Hemispherx Biopharma Receives New Composition of Matter Patent

Hemispherx Biopharma, Inc. recently announced it received formal notice on September 21, 2015, that the European Patent Office’s Examining Division granted the Company’s application titled “Double-Stranded Ribonucleic Acids with Rugged Physiochemical Structure and Highly Specific Biologic Activity” by inventors Carter, et al. and assignee Hemispherx Biopharma, Inc. The patent claims a novel form of rugged dsRNA. Rugged dsRNA are nucleic acids with a unique composition and physical characteristic identified with high specificity of binding to Toll-Like Receptor 3 (TLR3), thereby conveying an important range of therapeutic opportunities. This form of dsRNA has increased bioactivity and binding affinity to the TLR 3 receptor because of its reduced tendency to form branched dsRNA which can inhibit receptor binding.
Pharmaceutical formulations containing this nucleic acid as active ingredients, and methods of treatment with those formulations, are also described in the granted patent.

Hemispherx’s President, Thomas K. Equels, said “We are pleased to announce this significant step in our business plan for Ampligen. Success at this critical stage is important for implementation of our global program for Ampligen and, while formal approval by the member nations is still pending, this formal notification by the EPO of the granting of the composition of matter patent is an essential step in that approval process.”

With this important milestone achieved, the patent will enter a national validation phase taking several months that may include review and approval by up to approximately 25 countries. A similar application (U.S. patent 8,722,874) was granted in the US on May 13, 2014.

An earlier US composition of matter patent on Ampligen, an experimental biotherapeutic, was issued to Johns Hopkins University with results published in the Journal of Molecular Biology, and thereafter licensed to Hemispherx Biopharma, Inc. exclusively. Upon expiration of the original patent, Hemispherx relied on a continued research program and sizable portfolio of subsequently issued related patents to maintain a degree of proprietary protection for novel compositions and treatment methods based on Ampligen technology.

With issuance of US Patent 8,722,874, and this significant progress with the EPO, Hemispherx’s planned patent protection for novel formulations of Ampligen products may extend through 2029 and potentially beyond.

Patent extension is a possibility in the US because of patent law provisions (commonly known as the Hatch-Waxman Amendments), which permit patent extensions in certain cases to partially compensate for patent life lost due to a lengthy clinical drug development cycle.

Hemispherx is presently engaged in multiple clinical trials in the US related to Chronic Fatigue Syndrome (CFS) (AMP-511), enhancement of immune responses to influenza vaccines, and various immunotherapies for human cancers. Similar clinical studies are projected for the European Union and Australia under various collaborative agreements.

Hemispherx has identified an immune marker, low NK cell activity, which is associated with increased CFS symptom severity and responds favorably to Ampligen treatment in vitro. On September 15, 2015, Hemispherx announced the publication of a peer reviewed article in the Journal of Clinical and Cellular Immunology (Strayer D, et al, 2015;6:4 http://doi.org/10.4172/2155-9899.1000348) entitled “Low NK Cell Activity in Chronic Fatigue Syndrome (CFS) and Relationship to Symptom Severity” in which 17 studies were reviewed that evaluated NK cell cytotoxicity (NKCC) data and the relationship to different CFS case definitions and CFS disease severity. NK cells are an important component of the human immune response acting as a surveillance mechanism against invading pathogens and tumor cells. The review includes evidence that there is an association between decreased NK cell activity and increased CFS symptomatology. New data were reported in this publication, of in vitro exposure of peripheral blood mononuclear cells (PBMCs) from 15 CFS patients. These data indicated than in vitro exposure to Ampligen(R), an experimental biotherapeutic, produced a 178% increase in mean NK activity (NKCC) and a 100% increase in median NK activity (NKCC).

For cancers that were previously, largely untreatable, Hemispherx also has identified potential therapeutic regimens that combine Ampligen with newly proven immunotherapeutics with the goal of increasing overall effectiveness and survival. These new immunotherapeutics are also called “check-point inhibitors” because they unleash the human immune apparatus to accelerate the killing of human tumor cells.

On September 23, 2015, Hemispherx announced another new peer-reviewed article with senior authors from the Italian Ministry of Health and other related agencies published in Clinical and Cellular Immunology: 2015;6:4 entitled, “A Toll-Like Receptor 3 – Agonist as Promising Candidate in Multiple Sclerosis Treatment.” As stated in the Abstract of the article by the Italian authors: “The aim of this paper is to provide a brief overview of Ampligen(R) historical development, clinical pharmacology, clinical trials, and safety data and to discuss about its potential role in MS treatment in the context of existing treatment options.” In the article, the authors observe that Ampligen, an experimental therapeutic, unlike all other toll-like receptors (TLRs) studied to date, has a unique immune mechanism making it potentially adaptable as a treatment for many different disorders including multiple sclerosis (MS). They further state: “another winning point of TLR-targeting drugs is that they have fewer side effects and lower or no toxicity compared to other drugs used in MS treatment.”

The recently issued US patent, and its counterpart in the EU, indicate how dsRNAs acting through TLR3 receptor activation may prove to be potent antiviral compounds and anticancer agents and, through secondary immunomodulators, have the potential to enhance the bioactivity of vaccines and treat autoimmune diseases.

The significant extension of proprietary longevity via the new composition-of-matter patent in the US and the application now accepted by the EU is expected to favorably affect patent longevity of Ampligen in potentially up to an additional 25 countries.

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.

Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the clinical development of new drug entities for treatment of seriously debilitating disorders. 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. 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 US 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(R) N 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 US for commercial products. For more information, visit www.hemispherx.net.