Drug intelligence / Profile preview

MTX-241F

Development stage
Preclinical
Lead developer
MEKanistic Therapeutics
Modality
Small Molecules
Administration
Intravenous, Intraperitoneal, Oral
01

Overview

MTX-241F is an experimental small molecule inhibitor that targets both the Epidermal Growth Factor Receptor (EGFR) and Phosphoinositide 3-kinase (PI3K) signaling pathways. Developed by MEKanistic Therapeutics, it is being investigated for the treatment of aggressive brain tumors, including pediatric diffuse midline glioma (DMG) and adult glioblastoma (GBM). Research presented at AACR 2025 demonstrates that MTX-241F can modulate the tumor immunopeptidome by selectively upregulating specific peptides associated with MHC-I presentation, such as those stemming from the NCOA4, VATF, and THOC2 genes. This mechanism suggests that MTX-241F may enhance the visibility of tumor cells to the immune system, potentially offering a synergistic approach when combined with precision immunotherapies.

02

Targets

PIK3CA (Phosphoinositide 3-kinase alpha)DNA-PK (DNA-dependent protein kinase)EGFR (Epidermal growth factor receptor)

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