Safety Study of Single Administration Post-Exposure Prophylaxis Treatment for Ebola Virus

A Phase I Randomized, Double-Blind, Placebo-Controlled, Single-Dose, Dose-Escalation Study to Assess the Safety, Tolerability and Pharmacokinetics of AVI-6002 in Healthy Adult Volunteers

The purpose of this study is to characterize the safety and pharmacology of single administrations of AVI-6002, a post-exposure prophylaxis candidate treatment for Ebolavirus.

Ebola hemorrhagic fever (EHF) is a rare human disease caused by Ebola Virus (EBOV), a filamentous single-stranded, negative-sense RNA virus. Since 1976 several Ebolavirus outbreaks have occurred with fatality rates ranging from 57% to 90%, with most of these outbreaks traced to single EBOV species; EBOV-Z. No effective therapy is currently available for Ebolavirus.

AVI-6002 is an experimental combination of 2 phosphorodiamidate morpholino antisense oligomers with positive charges on selected subunits (PMOplus™). These oligomers specifically target viral messenger RNA encoding 2 Ebolavirus proteins thought to be important in viral replication and host immune suppression. The present study is designed to characterize the safety, tolerability and pharmacokinetics of escalating single-administration doses of AVI-6002 in healthy human subjects.

Inclusion Criteria:

• Male or female and between the ages of 18 and 50 years in good general health
• Volunteers must be willing to use barrier methods of contraception or be of non-childbearing potential
• Volunteers must be willing to undergo a urine screen for drugs of abuse

Exclusion Criteria:

• Pregnancy or breastfeeding.
• Any clinically relevant abnormalities in physical examinations, vital signs, ECG, clinical chemistry, hematology or urinalysis
• Positive test for human immunodeficiency virus, hepatitis B or hepatitis C or known history of HIV infection