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LGH447 + midostaurin is a combination of two investigational small molecule kinase inhibitors studied primarily for the treatment of acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). LGH447 is a selective inhibitor of cyclin-dependent kinase 9 (CDK9), while midostaurin is a multikinase inhibitor that targets FLT3 and other kinases. The combination aims to enhance anti-leukemic activity by simultaneously inhibiting key signaling pathways involved in leukemic cell proliferation and survival. Both agents have been evaluated together in early-phase clinical trials for patients with relapsed/refractory or previously untreated AML[2][3][4][8]. Midostaurin, marketed as Rydapt, is also approved for use in FLT3-mutated AML and certain forms of systemic mastocytosis[1][5][6][7].
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