A similar phase I clinical trial for Neumune is currently ongoing in the Netherlands. Acute Radiation Syndrome (ARS) is a potentially lethal condition caused by high-dose radiation exposure that might result from a nuclear or radiological terrorist attack or from an accident at a nuclear facility.
Hollis-Eden is developing Neumune under an FDA rule (the Animal Efficacy Rule) designed for medical countermeasures to weapons of mass destruction. According to this rule, for indications in which it would be unethical to conduct efficacy studies in humans (as is the case with radiation injury), marketing approval may be granted based on the demonstration of efficacy in relevant animal species and successful completion of phase I safety trials in humans.
Hollis-Eden has already conducted and reported on dose-ranging radiation studies in over 200 non-human primates. In animals receiving moderate doses of radiation, Neumune has demonstrated a reduction in the incidence of neutropenia (loss of white blood cells), thrombocytopenia (loss of platelets) and anemia (loss of red blood cells) compared to animals not receiving the compound. In animals receiving high doses of radiation, Neumune demonstrated a reduction in both febrile neutropenia and severe thrombocytopenia, as well as a survival benefit as compared to animals not receiving Neumune.
Pivotal studies in non-human primates, designed to demonstrate efficacy in support of a planned New Drug Application (NDA) filing, are expected to begin in the second half of 2005.
Under the phase I clinical trial program, in addition to analyzing safety, initial studies are being conducted to determine the concentration of Neumune that can be achieved in human blood. This pharmacokinetic (PK) information can then be used in selecting the final dose for the pivotal efficacy study in non-human primates, as well as a larger human clinical trial to demonstrate safety.
Under the Animal Efficacy Rule, matching the drug levels in humans with that which produces efficacy in the appropriate animal species is a requirement for approval. By comparing the concentration of Neumune that can be achieved in humans to that which has already been established in non-human primates, the company expects to be able to match the dose most likely to achieve efficacy in the pivotal studies with a dose that is achievable and tolerable in humans.
The studies to be conducted in the US are designed to complement the clinical program that has already commenced in the Netherlands.