Orphan Drug Designation Granted for New Agent in Hepatocellular Carcinoma

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CASI Pharmaceuticals, Inc, announced that its investigational agent ENMD-2076 has received orphan drug designation from the U.S. Food & Drug Administration (FDA) for the treatment of hepatocellular carcinoma. ENMD-2076 is an orally active Aurora A/angiogenic kinase inhibitor with a unique kinase selectivity profile and multiple mechanisms of action.

The drug has been shown to inhibit a distinct profile of angiogenic tyrosine kinase targets in addition to the Aurora A kinase, which is a key regulator of mitosis and is often overexpressed in human cancers. ENMD-2076 also targets the ­VEGFR, Flt-3, and FGFR3 kinases, which have been shown to play important roles in the pathology of several cancers.

ENMD-2076 previously received orphan drug designation from the FDA for the treatment of ovarian cancer, multiple myeloma, and acute myeloid leukemia. Phase II studies of ENMD-2076 in ovarian cancer, triple-negative breast cancer, metastatic soft-tissue sarcoma, and advanced ovarian clear cell carcinomas are currently underway. ■





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