Novartis ianalumab Phase III trial meets primary endpoint in immune thrombocytopenia
Basel: Novartis has 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.
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. 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.
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. The safety profile of ianalumab was consistent with what was previously observed in clinical studies, with no new safety signals.
“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. Many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control. 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.
“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. Recently, Novartis announced positive top-line results for ianalumab in adults with active Sjögren's disease.
