Drug Detail

Information about Omacetaxine mepesuccinate

Generic Name
Omacetaxine mepesuccinate
Brand Name (US)
ChemGenex Pharmaceuticals
Drug Type
Protein Translation Inhibitor
Approval Status
Phase 3
Overall Strategy
GIST cell based
Inhibit protein translation
Drug Category
Protein translation inhibitor

A semisynthetic formulation of the cytotoxic plant alkaloid homoharringtonine isolated from the evergreen tree Cephalotaxus with potential antitumor activity. Omacetaxine mepesuccinate binds to the 80S ribosome in eukaryotic cells and inhibits protein synthesis by interfering with chain elongation. This agent also induces differentiation and apoptosis in some cancer cell types.
Omacetaxine has demonstrated clinical activity in the highly resistant T315I mutation in CML.
Omacetaxine mepesuccinate is a first-in-class cetaxine with demonstrated clinical activity as a single agent in a range of hematological malignancies. Omacetaxine has a novel mechanism of action, specifically binding to the ribosomal A-site cleft and inhibiting protein translation of short-lived oncoproteins that are upregulated in leukemic cells (particularly Cyclin-D1, Mcl-1 and c-Myc). As omacetaxine acts independently of tyrosine kinase inhibitors, it may have a therapeutic advantage for patients who have developed resistance to TKIs. Omacetaxine is administered subcutaneously.