April 23, 2012

Medgenics Files for U.S. Orphan Drug Designation for INFRADURE for the Treatment of Hepatitis D



April 23, 2012, 10:00 a.m. EDT

Designation could open pathway to expedited approval of platform protein delivery technology and additional market exclusivity

MISGAV, Israel & SAN FRANCISCO, Apr 23, 2012 (BUSINESS WIRE) -- Medgenics, Inc., 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 for Orphan Drug Designation with the U.S. Food and Drug Administration (FDA) for INFRADURE(TM) for the treatment of hepatitis D. INFRADURE is based on Medgenics' proprietary tissue-based Biopump(TM) platform technology, which uses the patient's own tissue to continuously produce and deliver therapeutic proteins, such as interferon-alpha for use in the treatment of hepatitis.

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.

This application for Orphan Drug Designation follows Medgenics' recent submission of an Investigational New Drug (IND) application to the FDA for a Phase IIb anemia trial in dialysis patients using EPODURE(TM), a different implementation of the same Biopump platform that produces erythropoietin (EPO).

Marlene Haffner, M.D. MPH, former Director of Orphan Products Development at the FDA, and regulatory advisor to Medgenics said, "INFRADURE's application for the treatment of hepatitis D appears to meet the key criteria required for Orphan Drug Designation by the FDA, as the number of U.S. patients with this disease is estimated to be considerably fewer than 200,000. Furthermore, preclinical data support that INFRADURE has a reasonable rationale for treatment of the disease based on its potential ability to continuously delivery interferon alpha, the current standard of care for hepatitis D that is administered by years of repeat injections. Should Orphan Drug Designation be granted, the regulatory approval route for INFRADURE for the treatment of hepatitis D could be significantly expedited."

This Orphan Drug Designation filing marks the first application for Medgenics' Biopump technology seeking an expedited regulatory pathway in the U.S. Obtaining Orphan Drug Designation could potentially allow for substantially smaller pivotal clinical trials, as compared to a more wide-spread disease. This could lead to more immediate access of the treatment to a patient population in need. Medgenics expects to receive the FDA's initial response to the filing before the end of this quarter, and believes Orphan Designation could be confirmed during the third quarter.

Bruce Bacon, M.D., past President of the American Association for the Study of Liver Disease and a recognized global expert in hepatitis who serves on Medgenics' Strategic Advisory Board, commented, "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. 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. Medgenics' treatment is potentially a game changer not only as a treatment for hepatitis D, but also as a key element in the treatment of various other forms of hepatitis worldwide."

"Our application for Orphan Drug Designation for hepatitis D demonstrates Medgenics' commitment to the treatment of hepatitis. Obtaining Orphan Drug Designation could be the most rapid route for us to bring our Biopump technology to the U.S.," stated Andrew L. Pearlman, Ph.D., President and Chief Executive Officer of Medgenics. "We recently submitted an IND application with the FDA for a Phase IIb anemia trial for EPODURE, a version of the Biopump which produces a different protein, EPO, and have received approval in Israel to commence a Phase IIa trial for that same indication. We are very encouraged with the momentum we are building in 2012 as these important clinical advancements reflect the progress that various applications of our platform technology are making through regulatory approval processes as we continue to build shareholder value."

"We are eager to pursue this niche opportunity in hepatitis D, which would use a similar INFRADURE approach to that we will employ in treating hepatitis C in a Phase I/II study in Israel that is scheduled to commence in the third quarter of 2012 pending final regulatory approval. Receiving Orphan Drug Designation for our INFRADURE Biopump in the treatment of hepatitis D could lead not only to an expedited approval route based on clinical studies of moderate size for this specific rare indication, but could also represent a significant advancement of our entire portfolio of treatments. Such an approval may serve to pave the way for a more rapid pace at which our platform protein delivery technology can move through the FDA approval process for other multibillion-dollar clinical indications," added Dr. 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 Medgenics

Medgenics is developing and commercializing Biopump(TM), 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:

-- EPODURE(TM) 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, and is about to commence a Phase IIa safety/efficacy trial in dialysis patients in Q2 2012 in Israel. An IND has been filed with the U.S. Food and Drug Administration 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.

-- INFRADURE(TM) for sustained production and delivery of interferon-alpha for use in the treatment of hepatitis is awaiting final approval of two Phase I/II trials in Israel in hepatitis C, slated to commence Q3 2012.

-- HEMODURE(TM) for sustained production and delivery of clotting Factor VIII therapy for the sustained prophylactic treatment of hemophilia is now in development.

Medgenics is focused on the development and manufacturing of its innovative Biopumps, aiming to 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.

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.

SOURCE: Medgenics, Inc.


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