Novartis scores a Vayhit, but filing some way off

Following difficulties with pelabresib, Novartis has had good news with another Morphosys-originated asset: the company toplined a win on Tuesday with the anti-BAFF-R antibody ianalumab in immune thrombocytopenic purpura. The Vayhit2 trial combined ianalumab with Promacta in second-line disease and met its primary endpoint of time to treatment failure; Novartis is holding back details for an upcoming meeting. However, regulatory filings aren’t expected until 2027, after the company has reported results from the first-line Vayhit1 study – these were once due this year, but have been delayed until 2026. The FDA’s requirement for two phase 3 studies has tripped up Novartis’s ITP rival Argenx, which saw one win and one loss with its contender, Vyvgart, with intravenous and subcutaneous versions respectively. Argenx is now running a second phase 3 trial, Advance-Next, of its IV formulation, with results expected next year – potentially putting ianalumab and Vyvgart on a similar regulatory timeline. ITP is caused by low platelet levels that can lead to bruising and bleeding; steroids are a treatment mainstay, but carry long-term risks, while thrombopoietin receptor agonists like Promacta increase the risk of blood clots. In Vayhit2, ianulmab was given as four once-monthly doses, so could offer a more convenient schedule.

Notable trials of ianalumab

Note: *defined as platelet count <30g/L, start of a new ITP treatment, need for rescue treatment, ineligibility to taper or discontinue eltrombopag, death. Source: OncologyPipeline & clinicaltrials.gov.