These researchers are testing Tolimidone (also known as MTX228), which has a long history. It was developed by Pfizer as a treatment for gastric ulcers, but it failed to show enough benefit in a phase-II clinical trial. Years later, Melior Pharmaceuticals repurposed Tolimidone for type 2 diabetes, and ran a phase II clinical trial for that. However, the company has not issued press releases since 2021, and there has been no new news on Tolimidone for T2D since 2019.
Later researchers found the enzyme which Tolimidone activates, called Lyn kinase, helps determine whether beta cells survive or die. In their experiments, turning Lyn kinase off caused beta cells to die and led to diabetes in mice, while activating it protected beta cells and encouraged them to multiply (including human beta cells). Those findings led to this study.
Tolimidone is administered as a capsule or tablet.
The Study
This is an open-label study where everyone gets the treatment (no placebo group). There is no blinding. The different groups get different doses: 100 milligrams once a day, 100 milligrams twice a day, or 200 milligrams once a day, for three months. Adults between 18 and 65 who have had type 1 diabetes for at least a year can join.
The trial's main measurement is the change in C-peptide over 3 months, which reflects how much insulin the body is producing on its own. Other measurements collected through the trial's optional longer follow-up include changes in daily insulin dose, HbA1c, fasting blood sugar, the amount of time spent in a healthy blood sugar range as tracked by a wearable glucose sensor, and the number of low blood sugar episodes, and more C-peptide data.
Recruitment began in mid-2025. They hope for main results by October 2026 and publication by end of 2027.
The trial is currently taking place at a single site: University of Alberta, Edmonton, Alberta, Canada.
People interested in learning more about participating can contact Dominique Forrest at the University of Alberta by phone at 780-248-1770 or by email at dforres1@ualberta.ca.
Discussion
There is an obvious contradiction in how this study is structured. The drug is expected to work by saving beta cells, by preventing them from dying. However, the study is recruiting people who have had type-1 for more than a year, so are not in their honeymoon, and (presumably) have few to no beta cells left. You would think a study aimed at a drug to save beta cells would recruit people who still had beta cells, or more of them. I can't explain this.
I consider this a "phase-IIΔ" study, rather than a regular "Phase-II" study, because there has never been a phase-I study in type 1 diabetes. There has been for other diseases, so this is like a phase-I study with respect to effectiveness, but a phase-II study with respect to safety. The size (24 people) is closer to phase-I than phase-II.
The researchers are testing multiple doses in this clinical trial, so that they can run a follow on trial (probably another phase-II study) using the dose which shows the best results here.
More Information
- ClinicalTrials.gov listing: https://clinicaltrials.gov/study/NCT06474598
- Biodexa Pharmaceuticals: https://biodexapharma.com/
- Press release announcing the first patient enrolled: https://biodexapharma.com/biodexa-announces-recruitment-of-first-patient-in-phase-2-study-of-tolimidone-in-type-1-diabetes/
- Press release announcing Health Canada's approval of the study: https://biodexapharma.com/biodexa-announces-phase-2a-study-of-tolimidone-in-type-1-diabetes-approved-by-health-canada/
- Wikipedia page for Tolimidone: https://en.wikipedia.org/wiki/Tolimidone
- BioSpace coverage of the trial's first patient enrollment: https://www.biospace.com/press-releases/biodexa-hits-key-milestone-for-its-type-1-diabetes-candidate-tolimidone-enrolls-first-patient-in-phase-2a-study
- University of Alberta feature on the preclinical research behind the trial: https://www.ualberta.ca/en/folio/2023/11/could-a-pill-cure-diabetes.html
- Biodexa's interim financial results discussing the trial's progress: https://biodexapharma.com/interim-results-for-the-six-months-ended-june-30-2025/
