largest single trial for these diseases, including both adolescent and adult participants with native and post-transplant kidneys. Patients received pegcetacoplan or placebo bi-weekly for 26 weeks, followed by a 26-week open-label phase during which all received pegcetacoplan. The study primarily focused on urine protein-to-creatinine ratio (UPCR) changes at Week 26 compared to baseline.
Understanding C3 Glomerulopathy (C3G) and Primary IC-MPGN
C3G and primary IC-MPGN are rare, severe kidney disorders that can lead to complete kidney failure. They are marked by excessive C3c deposits, which contribute to kidney inflammation, damage, and eventual failure. Currently, no treatments effectively address the underlying causes. About 50% of individuals diagnosed with C3G and IC-MPGN may face kidney failure within five to ten years, necessitating transplants or lifelong dialysis. Alarmingly, 90% of patients who have had a kidney transplant may see a return of the disease. Together, these conditions are estimated to affect approximately 5,000 individuals in the U.S. and up to 8,000 in Europe.
About Pegcetacoplan
Pegcetacoplan is a targeted therapy addressing C3, aiming to regulate the complement cascade linked to various severe health issues. It is currently under investigation for several rare diseases, including those affecting the kidneys and blood systems. Pegcetacoplan has received approval for treating paroxysmal nocturnal hemoglobinuria (PNH) under the names EMPAVELI® in the U.S. and Aspaveli® in the E.U. and elsewhere.
Apellis and Sobi’s Collaboration Explained
Apellis and Sobi hold global co-development rights for pegcetacoplan, with Sobi managing commercialization outside the U.S. Meanwhile, Apellis retains exclusive U.S. rights and oversees worldwide rights related to ocular conditions, such as geographic atrophy.
About Apellis
Apellis Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing innovative therapies for challenging diseases. The company has pioneered a new class of complement medicine with two authorized treatments targeting the C3 protein. Their first therapy for geographic atrophy aims to combat a leading cause of blindness worldwide. More information can be found at apellis.com.
About Sobi
Sobi® specializes in biopharmaceutical solutions for rare diseases, ensuring access to innovative treatments in hematology, immunology, and specialty care. With around 1,800 employees globally, Sobi’s revenue reached SEK 26 billion in 2024 and is publicly traded on Nasdaq Stockholm (STO:SOBI). For more, visit sobi.com.
Forward-Looking Statements
This announcement includes statements regarding future expectations and plans that could be considered “forward-looking statements.” Terms such as “anticipate,” “believe,” and “expect” may indicate these forward-looking statements. Actual outcomes could differ significantly due to various factors, including the approval process from regulatory bodies for pegcetacoplan. For more details, refer to Apellis’ Annual Report and other SEC filings.
Media Contacts
Lissa Pavluk
media@apellis.com
617-977-6764
Investor Relations
Meredith Kaya
meredith.kaya@apellis.com
617-599-8178
Contact Information for Sobi
Click here for Sobi Investor Relations Team contacts. For media inquiries, click here.
Sources
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.
This article was initially published on Quiver News; see the complete story.
The views expressed here are those of the author and do not necessarily reflect Nasdaq, Inc.’s views.
