Apellis and Sobi Announce EMA Validation of Indication Extension Application for Aspaveli® (pegcetacoplan) for C3G and Primary IC-MPGN
Apellis Pharmaceuticals (APLS) and Sobi announced that the European Medicines Agency (EMA) has validated an indication extension application for Aspaveli® (pegcetacoplan) to treat C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare kidney diseases currently without approved treatments.
The application is supported by positive Phase 3 VALIANT study results, which showed a significant 68% reduction in proteinuria (p<0.0001) in pegcetacoplan-treated patients versus placebo at Week 26. The treatment demonstrated consistent results across subgroups, achieved kidney function stabilization (p=0.03), and showed reduced C3c staining intensity (p<0.0001). The safety profile remained favorable and consistent with previous findings.
If approved, pegcetacoplan could potentially launch in the U.S. for C3G and IC-MPGN in the second half of 2025.
Apellis Pharmaceuticals (APLS) e Sobi hanno annunciato che l'Agenzia Europea per i Medicinali (EMA) ha convalidato una domanda di estensione dell'indicazione per Aspaveli® (pegcetacoplan) per il trattamento della glomerulopatia da C3 (C3G) e della glomerulonefrite membranoproliferativa a complessi immuni primari (IC-MPGN), malattie renali rare attualmente senza trattamenti approvati.
La domanda è supportata dai risultati positivi dello studio VALIANT di Fase 3, che ha mostrato una significativa riduzione del 68% della proteinuria (p<0.0001) nei pazienti trattati con pegcetacoplan rispetto al placebo alla settimana 26. Il trattamento ha dimostrato risultati coerenti tra i sottogruppi, ha raggiunto la stabilizzazione della funzione renale (p=0.03) e ha mostrato una riduzione dell'intensità di colorazione C3c (p<0.0001). Il profilo di sicurezza è rimasto favorevole e coerente con i risultati precedenti.
Se approvato, il pegcetacoplan potrebbe potenzialmente essere lanciato negli Stati Uniti per C3G e IC-MPGN nella seconda metà del 2025.
Apellis Pharmaceuticals (APLS) y Sobi anunciaron que la Agencia Europea de Medicamentos (EMA) ha validado una solicitud de extensión de indicación para Aspaveli® (pegcetacoplan) para tratar la glomerulopatía por C3 (C3G) y la glomerulonefritis membranoproliferativa por complejos inmunes primarios (IC-MPGN), enfermedades renales raras que actualmente no tienen tratamientos aprobados.
La solicitud está respaldada por los resultados positivos del estudio VALIANT de Fase 3, que mostró una reducción significativa del 68% en la proteinuria (p<0.0001) en pacientes tratados con pegcetacoplan en comparación con placebo a la semana 26. El tratamiento demostró resultados consistentes entre subgrupos, logró estabilización de la función renal (p=0.03) y mostró una reducción en la intensidad de tinción de C3c (p<0.0001). El perfil de seguridad se mantuvo favorable y consistente con hallazgos previos.
Si se aprueba, el pegcetacoplan podría lanzarse potencialmente en EE. UU. para C3G y IC-MPGN en la segunda mitad de 2025.
Apellis Pharmaceuticals (APLS)와 Sobi는 유럽 의약품청(EMA)이 Aspaveli® (pegcetacoplan)의 C3 글로메룰로병증(C3G) 및 원발 면역 복합체 막증식성 사구체신염(IC-MPGN) 치료를 위한 적응증 연장 신청을 검증했다고 발표했습니다. 이들은 현재 승인된 치료법이 없는 희귀 신장 질환입니다.
이 신청은 3상 VALIANT 연구의 긍정적인 결과에 의해 뒷받침되며, pegcetacoplan 치료를 받은 환자에서 위약 대비 26주차에 단백뇨 68% 감소 (p<0.0001)를 보여주었습니다. 치료는 하위 그룹 전반에 걸쳐 일관된 결과를 나타내었고, 신장 기능 안정화(p=0.03)를 달성했으며, C3c 염색 강도가 감소함(p<0.0001)도 나타났습니다. 안전성 프로필은 이전 결과와 일관되게 유리한 상태를 유지했습니다.
승인될 경우, pegcetacoplan은 2025년 하반기에 C3G 및 IC-MPGN을 위해 미국에서 출시될 수 있습니다.
Apellis Pharmaceuticals (APLS) et Sobi ont annoncé que l'Agence européenne des médicaments (EMA) a validé une demande d'extension d'indication pour Aspaveli® (pegcetacoplan) afin de traiter la glomérulopathie C3 (C3G) et la glomérulonéphrite membranoproliférative à complexes immuns primaires (IC-MPGN), des maladies rénales rares actuellement sans traitements approuvés.
La demande est soutenue par des résultats positifs de l'étude VALIANT de phase 3, qui a montré une réduction significative de 68% de la protéinurie (p<0.0001) chez les patients traités par pegcetacoplan par rapport au placebo à la semaine 26. Le traitement a démontré des résultats cohérents dans tous les sous-groupes, a atteint une stabilisation de la fonction rénale (p=0.03) et a montré une réduction de l'intensité de coloration C3c (p<0.0001). Le profil de sécurité est resté favorable et cohérent avec les résultats précédents.
Si approuvé, le pegcetacoplan pourrait potentiellement être lancé aux États-Unis pour C3G et IC-MPGN dans la seconde moitié de 2025.
Apellis Pharmaceuticals (APLS) und Sobi haben angekündigt, dass die Europäische Arzneimittel-Agentur (EMA) einen Antrag auf Erweiterung der Indikation für Aspaveli® (pegcetacoplan) zur Behandlung der C3-Glomerulopathie (C3G) und der primären immun-komplexen membranoproliferativen Glomerulonephritis (IC-MPGN) validiert hat, seltene Nierenerkrankungen, für die derzeit keine zugelassenen Behandlungen existieren.
Der Antrag wird durch positive Ergebnisse der Phase-3-Studie VALIANT unterstützt, die eine signifikante Reduktion der Proteinurie um 68% (p<0.0001) bei mit pegcetacoplan behandelten Patienten im Vergleich zu Placebo in der Woche 26 zeigte. Die Behandlung zeigte konsistente Ergebnisse über die Subgruppen hinweg, erreichte eine Stabilisierung der Nierenfunktion (p=0.03) und zeigte eine verringerte C3c-Färbeintensität (p<0.0001). Das Sicherheitsprofil blieb günstig und konsistent mit früheren Ergebnissen.
Falls genehmigt, könnte pegcetacoplan möglicherweise in der zweiten Hälfte des Jahres 2025 in den USA für C3G und IC-MPGN auf den Markt kommen.
- Phase 3 VALIANT study met primary endpoint with 68% proteinuria reduction
- Demonstrated kidney function stabilization in treated patients
- Potential market expansion into new indications with no current approved treatments
- Consistent positive results across all patient subgroups
- Favorable safety and tolerability profile
- None.
Insights
The EMA validation for Aspaveli's indication extension represents a important development in the rare kidney disease space. The Phase 3 VALIANT study results are particularly compelling, with the 68% reduction in proteinuria being both statistically significant (p<0.0001) and clinically meaningful. To put this in perspective, proteinuria reduction of this magnitude typically correlates with significantly improved kidney survival rates and reduced progression to end-stage renal disease.
The market opportunity is substantial despite the rare disease classification. C3G and IC-MPGN patients currently face options, typically progressing to dialysis or kidney transplantation, which carry high healthcare costs. Aspaveli's potential approval would position it as the first targeted therapy for these conditions, potentially capturing a significant portion of this untapped market.
Several key factors enhance the commercial potential:
- Demonstrated efficacy across all patient subgroups, including transplant recipients, broadening the potential patient population
- Consistent safety profile with previous indications, reducing regulatory risks
- Established manufacturing and distribution infrastructure from existing PNH indication
- Potential U.S. approval in H2 2025, creating a near-term catalyst
The Apellis-Sobi partnership leverages complementary strengths, with Sobi's established rare disease presence in Europe potentially accelerating market penetration. This indication extension could significantly expand Aspaveli's revenue potential, building upon its existing PNH franchise and strengthening both companies' positions in the rare disease market.
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), which are rare, chronic kidney diseases with no approved treatments.
“C3G and IC-MPGN are severe and life-threatening kidney conditions, often leading to kidney failure and requiring a kidney transplant or dialysis for life,” said Lydia Abad-Franch, M.D., head of R&D and medical affairs and chief medical officer at Sobi. “With no approved treatments currently available, this important milestone reflects our dedication to improving outcomes for those affected by these rare kidney conditions.”
The submission includes positive data from the Phase 3 VALIANT study. The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful
Additionally, pegcetacoplan-treated patients achieved stabilization of kidney function (nominal p=0.03), as measured by estimated glomerular filtration rate, and a substantial proportion of patients achieved a reduction in C3c staining intensity (nominal p<0.0001). Pegcetacoplan showed favorable safety and tolerability, consistent with its established profile.
“There is an urgent need for an approved treatment for C3G and IC-MPGN that can prolong kidney function,” said Jeffrey Eisele, Ph.D., chief development officer at Apellis. “The EMA validation leads us one step closer toward potentially bringing this treatment to European patients in need. Additionally, we continue to advance the regulatory process in the U.S., with a potential launch of pegcetacoplan for C3G and IC-MPGN in the second half of 2025, if approved.”
Aspaveli first received a marketing authorization from the European Commission in 2021 for the treatment of paroxysmal nocturnal hemoglobinuria.
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 are 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 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 C3c deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. There are no treatments that target the underlying cause of these diseases. Approximately
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 nephrology and hematology. 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 specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 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 27, 2024 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.
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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
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What conditions is Apellis seeking approval for with the new Aspaveli indication?
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