On September 12, 2025 Biodexa Pharmaceuticals PLC (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, reported its unaudited interim results for the six months ended June 30, 2025 which will also be made available on the Company’s website at www.biodexapharma.com (Press release, Biodexa Pharmaceuticals, SEP 12, 2025, View Source [SID1234655947]).
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OPERATIONAL HIGHLIGHTS
The Company announced the following in the six months ended June 30, 2025:
· Allowance by the US Patent and Trademark Office of patent application No. 17/391-495 "Oral Rapamycin Nanoparticle Preparations and Use", exclusively licensed to the Company by Emtora Biosciences .
· Appointment of Precision for Medicine, LLC as the clinical research organization to conduct the European component of the upcoming registrational Phase 3 study of eRapa in FAP.
· A successful Type C meeting with the US Food and Drug Administration regarding the protocol for the Company’s registrational Phase 3 study of eRapa in FAP.
· Orphan Drug Designation granted for eRapa in FAP by the European Commission.
· Recruitment of the first patient in a Phase 2a study of tolimidone in Type 1 Diabetes in an Investigator Initiated Trial conducted by the University of Alberta Diabetes Institute.
· Selection of ‘Serenta’ as the brand name for its Phase 3 clinical study of eRapa in FAP together with launch of a dedicated website, www.serentatrial.com, to provide information and resources for patients, caregivers, and healthcare professionals.
· Activation of the first clinical study site in the US for its Serenta trial in patients with FAP.
Post period end:
· Filing of a Clinical Trial Application with the European Medicines Agency for its Serenta trial in patients with FAP.
· Enrolment of first patients in the Serenta trial by the Pan American Center for Oncology Trials in San Juan, Puerto Rico.
FINANCIAL HIGHLIGHTS
· Signing of a $35 million Equity Line of Credit with C/M Capital Master Fund LP, or C/M, pursuant to which the Company has the right, but not the obligation, to sell to C/M, and C/M is obligated to purchase newly issued ADSs for a period of 36 months.
· The Company’s collaboration partner, Emtora Biosciences, was awarded an additional grant of $3.0 million from the Cancer Prevention & Research Institute of Texas, bringing the total of non-dilutive grant funding to $20.0 million in support of the registrational Phase 3 program of eRapa in FAP.
· R&D costs decreased to £1.67 million in 1H25 (1H24: £2.19 million) reflecting a reduction in spend on the MAGIC-G1 study in recurrent glioblastoma and pre-clinical studies offset by an increase in expenditure (net of CPRIT grant income) on MTX230 (eRapa).
· Administrative costs increased to £2.38 million (1H24: £2.03 million) as a result of a foreign exchange charge offset by a reduction in transaction related costs.
· Net cash used in operating activities (after changes in working capital) in 1H25 was £3.30 million (1H24: £4.81 million).
· The Company’s cash balance at June 30, 2025 was £4.04 million.
Commenting, Stephen Stamp, CEO and CFO, said "The first half was extremely productive. Having secured Fast Track designation and successfully negotiated our way through a Type C meeting with the FDA, in August we enrolled the first patients into our pivotal Serenta trial of eRapa in FAP. In parallel, we secured orphan designation from the European Commission for eRapa in FAP in Europe and filed a Clinical Trial Application with the EMA, which sets us up to begin enrolment in the Serenta trial in Europe in the fourth quarter".
CHIEF EXECUTIVE’S REVIEW
Our main focus in the first half of 2025 was on preparatory activities for the start of our registrational Phase 3 trial of eRapa in Familial Adenomatous Polyposis ("FAP").
R&D update
In the first half of 2025 we materially advanced our R&D pipeline, moving eRapa in FAP into Phase 3 and tolimidone for Type 1 Diabetes ("T1D) into Phase 2:
eRapa
eRapa is a proprietary oral 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 tumorgenesis. Rapamycin is approved in the US for organ rejection in renal transplantation as Rapamune(Pfizer). Through the use of nanotechnology and pH sensitive polymers, eRapa is designed to address the poor bioavailability, variable pharmacokinetics and toxicity generally associated with the currently available forms of rapamycin. eRapa is protected by a number of issued patents which extend through 2035, with other pending applications potentially providing further protection beyond 2035.
Familial Adenomatous Polyposis ("FAP")
FAP is an orphan indication characterized by a proliferation of polyps in the colon and/or rectum, usually occurring in mid-teens. There is no approved therapeutic option for treating FAP patients, for whom active surveillance and surgical resection of the colon and/or rectum remain the standard of care. If untreated, FAP almost always leads to cancer of the colon and/or rectum. There is a significant hereditary component to FAP with a reported incidence of one in 5,000 to 10,000 in the US and one in 11,300 to 37,600 in Europe. eRapa has received Orphan Designation in the US and in Europe. Importantly, mTOR has been shown to be over-expressed in FAP polyps – thereby underscoring the rationale for using a potent and safe mTOR inhibitor like eRapa to treat FAP.
An open-label Phase 2 study (NCT04230499) was conducted by Emtora in seven U.S. centres of excellence in 30 adult patients. Patients were sequentially enrolled into three dosing cohorts of 10 patients each for a 12-month treatment period: 0.5mg every other day (Cohort 1), 0.5mg daily every other week (Cohort 2), and 0.5mg daily (Cohort 3). Upper and lower endoscopic surveillance occurred at baseline and after six months. Primary endpoints were safety and tolerability of eRapa and percentage change from baseline in polyp burden, as measured by the aggregate of all polyp diameters.
In May 2024 and June 2024, results of the Phase 2 study at six months and 12 months, respectively, were presented at prestigious scientific meetings by Carol Burke, MD, the Principal Investigator. In summary, at six months, eRapa appeared safe and well-tolerated with a significant 24% reduction in the total polyp burden at six months compared with baseline and an overall 83% non-progression rate. At 12 months, 21 of 28 (75%) patients were deemed to be non-progressors with a median reduction in polyp burden of 17%. In Cohort 2, the dosage regimen for Phase 3, eight of nine (89)% of patients were deemed non-progressors at 12 months with a median reduction in polyp burden of 29%. Over the course of 12 months, there were four related Grade 3 or higher and one related Serious Adverse Event reported during the trial and 95% compliance rate at 12 months. One patient was removed from the trial due to non-compliance.
The Phase 3 registrational study (NCT06950385) is a double-blind placebo-controlled design, recruiting 168 high risk patients diagnosed with germline or phenotypic FAP. The primary clinical endpoint is first progression free survival event which will comprise composite endpoints including major surgery. We had a successful ‘Type C’ meeting with the FDA in January 2025 to finalise the protocol. The first clinical site was initiated in June 2025 and the first patients enrolled by the Pan American Center for Oncology Trials in San Juan, Puerto Rico in August 2025. Europe is following closely behind; our contract research organisation, Precision for Medicine, was appointed in March 2025. Orphan Drug Designation for eRapa in FAP was granted by the European Commission in May 2025. A Clinical Trial Application was filed with the European Medicines Agency for the Serenta trial in July 2025 which, if approved, will facilitate the start of patient recruitment in Europe in 4Q25.
Non-muscle Invasive Bladder Cancer ("NMIBC")
NMIBC refers to tumors found in the tissue that lines the inner surface of the bladder. The most common treatment is transurethral resection of the bladder tumor followed by intravesical Bacillus Calmette-Guerin ("BCG") with chemotherapy depending upon assessment of risk of recurrence. NMIBC is the fourth most common cancer in men with an incidence of 10.1 per 100,000 and 2.5 per 100,000 in women.
The ongoing multi-centre, double-blind, placebo-controlled Phase 2 study in NMIBC (NCT04375813) is fully enrolled at 166 patients with primary endpoints of safety/tolerability and relapse free survival after 12 months of treatment. The study, which is supported by a $3.0 million non-dilutive grant from the National Cancer Institute, part of the National Institutes of Health, was transferred to the University of Texas, San Antonio as an Investigator Initiated Trial and is expected to read out in mid-2026.
MTD228 – Tolimidone
Tolimidone was originally discovered by Pfizer and was developed through Phase 2 for the treatment of gastric ulcers. Pfizer undertook a broad pre-clinical program to characterize the pharmacology, pharmacokinetics, metabolism and toxicology of tolimidone. Pfizer discontinued development of the drug due to lack of efficacy for that indication.
Tolimidone is a selective activator of the enzyme Lyn kinase which increases phosphorylation of insulin substrate-1, thereby amplifying the signalling cascade initiated by the binding of insulin to its receptor.
Type 1 Diabetes ("T1D")
Tolimidone’s potential utility in T1D has been demonstrated by several preclinical studies conducted by the University of Alberta, where Lyn kinase was identified as a key factor for beta cell survival and proliferation in in vitro and in vivo models. Most importantly, tolimidone appeared to induce proliferation in beta cells isolated from human cadavers. From a mechanism of action perspective, tolimidone has been shown to both prevent beta cell degradation and to stimulate beta cell proliferation. In a meta analysis of 1,202 articles and 193 studies, the incidence of T1D was shown to be 15 per 100,000 with a prevalence of 9.5 per 10,000 of the population.
As a first step in the continued clinical development of tolimidone, a Phase 2a Investigator Initiated Trial (IIT) at the University of Alberta Diabetes Institute (NCT06474598) is designed to establish the minimum effective dose of tolimidone in patients with T1D. The study, enrolled the first patient in June 2025 and is expected to recruit 12 patients initially across three dose groups. The study will measure C-peptide levels (a marker for insulin) and HbA1c (a marker for blood glucose) after three months compared with baseline and the number of hyperglycemic events.
MTX110
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 brain tumor, by-passing the blood-brain barrier and potentially avoiding systemic toxicity. All three types of brain cancer being studied are orphan.
Recurrent Glioblastoma ("rGBM")
Our Phase 1 MAGIC-G1 study (NCT05324501) of MTX110 in rGBM has completed the dose escalation part of the study with the recruitment of the fourth patient in Cohort A. Overall survival was reported as between 11 and 12 months. Glioblastoma virtually always recurs with median Progression Free Survival of 1.5–6.0 months and median Overall Survival of 2.0–9.0 months.
Diffuse Midline Glioma ("DMG")
In February 2024 we announced headline data from a Phase 1 IIT study conducted by Columbia University in newly diagnosed patients with DMG. As this was the first ever study of repeated infusions to the pons via an implanted CED catheter, the primary objective of the study was safety and tolerability and, accordingly, the number of infusions was limited to two, each of 48 hours, seven days apart in nine patients. One patient suffered a severe adverse event assessed by the investigators as not related to the study drug. Although not powered to reliably demonstrate efficacy, median Overall Survival of patients in the study was 16.5 months compared with median survival rate in a cohort of 316 cases of 10.0 months.
Study investigators subsequently presented the results of the trial at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) in Philadelphia, PA.
Medulloblastoma
An IIT Phase I study of MTX110 in medulloblastoma remains ongoing at the University of Texas.
Due to resource constraints, MTX110 has been de-prioritised and there are no current development activities being undertaken.
Financing
Equity Line of Credit
In January 2025, we entered into a securities purchase agreement, or equity line of credit ("ELOC"), with the newly formed C/M. Under the terms of the ELOC, we have the right, but not the obligation, to sell to C/M from time to time up to $35 million of newly issued ADSs over a 36-month period, unless the ELOC is terminated. As consideration for the execution and delivery of the ELOC, we agreed to pay a commitment fee ("Commitment Fee") of $875,000 in cash, of which (i) $612,500 was to be paid to C/M on signing the ELOC and (ii) the balance was to be paid pro rata, simultaneously with the delivery of any ADSs sold under the ELOC. We had the right to issue ADSs representing the value of the applicable portion of the Commitment Fee. We paid the initial Commitment Fee of $612,500 through the issuance of 140,080 Depositary Shares to the Purchaser.
We may direct C/M to purchase a specified number of ADSs not to exceed $2.5 million on any given day, at a price based on a formula, typically 95% of the closing price on the prior day. As of June 30, 2025, the Company had raised gross proceeds of $8.56 million from the ELOC.
Warrant Inducement
In May 2025 we entered into letter agreements with certain holders of outstanding Series E, Series H, Series J and Series K warrants to reduce the exercise price of such warrants to $0.31 per share. The holders exercised an aggregate of 200,433 warrants representing the same number of ADSs. We received gross proceeds of approximately $62,000, before offering expenses. The Company did not issue new warrants to replace the exercised warrants and did not engage a placement agent to facilitate the transaction.