AMX0035 for Type 1 Diabetes

(MetMod-T1D Trial)

This trial tests a new oral treatment called AMX0035 for individuals with Type 1 Diabetes. Researchers aim to assess how AMX0035, taken for about 6 months, affects diabetes management compared to a placebo (a treatment with no active drug). Participants will receive either AMX0035 or a placebo to evaluate its impact. Those who have managed Type 1 Diabetes for over a year and currently use insulin might be suitable candidates for this study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Previous studies have generally shown that patients tolerate AMX0035 well. Most experienced only mild to moderate side effects, with diarrhea being the most common. Importantly, no serious side effects led to the study's termination. Some studies also found that AMX0035 might increase bile acid levels and cause fluid retention, but these effects were not serious. Overall, the safety data suggests that AMX0035 is safe for humans, with manageable side effects.¹²³⁴⁵

Unlike the standard treatments for Type 1 Diabetes, which typically involve insulin therapy, AMX0035 targets cellular health differently. AMX0035 is a combination of two compounds, sodium phenylbutyrate and tauroursodeoxycholic acid, which work together to protect nerve cells from damage. Researchers are excited about this treatment because it offers a novel approach by potentially reducing inflammation and oxidative stress in pancreatic cells, which could preserve insulin production. This mechanism is different from the usual focus on managing blood sugar levels and may offer a new avenue for improving long-term outcomes for Type 1 Diabetes patients.

Research has shown that AMX0035 positively affects conditions like Amyotrophic Lateral Sclerosis (ALS). In ALS, AMX0035 slowed the disease's progression. Another study found that AMX0035 stabilized or improved symptoms in people with Wolfram Syndrome, which can lead to diabetes. This treatment protects cells from dying, particularly in the brain and pancreas. Although direct evidence for Type 1 Diabetes is limited, these findings offer hope that AMX0035, which participants in this trial may receive, might help manage the disease by protecting important cells.⁵⁶⁷⁸⁹