Alnylam Pharmaceuticals presents new data from its transthyretin-mediated amyloidosis program

Alnylam PharmaceuticalsNov 2 2009

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it presented new pre-clinical research findings from its transthyretin (TTR)-mediated amyloidosis (ATTR) program at the 60^th Annual Meeting of the American Association for the Study of Liver Diseases (“The Liver Meeting”). Alnylam is developing ALN-TTR, a systemically delivered RNAi therapeutic targeting the TTR gene for the treatment of ATTR, including familial amyloidotic cardiomyopathy (FAC) and familial amyloidotic polyneuropathy (FAP). There are more than 100 mutations that have been identified in the TTR gene. ALN-TTR targets a region of the gene common to wild-type and all known mutant forms of TTR, and therefore, has potential as a therapeutic for all patients with FAC and FAP.

“We are very encouraged by these significant and important pre-clinical findings, which continue to validate the potential benefit of an RNAi therapeutic targeting TTR for the treatment of ATTR,” said Dinah Sah, Ph.D., Vice President, Research, CNS and Oncology at Alnylam. “Our new in vivo studies represent a significant step forward as they demonstrate the ability of ALN-TTR to silence the TTR gene for a period of weeks after a single dose administration. These data provide continued validation of our RNAi therapeutics strategy, and we are looking forward to advancing this program towards the clinic.”

The new pre-clinical research findings presented at the meeting demonstrated dose-dependent ALN-TTR reduction of liver TTR messenger (mRNA) and serum TTR protein levels by greater than 80% in transgenic mice and non-human primates, with gene silencing effects found to be durable for more than three weeks following a single dose administration.

“I am quite excited by these new data, which strongly support the advancement of this program towards patients who have extremely limited therapeutic options,” said Maria Joao Saraiva, Ph.D., Professor of Biochemistry, Molecular Neurobiology Group, Institute for Molecular and Cellular Biology in Portugal. “ATTR, which presents severe manifestations in both FAP and FAC, is estimated to affect approximately 50,000 people worldwide and is associated with significant morbidity, including intractable peripheral sensory neuropathy, disabling dysfunction of the autonomic nervous system that leads to severe intestinal dysfunction, and in many cases severe cardiomyopathy.”

Alnylam expects to file regulatory applications for ALN-TTR by the end of 2009 with a goal of initiating a Phase I clinical trial in early 2010. ALN-TTR is being advanced using stable nucleic acid-lipid particles (SNALP) delivery technology in collaboration with Tekmira Pharmaceuticals Corporation.