Hemispherx Biopharma Presents Newphase III Data on Ampligen at the 7th International Conference for Chronic Fatigue Syndrome 7th International Conference for CFS PHILADELPHIA–(BUSINESS WIRE)–Oct. 11, 2004-
– Treadmill Improvement Data Exceeds Initial Indications; Patients Receiving Ampligen Perform at Twice the Level Historically Considered Medically Significant In Chronic Diseases Hemispherx Biopharma, Inc. (AMEX: HEB) announced today that Dr. David Strayer, Hemispherx Biopharma's Chief Scientific Officer, presented clinical data on the company's pivotal randomized, double-blinded, Phase III clinical trial of Ampligen(R) vs. placebo in Chronic Fatigue Syndrome (CFS) at the 7th International Conference for CFS held in Madison, Wisconsin on October 8-10, 2004.
In his oral presentation, Dr. Strayer gave commentary on "intent to treat" analysis, which included patients who completed less than 40 weeks of the study, which showed that patients receiving Ampligen(R) improved exercise treadmill performance 19.3% vs. 4.1% in the placebo group (p=0.037, analysis of covariance with baseline as covariate). The difference in improvement in exercise treadmill duration in the Ampligen cohort compared to placebo was 15.2% and over twice the minimum considered medically significant (6.5%). Moreover, there was no significant difference in the number of serious adverse events, missed dosages or dropouts (i.e., leaving the study prematurely) among patients receiving Ampligen versus those receiving placebo, suggesting that the experimental drug was generally well tolerated.
"We are very pleased to report unblinded information from the pivotal CFS trial with Ampligen(R). After additional analysis of the treadmill data over the course of the last few months, the treadmill improvement data actually further increased over what we previously thought. This study is particularly important because CFS is currently only managed symptomatically as there is as yet no recognized approved therapy for this major disease category," said Dr. William A. Carter, Chief Executive Officer of Hemispherx Biopharma. There has been a growing focus recently on developing a treatment for CFS. A number of drugs have been tested, and reported upon. Results from a clinical trail using Reminyl, a drug used for Alzheimer's disease, found no evidence that Reminyl, at any dose, was better than placebo at improving CFS symptoms. In addition, the Agency for Healthcare Research and Quality (AHRQ), a governmental Agency, reported on a systematic review of the scientific literature on treatment of CFS. In its report they noted that in four trials using corticosteroids, the evidence was insufficient to make a conclusion about effectiveness. Five clinical trials using antidepressants showed no consistent pattern of improvement. The AHRQ however did report that out of eleven clinical trials using immunological therapy, "Ampligen(R), an investigational drug…yielded the most promising results." (www.ahrq.gov/clinic/epcsums/cfssum.htm)
Study Design The reported clinical study is a well controlled, multi-center, double-blind, randomized, placebo-controlled Phase III pivotal study of the efficacy and safety of the experimental agent Ampligen, given 400 mg twice weekly, versus placebo in patients with severely debilitating CFS. The clinical trial randomized 234 patients at 12 centers across the U.S. to assess the effects of 40 weeks of treatment with Ampligen in patients suffering from CFS. The prospectively defined primary endpoint was improved physical performance as measured by Treadmill Exercise Tolerance Testing (ETT): Duration. Efficacy may be established by showing a medically significant increase (greater than or equal to 6.5%) in mean exercise duration (baseline compared to week 40) that is statistically significant (p less than or equal to 0.05) using a statistical method termed "analysis of covariance." The Ampligen clinical study design was previously reviewed by a specially convened panel of the Advisory Committee, and the case definition of CFS was developed and recommended by the U.S. Center for Disease Control and Prevention (CDC).
About Chronic Fatigue Syndrome CFS is defined by various governmental agencies worldwide as a serious debilitating disease in which patients suffer from flu-like symptoms, including primarily disabling fatigue. The CDC has added CFS to its top priority list of emerging infectious diseases. CFS is a worldwide disease estimated to affect over 500,000 people in the U.S., and a similar number in Europe.
About Ampligen Ampligen is an experimental, specifically-configured, double stranded RNA drug. The experimental product is currently in a phase 3 clinical trial for the potential treatment of CFS and in two Phase 2b clinical trials for other chronic debilitating diseases. The FDA has already granted Ampligen Treatment IND Status and Orphan Drug Status. Treatment IND status was granted after an earlier reported study of 24 weeks duration consisting of 94 patients with severe CFS randomized to receive either Ampligen or placebo. Only governmental regulatory agencies can make a definitive determination that the presumed efficacy / safety ratio of any new drug supports its commercial introduction for any disorder, including potential CFS treatment.
About Hemispherx Hemispherx Biopharma, based in Philadelphia, is a biopharmaceutical company engaged in the manufacture and clinical development of new drug entities. Its flagship products include Alferon N(R) and the experimental antivirals products, Ampligen(R) and Oragens(TM). These novel Alferon-N proteins, commercially available for a category of STD infection, and experimental nucleic acids are being developed for globally important chronic diseases and disorders of the immune system including HPV, HIV, CFS and Hepatitis. Its four major technology platforms include large-and small-agent components for potential treatment of various chronic viral infections, and are being developed with various corporate, governmental and academic collaborators worldwide. Hemispherx has approximately 400 patents comprising its core intellectual property estate, a fully commercialized product (Alferon N(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 investigational drugs and associated technologies of the company (including Ampligen(R) and Oragens(TM)) 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. Only Clinical Studies under well-controlled conditions can establish efficacy and safety of any product. Clinical trials for other potential indications of the approved biologic Alferon(R) do not imply that the product will ever be specifically approved commercially for these other treatment indications.