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MB01733 (also known as MB07133) is a liver-targeted prodrug of the antimetabolite cytarabine (ara-C), developed using the proprietary HepDirect technology platform. Originally discovered by Metabasis Therapeutics and later licensed to Chiva Pharmaceuticals by Ligand Pharmaceuticals, the compound is designed for the treatment of hepatocellular carcinoma (HCC). The HepDirect technology utilizes a cyclic phosphate prodrug moiety that remains stable in systemic circulation but is selectively cleaved by the cytochrome P450 enzyme CYP3A4, which is highly expressed in the liver. This targeted activation releases cytarabine monophosphate directly within hepatocytes, where it is subsequently converted to the active triphosphate form (ara-CTP). By concentrating the active drug in the liver, MB01733 aims to enhance therapeutic efficacy against liver tumors while significantly reducing systemic exposure and the associated side effects, such as myelosuppression, typically seen with conventional cytarabine therapy.
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