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M801 project

NMS-088 is a potent inhibitor of FLT3, KIT and CSF1R kinases which play a role in Acute Myeloid Leukemia (AML), with potential for best-in-class. Activating mutations of FLT3 are present in ~30% of AML cases and associated with poor prognosis. First generation FLT3 inhibitors have shown activity in AML as single agents and in combination with standard therapy. NMS-088 is the most active among the FLT3 comparators tested in our labs, a finding especially evident in the presence of the FLT3 “gatekeeper mutation which results in clinical resistance to treatment with other FLT3 inhibitors. The drug has shown synergy and tolerability in combination with standard of care treatment in preclinical models of AML. Furthermore, there is a rationale for use of the molecule in Chronic Myelomonocytic Leukemia (CMML) on the basis of the effects of CSF1R inhibition, a highly expressed and activated target in this tumor type. Based on our preclinical findings, a Phase I/II study in relapsed/refractory AML and CMML patients is ongoing and combination studies are planned.