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RegeneRx Receives Orphan Drug Designation for Thymosin Beta 4 for the Treatment of Epidermolysis Bullosa RegeneRx Receives Orphan Drug Designation for Thymosin Beta 4 for the Treatment of Epidermolysis Bullosa June 4, 2004 — Bethesda, Md RegeneRx Biopharmaceuticals, Inc. (OTCBB:RGRX) (www.regenerx.com) announced that it has received Orphan Drug designation from U.S. FDA's Office of Orphan Products Development (OOPD) for its drug, Thymosin beta 4 (Tß4), for the treatment of Epidermolysis Bullosa (EB). Orphan drug designation results in seven years of market exclusivity upon FDA approval of a marketing application for that use, the waiver of certain FDA user fees, tax credits of up to 50% of qualified U.S. clinical trial expenses, and eligibility for FDA research grants for clinical trials. "This designation," explains J.J. Finkelstein, RegeneRx's President & CEO, "offers us a number of opportunities that would not otherwise have been available. It is an important milestone -- the result of the growing body of scientific data supporting Tß4's development for this debilitating disease. Recent experimental studies in animal models have documented the ability of Tß4 to stimulate the production of laminin-5, a key adhesive protein missing or diminished in certain EB patients. We now look forward to establishing Tß4's safety and efficacy in clinical trials in patients with EB in order to obtain FDA marketing approval." EB is a rare genetic disease characterized by the presence of extremely fragile skin and recurrent blister formation, resulting from minor mechanical friction or trauma. Often these blisters can develop on the skin, eye, mouth, as well as other organs and tissues. An estimated 2 out of every 100,000 live births are affected with some type of EB. The disorder occurs in every racial and ethnic group throughout the world and affects both sexes equally. Patients with the most severe form of EB produce little or no laminin-5, a protein that induces both adhesion and migration in a wide variety of cell types, including epithelial cells, fibroblasts, neurons and leukocytes. Laminin-5 plays a key role in maintaining the structural integrity of the skin. Tß4 is a naturally occurring 43-amino acid peptide present in virtually all human cells. It is a drug that promotes endothelial cell differentiation and keratinocyte cell migration, down-regulates a number of inflammatory cytokines and chemokines, and has a very low molecular weight, allowing it to diffuse relatively long distances through tissues. A key mechanism of action is Tß4's ability to regulate the cell-building protein, actin, a vital component of the cytoskeleton. Additionally, Tß4 directly influences the production of laminin-5, a protein necessary for proper adhesion of certain types of mammalian cells. Tß4 has been the subject of a significant amount of research at the National Institutes of Health (NIH) and several other academic institutions. RegeneRx is a biopharmaceutical company developing products based on the Tß4 platform for the treatment of EB, as well as other applications. The Company holds an exclusive worldwide license from the NIH, holds several issued patents related to its technology, and has numerous patent applications pending in countries throughout the world. RegeneRx recently entered into a strategic license and supply agreement with Defiante Farmaceutica, Lda, a wholly-owned subsidiary of Sigma-Tau Group, headquartered in Rome (Italy), that will develop Tß4 in Europe and other contiguous and geographically relevant countries for certain applications. Sigma-Tau is a leading research-based Italian pharmaceutical company with annual revenues of approximately $700 million and over 2200 employees worldwide. The information in this press release may include certain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements concern the Company's current expectations regarding future events, including the regulatory approval process and the ongoing and prospective development and commercialization of Tß4 and possible future benefits to the Company, its shareholders, and patients. Due to the nature of clinical trials, product development and the regulatory approval process, the forward-looking statements contained in this press release are subject to risks and uncertainties, including those reflected in the Company's filings with the Securities and Exchange Commission, particularly its most recent Annual Report on Form 10-KSB. The Company assumes no obligation to update or revise any forward-looking statements made herein or any other forward-looking statements made by the Company. |
