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JBZ-001 is an orally bioavailable small-molecule inhibitor of dihydroorotate dehydrogenase (DHODH), the rate-limiting enzyme in the de novo pyrimidine nucleotide biosynthesis pathway. By inhibiting DHODH, JBZ-001 disrupts nucleotide production essential for cancer cell proliferation, leading to cytotoxic effects in tumor cells. The drug is being developed by Jabez Biosciences as a next-generation targeted therapy for advanced solid tumors and hematological malignancies, including non-Hodgkin lymphoma and acute myeloid leukemia (AML). Preclinical studies have shown robust anti-cancer activity and favorable safety profiles. As of mid-2025, JBZ-001 is undergoing Phase 1 clinical trials to assess its safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD), and preliminary efficacy in patients with advanced cancers[1][2][3][5][6][7].
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