May 28, 2014
Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, announced they have met all regulatory requirements to initiate a Phase I/II clinical trial of TKM-PLK1 in patients with Hepatocellular Carcinoma (HCC). Tekmira is also conducting a separate Phase I/II clinical trial evaluating TKM-PLK1 in patients with Gastrointestinal Neuroendocrine Tumors (GI-NET) or Adrenocortical Carcinoma (ACC).
“Hepatocellular carcinoma is associated with one of the poorest survival rates in oncology, and clearly new therapies are needed. We are encouraged with the positive data from our GI-NET and ACC clinical studies with TKM-PLK1 and there is sound rational for evaluating this agent in HCC,” said Dr. Mark J. Murray, Tekmira’s President and CEO.
“We are excited about reaching another important milestone for Tekmira by launching this new clinical trial, and also remain on track to share results from our GI-NET/ACC trial in the second half of this year,” added Dr. Murray.
This trial is an open-label, multi-center, Phase I/II dose escalation study in patients with advanced hepatocellular carcinoma. The study is designed to determine the safety, tolerability and clinical benefit of TKM-PLK1. The study will be conducted at sites in North America and Asia.
About Hepatocellular Carcinoma (HCC)
Hepatocellular carcinoma (HCC) is one of the most common cancers worldwide and the third most common cause of cancer-related death worldwide. HCC is overwhelmingly related to chronic liver disease, particularly hepatitis B and hepatitis C. Patients with HCC usually are asymptomatic until later stages. The prognosis of HCC generally is very poor, with a median survival of six to 20 months and less than five percent of symptomatic patients surviving more than two years. 350,000 to 1 million cases of HCC occur every year worldwide. (source:www.clinicalkey.com)
Tekmira’s lead oncology product candidate, TKM-PLK1, targets polo-like kinase 1 (PLK1), a protein involved in tumor cell proliferation and a validated oncology target. Inhibition of PLK1 expression prevents the tumor cell from completing cell division, resulting in cell cycle arrest and death of the cancer cell. PLK1 has been a target of interest for years, and evidence that patients with elevated levels of PLK1 in their tumors exhibit poorer prognosis and survival rates has been documented in the medical literature. By using an RNAi approach and exploiting its naturally occurring mechanism of action, Tekmira can potentially overcome the limitations of other approaches and effectively silence PLK1.
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.