Biodexa Provides Update on Progression Free and Overall Survival in Phase 1 Study of MTX110 in Recurrent Glioblastoma
Biodexa Pharmaceuticals PLC (Nasdaq: BDRX) has provided an update on its Phase 1 study of MTX110 in recurrent glioblastoma (rGBM). The study, known as MAGIC-G1, involves intermittent infusions of MTX110 administered by convection enhanced delivery (CED) via implanted refillable pump and catheter.
In Cohort A of the study:
- Two patients have deceased with overall survival (OS) of 12 and 13 months
- One patient had progression-free survival (PFS) of 6 months and current OS of 13 months
- One patient has not yet progressed, with current PFS and OS of 12 months
These results compare favorably with typical median PFS of 1.5–6.0 months and median OS of 2.0–9.0 months for rGBM. The data builds on previous promising results from two Phase 1 studies of MTX110 in Diffuse Midline Glioma (DMG), which showed median OS of 16.5 and 26.1 months, compared to a typical median OS of 10.0 months.
Biodexa Pharmaceuticals PLC (Nasdaq: BDRX) ha fornito un aggiornamento sul suo studio di Fase 1 di MTX110 nei casi di glioblastoma ricorrente (rGBM). Lo studio, noto come MAGIC-G1, prevede infusione intermittente di MTX110 somministrata tramite un sistema di erogazione migliorata per convezione (CED) attraverso una pompa ricaricabile impiantata e un catetere.
Nel Gruppo A dello studio:
- Due pazienti sono deceduti con una sopravvivenza complessiva (OS) di 12 e 13 mesi
- Un paziente ha avuto una sopravvivenza senza progressione (PFS) di 6 mesi e una OS attuale di 13 mesi
- Un paziente non ha ancora mostrato progressione, con PFS e OS attuali di 12 mesi
Questi risultati sono favorevoli rispetto ai valori medi tipici di PFS di 1,5–6,0 mesi e di OS di 2,0–9,0 mesi per rGBM. I dati si basano su risultati promettenti precedenti provenienti da due studi di Fase 1 su MTX110 in Glioma Diffuso della Linea Mediana (DMG), che hanno mostrato una OS mediana di 16,5 e 26,1 mesi, rispetto a una OS mediana tipica di 10,0 mesi.
Biodexa Pharmaceuticals PLC (Nasdaq: BDRX) ha proporcionado una actualización sobre su estudio de Fase 1 de MTX110 en glioblastoma recurrente (rGBM). El estudio, conocido como MAGIC-G1, implica infusiones intermitentes de MTX110 administradas por entrega mejorada por convección (CED) a través de una bomba y catéter implantados y recargables.
En el Cohorte A del estudio:
- Dos pacientes han fallecido con una supervivencia general (OS) de 12 y 13 meses
- Un paciente tuvo una supervivencia libre de progresión (PFS) de 6 meses y una OS actual de 13 meses
- Un paciente aún no ha progresado, con PFS y OS actuales de 12 meses
Estos resultados son favorables en comparación con la PFS media típica de 1.5 a 6.0 meses y la OS media de 2.0 a 9.0 meses para rGBM. Los datos se basan en resultados prometedores previos de dos estudios de Fase 1 de MTX110 en Glioma Difuso de Línea Media (DMG), que mostraron una OS mediana de 16.5 y 26.1 meses, en comparación con una OS mediana típica de 10.0 meses.
Biodexa Pharmaceuticals PLC (Nasdaq: BDRX)는 재발성 교모세포종(rGBM)에 대한 MTX110의 1상 연구에 대한 업데이트를 제공했습니다. MAGIC-G1로 알려진 이 연구는 이식 가능한 충전 펌프 및 카테터를 통해 대류 강화 전달(CED) 방식으로 MTX110를 간헐적으로 주입하는 것을 포함합니다.
연구의 A군에서는:
- 두 명의 환자가 12개월 및 13개월의 전체 생존(OS)으로 사망했습니다.
- 한 환자는 6개월의 무변화 생존(PFS)과 현재 13개월의 OS를 보였습니다.
- 한 환자는 아직 진행되지 않아 현재 PFS 및 OS가 12개월입니다.
이러한 결과는 rGBM의 전형적인 PFS 중간값인 1.5–6.0개월 및 OS 중간값인 2.0–9.0개월과 비교할 때 긍정적입니다. 이 데이터는 MTX110을 사용한 두 개의 DMG(확산 중심 뇌종양) 1상 연구에서 나왔던 이전의 유망한 결과를 바탕으로 하며, 그 연구는 10.0개월의 전형적인 OS에 비해 각각 16.5개월과 26.1개월의 OS 중간값을 보여주었습니다.
Biodexa Pharmaceuticals PLC (Nasdaq: BDRX) a fourni une mise à jour sur son étude de Phase 1 de MTX110 dans le glioblastome récurrent (rGBM). L'étude, connue sous le nom de MAGIC-G1, implique des perfusions intermittentes de MTX110 administrées par un système de délivrance améliorée par convection (CED) via une pompe implantée rechargeable et un cathéter.
Dans le Cohorte A de l'étude :
- Deux patients sont décédés avec une survie globale (OS) de 12 et 13 mois
- Un patient a eu une survie sans progression (PFS) de 6 mois et une OS actuelle de 13 mois
- Un patient n'a pas encore progressé, avec une PFS et une OS actuelles de 12 mois
Ces résultats se comparent favorablement aux médianes typiques de PFS de 1,5 à 6,0 mois et de OS de 2,0 à 9,0 mois pour rGBM. Les données s'appuient sur des résultats prometteurs précédents de deux études de Phase 1 sur MTX110 dans le gliome diffus de la ligne médiane (DMG), qui ont montré des moyennes de OS de 16,5 et 26,1 mois, par rapport à une OS médiane typique de 10,0 mois.
Biodexa Pharmaceuticals PLC (Nasdaq: BDRX) hat ein Update zu seiner Phase-1-Studie von MTX110 bei rezidivierendem Glioblastom (rGBM) bereitgestellt. Die Studie, bekannt als MAGIC-G1, umfasst intermittierende Infusionen von MTX110, die über eine implantierte nachfüllbare Pumpe und einen Katheter durch konvektionsverstärkte Abgabe (CED) verabreicht werden.
In Kohorte A der Studie:
- Zwei Patienten sind verstorben mit einer Gesamtüberlebenszeit (OS) von 12 und 13 Monaten
- Ein Patient hatte eine progressionsfreie Überlebenszeit (PFS) von 6 Monaten und eine aktuelle OS von 13 Monaten
- Ein Patient hat sich bisher nicht verschlechtert, mit einer aktuellen PFS und OS von 12 Monaten
Diese Ergebnisse sind im Vergleich zu den typischen medianen Werten von PFS von 1,5–6,0 Monaten und medianen OS von 2,0–9,0 Monaten für rGBM positiv. Die Daten bauen auf vorherigen vielversprechenden Ergebnissen von zwei Phase-1-Studien zu MTX110 bei diffus midline Glioma (DMG) auf, die mediane OS von 16,5 und 26,1 Monaten zeigten, verglichen mit einem typischen medianen OS von 10,0 Monaten.
- Cohort A patients show overall survival ranging from 12 to 13+ months, exceeding typical rGBM survival rates
- One patient maintains 12 months of progression-free survival, surpassing typical rGBM outcomes
- Results build on previous positive data from MTX110 studies in Diffuse Midline Glioma
- Two out of four patients in Cohort A have deceased
- sample size of only four patients in the current rGBM study
Insights
The Phase 1 study results for MTX110 in recurrent glioblastoma (rGBM) show promising early signs. With overall survival (OS) ranging from 12-13 months for the first two patients and ongoing survival for the other two, these outcomes appear favorable compared to typical survival rates for rGBM. The progression-free survival (PFS) of 6-12 months is also encouraging.
Importantly, these results build upon MTX110's performance in two Phase 1 studies for diffuse midline glioma (DMG), where median OS reached 16.5 and 26.1 months, substantially exceeding the typical 10-month median. This consistency across multiple aggressive brain cancers suggests MTX110 may have broad potential.
However, caution is warranted due to the small sample size and early stage of research. The convection-enhanced delivery method is novel and requires further evaluation for safety and efficacy. Overall, these results justify continued investigation of MTX110 as a potential treatment for aggressive brain cancers with poor prognoses.
October 4, 2024
Biodexa Provides Update on Progression Free and Overall Survival in
Phase 1 Study of MTX110 in Recurrent Glioblastoma
Cohort A Data on Overall Survival Compare Favorably with Published Survival Rates
Results Build on MTX110’s Performance in Two Phase 1 Studies in Diffuse Midline Glioma
Biodexa Pharmaceuticals PLC (“Biodexa” or “the Company”), (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs 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)(NCT 05324501) in patients with recurrent glioblastoma (rGBM).
MAGIC-G1 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. Patients received MTX110 via intermittent repeated CED infusions. As of today, the status of patients in Cohort A is as follows:
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.
Glioblastoma (GBM) is the most aggressive central nervous system (CNS) primary malignancy in adults with an annual incidence is approximately 3 per 100,000 population. The standard of care in newly diagnosed GBM includes maximal safe surgical resection, followed by concurrent radiotherapy and temozolomide (TMZ). GBM virtually always recurs with median PFS of 1.5–6.0 months and median OS of 2.0–9.0 months (source: Birzu et al. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7794906).
These interim data in rGBM build on the data announced by the Company on July 2, 2024, and presented at the recent 21st International Symposium on Paediatric Neuro-Oncology (ISPNO 2024) of a Phase 1 study of MTX110 in nine patients with Diffuse Midline Glioma (“DMG”) which showed, after only two infusions and a single patient at optimum dose, median OS of 16.5 months. In an earlier Phase 1 study conducted by the University of California San Francisco of MTX110 in seven patients with DMG which showed median OS of 26.1 months. These data compare favorably with median OS in a cohort of 316 cases of 10.0 months (source: Jansen et al, 2015. Neuro-Oncology 17(1):160-166).
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 tumor. 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) and recurrent medulloblastoma (NCT04315064). MTX110 is delivered directly into and around the patient’s tumor via a catheter system (e.g. CED or fourth ventricle infusions) to bypass the blood-brain barrier. This technique exposes the tumor 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 (DMG) and GBM tumor 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 DIPG (DMG) cell lines (Grasso et al, 2015. Nature Medicine 21(6), 555-559).
For more information, please contact:
Biodexa Pharmaceuticals PLC |
| Stephen Stamp, CEO, CFO |
| Tel: +44 (0)29 20480 180 |
| www.biodexapharma.com |
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 programs include eRapa, under development for Familial Adenomatous Polyposis and Non-Muscle Invasive Blader Cancer; tolimidone, under development for the treatment of type 1 diabetes; and MTX110, which is being studied in aggressive rare/orphan brain cancer indications.
eRapa is a proprietary oral tablet formulation of rapamycin, also known as sirolimus. Rapamycin is an mTOR (mammalian Target Of Rapamycin) inhibitor. mTOR has been shown to have a significant role in the signalling pathway that regulates cellular metabolism, growth and proliferation and is activated during tumorigenesis.
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 glycaemic 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 solubilized 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 tumor, 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. For more information visit www.biodexapharma.com.
Forward-Looking Statements
Certain statements in this announcement may constitute “forward-looking statements” within the meaning of legislation in the United Kingdom and/or United States. Such statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and are based on management’s belief or interpretation. All statements contained in this announcement that do not relate to matters of historical fact should be considered forward-looking statements. In certain cases, forward-looking statements can be identified by the use of words such as “plans”, “expects” or “does not anticipate”, or “believes”, or variations of such words and phrases or statements that certain actions, events or results “may”, “could”, “would”, “might” or “will be taken”, “occur” or “be achieved.” Forward-looking statements and information are subject to various known and unknown risks and uncertainties, many of which are beyond the ability of the Company to control or predict, that may cause their actual results, performance or achievements to be materially different from those expressed or implied thereby, and are developed based on assumptions about such risks, uncertainties and other factors set out herein.
Reference should be made to those documents that Biodexa shall file from time to time or announcements that may be made by Biodexa in accordance with the rules and regulations promulgated by the SEC, which contain and identify other important factors that could cause actual results to differ materially from those contained in any projections or forward-looking statements. These forward-looking statements speak only as of the date of this announcement. All subsequent written and oral forward-looking statements by or concerning Biodexa are expressly qualified in their entirety by the cautionary statements above. Except as may be required under relevant laws in the United States, Biodexa does not undertake any obligation to publicly update or revise any forward-looking statements because of new information, future events or events otherwise arising.
FAQ
What are the latest survival results for Biodexa's (BDRX) MTX110 Phase 1 study in recurrent glioblastoma?
How does MTX110's performance in recurrent glioblastoma compare to typical survival rates?
What were the results of previous MTX110 studies in Diffuse Midline Glioma (DMG)?
How is MTX110 administered in the BDRX Phase 1 recurrent glioblastoma study?
