Eltrombopag Diolamine

BASEL, Switzerland I August 12, 2025 I Novartis today announced positive top-line results from VAYHIT2, a Phase III trial evaluating ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids 1,2 . Ianalumab plus eltrombopag, compared to placebo plus eltrombopag, significantly prolonged the time to treatment failure (TTF), the primary endpoint that assesses how long patients maintain safe platelet levels during and after the treatment period 1,2 . Ianalumab is being investigated in other B cell-driven autoimmune diseases, including ongoing Phase III trials in first-line ITP and in second and later lines of warm autoimmune hemolytic anemia, with readouts expected in 2026 3,4 . In VAYHIT2, patients treated with ianalumab plus eltrombopag experienced a significantly higher rate of sustained improvements in platelet count at six months, the key secondary endpoint of the study 1 . The safety profile of ianalumab was consistent with what was previously observed in clinical studies, with no new safety signals 1 . “While current treatments for ITP are generally effective in raising platelet counts, many patients require life-long treatment to maintain safe levels, which can create a lasting treatment burden,” said Adam Cuker, M.D., Professor of Medicine and Chief, Section of Hematology, University of Pennsylvania. “The results from VAYHIT2 are encouraging, as they suggest that ianalumab may support longer periods of disease control and reduce the need for continuous treatment.” ITP is a rare autoimmune disorder characterized by low platelet counts leading to an increased risk of bleeding, bruising and chronic fatigue 5-7 . Many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control 7 . There is a need for other treatment options with novel mechanisms of action that offer durable responses while reducing the burden of long-term treatment 8 . “For many people living with ITP, chronic treatment can disrupt their daily life due to the burden of regular dosing, dose adjustments and side effects,” said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. “These positive top-line results from the Phase III study highlight the potential of ianalumab, if approved, to deliver long-term disease control with four once-monthly doses and enable extended time off treatment.” Data is expected to be presented at an upcoming medical meeting and included in future regulatory submissions in 2027 along with results from the ongoing first-line ITP trial, VAYHIT1. Ianalumab has been granted Orphan Drug Designation by the US Food and Drug Administration and the European Medicines Agency 9,10 . Recently, Novartis announced positive top-line results for ianalumab in adults with active Sjögren’s disease. 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) 2,4,11-16 . 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 survival 11 . In clinical trials, ianalumab showed promising efficacy and a favorable safety profile in Sjögren’s disease, systemic lupus erythematosus, and immune thrombocytopenia 17-19 . Ianalumab originates from an early collaboration with MorphoSys AG, a company which Novartis later acquired in 2024 20 . 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 clotting 5 . This can lead to symptoms such as prolonged bleeding, easy bruising and chronic fatigue, which can significantly impact daily life 5,6 . Despite available treatments, many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control 7 . Current options often focus on maintaining safe platelet levels and preventing bleeding complications and may require ongoing use 7,21 . The burden of chronic treatment and unpredictability of relapses can significantly impact quality of life 6,22 . There is a need for therapies that offer durable response while reducing the burden of long-term treatment 8 . 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 corticosteroids 2 . 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 placebo 2 . 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 death 2 . The key secondary endpoint is the percentage of patients with a stable platelet count response at Month 6 2 . 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 endpoints 2 . 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 SOURCE: Novartis

Primary immune thrombocytopenia is an autoimmune disorder characterized by a low platelet count.\n For Novartis, this week is all about ianalumab. A day after posting a pair of phase 3 wins for the tricky indication of Sjögren\'s syndrome, the Swiss pharma has unveiled a separate late-stage victory in another B-cell-driven autoimmune disease.The latest clinical success comes from a phase 3 study of two different doses of ianalumab, a dual-mechanism, B-cell-depleting antibody that targets the protein BAFF-R, in patients with a low platelet count disorder called primary immune thrombocytopenia (ITP). Participants also received Novartis\' thrombocytopenia drug Promacta as part of their treatment and had found that corticosteroids hadn’t resolved their condition.While Novartis didn’t dig into the data, the company confirmed that treatment with ianalumab plus eltrombopag “significantly prolonged” the time to treatment failure (TTF) when compared to patients who received Promacta alone, hitting the trial’s primary endpoint. TTF measures how long patients maintain safe platelet levels during and after treatment.When it came to a key secondary endpoint, the ianalumab-eltrombopag treatment also showed a “significantly higher” rate of sustained improvements in platelet count at six months, Novartis explained in an Aug. 12 release.Ianalumab is administered as four once-monthly doses to treat ITP. Novartis is touting the therapy as potentially offering “long-term disease control through a short course of treatment” that could “allow patients extended time off treatment.”This would help it stand out from the current crop of ITP therapies, which often require lifelong treatment.“For many people living with ITP, chronic treatment can disrupt their daily life due to the burden of regular dosing, dose adjustments and side effects,” Novartis Chief Medical Officer Shreeram Aradhye, M.D., said in the release. “These positive topline results from the phase 3 study highlight the potential of ianalumab, if approved, to deliver long-term disease control with four once-monthly doses and enable extended time off treatment.”Ianalumab\'s story begins with Novartis’ collaboration with MorphoSys, before the Big Pharma went on to buy the biotech last year. The pharma is currently eyeing 2027 for an approval application for ianalumab in ITP, for which it hopes to combine data from this morning’s study with an ongoing trial of the drug as a first-line treatment for the disorder. In the meantime, the company is expecting phase 3 readouts for ianalumab in other B-cell-driven autoimmune diseases next year, such as warm autoimmune hemolytic anemia.This morning’s late-stage win in ITP comes just a day after Novartis reported a pair of phase 3 successes in Sjögren\'s syndrome, an autoimmune disorder that has defeated some of the company’s peers. Novartis hailed the triumph yesterday as the “first ever global phase 3 trials to demonstrate statistically significant reduction in disease activity for Sjögren\'s disease.”Ianalumab hasn’t always been so lucky in the clinic. Only last month, the company reported that the B-cell-depleting antibody had failed to hit the efficacy threshold in a phase 2 study for a painful skin condition called hidradenitis suppurativa.