Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, has reported that Alnylam Pharmaceuticals, Inc. presented positive results from its phase II clinical trial with patisiran (ALN-TTR02), an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR), which is enabled by Tekmira's lipid nanoparticle (LNP) technology. The programme represents the most clinically advanced application of Tekmira's proprietary LNP delivery technology.
"Today's most significant RNAi advances within the clinic are driven by Tekmira's LNP technology. We are pleased to report patisiran (ALN-TTR02), which is enabled by Tekmira's leading LNP technology, has entered a phase III clinical trial. Our LNP delivery technology continues to be validated by positive clinical data, with these phase II results demonstrating a product that is effective, safe and well tolerated. Our LNP is enabling multiple RNAi products in clinical development in a variety of therapeutic areas, including our own robust product pipeline, as well as partner products," said Dr Mark J Murray, Tekmira's president and CEO.
Alnylam presented data at the IXth International Symposium on Familial Amyloidotic Polyneuropathy (ISFAP) being held in Rio de Janeiro, Brazil, November 10-13. Alnylam reported results showing that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96 per cent, with mean levels of TTR knockdown exceeding 85 per cent. Knockdown of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein. In addition, patisiran was found to be generally safe and well tolerated in this study.
Alnylam also announced the initiation of the APOLLO phase III trial of patisiran, with the study now open for enrollment, to evaluate efficacy and safety of patisiran in ATTR patients with Familial Amyloidotic Polyneuropathy (FAP). Tekmira is entitled to receive a US$ 5M milestone payment upon dosing of the first patient within this trial.
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.