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Posted: September 8, 2010
Tekmira Pharmaceuticals Receives Clearance From FDA to Initiate TKM-PLK1 Phase 1 Clinical Trial
(Nanowerk News) Tekmira Pharmaceuticals Corporation, a leader in RNA interference (RNAi) therapeutics, today announced that its Investigational New Drug (IND) application for TKM-PLK1 has been cleared by the United States Food and Drug Administration (FDA) allowing Tekmira to initiate a Phase 1 human clinical trial.
Dr. Mark J. Murray, Tekmira's President and CEO, said, "We are pleased to have received FDA approval of our IND for TKM-PLK1. With this approval, we remain on track to achieve the company's milestone of initiating a Phase 1 human clinical trial in patients with advanced solid tumors before the end of the year."
TKM-PLK1 is being developed as a treatment for patients with advanced solid tumor cancers who are not well served by current therapy. The Phase 1 clinical trial will be an open label, non-randomized, dose finding study designed to evaluate the safety, tolerability and pharmacokinetics of TKM-PLK1. TKM-PLK1 targets polo-like kinase 1, or PLK1, a cell cycle protein implicated in tumor cell proliferation and a validated oncology target. Inhibition of PLK1 prevents the tumor cell from completing cell division, resulting in cell cycle arrest and death of the cancer cell. PLK1 has been implicated as a target in a number of significant cancer indications including colorectal, breast, non-small cell lung, and ovarian cancers. These diseases collectively affect over five hundred thousand new patients each year in the United States.
TKM-PLK1 consists of a small interfering RNA (siRNA) designed to silence PLK1 encapsulated in a Tekmira proprietary lipid nanoparticle (LNP) formulation. TKM-PLK1 has been shown in preclinical animal studies to selectively kill cancer cells, while sparing normal cells in healthy tissue. The therapeutic activity of TKM-PLK1 has been demonstrated in preclinical models of liver cancer as well as tumors outside the liver.
TKM-PLK1 is licensed under Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) intellectual property. Additionally, Alnylam has the right to co-develop TKM-PLK1 and retains that right until the start of a TKM-PLK1 Phase 2 clinical trial.
About RNAi and Tekmira's LNP Technology
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. LNP technology is one of the most widely used siRNA delivery approaches for systemic administration. Tekmira's LNP technology (formerly referred to as stable nucleic acid-lipid particles or SNALP) encapsulates siRNAs with high efficiency in uniform lipid nanoparticles which 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 LNP technology. Further information about Tekmira can be found at www.tekmirapharm.com. Tekmira is based in Vancouver, B.C.