CARDIFF, UK — Biodexa Pharmaceuticals PLC, 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, today provided survival updates from its MAGIC-G1 study (NCT05324501) of MTX110 in patients with recurrent glioblastoma (“rGBM”) being conducted at Duke Cancer Institute and Baptist MD Anderson Cancer Center.
In October 2023, Biodexa announced completion of recruitment of Cohort A of an ongoing open-label Phase I dose-escalation study designed to assess the feasibility and safety of intermittent infusions of MTX110 administered by convection enhanced delivery (“CED”) via implanted refillable pump and catheter. Because no drug-related adverse events were observed within the first 30 days from start of treatment, the minimum number of four patients were recruited into Cohort A.
Patient #1 received weekly infusions of 60µM of MTX110 and survived for 12 months from the start of treatment (OS=12).
Patients #2, 3 and 4 each received weekly infusions of 90µM of MTX110, the expected optimum dose, and remain in the study.
GBM universally recurs and once it does median overall survival according to a retrospective analysis of 299 patients reported in the Journal of Neuro-Oncology is 6.5 months. Currently, no standard of care is established for rGBM.
About Glioblastoma Multiforme (GBM)
GBM is the most common and devastating primary malignant brain tumour in adults with incidence of 3 -4 per 100,000 population. Standard of care for treatment of GBM is typically maximal surgical resection followed by radiotherapy plus concomitant and maintenance temozolomide chemotherapy. Notwithstanding, the multidisciplinary approach, almost all patients experience tumour progression with nearly universal mortality.
About MTX110
MTX110 is a water-soluble form of panobinostat free base, achieved through complexation with hydroxypropyl-β-cyclodextrin (HPBCD), that enables convection-enhanced delivery (CED) at potentially chemotherapeutic doses directly to the site of the tumour. Panobinostat is a hydroxamic acid and acts as a non-selective histone deacetylase inhibitor (pan-HDAC inhibitor). The currently available oral formulation of panobinostat lactate (Farydak®) is not suitable for treatment of brain cancers owing to poor blood-brain barrier penetration and inadequate brain drug concentrations. Based on favourable translational science data, MTX110 is being evaluated clinically as a treatment for recurrent Glioblastoma (NCT05324501), Diffuse Midline Glioma (“DMG”) (NCT04264143) and recurrent medulloblastoma (NCT04315064), and preclinically for treatment of Leptomeningeal Disease. MTX110 is delivered directly into and around the patient’s tumour via a catheter system (e.g. CED or fourth ventricle infusions) to bypass the blood-brain barrier. This technique exposes the tumour to very high drug concentrations while simultaneously minimising systemic drug levels and the potential for toxicity and other side effects. Panobinostat has demonstrated high potency against DIPG tumour cells in in vitro and in vivo models, and in a key study it was the most promising of 83 anticancer agents tested in 14 patient-derived DMG cell lines (Grasso et al, 2015. Nature Medicine 21(6), 555-559).
About Biodexa Pharmaceuticals PLC
Biodexa Pharmaceuticals PLC (listed on NASDAQ: BDRX) is a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs. The Company’s lead development programmes include tolimidone, under development as a novel agent for the treatment of type 1 diabetes and MTX110, which is being studied in aggressive rare/orphan brain cancer indications, and
Tolimidone is an orally delivered, potent and selective inhibitor of lyn kinase. Lyn is a member of the Src family of protein tyrosine kinases, which is mainly expressed in hematopoietic cells, in neural tissues, liver, and adipose tissue. Tolimidone demonstrates glycemic control via insulin sensitization in animal models of diabetes and has the potential to become a first in class blood glucose modulating agent.
MTX110 is a solubilised formulation of the histone deacetylase (HDAC) inhibitor, panobinostat. This proprietary formulation enables delivery of the product via convection-enhanced delivery (CED) at chemotherapeutic doses directly to the site of the tumour, by-passing the blood-brain barrier and potentially avoiding systemic toxicity.
Biodexa is supported by three proprietary drug delivery technologies focused on improving the bio-delivery and bio-distribution of medicines. Biodexa’s headquarters and R&D facility is in Cardiff, UK.
Contacts
Biodexa Pharmaceuticals PLC
Stephen Stamp, CEO, CFO
Tel: +44 (0)29 20480 180
Edison Group (US Investor Relations)
Laine Yonker
Tel: +1 (610) 716 2868
Email: [email protected]