Hemispherx BioPharma, Inc (NYSEMKT:HEB) woke up to a nice 10% pop in the value of their shares, after the drug maker commenced full data analysis of an intranasal human safety study of Ampligen® plus FluMist® known as AMP-600. The study was previously closed, but the initiation of full data analysis awaited the FDA’s evaluation of preliminary reports of blinded study findings. That evaluation was completed per formal notification from the FDA on August 9, 2017. Intranasal Ampligen was generally well-tolerated in the study.
Hemispherx shares reacted to the news, soaring nearly 8% to $0.44 in Monday’s trading session. HEB has a 1-year high of $2.64 and a 1-year low of $0.30. The stock’s 50-day moving average is $0.46 and its 200-day moving average is $0.53.
This study is the first to evaluate the human safety of nasally-administered Ampligen. Test subjects in other human studies of Ampligen, including the pancreatic cancer treatment program currently underway in The Netherlands, have been administered Ampligen intravenously (IV). All told, approximately 100,000 IV doses of Ampligen have been administered and found to be generally well-tolerated.
Hemispherx believes that the top line data from the intranasal safety study, coupled with extensive pre-clinical data, support further development of Ampligen as a broadly applicable highly selective Toll-like Receptor 3 (TLR-3) based new generation, immune enhancing adjuvant for intranasally-administered vaccines.
“We’re very encouraged by the outcome of this study, establishing a foundation for Ampligen as an intranasal adjuvant. This shows Ampligen’s potential to take us to the next step of developing Ampligen either as a strain-specific or more universal influenza intranasal vaccine immune enhancing adjuvant”, said Thomas Equels, Hemispherx’s Chief Executive Officer. “This safety study was the first milestone to our ultimate goal, which is to combine Ampligen with a non-live virus vaccine to produce stronger and broader coverage and to confer longer immunity than is possible with the vaccine alone. I firmly believe that Ampligen’s multi-faceted impact on the immune system redefines the term adjuvant.”
Ampligen is a highly selective TLR-3 agonist that induces the innate immune responses required for adaptive protective immunity. Monkeys immunized with H5N1 vaccine and Ampligen showed enhanced protection after being challenged with a homologous highly pathogenic H5N1 virus. Pre-clinical studies also showed cross-protection against H5N1 viruses using trivalent seasonal influenza vaccine in mouse models. In theory, Ampligen induced cross-protection may extend to any vaccine for viruses subject to a high mutation rate and genetic drift.
Ampligen is currently being developed as an immuno-oncology agent in solid cancer tumors and as a treatment in Chronic Fatigue Syndrome (CFS). Studies have shown that the two totally different indications both benefit from Ampligen’s foundational strength as an immuno-therapeutic. The company has completed a pivotal Phase 3 clinical trial in ME/CFS, where it is the only late-stage drug in the pipeline for this disease. The U.S. FDA has advised that potential approval for commercial sale in the U.S. for a ME/CFS indication will require the conduct and review of a follow up confirmatory clinical trial. Hemispherx is supplying the drug for use by pancreatic cancer patients in an Early Access Program sponsored by the Dutch government.
In AMP-600, Ampligen was nasally-administered as an adjuvant in conjunction with FluMist®, a seasonal live-virus flu vaccine, to healthy human volunteers at the University of Alabama at Birmingham under the auspices of Paul Goepfert, MD, Associate Professor of Medicine in the Division of Infectious Diseases and Director of the Alabama Vaccine Research Clinic. Twenty-five subjects were enrolled, twelve in Stage 1 and thirteen in Stage 2. The data from the AMP-600 safety study extends the pre-clinical results into humans. In the study, Ampligen was generally well-tolerated when administered intranasally with a live-virus influenza vaccine. Future studies with non-live virus vaccines will seek to replicate Ampligen’s efficacy as shown in the animal model results.
“Our next milestone for intranasal Ampligen is to collaborate with an established vaccine maker to administer Ampligen intranasally with a non-live virus vaccine,” said Equels, “this next milestone will allow us to determine Ampligen’s clinical efficacy as a viral vaccine adjuvant and to evaluate it as a mechanism for providing cross protection from virus mutations as well as other forms of the virus. We have every expectation of results similar to the prior animal studies.”
Hemispherx Biopharma, Inc. is a biopharmaceutical company, which engages in the clinical development of new drug therapies based on natural immune system enhancing technologies for the treatment of viral and immune based disorders. Its products include Alferon N Injection and the experimental immunotherapeutic/antiviral Ampligen. Alferon N Injection is a purified, natural source, glycosylated, multispecies alpha interferon product, composed of eight forms of high-purified alpha interferon. Ampligen includes application as a treatment for Chronic Fatigue Syndrome and as a vaccine enhancer for therapeutic and/or preventative development related to influenza and cancer treatments.