|May 03, 2004
|HEMISPHERX BIOPHARMA ANNOUNCES PHASE 3 CHRONIC FATIGUE SYNDROME TRIAL MEETS PRIMARY ENDPOINT
|Patients Receiving Ampligen Improve at Twice the Level Considered Medically Significant
Philadelphia, PA, Monday, May 03, 2004: Hemispherx Biopharma, Inc. (AMEX: HEB) announced today that its pivotal Phase III Chronic Fatigue Syndrome (CFS) trial of Ampligen met its primary endpoint with patients receiving Ampligen for 40 weeks. A statistically significant increase (p=0.022) in patients’ physical performance as measured by Treadmill Exercise Tolerance Testing (ETT) was found, compared to placebo. Ampligen represents one of a new class of drugs called dsRNA drugs for the potential treatment of CFS that afflict more than 500,000 people in the United States. Following peer review, the Ampligen study will be presented today at the 17th International Conference on Antiviral Research, held in Tucson, Arizona, in Oral Session II.
“We are very pleased to report these new results from the pivotal CFS trial with Ampligen. This study is particularly important because CFS is currently only treated symptomatically as there is as yet no FDA approved therapy,” said Dr. William A. Carter, Chief Executive Officer of Hemispherx Biopharma. “With this data, we now focus on the preparation of new drug applications nationally and internationally. We expect that these results will generate significant interest from the pharmaceutical industry.”
The reported new 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 (≥6.5%) in mean exercise duration (baseline compared to week 40) that is statistically significant (p≤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 FDA’s Antiviral Advisory Committee, and the case definition of CFS was developed and recommended by the U.S. Center for Disease Control and Prevention (CDC).
Patients receiving Ampligen for 40 weeks improved exercise treadmill performance 19.4% vs. 5.1% in the placebo group (p=0.022, analysis of covariance with baseline as covariate). An “intent to treat” analysis included patients who completed less than 40 weeks. Similar results were obtained with 17.7% improvement in exercise treadmill duration in the Ampligen cohort compared to 4.3% in patients receiving the placebo (p=0.047). Improvements in exercise treadmill duration in the patients receiving Ampligen compared to placebo were greater than 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 vs. those receiving placebo, suggesting that the experimental drug was generally well tolerated.
About Chronic Fatigue Syndrome
CFS is defined by various governmental agencies worldwide as a serious debilitating disease in which patients suffer from complex symptoms, including primarily disabling fatigue, associated with flu-like symptoms, headaches, sore throat, joint and muscle pain, mental fogginess, depression and sleep disorders. 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 an equal number in Europe.
Ampligen is an experimental, specifically-configured, double stranded RNA drug acting potentially as an immunomodulator and antiviral. Based on in vitro studies, Ampligen is believed to modulate the immune system as well as inhibit the virus directly. 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 the potential treatment of HIV/AIDS. 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.
Hemispherx Biopharma, based in Philadelphia, is a biopharmaceutical company engaged in the manufacture and clinical development of new drug entities for treatment of viral and immune-based chronic disorders. Its flagship products include Alferon N® and the experimental immunotherapeutics/antivirals Ampligen® and Oragens™. These novel proteins, commercially available for a category of STD infection, and experimental nucleic acids are being developed for globally important chronic viral 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®) and GMP certified manufacturing facilities for its novel pharma products. For more information please visit www.hemispherx.net
Hemispherx Biopharma, Inc.,
Dianne Will, Investor Relations
(518) 398-6222, Fax: (518) 398-6369
HEB's Web Site: 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 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. 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 do not imply that the product will ever be specifically approved commercially for these other treatment indications.