Medgenics, Inc. (NYSE MKT: MDGN and AIM: MEDU, MEDG), the developer of Biopump TM, a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announced that its INFRADURE™ Biopump has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) for the treatment of hepatitis D. INFRADURE is the version of Medgenics’ Biopump technology platform which produces interferon alpha, commonly used to treat hepatitis. This marks the first Orphan Drug Designation granted for treatment of a clinical indication using the Biopump. Orphan Drug Designation carries multiple benefits, including the availability of grant money, certain tax credits and seven years of market exclusivity, as well as the possibility of an expedited regulatory process. Marlene Haffner, M.D. MPH, former Director of Orphan Products Development at the FDA, and regulatory advisor to Medgenics said, “I am excited by this grant of Orphan Drug Designation of INFRADURE in hepatitis D. The novel Biopump platform potentially offers significant advance over current treatment not just for an orphan disease, but also for other diseases where protein therapy is a potential treatment. Designated Orphan Products have frequent access for advice to both FDA’s OOPD and to the FDA review divisions. They are also frequently approved by FDA via an accelerated pathway of Priority Review, which is granted for drugs offering a significant advance in the treatment of a serious and life threatening disease, or when there is no approved treatment. The INFRADURE Biopump seems to meet these criteria, particularly since hepatitis D is a serious disease with no approved treatment.” Bruce R. Bacon, M.D., past President of the American Association for the Study of Liver Disease, a recognized global expert in hepatitis and a member of Medgenics’ Strategic Advisory Board, commented, “INFRADURE offers the potential for a superior treatment for the 15 million people suffering from hepatitis D worldwide. The current treatment for hepatitis D requires years of weekly injections of interferon alpha, which leads to patient discomfort and substantial compliance challenges. Oral antiviral treatments have proven to be ineffective in treating hepatitis D. INFRADURE is intended to be implanted infrequently, with a single administration potentially replacing many months of weekly injections. This could offer a safe and efficacious treatment that could greatly improve patient compliance. The treatment also has potential for efficacy with greater patient compliance for other forms of hepatitis including the 170 million people infected with hepatitis C and the over 350 million people infected with hepatitis B.”
“The FDA’s timely approval of our Orphan Drug Designation for INFRADURE to treat hepatitis D is a key milestone in our broader hepatitis program as data gathered through clinical trials for INFRADURE in hepatitis D may serve as relevant support for other clinical uses of INFRADURE including hepatitis C and hepatitis B,” stated Andrew L. Pearlman, Ph.D., President and Chief Executive Officer of Medgenics. “Following on the FDA’s recent clearance to proceed with our Phase IIb study of the EPODURE™ Biopump technology to treat anemia, and the positive comments of the National Institutes of Health Recombinant DNA Advisory Committee, we see this Orphan Drug Designation from the FDA as another important step in the regulatory pathway for our Biopump platform. As Dr. Haffner has indicated, we are hopeful that this will lead to a streamlined regulatory pathway to approval of INFRADURE in hepatitis D and believe that it could also help advance the regulatory pathway for other applications of our Biopump technology in the U.S.”“We look forward to updating our clinical trial plans in view of the Orphan Drug Designation. We anticipate that hepatitis D clinical studies will use INFRADURE Biopumps that are fundamentally identical to those which will be used for the treatment of hepatitis C in our two Phase I/II studies in Israel which are now awaiting final approval from the Israeli Ministry of Health.” added Pearlman. About Hepatitis D According to the U.S. Centers for Disease Control and Prevention, hepatitis D, also known as "delta hepatitis," is a serious liver disease caused by infection with the hepatitis D virus (HDV), which is an RNA virus structurally unrelated to the hepatitis A, B or C viruses. Hepatitis D, which can be acute or chronic, is not common in the United States. HDV is an incomplete virus that requires the helper function of the hepatitis B virus (HBV) to replicate and only occurs among people who are infected with HBV. HDV is transmitted through percutaneous or mucosal contact with infectious blood and can be acquired either as a co-infection with HBV or as a super-infection in persons with HBV infection. There is no vaccine for hepatitis D, but it can be prevented in persons who are not already HBV-infected by administrative of the hepatitis B vaccination. Hepatitis D infects about 15 million people worldwide.
About MedgenicsMedgenics 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, and hemophilia, among others. 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:
- EPODURETM to produce and deliver erythropoietin for many months from a single administration, which has demonstrated elevation and stabilization of hemoglobin levels in anemic patients for periods of six months to more than 36 months in a Phase I/II dose-ranging trial in Israel and has received approval for a Phase IIa trial in dialysis patients due to launch in Q2 2012 in Israel. An Investigational New Drug application has been cleared by the FDA to initiate a Phase IIb study to evaluate the safety and efficacy of EPODURE in the treatment of anemia in dialysis patients in the U.S.
- INFRADURETM for sustained production and delivery of interferon-alpha for use in the treatment of hepatitis is awaiting final approval of the Israeli Ministry of Health of two Phase I/II trials in Israel in hepatitis C, slated to commence Q3 2012.
- HEMODURETM for sustained production and delivery of clotting Factor VIII therapy for the sustained prophylactic treatment of hemophilia is now in development.
Forward-looking StatementsThis 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.