First patient dosed in Greater China in global registrational clinical trial of efgartigimod in primary immune thrombocytopenia.

The ADVANCE-SC study is a randomized, double-blind, placebo-controlled, multi-center Phase III trial evaluating the efficacy and safety of subcutaneous (SC) efgartigimod in approximately 156 adult patients with primary ITP.

In a prior Phase II study, patients treated with efgartigimod exhibited clinically meaningful improvement in platelet counts across doses and types of ITP patients (newly diagnosed, persistent and chronic), and this benefit strongly correlated with reduction in IgG levels. This data highlights the potency of efgartigimod in this difficult-to-treat disease. Efgartigimod has the potential to treat ITP in a novel way, targeting the disease at its source by eliminating autoantibodies and restoring platelet counts. Also, efgartigimod appears to be well tolerated, potentially because of its unique structure and ability to bind to the FcRn receptor.

ITP is a rare and often chronic bleeding disorder in which reduced numbers of platelets can lead to an increased risk of bleeding and bruising, which impairs quality of life. In ITP, the immune system produces IgG autoantibodies that destroy platelets and affect platelet production. While ITP can be asymptomatic, minor bleeding episodes are not uncommon. Some ITP patients experience more severe bleeding episodes, including intracerebral hemorrhage.