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MB07133 is a HepDirect prodrug of an activated form of cytarabine (araC), an anti-cancer drug that is used to treat leukemia but is ineffective against primary liver cancer. AraC's anti-cancer activity is associated with its ability to be converted to its biologically active form, araCTP. However, araC is only slowly converted to araCTP in the liver or in primary liver tumors due to low levels of an enzyme in the liver that is required for the conversion of araC to araCMP, the first step in the activation pathway of the drug. Higher doses of araC cannot be used to overcome this limitation due to bone marrow toxicity resulting from rapid activation in that tissue. MB07133 uses our HepDirect technology to target a prodrug form of araCMP specifically to the liver. Once in the liver cell, the prodrug is rapidly cleaved, producing araCMP, bypassing the rate-limiting enzyme. Once generated in the target tissue, araCMP is rapidly converted to araCTP, the cancer-killing active form.
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