Posted in | Nanomedicine

Alnylam Presents Clinical Data for Patisiran Phase II OLE Study for Familial Amyloidotic Polyneuropathy

Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, today reported that Alnylam Pharmaceuticals, Inc. presented six-month clinical data for the ongoing Patisiran Phase II Open Label Extension (OLE) study in patients with Familial Amyloidotic Polyneuropathy (FAP).

Patisiran (ALN-TTR02) is an RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of TTR-mediated amyloidosis (ATTR), which is enabled by Tekmira's lipid nanoparticle (LNP) technology.

"We are pleased with the positive results reported today in Alnylam's patisiran development program, demonstrating sustained knockdown of serum TTR of up to 90% and a favorable tolerability profile out to one year of treatment. This program is enabled by Tekmira's LNP technology and represents the most clinically advanced application of Tekmira's proprietary LNP delivery technology. Our LNP delivery technology underpins the most significant RNAi advances in the field, enabling multiple RNAi products in clinical development in a variety of therapeutic areas to address significant unmet medical needs. Today's announcement reinforces how our LNP technology continues to be the industry gold standard, and is associated with positive outcomes and clinical benefit," said Dr. Mark J. Murray, Tekmira's President and CEO.

Alnylam presented data at the American Neurological Association's 2014 Annual Meeting being held October 12 - 14 in Baltimore. Results showed a mean 0.95 point decrease in modified Neuropathy Impairment Score (mNIS+7) at six months in 19 patients with mNIS+7 data available for the current analysis. This decrease in neuropathy progression compares favorably with the 7 to 10 point increase in mNIS+7 at six months that can be estimated from historical data sets in untreated FAP patients with similar baseline characteristics (Adams et al., International Symposium on Amyloidosis, April 2014; Berk et al., JAMA 310: 26588-67, 2013; Tafamidis European Medicines Agency Assessment Report, 2011). In addition, patisiran treatment achieved a sustained mean serum TTR knockdown at the 80% target level for over nine months, with an up to 89.6% knockdown achieved between doses. Patisiran was found to be generally well tolerated in this study out to one year of therapy, with no drug-related serious adverse events to date, and all 27 patients enrolled in the study continue to receive drug treatment. Infusion reactions were infrequent, mild and did not result in any discontinuations.


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