panobinostat (MTX110) / Biodexa Pharma - LARVOL DELTA

Infusion of Panobinostat (MTX110) Into the Fourth Ventricle in Children and Adults with Recurrent Medulloblastoma (clinicaltrials.gov) - P1 | N=2 | Completed | Sponsor: The University of Texas Health Science Center, Houston | Recruiting ➔ Completed

Trial completion • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor

MAGIC-G1: A Study of Intra-tumoral Administered MTX110 in Patients With Recurrent Glioblastoma (clinicaltrials.gov) - P1 | N=4 | Terminated | Sponsor: Biodexa Pharmaceuticals | N=36 ➔ 4 | Trial completion date: Aug 2028 ➔ Sep 2024 | Active, not recruiting ➔ Terminated | Trial primary completion date: Aug 2028 ➔ Sep 2024; Strategic decision

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

Biodexa's MX-110 Continues to Show Promise in Recurrent Glioblastoma (rGBM), the Deadliest Brain Cancer (ACCESSWIRE) - P1 | N=36 | MAGIC-G1 (NCT05324501) | Sponsor: Biodexa Pharmaceuticals | "Biodexa Pharmaceuticals PLC...released positive patient updates on progression-free survival (PFS) and overall survival (OS) from the ongoing MAGIC-1 study of MTX110 in recurrent glioblastoma....The study (NCT05324501) is designed to evaluate the feasibility, safety and efficacy of treating rGBM patients with MTX110. The company earlier reported patients #1 and #2 in Cohort A achieved overall survival (OS) of 12 and 13 months, respectively. The remaining two patients in Cohort A remain on follow-up. Patient #3 has achieved 13 months OS to date, with six months progression free survival (PFS). Patient #4 has not progressed and achieved 12 months PFS and 12 months OS to date."

P1 data • Glioblastoma

Biodexa Provides Update on Progression Free and Overall Survival in Phase 1 Study of MTX110 in Recurrent Glioblastoma (GlobeNewswire) - P1 | N=36 | NCT05324501 | Sponsor: Biodexa Pharmaceuticals | "Biodexa Pharmaceuticals PLC...announces an update in respect of its open label Phase 1 study of MTX110 in recurrent glioblastoma...In October 2023, the Company announced completed recruitment of four patients into cohort A of its Phase 1 study of MTX110 (also known as MAGIC-G1 study) (NCT05324501) in patients with recurrent glioblastoma (rGBM)...Patients #1 and #2 have deceased with overall survival (OS) since start of treatment of 12 months and 13 months, respectively....Patients #3 and #4 remain in post-study follow-up. Patient #3 had progression free survival (PFS) of six months and OS thus far of 13 months since start of treatment. Patient #4 has not yet had confirmed progression and, as of today, has PFS and OS of 12 months since start of treatment."

P1 data • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

Interim results for the six months ended June 30, 2024 (GlobeNewswire) - "R&D costs decreased to £2.19 million in 1H24 (1H23: £2.25 million) reflecting a reduction in spend on the MAGIC-G1 study in rGBM...ministrative costs decreased to £2.03 million (1H23: £2.29 million) as a result of a positive reversal in foreign exchange and a reduction in professional fees offset by increases in share-based payment charge and sundry other costs."

Commercial • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

Synergistic Efficacy of CDK4/6 Inhibitor Abemaciclib and HDAC Inhibitor Panobinostat in Pancreatic Cancer Cells (Multidisciplinary Digital Publishing Institute) - "The Abe-Pan combination exhibited excellent efficacy and produced synergistic effects, altering the expression of cell cycle proteins and epigenetic markers. Pan, alone and in combination with Abe, caused apoptosis in pancreatic cancer cells. Abe-Pan co-treatment showed relative safety in normal human dermal fibroblasts. Our novel combination treatment of Abe and Pan shows synergistic effects on PDAC cells."

Preclinical • Pancreatic Cancer

A Phase I study examining the feasibility of intermittent convection-enhanced delivery (CED) of MTX110 for the treatment of children with newly diagnosed diffuse midline gliomas (ISPNO 2024) - "Using an implantable subcutaneous pump connected with a catheter directly implanted into the pons we have conducted a phase I, 3+3 dose escalation study to investigate the safety and feasibility of repeated infusions of MTX110 (Biodexa Ltd), a water-soluble formulation of panobinostat, via CED. The study has demonstrated safety and feasibility of repeated infusions of MTX110 via CED in DMG patients with a favorable trend in overall survival warranting further trials with higher number of infusion pulses."

Clinical • P1 data • Brain Cancer • CNS Tumor • Diffuse Midline Glioma • Glioma • Hematological Disorders • Oncology • Solid Tumor

Positive Phase 1 Clinical Data of MTX110 in DMG Brain Cancer Demonstrating Increased Survival Presented at ISPNO 2024 (GlobeNewswire) - "Biodexa Pharmaceuticals PLC...announces data from a Phase 1 study of MTX110 in Diffuse Midline Glioma ('DMG') f/k/a Diffuse Intrinsic Pontine Glioma, or DIPG, an orphan pediatric brain cancer were presented over the weekend at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) in Philadelphia, PA....Although the study was not powered to reliably demonstrate efficacy, median progression free survival (PFS) was 10 months (range 8 to 20 months) and overall survival (OS) of patients in the study was 16.5 months (range 12 to 35 months). This compares favourably with median OS in a cohort of 316 cases of 10.0 months."

P1 data • Brain Cancer • CNS Tumor • Glioma • Oncology • Solid Tumor

MAGIC-G1: A Study of Intra-tumoral Administered MTX110 in Patients With Recurrent Glioblastoma (clinicaltrials.gov) - P1 | N=36 | Active, not recruiting | Sponsor: Biodexa Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

Biodexa Announces Positive Top Line Phase I Clinical Trial Results for Diffuse Midline Glioma and Provides R&D Update (GlobeNewswire) - P1 | N=9 | NCT04264143 | "Biodexa Pharmaceuticals PLC...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...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....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."

P1 data • Astrocytoma • Brain Cancer • CNS Tumor • Diffuse Midline Glioma • Glioma • Oncology • Solid Tumor

Biodexa Reports 12 Month Survival in MAGIC-G1 Study of MTX110 in Recurrent Glioblastoma Patients (GlobeNewswire) - P1 |N=36 | MAGIC-G1 (NCT05324501) | Sponsor: Biodexa Pharmaceuticals | "Biodexa Pharmaceuticals PLC...provided survival updates from its MAGIC-G1 study (NCT05324501) of MTX110 in patients with recurrent glioblastoma....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."

P1 data • Glioblastoma

Biodexa CEO Issues Shareholder Letter Highlighting Progress in 2023 and Expected Milestones in 2024 (GlobeNewswire) - "MTX110...Because we saw no drug-related adverse events during dose escalation, we were able to recruit the minimum number in the first cohort of patients in our Phase I study (called the MAGIC-G1 study) in recurrent glioblastoma multiforme, or GBM...We expect to announce Progression Free Survival data on the first cohort of patients in mid-2024."

P1 data • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

CED of MTX110 Newly Diagnosed Diffuse Midline Gliomas (clinicaltrials.gov) - P1 | N=9 | Completed | Sponsor: Luca Szalontay | Recruiting ➔ Completed

Trial completion • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Glioma • Oncology • Solid Tumor

MAGIC-G1: A PHASE 1 DOSE-ESCALATION TRIAL OF WEEKLY INTRATUMORAL ADMINISTRATION OF THE SOLUBILISED FORMULATION OF PANOBINOSTAT (MTX110) IN PATIENTS WITH RECURRENT GLIOBLASTOMA (RGBM) (EANO 2023) - "MATERIAL AND METHODSEligibility includes adult rGBM patients with a single enhancing lesion ≤3cm in maximal diameter; ≥4 weeks after chemotherapy, bevacizumab, or study drug; adequate organ function; and KPS ≥60%...Patients in cohort 2 will receive MTX110 in combination with lomustine at a starting MTX110 dose of 60 μM and following the same dose-escalation approach...CONCLUSION Weekly intratumoral administration of MTX110 via a fully internally implanted CED pump system is feasible. Updated results will be presented."

Clinical • P1 data • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

Biodexa Completes Recruitment of Cohort A in Study of MTX110 in Patients with Recurrent Glioblastoma (GlobeNewswire) - "Biodexa Pharmaceuticals PLC...is pleased to announce that it has completed recruitment into cohort A of its ongoing Phase 1 study of MTX110...in patients with recurrent glioblastoma (rGBM)....Enrolment in cohort B will commence upon approval by the study DSMB, which is anticipated to be received towards the end of October 2023....In addition, the Company is planning to add two more investigational centres into the study with activation expected in December 2023 and January 2024, respectively."

DSMB • Enrollment status • Trial status • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

Biodexa to Present Recruitment and Treatment Update in Phase 1 Study of MTX-110 (MAGIC-G1 Study) in Patients with Recurrent Glioblastoma at 2023 Annual European Association of Neuro-Oncology Meeting (EANO) (GlobeNewswire) - P1 | N=36 | MAGIC-G1 (NCT05324501) | Sponsor: Biodexa Pharmaceuticals | "Biodexa Pharmaceuticals PLC...is pleased to announce that it will be presenting an update on the recruitment and treatment of patients in the ongoing Phase 1 study of MTX-110 (also known as MAGIC-G1 study) in patients with recurrent glioblastoma (rGB) during a poster session on 22 September 2023, at the 2023 annual EANO meeting in Rotterdam, the Netherlands (NCT05324501)....As of the time of this announcement, three patients have been dosed in the first cohort of the study. No dose-limiting toxicities have been observed at any dose level; and all study-related, non-surgical adverse events were grade 1 or 2 and correlated with the location of the lesion being treated."

P1 data • Trial status • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

Completion of Enrolment and Treatment in the Phase 1 Study of MTX110 in the Treatment of Children with Newly Diagnosed Diffuse Midline Gliomas (DMGs) (GlobeNewswire) - "Biodexa Ltd...is pleased to announce completion of enrolment and treatment of nine paediatric patients into the ongoing investigator-sponsored Phase I study of MTX-110 in newly diagnosed DMGs (NCT04264143). All of the patients (age range 4-17 years) were enrolled at the Columbia University Irving Medical Centre and received radiation therapy as per the institution’s standard of care....Full study results are expected to be made public around the 1st quarter of 2024."

Enrollment closed • P1 data • Trial status • Brain Cancer • CNS Tumor • Glioma • Oncology • Solid Tumor

Publication of Manuscript from the PNOC015 (a.k.a. MTD-201) Study in Patients with Diffuse Midline Glioma (DMG) (GlobeNewswire) - "Biodexa Pharmaceuticals PLC...is pleased to announce the publication of the results of an investigator-initiated Phase I study PNOC015 (also known as MTD-201) of MTX110 in the treatment of pediatric patients with DMG (formerly known as Diffuse Intrinsic Pontine Glioma, or DIPG) in the Journal of Neuro-Oncology (NCT 03566199). The study recruited seven pediatric patients with confirmed diagnosis of DMG, after radiation therapy. Convection Enhanced Delivery (CED) of MTX110 at doses escalating from 30µM to 90μM was performed, with a total of 48 infusions across all patients. The study has demonstrated acceptable safety profile of the treatment (three patients reported dose-limiting toxicities) and encouraging efficacy with median overall survival (OS) of 26.1 months comparing favourably with historical data of OS of 10.0 months."

P1 data • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Glioma • Oncology • Solid Tumor

Infusion of Panobinostat (MTX110) Into the Fourth Ventricle in Children and Adults With Recurrent Medulloblastoma (clinicaltrials.gov) - P1 | N=5 | Recruiting | Sponsor: The University of Texas Health Science Center, Houston | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Dec 2024 ➔ Dec 2025

Trial completion date • Trial primary completion date • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor

PNOC015: Repeated convection enhanced delivery (CED) of MTX110 (aqueous panobinostat) in children with newly diagnosed diffuse intrinsic pontine glioma (DIPG). (PubMed, Neuro Oncol) - "Repeat CED of MTX110 with real time imaging with gadoteridol is tolerable for patients with DIPG. Median OS of 26.1 months compares favorably with historical data for children with DIPG. The results support further investigation of this strategy in a larger cohort."

Journal • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Glioma • Oncology • Solid Tumor

Initiation of a phase 1 dose-escalating study of weekly direct-to-brain administration of solubilised formulation of panobinostat (MTX110) in patients with recurrent glioblastoma (MAGIC-G1 study) (SNO 2022) - " MAGIC-G1 study is a Phase 1 two-cohort dose-escalating study that will assess safety and feasibility of intermittent intratumoral administration of MTX110 by CED to rGBM patients; either as a single agent (Cohort 1) or in combination with lomustine (Cohort 2). Cohort 1 recruitment will be completed before Cohort 2 starts dosing. Study results are expected in 2023."

Clinical • New P1 trial • P1 data • Brain Cancer • Diffuse Intrinsic Pontine Glioma • Glioblastoma • Oncology • Pediatrics • Solid Tumor

MAGIC-G1: A Study of Intra-tumoral Administered MTX110 in Patients With Recurrent Glioblastoma (clinicaltrials.gov) - P1 | N=36 | Recruiting | Sponsor: Midatech Ltd | Trial completion date: Sep 2024 ➔ Aug 2028 | Trial primary completion date: Sep 2024 ➔ Aug 2028

Combination therapy • Monotherapy • Trial completion date • Trial primary completion date • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

Midatech Pharma PLC Announces Planned Dose Escalation in MTX-110 Phase 1 Study (Issuer Direct) - "Midatech Pharma PLC...is pleased to announce that following completion of one month of treatment with MTX-110 in the first patient, its Phase I study of MTX-110 in recurrent glioblastoma ('rGB') (NCT05324501) will continue with a planned dose escalation following a positive recommendation from the study's Data Safety Monitoring Board ('DSMB'). The Phase I study, the MAGIC-G1 study, is an open-label, dose escalation study designed to assess the feasibility and safety of intermittent infusions of MTX-110 administered by convection enhanced delivery (CED) via implanted refillable pump and catheter....The first patient in the study was dosed at 60uM of MTX-110 via direct-to-tumour delivery and has received four 48-hour infusions over a period of four weeks....Patient 1 continues treatment as per the study protocol and patient 2 is expected to start treatment by the end of January 2023 and will receive the 90uM dose."

Trial status • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

Midatech Pharma PLC - First Patient Enrolled in Phase 1 Study of MTX-110 (Issuer Direct) - "Midatech Pharma PLC...is pleased to announce the enrolment of the first patient into its Phase 1 study of MTX110 in recurrent glioblastoma (rGB) (NCT05324501) at the Preston Robert Tisch Brain Tumor Center at Duke University, USA. The Phase I study is an open-label, 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. The study aims to recruit two cohorts, each with a minimum of four patients; the first cohort will receive MTX110 only and the second cohort will receive MTX110 in combination with lomustine."

Trial status • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

[VIRTUAL] PNOC015: Phase 1 study of MTX110 delivered by Convection Enhanced Delivery (CED) in children with newly diagnosed diffuse intrinsic pontine glioma (DIPG) previously treated with radiation therapy (SNO 2020) - "Repeat CED of MTX110 at the highest dose is tolerable. Tissue concentrations are likely to be substantially higher compared to oral dosing. Pharmacodynamic effects will be presented."

Clinical • P1 data • Diffuse Intrinsic Pontine Glioma • Glioma • Oncology • Peripheral Neuropathic Pain • Solid Tumor