Full Press Release Details
Biodexa Pharmaceuticals
or the "Company" or, together with its subsidiaries, the "Group")
for the Year Ended 31 December 2022
Biodexa Pharmaceuticals
PLC (Nasdaq: BDRX), a clinical-stage biopharmaceutical company developing a pipeline of products aimed at primary and metastatic cancers
of the brain, announces its audited preliminary results for the year ended 31 December 2022.
For more information,
Biodexa Pharmaceuticals
Stephen Stamp, CEO, CFO
Tel: +44 (0)29 2048 0180
(US Investor Relations)
Email: afactor@edisongroup.com
About Biodexa Pharmaceuticals
Biodexa Pharmaceuticals
PLC (listed on NASDAQ: BDRX) is a clinical stage biopharmaceutical company developing a pipeline of products aimed at primary and metastatic
cancers of the brain. The Company's lead candidate, MTX110, is being studied in aggressive rare/orphan brain cancer indications
including recurrent glioblastoma and diffuse midline glioma.
formulation of the histone deacetylase (HDAC) inhibitor, panobinostat. This proprietary formulation enables delivery of the product
via convection-enhanced delivery (CED) at potentially chemotherapeutic doses directly to the site of the tumour, by-passing the blood-brain
barrier and avoiding systemic toxicity.
headquarters and R&D facility is in Cardiff, UK. For more information, please visit www.biodexapharma.com
in this announcement may constitute "forward-looking statements" within the meaning of legislation in the United Kingdom and/or
United States Private Securities Litigation Reform Act. All statements contained in this announcement that do not relate to matters of
historical fact should be considered forward-looking statements.
be made to those documents that Biodexa shall file from time to time or announcements that may be made by Biodexa in accordance with regulations
promulgated by the US Securities and Exchange Commission, which contains and identifies 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 the relevant law 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 otherwise arising.
Cardiff, UK, and quoted on NASDAQ in the US, Biodexa is a clinical-stage biotechnology company with three enabling drug delivery technologies.
The Company de-listed from the AIM market as of 26 April 2023.
Review, and throughout 2022, we pursued a strategy of broadening our R&D pipeline by initiating internal programmes, collaborating
with third party pharmaceutical companies on their proprietary active pharmaceutical ingredients, or APIs, and adding new indications
to MTX110. New internal programmes were selected and prioritised based on the expected time to deliver proof-of-concept data for potential
a second R&D collaboration with Janssen Pharmaceutica NV, for reasons not always within our control, we were not successful in finding
partners for our internal Q-Sphera pipeline. As our cash runway ran down, and the market for biotech financing worsened in 2022, we considered
the opportunities for refinancing the Company as a drug delivery company were limited. Accordingly, we concluded that repositioning the
Company as a therapeutics company, focused on rare and orphan products in a merger with Bioasis Technologies, Inc. (Bioasis) with an attendant
$10.0 million financing was the optimal solution for the Company. Although the merger and financing did not proceed, we were successful
in raising $6.0 million, repositioning the Company as a clinical-stage therapeutics company supported by enabling technologies. In line
with that repositioning, we undertook a restructuring in March 2023 including a cost reduction programme and termination of our internal
Q-Sphera programmes. These initiatives are described more fully in the Chief Executive's Review.
Following the repositioning
of the Company, our priorities for 2023 reflect our modified strategy as follows:
| Strategic Imperatives | Progress in 2022 | Priorities for 2023 |
| Advance our clinical -stage assets through to proof-of-concept data | We announced the recruitment of our first patient in the first cohort of a Phase I study of MTX110 in recurrent GBM (rGBM) in two clinical centres in the US. | In respect of our Phase I study in rGBM, deliver interim safety and efficacy data (in the form of Progression Free Survival data) in respect of Cohort A patients and recruit Cohort B patients. Finalise recruitment of our second Phase I study in DIPG and report safety data. Accelerate recruitment of our Phase I study in medulloblastoma. |
| Develop and broaden applications for our three primary drug delivery technologies | Having discovered we could encapsulate and retain the functional integrity of a monoclonal antibody, we explored additional potential applications for our Q-Sphera technology including antibody drug conjugates (ADCs) and BiTes. We completed our assignment under our first R&D collaboration with Janssen, including optimising drug loading of Janssen's proprietary large molecule using our Q-Sphera technology. | Generate in vivo data to demonstrate intratumoral delivery of drugs using our proprietary technology. Explore opportunities for MTX110 in combination therapy for brain and metastatic CNS cancer. Expand further our patent portfolio to cover new inventions and divisionals to strengthen existing patent families. |
| Enter into R&D collaborations at the feasibility stage and/or licenses at proof-of-concept stage with third parties. | In January 2022 and again in March 2022, we announced two R&D collaborations with Janssen. We have been tasked with maximizing drug loading and optimizing in vitro duration of release for two of Janssen's experimental large molecules using our Q-Sphera technology. | Enter into R&D collaborations with third parties to formulate their proprietary molecules using our technology platforms. Seek a partner to develop, or co-develop, MTX110 once preliminary data from our Phase I study in recurrent GBM become available. |
| Seek opportunities to diversify our pre-clinical and clinical-stage pipeline | We explored additional indications for MTX110 during the year although the decision to reposition the Company into a therapeutics (as opposed to drug delivery) company was actioned in early 2023. We added a new research programme coded MTD217 focused on developing new therapeutics options for metastatic cancers including leptomeningeal disease. | Identify and acquire at least one development asset to in-license, ideally in oncology and for a rare/orphan indication. Initiate additional preclinical studies to assess the potential for MTD217 inhibitors in leptomeningeal disease. |
| Provide a healthy and stimulating environment in which our staff members can continue to thrive and innovate | We have been compliant with ISO 9001 since 2014. During the year, we introduced a new COSHH assessment procedure to better quantify the safety of chemicals and third parties' APIs being deployed in our laboratories. | Continue to monitor third party advice and regulation to maintain a safe environment for our staff members. Develop individualised learning programmes for staff members through participation in conferences, webinars and/or training programmes. |
Following our Strategic
Review in March 2020, we reverted to a traditional biotech business model. We aimed to deploy our proprietary technologies to develop
proof-of-concept formulations and then enter into licensing agreements with third party pharmaceutical companies.
the Company more investable and secure additional financing, the Board decided to reposition the Company as a therapeutics (as opposed
to drug delivery) company in early 2023. As a result, the delivery of proof-of-concept clinical data is the primary focus of our business
model going forward.
to build a balanced portfolio of clinical-stage development assets, ideally focused on oncology and on rare/orphan indications. Our only
current clinical-stage asset, MTX110 is in Phase I development for three rare/orphan brain cancers.
may, like MTX110, be based on one or more of our enabling technologies.
into R&D collaborations with third parties to develop proof-of-concept formulations of their proprietary compounds using our proprietary
drug delivery technologies. We will not be expanding our internal pipeline of drug delivery based programmes.
To establish proof-of-concept
in pre-clinical studies for potential licensees, we are able to manufacture non-GMP Q-Sphera products at pilot scale at our Cardiff facility.
Upon securing a license partner who wishes to start clinical studies, our intention would be to technology transfer GMP manufacture of
clinical trial supplies and ultimately full GMP commercial manufacture to a third party CMO. We would expect a licensee to assume the
cost of manufacturing GMP product and commercial scale-up pursuant to a technology transfer agreement.
being manufactured to GMP standards at a CMO.
Once proof-of-concept
has been established, we intend to seek to license our products to a partner who would complete the clinical development and subsequently
market and sell them in the licensed territory. In addition to reimbursement of development costs, the partner would be expected to make
milestone payments based on sales targets and royalty payments.
pipeline includes six projects of which one is partnered with Janssen:
technology in combination with panobinostat, an otherwise insoluble drug, MTX110 is designed for direct-to-tumour treatment of intractable
brain cancers. Panobinostat is currently marketed under the brand Farydak which is used orally in combination therapy for the treatment
of multiple myeloma. We are currently researching the utility of MTX110 to proof-of-concept stage in three indications:
common and aggressive form of brain cancer in adults, usually occurring in the white matter of the cerebrum. Treatments include radiation,
surgical resection and chemotherapy, although in almost all cases tumours recur. There are approximately 2-3/100,000(1) population
diagnoses of GBM per annum. Survival with standard of care treatment ranges from approximately 13 months in unmethylated MGMT patients
to approximately 30 months in highly methylated MGMT patients(2).
Following IND approval
in December 2021, we are in the process of recruiting patients in a Phase I study to assess the utility of MTX110 in recurrent GBM. 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
Pontine Glioma (DIPG):
located in the pons (middle) of the brain stem and are diffusely infiltrating. Occurring mostly in children, approximately 1,000 patients(3)
worldwide are diagnosed with DIPG per annum and median survival is approximately 10 months(4). There is no effective treatment
since surgical resection is not possible. The standard of care is radiotherapy, which transiently improves symptoms and survival. Chemotherapy
does not improve survival and one likely reason is that many anti-cancer drugs cannot cross the blood-brain barrier to access the tumour.
we reported the first-in-human study by the University of California, San Francisco ("UCSF") of MTX110 in DIPG using a convection
enhanced delivery ("CED") system. The Phase I study established a recommended dose range for Phase II, a good safety and tolerability
profile but also encouraging survival data in the seven patients treated.
are malignant embryonal tumours that start in the cerebellum. They are invasive and, unlike most brain tumours, spread through the cerebrospinal
fluid ("CSF") and frequently metastasize to different locations in the brain and spinal cord. Treatments include
resection, radiation and chemotherapy. Approximately 350 patients(5) are diagnosed with medulloblastoma per annum and 3,800
people are living with the disease in the US. The cumulative survival rate is approximately 60%, 52%, and 47% at 5 years, 10 years, and
20 years, respectively(6); however, recurrence is nearly always fatal with no established standard of care.
of Texas is undertaking a Phase I exploratory study in recurrent medulloblastoma patients using direct administration of MTX110 into the
fourth ventricle, enabling it to circulate throughout the CSF.
Our Q-Sphera technology
employs 3-D printing techniques to encapsulate medicines in polymer-based bioresorbable microspheres. The microspheres may be injected
to form depots in the body which release drug over predictable, sustained periods from one week up to several months. The features and