Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, announced that its collaboration partner, the National Cancer Institute (NCI), will present preclinical data at the annual meeting of the American Association for Cancer Research (AACR) in a poster session beginning at 8:00 am ET today entitled "Nanoparticle-Based Combinatorial siRNA Therapy against Human Hepatocellular Carcinoma (HCC)."
"In addition to the encouraging results from our Phase I TKM-PLK1 clinical trial that were presented yesterday at AACR, our collaborators at the NCI have identified the novel cancer genes WEE1 and CSN5 from human tumor samples, and together we have generated promising preclinical data with this combination by leveraging our expertise in siRNA design and delivery," said Dr. Mark J. Murray, Tekmira's President and CEO.
The preclinical results summarized in the poster indicate that a statistically significant decrease in tumor size was observed in the animals treated with either CSN5 siRNA or a combination of CSN5 and WEE1 siRNA. The results also indicate that the treatment does not affect the global gene expression in the surrounding liver following siRNA therapy compared to that seen in the tumor tissue, supporting the tumor-specific nature of the effect. Tekmira has an ongoing collaboration with the NCI to identify novel cancer genes for RNAi applications to meet unmet needs.
Tekmira is currently evaluating several preclinical candidates with potential in diverse therapeutic areas. The Tekmira team will continue to generate data to support the advancement of the most promising of these and expects to nominate the next product candidate for development in 2013.
The Phase I TKM-PLK1 data were presented yesterday at the AACR Annual Meeting 2013 in an oral presentation entitled "A phase I dose escalation study of TKM-080301, a RNAi therapeutic directed against PLK1, in patients with advanced solid tumors."
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.