FDA grants Orphan Drug Designation to CG’806 for the treatment of AML

Last month, the US Food and Drug Administration (FDA) granted Orphan Drug Designation to CG’806, a multi-kinase inhibitor, for the treatment of patients with Acute Myeloid Leukemia (AML).

CG’806 is a highly potent, first-in-class, oral inhibitor of FMS like Tyrosine Kinase 3 (FLT3)/ Bruton’s Tyrosine Kinase (BTK). Preclinical studies have demonstrated that, CG’806 can inhibit AML harbouring FLT3- Internal Tandem Duplication (FLT3-ITD), Tyrosine Kinase Domain (TKD) mutations or both by inhibiting FLT3/BTK/Aurora Kinase (AuroK) activation.  Additionally, CG’806 exhibits a profound suppression of cell proliferation in FLT3-mutant AML cells.

Aptose Biosciences, the drug manufacturers, noted that based on the encouraging preclinical data of CG’806, they are eager to begin clinical trials in AML later this year.