Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, reported that Alnylam Pharmaceuticals, Inc. presented clinical results from its completed phase 1 clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR), which utilizes Tekmira's lipid nanoparticle (LNP) technology and is manufactured by Tekmira.
"We are pleased that data from the ALN-TTR01 phase 1 human clinical trial continues to demonstrate that Tekmira's technology is well tolerated and is enabling the development of RNAi products. Tekmira continues to develop and advance its industry-leading LNP delivery platform to enable RNAi therapeutics for a variety of clinical indications," said Dr. Mark J. Murray, Tekmira's president and CEO.
The new data were presented at the XIII International Symposium on Amyloidosis held in Groningen, The Netherlands updating interim data that was released late last year. Alnylam reported results that showed that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein, in ATTR patients. Knockdown of TTR, the disease-causing protein, was found to be dose dependent, rapid, and durable after just a single dose. ALN-TTR was found to be generally safe and well tolerated in this study.
Alnylam is developing a second product candidate called ALN-TTR02, which also utilizes Tekmira's LNP technology and is manufactured by Tekmira. Alnylam has initiated a phase 1 trial with ALN-TTR02 aimed at evaluating safety, tolerability, and clinical activity of ALN-TTR02 in healthy volunteers. Alnylam expects to present data from this study in the third quarter of 2012.
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 "siRNA," 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.