Hangzhou HealZen Therapeutics Co., Ltd recently announced that its independently developed Class 1 innovative drug, HZ-A-018 capsules (Bruton's tyrosine kinase inhibitor), has been officially approved by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA), China, to conduct a pivotal single-arm Phase II clinical study for the monotherapy of relapsed/refractory primary central nervous system lymphoma (R/R PCNSL).

The HZ-A-018-102 study is a multicenter, open-label Phase I/II clinical trial, with the Phase II portion designed to evaluate the efficacy and safety of HZ-A-018 monotherapy in the treatment of R/R PCNSL. The primary endpoint of the study was the overall response rate (ORR) assessed by the Independent Review Committee (IRC).

Primary central nervous system lymphoma (PCNSL) is a rare and aggressive type of non-Hodgkin's lymphoma (NHL), primarily characterized by diffuse large B-cell lymphoma (DLBCL) as its pathological subtype. PCNSL has a short disease course, rapid progression, poor prognosis, and is highly lethal with a 5-year survival rate of approximately 22 to 40%[1-2]. Combined chemotherapy regimens containing high-dose methotrexate (HD-MTX) are currently the first-line choice for the initial treatment of PCNSL. However, about 30 to 60% of PCNSL patients relapse within 1 to 2 years of initial treatment, about 10 to 15% of patients are primarily refractory, and other patients become refractory after first-line treatment[2]. Currently, there are no effective treatment options for R/R PCNSL, and authoritative guidelines both domestically and internationally prioritize clinical trials as the recommended approach.

HZ-A-018 is a highly selective small-molecule BTK inhibitor that can block the B-cell receptor (BCR) and Toll receptor signaling pathway and has a high permeability rate through the blood-brain barrier, effectively inhibiting the proliferation of B cells and promoting apoptosis. The safety of HZ-A-018 is controllable, and the efficacy of monotherapy for R/R PCNSL is outstanding, which is expected to bring a new treatment option for R/R PCNSL patients. In September 2023, HZ-A-018 was included in the breakthrough therapy program by CDE for the treatment of relapsed/refractory primary central nervous system lymphoma.

About HZ-A-018 Capsules

HZ-A-018 is a highly selective small molecule BTK inhibitor independently developed by Hangzhou HealZen Therapeutics Co., Ltd, which has been approved for the monotherapy of B-cell lymphoma, Relapsed/refractory primary central nervous system lymphoma, chronic/persistent adult primary immune thrombocytopenia, multiple sclerosis, and neuromyelitis optica spectrum disorders as well as combination therapy with HZ-H08905 tablets for the treatment of relapsed or refractory B-cell lymphoma.

About Hangzhou HealZen Therapeutics Co., Ltd

Hangzhou HealZen Therapeutics Co., Ltd is an innovative small-molecule drug development company driven by innovative technology in the clinical stage, dedicated to addressing unmet clinical needs. The company independently develops targeted protein degradation drugs and an intelligent drug development platform, focusing on drug development in disease areas such as hematological tumors and the central nervous system. The company has established project pipelines with over 10 Class 1 new drugs, of which 6 molecules have entered the clinical research stage, 1 molecule is in clinical phase III, and 1 molecule has been recognized as a breakthrough therapy. In addition, the company has accumulated 4 new drug pipelines for external cooperation, and the drug discovery capability has been widely recognized in the industry.

References:

[1] National Comprehensive Cancer Network. NCCN Guidelines Version 2.2022, Central Nervous System Cancers: Primary CNS Lymphoma.

[2] Jahnke K, et al. Relapse of Primary central nervous system lymphoma: clinical features, outcome and prognostic factors. J Neurooncol, 2006, 80:159-165.