Drug intelligence / Profile preview

CPX-351 + midostaurin

Development stage
Unknown
Lead developer
Jazz Pharmaceuticals
Modality
Small Molecules, Liposomes → Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous, Oral
01

Overview

CPX-351 + midostaurin is an investigational combination therapy for acute myeloid leukemia (AML) with FLT3 mutations. CPX-351 is a dual-drug liposomal formulation encapsulating daunorubicin and cytarabine in a synergistic 1:5 molar ratio, optimizing interaction between these classic anticancer agents. Midostaurin is a small-molecule oral inhibitor targeting FLT3 (FMS-like tyrosine kinase 3) and additional kinases, intended to block leukemogenic signaling in FLT3-mutated AML. This combination is under clinical investigation (not approved as a combination regimen) primarily for newly diagnosed AML with FLT3 mutations, where preclinical and early clinical trials (e.g., V-FAST) suggest a feasible safety profile and promising remission rates[1][3].

Brand names
VyxeosRydapt
Other names
Vyxeos liposomaldaunorubicin + cytarabine liposomePKC412PKC-412PKC 412
02

Targets

FLT3 (Fms related receptor tyrosine kinase 3)DNA polymerase familyTOP2A (DNA topoisomerase II)PDGFR (PDGFR family)VEGFR2 (Vascular endothelial growth factor receptor 2)KIT (c-KIT proto-oncogene receptor tyrosine kinase)DNA

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