Avi plans to conduct the trial as part of its continued collaboration with the US Army Medical Research Institute of Infectious Diseases (USAMRIID). Preclinical results of AVI-6002 demonstrated a reproducible and high rate of survival in non-human primates challenged with a lethal infection of Ebola, the company said.
In repeated trials, monkeys were dosed with well-tolerated amounts of drug and survived a challenge of roughly 1,000 times the minimum lethal dose. Treatment of Ebola infected animals with AVI-6002 resulted in 75% survival of the infected animals at 15 days post infection when the treatment period ended and circulating viral titer was below detectable levels.
Avi is conducting this research pursuant to the FDA’s Animal Efficacy Rule, which is designed for the development of new drug products for indications in which clinical studies in humans cannot be conducted ethically. According to this rule, marketing approval may be granted based on the demonstration of efficacy in appropriate animal species and additional supporting data.
Leslie Hudson, president and CEO of Avi, said: We are extremely pleased to be advancing AVI-6002 into clinical development for the treatment of Ebola virus based on the unprecedented results demonstrated by this product candidate in preclinical studies conducted in collaboration with USAMRIID.
The clinical development pathway for biodefense agents like AVI-6002 provides for approval of a product based upon animal efficacy data and supporting human safety data. Our success with this drug candidate demonstrates the strength of Avi’s biodefense program and the potential for RNA-based therapeutics in the treatment of infectious diseases, including bioterrorism agents.