Novartis AG has announced significant findings from its VAYHIT2 Phase 3 trial, which assessed the combination of ianalumab and eltrombopag in patients suffering from primary immune thrombocytopenia (ITP) who had previously been treated with corticosteroids. This announcement follows the release of topline results in August, showcasing a notable advancement in the management of this rare blood disorder.
The trial demonstrated that the combination therapy extended the duration of disease control by 45%. This conclusion was based on the primary endpoint of time to treatment failure (TTF), which measures how long patients can maintain safe platelet levels during and after treatment. Patients receiving the ianalumab plus eltrombopag treatment experienced a median TTF of 13.0 months, compared to 4.7 months for those on a placebo combined with eltrombopag.
Key Findings in Disease Management and Patient Outcomes
The detailed data were presented at the American Society of Hematology Annual Meeting & Exposition (ASH) and were also published in The New England Journal of Medicine. In addition to improved TTF, patients receiving the combination therapy achieved a significantly higher rate of sustained platelet count improvement at six months, with 62% of participants showing improvement compared to 39% in the placebo group.
Furthermore, the treatment also demonstrated a reduction in fatigue levels, with a mean decrease of 7.7 points in the ianalumab plus eltrombopag group, compared to 3.6 points for those receiving placebo. The estimated probability of being free from treatment failure at 12 months was calculated as 54% for the 9 mg/kg group, 51% for the 3 mg/kg group, and 30% for the placebo group.
Future Prospects for Ianalumab in Autoimmune Diseases
Ianalumab is currently under investigation for other B-cell-driven autoimmune conditions. Ongoing Phase 3 trials are exploring its efficacy in first-line ITP and in treatment for warm autoimmune hemolytic anemia. Results from these studies are anticipated in 2026, which could further expand the therapeutic applications of this investigational drug.
At the time of publication, Novartis shares rose by 1.46%, trading at $132.07. The promising results from the VAYHIT2 trial may bolster investor confidence and enhance Novartis’ position in the competitive pharmaceutical market.
As research continues, the medical community remains hopeful that advancements like those from Novartis will lead to improved outcomes for patients battling complex blood disorders.
