Date: 2014-10-16
Type of information: Results
phase: 1
Announcement: results
Company: Sarepta Therapeutics (USA - MA)
Product: AVI-6002
Action
mechanism: AVI-6002 for the treatment of Ebola is a combination therapy of two phosphorodiamidate morpholino oligomers (PMOs AVI-7537 and AVI-7539), which target the viral matrix proteins VP24 and VP35, respectively. Subsequent animal studies demonstrated that for each combination therapy, only one oligomer contributed to efficacy, and therefore, the lead drug candidate for Ebola has since become the single compounds AVI-7537. PMOplus® chemistry is an advanced generation of Sarepta\'s phosphorodiamidate morpholino oligomer, or PMO, technology pioneered by Sarepta. The PMO platform is designed to provide a stable chemistry backbone with drug-like characteristics for Sarepta\'s advanced RNA-based therapeutics. PMOplus® chemistry includes specific molecular charges positionally inserted into the PMO\'s inherent charge-neutral backbone.
Disease: Ebola fever
Therapeutic area: Infectious diseases
Country:
Trial
details: The Phase I clinical study was a randomized, double-blind, placebo-controlled trial designed to characterize the safety, tolerability and pharmacokinetics of single doses of intravenous formulations of AVI-6002 in healthy adult volunteers. 30 subjects were enrolled in six cohorts receiving up to 9 mg/kg of the combination drug candidates (4 active:1 placebo per cohort) for a total of 60 subjects.
Latest
news: * On October 16, 2014, Sarepta Therapeutics, a developer of innovative RNA-based therapeutics, announced the publication of results from two single ascending-dose studies that demonstrated no clinical or toxicologic safety concerns with the company’s drug candidates for the treatment of Ebola and Marburg virus, respectively. AVI-6002 for the treatment of Ebola is a combination therapy of two phosphorodiamidate morpholino oligomers (PMOs AVI-7537 and AVI-7539), which target the viral matrix proteins VP24 and VP35, respectively. Results from previous viral challenge studies of AVI-6002 in non-human primates demonstrated prevention of disease development and death following exposure to Ebola virus. Subsequent animal studies demonstrated that for each combination therapy, only one oligomer contributed to efficacy, and therefore, the lead drug candidate for Ebola has since become the single compounds AVI-7537. Results showed the compound to be well-tolerated with no dose limiting level demonstrated. No clinically significant or dose-dependent effects were observed at any of the safety endpoints evaluated. The safety and pharmacokinetics of the PMOplus® compounds were similar, regardless of the target RNA sequence. The study results are to be published in the November issue of the American Society for Microbiology’s journal, Antimicrobial Agents and Chemotherapy and are available online at dx.doi.org/10.1128/AAC.03442-14.
