Philadelphia, PA, Monday, March 19, 2012: Hemispherx Biopharma, Inc. (NYSE Amex: HEB) (the “Company” or “Hemispherx”) announced the publication of a peer-reviewed article providing the results from the AMP-516 Phase III Clinical Trial of Ampligen® [rintatolimod, Poly (I) • (C12,U)], an experimental therapeutic, in the high impact, online journal, PLoS ONE. The report is entitled “A Double-Blind, Placebo-Controlled, Randomized, Clinical Trial of the TLR-3 Agonist Rintatolimod in Severe Cases of Chronic Fatigue Syndrome”. Recently, researchers from the Centers for Disease Control and Prevention (“CDC”) and Harvard School of Public Health published new data showing the profound economic impact of CFS on increasing healthcare costs of $452 million and decreasing CFS patient productivity by $1.2 billion in Georgia, a state with approximately 5.5 million people age 18-59 (Cost Effectiveness and Resource Allocation, 9:1, 2011).
In the current PLoS One publication, a Phase III, FDA authorized study in CFS evaluated the safety and therapeutic effectiveness of Ampligen®, an experimental therapeutic, in 234 subjects with debilitating CFS at 12 clinical sites in the United States. The Ampligen® treatment was generally well-tolerated. The article can be found at http://dx.plos.org/10.1371/journal.pone.0031334.
The primary endpoint, exercise tolerance, improved an average of 21% in subjects receiving Ampligen® compared to placebo and the proportions of patients with exercise improvements of at least 25% and 50% were 1.7 and 1.9-fold greater for the Ampligen® group versus placebo (p<0.05). An ad hoc continuous responder analysis of exercise improvement between 25% and 50% at 5% increments demonstrated a significantly greater response for patients receiving Ampligen® compared to placebo.
The Ampligen® cohort also reduced dependence on medications used to reduce symptoms of CFS compared to the placebo group (p<0.05), adding additional insight to the recent CDC/Harvard study which emphasizes the overwhelming economic burden of medical care for CFS sufferers.
In 2010, Hemispherx published new data showing that a greater proportion of placebo patients in this Phase III trial were found to have a significant prolongation of the EKG QT interval compared to patients who received Ampligen®. Prolongation of the QT interval, which is a risk factor for sudden cardiac death and arrhythmias, was associated with continued use of certain drugs by CFS sufferers known to prolong the QT interval (Journal of Applied Research, 10:3, 2010). CFS patients are considered to be at increased risk for catastrophic cardiac events despite their relatively young age and the preponderance of women (approximately 2-3 women for each man) who suffer from this chronically debilitating disease.
On January 11, 2012, Hemispherx announced that the FDA had granted an extension of its pending New Drug Application (“NDA”) for potential treatment of CFS. The Company is currently conducting “open-label” treatment protocol in the U.S. and evaluating new diagnostic modalities to provide additional insights into the CFS disorder.
The FDA originally concluded (Complete Response Letter received 11/25/09) that this Phase III study was inadequate to support approval of the NDA. However, the new analyses and other insights in the PLoS One report supplement the original study findings. The Company believes that continued efforts to understand existing data and to advance the development of new data and information, will ultimately support a re-filing of the NDA.
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® (FDA approved for a category of sexually transmitted diseases) 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. Hemispherx’s platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Hemispherx has patents comprising its core intellectual property estate and a fully commercialized product (Alferon N Injection®). 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.
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 investigational drugs and associated technologies of the Company (including Ampligen® and Alferon® LDO) 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. The planning, completion, results or submission of clinical trials do not imply that any study product will ever be approved commercially for the studied or other treatment indications.