Argenx announces ADVANCE-SC study did mot meet primary, secondary endpoints

Argenx announced topline results from the ADVANCE-SC study evaluating Vyvgart Hytrulo in adults with primary immune thrombocytopenia, or ITP. The study did not meet the primary endpoint of a sustained platelet count response in chronic ITP patients. Additional analyses of the dataset are ongoing and the full results will be presented at an upcoming medical meeting and in a peer-reviewed publication. ADVANCE-SC is the second of two registrational trials conducted as part of the ongoing ITP development program for Vyvgart and enrolled 207 adult patients with chronic and persistent ITP. Patients were heavily pre-treated and 75% of patients had received three or more prior ITP therapies. Primary endpoint was not met; 13.7% of treated patients demonstrated a sustained platelet count response compared to 16.2% of placebo patients. Secondary endpoints were not met, including additional endpoints on International Working Group, or IWG, responder status and mean platelet count change from baseline. Vyvgart Hytrulo was well-tolerated in ADVANCE-SC; the observed safety and tolerability profile was consistent with ADVANCE-IV and the confirmed safety profile of Vyvgart and Vyvgart Hytrulo. Results from the first study in the ITP registrational program, ADVANCE-IV, were reported in May 2022. The study met its primary and key platelet-derived secondary endpoints. ADVANCE-IV formed the basis of the regulatory submission for approval of Vyvgart IV for ITP in Japan, where a decision is expected in the first quarter of 2024. Vyvgart is currently being evaluated in 13 severe autoimmune diseases, including the registrational ADDRESS study for pemphigus from which topline results are expected around year-end 2023.