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WJ01024 is an orally bioavailable small molecule inhibitor of the nuclear export protein exportin-1 (XPO1; also known as chromosome region maintenance 1 protein homolog or CRM1). By inhibiting XPO1, WJ01024 blocks the nuclear export of various tumor suppressor proteins such as p53, thereby enhancing their tumor-suppressive functions. This mechanism leads to antineoplastic and pro-apoptotic activities. Preclinical studies have shown that WJ01024 inhibits the growth and induces death of a variety of tumor cells in vitro and can inhibit both blood and solid tumors in animal models. Clinically, it is being developed for advanced cancers including hematologic neoplasms and myelofibrosis (both post-essential thrombocythemia myelofibrosis and post-polycythemia vera myelofibrosis), with ongoing trials evaluating its use alone or in combination with ruxolitinib to enhance therapeutic efficacy[1][2][3][6].
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