Medgenics, Inc. (NYSE Amex: MDGN and AIM: MEDU, MEDG), the developer of a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announced that it has filed an Investigational New Drug (“IND”) application with the U.S. Food and Drug Administration (“FDA”) to initiate a Phase IIb multi-center, 100-patient clinical trial. The trial is designed to evaluate the safety and efficacy of sustained erythropoietin (“EPO”) therapy delivered via the Company’s EPODURE Biopump™ for the treatment of anemia in dialysis patients with end-stage renal disease (“ESRD”). EPODURE is an autologous dermal Biopump, a small tissue implant made from the patient’s own dermal (skin) tissue. EPODURE Biopumps are processed to enable the continuous production of EPO, and are subsequently implanted subcutaneously. Positive data from the Company’s Phase I/II dose-ranging study of EPODURE in pre-dialysis patients were presented at the 2010 and the 2011 American Society of Nephrology Annual Meetings, and demonstrated that a single administration can raise and maintain hemoglobin levels for many months without any injections of EPO or other erythropoietin stimulating agents (“ESAs”). The filing of this IND application follows two key events that occurred in March 2012: the regulatory approval for a smaller Phase IIa study for the same indication in Israel; and positive meetings with the National Institutes of Health’s Recombinant DNA Advisory Committee in the U.S. “This IND filing is our first with the FDA and marks a significant milestone for Medgenics. It is one of many key catalysts we have targeted for our Company in 2012. We anticipate receiving the FDA’s comments in May, which we believe could lead to trial approval in the third quarter of 2012,” said Andrew L. Pearlman, Ph.D., President and Chief Executive Officer of Medgenics. “Filing for the anemia indication of our platform technology is a key step not only for EPODURE, but helps pave the way for multiple future indications that address multibillion-dollar markets.”
About AnemiaAnemia is a common result of renal failure primarily caused by insufficient production of the hormone EPO by the damaged kidneys, which leads to a decrease in production of red blood cells. Treatment with EPO typically involves intravenous or subcutaneous administration by injection at regular intervals followed by frequent laboratory tests to monitor hemoglobin concentration. Due to the short half-life of the protein, it is usually administered at doses that result in supra-physiological levels far above normal range, which then decline through the range and often drop below it until the next injection. This variability in levels, particularly the peak levels far above physiologic range, is in stark contrast to the gradual changes maintained by the kidneys, and have been suggested by researchers to increase the risk of adverse cardiovascular effects and may make maintenance of steady hemoglobin levels more challenging. EPODURE is designed to provide a gradual supply of EPO, to follow the natural model and to avoid the risks that swings in EPO and hemoglobin levels are thought to increase. The need for frequent injections and laboratory tests, medication compliance and lifestyle adjustment are potential barriers to achieving the desired hemoglobin goal. These barriers, together with the high costs of the currently available recombinant EPO products, provide an incentive to improve care with a sustained therapy approach. About Medgenics Medgenics is developing and commercializing Biopump™, a proprietary tissue-based platform technology for the sustained production and delivery of therapeutic proteins using the patient's own skin biopsy for the treatment of a range of chronic diseases including anemia, hepatitis C and hemophilia. Medgenics believes this approach has multiple benefits compared with current treatments, which include regular and costly injections of therapeutic proteins. Medgenics has three long-acting protein therapy products in development based on this technology:
- EPODURE to produce and deliver erythropoietin for many months from a single administration, has demonstrated elevation and stabilization of hemoglobin levels in anemic patients for six to more than 36 months in a Phase I/II dose-ranging trial in CKD patients, and is about to commence a Phase IIa safety/efficacy trial in ESRD dialysis patients in Q2 2012 in Israel.
- INFRADURETM for sustained production and delivery of interferon-alpha for use in the treatment of hepatitis is awaiting final approval for two Phase I/II trials in hepatitis C in Israel, slated to commence Q3 2012.
- HEMODURETM for sustained production and delivery of clotting Factor VIII therapy for the prophylactic treatment of hemophilia is now in development.
In addition to treatments for anemia, hepatitis and hemophilia, Medgenics plans to develop and/or out-license a pipeline of future Biopump products targeting the large and rapidly growing global protein therapy market, which is forecast to reach $132 billion in 2013. Other potential applications for Biopumps include multiple sclerosis, arthritis, pediatric growth hormone deficiency, obesity and diabetes.Forward-looking Statements This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and as that term is defined in the Private Securities Litigation Reform Act of 1995, which include all statements other than statements of historical fact, including (without limitation) those regarding the Company's financial position, its development and business strategy, its product candidates and the plans and objectives of management for future operations. The Company intends that such forward-looking statements be subject to the safe harbors created by such laws. Forward-looking statements are sometimes identified by their use of the terms and phrases such as "estimate," "project," "intend," "forecast," "anticipate," "plan," "planning, "expect," "believe," "will," "will likely," "should," "could," "would," "may" or the negative of such terms and other comparable terminology. All such forward-looking statements are based on current expectations and are subject to risks and uncertainties. Should any of these risks or uncertainties materialize, or should any of the Company's assumptions prove incorrect, actual results may differ materially from those included within these forward-looking statements. Accordingly, no undue reliance should be placed on these forward-looking statements, which speak only as of the date made. The Company expressly disclaims any obligation or undertaking to disseminate any updates or revisions to any forward-looking statements contained herein to reflect any change in the Company's expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based. As a result of these factors, the events described in the forward-looking statements contained in this release may not occur.