UPDATE: Sarepta Announces Positive Safety Results from Phase I Trial of Marburg Drug Candidate

Sarepta Therapeutics, Inc.

, a developer of innovative RNA-based therapeutics, today announced positive safety results from a Phase I multiple ascending dose study of AVI-7288 in healthy volunteers. AVI-7288, which uses Sarepta's advanced and proprietary PMOplus™ chemistry, is the company's lead drug candidate for the treatment of Marburg virus infection. Sarepta has been developing AVI-7288 under a Department of Defense contract managed by the Medical Countermeasure Systems BioDefense Therapeutics (MCS-BDTX) Joint Product Management Office. The Phase I clinical study was a randomized, double-blind, placebo-controlled trial that enrolled 40 healthy adult volunteers, and was designed to characterize the safety, tolerability and pharmacokinetics of AVI-7288 after daily repeat dosing. In each of five cohorts, six subjects received AVI-7288 and two subjects received placebo, daily for 14 days. Results showed that AVI-7288 was well tolerated through the highest dose tested, 16 mg/kg per day, which is higher than the anticipated therapeutic dose, with no reported serious or clinically significant adverse events. An independent Data and Safety Monitoring Board reviewed blinded safety results from the study and recommended continued clinical development of AVI-7288. “We are very encouraged by the AVI-7288 study results announced today,” said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. “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.” "The new data for AVI-7288 add to a growing body of evidence supporting the safety and activity of Sarepta's PMO-based chemistries,” said Art Krieg, M.D., senior vice president and chief scientific officer of Sarepta Therapeutics. “Our proprietary RNA technologies offer a versatile drug development platform with broad potential utility across a spectrum of therapeutic areas.”