The new peer-reviewed study shows conclusively that the biologic properties of Ampligen(R) are conveyed exclusively by a unique naturally occurring receptor on the cell surface called "TLR-3." Toll-like receptors or "TLRs" such as TLR-3 serve as "pattern recognition" receptors in the early detection of pathogens and the establishment of early defense mechanisms (innate immunity). As such, they are critical to the first line of immunological defense against a broad range of pathogens, such as otherwise lethal viruses and even various forms of cancer.
When the dormant alarm signals of TLRs are activated (as by exposure to a pathogen or a stimulant agent such as Ampligen(R)), TLRs in effect cause an overreaction, driving the body to proliferate broad-spectrum defenses against many types of pathogens.
New research shows that these receptors present in virtually all life forms, from mosquitoes to man (in scientific terms, they are remarkably "evolutionarily conserved"). This suggests that TLRs are critical to retaining healthy life in a constantly changing environment full of hostile pathogens, many of which the subject has never encountered before.
The study was conducted in TLR-3 "knockout" animals at Utah State University under a NIH contract and with the aid of a variety of collaborators representing a consortium of institutions (Yale University, Vanderbilt University, and the Centre d'Immunologie de Marseille-Luminy), to determine if TLR-3 is essential for the activities of dsRNAs for the induction of innate immunity. Unlike the toxic Poly I:Poly C, Ampligen is directed towards the activation of TLR-3 in the induction of innate immune responses.
The closely allied compound, Poly I : Poly C, has been used in clinical trials but has a broad range of side-effects, some of which are life-threatening. Where the studies found specific involvement of TLR-3 in conveying Ampligen(R)'s biologic properties, no such specificity was seen with poly I : poly C, thus explaining its side effect profile without compromising its potency as an immunotherapeutic.
The findings further establish the remarkable specificity of Ampligen(R) in activating the critical first line of defense against various types of pathogenic elements. The findings are pertinent to many cutting edge therapeutic topics in biotechnology today, including the enhancement of vaccine efficacy (both antiviral and anticancer) as well as strengthening biologic therapy (interferons, interleukins, etc.) of various serious human diseases as well as enhancing traditional antivirals such as Tamiflu(R) and Relenza(R).
The experimental therapeutic Ampligen(R) is perhaps the best studied TLR activator in human clinical trials to date, having been administered more than 75,000 times in approximately 15 clinical trials of which four were very well-controlled (placebo-controlled, randomized, multi-center, double-blinded).
"These scientific findings go a long way toward explaining how a single, relatively simple compound, Ampligen(R), can repeatedly show therapeutic promise in such a broad array of indications. The findings may also have broad economic implications for the American biotechnology sector," commented Dr. William A. Carter, Chairman and CEO of Hemispherx Biopharama, Inc. "According to a recent KPMG Bioscience report, our sector recently achieved a new high of $70 billion in revenues a year, but is now faced with many very short product life cycles, large po rtfolios of expiring patents and the corresponding potential for global generic competition. However, specific TLR promoters, such as Ampligen(R), may have the potential to increase the efficacy of a broadly diverse set of previously licensed biological products, thus effectively extending their useful commercial life cycles." Hemispherx has a set of patent applications pending worldwide related to safely enhancing the therapeutic responses of biological products.
About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is a biopharmaceutical 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(R) and the experimental therapeutics Ampligen(R) and Oragens(R). Alferon N Injection(R) is approved for a category of STD infection, and Ampligen(R) and Oragens(R) represent experimental RNA nucleic acids being developed for globally important viral diseases and disorders of the immune system. Hemispherx's platform technology includes large and small agent components for potential treatment of various severely debilitating and life threatening diseases. Hemispherx has in excess of 100 patents comprising its core intellectual property estate, a fully commercialized product (Alferon N Injection(R)) and GMP certified manufacturing facilities for its novel pharma products. For more information please visit www.hemispherx.net
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, change 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. Any specifically referenced investigat ional drugs and associated technologies of the Company (including Ampligen(R), Alferon LDO and Oragens) are experimental in nature and as such are not designated safe and effective by a regulatory authority for general use and are legally available only through clinical trials with the referenced disorders. The forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements. Clinical trials for other potential indications of the approved biologic Alferon N Injection(R) do not imply that the product will ever be specifically approved commercially for these other treatment indications.
NOTE TO EDITORS: In the expression Poly C(12)U, which appears in the first paragraph, the "12" is a subscript. This placement may not appear properly in some systems.
Hemispherx Biopharma, Inc.
Dianne Will, 518-398-6222
CCG Investor Relations
Sean Collins, Sr. Partner, 310-477-9800