ASH 2025 | VAYHIT2 primary results: ianalumab and eltrombopag versus placebo and eltrombopag in 2L primary ITP

VAYHIT2 is a pivotal, phase 3, randomized, placebo-controlled study evaluating combination eltrombopag with ianalumab, a novel anti-BAFF receptor monoclonal antibody, very potent inhibitor of B-cell maturation as well as a very potent depletor of B-cells, versus eltrombopag plus placebo in patients with ITP in the second line. So these are patients that have just failed corticosteroids. They’re relatively early on in their disease course. VAYHIT2 demonstrated significant improvements in time-to-treatment failure in patients receiving ianalumab plus eltrombopag versus patients receiving only eltrombopag with placebo. And two different doses of ianalumab were evaluated, and the higher dose of ianalumab also demonstrated a significant increase in sustained response at six months. 

Basically, this study, which was a very different way of evaluating new agents in ITP, a very bold way, if I may say so, which kudos to the sponsor of the study, Novartis, for that, for evaluating this drug in a setting that most ITP therapies are never looked at in, which is early in disease, with the idea of maybe we can modify the course of the disease early on, before it becomes so immunologically complex, before the patient has a lot of oligoclonal T-cell clones that are causing issues, that are making disease very difficult to treat. We know the longer people have ITP, the worse it gets, and the more challenging it is to manage. 

So this study evaluated, you know, ianalumab plus eltrombopag versus placebo plus eltrombopag. So everybody got established therapeutic eltrombopag. And yet the patients receiving ianalumab did much better in terms of time to treatment failure after eltrombopag tapers were done in the patients on the study. And it did that with no increase in the severity or frequency of infections, a really, really quite good side effect profile. It’s given us four intravenous infusions once a month for four months. So not a huge burden for patients and an opportunity for, at a minimum, a nice long duration durable, treatment-free time where people don’t have to be on any ITP therapy, but may also, and we’ll find this with follow-up, may also actually induce some durable disease modification in ITP, which has been, in many ways, a holy grail in this disease.

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