Alnylam Publishes Results from Phase II GEMINI Trial with ALN-RSV01

CAMBRIDGE, Mass., Apr 27, 2010 (BUSINESS WIRE) -- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has published results from its Phase II GEMINI study in the Proceedings of the National Academy of Sciences (PNAS) (DOI: 10.1073/pnas.0912186107). These data, previously presented at the International Symposium on Respiratory Viral Infections in Singapore in 2008, showed human proof of concept with an RNAi therapeutic in a randomized, double blind, placebo-controlled study. In this study, treatment with ALN-RSV01 showed statistically significant anti-viral activity, including a decreased RSV infection rate and an increase in the number of subjects who remained free of infection. "The positive results generated from GEMINI established encouraging human proof of concept with an RNAi therapeutic," said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research at Alnylam. "GEMINI also set the stage for us to advance ALN-RSV01 into the naturally RSV-infected adult lung transplant population where we are currently conducting a Phase IIb clinical study. We are encouraged by the cumulative data to date in this program and look forward to the program's continued advancement." GEMINI was a double blind study, where 88 adult subjects experimentally infected with RSV were randomized to receive ALN-RSV01 or placebo. Administration of ALN-RSV01 resulted in statistically significant anti-viral activity with a 38% relative reduction in RSV infection rate (p<0.01) resulting in a 95% increase in the number of infection-free subjects as compared with placebo (p<0.01). ALN-RSV01 was also found to be safe and well tolerated. "Results from the GEMINI study are notable, as they established anti-viral activity for an RNAi therapeutic in a major respiratory disease and provide a solid foundation for ongoing development of ALN-RSV01," said John P. DeVincenzo, M.D., Professor of Pediatrics and Infectious Diseases and Professor of Molecular Sciences at the University of Tennessee Health Science Center and Principal Investigator for the GEMINI study. "I am enthusiastic about the continued development of RNAi therapeutics for the treatment of RSV infection, a major infectious disease in both adult and pediatric populations where there are no effective treatments used widely today." Earlier this year, Alnylam initiated a randomized, double blind, placebo-controlled Phase IIb trial of ALN-RSV01 in RSV-infected adult lung transplant patients. The primary endpoint of this study is the incidence of new or progressive bronchiolitis obliterans syndrome (BOS), a life-threatening complication of RSV infection in the transplanted lung resulting in approximately 50% mortality within three to five years of onset. The Phase IIb study is expected to enroll up to 76 patients. The ALN-RSV program is partnered with Kyowa Hakko Kirin Co., Ltd. in Asia, and Cubist Pharmaceuticals, Inc. worldwide except Asia. In parallel with Alnylam's development of ALN-RSV01 for the treatment of RSV in lung transplant patients, Alnylam and Cubist are developing a second-generation compound, ALN-RSV02, which will be focused on the pediatric patient population. Cubist will take the lead in advancing ALN-RSV02 in the pediatric setting in continued collaboration and 50-50 funding with Alnylam, and Cubist retains an opt-in right for ALN-RSV01 in the adult transplant indication. 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; its most advanced program is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including liver cancers, TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntington's disease. 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-based therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com . Alnylam Forward-Looking Statement 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 risks related to: Alnylam's approach to discover and develop novel drugs, which is unproven and may never lead to marketable products, including ALN-RSV01 or any successor product thereto; whether pre-clinical testing or early clinical trials of a product candidate, including ALN-RSV01, are predictive of the results that will be obtained in subsequent human clinical trials; Alnylam's dependence on third parties for development, manufacture, marketing, sales and distribution of products, including Alnylam's partners for its ALN-RSV program, Cubist and Kyowa Hakko Kirin; obtaining regulatory approval for products in different jurisdictions; and competition from others using technology similar to Alnylam's and others developing products for similar uses; 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. SOURCE: Alnylam Pharmaceuticals, Inc. Alnylam Pharmaceuticals, Inc. Cynthia Clayton (Investors), 617-551-8207 or Spectrum Amanda Sellers (Media), 202-955-6222 x2597 Copyright Business Wire 2010

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