Immune thrombocytopenia (ITP) is a rare autoimmune disease in which the immune system destroys platelets. Treatments for patients with ITP include corticosteroids, intravenous immunoglobulin (IVIG), anti-D, splenectomy, and rituximab. However, despite its treatments, there are quite a few patients with persistently low platelet counts. Therefore, the key unmet needs for immune thrombocytopenia therapy are achievement of adequate therapeutic efficacy.
Patient with persistent and chronic ITP
SKI-O-703 is currently in phase IIa clinical trial in US, EU, and Korea.
R788 (Fostamatinib, Tavlesse)
R788 is the first small molecule SYK inhibitor. R788 new drug application has been approved to US (2018) and EU (2020) for the treatment of patients with refractory ITP despite its marginal efficacy in clinical trials. SKI-O-703 displayed high selectivity and potency against SYK in both biochemical and cell-based assays. Selective SYK inhibition by SKI-O-703 prevents FcγR mediated phagocytosis in vitro and downregulates cluster of differentiation (CD) 63 expression on peripheral blood basophils in healthy adults.