TolerRx Gets Orphan Drug Designation

TolerRx, Inc. today announcedthat 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 1diabetes mellitus. TolerRx is currently conducting a clinical study of TRX4 inthe U.S. in subjects with Type 1 diabetes. Orphan drug designation would entitle TolerRx to exclusive TRX4 marketingrights in the United States for seven years should TolerRx be the firstcompany to receive marketing approval for this type of therapeutic drugproduct. In addition, the designation would allow TolerRx to apply forresearch funding, tax credits for certain research expenses, and a waiver fromthe 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 inthe treatment of new-onset Type 1 diabetes," said Douglas J. Ringler, ChiefExecutive Officer of TolerRx. "This designation is one in a number of plannedstrategic initiatives that will provide us with financial and regulatorybenefits and market exclusivity." In a study of subjects with new-onset Type 1 diabetes published in theJune 23, 2005 edition of the New England Journal of Medicine, TRX4 (ChAglyCD3)was shown to preserve the function of insulin-producing beta cells in thepancreas and reduce the amount of administered insulin needed to control bloodglucose 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 withtransient symptoms of flu-like syndrome and transient EBV reactivation.TolerRx recently initiated a clinical trial in the U.S. in subjects with Type1 diabetes designed to optimize and select a TRX4 dosing regimen to be used inlater phase trials. About Type 1 Diabetes Diabetes (medically known as diabetes mellitus) is the name given todisorders in which the body has difficulty regulating its blood glucose, orblood 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 adisorder of the body's immune system. In Type 1 diabetes, the pancreasproduces little or no insulin as a result of the immune system attacking anddestroying the insulin-producing beta cells in the pancreas. Therefore, Type 1diabetes patients require frequent administration of insulin therapy each dayto control their blood sugar levels. In the United States, approximately 1.3 million people have Type 1diabetes, and each year approximately 30,000 new patients are diagnosed withthe disease, including 13,000 children. About TRX4 TRX4 is a monoclonal antibody that binds to a receptor found on all Tcells called CD3, which is involved in normal T cell signaling. TRX4 isdesigned to block the function of autoreactive T-effector cells that attackthe body's tissues and cause autoimmune disease. Because T-effector cells andT-regulatory cells utilize different signaling pathways for activation, TRX4is expected to suppress autoreactive T cells while promoting T-regulatory cellactivity, resulting in a state of immunological tolerance. In addition to Type1 diabetes, TolerRx is also developing TRX4 for the treatment of psoriasis andis currently enrolling subjects in a U.S. Phase Ib study of subjects withmoderate-to-severe psoriasis.