Novartis announces both ianalumab Phase III clinical trials met primary endpoint in patients with Sjögren's disease
Novartis (NYSE:NVS) announced significant breakthrough in Phase III clinical trials for ianalumab in treating Sjögren's disease. Both NEPTUNUS-1 and NEPTUNUS-2 trials met their primary endpoint, demonstrating statistically significant improvements in disease activity measured by EULAR Sjögren's syndrome disease activity index (ESSDAI).
Ianalumab, which features a dual mechanism of action through B-cell depletion and BAFF-R inhibition, could become the first targeted treatment approved for Sjögren's disease. The drug demonstrated a favorable safety profile and was well-tolerated in trials. Having received FDA Fast Track Designation, Novartis plans to present detailed trial data at an upcoming medical meeting and submit to health authorities globally.
Novartis (NYSE:NVS) ha annunciato una svolta significativa negli studi clinici di fase III su ianalumab per il trattamento della sindrome di Sjögren. Entrambi gli studi NEPTUNUS-1 e NEPTUNUS-2 hanno raggiunto il loro endpoint primario, mostrando miglioramenti statisticamente significativi dell'attività di malattia misurata dall'indice EULAR di attività della sindrome di Sjögren (ESSDAI).
Ianalumab, che presenta un doppio meccanismo d'azione tramite deplezione delle cellule B e inibizione del recettore BAFF (BAFF-R), potrebbe diventare il primo trattamento mirato approvato per la sindrome di Sjögren. Il farmaco ha mostrato un profilo di sicurezza favorevole ed è stato ben tollerato negli studi. Dopo aver ottenuto la designazione Fast Track della FDA, Novartis prevede di presentare i dati dettagliati degli studi a una prossima conferenza medica e di sottoporre le richieste di autorizzazione alle autorità sanitarie di tutto il mondo.
Novartis (NYSE:NVS) anunció un avance importante en los ensayos clínicos de fase III con ianalumab para el tratamiento del síndrome de Sjögren. Tanto NEPTUNUS-1 como NEPTUNUS-2 alcanzaron su objetivo primario, mostrando mejoras estadísticamente significativas en la actividad de la enfermedad medidas por el índice EULAR de actividad del síndrome de Sjögren (ESSDAI).
Ianalumab, que presenta un doble mecanismo de acción mediante la depleción de células B y la inhibición del receptor BAFF (BAFF-R), podría convertirse en el primer tratamiento dirigido aprobado para el síndrome de Sjögren. El fármaco mostró un perfil de seguridad favorable y fue bien tolerado en los ensayos. Tras recibir la designación Fast Track de la FDA, Novartis planea presentar datos detallados de los ensayos en una próxima reunión médica y presentar solicitudes ante las autoridades sanitarias a nivel mundial.
Novartis (NYSE:NVS)가 쇼그렌 증후군 치료를 위한 3상 임상시험에서 중요한 성과를 발표했습니다. NEPTUNUS-1과 NEPTUNUS-2 두 시험 모두 1차 평가변수를 충족했으며, EULAR의 쇼그렌 증후군 활동도 지수(ESSDAI)로 측정한 질병 활동도에서 통계적으로 유의한 개선을 보였습니다.
Ianalumab은 이중 작용 기전으로 B세포 제거와 BAFF 수용체(BAFF-R) 억제를 병행하며, 쇼그렌 증후군에 대해 최초의 표적 치료제로 허가받을 가능성이 있습니다. 약물은 안전성 프로필이 양호했고 임상에서 내약성이 우수했습니다. FDA의 패스트트랙 지정을 받은 상태로, 노바티스는 향후 학회에서 상세한 임상 데이터를 발표하고 전 세계 규제 기관에 허가 신청을 제출할 계획입니다.
Novartis (NYSE:NVS) a annoncé une avancée majeure dans les essais cliniques de phase III d'ianalumab pour le traitement du syndrome de Sjögren. Les essais NEPTUNUS-1 et NEPTUNUS-2 ont tous deux atteint leur critère principal, montrant des améliorations statistiquement significatives de l'activité de la maladie mesurées par l'indice EULAR d'activité du syndrome de Sjögren (ESSDAI).
Ianalumab, qui présente un double mécanisme d'action combinant la déplétion des cellules B et l'inhibition du récepteur BAFF (BAFF-R), pourrait devenir le premier traitement ciblé approuvé pour le syndrome de Sjögren. Le médicament a affiché un profil de sécurité favorable et a été bien toléré lors des essais. Ayant reçu la désignation Fast Track de la FDA, Novartis prévoit de présenter les données détaillées des essais lors d'une prochaine réunion médicale et de soumettre des demandes d'autorisation auprès des autorités de santé du monde entier.
Novartis (NYSE:NVS) gab einen bedeutenden Durchbruch in den Phase-III-Studien mit Ianalumab zur Behandlung des Sjögren-Syndroms bekannt. Sowohl NEPTUNUS-1 als auch NEPTUNUS-2 erfüllten den primären Endpunkt und zeigten statistisch signifikante Verbesserungen der Krankheitsaktivität, gemessen am EULAR-Index zur Aktivität des Sjögren-Syndroms (ESSDAI).
Ianalumab, das einen doppelten Wirkmechanismus durch B-Zell-Depletion und Hemmung des BAFF-Rezeptors (BAFF-R) aufweist, könnte das erste zielgerichtete zugelassene Medikament für das Sjögren-Syndrom werden. Das Präparat zeigte ein günstiges Sicherheitsprofil und wurde in den Studien gut vertragen. Nach Erhalt des Fast-Track-Status der FDA plant Novartis, detaillierte Studiendaten auf einer bevorstehenden medizinischen Tagung vorzustellen und weltweit bei Zulassungsbehörden einzureichen.
- Both Phase III trials successfully met primary endpoint for Sjögren's disease treatment
- Potential to become first-ever targeted treatment approved for Sjögren's disease
- Drug demonstrated favorable safety profile and was well-tolerated
- FDA Fast Track Designation already granted for ianalumab
- Detailed efficacy data and specific improvement metrics not yet disclosed
- Timeline for regulatory submissions and potential approval not specified
Insights
Novartis achieves breakthrough in Sjögren's disease treatment with successful Phase III trials of ianalumab, addressing significant unmet medical need.
The successful completion of the NEPTUNUS-1 and NEPTUNUS-2 Phase III trials represents a landmark achievement in Sjögren's disease treatment. These are the first global Phase III trials to demonstrate statistically significant reduction in disease activity for this condition, measured by the validated EULAR Sjögren's syndrome disease activity index (ESSDAI).
Ianalumab's dual mechanism of action - combining B-cell depletion with BAFF-R inhibition - addresses the underlying pathology of Sjögren's, an autoimmune condition characterized by lymphocytic infiltration of exocrine glands. This approach targets the disease process rather than just managing symptoms, which is the current standard of care.
The clinical significance cannot be overstated. Sjögren's disease affects approximately
The FDA Fast Track Designation highlights the recognized unmet need and could accelerate regulatory review. With plans to present at an upcoming medical congress and submit to health authorities globally, Novartis is positioning ianalumab to potentially become the first approved targeted therapy for Sjögren's disease - opening a new market segment with limited competition.
The favorable safety profile mentioned is particularly important for chronic autoimmune conditions requiring long-term treatment, though specific safety data details are pending full disclosure at upcoming medical meetings.
Ad hoc announcement pursuant to Art. 53 LR
- NEPTUNUS-1 and NEPTUNUS-2 are the first ever global Phase III trials to demonstrate statistically significant reduction in disease activity for Sjögren's disease1
- Ianalumab has the potential to become the first and only targeted treatment approved for patients with Sjögren's disease
- Ianalumab was well tolerated and demonstrated a favorable safety profile in Sjögren's disease1,2
- Novartis plans to present its data at an upcoming medical congress and submit to health authorities globally
"Sjögren's disease is a serious, progressive, systemic autoimmune disease, often unrecognized or misdiagnosed with a significant detrimental impact to quality of life, with very limited treatment options and an established unmet need. Both Phase III trials demonstrate that ianalumab improves disease activity in patients with Sjögren's disease," said Shreeram Aradhye, M.D., President of Development and Chief Medical Officer at Novartis. "These Phase III studies mark a significant milestone. We look forward to engaging with health authorities to discuss these findings in the near future."
The NEPTUNUS pivotal trials achieved the primary endpoint of improving disease activity measured by a reduction in EULAR Sjögren's syndrome disease activity index (ESSDAI), a multi-dimensional disease activity measurement compared to placebo1. Ianalumab was well tolerated and demonstrated a favorable safety profile in Sjögren's disease1,2.
Novartis plans to present the NEPTUNUS-1 and NEPTUNUS-2 data at an upcoming medical meeting and submit ianalumab, which was granted Fast Track Designation by the US Food and Drug Administration (FDA), to health authorities globally6.
About ianalumab
Ianalumab (VAY736) is a novel fully human monoclonal antibody being investigated for its potential to treat various B cell-driven autoimmune diseases, including Sjögren's disease, immune thrombocytopenia (ITP), systemic lupus erythematosus (SLE), lupus nephritis (LN), warm autoimmune hemolytic anemia (wAIHA) and diffuse cutaneous systemic sclerosis (dcSSc)3,7-13. Its mechanism of action targets B cells in two ways, namely combining B cell depletion via antibody-dependent cellular toxicity (ADCC) and interruption of BAFF-R mediated signals of B cell function and survival3. In clinical trials, ianalumab showed promising efficacy and a favorable safety profile in Sjögren's disease, systemic lupus erythematosus, and immune thrombocytopenia2,14,15. Ianalumab originates from an early collaboration with MorphoSys AG, a company which Novartis later acquired in 202416.
About NEPTUNUS-1 and NEPTUNUS-2
The Phase III clinical trials, NEPTUNUS-1 and NEPTUNUS-2, are global, multicenter, pivotal studies evaluating the efficacy and safety of ianalumab in patients with Sjögren's disease7,8. These trials were designed to provide comprehensive data on ianalumab's potential as a targeted treatment for Sjögren's disease, in patients with active extraglandular disease3,7,8.
NEPTUNUS-1 is a randomized, double-blind, 2-arm multicenter Phase III trial (N=275) to evaluate the clinical efficacy, safety, and tolerability of ianalumab 300 mg subcutaneous (s.c.) monthly compared with placebo for 52 weeks7. NEPTUNUS-2 is a randomized, double-blind, 3-arm multicenter Phase III trial (N=504) to evaluate the clinical efficacy, safety, and tolerability of ianalumab 300 mg s.c. monthly or every 3 months compared with placebo for up to 52 weeks8.
The primary endpoint was measured by improvements in systemic disease activity using ESSDAI (EULAR Sjögren's syndrome disease activity index)7,8. Patients currently enrolled in NEPTUNUS-1 and NEPTUNUS-2 have been given the opportunity to continue follow-up in these studies or enter a long-term extension trial17.
About Sjögren's disease (previously called Sjögren's syndrome)
Sjögren's disease is a systemic, chronic autoimmune disorder that causes inflammation and tissue damage, impacting the entire body4. It primarily affects exocrine glands, leading to excessive dryness, with over 90 percent of patients experiencing dry eyes and dry mouth4,18. The disease is heterogenous, patients experience dryness, fatigue and widespread pain and 30-40 percent of patients will also show extraglandular organ involvement5,19. Extraglandular manifestation can be very diverse and can affect skin, musculoskeletal system, kidneys, lungs and other organs19. The risk of lymphoma is increased in patients with Sjögren's5.
Sjögren's is one of the most prevalent rheumatic autoimmune diseases, affecting approximately 0.25 percent of the population with an estimated 50 percent undiagnosed 20-21. Sjögren's is nine times more common in women than men4. B cell dysfunction plays a significant role in the disease by causing an autoimmune response that leads to inflammation and tissue damage3,4. There are no systemic treatments approved, with only limited symptomatic treatments available to provide temporary and partial symptomatic relief, highlighting the need for effective targeted therapies3.
Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "may," "could," "would," "investigational," "pipeline," "upcoming," "intends," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for ianalumab, or regarding potential future revenues from ianalumab. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that ianalumab will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that ianalumab will be commercially successful in the future. In particular, our expectations regarding ianalumab could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
About Novartis
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.
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References
- Novartis data on file
- Bowman S et al, Safety and efficacy of subcutaneous ianalumab (VAY736) in patients with primary Sjögren's syndrome: a randomized, double-blind, placebo-controlled, phase 2b dose-finding trial, Lancet 2022; 399:161-71
- Dorner T et al, Safety and Efficacy of ianalumab in patients with Sjögren's disease:52-week results from a randomized, placebo-controlled, phase 2b dose-ranging study, Arthritis and Rheumatology 2025, 77(5):560-570
- Negrini S et al, Sjögren's syndrome: a systemic autoimmune disease, Clin Exp Med. 2022; 22(1): 9–25
- Mariette, Primary Sjögren's symptoms, New England Journal of Medicine, 2018, 378;10
- Ianalumab, Department of Health and Human Services, Fast Track Designation, US Food and Drug Administration, 2025
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- Phase 2 safety and efficacy of subcutaneous (s.c.) dose ianalumab (VAY736; Anti-BAFFR mAB) administered monthly over 28 weeks in patients with Systemic Lupus Erythematosus (SLE) of moderate-to-severe activity, ACR congress, available at: Phase 2 Safety and Efficacy of Subcutaneous (s.c.) Dose Ianalumab (VAY736; Anti-BAFFR mAb) Administered Monthly over 28 Weeks in Patients with Systemic Lupus Erythematosus (SLE) of Moderate-to-Severe Activity - ACR Meeting Abstracts [Last accessed: August 2025]
- A Phase 2 Study of ianalumab in patients with primary immune thrombocytopenia previously treater with at least two lines of therapy, EHA congress, available at: EHA Library - The official digital education library of European Hematology Association (EHA) [Last accessed: August 2025]
- Novartis to strengthen oncology pipeline with agreement to acquire Morphosys [AG Press release]. [Press release]. Available at: Novartis to strengthen oncology pipeline with agreement to acquire MorphoSys AG for
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- Maleki Fischbach M, et al, Manifestations and management of Sjögren's disease, Arthritis Res Ther. 2024;26(1):43
- Kerry Gairy et al, Burden of illness among subgroups of px with primary SjD and systemic involvement, Rheumatology 2021, Volume 60, Issue 4, April 2021, Pages 1871–1881
- Conrad N, et al, Incidence, prevalence, and co-occurrence of autoimmune disorders over time and by age, sex, and socioeconomic status: a population-based cohort study of 22 millions individuals in the
UK , Lancet. 2023;401(10391):1878-1890 - Narváez J et al, Prevalence of Sjögren's syndrome in the general adult population in
Spain : estimating the proportion of undiagnosed cases, Sci Rep. 2020;10(1):10627
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SOURCE Novartis Pharmaceuticals Corporation
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