Nucleic acid compounds represent a potential new class of pharmaceutical products that are designed to act at the molecular level for treatment of human diseases. The Hemispherx Biopharma drug technology utilizes specifically-configured RNA. The Hemispherx double-stranded RNA drug product, trademarked Ampligen®, an experimental, unapproved drug, which is administered intravenously, is in human clinical development for various therapeutically oriented studies, including treatment for CFS/ME, HIV, renal cell carcinoma and malignant melanoma.
Based on the results of pre-clinical studies and clinical trials, Hemispherx believes that Ampligen®, an experimental agent, may have broad-spectrum anti-viral and anti-cancer properties. Approximately 500 patients have received Ampligen® in clinical trials authorized by the Food and Drug Administration (“FDA”) at over twenty clinical trial sites across the U.S., representing the administration of more than 40,000 doses of this drug, and Ampligen® is available only through clinical trials for limited indications.
Clinical trials already conducted by Hemispherx U.S. include treatments of CFS/ME, other infectious diseases such as Hepatitis B, HIV, and clinical trials for cancer including patients with renal cell carcinoma and malignant melanoma. Certain of these studies to date have not been well-controlled and accordingly additional tests will be necessary for support of regulatory approvals.
Hemispherx U.S. has been issued certain patents on the use of Ampligen® alone and Ampligen® in combination with certain other drugs including AZT, ddI, ddC, interferon and/or IL-2, for the treatment of HIV.
A second experimental drug that Hemispherx U.S. has patented is Poly A:Poly U for Hepatitis B, which has been studied in advanced clinical trials primarily conducted by a major European pharmaceutical company. Hemispherx U.S. also has licensed a series of patents on Oragen™, experimental drugs believed by the company to be a broad-spectrum oral anti-viral drugs of the second-generation RNA drugs developed at Temple University. Hemispherx's experimental compounds, no members of which have yet been determined "safe and effective" by regulatory authorities, are accordingly only available legally in certain authorized trials and tests; in vitro (outside the body) tests are also not necessarily indicative of any evidence of clinical benefits or advantages. But the focus of Hemispherx is on Ampligen® as a treatment for CFS/ME and HIV.
The Company believes that Ampligen® has been generally well tolerated with a low incidence of clinical toxicity, particularly given the severely debilitating or life threatening diseases that have been treated. A mild flushing reaction has been observed in approximately 15% of patients treated in our various studies. This reaction is occasionally accompanied by erythema, a tightness of the chest, tachycardia, anxiety, shortness of breath, subjective reports of "feeling hot," sweating and nausea. The reaction is usually infusion-rate related and can generally be controlled by slowing the infusion rate. Other adverse side effects include liver enzyme level elevations, diarrhea, itching, urticaria (swelling of the skin), bronchospasm, hypotension, photophobia, rash, bradycardia, transient visual disturbances, arrhythmias, decreased platelets and white blood cell counts, anemia, dizziness, confusion, elevation of kidney function tests, occasional temporary hair loss and various flu-like symptoms, including fever, chills fatigue, muscular aches, joint pains, headaches, nausea and vomiting. These flu-like side effects typically subside within several months.
Alferon Low Dose Oral
Hemispherx's oral form of
our highly purified,
natural source alpha interferon
is a new dosage form of our FDA approved drug, Alferon N®, the only
natural interferon currently FDA approved and available in the
marketplace. Hemispherx has initiated clinical trials as
part of an accelerated evaluation of the experimental
bio-therapeutic Alferon LDO (Low Dose Oral Interferon Alfa-n3
(Human Leukocyte Derived)) as a potential new experimental
therapy for Avian Flu and other lethal viral diseases, which
have high acute death rates. Clinical trials in human volunteers
(being conducted in both the U.S. at Drexel University,
Philadelphia, and shortly to commence in Hong Kong at the
Princess Margaret Hospital) are designed to determine whether
Alferon N, delivered in a new, experimental oral drug delivery
format, can resuscitate the broad-spectrum antiviral and
immunostimulatory genes. These human genes are shut down by
acute lethal viral infections such as avian flu and smallpox.
Positive results in the ongoing
clinical studies would potentially make Alferon LDO the first
orally active interferon drug candidate and would open an
opportunity leading to a wide range of potential new
Polyadenur™ is the trademark of Poly A : Poly U RNA developed and tested independently of Hemispherx U.S. by laboratories Beaufour Ispon, a French corporation. Beaufour Ispon conducted Phase II/III trials in Hepatitis B in conjunction with interferon. The results as compared with the use of interferon alone were encouraging based on a reduction in virus and improvement in liver parameters. Poly A : Poly U application to Hepatitis B is covered by Hemispherx U.S. patents and this has been confirmed recently by the European patent application review board. The strategy to follow is still to be finalized as Ampligen® also had been studied in Hepatitis B.