Patient enrollment in Tolerx's Phase 3 clinical study DEFEND-1 completed

Jan 8 2010

Tolerx, Inc., today announced the completion of patient enrollment in its Phase 3 clinical study DEFEND-1, which is evaluating the safety and efficacy of otelixizumab, a targeted T cell immunomodulator, in patients with new-onset autoimmune type 1 diabetes.

The DEFEND-1 (Durable Response Therapy Evaluation For Early or New-Onset Type 1 Diabetes) study enrolled 240 patients, aged 12-45 years with newly diagnosed autoimmune type 1 diabetes. The DEFEND-1 study is investigating the ability of otelixizumab to preserve beta cell function, which may reduce the risk of both short- and long-term complications of the disease. Patients will be monitored during the 12-month follow-up period and c-peptide levels (a surrogate measure of beta cell function) will be measured as the primary endpoint. Secondary endpoints will evaluate the patient's ability to maintain excellent glycemic control as measured by HbA1c levels and the amount of daily injected insulin required.

Dr. Paolo Pozzilli, Professor of Endocrinology & Metabolic Diseases at the University Campus Bio-Medico in Rome, Italy, and a DEFEND-1 investigator commented: "Reaching full enrollment in DEFEND-1 is a major accomplishment for the type 1 diabetes community and furthers the development of innovative immunomodulating therapies for our patients. We will continue to work with Tolerx to further otelixizumab's clinical development and to validate its promise of beta cell preservation in new-onset autoimmune type 1 diabetes patients."

Peter A. Gottlieb, MD, Associate Professor of Pediatrics and Medicine at the Barbara Davis Center at the University of Colorado at Denver, known for his involvement in many types of clinical trials for the prevention and treatment of diabetes, noted that, "The continuous otelixizumab dose regimen optimization efforts have been a major translational research focus. These important research efforts are enabling us, in the DEFEND development program, to evaluate the potential ability of otelixizumab to provide a long-term immunologic remission after a short course of therapy. If successful in the clinic, this would be a significant step forward."

Tolerx also announced today its intention to conduct a second confirmatory Phase 3 study of otelixizumab in new-onset autoimmune type 1 diabetes. Further details of the design and timing of the study, to be named DEFEND-2, will be forthcoming.

"The on-time completion of patient enrollment in DEFEND-1 represents a major milestone for Tolerx," said Dr. Douglas J. Ringler, President and Chief Executive Officer of Tolerx. "We are very grateful for the dedication of the patients, their caregivers and our clinical trial investigators for making it possible to reach our enrollment target. As part of our clinical development program to reach regulatory approval for otelixizumab, we will now quickly transition to the launch of DEFEND-2, a confirmatory study, to maintain enrollment momentum and enthusiasm generated to date in the type 1 diabetes community."