AVI BioPharma gets FDA approval to proceed with Marburg virus infection study 2 hours 37 minutes ago - Datamonitor via Comtex
AVI BioPharma, Inc., a developer of RNA-based therapeutics, has received the FDA approval to proceed with a single oligomer, AVI-7288, in studies in both humans and non-human primates to support the safety and efficacy of post-exposure prophylaxis against Marburg virus infection.
AVI-7288 is one of two components that make up AVI-6003. Studies conducted to date have shown that efficacy in non-human primates can be attributed to this single component, while the second component, AVI-7287, does not appear to contribute to efficacy.
AVI is conducting this work under a Department of Defense contract managed by the Joint Project Manager Transformational Medical Technologies (JPM-TMT) Project Management Office, a component of the Joint Program Executive Office for Chemical and Biological Defense (JPEO-CBD). The FDA approved proceeding with the Marburg program using the single oligomer AVI-7288 under the original IND.
AVI will proceed with dosing AVI-7288 in the Phase I multiple ascending dose studies planned to characterize the safety, tolerability and pharmacokinetics of multiple doses of the drug in healthy adult volunteers. The randomized, double-blind placebo controlled study will be overseen by a DSMB, who will review safety and clinical laboratory data after each dose cohort prior to enrolling the next highest dose cohort. AVI will also proceed using AVI-7288 in non-human primate studies to continue to evaluate efficacy.
The company will present the results of a non-human primate confirmatory study conducted to evaluate the optimal dose and components of AVI-6003 during the upcoming Association of Microbiology BioDefense and Emerging Diseases Research Meeting in Washington, DC. In the confirmatory study at higher doses of PMOplus at 15 mg/kg/component, survival was 90%, 100% and 0% in the AVI-6003, AVI-7288 and placebo groups, respectively. Based on these results, AVI concluded that AVI-7288 is the active component in AVI-6003 and that further development would proceed accordingly with the single oligomer component AVI-7288.
AVI-7288 is a single oligomer, one of two composing AVI-6003. AVI-7288 employs AVI's patented PMOplus technology that selectively introduces positive charges to its phosphorodiamidate morpholino oligomer (PMO) backbone to improve interaction between the drug and its target.
"While we have not seen any toxicity in humans to date with AVI-6003, by removing AVI-7287 we have a simpler development path and may improve the therapeutic window by evaluating a single action agent," said Chris Garabedian, president and CEO of AVI BioPharma. "We are pleased to be able to explore this single oligomer as a potentially safe and effective treatment against this lethal hemorrhagic fever virus."