News Feature | February 20, 2014

Sarepta's AVI-7288 Shows Positive Phase I Results For Marburg Virus

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By Cyndi Root

Sarepta Therapeutics, Inc. announced in a press release that a Phase I study of AVI-7288 showed positive results. AVI-7288 is a drug candidate for Marburg virus infection. Sarepta developed the drug with the Department of Defense. The new drug formulation uses Sarepta’s proprietary PMOplus™ chemistry platform. Chris Garabedian, President and Chief Executive Officer of Sarepta Therapeutics, said, “These safety data, combined with previously reported efficacy results showing up to 100 percent survival in infected animals, differentiates AVI-7288 as the most advanced medical countermeasure in development for the treatment of Marburg infection.”

Marburg virus, also known as Marburg hemorrhagic fever, is a rare fever that strikes humans and primates. A member of the filovirus family, hemorrhagic fever is caused by a unique RNA virus. First described in 1967, its name came from an outbreak of the fever in Marburg, Germany when imported monkeys infected laboratory workers. Endemic to Africa, the CDC classified it as a possible bioterrorism agent and a threat to U.S. interests. The virus strikes suddenly with symptoms of nausea, vomiting, fever, and chills. It progresses to major organ failure and hemorrhaging. No treatments are available.

AVI-7288 was designed to alter the viral nucleoprotein (NP) gene by inhibiting mRNA translation. AVI-7288 is a part of Sarepta’s PMOplus™ technology. The platform combines chemistry and RNA-based therapeutics. In the Phase I study, 40 healthy adults participated. The placebo-controlled trial tested tolerability, safety, and pharmacokinetics. At the highest dose of AVI-7288 (16 mg/kg per day), participants tolerated the dose and no serious side effects or adverse events were reported. An independent panel reviewed the data and endorsed the drug for further study.

About Sarepta Therapeutics

Sarepta Therapeutics, formerly AVI BioPharma, develops RNA-based therapies for serious or infectious diseases. Eteplirsen for muscular dystrophy is a treatment in the pipeline. The Phase I trial of AVI-7288 was conducted with the U.S. Department of Defense’s Joint Program Executive Office for Chemical and Biological Defense. The agency works to develop chemical and biological countermeasures like drugs for bioterrorism. The Medical Countermeasure Systems Joint Project Management Office (JPM-MCS) aided in management of the project.