Exploratory Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab (VAY RE-HIT)

This study is intended to explore the efficacy and safety of a second course of ianalumab after experiencing treatment failure in the pivotal Primary Immune Thrombocytopenia (ITP) trials (CVAY736I12301, CVAY736Q12301) and after loss of durable response in the pivotal Warm Autoimmune Hemolytic Anemia (wAIHA) trial (CVAY736O12301).

Start Date30 Jan 2026

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06711887

/ RecruitingPhase 3

An Open-label Extension Study to Assess the Efficacy and Safety of Ianalumab With or Without Study Treatment Withdrawal in Adult Participants With Lupus Nephritis Who Have Completed Study Treatment in the CVAY736K12301 Core Study (SIRIUS-LN Extension)

The purpose of this 2-year extension study is the evaluation of the efficacy and safety
1. after study treatment withdrawal in patients with lupus nephritis (LN) who achieved response (complete renal response [CRR] or partial renal response [PRR]) on double-blind treatment at the end of the SIRIUS-LN core study, and
2. of open-label ianalumab 300 mg treatment in patients who, at the end of the SIRIUS-LN core study, were either already receiving ianalumab open-label treatment or did not meet CRR/PRR criteria on double-blind treatment at the end of the SIRIUS-LN core study.

Start Date19 May 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

NCT06411639

/ WithdrawnPhase 1

A Multicenter, Open-label, Single-arm, Multiple-dose Study to Characterize the Pharmacokinetics, Safety and Tolerability of Subcutaneous Ianalumab in Chinese Adult Participants With Autoimmune Diseases

The purpose of this pharmacokinetic (PK) study is to describe the PK profile of ianalumab following s.c. administration in Chinese participants with systemic lupus erythematosus (SLE) and/or Sjögren's disease (SjD). Collection of intensive PK data from Chinese population had been designed in the ianalumab Phase 3 studies of SjD CVAY736A2302 (NCT05349214) and lupus nephritis (LN) CVAY736K12301 (NCT05126277) on an optional basis. This study is conducted to provide supplementary Chinese PK data in addition to the intensive PK data from the two Phase 3 studies .

Start Date25 Nov 2024

Sponsor / Collaborator

Novartis Pharmaceuticals Canada, Inc.

100 Clinical Results associated with Ianalumab

100 Translational Medicine associated with Ianalumab

100 Patents (Medical) associated with Ianalumab

Literatures (Medical) associated with Ianalumab

01 May 2025·Arthritis & Rheumatology

Safety and Efficacy of Ianalumab in Patients With Sjögren's Disease: 52‐Week Results From a Randomized, Placebo‐Controlled, Phase 2b Dose‐Ranging Study

Article

Author: Hueber, Wolfgang ; Fox, Robert ; Junge, Guido ; Schulze‐Koops, Hendrik ; Papas, Athena ; Luo, Wen‐Lin ; Bowman, Simon J. ; Grader‐Beck, Thomas ; Woznicki, Janice ; Moots, Robert J. ; Fisher, Benjamin A. ; De Vita, Salvatore ; Sopala, Monika ; Mariette, Xavier ; Barcelos, Filipe ; Avrameas, Alexandre ; Dörner, Thomas

Objective:

The objective of this study was to report 52‐week safety and efficacy of ianalumab from phase 2b dose‐finding study in patients with Sjögren's disease (SjD).

Methods:

Patients randomly received (1:1:1:1) ianalumab (5, 50, or 300 mg) or placebo subcutaneously every 4 weeks until week 24 (treatment period [TP]1). At week 24, patients on 300 mg were rerandomized to continue 300 mg or receive placebo until week 52 (TP2), patients on placebo were switched to ianalumab 150 mg, and patients on 5 and 50 mg directly entered posttreatment safety follow‐up. Patients who discontinued treatment early or completed treatment entered safety follow‐up (≥20 weeks).

Results:

During TP1, 190 patients were randomized (placebo = 49, 5 mg = 47, 50 mg = 47, 300 mg = 47). Of these 190 patients, 90 (47.4 %; 43 continued 300 mg and 47 received placebo) entered TP2, and 81 of 90 (90.0%) completed the study treatment. By week 52, efficacy was sustained in patients who continued 300 mg in TP2 (EULAR Sjögren's Syndrome Disease Activity Index, EULAR Sjögren's Syndrome Patient Reported Index, patient global assessment, and physician global assessment change from week 24: −1.45, −0.46, −4.69, and −6.86, respectively). Stimulated salivary flow rates and autoantibody levels numerically improved in the 300 mg group. Treatment‐emergent adverse events were not dose‐dependent, except for injection‐site reactions. Cases of decreased neutrophil counts (Common Terminology Criteria for Adverse Events v4.03 grade 3 according to laboratory listings) were observed in three patients during the posttreatment follow‐up, occurring at 3.5, 5.5, and 3 months, after the last ianalumab administration. None were associated with infection except one incidental finding of asymptomatic cytomegalovirus infection (IgM‐positive).

Conclusion:

In patients with SjD, ianalumab 300 mg demonstrated sustained efficacy through week 52 and a favorable safety profile up to two years of follow‐up.

01 Sep 2024·Expert Review of Hematology

Novel treatments for immune thrombocytopenia: targeting platelet autoantibodies

Review

Author: Arnold, Donald M. ; Harnett, Brian ; Al‐Samkari, Hanny ; Dalmia, Shreyash ; Al-Samkari, Hanny ; Arnold, Donald

INTRODUCTION:

Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by low platelets and an increased risk of bleeding. Platelet autoantibodies target major platelet glycoproteins and cause Fc-mediated platelet destruction in the spleen and reticuloendothelial systems. As mechanisms of disease, platelet autoantibodies are important therapeutic targets. Neonatal Fc receptor (FcRn) antagonists are a new class of therapeutics that reduce the half-life of immunoglobulin G including pathogenic platelet autoantibodies. Spleen tyrosine kinase (Syk) inhibitors interfere with Fc-mediated platelet clearance. Bruton's tyrosine kinase (BTK) inhibitors and B-cell activating factor (BAFF) inhibitors reduce antibody production. The efficacy of these targeted therapies provides new support for the role of platelet autoantibodies in pathogenesis of ITP even these antibodies can be difficult to detect.

AREAS COVERED:

This review includes an in-depth exploration of the pathophysiologic mechanisms of ITP, focusing on autoantibodies. Treatments outlined in this review include a) FcRn antagonists, b) complement inhibitors, c) B-cell directed therapies such as BTK inhibitors, and anti-BAFF agents, d) Syk inhibitors, e) plasma-cell directed therapies, and f) novel cellular therapeutic products.

EXPERT OPINION:

Platelet autoantibodies are often elusive in ITP, yet novel treatments targeting this pathway reinforce their role in the pathogenesis of this autoimmune platelet disorder.

01 Jun 2024·DEUTSCHE MEDIZINISCHE WOCHENSCHRIFT

Review

Author: Witte, Torsten ; Ernst, Diana ; Zehrfeld, Nadine

News (Medical) associated with Ianalumab

09 Dec 2025

·www.globenewswire.com

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

Ianalumab (9 mg/kg) plus eltrombopag extended ITP disease control by 45% with patients maintaining disease control 2.8 times longer than with placebo plus eltrombopag1,2 62% of patients treated with ianalumab plus eltrombopag achieved sustained platelet response at six months compared to 39% with placebo plus eltrombopag1,2Ianalumab, administered as four once-monthly intravenous doses in the ITP setting, has the potential to reduce the need for chronic treatment and deliver durable disease control in ITPNovartis plans to submit VAYHIT2 data from second-line ITP with results from ongoing first-line ITP trial, VAYHIT1, to health authorities in 2027 Basel, December 9, 2025 – Novartis today announced positive results from VAYHIT2, a Phase III trial evaluating ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids1-3. Ianalumab (9 mg/kg) plus eltrombopag extended ITP disease control by 45%, based on the primary endpoint of time to treatment failure (TTF), which assesses how long patients maintain safe platelet levels during and after the treatment period1,2. The median time to treatment failure for patients receiving ianalumab plus eltrombopag was 2.8 times longer than those on placebo plus eltrombopag (13.0 months vs. 4.7 months)1,2.Detailed data will be presented during the Late-Breaking Abstract Session at the 67th American Society of Hematology Annual Meeting & Exposition (ASH) and simultaneously published in The New England Journal of Medicine1,2.“Treatments for ITP have historically focused on raising platelet counts, often requiring chronic therapy to control ITP. This means many patients remain on treatment long-term, facing persistent disease burden and symptoms like fatigue,” said Hanny Al-Samkari, M.D., Peggy S. Blitz Endowed Chair in Hematology/Oncology, Mass General Brigham, and Associate Professor of Medicine, Harvard Medical School. “The VAYHIT2 trial results are encouraging, as they demonstrated improved disease control even while patients spend time off treatment, pointing toward possible progress for people living with ITP.”Patients receiving ianalumab (9 mg/kg) plus eltrombopag also achieved a significantly higher rate of sustained platelet count improvement at six months versus placebo plus eltrombopag (62% vs. 39%), meeting the key secondary endpoint1,2. Fatigue improvement, as measured by PROMIS Fatigue, showed a mean reduction of 7.7 points with ianalumab plus eltrombopag versus 3.6 points with placebo plus eltrombopag1,2.“B cells drive the autoimmune response that leads to platelet destruction and increased bleeding risk in ITP. The novel dual mechanism of action of ianalumab aims to deplete B cells while blocking their survival signals,” said Mark Rutstein, M.D., Global Head, Oncology Development, Novartis. “Guided by our decades-long experience advancing ITP care, the VAYHIT2 findings underscore the potential of ianalumab to deliver durable control with a short course of four once-monthly doses, offering patients the possibility of achieving disease stability without ongoing treatment.”Two doses of ianalumab were assessed in VAYHIT2 with ianalumab 9 mg/kg demonstrating statistically significant improvements across both the primary and key secondary endpoints, and ianalumab 3 mg/kg demonstrating statistically significant improvements in the primary endpoint and numerical improvements in the key secondary endpoint1-3. Ianalumab 9 mg/kg + eltrombopag (N=50) Ianalumab 3 mg/kg + eltrombopag (N=51) Placebo + eltrombopag (N=51) Primary endpoint: Time to treatment failure (TTF) 13.0 months (HR 0.55; 95% CI: 0.32, 0.92; p=0.021a) Not estimable (HR 0.58; 95% CI: 0.34, 0.98; p=0.023a) 4.7 months Key secondary endpoint: Stable response at 6 months (SR6) 62.0% (p=0.023a) 56.9% (p=0.035a) 39.2% a. Required p-value for statistical significance is one-sided <0.025Ianalumab was well tolerated with no new safety signals, and the side effect profile was consistent with previous studies1,2. 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 interruption1,2. No on-treatment adverse event led to permanent discontinuation of therapy1,2. VAYHIT2 marks the third positive Phase III trial with ianalumab, following two positive trials in adults with active Sjögren's disease1,4. 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 Agency5,6. *An adverse event of special interest encompassing several terms related to low levels of neutrophils, neutrophil precursors and leukocytesAbout 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 thrombocytopenia4,14-16. 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 life18,19. 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 use20,21. The burden of chronic treatment and unpredictability of relapses can significantly impact quality of life19,22. 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. DisclaimerThis 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 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.Cuker A, Stauch T,Cooper N et al. Ianalumab plus Eltrombopag in Immune Thrombocytopenia. N Engl J Med. DOI: 10.1056/NEJMoa2515168Clinicaltrials.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/NCT05653219Thomas 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.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=1018924European 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.htmClinicaltrials.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/NCT05648968Dörner 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.43059Clinicaltrials.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/NCT05350072Clinicaltrials.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/NCT05349214Clinicaltrials.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/NCT05639114Clinicaltrials.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/NCT05126277Clinicaltrials.gov. NCT06470048. A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis. Accessed November 14, 2025. https://clinicaltrials.gov/study/NCT06470048Bowman 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)02251-0Shen 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/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.htmlNovartis. 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-cashRodeghiero 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-2008-07-162503Kuter 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;87:558-61Kuter DJ. The treatment of immune thrombocytopenia (ITP)—focus on thrombopoietin receptor agonists. Ann Blood. 2021;6:27. doi:10.21037/aob-2021-itp-04Provan D, Newland AC. Current management of primary immune thrombocytopenia. Adv Ther. 2015;32(10):875-887. doi:10.1007/s12325-015-0240-zCooper 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.26083Mingot-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 # # # Novartis Media RelationsE-mail: media.relations@novartis.com Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com

Clinical ResultPhase 2Phase 3ASHOrphan Drug

09 Dec 2025

·www.fiercebiotech.com

ASH: Novartis details ianalumab\'s phase 3 win in rare blood disease

Novartis plans to submit ianalumab for FDA approval in primary immune thrombocytopenia in 2027, using data from both the VAYHIT2 and VAYHIT1 phase 3 trials.\n ORLANDO, Fla.—Novartis is divulging details from ianalumab’s summertime phase 3 win in a rare autoimmune blood disorder, and the data suggest the treatment could delay the progression of patients to more serious forms of the disease.Patients with primary immune thrombocytopenia (ITP) given ianalumab alongside Novartis’ Promacta (eltrombopag) saw their low platelet counts boosted to safe levels for 13 months after treatment, compared to just 4.7 months for patients given eltrombopag and placebo, translating into an extension of ITP disease control by 45%.This benefit was seen in patients given four monthly 9-mg/kg doses of Novartis’ combo therapy. A 3-mg/kg dose of the combo also achieved a statistically significant 42% improvement compared with the control group, even though the specific length of the measure—known as time to treatment failure—couldn’t be determined yet because the experimental treatment was still working in too many patients. Patients given the investigational combo also reported greater improvements in fatigue, a common symptom of ITP and a secondary endpoint in the trial.“The study achieved my high hopes and expectations,” Hanny Al-Samkari, M.D., a hematologist at Massachusetts General Hospital and Harvard Medical School who is an investigator for the trial, told Fierce Biotech.Al-Samkari presented the results from the trial, called VAYHIT2, at the American Society of Hematology (ASH) meeting in Orlando, Florida, on Dec. 9. Novartis announced the results in a press release on the same day.The results were also published in the New England Journal of Medicine on Dec. 9. Patients given ianalumab had slightly more infusion reactions than those given eltrombopag and placebo, Al-Samkari said, but most of these were mild to moderate in severity. A bigger safety question was whether patients given ianalumab would develop more infections, because the antibody is designed to deplete B cells to combat the autoimmune activity that destroys ITP patients’ platelets.Infections were no worse or more common in either ianalumab arm compared to placebo, Al-Samkari said. “That really helps us to feel comfortable that this is a safe medication to use.”ITP, which increases the risk of bruising and dangerous bleeding, can vary greatly. Of adults who develop the disease, 20% go into remission after one year, Al-Samkari explained, while 80% become chronic patients. And, of those with chronic disease, some experience only mild symptoms while others are frequently in and out of the hospital.The VAYHIT2 trial focused on patients who had been diagnosed in the last three or four months and been given first-line treatment—corticosteroids—and not had success. This design is bold and unusual in the field, Al-Samkari said, where other trials often focus on controlling disease in patients who have had ITP for years and failed multiple lines of treatment.“There was an ASH plenary last year for rilzabrutinib that is now approved in ITP as a chronic controller medication,” he added, referring to Sanofi’s late-line BTK inhibitor Wayrilz. “This drug is being evaluated totally differently.”Al-Samkari’s hope is that by resetting patients’ B cells, the ianalumab-eltrombopag combo can “defang” the disease, preventing recently diagnosed patients from progressing to more severe disease.“It’s promising what we’ve seen so far, but we need more time, another couple years,” he said. If the therapy proves successful at staving off disease progression, and if another trial testing the combo in first-line ITP, VAYHIT1, also scores a win, “it will be very strong evidence to change the treatment paradigm for this disease.”Novartis plans to submit ianalumab for FDA approval in ITP in 2027, according to the company’s Dec. 9 release, using data from both VAYHIT2 and VAYHIT1.The Swiss pharma originally announced its VAYHIT2 success in August, the day after sharing that ianalumab also succeeded in a pair of phase 3 trials for Sjögren’s syndrome, another B-cell-driven autoimmune disease. The company originally developed the antibody with MorphoSys, a German biotech Novartis went on to buy in 2024.The Big Pharma is also testing ianalumab in a host of other indications, including ongoing phase 3 trials in systemic lupus erythematosus, lupus nephritis and other diseases where rogue B cells target blood cells.Editor\'s note: This story was updated at 8:40 a.m. ET on Dec. 9 to include a link to the published study.

Phase 3Clinical ResultASHDrug Approval

09 Dec 2025

·endpoints.news

Novartis says antibody helps prolong control of rare blood disorder

Novartis said its latest experimental approach for a rare blood disorder prolonged patients’ disease control by 45% compared to a placebo arm. The Phase 3 results, which follow a topline readout in August, are for second-line patients with primary immune thrombocytopenia, or ITP. Patients who took the highest dose of Novartis’ ianalumab combined with the company’s already approved ITP treatment Promacta saw a median of 13 months before treatment failure. Patients who received a placebo instead of ianalumab saw a median of 4.7 months. The endpoint measured how long patients maintained safe platelet levels during and after their treatment. Patients received four once-monthly infusions of ianalumab along with Promacta, then all patients tapered off Promacta. For a cohort of patients who received a lower dose of ianalumab, the median time to treatment failure had not yet been reached. “It’s fantastic that we have a medication here, in ianalumab, that has shown in the pivotal Phase 3 study to be not only very safe, but very effective at giving people the long-term disease control,” Hanny Al-Samkari, an investigator on the trial, told Endpoints News in an interview . ITP occurs when B cells produce autoantibodies that attack and destroy platelets, potentially causing bleeding, bruising and chronic fatigue. Many patients cycle through treatments without getting long-term control of their disease. Ianalumab is a monoclonal antibody, unlike Promacta, which is a platelet-stimulating agent. It’s designed to deplete the B cells responsible for the issues that occur with ITP, as well as block their survival signals. “The goal of this study was actually to attack the disease in a totally different way,” Al-Samkari, a hematologist at Mass General Brigham Cancer Institute, said. By adding ianalumab to Promacta, Novartis hoped to “hit the disease early in the course” and give patients longer-lasting control of their condition. In the trial, ianalumab was given along with Promacta to patients who had failed first-line treatment with corticosteroids. At six months, 55% of the ianalumab patients and 26% of the placebo patients saw a complete response, meaning their disease was under “optimal control” with platelet counts “very close to normal,” Al-Samkari said. In addition, ianalumab outperformed placebo in reducing fatigue. Promacta was approved in 2008 for patients who had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. It generated $2.2 billion in 2024 sales. Novartis is also testing ianalumab combined with first-line corticosteroids in a Phase 3 study expected to read out next year. The company said it plans to submit the data from both trials to the FDA in 2027. Novartis originally developed ianalumab in collaboration with MorphoSys, which it acquired in 2024. Editor’s note: This piece has been updated to include Hanny Al-Samkari’s affiliation with Mass General Brigham Cancer Institute.

Clinical ResultPhase 3Drug Approval

100 Deals associated with Ianalumab

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KEGG	Wiki	ATC	Drug Bank
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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Systemic Lupus Erythematosus	Phase 3	Japan	China Novartis Institutes for BioMedical Research Co., Ltd.	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Japan	Novartis Pharma Stein AG	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Japan	Novartis Pharmaceuticals Corp.	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Japan	Novartis Pharma AG	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Brazil	China Novartis Institutes for BioMedical Research Co., Ltd.	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Brazil	Novartis Pharma Stein AG	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Bulgaria	Novartis Pharmaceuticals Corp.	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Canada	Novartis Pharma AG	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Canada	China Novartis Institutes for BioMedical Research Co., Ltd.	02 Mar 2023
Systemic Lupus Erythematosus	Phase 3	Canada	Novartis Pharma Stein AG	02 Mar 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05653219 (VAYHIT2, N Engl J Med \| Company_Website) Manual	Phase 3	Purpura, Thrombocytopenic, Idiopathic Second line	152	Ianalumab 9 mg + Eltrombopag	fjhhdmxaol(xhuewylxlx) = eohgawbrna ntucsrlesq (sasoaaquot, 39 - 67) View more	Positive	09 Dec 2025
				Ianalumab 3 mg + Eltrombopag	fjhhdmxaol(xhuewylxlx) = jahtziyqhi ntucsrlesq (sasoaaquot, 36 - 64) View more
NCT05653219 (VAYHIT2, ASH2025)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	152	Ianalumab 9mg/kg + Eltrombopag	juaifpiixe(kjyabdicpi) = jrgpyaucup xjwhuxqgcx (mhrmeojuxo ) View more	Positive	06 Dec 2025
				Ianalumab 3mg/kg + Eltrombopag	juaifpiixe(kjyabdicpi) = nsinxjmnzh xjwhuxqgcx (mhrmeojuxo ) View more
NCT05124925 (ACR2025)	Phase 2	Sjogren's Syndrome	21	Ianalumab 300 mg	rzzwtvrgjj(dvdxncuipr) = vxshksxaxm nczpbnymrh (bfkhrqkfdd )	Positive	24 Oct 2025
NCT05349214 (NEPTUNUS-2, Company_Website) Manual	Phase 3	Sjogren's Syndrome	504	ianalumab 300 mg s.c. monthly	bpnvqoxsfm(hhmxogfjdq) = met primary endpoint in patients with Sjögren’s disease. dxsgcmawxv (nadpjkoila ) Met View more	Positive	11 Aug 2025
				ianalumab every 3 months
NCT05350072 (NEPTUNUS-1, Company_Website) Manual	Phase 3	Sjogren's Syndrome	275	ianalumab 300 mg	reqadymjor(zgxflmhyak) = met primary endpoint in patients with Sjögren’s disease. luywuermuy (lapnulvpez ) Met View more	Positive	11 Aug 2025
				Placebo
NCT05885555 (PS2237, EHA2025)	Phase 2	Purpura, Thrombocytopenic, Idiopathic	10	Ianalumab	gexixjwngx(thgnjevbmr) = uspmwakyau zjrcgqwiua (iygsospoeb ) View more	Positive	14 May 2025
NCT05885555 (VAYHIT3, EHA2025)	Phase 2	Purpura, Thrombocytopenic, Idiopathic	41	Ianalumab 9 mg/kg	zoxrdobxmq(fnqbmnatfq) = 82% of pts experienced at least one adverse event xhxgnfhpcd (zrvvmxchfi ) View more	Positive	14 May 2025
NCT05885555 (ASH2024)	Phase 2	Purpura, Thrombocytopenic, Idiopathic	-	Ianalumab 9 mg/kg	qymqmndxcp(ojijonmibf) = all 10 patients (100%) and 3 patients (30%) experienced any-grade adverse events (AEs) and Grade ≥3 AEs, respectively. Six patients had infections and 2 patients had infusion-related reactions; all of these events were Grade 1 or 2. No patients discontinued treatment due to AEs. After Week 25 there was one death due to pulmonary edema, which was assessed as unrelated to ianalumab. No safety signal was detected up to data cutoff. aulgkjptir (fnhiaecrwm ) View more	-	09 Dec 2024
NCT03656562 (CVAY736X2208, EULAR 12024 \| EULAR 22024) Manual	Phase 2	Systemic Lupus Erythematosus	67	ianalumab 300 mg subcutaneous	siazvsuvqq(pynbjqrpem) = azipykwogo dmbdekvach (pwdrjslfch )	Positive	14 Jun 2024
				placebo	siazvsuvqq(pynbjqrpem) = tecxsnxsjr dmbdekvach (pwdrjslfch )
NCT03656562 (SIRIUS-SLE 1 & 2, and SIRIUS-LN, ACR2023)	Phase 2	Systemic Lupus Erythematosus ANA ≥1:80 \| SLEDAI-2K score ≥6 \| BILAG-2004 ≥1 A or ≥2 B scores	-	Ianalumab 300 mg	rraizpctjd(qbdqmiuslm) = jkexxurekg gfbehdahge (aejnmhbhbk, 0 - 30)	Positive	14 Nov 2023
				Placebo	rraizpctjd(qbdqmiuslm) = tfjpzmoynz gfbehdahge (aejnmhbhbk, 0 - 27.5)

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