A phase 2 clinical trial demonstrated that a 2-week course of teplizumab was able to delay the onset of clinical type 1 diabetes (T1D) by 3 years compared with patients taking a placebo, according to data presented at the American Diabetes Association's 80th Scientific Sessions.

T1D, a chronic autoimmune disease that affects nearly 1.6 million Americans, causes the destruction of insulin-producing beta cells and dependence on exogenous insulin for survival. Some past treatments have successfully delayed the loss of insulin production in patients with T1D, but further interventions were still needed in order to affect clinical progression before diagnosis.

In 2019, teplizumab was granted Breakthrough Therapy Designation by the FDA and PRIority MEdicines (PRIME) designation by the European Medicines Agency for the prevention or delay of T1D in individuals at-risk of developing the disease.

The results from this recent study showed that patients treated with teplizumab had higher rates of insulin secretion and C-peptide levels than those treated with the placebo. This was able to demonstrate a change in the normal progression of T1D, according to the study authors.

Additionally, participants who took the placebo demonstrated no change since the following year’s reporting in the median time to diagnosis of T1D during the follow-up period. These patients also maintained a decline in insulin and C-peptide production, which is consistent with the beta-cell destruction that is caused by T1D.

All of the participants in the trial were relatives of patients with T1D who were at high risk for its development based on the presence of 2 or more autoantibodies that were detectable through a screening test.

This study’s results add another year to the average time reported to reach insulin dependence in participants treated with teplizumab compared with participants treated with the placebo, according to the study authors.

"We are excited and hopeful about what the newly released trial results mean for the type 1 diabetes community. This is the first disease-modifying drug with data showing a long-term delay to insulin dependence," said Aaron J. Kowalski, PhD, president and CEO of Juvenile Diabetes Research Foundation, in a press release. "This is a major milestone in the global effort to comprehensively understand T1D and support the advancement towards prevention and cures for this disease."

New findings from groundbreaking study shows extended delay in onset of type 1 diabetes [news release]. New York, NY: Juvenile Diabetes Research Foundation; June 15, 2020. eurekalert.org/pub_releases/2020-06/j-nff061520.php. Accessed June 19, 2020.