SAN DIEGO--(BUSINESS WIRE)--April 7, 2003-- Hollis-Eden Pharmaceuticals, Inc. (NASDAQ:HEPH - News) today announced positive preliminary results from a study in non-human primates that demonstrated its immune regulating hormone HE2100 is providing significant protection from the acute life threatening effects of whole body radiation exposure. High levels of whole body radiation damages a person's bone marrow resulting in neutropenia -- a severe loss of neutrophils, or key white blood cells -- which results in a high risk of infection, hospitalization and potential death. Preliminary results from the Company's pilot study indicate when HE2100 is given 24 hrs before or 2 to 4 hrs after radiation exposure, a statistically significant reduction in the occurrence of severe neutropenia is observed as compared to control animals not receiving drug. The data were presented today at the 43rd Annual Scientific Meeting of the British Society For Haematology, being held April 7-9, 2003 in Glasgow, Scotland.
Under a new U.S. Food and Drug Administration (FDA) rule, where it would be unethical to expose healthy humans to radiation in an effort to determine clinical efficacy, marketing approval as a drug to provide protection from acute radiation injury may be gained solely on the demonstration of safety in humans and efficacy in relevant animal species. Hollis-Eden believes that the non-human primate is the most relevant animal species to demonstrate efficacy that would correlate in humans. This initial pilot study is the first of several to be conducted to determine the optimal dosing regimen. Following these pilot studies, Hollis-Eden plans to conduct a pivotal efficacy study in non-human primates, which the Company believes is comparable to a Phase III clinical trial to demonstrate efficacy of HE2100 under the new FDA rule. The Company expects to apply for approval of HE2100 for radioprotection in 2004.
In the pilot study, animals were given only one dose of HE2100 24 hours prior to being exposed to 400cGy of radiation, and other animals were given daily doses of HE2100 beginning 2 to 4 hours after radiation exposure. A control group of animals received no medicine. By day 8 after radiation exposure, the majority of the control animals experienced severe neutropenia and were administered preventative antibiotic therapy. Three weeks after radiation, the average percentage of days of severe neutropenia (less than 500 neutrophils/uL) was 47% for the control group versus 17% for a group receiving a single dose of HE2100 before radiation exposure and 11% for a group of animals treated with HE2100 after radiation exposure. This represents a 4-fold decrease in the percentage of days the animals in the post-radiation treatment group were at high risk for infection -- the leading cause of mortality following whole body radiation.
"These data mean that in a scenario of a nuclear event, such as a dirty bomb or nuclear accident, where tens of thousands of people are potentially exposed to high levels of radiation, HE2100 may offer a cost-effective treatment that could significantly improve the chance of survival and reduce the necessity of hospitalization at a time when medical facilities would be overwhelmed," said Dwight R. Stickney, M.D., Radiation Oncologist and Medical Director, Hollis-Eden Pharmaceuticals, Inc. "The data are still being developed from this pilot study and there is more work to be done to optimize the administration parameters; however, we are very excited by the significant protection that was afforded the animals from the toxicity of radiation in a model relevant to man."
Hollis-Eden is co-developing HE2100 with an agency within the U.S. Department of Defense, where HE2100 has emerged as the agency's leading candidate for radioprotection. Results of several previous studies conducted in mice have been published in the International Journal of Immunopharmacology and in Radiation Research. These studies showed that HE2100, when given to animals shortly before or shortly after exposure to lethal doses of radiation, provided significant survival advantages in HE2100-treated animals versus placebo-treated animals. In that study, 100% of animals treated with HE2100 24 hours prior to being exposed to 900cGy of radiation survived versus 100% mortality in the animal group receiving no drug. Investigators conducting the study attributed the survival advantage to HE2100's ability to increase a number of cell types associated with immune protection, including neutrophils and platelets.
The leading drug currently marketed for neutropenia is Amgen's Neupogen, which is used in the setting of chemotherapy-induced neutropenia. Neupogen costs the patient approximately $2,500 per treatment regimen and generates annual revenue of approximately $1.5 billion. Neupogen received FDA approval on the basis of the reduction of infections by shortening the time of neutropenia in cancer patients undergoing chemotherapy.
There is an urgent, unmet medical need for a safe and practical countermeasure for the significant risk of neutropenia resulting from high levels of radiation exposure. The only drug that is currently available for stockpiling in the event of radiation injury is potassium iodide. However, potassium iodide is only effective against the long-term risk of thyroid cancer, and does not protect the body from the acute effects on the bone marrow, which can lead to rapid fatalities. Despite this limitation, potassium iodide has been stockpiled broadly for years in Europe and Japan for civilians living within close proximity of nuclear power plants, and the U.S. has recently begun purchasing millions of doses of the drug for similar uses in this country. In addition, the Strategic National Stockpile, a program of the Department of Homeland Security, has already stockpiled enough drugs to treat 100 million people exposed to plague, 50 million people exposed to tularemia and 12 million people exposed to anthrax. The agency claims it can deliver the drugs anywhere in the country in 12 hours or less. Given that HE2100 may be useful in protecting against the immediate life threatening effects of radiation, the Company believes there may be strong interest by government agencies to adopt a similar stockpiling strategy if HE2100 is successfully developed.
Hollis-Eden also presented data at this meeting updating previously disclosed research conducted late last year demonstrating that both HE2100 and HE2200, another investigational drug being developed by Hollis-Eden, significantly reduced or completely prevented neutropenia in non-human primates exposed to carboplatin, a commonly used chemotherapy agent. In that study, primates were administered a dose of carboplatin followed 36 hours later by daily doses of either HE2100, HE2200, or no drug for a period of 10 days. Toxicity from chemotherapy can result in patients becoming at risk for infection due to the significant loss of neutrophils. The primary endpoint of the study was the total number of observations of neutropenia (less than 1200 neutrophils/uL) during the 20-day study period. In the no drug group, animals were neutropenic for a total of 41% of the observations. In contrast, both HE2200 and HE2100 showed statistically significant improvement in the number of observations of neutropenia, with HE2200-treated animals showing neutropenia for only 15% of the observations and HE2100 showing 0% neutropenia for all animals during the entire 20-day observation period. In addition, histological data was presented illustrating significant chemotherapy-induced toxicity to the bone marrow of untreated animals when compared to the HE2100-treated animals at the time of maximum chemotherapy-induced damage to the bone marrow. Both investigational drugs were well tolerated with no serious side effects reported.
"The data from this radiation protection study marks a key significant milestone achievement for the Company and we believe radically advances the vision we have for our technology and drug candidates," said Richard Hollis, Chairman and CEO, Hollis-Eden Pharmaceuticals, Inc. "While the new FDA rule provides a pathway to commercial approval without the need to conduct traditional Phase II and Phase III efficacy studies in humans, the agency will nevertheless require robust proof of effectiveness in a relevant animal species and safety in humans that clearly and definitively establishes the drug candidate's value. This is still a very rigorous FDA regulatory approval process. However, this is also why these results are so relevant and vital to the development of this extremely important drug candidate as a countermeasure to the most feared weapon of mass destruction, radiation. The ability of HE2100 to significantly protect these animals from the loss of infection-fighting white blood cells in our first pilot study in primates is a major medical and scientific accomplishment. We are now in a position to accelerate HE2100 development in relevant animal trials for commercial approval and to make HE2100 available for our national pharmaceutical stockpiling efforts. We realize the importance of this drug candidate's contribution to Homeland Security and to the security of our soldiers, rescue workers and citizens in this new era of terrorist threats. We are also excited about the roles HE2100 and HE2200 may play in the cancer market, which represents another enormous market opportunity for our drug candidates."
Hollis-Eden Pharmaceuticals, Inc. is a development-stage pharmaceutical company based in San Diego, California, working to become the world leader in the development of a new class of investigational drugs known as Immune Regulating Hormones (IRHs). The goal of IRH therapy is to direct, through controlling gene expression, the production of key cytokines and enzymes that re-regulate immune and metabolic functions toward homeostasis, a profile that could be useful in a wide variety of diseases. The Company has a number of investigational IRHs under development, including HE2000, which is currently being studied in clinical trials in a number of infectious diseases. Hollis-Eden is also developing an additional IRH, HE2200, for improving vaccine responses in the elderly and for lowering cholesterol in conditions of hypercholesterolemia. For more information on Hollis-Eden, contact the Company's website at holliseden.com |
