Biodexa Pharmaceuticals PLC (Nasdaq: BDRX) has announced the enrollment of the first patient in a Phase 2 clinical trial evaluating tolimidone for the treatment of Type 1 diabetes (T1D). The milestone marks a significant step forward for the clinical-stage biopharmaceutical company's efforts to address unmet medical needs in diabetes care.
Trial Design and Objectives
The Phase 2 study is being conducted as an Investigator Initiated Trial (IIT) by the University of Alberta Diabetes Institute. The trial will initially enroll 12 patients across three dose groups, with the potential for expansion based on initial results.
The primary focus of the study centers on measuring key biomarkers of diabetes management over a three-month period. Researchers will assess C-peptide levels, which serve as a marker for insulin production, and HbA1c levels, which indicate blood glucose control over time. The study will also track the frequency of hyperglycemic events, comparing all measurements to baseline values established at the start of treatment.
Preclinical Foundation
The decision to advance tolimidone into Phase 2 testing for Type 1 diabetes stems from compelling preclinical evidence generated at the University of Alberta. Research conducted at the institution identified Lyn kinase as a critical factor for beta cell survival and proliferation in both in vitro and in vivo experimental models.
Most notably, tolimidone demonstrated the ability to induce proliferation in beta cells isolated from human cadavers, a finding that suggests potential therapeutic relevance for patients with Type 1 diabetes who have lost beta cell function.
Mechanism of Action
Tolimidone functions as a selective activator of the enzyme Lyn kinase, which plays a crucial role in insulin signaling pathways. The drug increases phosphorylation of insulin substrate-1, thereby amplifying the signaling cascade that begins when insulin binds to its receptor. This mechanism positions tolimidone as a potential first-in-class blood glucose modulating agent.
Lyn kinase belongs to the Src family of protein tyrosine kinases and is primarily expressed in hematopoietic cells, neural tissues, liver, and adipose tissue. In animal models of diabetes, tolimidone has demonstrated glycemic control through insulin sensitization.
Drug Development History
Tolimidone has a unique development history, having been originally discovered and developed by Pfizer Inc. through Phase 2 clinical trials for the treatment of gastric ulcers. Pfizer conducted extensive preclinical characterization of the drug's pharmacology, pharmacokinetics, metabolism, and toxicology profile before discontinuing development due to lack of efficacy in the gastric ulcer indication.
Biodexa has since acquired the rights to tolimidone and repositioned it for diabetes treatment based on its mechanism of action and the preclinical evidence supporting its potential utility in Type 1 diabetes.
Company Pipeline Context
Tolimidone represents one of three lead development programs in Biodexa's pipeline. The company is also advancing eRapa, a proprietary oral formulation of rapamycin for Familial Adenomatous Polyposis and Non-Muscle Invasive Bladder Cancer, and MTX110, a solubilized formulation of panobinostat for aggressive rare brain cancer indications.
The Cardiff, UK-based company supports its drug development efforts through three proprietary drug delivery technologies designed to improve bio-delivery and bio-distribution of therapeutic agents.
