The US Food and Drug Administration (FDA) has granted Orphan Drug designation to CASI Pharmaceuticals' orally-active, Aurora A/angiogenic kinase inhibitor, ENMD-2076 for the treatment of hepatocellular carcinoma (HCC).

Orphan Drug designation is granted by the FDA Office of Orphan Products Development, which evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan Drug designation is granted to therapeutics treating rare diseases affecting less than 200,000 people in the US.  The designation entitles the sponsor to seven years of market exclusivity as well as opportunities for additional funding and expert protocol assistance.

Dr Ken Ren, chief executive officer of CASI, commented, “We are pleased with the Orphan Drug designation as it confirms our belief in the versatility of ENMD-2076 as a promising treatment for HCC, and for other tumor types that we are currently evaluating in the clinic. Orphan drug status also enhances the commercial value of ENMD-2076 to treat HCC, a disease which is difficult to treat. We are finalizing our next steps for ENMD-2076 in HCC and/or in fibrolamellar carcinoma, a subset of HCC and for which there is no treatment available and look forward to advancing our overall development plan for ENMD-2076.”

ENMD-2076 is an orally-active, Aurora A/angiogenic kinase inhibitor with a unique kinase selectivity profile and multiple mechanisms of action. ENMD-2076 has been shown to inhibit a distinct profile of angiogenic tyrosine kinase targets in addition to the Aurora A kinase. Aurora kinases are key regulators of mitosis (cell division), and are often over-expressed 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 has shown promising activity in Phase I clinical trials in solid tumor cancers including ovarian, breast, liver, renal and sarcoma, as well as in leukemia and multiple myeloma. CASI is completing a phase II trial of ENMD-2076 in ovarian cancer. In addition, CASI is conducting a dual-institutional Phase II study of ENMD-2076 in triple-negative breast cancer, a Phase II study in advanced/metastatic soft tissue sarcoma and a Phase II study in advanced ovarian clear cell carcinomas. ENMD-2076 has received orphan drug designation from the US FDA for the treatment of ovarian cancer, multiple myeloma and acute myeloid leukemia.

CASI is a clinical-stage pharmaceutical company employing a drug-development strategy that leverages resources in both North America and in China to develop therapeutics for the treatment of cancer and other diseases.