Novartis has reported that its experimental drug ianalumab met the primary goal in a phase 3 trial for primary immune thrombocytopenia (ITP), a rare autoimmune condition where the immune system destroys platelets, increasing the risk of bleeding and fatigue.
The study, VAYHIT2, investigated ianalumab alongside eltrombopag in patients whose disease had not responded to corticosteroids – a conventional means of treatment. Results showed that patients receiving ianalumab maintained safe platelet levels for longer than those given a placebo. Patients on ianalumab also had higher rates of sustained improvements in platelet counts at six months, the key secondary endpoint.
The treatment was given as four once-monthly doses, potentially allowing patients longer periods without continuous treatment. Safety findings were consistent with previous studies, with no new concerns reported.
Adam Cuker, professor of medicine at the University of Pennsylvania, US, said: “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.”
Data from the trial will be presented at an upcoming medical meeting and submitted to regulators in 2027, alongside results from an ongoing first-line ITP trial. Ianalumab has been granted Orphan Drug Designation by the US Food and Drug Administration and the European Medicines Agency.
Ianalumab is also being studied in other autoimmune diseases, including Sjögren’s disease and lupus.
