InnoCare Pharma, a leading biopharmaceutical company, announced that the first patient has been dosed in phase II clinical trial of its Bruton’s tyrosine kinase (BTK) inhibitor orelabrutinib for the treatment of primary immune thrombocytopenia (ITP) in China.

Primary immune thrombocytopenia (ITP), also referred to as idiopathic thrombocytopenic purpura, is an acquired immune mediated disorder characterized by a decrease in peripheral blood platelet counts, resulting in an increased risk of bruising and bleeding. The main pathogenesis of ITP is the loss of immune tolerance to platelet auto-antigens. This immune intolerance leads to increased platelet destruction and decreased platelet production from megakaryocytes by autoantibodies and cytotoxic T lymphocytes.

Only about 70% patients respond to first-line treatments, some patients are still refractory or relapsed after combined therapies, therefore it is necessary to explore new therapeutic targets. Inhibition of BTK may reduce platelet destruction by inhibiting B cell activation and autoantibody production.

The annual incidence of ITP in adults is approximately 2–10 new cases per 100,000 people. The elderly over 60 years old is at high risk of ITP, and females of childbearing have a higher risk than males of the same age group.

Dr. Jasmine Cui, the co-founder, Chairwoman and CEO of InnoCare said, “No BTK inhibitor has been approved for the treatment of patients with ITP in the world. BTK is a key kinase in the B cell receptor signaling pathway, which is essential for the activation of B lymphocytes, macrophages and other immune cells as well as the production of antibodies in the pathological process of ITP. Orelabrutinib has high target selectivity with safety profile, which is expected to bring novel treatment options to ITP patients.”

Orelabrutinib is a small molecule Bruton’s tyrosine kinase inhibitor (BTKi) developed for the treatment of cancer and autoimmune diseases.

Orelabrutinib had received approval on Dec. 25, 2020 from the China National Medical Products Administration (NMPA) in two indications: the treatment of patients with r/r chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL), and the treatment of patients with r/r mantle cell lymphoma (MCL). In addition, multi-center, multi-indication clinical trials are underway in the US and China for orelabrutinib as monotherapy or in combination therapies for the treatment of marginal zone lymphoma (MZL), central nervous system lymphoma (CNSL), Waldenstrom’s macroglobulinemia (WM), diffuse large B-cell lymphoma (DLBCL), etc.

Orelabrutinib was granted as Breakthrough Therapy Designation for the treatment of r/r MCL by US Food and Drug Administration (FDA).

Attributed to its excellent selectivity and clinical safety profiles, orelabrutinib is also evaluated in the global phase II studies for the treatment of Multiple Sclerosis (MS), phase II clinical trials for the treatment of systemic lupus erythematosus (SLE) and primary immune thrombocytopenia (ITP) in China.

InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancer and autoimmune diseases.