OncotoX-AML

OncotoX-AML is an engineered biologic therapeutic being developed by Omeros Corporation for the treatment of acute myeloid leukemia (AML). It is designed to selectively target and eliminate both AML blasts and leukemia stem cells (LSCs), which are often responsible for disease relapse. The molecule is approximately half the size of a standard antibody and functions by delivering a toxic payload directly into cancer cells. Its mechanism of action is mutation-agnostic, meaning it remains effective regardless of common AML genetic mutations such as TP53, NPM1, KMT2A, and FLT3. In preclinical models, OncotoX-AML has shown superior efficacy compared to the standard-of-care combination of venetoclax and azacitidine, and primate studies have demonstrated significant reduction in myeloid progenitor cells without causing neutropenia or significant hematologic changes.