Novartis has reported positive outcomes from the VAYHIT2 Phase III trial assessing ianalumab plus eltrombopag to treat patients with primary immune thrombocytopenia (ITP) who had prior corticosteroid treatment.
The trial results showed that four once-monthly doses of ianalumab (9mg/kg) plus eltrombopag extended disease control by 45%, based on the time to treatment failure (TTF) compared to placebo plus eltrombopag.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
Median TTF for the ianalumab plus eltrombopag group was 13.0 months, 2.8 times longer than the 4.7 months seen in the placebo group.
At six months, 62% of patients given ianalumab at 9mg/kg in combination with eltrombopag experienced sustained improvements in platelet counts while 39% of those in the placebo group reached this outcome, fulfilling the key secondary endpoint.
Additionally, patients in the ianalumab group reported a mean reduction in fatigue of 7.7 points on the PROMIS Fatigue scale, compared to a 3.6-point reduction among those receiving placebo.
VAYHIT2 tested two ianalumab doses. The 9mg/kg dose showed statistically significant improvements in both primary and key secondary endpoints while the 3mg/kg dose achieved significant improvement in the primary endpoint.
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalDataIanalumab was observed to be well tolerated and no new safety signals were reported.
Infusion-related reactions and headache were the most common adverse events. Neutropenia was seen more frequently with ianalumab but resolved without intervention. No adverse event resulted in permanent discontinuation.
Novartis oncology development global head Mark Rutstein said: “B cells drive the autoimmune response that leads to platelet destruction and increased bleeding risk in ITP. The novel dual mechanism of action of ianalumab aims to deplete B cells while blocking their survival signals.
“Guided by our decades-long experience advancing ITP care, the VAYHIT2 findings underscore the potential of ianalumab to deliver durable control with a short course of four once-monthly doses, offering patients the possibility of achieving disease stability without ongoing treatment.”
VAYHIT2 is the third positive Phase III study for ianalumab, after previous studies involving adults with active Sjögren’s disease.
The company intends to submit the data from VAYHIT2, along with results from the ongoing first-line ITP trial, VAYHIT1, in 2027.
In June 2025, Novartis reported positive outcomes from the multi-centre Phase IIIB APPULSE-PNH study of oral Fabhalta (iptacopan) in adults with paroxysmal nocturnal haemoglobinuria (PNH).
