Novartis' Investigational Drug Reports Longer Disease Control In Patients With Rare Blood Disorder

Novartis AG (NYSE:NVS) on Tuesday shared results from the VAYHIT2 Phase 3 trial of ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids.

In August, Novartis shared topline results from the VAYHIT2 trial.

Ianalumab (9 mg/kg) plus eltrombopag extended ITP disease control by 45%, based on the primary endpoint of time to treatment failure (TTF), which assesses how long patients maintain safe platelet levels during and after the treatment period.

The median time to treatment failure for patients receiving ianalumab plus eltrombopag was 2.8 times longer than those on placebo plus eltrombopag (13.0 months vs. 4.7 months).

Detailed data were presented at the American Society of Hematology Annual Meeting & Exposition (ASH) and simultaneously published in The New England Journal of Medicine.

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Patients receiving ianalumab (9 mg/kg) plus eltrombopag also achieved a significantly higher rate of sustained platelet count improvement at six months versus placebo plus eltrombopag (62% vs. 39%), meeting the key secondary endpoint.

Fatigue improvement showed a mean reduction of 7.7 points with ianalumab plus eltrombopag versus 3.6 points with placebo plus eltrombopag.

The estimated probability of being free from treatment failure at 12 months was 54% in the 9-mg group, 51% in the 3-mg group, and 30% in the placebo group.

Ianalumab is being investigated in other B-cell-driven autoimmune diseases, including ongoing Phase 3 trials in first-line ITP and in second and later lines of warm autoimmune hemolytic anemia, with readouts expected in 2026.

NVS Price Action: Novartis stock is up 1.46% at $132.07 at publication on Tuesday.

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