FDA Authorizes Expansion of Ampligen/FluMist Intranasal Clinical Trial

Mon, 12/09/2013 - 8:48am

Hemispherx Biopharma today announced that its Phase 1/2 clinical trial at the University of Alabama Vaccine Research Clinic is now being expanded based on an analysis of safety results to date conducted by both the Food and Drug Administration and a Data Monitoring Committee, the latter group comprised of a team of independent clinical, basic research, and statistical professionals.

The ongoing clinical study is the first extension to humans of preclinical studies, previously published in the peer-reviewed literature, indicating that the combination of Ampligen(R) (an experimental therapeutic) with commercially available seasonal influenza vaccine when applied intranasally, may provide enhanced protection against certain potentially pandemic influenza strains (Ichinohe T, et al. 2007, JID 196:1313-1320).

The Spanish flu pandemic killed between 20 and 100 million people in 1918-1919, a similar epidemic based on the worldwide population in 2004 has been estimated to result in approximately 60 million deaths (Murray CJ, et al. 2006, Lancet 368:2211-2218). Influenza often leads to death in patients suffering from underlying diseases; complications are especially prevalent in the elderly population. Hence, the objective of identification of an influenza vaccine and/or vaccine adjuvant "cocktail" offering broad-spectrum protective properties (sometimes referred to as a "Universal flu vaccine"), remains one of the highest public health priorities. Successful animal experimentation does not necessarily predict clinical effectiveness and no assurance can be given that the projected clinical tests will demonstrate either acceptable safety or efficacy.

The pioneering preclinical influenza research was conducted over several years at the Japanese National Institutes of Health (JNIH) under the direction of Dr. Hideki Hasegawa, Director, Department of Pathology. The research was conducted under a standard Material Transfer Agreement with the Company, and Dr. Hasegawa was an independent investigator who received no financial support from Hemispherx for his experimental work.

The expanded clinical study includes a search for immune molecules elicited by the treatment drugs which may be protective against pandemic and pre-pandemic influenza strains. In the cited preclinical studies with Ampligen(R), a broad range of potentially protective immune molecules were identified in both nasal washings as well as in the blood stream. In animals, the elicitation of both adaptive immunity molecules (immunoglobulins IgA and IgG) as well as innate immunity mechanisms (e.g., natural killer (NK) cells) have been detected as secondary to Ampligen(R)'s mechanism of action.

On April 30, 2012, when the first stage of the study received FDA authorization, Dr. Goepfert, Associate Professor of Medicine in the Division of Infectious Diseases and Director of the Alabama Vaccine Research Clinic, stated that "the normal route of transmission of the flu virus is through the nasal passage. We are excited about commencing this study, which utilizes the mucosal immune system and thus offers significant potential to protect large numbers of individuals by administering enhanced flu vaccines. In his animal studies, Dr.
Hasegawa showed that Ampligen(R) helped to expand viral epitope recognition and thereby provided cross-protection to flu virus strains which were not expected to be conveyed by the administered vaccine."

Highly pathogenic avian H5N1 viruses have extraordinary lethality in humans. Human disease has been limited thus far due to the inability of those viruses to infect the upper airways of humans -- although there has been concern that viral mutations could provide this capacity.
Scientists in Wisconsin and the Netherlands successfully mutated the
H5N1 virus so that it can more easily spread among humans (Nature News 20th December, 2011). This research was done to test the potential for avian H5N1 to mutate into a form that can spread easily in humans through coughing or sneezing. Nevertheless it has caused concern that such work may get into the hands of potential bio-terrorists and/or accidentally be released from experimental labs into the general environment. The Company believes that the expanded human trial at the University of Alabama will provide the first data in humans as to whether the epitope expansion observed by Dr. Hasegawa in experimental animals occurs in humans. Successful animal experimentation does not necessarily predict clinical effectiveness and no assurance can be given that the projected clinical tests will demonstrate either acceptable safety or efficacy.

It is anticipated that FluMist(R) combined with an effective adjuvant may be able to provide easily administered protection in humans even against mutated strains of flu virus including both H7N9 and H5N1 strains. Successful enhancement of immune responses in the current study group of young adults by Ampligen(R), an experimental therapeutic, would provide justification for extension of studies to the elderly who, as a group, could benefit greatly from more effective vaccines.



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