CAMBRIDGE, Mass., Feb. 14 /PRNewswire/ -- TolerRx, Inc. today announced that its lead product, TRX4, has received orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of new-onset Type 1 diabetes mellitus. TolerRx is currently conducting a clinical study of TRX4 in the U.S. in subjects with Type 1 diabetes.
Orphan drug designation would entitle TolerRx to exclusive TRX4 marketing rights in the United States for seven years should TolerRx be the first company to receive marketing approval for this type of therapeutic drug product. In addition, the designation would allow TolerRx to apply for research funding, tax credits for certain research expenses, and a waiver from the FDA application user fee required by the Prescription Drug User Fee Act (PDUFA).
"We are pleased to have received this orphan drug designation for TRX4 in the treatment of new-onset Type 1 diabetes," said Douglas J. Ringler, Chief Executive Officer of TolerRx. "This designation is one in a number of planned strategic initiatives that will provide us with financial and regulatory benefits and market exclusivity."
In a study of subjects with new-onset Type 1 diabetes published in the June 23, 2005 edition of the New England Journal of Medicine, TRX4 (ChAglyCD3) was shown to preserve the function of insulin-producing beta cells in the pancreas and reduce the amount of administered insulin needed to control blood glucose levels for at least 18 months after a single six day course of TRX4. At the doses used in this study, TRX4 administration was associated with transient symptoms of flu-like syndrome and transient EBV reactivation. TolerRx recently initiated a clinical trial in the U.S. in subjects with Type 1 diabetes designed to optimize and select a TRX4 dosing regimen to be used in later phase trials.
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, or blood sugar, levels. There are two major types of diabetes: Type 1 and Type 2. Type 1, also called juvenile diabetes or insulin-dependent diabetes, is a disorder of the body's immune system. In Type 1 diabetes, the pancreas produces little or no insulin as a result of the immune system attacking and destroying the insulin-producing beta cells in the pancreas. Therefore, Type 1 diabetes patients require frequent administration of insulin therapy each day to control their blood sugar levels.
In the United States, approximately 1.3 million people have Type 1 diabetes, and each year approximately 30,000 new patients are diagnosed with the disease, including 13,000 children.
About TRX4
TRX4 is a monoclonal antibody that binds to a receptor found on all T cells called CD3, which is involved in normal T cell signaling. TRX4 is designed to block the function of autoreactive T-effector cells that attack the body's tissues and cause autoimmune disease. Because T-effector cells and T-regulatory cells utilize different signaling pathways for activation, TRX4 is expected to suppress autoreactive T cells while promoting T-regulatory cell activity, resulting in a state of immunological tolerance. In addition to Type 1 diabetes, TolerRx is also developing TRX4 for the treatment of psoriasis and is currently enrolling subjects in a U.S. Phase Ib study of subjects with moderate-to-severe psoriasis.
About TolerRx, Inc.
TolerRx is a biopharmaceutical company specializing in the discovery, development, and commercialization of novel therapies to treat patients with immune-mediated diseases. TolerRx has two antibodies in clinical development: TRX4 in Type 1 diabetes and psoriasis, and TRX1 in cutaneous lupus erythematosus (CLE). TRX1 is a humanized anti-CD4 antibody that is being developed in collaboration with Genentech, Inc. TRX4 is an anti-CD3 monoclonal antibody to which TolerRx owns exclusive rights. TolerRx is also developing new products to induce immunological tolerance for the treatment of autoimmune diseases and to remove tolerance for the treatment of cancer or chronic viral diseases. For more information, please visit http://www.tolerrx.com.
Safe Harbor Statement
This press release may contain or incorporate by reference certain statements that are not historical facts, including statements preceded by, followed by or that include the words "may," "believes," "expects," "anticipates," or the negation thereof, or similar expressions, which constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 (the "Reform Act"). All statements that address events, transactions or developments that are expected or anticipated to occur in the future are forward-looking statements within the meaning of the Reform Act. Such forward-looking statements involve risks, uncertainties and other factors that may cause the actual performance or achievements of TolerRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. For those statements, TolerRx claims the protection of the safe harbor for forward-looking statements contained in the Reform Act. TolerRx will not undertake and specifically declines any obligation to publicly release the result of any revisions that may be made to any forward-looking statements to reflect events or circumstances after the date of such statements or to reflect the occurrence of anticipated or unanticipated events.
TolerRx, Inc. Contact: Robert Kirsch, RAC Vice President, Regulatory Affairs (617) 452-1300 rkirsch@tolerrx.com
Source: TolerRx, Inc.
