PHILADELPHIA, March 24, 2015 (GLOBE NEWSWIRE) -- Hemispherx Biopharma, Inc. (NYSE MKT:HEB) (the "Company" or "Hemispherx") announces that the European subsidiary, "Hemispherx Biopharma Europe N.V./S.A" gained a positive opinion from the COMP (Committee on Medical Products) regarding its Orphan Medicinal Product Application for Ampligen®, an experimental therapeutic, to treat Ebola Virus Disease (EVD).
Orphan designation by the European Medicines Agency (EMA) is granted to promote the clinical development of drugs that target rare life-threatening conditions and which are expected to provide significant therapeutic advantage over any existing treatments and includes some tropical diseases primarily found in developing nations as is the case for EVD. Hemispherx submitted in vitro and in vivo data in appropriate preclinical models relevant to the EVD indication to the European Union (EU) Committee for Orphan Medicinal Products. In addition to the efficacy data, clinical safety information was also included in the comprehensive application.
The dossier submitted to the EU contained an inventory of relevant pre-clinical research in EVD therapeutically-oriented models, as well as, clinical safety information derived from non-EVD clinical studies of Ampligen®. Of potentially high relevance to the favorable COMP opinion were experiments, conducted in Italy, which showed that, in vitro, Ampligen® successfully competed with Ebola viral dsRNA in a manner which may result in loss of virulence by EBOV. Basing experimental design on an earlier report (Prins, et al. J Virol 2010;84(6):3004), independent researchers showed that Ampligen®, an experimental therapeutic, successfully competed with dsRNA for Ebola VP35 binding with a remarkably low Ampligen® concentration reflected by an IC50=1.1 µg/ml. The Italian researchers received a grant-in-aid from Hemispherx Biopharma for the conduct of these experiments.
There are significant benefits for achieving Orphan Designation, including eligibility for grants from EU and Member State programs as well as initiatives supporting research and development encompassing clinical protocol design assistance. Designated orphan medicines are assessed for marketing authorization centrally in the European Union with reductions/waivers in the fees and costs of the overall regulatory process. The designation allows companies to make a single application to the European Medicines Agency, resulting in a single opinion and a single decision from the European Commission, valid in all EU Member States. Authorized orphan medications, once commercially approved, receive benefits including ten years of complete protection from market competition with similar medicines. This period of protection from competition is extended by two years for medicines that also have complied with an agreed pediatric investigation plan. Sponsors may also have access via orphan designation to conditional approval, which is also conducted under the centralized procedure.
The EU Orphan application process consists of multiple steps and a final decision from the European Commission normally occurs sometime after the summary report of the COMP. No assurances can be given that the final decision will designate Ampligen as an Orphan Medical Product for treatment of EVD.
About Ampligen®
Ampligen®, an experimental therapeutic, is a new class of specifically-configured ribonucleic acid (RNA) compounds targeted as potential treatment of diseases with immunologic defects and/or viral causation. Ebola virus specifically inhibits the dsRNA within cells via a sequestration process. Such RNA would otherwise cause a robust antiviral response to be mounted: Ampligen® may be able to overcome this deficiency in host response. Positive results against Ebola in vitro, and/or in an animal model, have been reported to the Company by the United States Army Medical Research Institute of Infectious Diseases (USAMRIID) and other research/academic institutions in the US and abroad. Clinical protocol development is being pursued. Clinical trial data will be necessary to establish human efficacy, if any, of Ampligen® for Ebola viruses.
About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the manufacture and clinical development of new drug entities ("biological modifiers") for treatment of seriously debilitating disorders especially life-threatening viruses. Hemispherx's flagship products include Alferon® N Injection® and the experimental therapeutics Ampligen® and Alferon® LDO. Ampligen® is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system, including Chronic Fatigue Syndrome. Hemispherx's platform technology includes components for potential treatment of various severely debilitating and life threatening diseases including cancers. Because both Ampligen® and Alferon® LDO are experimental in nature, they are not designated safe and effective by a regulatory authority 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. The Company's Alferon N Injection® approval in Argentina includes the use of Alferon N Injection® (under the brand name "Naturaferon") for use in any patients who fail, or become intolerant to recombinant interferon, including patients with chronic active hepatitis C infection. The Company 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.
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 Chronic Fatigue Syndrome in the United States. The production of new Alferon® API inventory commenced in February 2015. While the facility is approved by the FDA under the Biological License Application ("BLA") for Alferon®, this status will need to be reaffirmed by an FDA pre-approval inspection. We will also need FDA's approval to release commercial product once we have submitted satisfactory stability and quality release data. 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. 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 an Ampligen® NDA in the United States and other countries. Any failure to satisfy the FDA regulatory requirements or the requirements of other countries could significantly delay, or preclude outright, approval of an 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. For instance, the strategies and operations of Hemispherx involve risk of competition, changing market conditions, changes in laws and regulations affecting these industries and numerous other factors discussed in this release and in the Company's filings with the Securities and Exchange Commission. The final results of these efforts could vary materially from Hemispherx's expectations. Findings from animal studies may not correlate with results from humans. Clinical trial data will be necessary to establish human efficacy of Ampligen® or Alferon® for Ebola virus disease.
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," 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, above, 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. No evidence is suggested that Alferon® N, Alferon® LDO and/or Ampligen will ever be commercially approved for the new potential treatment indications mentioned in this release.