Posted in | Nanomedicine

Positive Results from Tekmira's Lipid Nanoparticle Technology-Enabled ALN-TTR02 Phase II Trial for ATTR

Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, today reported that Alnylam Pharmaceuticals, Inc. presented positive results from its Phase II clinical trial with 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.

"These positive Phase II results from the ALN-TTR02 program represent the most advanced non-oncology product candidate enabled by our industry-leading LNP delivery technology. These data show that multiple doses of ALN-TTR02 provide rapid, dose-dependent and statistically significant knock-down of TTR protein and that our LNP technology was well tolerated. Alnylam has disclosed plans to initiate a Phase III clinical trial by the end of 2013 that will trigger a $5 million milestone payment to Tekmira. Tekmira's LNP delivery technology continues to lead the RNAi field by enabling multiple products in clinical development in a variety of therapeutic areas," said Dr. Mark J. Murray, Tekmira's President and CEO.

The new data were presented at the Biennial Meeting of the Peripheral Nerve Society being held June 29-July 3 in St. Malo, France. Alnylam reported results from 19 patients that showed significant knock-down of up to 93% of circulating wild-type and mutant TTR in a multi-dose study. Multiple doses of ALN-TTR02 were reported to be generally safe and well tolerated. There were no significant adverse events or discontinuations associated with the drug.

Alnylam disclosed that it intends to present the final data from this ALN-TTR02 Phase II study at the IXth International Symposium on Familial Amyloidotic Polyneuropathy (ISFAP) to be held in Rio de Janeiro, Brazil, November 10-13, 2013. Tekmira will receive a $5 million milestone payment when ALN-TTR02 enters a Phase III clinical trial, which Alnylam has guided should occur by the end of 2013. Tekmira is eligible to receive royalty payments based on commercial sales of ALN-TTR02.

For more detailed information about the Phase II data for ALN-TTR02, please refer to the Alnylam news release dated June 30, 2013 and the presentation of these data, which can be found on Alnylam's website at


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