Novartis two for two as ianalumab scores again

A mere 24 hours after Novartis' ianalumab hit the mark in two phase 3 trials in Sjogren's syndrome, the drug has delivered another win in primary immune thrombocytopenia (ITP), another autoimmune disorder.

In the VAYHIT2 trial, the anti-BLyS/BAFF antibody was compared to placebo, both given on top of Novartis' established ITP therapy Promacta (eltrombopag), which made more than $2.2 billion in sales last year. Subjects in the study had previously been treated with corticosteroids for ITP, in which the immune system mistakenly targets and destroys blood platelets.

The combination of ianalumab and Promacta significantly prolonged the time to treatment failure (TTF), the trial's primary endpoint, which gauges how long patients maintain safe platelet levels during and after the treatment period. There was also a significantly higher rate of sustained improvements in platelet count at six months, according to Novartis.

Many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control, so there is a pressing need for new treatment options, the company added.

"While current treatments for ITP are generally effective in raising platelet counts, many patients require lifelong treatment to maintain safe levels, which can create a lasting treatment burden," said Adam Cuker, a haematologist at the University of Pennsylvania who is one of the VAYHIT2 investigators.

"The results from VAYHIT2 are encouraging, as they suggest that ianalumab may support longer periods of disease control and reduce the need for continuous treatment," he added.

Novartis acquired ianalumab when it bought MorphoSys for $2.9 billion last year, and the new data in ITP comes right after the drug showed efficacy in Sjögren's, a condition that affects millions of people worldwide and causes dryness in mucosal surfaces like the eyes and mouth, as well as organ damage and an increase in the risk of life-threatening cancers.

That was the time that a drug has been shown to address the underlying pathology of Sjögren's in phase 3 trials, and Novartis is now moving swiftly ahead with plans to file ianalumab for approval in Sjögren's as it continues to develop the antibody for ITP and other B cell-driven autoimmune diseases.

Along with a phase 3 trial of the drug as a first-line therapy for ITP, trials are also on the go in warm autoimmune haemolytic anaemia, with readouts from all those studies due next year.

The ITP data is due to be presented later this year, with a regulatory filing anticipated for 2027, after the readout of the first-line study (VAYHIT1).