Alnylam to present data on RNAi therapeutics for fibrotic diseases at The Liver Meeting

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today it will present several poster presentations at the 61^st Annual Meeting of the American Association for the Study of Liver Diseases ("The Liver Meeting") being held in Boston, Mass from October 29 - November 2, 2010. At the meeting, new research related to the company's pre-clinical and clinical pipeline efforts will be presented, including new data showing effective delivery of RNAi therapeutics to hepatic stellate cells. Pre-clinical data will also be presented on Alnylam's key development programs, including ALN-TTR01 for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-VSP for the treatment of liver cancers, and ALN-PCS for the treatment of hypercholesterolemia.

“The data we are presenting at this meeting highlight the significant progress we are making in advancing RNAi therapeutics to patients”

"The data we are presenting at this meeting highlight the significant progress we are making in advancing RNAi therapeutics to patients," said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research. "It is notable that we are now showing effective systemic delivery to stellate cells, creating a potentially new approach for the treatment of liver fibrosis with siRNAs targeting collagen 1a1. We are also pleased with additional updates from some of our key clinical development programs, including ALN-VSP, ALN-TTR01, and ALN-PCS."

In a poster titled "Liposome Mediated Delivery of siRNA to Hepatic Stellate Cells," Alnylam scientists will present new data on the systemic delivery of RNAi therapeutics to hepatic stellate cells (HSCs). HSCs play a key role in the initiation and progression of liver fibrosis, the excessive accumulation of tough, fibrous scar tissue that occurs in most types of chronic liver diseases. These new data show that siRNAs formulated in LNPs comprising the novel cationic lipid "C12-200" result in effective silencing of the HSC-specific gene target, collagen 1a1 (col1a1). These data point to a potential strategy for development of RNAi therapeutics for the treatment of fibrotic diseases.

Other presentations from Alnylam scientists at The Liver Meeting include the following:

• a poster titled, "Carbohydrate Conjugation to siRNA for Liver-Specific Delivery" will describe progress made on improving stability and activity of GalNAc-conjugated siRNAs in vitro and their translation to improved in vivo gene silencing efficacy;
• a poster titled, "RNAi Therapeutics Targeting PCSK9 Result in Significant and Durable LDLc Lowering" will highlight pre-clinical data from the company's PCSK9 program which blocks the production of both intracellular and extracellular PCSK9 and uses a second generation LNP formulation;
  • new data included in the poster will show the ability to utilize siRNA combination approaches to achieve efficient silencing of several genes at one time to achieve improved cholesterol lowering;
  • in addition, new data will also be presented showing that lowering of PCSK9 in an LDLR heterozygous animal model of familial hypercholesterolemia results in a significant lowering of total cholesterol;
• a poster titled, "ALN-TTR, an RNAi Therapeutic for the Treatment of Transthyretin (TTR) Amyloidosis, Mediates Regression of Peripheral TTR Protein" will highlight pre-clinical data for the company's ALN-TTR01 program, which is currently in a Phase I clinical trial in patients with ATTR; and,
• a poster titled, "ALN-VSP, an Experimental RNAi Therapeutic, Demonstrates Efficacy in Tumor Model Studies" will review pre-clinical data for Alnylam's ALN-VSP program, which is currently in a Phase I clinical trial in patients with advanced solid tumors with liver involvement. Alnylam plans on providing an additional clinical update on the ALN-VSP program by the end of 2010.