Sarepta Therapeutics, Inc.(formerly AVI BioPharma) has announced that its lead therapeutic drug candidate for the Marburg virus, AVI-7288, demonstrated up to 100 per cent survival in a non-human primate (NHP) study exploring the drug's effect when treatment is delayed to various time points post-infection. The study demonstrated a significantly higher rate of survival among NHPs treated with AVI-7288 compared to the placebo-treated group when treatment was administered up to 96-hours post infection. Sarepta is conducting this work under a US Department of Defense (DoD) 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).

"These results are unprecedented and demonstrate a compelling proof of concept with our PMOplus chemistry platform and its ability to treat the most lethal and fast-acting viruses, without compromising efficacy of survival even after up to a four-day delay in the initiation of treatment," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "These results represent a significant advancement toward the protection of our service members and the civilian population in the event of a bioterrorist attack. Extending the window of opportunity for effective medical intervention against lethal infections may translate to more lives saved."

This study showed a high degree of survival between 83 per cent and 100 per cent in each of four post-exposure cohorts that received daily treatments with AVI-7288 beginning one-, 24-, 48-, or 96-hours after infection, compared to 0 per cent survival in the placebo-treated control group. Currently at Day 27, the study will continue to monitor the surviving non-human primates until study termination at Day 41.

The work is a collaborative effort between Sarepta and scientists at the U.S. Army Medical Research Institute of Infectious Diseases (USAMRIID), the DoD's leading medical research laboratory for biological defense, which has the DoD's only maximum containment, or Biosafety Level 4, capability.

Marburg hemorrhagic fever is a severe and potentially fatal disease in humans first recognized in 1967. It is caused by an RNA virus of the Filoviridae family and is understood to be endemic to Africa. The Marburg virus is classified as a Category A bioterrorism agent by the Centers for Disease Control and Prevention (CDC) and was determined to pose a material threat to national security and public health by the Secretary of Homeland Security in 2006. Onset of the disease is often sudden, and the symptoms include fever, chills, nausea, vomiting, chest pain and diarrhea. Increasingly severe symptoms may also include massive hemorrhaging and multiple organ dysfunctions. There are currently no treatments for Marburg virus infection beyond supportive care.

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 superior 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. PMOplus® has potentially broad therapeutic applications and has thus far shown to be particularly effective in increasing the potency of PMO-based oligomers.

JPM-TMT is a component of the US Department of Defense's Joint Program Executive Office for Chemical and Biological Defense (JPEO-CBD). JPM-TMT aims to protect the Warfighter from emerging infectious diseases, genetically altered, and unknown biological threats. Through strategic investments and partnerships with innovative biotech firms, pharmaceutical corporations, other government agencies, and academic institutions, JPM-TMT facilitates the advanced development and acquisition of adaptable platform technologies, broad-spectrum medical countermeasures, and innovative systems to enhance our nation's biodefense response capability.

US Army Medical Research Institute of Infectious Diseases (USAMRIID), located at Fort Detrick, Maryland, is the lead medical research laboratory for the US Department of Defense's Biological Defense Research Program, and plays a key role in national defense and in infectious disease research.

Sarepta Therapeutics -- formerly AVI BioPharma -- is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases.