Novartis Announces Positive Phase III Results for Ianalumab in Primary Immune Thrombocytopenia (ITP)
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Basel, August 12, 2025 – 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 corticosteroids1,2. 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 period1,2. 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 20263,4.
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 study1. The safety profile of ianalumab was consistent with what was previously observed in clinical studies, with no new safety signals1.
“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 fatigue5-7. Many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control7.Ther is a need for other treatment options with novel mechanisms of action that offer durable responses while reducing the burden of long-term treatment8.
“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.
Key Highlights:
Ianalumab prolonged the duration of safe platelet levels during and after treatment in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids1,2. Patients treated with ianalumab also experienced a significantly higher rate of sustained improvements in platelet count, the key secondary endpoint of the study1.
* Ianalumab, administered as four once-monthly doses in the ITP setting, could offer long-term disease control and perhaps allow patients extended time off treatment, if approved.Forward-Looking Statement
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 openness; 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.
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Ianalumab Shows Promise in Autoimmune diseases
Ianalumab, an investigational antibody targeting B-cell activating factor (BAFF), is currently being evaluated in several clinical trials for its potential to treat autoimmune diseases like Sjögren’s syndrome and systemic lupus erythematosus (SLE). Early data suggests it could offer a new treatment option for patients who haven’t found relief with existing therapies.
Sjögren’s Syndrome Trials (NEPTUNUS-1 & NEPTUNUS-2)
Two Phase 3 trials, NEPTUNUS-1 (NCT05350072) and NEPTUNUS-2 (NCT05349214), are assessing the efficacy and safety of Ianalumab in individuals with active Sjögren’s syndrome. These studies are designed to determine if Ianalumab can improve symptoms and quality of life for those living with this chronic autoimmune condition. The trials are actively recruiting participants,indicating ongoing research into the drug’s potential benefits.
Systemic Lupus Erythematosus Trials (SIRIUS-SLE 1 & SIRIUS-LN)
Ianalumab is also under investigation for its use in SLE. The SIRIUS-SLE 1 trial (NCT05639114) is a Phase 3 study evaluating Ianalumab in combination with standard-of-care therapy. Researchers want to see if adding Ianalumab can improve outcomes for patients with SLE.
Another trial, SIRIUS-LN (NCT05126277), focuses specifically on lupus nephritis – kidney inflammation caused by SLE.This study is evaluating Ianalumab’s safety, efficacy, and tolerability when combined with standard treatment. Lupus nephritis can be a serious complication of SLE,and new treatment options are urgently needed.
Ongoing Research
A new clinical trial, NCT06470048, is also underway, further expanding the research into Ianalumab’s potential applications. While details are still emerging,this continued investigation highlights the significant interest in this drug as a potential therapeutic agent for autoimmune diseases.
It’s vital to remember that these are ongoing studies, and the results are not yet finalized. However, the breadth of research surrounding Ianalumab suggests it could become an important tool in managing these challenging conditions.Patients interested in learning more about these trials should discuss them with their healthcare providers.
Sources:
- Clinicaltrials.gov. NCT05350072. Phase 3 Study to Assess Efficacy and safety of Ianalumab (VAY736) in Patients With Active Sjogren’s Syndrome (NEPTUNUS-1). accessed August 9, 2025. https://clinicaltrials.gov/study/NCT05350072
- Clinicaltrials.gov. NCT05349214.Three-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren’s Syndrome (NEPTUNUS-2). Accessed August 9, 2025. https://clinicaltrials.gov/study/NCT05349214
- 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 August 9, 2025.https://www.clinicaltrials.gov/study/NCT05639114
- Clinicaltrials.gov. NCT05126277. Safety,Efficacy and Tolerability of Ianalumab Versus Placebo,Combination With SoC Therapy,in Participants With Active Lupus Nephritis (SIRIUS-LN). Accessed August 9, 2025. https://clinicaltrials.gov/study/NCT05126277
- Clinicaltrials.gov. NCT06470048.A Clinical Trial of Ianalumab in Participants With Autoimmune Diseases. Accessed August 9, 2025.