Avi plans to conduct the trial as part of its continued collaboration with the US Army Medical Research Institute of Infectious Diseases. Preclinical results of AVI-6003 demonstrated a reproducible and high rate of survival in non-human primates challenged with a lethal infection of Marburg virus.
In repeated trials, monkeys were dosed with well-tolerated amounts of drug and survived a challenge of roughly 1000 times the minimum lethal dose of virus. This level of infectious challenge normally results in uniform death of untreated monkeys within seven to 10 days. Treatment of Marburg infected animals with AVI-6003 resulted in 100% survival at 15 days, the company said.
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 relevant animal species and additional supporting data.
Leslie Hudson, president and CEO of Avi, said: The outstanding results demonstrated in preclinical studies of AVI-6003 for the treatment of Marburg infection warrant its advancement to studies that may ultimately lead to the development of the first effective therapeutic for this lethal infection.
We are excited by the potential shown by Avi’s RNA therapeutics for the treatment of infectious diseases including bioterrorism agents such as Marburg and Ebola virus.