Enrollment complete in Diamyd Phase II study for type 1 diabetes

A total of 50 children aged four and older with a high risk of developing type 1 diabetes have been enrolled in a researcher-initiated Phase II study, DiAPREV-IT, with Diamyd Medical's diabetes vaccine Diamyd®. The study is thus fully recruited. The purpose of the study is to evaluate whether preventive treatment with Diamyd® can delay or halt the progression of the disease so that the children do not develop clinical symptoms of type 1 diabetes.

The children enrolled in the study are healthy, but have all been found to have an ongoing autoimmune process, where their blood sugar-regulating beta cells in the pancreas are being destroyed by their own immune system. If the destruction is not stopped, it can with time lead to type 1 diabetes. Preventive treatment with Diamyd® is intended to intervene in the autoimmune process at an early stage, and thus prevent the disease from developing. Today there is no treatment that can halt the progress of disease in type 1 diabetes.

"It is very exciting to follow the children in this prevention study, which is the first of its kind," says Peter Zerhouni, President and CEO of Diamyd Medical. "This study provides valuable information that can form the basis for larger prevention studies. If it turns out that preventive treatment with our diabetes vaccine reduces the risk of developing type 1 diabetes in these children it would be a great step forward in diabetes research."

The study is being conducted by a research group at Lund University and is led by Dr. Helena Elding Larsson, a pediatrician in Malmö and researcher at Lund University. The study is funded by research grants, but Diamyd Medical has participated in the design of the study and can utilize the study results.

"I think there is a good chance that the diabetes vaccine can have effect in the preventive treatment of children who have not yet been diagnosed with type 1 diabetes," says Helena Elding Larsson. "In DiAPREV-IT, we vaccinate the children early in the disease process when they still have many beta cells left to save and the disease process is not as aggressive as in newly diagnosed, which should increase the chance that the vaccine will have an effect."

DiAPREV-IT, which was started in 2009, is a double-blind, placebo-controlled Phase II study including a total of 50 children aged four and older who through analysis of diabetes markers, so-called auto-antibodies, in the blood are demonstrated to be at high risk of developing type 1 diabetes. Study participants have auto-antibodies against GAD65 and at least one other auto-antibody. Half of the children receive two injections of Diamyd®, and the remaining half receive placebo (inactive substance). The children will be monitored for a total of five years by means of sampling and glucose tolerance tests to evaluate the beta cell function, a measure of the body's own ability to regulate blood sugar. The first results are expected to be compiled three years after the last participant is enrolled, and can thereby be presented in 2015.

Diamyd® has previously been evaluated in a Phase III study of children already diagnosed with type 1 diabetes. The Phase III study did not met the primary efficacy endpoint of preserving beta cell function. Safety data from the study showed that Diamyd® was well tolerated, as demonstrated by a similar number of adverse events reported across all treatment groups.