Tekmira Pharmaceuticals Corporation, a leader in RNA interference (RNAi) therapeutics, announced it has filed an Investigational New Drug (IND) application seeking approval from the United States Food and Drug Administration (FDA) to begin a phase 1 human clinical trial to evaluate PLK1 SNALP as a treatment for solid tumour cancers.

Dr Mark J. Murray, Tekmira's president and CEO, said, “With the filing of our PLK1 SNALP IND, we are on track to begin a phase 1 human clinical trial with our second RNAi clinical product candidate in the second half of this year. This is another important milestone for Tekmira as we continue to both advance our own pipeline of RNAi drug candidates and support our partners as they move forward with their own RNAi programmes using our leading SNALP delivery technology.”

The FDA has 30 days to review Tekmira’s IND application for PLK1 SNALP and to request any additional information before permitting the clinical trial to proceed. At the end of the FDA review period, Tekmira will provide additional details of the PLK1 SNALP phase 1 human clinical trial, which is expected to begin in the second half of 2010.

Tekmira’s therapeutic approach to treating cancer is to target PLK1 (polo-like kinase 1), a protein involved in tumour cell proliferation and a validated oncology target. In preclinical studies, PLK1 SNALP displayed potent and specific anti-tumour effects in a variety of tumour models in animals including preclinical models of tumours outside the liver. Tekmira’s work on PLK1 SNALP was published in the Journal of Clinical Investigation (Judge et al, Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics. JCI. 23 Feb 09).

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. Lipid nanoparticles (LNPs) are one of the most widely used siRNA delivery approaches for systemic administration. Tekmira’s SNALP (stable nucleic acid-lipid particles) technology is the leading class of LNPs being used in clinical development. SNALP technology encapsulates siRNAs with high efficiency in uniform lipid nanoparticles which are effective in delivering RNAi therapeutics to disease sites in numerous preclinical models. SNALP formulations are manufactured by a proprietary method which is robust, scalable and highly reproducible. SNALP-based products have been reviewed by multiple FDA divisions for use in clinical trials. SNALP 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.