Tekmira Pharmaceuticals Corporation (TSX:TKM) announced today 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 the Company's lead product candidate ApoB SNALP as a treatment for elevated low-density lipoprotein (LDL) cholesterol.
Dr. Mark J. Murray, Tekmira's President and CEO, said, "The filing of the ApoB SNALP IND marks an important transition for Tekmira as our technology moves towards human clinical development and supports Tekmira's leadership position in the advancement of RNAi therapeutics."
The FDA has 30 days to review Tekmira's IND application and to request any additional information before allowing the clinical trial to proceed. Tekmira will provide guidance at the end of the FDA review period about the initiation and the details of the Phase 1 clinical trial, which is expected to begin before mid-year 2009.
Tekmira's therapeutic approach is to address the underlying cause by targeting ApoB, a protein synthesized in the liver that is essential to the assembly and secretion of very low density lipoprotein (VLDL), a precursor to LDL, both of which are required for the transport and metabolism of cholesterol. ApoB SNALP has been shown in preclinical studies to reduce diet-induced high LDL cholesterol, or "bad" cholesterol, and return blood cholesterol levels to normal with a single treatment.
RNAi drugs have the potential to treat human diseases by "switching-off" disease causing genes. The technology, representing one of the most promising and rapidly advancing frontiers in biology and drug discovery, was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi drugs, such as siRNA, require delivery technology to be administered systemically. In preclinical studies, Tekmira's SNALP (stable nucleic acid-lipid particles) technology has been shown to be a safe and effective way to deliver RNAi drugs to disease sites. Tekmira believes it has a leading intellectual property position in the field of siRNA delivery.