Biodexa Hits Key Milestone For Its Type 1 Diabetes Candidate Tolimidone, Enrolls First Patient In Phase 2a Study

Rhea-AI Summary

Biodexa Pharmaceuticals (NASDAQ:BDRX) has enrolled its first patient in a Phase 2a trial for Tolimidone, a potential treatment for type 1 diabetes. Originally discovered by Pfizer for gastric ulcers, Tolimidone is being repurposed as it activates the Lyn kinase enzyme, which could help the body produce insulin. The Phase 2a dose confirmation study, conducted with the University of Alberta, will measure C-peptide and HbA1c levels across 12 patients in three dose groups over three months. Preclinical studies at the University of Alberta showed promising results, including beta cell proliferation in human cadaver samples. The global type 1 diabetes treatment market, currently valued at $16.97 billion, is projected to reach $26.22 billion by 2032, growing at a 6.9% CAGR.

Positive

• Tolimidone showed promising preclinical results, including beta cell proliferation in human cadaver samples
• The drug could potentially become a first-in-class blood glucose modulating agent taken as a pill instead of insulin injections
• Large market opportunity with type 1 diabetes treatment market projected to reach $26.22 billion by 2032
• Drug already has extensive safety data from previous Pfizer trials for different indication

Negative

• Small initial trial size of only 12 patients
• Previous development by Pfizer was discontinued due to lack of efficacy for original indication
• Early stage development (Phase 2a) with no guarantee of success
• Faces competition in a well-established insulin therapy market

Insights

Biotechnology Research Analyst

Biodexa's Type 1 diabetes drug enters Phase 2a testing; promising mechanism but still early-stage with significant hurdles ahead.

Biodexa Pharmaceuticals has reached a significant but early milestone by enrolling the first patient in its Phase 2a trial for Tolimidone in Type 1 diabetes. This represents the initial clinical validation step for a drug candidate with an interesting scientific premise.

The mechanism here is noteworthy - Tolimidone selectively activates Lyn kinase, potentially stimulating beta cell survival and proliferation. What makes this particularly intriguing is that preclinical work at the University of Alberta demonstrated Tolimidone could induce proliferation in beta cells from human cadavers, suggesting potential regenerative capabilities.

This Phase 2a study structure reveals much about where we stand in development: only 12 initial patients across three dose groups indicates this is primarily a dose-finding study with early efficacy signals. The biomarkers being tracked - C-peptide (indicating insulin production) and HbA1c (showing glucose control) - are appropriate surrogate endpoints for this stage.

The drug development strategy here is clever - repurposing a compound previously developed by Pfizer for gastric ulcers reduces early development risks since safety data already exists. However, investors should recognize that Phase 2a represents an early step in clinical development, with Phase 2b, Phase 3, and regulatory review still ahead before potential commercialization.

The $16.97 billion global Type 1 diabetes market provides significant opportunity, but Tolimidone faces substantial hurdles to prove it can meaningfully impact beta cell function in actual patients. While the preclinical evidence is promising, the vast majority of compounds showing preclinical promise fail to demonstrate sufficient efficacy in human trials.

Endocrinology Specialist

Tolimidone's beta cell regeneration approach could revolutionize Type 1 diabetes treatment, but significant clinical proof still needed.

The entry of Biodexa's Tolimidone into Phase 2a testing represents a fascinating approach to Type 1 diabetes treatment. The current standard of care - lifelong insulin therapy via injections or pumps - addresses symptoms but not the underlying autoimmune destruction of beta cells. A therapy that could potentially regenerate these insulin-producing cells would fundamentally change treatment paradigms.

The scientific mechanism targets a specific biological pathway: Tolimidone activates Lyn kinase, increasing phosphorylation of insulin substrate-1, essentially amplifying insulin receptor signaling. More importantly, preclinical work suggests it may promote beta cell survival and proliferation - addressing the core pathology of Type 1 diabetes.

However, clinical realities warrant caution. The three-month measurement period in this small initial study is sufficient to detect early signals but not long-term efficacy or safety. The study will track C-peptide levels (indicating endogenous insulin production) and HbA1c (showing overall glucose control) - both critical metrics for diabetes intervention.

For context, 1.7 million American adults currently manage Type 1 diabetes with insulin therapy. A pill-based treatment that could reduce insulin dependence would significantly improve quality of life, but complete insulin independence would require substantial beta cell regeneration.

The truly transformative potential - if Tolimidone could stimulate meaningful beta cell regeneration in humans - remains theoretical until clinical data validates these effects. The Phase 2a results will provide crucial early indicators of whether this mechanism translates from laboratory to clinical settings.

CARDIFF, UNITED KINGDOM / ACCESS Newswire / June 18, 2025 / When it comes to type 1 diabetes, treating this chronic condition can be difficult. After all, with this type of diabetes, the body's immune system attacks and destroys the beta cells that produce insulin. Without the natural ability to produce insulin, glucose from the bloodstream isn't absorbed into cells to provide energy. Since there is no cure, patients with type 1 diabetes need lifelong insulin therapy, typically via injections or a pump. Although type 1 diabetes typically develops in childhood or adolescence, it can happen at any age and is more likely to occur in individuals who are obese or inactive, or both. 1.7 million adults aged 20 or older - or 5.7% of U.S. adults - have the disease and are receiving insulin via shots or a pump.

As it stands, the global market for treating type 1 diabetes is $16.97 billion and projected to reach $26.22 billion by 2032, growing at a CAGR of 6.9% from now until then.

Biodexa Believes It Has The Answer

That's why a handful of companies, including Biodexa Pharmaceuticals PLC (NASDAQ:BDRX), a clinical stage biopharmaceutical company developing treatments for unmet medical needs, are working on next-generation drugs and therapies to treat this insidious disease. Biodexa is developing Tolimidone to treat type 1 diabetes and has recently enrolled its first patient in a phase 2a trial.

Tolimidone was first discovered by Pfizer Inc. and was developed to treat gastric ulcers. The drug made it through phase 2 trials, but Pfizer discontinued developing the drug because of a lack of efficacy for gastric ulcers. But for type 1 diabetes, it holds promise given Tolimidone is a selective activator of the enzyme Lyn kinase, which increases phosphorylation of insulin substrate-1, thereby amplifying the signaling cascade initiated by the binding of insulin to its receptor. In layman's terms, it can potentially help the body produce insulin. 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.

The use of Tolimidone in type 1 diabetes has been demonstrated in a number of preclinical studies conducted at the University of Alberta. The studies identified Lyn kinase as a key factor for beta cell survival and proliferation in in vitro and in vivo models, reports Biodexa. Very promising, noted the company, is the fact that Tolimidone was able to induce proliferation in beta cells isolated from human cadavers. Biodexa believes the drug has the potential to become a first-in-class blood glucose modulating agent. That would be welcome news to type 1 diabetes patients who are forced to take insulin shots or use an insulin pump. Taking just a pill to manage their disease could prove game changing.

Phase 2a Trial Gets Its First Patient

Currently, Biodexa is working with the University of Alberta on a phase 2a dose confirmation study, and the enrollment of the first person in that study is a big milestone for Biodexa. The dosing study was already approved by Health Canada and will be conducted by the University of Alberta, which 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 initially in 12 patients across three dose groups. The study may be expanded.

"We are excited to initiate our clinical program in type 1 diabetes with the University of Alberta and build on the extensive tolimidone data package put together by Pfizer, Melior and Bukwang," said Stephen Stamp, CEO and CFO of Biodexa, when the study was first announced. The study is designed to build on the preclinical data that Biodexa said suggested Tolimidone could have a proliferative impact on pancreatic beta cells, the cells responsible for insulin production.

Type 1 diabetes may not be the most common form of diabetes, but it can be among the most life-altering, requiring patients to rely on insulin for the rest of their lives. Biodexa is trying to make that easier for sufferers, taking a drug already developed and putting it to use in another form, one that the company believes holds a lot of potential.

With the first patient enrolled in its phase 2a trial, Biodexa is much closer to bringing relief to type 1 diabetes patients. Stay tuned to hear more about Tolimidone and Biodexa's progress in fighting a disease that impacts millions of people in the U.S. and abroad.

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SOURCE: Biodexa Pharmaceuticals

View the original press release on ACCESS Newswire

FAQ

What is Biodexa's (BDRX) Tolimidone drug candidate for type 1 diabetes?

Tolimidone is a selective activator of Lyn kinase enzyme that could help the body produce insulin, potentially offering an oral alternative to insulin injections for type 1 diabetes patients.

What phase is Biodexa's (BDRX) Tolimidone currently in?

Tolimidone has just enrolled its first patient in a Phase 2a dose confirmation study, which will evaluate the drug across 12 patients in three dose groups over three months.

How does Tolimidone work for type 1 diabetes treatment?

Tolimidone activates the Lyn kinase enzyme, which increases phosphorylation of insulin substrate-1, amplifying the insulin receptor signaling cascade and potentially helping the body produce insulin.

What are the market prospects for Biodexa's (BDRX) type 1 diabetes treatment?

The global type 1 diabetes treatment market is currently $16.97 billion and is expected to reach $26.22 billion by 2032, growing at a 6.9% CAGR.

What results has Tolimidone shown in preclinical studies?

Preclinical studies at the University of Alberta demonstrated that Tolimidone could induce proliferation in beta cells isolated from human cadavers and showed promise for beta cell survival.