Positive Phase 1 Clinical Data of MTX110 in DMG Brain Cancer Demonstrating Increased Survival Presented at ISPNO 2024

Core Insights - Biodexa Pharmaceuticals PLC presented positive Phase 1 clinical data for MTX110 in treating Diffuse Midline Glioma (DMG), showing a median overall survival of 16.5 months compared to 10.0 months in historical cohorts [1][2][8] Study Design and Results - The Phase 1 study was an open-label, investigator-initiated trial conducted by Columbia University, involving nine patients newly diagnosed with DMG [3][8] - Patients received two infusions of MTX110 via convection enhanced delivery (CED), with a median progression-free survival of 10 months and overall survival ranging from 12 to 35 months [2][7] - The primary endpoint was to determine the dosage regimen for a proposed Phase II study, with preliminary data suggesting a dose between 60μM and 90μM for future trials [9] Safety and Tolerability - The treatment was well tolerated, with one Grade 4 adverse event deemed unrelated to the drug, while most other adverse events were classified as Grade 2 to 3 [7][8] - The study was not powered to demonstrate statistical significance for efficacy, but the results indicate a favorable outcome compared to historical data [2][10] Product Information - MTX110 is a solubilized formulation of the histone deacetylase (HDAC) inhibitor panobinostat, designed to bypass the blood-brain barrier and minimize systemic toxicity [13][20] - The company is also developing other products, including eRapa for Familial Adenomatous Polyposis and Non-Muscle Invasive Bladder Cancer, and tolimidone for type 1 diabetes [11][12]