Novartis ianalumab Phase III trial meets primary endpoint in ITP, demonstrating statistically significant improvement in time to treatment failure

Core Insights - Novartis announced positive top-line results from the Phase III trial VAYHIT2, evaluating ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids, showing significant prolongation of time to treatment failure (TTF) [1][6] - The study demonstrated a higher rate of sustained improvements in platelet count at six months for patients treated with ianalumab plus eltrombopag, indicating potential for long-term disease control [2][6] - Ianalumab is being investigated for various B cell-driven autoimmune diseases, with ongoing trials expected to yield further data in 2026 and 2027 [1][4] Company Overview - Ianalumab (VAY736) is a fully human monoclonal antibody targeting B cells, showing promising efficacy and safety in treating autoimmune diseases such as ITP and Sjögren's disease [5][6] - Novartis has received Orphan Drug Designation for ianalumab from both the US FDA and the European Medicines Agency, highlighting its potential in treating rare diseases [4][5] - The company aims to reduce the treatment burden for ITP patients by offering a regimen of four once-monthly doses, potentially allowing extended time off treatment [4][6] Industry Context - Primary immune thrombocytopenia (ITP) is a rare autoimmune disorder characterized by low platelet counts, leading to increased bleeding risk and chronic fatigue, necessitating new treatment options [3][7] - Current ITP treatments often require lifelong management, creating a significant treatment burden for patients, which underscores the need for therapies that provide durable responses [3][8] - The VAYHIT2 trial results suggest that ianalumab may address unmet needs in the ITP treatment landscape by offering a novel mechanism of action and improved patient quality of life [3][8]