Biodexa Updates on MTX110 Phase 1 Study in Recurrent Glioblastoma

Biodexa Pharmaceuticals PLC, a clinical stage biopharmaceutical company listed on Nasdaq under the symbol BDRX, has provided an update on its Phase 1 study of MTX110 in patients with recurrent glioblastoma (rGBM). This open-label study, known as MAGIC-G1 (NCT 05324501), aims to evaluate the feasibility and safety of administering MTX110 via convection enhanced delivery (CED). The company recently completed recruitment for Cohort A, consisting of four patients.

In this study, MTX110, a water-soluble form of panobinostat, is delivered intermittently through an implanted refillable pump and catheter system. This method allows high drug concentrations to be administered directly to the tumor, bypassing the blood-brain barrier and minimizing systemic exposure and potential side effects.

The current status of patients in Cohort A is as follows:

- Patient #1 and Patient #2 have passed away, with overall survival (OS) of 12 months and 13 months, respectively, since the start of treatment.
- Patient #3 is still in post-study follow-up, with progression free survival (PFS) of six months and OS thus far of 13 months.
- Patient #4 has not yet experienced confirmed progression and currently shows PFS and OS of 12 months since beginning treatment.

Glioblastoma (GBM) is a highly aggressive primary malignancy of the central nervous system (CNS) in adults, with an annual incidence of approximately 3 per 100,000 people. The standard treatment for newly diagnosed GBM typically involves the maximum safe surgical resection, followed by concurrent radiotherapy and temozolomide (TMZ). Despite these measures, GBM almost always recurs, with median PFS ranging from 1.5 to 6.0 months and median OS from 2.0 to 9.0 months, according to a study by Birzu et al.

The interim data from this rGBM study build on previous findings. In July 2024, Biodexa presented data from a Phase 1 study of MTX110 in nine patients with Diffuse Midline Glioma (DMG) at the 21st International Symposium on Paediatric Neuro-Oncology (ISPNO 2024). This study showed a median OS of 16.5 months after only two infusions and a single patient receiving the optimum dose. An earlier Phase 1 study conducted by the University of California San Francisco reported a median OS of 26.1 months in seven DMG patients treated with MTX110. These figures compare favorably with a median OS of 10.0 months in a cohort of 316 cases, as reported by Jansen et al.

MTX110’s formulation involves complexation with hydroxypropyl-β-cyclodextrin (HPBCD) to enable CED at potentially therapeutic doses. Panobinostat, the active component, is a non-selective histone deacetylase inhibitor (pan-HDAC inhibitor) known for its high potency against DIPG (DMG) and GBM tumor cells in both in vitro and in vivo models. The oral form of panobinostat lactate (Farydak®) is ineffective for brain cancers due to poor blood-brain barrier penetration and low brain concentrations.

In addition to MTX110, Biodexa's primary development programs include eRapa, an oral tablet formulation of rapamycin for Familial Adenomatous Polyposis and Non-Muscle Invasive Bladder Cancer; and tolimidone, an orally delivered inhibitor of Lyn kinase for type 1 diabetes treatment. The company’s proprietary drug delivery technologies aim to enhance the bio-delivery and bio-distribution of medicines.

Biodexa is headquartered in Cardiff, UK, where it also maintains its research and development facility. The company is dedicated to advancing innovative treatments for diseases with unmet medical needs.