August 6, 2008
Tolerx Initiates Dosing of Otelixizumab, a Novel Type 1 Diabetes Agent, in DEFEND, a Phase 3 Clinical Trial
CAMBRIDGE, Mass., Aug. 6 /PRNewswire/ -- Tolerx, Inc., a biopharmaceutical company engaged in the discovery and development of novel therapies for immune-mediated diseases, today announced the initiation of a pivotal Phase 3 clinical trial that is evaluating otelixizumab in autoimmune new onset type 1 diabetes. As a result of the initiation of patient dosing in DEFEND (Durable Response Therapy Evaluation For Early or New Onset Type 1 Diabetes), Tolerx will receive a $15 million milestone payment from GlaxoSmithKline.
DEFEND is a randomized, placebo controlled Phase 3 trial designed to enroll approximately 240 adult patients, age 18 to 35, with newly diagnosed autoimmune type 1 diabetes. DEFEND is being conducted at multiple centers in North America and Europe. The trial will evaluate whether a single course of otelixizumab, administered not more than 90 days after the initial diagnosis of autoimmune type 1 diabetes, will reduce the amount of administered insulin required to control blood glucose levels by inhibiting the destruction of beta cells. The primary endpoint will be a measurement of C-peptide, a surrogate measure of beta cell function. Maintenance of beta cell function has been associated with improved glycemic control (HbA1c levels), fewer hypoglycemic events, and a reduction in long-term disease complications in established type 1 diabetics in the Diabetes Control and Complications Trial (DCCT).
"We are excited to have dosed the first patient in DEFEND and look forward to continued patient enrollment in this Phase 3 clinical trial. The initiation of DEFEND represents the culmination of a methodical, dose-ranging development plan, including the successful outcome from an end-of-Phase 2 meeting at FDA. We believe otelixizumab has the potential to provide an important new treatment option for patients with newly diagnosed autoimmune type 1 diabetes," said Dr. Douglas J. Ringler, President and Chief Executive Officer of Tolerx.
For additional information about DEFEND, please visit http://www.defendagainstdiabetes.com/.
About Type 1 Diabetes
Diabetes (medically known as diabetes mellitus) is the name given to disorders in which the body has difficulty regulating its blood glucose (sugar) level. There are two major types of diabetes: type 1 and type 2. Type 1, previously known as juvenile diabetes or insulin-dependent diabetes, is a disorder of the body's immune system. In type 1 diabetes, the immune system attacks and destroys the insulin-producing beta cells in the pancreas. The decrease in endogenous (natural) insulin production means that patients must monitor their glucose levels frequently and take insulin regularly to control their blood glucose levels.
Otelixizumab is a monoclonal antibody that binds to CD3, a T lymphocyte receptor involved in normal cell signaling. Otelixizumab is thought to work by blocking the function of effector T cells that attack the body's tissues and cause autoimmune disease while inducing a subset of T cells known as regulatory T cells. It is thought that the regulatory T cells may protect against effector T cell damage well after the antibody has been eliminated from the body. In addition to DEFEND, Tolerx is also continuing to evaluate otelixizumab in subjects with type 1 diabetes in its ongoing Phase 2 study, TTEDD (TRX4 Therapeutic Evaluation of Different Dosing Regimens). A principal purpose of TTEDD is to evaluate different dosing regimens in an effort to reduce side effects and to acquire additional information about otelixizumab's clinical activity. Tolerx has conducted dose optimization studies in type 1 diabetes and has identified a dosing regimen that thus far has significantly reduced side effects while maintaining important biological activity. This dosing regimen is being used in DEFEND. For more information on DEFEND, please see http://www.defendagainstdiabetes.com/.
Tolerx Collaboration with GlaxoSmithKline
In October 2007, Tolerx and GlaxoSmithKline (GSK) entered into a global alliance to develop and commercialize otelixizumab in numerous indications, including type 1 diabetes. Under the terms of the agreement, Tolerx is responsible for conducting the Phase 3 clinical program for type 1 diabetes in the US and if successful, for submitting the biologics license application (BLA) to the FDA. Tolerx has the option to co-promote otelixizumab in type 1 diabetes in the US with GSK while GSK has exclusive rights to develop and commercialize otelixizumab in all other indications worldwide, including pediatric type 1 diabetes.
Tolerx is a biopharmaceutical company engaged in the discovery and development of novel therapies for immune-mediated diseases. Tolerx currently has two antibodies in clinical development: otelixizumab and a modified version of TRX1, MTRX1011A. MTRX1011A is a humanized anti-CD4 monoclonal antibody being developed in collaboration with Genentech, Inc. Tolerx is also engaged in preclinical development of new monoclonal antibody product candidates that engage the body's immune system and promote and enhance immune responses. These antibodies are being assessed for potential benefit in the treatment of cancer, chronic viral diseases, and as an adjunct to vaccines. For more information, please visit http://www.tolerx.com/.
Tolerx Forward-Looking Statements
This press release contains certain statements that are preceded by, followed by, or that include the words "will evaluate,""will,""look forward,""believe," and "has the potential to," or similar expressions or the negation thereof. Such statements, which are not historical facts, constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 (the "Reform Act"). Statements regarding the outcomes sought to be realized in the Phase 3 clinical trial, the possibility that otelixizumab may have the potential to change type I diabetes treatment paradigms, the anticipated progress and development of otelixizumab, in type 1 diabetes and other disease indications, are all forward-looking statements. Such forward-looking statements involve risks, uncertainties and other factors that may cause the actual performance or achievements of Tolerx to be materially different from any future results, performance, or achievements expressed or implied by such forward-looking statements. Factors and risks that may impact management's expectations and affect the forward-looking statements shall include but not be limited to results of DEFEND and other future clinical studies, the ability to obtain regulatory approval for otelixizumab for type 1 diabetes or any other indication, the continuation of the collaboration with GSK, the introduction of competing therapies by other companies, and changes in the company's business plan or objectives. These statements reflect the view of Tolerx as of the date of this press release and should not be relied upon as reflecting the company's views at any date subsequent to this release. Tolerx undertakes no obligation to update or revise any forward-looking statements to reflect events or circumstances after the date of this press release.
CONTACT: Cory Tromblee of Tolerx, Inc., +1-617-897-8294
Web site: http://www.tolerx.com/http://www.defendagainstdiabetes.com/