Ianalumab Plus Eltrombopag Extends Disease Control in Phase 3 ITP Study Published in NEJM

Overview

• Investigators at ASH reported that four monthly infusions of ianalumab combined with a limited course of eltrombopag significantly delayed time to treatment failure versus placebo plus eltrombopag in adults with ITP after steroid failure.
• The higher‑dose ianalumab arm reached a median 13 months to treatment failure versus 4.7 months on placebo, the lower dose was not estimable due to few events, and stable platelet response at six months was 62% and 56.9% for high and low doses versus 39.2% for placebo.
• Overall adverse event rates were similar across groups, with transient neutropenia more frequent on ianalumab yet no increase in infection rates, and one severe drug‑related event of palpitations reported.
• The double‑blind, randomized VAYHIT2 trial enrolled 152 patients across 24 countries, and the estimated 12‑month probability of avoiding treatment failure was 54.2% with high‑dose ianalumab and 50.5% with low dose versus 30% with placebo.
• Participants will be followed for up to 39 months to assess durability, ianalumab remains unapproved, and Novartis is advancing the program it acquired with MorphoSys, including the ongoing VAYHIT1 study in earlier‑line disease.