Hemispherx Biopharma Repurposes Ampligen in Immuno-Oncology

PHILADELPHIA, May 31, 2017 (GLOBE NEWSWIRE) — Hemispherx Biopharma (NYSE MKT:HEB) (“Hemispherx” or the “Company”) announced today that it continues to bolster its scientific capabilities by expanding the role of Joseph C. Horvath, M.D., to Deputy Chief Medical Officer.

In this newly-created position, Dr. Horvath will assist the Company on various scientific aspects of development, manufacturing, regulatory, and medical affairs with a focus on assisting with the oversight of the current Ampligen Early Access Program (EAP) in place for pancreatic cancer.

The Ampligen pancreatic cancer EAP was recently extended by the Netherlands, establishing a treatment program for up to 50 pancreatic cancer patients and creating a funding mechanism to compensate Hemispherx for the use of Ampligen® in the program. Efforts are underway to further expand the program in the Netherlands as well as commence similar therapeutic cancer programs in other major European countries that allow for early access with full governmental reimbursement.

We are repurposing Ampligen® as a broadly indicated immuno-oncology agent. By repurposing the experimental drug from a sole focus on Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), we take full advantage of a fully developed safety profile that emerged from the approximately 100,000 doses utilized in prior clinical studies showing that Ampligen was generally well-tolerated. No other TLR agonist comes into the Cancer space with such a strong and positive safety profile,” said Thomas Equels, CEO of Hemispherx Biopharma.

Our belief in Ampligen’s potential as a therapy in oncology is based not only on its direct antitumor cell activity, but also on its immuno-modulatory activities, which may be important in providing a favorable tumor microenvironment for therapy,” Equels added.  “Dr. Horvath’s vast experience in both oncology and microbiology will help us in achieving our near term goal of repurposing Ampligen into a serious player in immune-oncology.  We are deeply committed to providing a therapeutic solution to the large unmet medical need in pancreatic cancer, which is one of the leading causes of cancer deaths in Europe as research shows.

Dr. Horvath has considerable experience in immuno-oncology research, which is anticipated to be especially helpful in the Company’s expanding efforts in pancreatic cancer programs and the on-going work in ME/CFS.  His experience is expected to ensure the continuation of successful organization and planning of upcoming clinical studies in the immuno-oncology space.

Dr. Horvath stated, “I am professionally excited with this opportunity to be at the leading edge of developing Ampligen as a standalone and combination therapy in immuno-oncology. Ampligen has been shown to be generally well-tolerated in multiple clinical trials in the US and Europe.  This safety record, in combination with Ampligen’s Toll-like Receptor 3 mechanism activating both innate and adaptive immunity, requires that our team at Hemispherx work hard to develop Ampligen as an immuno-stimulant to combat those forms of cancer, such as pancreatic, where a relation between a favorable prognosis and a positive immune profile has been reported. A body of clinical research data suggests that Ampligen favorably changes these immune parameters in another GI solid tumor, colorectal carcinoma.

Dr. Horvath has more than 40 years of experience in biomedical research in the fields of virology, immunology and molecular biology.  He obtained a doctorate in medicine (1971) and board certification in clinical pathology (1976) and virology (1980) from Semmelweis University Medical School in Budapest, Hungary.

Dr. Horvath was a postdoctoral fellow, then a research professor at the Department of Microbiology at the University of Sherbrooke in Canada where he conducted studies regarding the effects of viral and cellular oncogenes and anti-oncogenes in carcinogenesis. He served as Hemispherx’ Director of Research and Development for seven years.  He also served as Scientific Director of the Biotherapy Research Laboratories at St. Joseph Hospital’s Cancer Institute in Tampa, FL. 

A chart accompanying this release is available at http://www.globenewswire.com/NewsRoom/AttachmentNg/a9bf8a40-2c23-4454-81e9-1dee949d68b9

According to United European Gastroenterology (UEG), a professional nonprofit organization focusing on digestive diseases, the death rate from pancreatic cancer in Europe is projected to increase to 112,000 deaths per year. In the US pancreatic cancer is projected to increase in both incidence and cancer related deaths over the next decade, while the other major cancer killers are projected to have declining death rates (https://www.pancan.org/wp-content/uploads/2013/01/incidence_report_2012.pdf).  The overall survival rate from pancreatic cancer at five years is only 6%, therefore making it a largely unmet medical need.

About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the manufacture and clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx’s flagship products include Alferon N Injection® and the experimental therapeutics rintatolimod (tradenames Ampligen® or Rintamod®) and Alferon® LDO. Rintatolimod is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system, including Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). Hemispherx’s platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Because both rintatolimod and Alferon® LDO are experimental in nature, they are not designated safe and effective by the FDA for general use and are legally available only through clinical trials. Hemispherx has patents comprising its core intellectual property estate and a fully commercialized product (Alferon N Injection®), approved for sale in the U.S. and Argentina. The FDA approval of Alferon N Injection® is limited to the treatment of refractory or recurrent external genital warts in patients 18 years of age or older. Our Alferon N® approval in Argentina includes the use of Alferon N Injection® (under the pending brand name “Naturaferon”) for use in any patients who fail or become intolerant to recombinant interferon, including patients with chronic active hepatitis C infection. Rintamod® has received approval in the Argentine Republic to treat severe cases of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS). It will be marketed by GP Pharm, Hemispherx’s commercial partner in Latin America. Hemispherx wholly owns and exclusively operates a GMP certified manufacturing facility in the United States for commercial products. For more information please visit www.hemispherx.net.

Forward-Looking Statements
To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as “intends,” “plans,” “potential,” “potentially,” “possible,” and similar expressions are intended to identify forward-looking statements. The inclusion of forward-looking statements should not be regarded as a representation by Hemispherx that any of its plans will be achieved. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond Hemispherx’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. Examples of such risks and uncertainties include those set forth in the Disclosure Notice, below, market and other conditions, the impact of general economic, industry or political conditions in the United States or internationally, as well as the risks described in Hemispherx’s filings with the Securities and Exchange Commission, including the most recent reports on Forms 10-K, 10-Q and 8-K. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Hemispherx undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise revise or update this release to reflect events or circumstances after the date hereof.

Disclosure Notice
The information in this press release includes certain “forward-looking” statements including without limitation statements about additional steps which the FDA may require and Hemispherx may take in continuing to seek commercial approval of the Ampligen® NDA for the treatment of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) in the United States. The final results of these and other ongoing activities could vary materially from Hemispherx’s expectations and could adversely affect the chances for approval of the Ampligen® NDA in the United States and other countries. The clinical studies referenced herein have been previously reviewed by the FDA and are not, in and of themselves, a sufficient basis for approval in the United States. Any failure to satisfy the FDA regulatory requirements or the requirements of other countries could significantly delay, or preclude outright, approval of the Ampligen® NDA in the United States and other countries.

Information contained in this news release, other than historical information, should be considered forward-looking and is subject to various risk factors and uncertainties including, but not limited to, general industry conditions and competition; general economic factors; our ability to adequately fund our projects; the impact of pharmaceutical industry regulation and healthcare legislation in the United States and internationally; trends toward healthcare cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; our ability to accurately predict the future market conditions; manufacturing difficulties or delays; dependence on the effectiveness of our patents and other protections for products; and the exposure to litigation, including patent litigation, and/or regulatory actions; and numerous other factors discussed in this release and in our filings with the Securities and Exchange Commission. The production of new Alferon® API inventory will not commence until the validation phase is complete. While the facility is approved by FDA under the Biological License Application (“BLA”) for Alferon®, this status will need to be reaffirmed by a successful Pre-Approval Inspection by the FDA prior to commercial sale of newly produced inventory product. The validation phase was delayed because of the damage caused by the flood that occurred in January 2016 at the facility. At this time, we believe that all repairs to the manufacturing facility have been completed. If and when we obtain a reaffirmation of FDA BLA status and have begun production of new Alferon® API, we will need FDA approval as to the quality and stability of the final product to allow commercial sales to resume. With regard to our NDA for Ampligen® to treat CFS, we note that there are additional steps which the FDA has advised us to take in our seeking approval. The final results of these efforts and/or any other activities could vary materially from our expectations. Any failure to satisfy the FDA regulatory requirements or the requirements of other countries could significantly delay, or preclude outright, approval of Ampligen® in the United States and other countries. No evidence is suggested that Ampligen® will be commercially approved for any treatment or that Alferon N Injection® will be commercially approved for potential new treatment indications or for new manufacturing procedures. Approval of Rintamod® for CFS in the Argentine Republic does not in any way suggest that the Ampligen® NDA in the United States will obtain commercial approval. Also, it is noted that ANMAT approval is only an initial, but important, step in the overall successful commercialization. Namely, additional steps required for commercialization in Argentina will require, among others, an appropriate reimbursement level, appropriate marketing strategies, completion of manufacturing preparations for launch (including possible requirements for approval of final manufacturing, etc., and there are no assurances as to whether or when such multiple subsequent steps will be successfully performed to result in an overall successful commercialization and product launch).  Expansion of the Early Access Program with myTomorrows into pancreatic cancer is no assurance of commercial approval in the US or elsewhere.

CONTACT: Company/Investor Contact:
Charles Jones
CJones & Associates Public Relations