On December 26, 2017 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CG’806, a highly potent pan-FLT3/pan-BTK inhibitor, for the treatment of patients with acute myeloid leukemia (AML) (Press release, Aptose Biosciences, DEC 26, 2017, View Source;p=RssLanding&cat=news&id=2324031 [SID1234522772]). AML is a particularly devastating cancer of the blood and bone marrow and is the most common type of acute leukemia among adults, with an annual incidence of approximately 21,000 patients and causing more than 10,000 deaths each year in the U.S.

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"We are pleased that the FDA has recognized the unique potential of CG’806 to address AML and has assigned CG’806 the status of orphan drug designation," said William G. Rice, Ph.D., Chairman, President and CEO. "Results from non-clinical studies that we and our research collaborators have generated are promising and give reason for our eagerness to begin clinical trials in both AML and B-Cell malignancies in 2018."

AML cells utilize multiple forms of the FLT3 receptor tyrosine kinase and other pathways to promote rapid proliferation and to escape the inhibitory activities of many therapeutics. CG’806 is a highly potent inhibitor that simultaneously targets all known forms of FLT3 and other key oncogenic pathways that drive the proliferation of AML cancer cells, thereby providing CG’806 with a broad range of activity against AML and a strategy to delay mutational escape.

The FDA’s Office of Orphan Drug Products assigns orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides Aptose certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

About CG’806

CG‘806 is an oral, first-in-class pan-FLT3/pan-BTK inhibitor. This small molecule demonstrates potent inhibition of all wild type and mutant forms of FLT3 tested (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), suppresses multiple oncogenic pathways operative in AML, eliminates AML tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with FLT3-driven AML. Likewise, CG’806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinases operative in B cell malignancies, suggesting CG’806 may also be developed for CLL and MCL patients that are resistant/refractory/intolerant to covalent BTK inhibitors.