Biodexa Announces Positive Top Line Phase I Clinical Trial Results for Diffuse Midline Glioma and Provides R&D Update

Biodexa Pharmaceuticals PLC
(“Biodexa” or the “Company”)

Biodexa Announces Positive Top Line Phase I Clinical Trial Results for Diffuse Midline Glioma and Provides R&D Update

Median Overall Survival of 16.5 Months vs 10.0 months in Comparable Cohort

CARDIFF, United Kingdom, February 23, 2024 (GLOBE NEWSWIRE) – Biodexa Pharmaceuticals PLC, (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs including Type 1 diabetes and rare / orphan brain cancers, announces an R&D update including positive top-line clinical trial results of a recently completed Phase 1 study of MTX110 in patients with diffuse midline glioma, or DMG, and results of a preclinical experiment designed to demonstrate tolimidone’s potential for beta cell proliferation in an in vitro model.

MTX110

In an investigator initiated study conducted by Columbia University Irving Medical Center, patients newly diagnosed with DMG were administered MTX110 via convection enhanced delivery (“CED”) using a subcutaneous pump connected to a catheter directly implanted into the pons in a 3+3 dose-escalating design (NCT 04264143). As this was the first ever study of repeated infusions to the pons via an implanted CED catheter, the primary objective of the study was safety and tolerability and, accordingly, the number of infusions was limited to two, each of 48 hours, 7 days apart. Nine patients were treated in the study (30 M group, n=3; 60 M group, n=4; 90 M group (optimal dose), n=2). One patient in the 60 M group suffered a severe adverse event assessed by the investigators as not related to the study drug but related to the infusion and tumor anatomy. Although the study was not powered to reliably demonstrate efficacy, median overall survival (OS) of patients in the study was 16.5 months. This compares favourably with median survival rate in a cohort of 316 cases of 10.0 months (Jansen et al, 2015. Neuro-Oncology 17(1):160-166). 

Study investigators are planning to present detailed results of the trial at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) being held on June 28-July 2, 2024 in Philadelphia, PA.

Tolimidone

On the Company’s behalf, a CRO conducted an in vitro experiment designed to demonstrate tolimidone’s potential for beta cell proliferation using reaggregated pancreatic islets. The results of the experiment were inconclusive in that they did not correlate with the results previously seen in in vitro and in vivo studies of tolimidone. The Company believes there are a few possible explanations to the outcome of this in vitro study and accordingly, plans to move ahead rapidly with an in vivo preclinical study with similar objectives while continuing preparations for its planned Phase IIa open-label study of tolimidone in patients with Type 1 diabetes due to start recruitment later this year.