Ianalumab lengthens time patients can maintain safe platelet levels

A Phase 3 clinical trial testing ianalumab (VAY736) in combination with Promacta (eltrombopag) in people with primary immune thrombocytopenia (ITP) has met its main goal, showing that the addition of the experimental infusion therapy can significantly prolong the time patients maintain safe platelet levels during and after treatment.

According to therapy developer Novartis, these initial results show ianalumab “could offer long-term disease control through a short course of treatment.” That’s according to a company press release announcing the positive trial data from the ongoing, global VAYHIT2 study (NCT05653219).

“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, MD, president of development and chief medical officer at Novartis. “These positive top-line 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.”

Novartis said data readouts, or fuller details of the trial results, are expected in 2026.

Ianalumab tested in adults already treated with corticosteroids

ITP is an autoimmune disorder in which the immune system makes antibodies that attack platelets, which are the cell fragments that help blood clot. Low platelet levels in ITP mean blood can’t clot correctly, resulting in symptoms like easy and prolonged bleeding. In primary ITP, the disorder develops for unknown reasons, rather than as a consequence of another health issue, as happens in secondary ITP.

“While current treatments for ITP are generally effective in raising platelet counts, many patients require lifelong treatment to maintain safe levels, which can create a lasting treatment burden,” said Adam Cuker, MD, a professor of medicine and chief of the hematology section at the University of Pennsylvania.

An antibody therapy, ianalumab is designed to deplete and reduce the activity of B-cells, the immune cells responsible for producing antibodies — including those driving autoimmune diseases. By lowering the number and reducing the activity of B-cells, ianalumab is also expected to lower the levels of the harmful antibodies that drive ITP and other autoimmune disorders.

The VAYHIT2 trial enrolled 153 adults with primary ITP whose disease had not been adequately controlled with corticosteroids, which are anti-inflammatory therapies commonly used as a first-line ITP treatment.

All participants in the trial were given the approved ITP therapy Promacta. Some participants also were treated with a low or high dose of ianalumab — given as four once-monthly intravenous, or into-the-vein, infusions — while others were instead given a placebo.

Therapy also found to maintain safety profile

The trial’s main goal was to see if the time to treatment failure — a dip in platelet levels and/or needing other medications for disease management — would be longer in patients treated with ianalumab.

Novartis reported that the trial hit this goal, as well as the key secondary goal of showing that patients given ianalumab experienced a significantly higher rate of sustained improvements in platelet counts. Ianalumab’s safety profile was also consistent with that seen in previous studies, with no new safety issues identified.

[These results] are encouraging, as they suggest that ianalumab may support longer periods of disease control and reduce the need for continuous treatment.

The company did not provide further details, noting that in-depth results will be presented at a future medical meeting.

Cuker said the results “are encouraging, as they suggest that ianalumab may support longer periods of disease control and reduce the need for continuous treatment.”

A separate Phase 3 clinical trial called VAYHIT1 (NCT05653349) is testing ianalumab against a placebo when given in addition to first-line corticosteroids in adults newly diagnosed with primary ITP. That trial is still enrolling participants at more than 150 sites worldwide. Its goal also is to show that the time to treatment failure is significantly delayed by the addition of ianalumab.

Novartis intends to use data from both the VAYHIT2 and VAYHIT1 trials as a basis to apply for regulatory approvals of ianalumab in ITP, the release stated. The company said it is planning to submit such applications in 2027.