argenx reports topline results from ADVANCE-SC study of VYVGART Hytrulo in primary immune thrombocytopenia
argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced topline results from the ADVANCE-SC study evaluating VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in adults with primary immune thrombocytopenia (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.
“This is not the outcome we had hoped for patients, but setbacks are part of pioneering a new class of medicines and these data will provide insights into the broader understanding of FcRn and ITP,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. “We are very grateful to everyone involved in the ADVANCE-SC study, especially the patients and their families, the investigators, and our internal team who worked tirelessly to complete this global study. We remain committed to the ITP patient community who urgently needs additional treatment options to manage this challenging disease, and continue to move forward in our deeper analysis of these results.“
ADVANCE-SC Study Data
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 (p=0.5081); 13.7% (17/124) of treated patients demonstrated a sustained platelet count response compared to 16.2% (11/68) of placebo patients
Secondary endpoints were not met, including additional endpoints on International Working Group (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.