Sarepta's Marburg drug candidate demonstrates positive safety results in trial

Sarepta Therapeutics, Inc., a Cambridge, Massachusetts-based RNA therapeutics developer, announced positive safety results on Monday from a Phase I clinical study of a drug candidate to treat Marburg virus infection.

Sarepta tested AVI-7288 in a Phase I randomized, double-blind, placebo-controlled trial that enrolled 40 healthy adult volunteers. Sarepta's AVI-7288 was found to be well tolerated through the highest dose tested with no reported serious or clinically significant adverse events. The company is developing the drug candidate under a contract managed by the U.S. Department of Defense's Medical Countermeasure Systems BioDefense Therapeutics Joint Product Management Office.

"We are very encouraged by the AVI-7288 study results announced today," Chris Garabedian, the 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 hemorrhagic fever is a potentially deadly disease caused by Marburg virus. The virus, which is endemic to Africa, is classified by the U.S. Centers for Disease Control and Prevention as a Category A bioterrorism agent.

"The new data for AVI-7288 add to a growing body of evidence supporting the safety and activity of Sarepta's (phosphorodiamidate morpholino oligomer)-based chemistries," Art Krieg, the senior vice president and chief scientific officer of Sarepta Therapeutics, said. "Our proprietary RNA technologies offer a versatile drug development platform with broad potential utility across a spectrum of therapeutic areas."

Sarepta Therapeutics develops RNA-based therapeutics meant to improve and save the lives of individuals affected by life-threatening rare and infectious diseases.