BHT-3021 - Program Overview


About Type 1 Diabetes (T1D)

T1D, also known as juvenile diabetes, is an autoimmune disease that results from the immune system attacking and destroying islet cells (insulin-producing cells in the pancreas), and proinsulin, which is the prohormone precursor to insulin widely thought of as the most important self-antigen related to the disease.

Approximately 80% of patients diagnosed with T1D have detectable antibodies to islet cell self-proteins including insulin IA-2, a major autoantigen in T1D, and glutamic acid decarboxylase, a target of autoantibodies in people who later become insulin dependent. The islets in the pancreas display insulitis, which is characterized by infiltration of T cells, B cells and macrophages into the islet of Langerhans (the group of hormone-producing cells in the pancreas).

Control of blood glucose is critical to both the long- and short-term health of T1D patients. Short-term effects of poor glucose control include diabetic ketoacidosis, which is a condition that can lead to coma. Long-term effects include elevated glucose levels, blindness, kidney disease, heart disease, peripheral vascular disease and stroke. In order to keep blood glucose at or near normal levels, T1D patients require the life-long administration of self-injectable insulin, which is the only approved therapy for T1D.

According to the Juvenile Diabetes Research Foundation International, as many as three million people in the United States may have T1D, with over 15,000 children newly diagnosed each year. Although primarily diagnosed in children, the incidence of disease among adults, classified as Latent Autoimmune Diabetes of Adult (LADA), is becoming more common. Although difficult to quantify, the costs of managing the numerous long-term complications of T1D are substantial.


Current Treatments for T1D
There are currently no treatments for diabetes, and insulin is the only current option to replace the effects of a non-functioning pancreas.


About BHT-3021
BHT-3021 is a plasmid encoding proinsulin designed to tolerize the immune system to proinsulin, thereby turning off the self directed immune attack. This product candidate’s potential to improve glucose control could reduce or eliminate insulin dependence and long-term complications of T1D, which would address a major unmet need and capitalize on a significant commercial market opportunity.

Patient enrollment is currently underway in a Phase I/II placebo-controlled clinical trial of BHT-3021 to evaluate safety and pharmacodynamics, immune tolerance and pancreatic function in patients with T1D.  Preliminary data from the trial have been positive and the announcement of top-line results is expected in the first half of 2009.


Clinical trial