MTX-241

MTX-241 is a novel, small molecule dual inhibitor of the epidermal growth factor receptor (EGFR) and phosphoinositide 3-kinase (PI3K) signaling pathways. Developed by Mekanistic Therapeutics, it was designed using computational modeling to exploit structurally related ATP binding sites of both kinases, aiming to overcome resistance mechanisms and compensatory signaling common in single-agent kinase inhibition. MTX-241 is specifically engineered for blood-brain barrier (BBB) penetrance and has demonstrated potent cytotoxic activity in preclinical models of adult and pediatric high-grade gliomas, including glioblastoma (GBM) and diffuse intrinsic pontine glioma (DIPG). Beyond kinase inhibition, the compound exhibits unique metabolic effects by suppressing both glycolytic pathways and oxidative phosphorylation in malignant cells.