Definition

An orally bioavailable inhibitor of the enzyme N-myristoyl transferase (NMT), with potential antineoplastic activity. Upon oral administration, NMT inhibitor PCLX-001 targets and binds to NMT, especially NMT type 2 (NMT2). This prevents NMT-mediated signaling and myristoylation. This inhibits proliferation of certain cancer cells in which NMT expression is lost. PCLX-001 also inhibits B-cell receptor (BCR) signaling and reduces the levels of Src-family tyrosine kinases (SFKs). NMTs mediate myristoylation, a key process by which the fatty acid myristate is added to proteins and allows proteins to interact with cell membranes and become part of the cell signaling system. NMT expression is lost in numerous cancers, such as blood cancer cells, thereby making these cells more sensitive to PCLX-001 compared to normal cells. The loss of NMT expression may promote tumorigenesis.