MISGAV, Israel & SAN FRANCISCO--(BUSINESS WIRE)--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 received formal notification of recommendations from the National Institutes of Health (“NIH”) Recombinant DNA Advisory Committee (“RAC”) reflecting the Company’s successful presentation of study findings during a meeting held on March 8, 2012. Completion of the RAC process is an important step in advancing toward the Company’s proposed U.S. Phase II clinical study evaluating 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”).
The RAC meeting was attended by an audience from the general public and was webcast live. An archive of this webcast is available at http://videocast.nih.gov/summary.asp?Live=11074.
EPODURE is an autologous dermal Biopump capable of the sustained secretion of therapeutic EPO in the body using a small tissue explant from the patient’s own skin. The EPODURE Biopump is subsequently implanted subcutaneously into the patient to provide continuous delivery of EPO.
“We are delighted that the RAC has given such positive responses regarding our proposed Phase II clinical protocol for EPODURE to treat ESRD and we have already addressed most of their recommendations in our study planning,” said Andrew L. Pearlman, Ph.D., President and Chief Executive Officer of Medgenics. “Clinical trials involving recombinant DNA products intended to be conducted in the U.S. generally need to satisfy review by the RAC, which is widely recognized as a most thorough scientific and clinical review by leading experts in relevant areas. Key to this positive response was the safety and sustained clinical activity we showed from the EPODURE Phase I/II trial conducted in Israel. We believe the RAC recommendations can be incorporated into our proposed protocol without difficulty and will not pose any significant delay or expense. Following a positive pre-Investigational New Drug (“IND”) application meeting with the U.S. Food and Drug Administration (“FDA”) last fall, completion of the RAC process clears an important hurdle toward the successful submission of our IND application and is a significant step in implementing our clinical strategy.
“We look forward to submitting our IND to the FDA in the coming months and to obtaining FDA clearance for our Phase II clinical trial, which we expect will affirm the positive results from our earlier Phase I/II study of EPODURE in pre-dialysis patients,” added Dr. Pearlman. “In that study we demonstrated that a single administration can raise and maintain hemoglobin levels for many months without any injections of erythropoietin stimulating agents.”
Anemia is a common complication of renal failure resulting primarily from 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 super physiological levels, which then decline into a more physiologic range. This variability in levels, and in particular the peak levels which then decline into a more physiologic range. This variability in levels, and in particular the peak levels, has been hypothesized to potentially increase the risk of adverse cardiovascular effects and may make maintenance of steady hemoglobin levels more challenging. 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 the National Institutes of Health Recombinant DNA Advisory Committee
The objective of the Recombinant DNA Advisory Committee is to provide advice to the Director of the NIH on matters related to the conduct and oversight of research involving recombinant DNA, including the content and implementation of the NIH Guidelines for Research Involving Recombinant DNA Molecules and other NIH activities pertinent to recombinant DNA technology. The RAC comprises experts in a wide range of scientific and medical disciplines and also includes medical ethicists and members of patient and other lay communities. Because of the dedication, effort, and thoughtful contributions of its members over the past 30 years, the RAC has been a vital national forum promoting critically important scientific progress in a transparent, responsible, and safe manner and enhancing public trust in the science.
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 (now completing a Phase I/II dose-ranging trial) 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;
- INFRADURETM (planning to commence a Phase I/II trial in Israel in 1H12 in hepatitis C) to produce a sustained therapeutic dose of interferon-alpha for use in the treatment of hepatitis;
- HEMODURETM is a sustained Factor VIII therapy for the prophylactic treatment of hemophilia, now in development.
Medgenics intends to develop its innovative products and bring them to market via strategic partnerships with major pharmaceutical and/or medical device companies.
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.
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.