InnoCare Pharma, a leading biopharmaceutical company, announced today the Investigational New Drug (IND) approval of its Bruton's tyrosine kinase (BTK) inhibitor orelabrutinib by China National Medical Products Administration (NMPA) for starting phase II clinical trial in primary immune thrombocytopenia (ITP) in China.

This is a randomized, multicenter, adaptive phase IIa/IIb study to evaluate the efficacy and safety of orelabrutinib in adult ITP patients.

Primary immune thrombocytopenia (ITP), also referred to as idiopathic thrombocytopenic purpura, is an acquired immune mediated disorder characterized by a decrease in the number of peripheral blood platelet counts, resulting in an increased risk of bruising and bleeding[i].

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. 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.

The adult annual incidence rate of ITP is approximately 2–10 new cases per 100,000 population per year[ii],[iii]. It is more common in patients older than 60 years of age. Females of childbearing has a higher risk of ITP than males of the same age group.

Dr. Jasmine Cui, the co-founder, Chairwoman and CEO of InnoCare, said, "Globally, no BTK inhibitor has been approved for the treatment of patients with ITP. Orelabrutinib has high target selectivity with safety profile, which is expected to bring novel treatment options to patients with ITP."

About Orelabrutinib

Orelabrutinib is a highly selective BTK inhibitor developed by InnoCare for the treatment of cancers and autoimmune diseases.

On Dec. 25, orelabrutinib received approval from the China National Medical Products Administration (NMPA) in two indications: the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL), and the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL). Both new drug applications (NDAs) were previously granted priority review by the Center for Drug Evaluation (CDE) of the NMPA.

In addition to the approved indications, multi-center, multi-indication clinical trials are underway in the U.S. and China with orelabrutinib as monotherapy or in combination therapies.

About InnoCare

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. We strategically focus on lymphoma, solid tumors, and autoimmune diseases with high unmet medical needs in China and worldwide. InnoCare has branches in Beijing, Nanjing, Shanghai, Guangzhou, New Jersey, and Boston.

[i] Thrombosis and Hemostasis Group, Chinese Society of Hematology, Chinese Medical Association. Chinese Guideline on the diagnosis and management of adult primary immune thrombocytopenia (version 2020). Zhonghua Xue Ye Xue Za Zhi. 2020, 41(8): 617-623.

[ii] Lee JY, Lee JH, Lee H, et al. Epidemiology and management of primary immune thrombocytopenia: a nationwide population-based study in Korea. Thromb Res. 2017, 155:86-91.

[iii] Moulis G, Palmaro A, Montastruc JL, et al. Epidemiology of incident immune thrombocytopenia: a nationwide population-base study in France. Blood. 2014, 124(22):3308-3315.