Tolerx initiates confirmatory Phase 3 clinical trial to evaluate otelixizumab in new-onset type 1 diabetes

Tolerx, Inc., a biopharmaceutical company developing novel therapies to treat autoimmune diseases and cancer by modulating T cell activity, today announced the initiation of a confirmatory Phase 3 clinical trial to further evaluate otelixizumab in autoimmune new-onset type 1 diabetes. The confirmatory Phase 3 clinical trial is called DEFEND-2 (Durable-Response Therapy Evaluation For Early or New-Onset Type 1 Diabetes) and immediately follows successful completion of enrollment in the initial Phase 3 clinical trial, DEFEND-1, with results from DEFEND-1 expected in the first half of 2011.

"For decades, our primary treatment option for patients with type 1 diabetes has been the use of insulin alone to control blood glucose levels, while the underlying autoimmune disease continues to progress," said Dr. Thomas B. Repas, Clinical Assistant Professor, Department of Internal Medicine, University of South Dakota, Sanford School of Medicine, and an investigator in the DEFEND-2 study.  "The future potential of otelixizumab opens up the opportunity for an entirely new treatment approach that can intervene at early onset of type 1 diabetes and potentially break the cycle of the continued progression of this disease that patients manage over their entire lifetime."

DEFEND-2 is a randomized, placebo-controlled confirmatory Phase 3 trial designed to enroll up to 400 patients, age 12 to 45, with newly diagnosed autoimmune type 1 diabetes. DEFEND-2 is being conducted at more than 200 sites in North America and Europe. Building on DEFEND-1, the DEFEND-2 clinical trial similarly evaluates the efficacy and safety of otelixizumab in an additional study population of patients age 12 to 45 with new-onset type 1 diabetes. In the confirmatory study, otelixizumab is administered as a single course, given not more than 90 days after the initial diagnosis of autoimmune type 1 diabetes.  The primary endpoint will be a measurement of C-peptide, a surrogate measure of beta cell function that has been endorsed by the U.S. Food and Drug Administration (FDA) at 12 months after dosing.  Maintenance of beta cell function has been associated with improved glycemic control (HbA1c levels), fewer hypoglycemic events, fewer hyperglycemic excursions, and a reduction in long-term disease complications in established type 1 diabetes patients, as referenced in the Diabetes Control and Complications Trial (DCCT).

"Tolerx is aggressively moving forward in our clinical development of otelixizumab.  We are excited by the potential of this novel drug candidate to change the type 1 diabetes treatment paradigm by inhibiting disease progression and perhaps halting the disease by inducing an immunologic remission," said Dr. Douglas J. Ringler, President and Chief Executive Officer of Tolerx.  "The first patients have been dosed in the DEFEND-2 study, and we believe our study is designed with the robustness and rigor to confirm the efficacy and safety of otelixizumab in a broad patient population."