zelenirstat

Zelenirstat is a first-in-class, oral small molecule inhibitor of N-myristoyltransferase 1 (NMT1) and N-myristoyltransferase 2 (NMT2), enzymes responsible for the myristoylation of proteins—a modification critical for membrane localization and function of many signaling proteins in cancer cells[2][3][5]. By inhibiting both NMT1 and NMT2, zelenirstat disrupts key oncogenic pathways including those involving EGFR, VEGFR, B-cell receptor signaling, Src family kinases (SFKs), and mitochondrial complex I assembly required for oxidative phosphorylation[6][8]. This dual mechanism leads to impaired cell signaling and energy production in cancer cells. Zelenirstat is being developed by Pacylex Pharmaceuticals primarily as an anti-cancer agent for hematologic malignancies such as acute myeloid leukemia (AML) and lymphomas as well as solid tumors; it also shows potential antiviral activity against viruses dependent on myristoylated proteins[1][4][5]. The drug has completed phase 1 clinical trials demonstrating safety and preliminary efficacy in heavily pretreated patients with advanced solid tumors or relapsed/refractory B-cell lymphoma[2][7].

Other names

PCLX-001PCLX001PCLX 001DDD86481DDD-86481DDD 86481zelenirstat