-- Research Highlights Include Potential New Therapeutic Applications for RNAi Therapeutics in Liver Fibrosis --
CAMBRIDGE, Mass., Oct 29, 2010 (BUSINESS WIRE) -- Alnylam Pharmaceuticals, Inc. /quotes/comstock/15*!alny/quotes/nls/alny (ALNY 13.14, -0.15, -1.13%) , a leading RNAi therapeutics company, announced today it will present several poster presentations at the 61st 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," 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.
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is applying its therapeutic expertise in RNAi to address significant medical needs, many of which cannot effectively be addressed with small molecules or antibodies, the current major classes of drugs. Alnylam is leading the translation of RNAi as a new class of innovative medicines with peer-reviewed research efforts published in the world's top scientific journals including Nature, Nature Medicine, and Cell. The company is leveraging these capabilities to build a broad pipeline of RNAi therapeutics for the treatment of a wide range of disease areas, including respiratory syncytial virus (RSV), liver cancers, TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntington's disease. In addition, Alnylam formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies. The company's leadership position in fundamental patents, technology, and know-how relating to RNAi has enabled it to form major alliances with leading companies including Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. Alnylam and Isis are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development, and commercialization of microRNA therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit http://www.alnylam.com/.
Alnylam Forward-Looking Statement
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, its expectations regarding the development of effective and efficient delivery approaches for RNAi therapeutics, including through delivery of RNAi therapeutics to hepatic stellate cells, and its expectations regarding the progress of its ongoing research and development programs for its RNAi therapeutic candidates, including ALN-VSP, ALN-TTR01, and ALN-PCS, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including Alnylam's ability to continue advancing its delivery efforts, including its efforts around delivery to hepatic stellate cells, discover and develop novel drug candidates, and successfully demonstrate efficacy and safety of its drug candidates, including ALN-VSP and ALN-TTR01, in human clinical trials, as well as those risks more fully discussed in the "Risk Factors" section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.
SOURCE: Alnylam Pharmaceuticals, Inc.
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