Novartis ianalumab significantly extends disease control in patients with immune thrombocytopenia with only four once-monthly doses

_{a. Required p-value for statistical significance is one-sided <0.025}

Ianalumab was well tolerated with no new safety signals, and the side effect profile was consistent with previous studies^1,2. Adverse events were comparable between the ianalumab and placebo arms, with the most common AEs for ianalumab plus eltrombopag being headache (14% with 9 mg/kg, 10% with 3 mg/kg vs. 8% with placebo) and infusion-related reactions (14% with 9 mg/kg, 8% with 3 mg/kg vs. 8% with placebo)^1,2. Neutropenia* occurred more frequently in the ianalumab groups (16% with 9 mg/kg, 12% with 3 mg/kg) compared to placebo (2%) with most cases resolving without requiring treatment or dose interruption^1,2. No on-treatment adverse event led to permanent discontinuation of therapy^1,2.

VAYHIT2 marks the third positive Phase III trial with ianalumab, following two positive trials in adults with active Sjögren's disease^1,4. Novartis plans to submit the data from VAYHIT2 along with results from the ongoing first-line ITP trial, VAYHIT1, in 2027. Ianalumab has been granted Orphan Drug Designation by the US Food and Drug Administration and the European Medicines Agency^5,6.

*An adverse event of special interest encompassing several terms related to low levels of neutrophils, neutrophil precursors and leukocytes

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 survival⁸. In clinical trials, ianalumab showed promising efficacy and a favorable safety profile in Sjögren’s disease, systemic lupus erythematosus, and immune thrombocytopenia^4,14-16. Ianalumab originates from an early collaboration with MorphoSys AG, a company which Novartis later acquired in 2024¹⁷.

About primary immune thrombocytopenia 
Primary immune thrombocytopenia (ITP) is a rare, autoimmune disorder in which the immune system mistakenly targets and destroys platelets, the cells essential for blood clotting¹⁸. This can lead to symptoms such as prolonged bleeding, easy bruising and chronic fatigue, which can significantly impact daily life^18,19. 

Despite available treatments, many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control²⁰. Current options often focus on maintaining safe platelet levels and preventing bleeding complications and may require ongoing use^20,21. The burden of chronic treatment and unpredictability of relapses can significantly impact quality of life^19,22. There is a need for therapies that offer durable response while reducing the burden of long-term treatment²³. 

About VAYHIT2  
VAYHIT2 (NCT05653219) is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of two different doses of ianalumab versus placebo, in addition to eltrombopag, in adults with primary immune thrombocytopenia (ITP) (platelet count <30 G/L) who failed previous first-line treatment with corticosteroids³. Alongside eltrombopag, patients were randomized 1:1:1 to receive four once-monthly intravenous infusions of ianalumab at 3 mg/kg, ianalumab at 9 mg/kg or placebo³. The primary endpoint was time to treatment failure, which is defined as the time from randomization until either: a platelet count of less than 30 G/L later than 8 weeks from randomization; the need for rescue therapy later than 8 weeks from randomization; initiation of a new ITP treatment at any time; ineligibility or inability to taper/discontinue eltrombopag; or death³. The key secondary endpoint is the percentage of patients with a stable platelet count response at Month 6³. Other secondary endpoints include measures of depth and duration of platelet response as well as patient-reported outcomes that measure quality of life and fatigue, among other endpoints³.

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,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. 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 the investigational or approved products described in this press release 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 such products will be commercially successful in the future. In particular, our expectations regarding such products 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.

Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram.

References

1. Primary results from VAYHIT2, a randomized, double-blind, phase 3 trial of ianalumab plus eltrombopag versus placebo plus eltrombopag in patients with primary immune thrombocytopenia (ITP) who failed first-line corticosteroid treatment. Oral presentation at ASH Annual Meeting & Exposition: December 6-9, 2025.
2. Cuker A, Stauch T,Cooper N et al. Ianalumab plus Eltrombopag in Immune Thrombocytopenia. N Engl J Med. DOI: 10.1056/NEJMoa2515168
3. Clinicaltrials.gov. NCT05653219. A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids (VAYHIT2). Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05653219
4. Thomas GB, Xavier M, Stephanie F et al. Ianalumab demonstrates significant reduction in disease activity in patients with Sjögren’s Disease: Efficacy and safety results from two global Phase 3, randomized, placebo-controlled double-blind studies (NEPTUNUS-1 and NEPTUNUS-2). Presented at the American College of Rheumatology (ACR) Congress; October 24-29, 2025; Chicago, Illinois.
5. US Food and Drug Administration. Orphan drug designation: ianalumab—treatment of primary immune thrombocytopenia. Published February 13, 2025. Accessed November 14, 2025. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=1018924
6. European Commission. Community register of orphan medicinal products: ianalumab. Updated October 14, 2025. Accessed November 14, 2025. https://ec.europa.eu/health/documents/community-register/html/o3036.htm
7. Clinicaltrials.gov. NCT05648968. A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia (VAYHIA) Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05648968
8. Dörner T, Bowman SJ, Fox R, 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 Rheumatol. 2025;77(5):560-570. doi:10.1002/art.43059
9. Clinicaltrials.gov. NCT05350072. Two-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome (NEPTUNUS-1). Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05350072
10. Clinicaltrials.gov. NCT05349214. Three-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome (NEPTUNUS-2). Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05349214
11. Clinicaltrials.gov. NCT05639114. Phase 3 Study to Evaluate Two Regimens of Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 1) (SIRIUS-SLE 1). Accessed November 14, 2025. https://www.clinicaltrials.gov/study/NCT05639114
12. Clinicaltrials.gov. NCT05126277. Safety, Efficacy and Tolerability of Ianalumab Versus Placebo, Combination With SoC Therapy, in Participants With Active Lupus Nephritis (SIRIUS-LN). Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT05126277
13. Clinicaltrials.gov. NCT06470048. A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis. Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT06470048
14. Bowman SJ, Fox R, Dörner T, et al. Safety and efficacy of subcutaneous ianalumab (VAY736) in patients with primary Sjögren's syndrome: a randomised, double-blind, placebo-controlled, phase 2b dose-finding trial. Lancet. 2022;399(10320):161-171. doi:10.1016/S0140-6736(21)02251-0
15. Shen N, Ignatenko S, Gordienko A, et al. 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 [abstract]. Arthritis Rheumatol. 2023; 75 (suppl 9). Accessed November 14, 2025. https://acrabstracts.org/abstract/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/
16. Bradbury C, Elverdi T, Trautmann K, et al. A phase 2 study of ianalumab in patients with primary immune thrombocytopenia previously treated with at least two lines of therapy (VAYHIT3). HemaSphere. 2025;9(Suppl 1):Abstract S238. Presented at European Hematology Association (EHA) Congress, June 12‑15, 2025. Milan, Italy. Accessed November 14, 2025. https://library.ehaweb.org/eha/2025/eha2025-congress/4159389/charlotte.bradbury.a.phase.2.study.of.ianalumab.in.patients.with.primary.html
17. Novartis. Press release. Novartis to strengthen oncology pipeline with agreement to acquire MorphoSys AG for EUR 68 per share or an aggregate of EUR 2.7bn in cash. February 5, 2024. Accessed November 14, 2025. https://www.novartis.com/news/media-releases/novartis-strengthen-oncology-pipeline-agreement-acquire-morphosys-ag-eur-68-share-or-aggregate-eur-27bn-cash
18. Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009;113(11):2386-2393. doi:10.1182/blood-2008-07-162503
19. Kuter DJ, Mathias SD, Rummel M, et al. Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care. Am J Hematol. 2012;87:558-61
20. Kuter DJ. The treatment of immune thrombocytopenia (ITP)—focus on thrombopoietin receptor agonists. Ann Blood. 2021;6:27. doi:10.21037/aob-2021-itp-04
21. Provan D, Newland AC. Current management of primary immune thrombocytopenia. Adv Ther. 2015;32(10):875-887. doi:10.1007/s12325-015-0240-z
22. Cooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) World Impact Survey (I-WISh): impact of ITP on health-related quality of life. Am J Hematol. 2021;96(2):199-207. doi:10.1002/ajh.26083
23. Mingot-Castellano ME, Bastida JM, Caballero-Navarro G, et al. Novel therapies to address unmet needs in ITP. Pharmaceuticals (Basel). 2022;15(7):779. doi:10.3390/ph15070779

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