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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced the publication of new data in the journal Molecular Therapy by Alnylam scientists and collaborators from the David H. Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology (MIT). These new data describe the further development of lipidoid formulations for the systemic delivery of small interfering RNAs (siRNAs), the molecules that mediate RNAi.
"We are very encouraged with the progress we have made with systemic delivery of siRNAs since the continued advancements we and our collaborators are making with this technology is enabling the ability to address the broadest potential of RNAi therapeutics," said Akin Akinc, Ph.D., Associate Director, Research at Alnylam. "Formulations based on lipid and lipid-like materials called ‘lipidoids' show great promise for the systemic delivery of RNAi therapeutics. These new findings further expand our understanding of lipidoids as part of our ongoing efforts to optimize these formulations to maximize efficacy in vivo."
Lipidoids are a new class of lipid-based molecules which are used to form novel nanoparticle formulations for systemic delivery of RNAi therapeutics. A previous study by Alnylam scientists in collaboration with scientists from MIT (Akinc et al., Nature Biotechnology 26, 561-569 (01 May 2008)), showed successful delivery of siRNAs encapsulated in lipidoid formulations when administered in multiple animal species including mice, rats, and non-human primates.
Data published from this new study (Akinc et al., Molecular Therapy advance online publication 3 March 2009; doi:10.1038/mt.2009.36) extends the work from the previous study by:
* identifying key parameters affecting the pharmacodynamics of this type of formulation, including increasing the anchor length of synthesized PEG lipids, maximizing siRNA loading, and reducing particle size to more efficiently access hepatocytes;
* demonstrating that lipidoid formulations achieve delivery of greater than 90% of the administered siRNA dose to the liver and maintain robust in vivo activity following repeat administration over a period of several months, and indicating no evidence of neutralizing antigenicity or tachyphylaxis; and,
* characterizing the long-term stability of the formulation.
Lipidoid formulations represent one of several approaches Alnylam is pursuing for systemic delivery of RNAi therapeutics. Additional approaches include other lipid nanoparticle formulations and siRNA conjugation strategies. Recently, Alnylam received clearance from the U.S. Food and Drug Administration (FDA) for its investigational new drug (IND) application to initiate a Phase I study for its ALN-VSP liver cancer program, which it expects to begin enrolling in the first half of 2009. ALN-VSP employs a lipid nanoparticle formulation known as "SNALP" developed in collaboration with Tekmira Pharmaceuticals Corporation.
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. RNAi therapeutics target the cause of diseases by potently silencing specific messenger RNAs (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, Inc.
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; its most advanced program is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection and is partnered with Cubist and Kyowa Hakko. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including liver cancers, hypercholesterolemia, Huntington’s disease, and TTR amyloidosis. 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, and Cubist. To reflect its outlook for key scientific, clinical, and business initiatives, Alnylam established “RNAi 2010” in January 2008 which includes the company’s plan to significantly expand the scope of delivery solutions for RNAi therapeutics, have four or more programs in clinical development, and to form four or more new major business collaborations, all by the end of 2010. 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 www.alnylam.com.
About the Koch Institute
The MIT Center for Cancer Research (CCR) has changed its name to the David H. Koch Institute for Integrative Cancer Research at MIT (Koch Institute – pronounced “coke”) effective March 2008. This name change is linked to generous funding received in support of the creation of a new building and endeavor, to be completed by the year 2010, to house expanded and innovative cancer research at MIT. Note that all CCR facilities and faculty members have been incorporated into the Koch Institute.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, 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 the company’s ability to successfully research and develop products, such as ALN-VSP for the treatment of liver cancers, as well as those risks more fully discussed in the “Risk Factors” section of its most recent annual report on Form 10-K 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.
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Alnylam Pharmaceuticals, Inc.
Cynthia Clayton (Investors)
Yates Public Relations
Adriana Jenkins (Media)
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