Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, announced today that preclinical data demonstrating potent anti-viral activity of TKM-Marburg were presented at DIA/FDA Oligonucleotide-based Therapeutics Conference taking place in Washington, DC from September 25-27, 2013 by Tekmira's Chief Scientific Officer, Dr. Ian MacLachlan.
"Our collaborative work with Dr. Tom Geisbert and the team at UTMB has resulted in new data that builds upon our work in viral diseases and further validates the broad applicability of Tekmira's industry-leading LNP technology platform. We are excited to see promising new preclinical data showing complete protection of non-human primates from the most pathogenic strain of Marburg virus, Angola, even when the TKM-Marburg treatment regimen is not initiated until 24 hours after virus exposure," said Dr. Mark J. Murray, Tekmira's President and CEO.
"We expect to continue to build on these data and pursue additional funding opportunities for TKM-Marburg. In addition to TKM-Marburg, we are developing TKM-Ebola under a $140 million contract awarded by the U.S. Government and we will enter a Phase I clinical trial early in 2014. Tekmira's leadership in the area of anti-viral therapy for hemorrhagic fever viruses has formed a strong foundation for future anti-viral therapeutics," added Dr. Murray.
In a presentation entitled "Medical Countermeasures for Filovirus Infection: Development of siRNA Therapeutics Under the Animal Rule" data were presented that showed successful anti-viral therapy with the application of Tekmira's LNP technology to hemorrhagic fever viruses, including multiple strains of the Ebola and Marburg viruses. Newly presented data resulting from a collaboration between Tekmira and the University of Texas Medical Branch (UTMB) showed 100% survival in non-human primates infected with the Angola strain of the Marburg virus in two separate studies. In the first study, 100% survival was achieved when dosing at 0.5 mg/kg TKM-Marburg began one hour after infection with otherwise lethal quantities of the virus. Dosing then continued once daily for seven days. In the second study, 100% survival was achieved even though treatment did not begin until 24 hours after infection.
Marburg and Ebola are members of the filovirus family of hemorrhagic fever viruses. Regularly occurring natural outbreaks with of the Marburg Angola strain have resulted in mortality in approximately 90% of infected individuals, matching that of the most lethal Ebola strains, while in laboratory settings experimental infection with either virus is uniformly lethal. There are currently no approved therapeutics available for the treatment of Marburg infection.
These new results build upon a study published last month in the Journal of Infectious Disease showing 100% protection in guinea pig models of infection with Angola, Ci67 and Ravn strains of the Marburg virus using a broad spectrum RNAi therapeutic enabled by Tekmira's LNP. In 2010, Tekmira and UTMB were awarded a National Institutes of Health (NIH) grant to support research to develop RNAi therapeutics to treat Ebola and Marburg hemorrhagic fever viral infections.
TKM-Ebola, an anti-Ebola viral therapeutic, is being developed under a contract with the U.S. Department of Defense's (DoD) Joint Project Manager Medical Countermeasure Systems (JPM-MCS) , with a total contract value of approximately $140 million. Earlier preclinical studies were published in the medical journal The Lancet and demonstrated that when siRNA targeting the Ebola virus and delivered by Tekmira's LNP technology were used to treat previously infected non-human primates, the result was 100 percent protection from an otherwise lethal dose of Zaire Ebola virus (Geisbert et al., The Lancet, Vol 375, May 29, 2010). Tekmira's productive collaboration with the JPM-MCS was recently modified and expanded to include significant advances in LNP formulation technology since the initiation of the program in 2010.
About Joint Project Manager Medical Countermeasure Systems (JPM-MCS)
JPM-MCS, a component of the Joint Program Executive Office for Chemical and Biological Defense, aims to provide U.S. military forces and the nation with safe, effective, and innovative medical solutions to counter chemical, biological, radiological, and nuclear threats. JPM-MCS facilitates the advanced development and acquisition of medical countermeasures and systems to enhance U.S. biodefense response capability. For more information, visit www.jpeocbd.osd.mil.
About RNAi and Tekmira's LNP
RNAi therapeutics have the potential to treat a broad number of human diseases by "silencing" disease causing genes. The discoverers of RNAi, a gene silencing mechanism used by all cells, were awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi therapeutics, such as "siRNAs," require delivery technology to be effective systemically. Tekmira believes its LNP technology represents the most widely adopted delivery technology for the systemic delivery of RNAi therapeutics. Tekmira's LNP platform is being utilized in multiple clinical trials by both Tekmira and its partners. Tekmira's LNP technology (formerly referred to as stable nucleic acid-lipid particles or SNALP) encapsulates siRNAs with high efficiency in uniform lipid nanoparticles that are effective in delivering RNAi therapeutics to disease sites in numerous preclinical models. Tekmira's LNP formulations are manufactured by a proprietary method which is robust, scalable and highly reproducible, and LNP-based products have been reviewed by multiple FDA divisions for use in clinical trials. LNP formulations comprise several lipid components that can be adjusted to suit the specific application.
Tekmira Pharmaceuticals Corporation is a biopharmaceutical company focused on advancing novel RNAi therapeutics and providing its leading lipid nanoparticle delivery technology to pharmaceutical partners. Tekmira has been working in the field of nucleic acid delivery for over a decade and has broad intellectual property covering LNPs. Further information about Tekmira can be found at www.tekmirapharm.com. Tekmira is based in Vancouver, B.C.