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FAQs Q. Who is RegeneRx Biopharmaceuticals? Q. Who is RegeneRx Biopharmaceuticals? A. RegeneRx Biopharmaceuticals, Inc. was founded in 1982 and is a biopharmaceutical research and development company focusing on the development of products to treat a variety of human diseases. In particular, RegeneRx is focused on developing drugs that treat chronic wounds such as chronic pressure ulcers (bed sores), diabetic foot ulcers, as well as "orphan" diseases such as epidermolysis bullosa. The Company also has a strong interest in ophthalmic wound healing, as well as cardiovascular and neurovascular repair. The Company went public in 1986 and has remained a publicly-traded company since that time. RegeneRx is headquartered in Bethesda, Maryland. [Top] Q. Where is the Company's stock listed? A. RegeneRx is listed on the American Stock Exchange under the symbol RGN, which may be found by visiting the AMEX website. [Top] Q. What is the Company's recent financial and operational history? A. Year-end financial results, as well as quarterly results, may be obtained through RegeneRx’s most current 10-K and 10-Q SEC filings. Operationally, the Company has successfully initiated and completed a Phase I clinical trial for chronic dermal wound healing, while undertaking non-clinical studies in support of its clinical efforts. RegeneRx is sponsoring three Phase II dermal wound healing clinical trials, a Phase II ophthalmic wound healing trial in diabetic patients undergoing eye surgery and a Phase I study in support of a proposed Phase II acute myocardial infarction clinical trial. Additionally, RegeneRx has developed three distinct drug formulations for each of its three clinical areas of interest, including: a parenteral "injectable" formulation that may be used for systemic administration of Tβ4, and a topical gel and sterile eye drops. [Top] Q. What are the Company's primary product candidates? A. RegeneRx's principal investigational drug is based upon Thymosin Beta 4 (Tß4), a chemically synthesized copy of a natural human peptide that circulates in the blood and plays a vital role in the regeneration, remodeling, and healing of tissues. Tß4 represents a first-in-class, investigational drug being developed to speed the healing of injured tissues, accelerate the growth of blood vessels, and reduce inflammation. Tß4 is the foundation of a technology platform that may have broad clinical applications -- from dermal wound repair to ocular repair to preventing damage and repairing internal organs and tissues. To date, RegeneRx has formulated three Tβ4-based drug candidates that are, or will be, used in its phase II clinical trial program. [Top] Q. What is the RegeneRx business strategy? A. The Company's strategy is to create a flexible business model that will allow it to operate as efficiently as possible without building unnecessary infrastructure. This strategy has allowed RegeneRx to quickly meet its goals of initiating clinical trials with its product while maintaining flexibility to modify expenditures as the capital markets allow. This approach is sometimes referred to as a "virtual" company model due to the emphasis on outsourcing key projects (e.g., manufacturing, non-clinical studies, clinical trials) rather than building in-house capability. [Top] Q. Does the Company have strategic alliances with any other companies? A. In January 2004, the Company out-licensed certain rights to Tß4 to Defiante Farmaceutica Lda, a wholly owned subsidiary of the Sigma-Tau Group, a global pharmaceutical firm headquartered in Rome, Italy. Under the terms of the license, Defiante/Sigma-Tau is developing Tß4 in Europe and a number of other contiguous and geographically relevant countries for internal and external wound healing. Defiante/Sigma-Tau will pay a royalty to RegeneRx and also exclusively purchase all Tß4 from RegeneRx throughout the term of the license. Defiante/Sigma-Tau has certain product development obligations and performance criteria related to commercial sales. [Top] Q. What distinguishes Tß4 from other wound healing compounds? A. Tß4 has a number of unique and important properties. It is a first-in-class molecule that performs a number of biological functions such as regulating actin and plays a vital role in the healing of injured or damaged tissues. Tß4 is present in concentrations that are high enough to sequester most of the G-actin in cells, inhibiting its polymerization. For more information view our Technology page. In non-clinical studies, Tß4 has also been shown to promote endothelial cell migration and angiogenesis in certain tissues (dermal but not ocular), and induce collagen deposition and production of laminin-5. Tß4 can prevent apoptosis and down-regulates certain inflammatory cells. A recent scientific paper, published in Nature in January 2007 showed that Tß4 stimulated adult epicardial stem cells to differentiate into cardiac blood vessels. This data has very important implications for not only treating heart attacks but also for other ischemic events such as stroke. Also of interest, Tß4 is a "conserved" molecule, which means it is identical from species to species. Most experts would agree that there is a reason a molecule is "conserved" in that it likely plays an important biological role, thus, it has not been subject to change through evolution or from species to species. [Top] Q. How is Tß4 produced and at what cost? A. Tß4 is a 43-amino acid peptide that is manufactured through solid-phase chemical synthesis. This process, which was partially developed by Dr. Su Sun Wang, a retired former company executive, allows large quantities of the product to be manufactured efficiently. Based on current estimates, the production costs will decrease as we scale-up quantities and, therefore, be consistent with cost structures of other pharmaceutical products. [Top] Q. Is there much scientific data on Tß4? A. There is a large body of scientific data on Tß4 from researchers around the world that describes its activity both in vitro and in animal models, much of which is the basis for our current development endeavors. Researchers at the NIH, and other major research institutions, have published numerous articles on Tß4 showing the compound can accelerate dermal and ocular wound healing. In one of the original NIH publications, the authors concluded that, "These results suggest that Tß4 is a potent wound healing factor with multiple activities that may be useful in the clinic." In the November 24, 2004 issue of Nature, researchers at the University of Texas Southwestern Medical Center reported that Tß4, when administered within 24 hours of a myocardial infarction in mice (induced by ligating the coronary artery), was able to prevent or reduce the damage to the heart tissue. There was a statistically significant reduction in scar tissue and increase in cardiac function compared to a placebo. According to Alan Wasserman, M.D., a noted cardiologist, and Professor and Chairman of the Department of Medicine at The George Washington University School of Medicine: “This paper by Dr. Srivastava and colleagues is one of the most exciting works in the field of acute myocardial infarction I have seen. The ability to repair [cardiac] muscle after an acute infarction makes the use of Tß4, potentially, the most important discovery since the advent of thrombolytic therapy.” Scientific publications relevant to the Company's interests can be seen by viewing our Publications page. [Top] Q. Is the mechanism of action of Tß4 known? A. Yes, we believe there are several mechanisms of action, please view our Technology page. [Top] Q. What is the status of human clinical trials? A. RegeneRx previously successfully completed a Phase I dermal clinical trial and is now sponsoring three Phase II dermal wound healing clinical trials with Tß4, two in the U.S. and one in Europe through our partner Sigma-Tau. RegeneRx is also sponsoring a Phase II clinical trial targeting repair of the cornea (outer surface of the eye) after surgery in diabetic patients and a Phase I study to support a planned Phase II acute myocardial infarction (heart attack) trial. For more information view our Clinical Trials page. [Top] Q. How does the FDA regulate the Company's products? A. Pre-clinical testing must be conducted to evaluate the potential efficacy and the safety of an investigational drug in animals prior to its use in human clinical trials. The results of these studies are submitted to the U.S. FDA as part of an Investigational New Drug (IND) submission, which must be reviewed before clinical testing can begin. Clinical evaluation involves a three-stage process. In Phase I, trials are conducted with a small number of subjects to determine the safety profile, the pattern of drug distribution and metabolism. In Phase II, trials are conducted with patients afflicted with a specific disease in order to determine preliminary efficacy, optimal dosages, and expanded evidence of safety. In Phase III, large scale, multi-center trials are conducted with patients afflicted with a target disease in order to provide enough data for statistical proof of safety and efficacy required by the FDA. The results of the pre-clinical and clinical testing with detailed information on manufacturing are then submitted to the FDA in the form of a New Drug Application (NDA) for approval to commence commercial sales. Q. How would the Company characterize the markets in which it is conducting or plans to conduct clinical trials? A. Impaired wound healing is a problem for the elderly, diabetics, immunosuppressed and immobilized individuals. The cost of treating all chronic wounds is estimated to be over $8 billion per year and is expected to increase 10% annually. It is estimated that of the 14 million diabetics in the United States, some 1.5 million suffer from impaired healing problems. According to the Centers of Medicare and Medicaid Services, the number of lower-extremity, diabetes-related amputations has risen from 56,000 in 1994 to 87,000 in 2000. The incidence of pressure ulcers is even greater with 4.9 million in the acute care setting, 1.2 million in the long-term health care setting, and 1.1 million in the home health setting. At present, the cost of treating a pressure ulcer can be as high as $50,000 per patient and require several months or even years to heal. There are numerous opportunities for an ophthalmic wound healing product. For example, people with diabetes often have difficulty healing after ophthalmic surgeries, such as vitrectomies. Injuries to the cornea, which may eventually heal, can result in eye damage due to inflammation within the cornea. Also, certain eye drops with preservatives to keep out bacteria, can cause inflammation that can adversely affect the eye. It is estimated there are 200,000 vitrectomies (removal of the gel-like fluid inside the middle of the eye) in the U.S. each year and another 360,000 in the rest of the world. Many of these surgeries are performed in diabetic patients who often have difficulty healing. Due to an aging population, epidemic increases in diabetes and diabetic retinopathy, the incidence of vitrectomy surgery is growing at 5.5% per year and more retinal specialists are being trained to perform vitrectomies. Heart attacks occur in a million people annually in the U. S. and 13+ million suffer from coronary artery disease. Heart attacks are the single largest cause of death in U.S. and result in $250 billion in direct costs per year. Reperfusion (reopening a blocked vessel) can cause significant myocardial damage as well. The company believes that if Tβ4 is efficacious in preventing damage to the heart after a heart attack, it may similarly prove efficacious in the brain after ischemic damage. Strokes occur in over 700,000 people annually in U.S. and are the 3rd leading cause of death and leading cause of disability among adults. Strokes result in $54 billion in direct and indirect costs per year in the U.S. [Top] Q. What is the Company's competitive advantage in this market? A. Although it is recognized that wound healing is a complex process, most companies working to develop new drugs in this area have focused primarily on adding growth factors to stimulate healing and have, to date, failed to demonstrate dramatic improvements in the healing process. In fact, for most recent clinical studies in patients with non-healing chronic leg ulcers it has been found that there is no deficiency in either growth factors or inflammation mediators. Rather, these new studies suggest that healing may be impaired by inflammatory molecules rather than inhibited by a deficiency of growth factors. Because Tß4 is not a growth factor nor a cytokine, its mechanism of action is different in many important ways as described above. Since Tß4 has a number of documented biological properties, each of which appears to play a role in tissue and organ repair, we believe this multi-faceted compound may allow a more versatile and comprehensive approach to wound healing. [Top] Q. Has the Company developed other products? A. The basis on which the Company was founded in 1982 was the discovery of Thymosin alpha 1 (Tα1) by Dr. Allan Goldstein, RegeneRx's Chairman and Chief Scientific Advisor and Chairman of Biochemistry and Molecular Biology at the George Washington University Medical School in Washington, D.C. Tα1 was licensed to a European pharmaceutical company in the 1980's and reached the Italian market shortly thereafter. The Company subsequently licensed (and eventually sold its rights) to Tα1 to a U.S. biopharmaceutical company in the 1990's which is marketing it in over 30 countries outside the U.S. for the treatment of hepatitis B and C, has marketing applications pending in several other countries, and is conducting Phase III human clinical trials in the U.S. [Top] Q. What is the intellectual property status of Tß4? A. Currently the Company holds several issued patents specifically related to its technology platform, has obtained world-wide rights to several pending patents under an exclusive license from NIH, and has filed nearly 60 patent applications in total for analogues, fragments and other compositions and combinations, as well as methods of use and delivery. The company believes that these patents, and those it expects to receive, will give it a broad spectrum of coverage for the identified medical uses for Tß4. [Top] Q. Does the Company collaborate with outside researchers? A. For the past several years, the Company has been working with the NIH to explore Tß4 for the treatment of chronic wounds. Through this collaboration the Company has received an exclusive license from the NIH. The Company has also supported work or collaborated with researchers at the University of Texas - Southwestern in Dallas, Children's National Medical Center in Washington, D.C., Wayne State University in Detroit, The George Washington University Medical School in the District of Columbia, and with scientists at other research institutions across the U.S. and abroad. Typically, such collaborations are conducted under Material Transfer Agreements whereby RegeneRx retains an option to exclusively license any rights to new technology that may be developed resulting from the collaboration. [Top] Q. What is the Company's plan for commercializing Tß4? A. Because of Tß4's well-documented biological activities, it may have important applications in a number of common diseases or injuries. RegeneRx is not currently able to address all of these potential applications and is considering partners to co-develop Tß4 in the ophthalmic, neurological and cardiac fields in territories outside the U.S. The Company is planning to continue to independently develop the product for the treatment of such indications in the United States. Pursuant to this product development strategy, in January 2004 the Company entered a strategic alliance with Defiante Farmaceutica, Lda, a wholly-owned subsidiary of Sigma-Tau Group, a European pharmaceutical company with annual revenues of approximately $1 billion. RegeneRx licensed to Defiante/Sigma-Tau certain European rights to Tß4 for the treatment of internal and external wounds. Defiante/Sigma-Tau will develop Tß4 for that market and pay royalties to the Company upon commercial sales. In addition to royalties, RegeneRx will exclusively supply Tß4 to Defiante/Sigma-Tau and, thus, have another source of recurring product revenue. An important aspect of the strategic alliance is the exchange of information and data. Both partners should benefit from the independent development of non-clinical and clinical data related to Tß4. These data will be exchanged among the parties and should facilitate product development in each territory of interest. The Company believes strategic relationships with partners such as Defiante/Sigma-Tau are highly complementary and offer stockholders a more diversified portfolio of product development opportunities. [Top] Q. Where would one find additional information on the Company and its activities? A. The most comprehensive source would be to read the Company's latest 10-K by clicking here. [Top] |