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MTX110 is a novel, investigational formulation of the anticancer drug panobinostat designed for direct delivery to brain tumors. It consists of a water-soluble form of the free base of panobinostat achieved through complexation with hydroxypropyl-β-cyclodextrin (HPBCD), enabling convection-enhanced delivery (CED) directly into and around brain tumors. Panobinostat is a non-selective histone deacetylase (HDAC) inhibitor that induces cell cycle arrest and apoptosis in tumor cells by increasing acetylation of histones and other proteins, leading to chromatin relaxation and transcriptional activation. The standard oral form of panobinostat has poor blood-brain barrier penetration; MTX110 overcomes this limitation by local administration via catheter systems such as CED or fourth ventricle infusions. This approach achieves high intratumoral concentrations while minimizing systemic exposure and toxicity. MTX110 is being developed primarily for aggressive pediatric brain cancers like diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG), as well as recurrent glioblastoma in adults[2][3][4][5][6][7][8].
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