Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, announced the publication of two articles in peer-reviewed scientific journals — the Journal of Infectious Diseases and New England Journal of Medicine — that highlight results enabled by Tekmira's lipid nanoparticle (LNP) technology.
"These recently published data further validate the broad applicability of Tekmira's industry-leading LNP technology platform. Our work with our collaborators at UTMB has resulted in the first report of complete post-exposure protection against the most pathogenic strain of Marburg virus. These findings build upon our work in infectious diseases — including our TKM-Ebola program, an anti-Ebola viral therapeutic currently in development under a $140 million contract awarded by the U.S. Government and entering a Phase I clinical trial early in 2014 — and provide a foundation for future infectious disease therapeutics," said Dr. Mark J. Murray, Tekmira's President and CEO.
"Tekmira's LNP technology is also enabling the rapid, dose-dependent, durable, and specific knockdown of TTR in clinical trials of Alnylam's ALN-TTR01 and ALN-TTR02 RNAi therapeutic products. Specifically, the ALN-TTR02 data points to our LNP delivery technology providing improved potency and demonstrating up to a 94% reduction of serum TTR with ALN-TTR02," added Dr. Murray.
The study published in the Journal of Infectious Diseases results from a collaboration between Tekmira and the University of Texas Medical Branch (UTMB). The paper, entitled "Protection against Lethal Marburg Virus Infection Mediated by Lipid Encapsulated siRNA" showed 100% protection in guinea pig models against the Angola, Ci67 and Ravn strains of the Marburg virus using a broad spectrum RNAi therapeutic enabled by Tekmira's LNP (Ursic-Bedoya et al., J Infect Dis.(2013) [Online early access]. doi: 10.1093/infdis/jit465. First published online: August 29, 2013).
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. The grant is supporting ongoing work at Tekmira and at UTMB including work advancing these promising results into non-human primates.
Complete study results from Phase I trials with ALN-TTR01 and ALN-TTR02 were published in the New England Journal of Medicine in a paper entitled "Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis" (Coelho et al., N Engl J Med 2013; 369:819-29). ALN-TTR01 and ALN-TTR02 are systemically delivered RNAi therapeutics that use Tekmira's LNP technology and target transthyretin (TTR), the disease-causing protein in TTR-mediated amyloidosis (ATTR). ALN-TTR01 and ALN-TTR02 are being developed by Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY). More detailed information about the Phase I trials with ALN-TTR01 and ALN-TTR02 can be found in Alnylam's news release dated August 28, 2013, which has been posted at www.alnylam.com.
Tekmira has granted Alnylam a license to use Tekmira's LNP technology to advance RNAi therapeutic products, and Tekmira is eligible to receive milestones and royalties as Alnylam's LNP enabled products are developed and commercialized. Tekmira is entitled to receive a $5 million milestone payment when ALN-TTR02 enters a pivotal or Phase III clinical trial, which Alnylam has guided should occur by the end of 2013. Tekmira is eligible to receive royalty payments based on commercial sales of ALN-TTR02.
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.