On 29 March 2018, the US Food and Drug Administration (FDA) granted Orphan Drug Designation to MAX-40279, a multi-kinase targeted inhibitor, for the treatment of patients with Acute Myeloid Leukemia (AML).
Mutations in the fms like tyrosine kinase (FLT3) gene represent one of the most commonly encountered, and clinically challenging, classes of AML mutations and it is expressed in approximately 30% of patients. MAX-40279 is a dual inhibitor of FLT3 and fibroblast growth factor receptor (FGFR). According to the drug manufacturers, MaxiNovel Pharmaceuticals, MAX-40279 “demonstrated potent inhibition of both FLT3 and FGFR with excellent drug concentration in the bone marrow” in preclinical studies. Additionally, MAX-40279 was designed to overcome the drug resistance experienced by current FLT3 inhibitors due to bone marrow FGF/FGFR pathway activation.
At present, MAX-40279 is being investigated in a phase I dose escalation study (NCT03412292), which is evaluating the safety and tolerability of MAX-40279 in patients with AML. The primary endpoints of the study include incidence of adverse events and determination of the maximum tolerated dose.