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 News Release - January 11, 2010

Alnylam Provides Key Goals and Guidance for Next 12 to 18 Months

Expects Four RNAi Therapeutic Programs in Clinical Development: ALN-RSV, ALN-VSP, ALN-TTR, and ALN-PCS

Will Continue to Advance Major Breakthroughs in Delivery Technology and Science

Expects to Form New Partnerships and Create New Business Opportunities

CAMBRIDGE, Mass.--(HSMN NewsFeed)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY ), a leading RNAi therapeutics company, announced today its key pipeline, scientific and business goals for the next 12 to 18 months, including 2010 financial guidance.

“We believe that we are entering a period that may be viewed as an ‘RNA Decade,’ where the world’s scientific understanding of the roles of RNA is redefining modern biology and where the industry’s advancement of RNA-based therapeutics holds the potential to transform medicine,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “In this new era, Alnylam is extremely well positioned to build a significant biopharmaceutical company, which remains our mission and our passion. Our scientific leadership, particularly related to delivery of RNAi therapeutics, is now driving a growing Alnylam pipeline of innovative medicines that can address human disease in a fundamentally new way. In fact, these efforts place Alnylam’s multiple RNAi therapeutic programs on the frontier of demonstrating human proof of concept, a key milestone for the entire drug class. Further, our intellectual property estate remains unmatched and, together with our scientific and pipeline progress, will fuel continued formation of new partnerships and new business opportunities.”

“Accomplishments in 2009 reflect the substantial progress we are making as we continue to lead the industry’s efforts in the discovery and development of RNAi therapeutics, and we are even more excited about the next 12 to 18 months as we also fulfill the ‘RNAi 2010’ goals that we described two years ago,” said Barry Greene, President and Chief Operating Officer of Alnylam. “Over this period, we expect to have four RNAi therapeutic programs in clinical development – ALN-RSV, ALN-VSP, ALN-TTR, and ALN-PCS – providing us with the opportunity to demonstrate human proof of concept for multiple programs. Further, we expect to continue our leadership in RNAi with continued delivery advances. We also expect to form new partnerships across our efforts at Alnylam, Regulus, and through our Biotherapeutics initiative, as there remains significant interest across the industry in RNA therapeutics based on Alnylam’s scientific and intellectual property leadership.”

“We are on track to meet our guidance of ending 2009 with a cash position of greater than $430 million, a financial position that allows us to invest in the advancement of our platform and pipeline of novel RNAi therapeutic products,” said Patricia Allen, Vice President, Finance and Treasurer of Alnylam. “We expect to finish 2010 with greater than $325 million in cash, which excludes the potential payment from Novartis should they decide to execute their adoption license later this year.”

Pipeline Goals

  • Advance ALN-RSV01 in Phase IIb Clinical Study for the Treatment of Respiratory Syncytial Virus (RSV) Infection. Alnylam plans to advance ALN-RSV01 in a new Phase IIb clinical trial in RSV-infected adult lung transplant patients, and expects to begin enrollment in this study in the first quarter of 2010. The objective of this study is to repeat and extend the clinical results observed in a recently completed Phase IIa study, also in the lung transplant patient population. Alnylam is funding the continued advancement of ALN-RSV01, and its partner Cubist has retained an opt-in right for the product in the adult transplant indication. Cubist is taking the lead in advancing ALN-RSV02 in the pediatric setting in continued collaboration and 50-50 funding with Alnylam. The ALN-RSV program is partnered with Kyowa Hakko Kirin Co., Ltd. in Asia, and Cubist worldwide except Asia.
  • Continued Clinical Development of ALN-VSP for the Treatment of Liver Cancers. Alnylam continues to enroll patients in a Phase I multi-center, open label, dose escalation trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALN-VSP in patients with advanced solid tumors with liver involvement, including hepatocellular carcinoma (HCC). A significant number of patients have been enrolled across multiple dose cohorts, and Alnylam expects to present preliminary data from the Phase I trial in mid-2010. This is the company’s first systemic RNAi program, and represents Alnylam’s first clinical program in an oncology indication.
  • Advance ALN-TTR Program for Transthyretin (TTR)-Mediated Amyloidosis (ATTR) in Phase I Clinical Study. Alnylam has filed regulatory applications for ALN-TTR01 and plans to initiate a Phase I trial in ATTR patients in the first half of 2010. ALN-TTR01 is a systemically delivered RNAi therapeutic that employs first-generation lipid nanoparticles (LNPs). Pre-clinical studies have demonstrated potent and durable silencing of both the normal and mutated TTR gene in rodents and non-human primates. Further, administration of the RNAi therapeutic has been shown to reduce the pathogenic accumulation of mutant TTR in peripheral tissues in studies performed in a transgenic mouse model. In parallel, Alnylam is also advancing ALN-TTR02, which utilizes second-generation LNPs.
  • Expand Clinical Development Pipeline with ALN-PCS for the Treatment of Hypercholesterolemia. Alnylam expects to advance its ALN-PCS program toward the clinic with a goal of initiating a Phase I clinical trial in 2011. ALN-PCS is a systemically delivered RNAi therapeutic targeting the gene PCSK9, which is involved in the metabolism of LDL (“bad”) cholesterol. The role of PCSK9 in LDL metabolism has been validated in human genetic studies. Pre-clinical studies with ALN-PCS have demonstrated a greater than 50% reduction in levels of LDL cholesterol in non-human primates. ALN-PCS is being advanced using second-generation LNPs for systemic delivery.
  • Advance Additional Pre-Clinical RNAi and microRNA Therapeutic Programs. Alnylam also expects to advance additional pre-clinical programs, including ALN-RSV02, in collaboration with Cubist, for the treatment of RSV infection in the pediatric setting; ALN-HTT, in collaboration with Medtronic, for the treatment of Huntington’s disease; amongst several additional RNAi therapeutic programs. Further, Alnylam is advancing a pipeline of microRNA therapeutics through its co-ownership of Regulus Therapeutics Inc.

Scientific Goals

  • Expand Platform for Delivery of RNAi Therapeutics. Alnylam plans to continue advancing and developing novel delivery solutions, on its own and through its multiple industry and academic collaborations, for the systemic delivery of RNAi therapeutics.
  • Continue Scientific Leadership in RNAi Therapeutics with 15 or More Peer-Reviewed Papers. Alnylam intends to continue its leading scientific efforts through the publication of key findings related to RNAi therapeutics and the company’s broader technology platform, including microRNA therapeutics, in high-quality, peer-reviewed scientific journals.

Business and Intellectual Property Goals

  • Form New Partnerships. Alnylam expects to form additional new alliances, which could include: platform alliances, such as the company’s partnerships with Takeda and Roche; product alliances, such as those the company has formed with Medtronic, Kyowa Hakko Kirin, and Cubist; alliances with Regulus Therapeutics, such as that formed with GlaxoSmithKline; alliances with the Alnylam Biotherapeutics initiative; as well as the formation of new business ventures.
  • Continue Intellectual Property (IP) Dominance with Greater than 30 New Patent Grants. Alnylam expects to further strengthen its dominant IP position by receiving greater than 30 additional patent issuances or grants from patent offices around the world.
  • Maintain Solid Financial Performance. Alnylam is on track to achieve a 2009 year-end cash position of greater than $430 million. In 2010, Alnylam aims to maintain its solid financial position and expects to end the year with greater than $325 million in cash. This guidance excludes the potential payment from Novartis should they decide to execute their adoption license later this year.

About RNAi 2010

In January 2008, Alnylam launched its ‘RNAi 2010’ plan as a new initiative focused on achieving major scientific, clinical, and business milestones by the end of 2010. The specific objectives of the RNAi 2010 plan that were established at the beginning of 2008 were as follows:

  • Scientific Leadership. By the end of 2010, Alnylam expects to broaden its leadership and significantly expand the scope of delivery solutions for RNAi therapeutics. This will be achieved by the continued scientific leadership of Alnylam scientists and current academic and industry collaborators, but also a significant external effort to form new delivery technology partnerships. Further, this effort will include the significant expansion of the range of tissues and cell types where the company aims to achieve efficient delivery of RNAi therapeutics with both direct and systemic delivery approaches.
  • Clinical Pipeline. By the end of 2010, Alnylam expects to have four or more RNAi therapeutic programs in clinical development. These include direct and systemic RNAi programs, Alnylam proprietary and 50-50 partnership programs, and siRNA and microRNA therapeutics.
  • New Business Collaborations. Based on its scientific, clinical, and intellectual property leadership, the company also expects to form four or more new major business collaborations by the end of 2010. These are expected to include the completion of additional broad platform alliances similar to the company’s July 2007 partnership with Roche. Completion of these business collaborations is expected to provide the company significant resources and funding to advance Alnylam’s proprietary and 50-50 partnership pipeline programs.

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 and is partnered with Cubist and Kyowa Hakko Kirin. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including liver cancers, TTR amyloidosis, 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. 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-based therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit

Alnylam Forward-Looking Statement

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, the need for novel RNAi therapeutics, Alnylam’s views with respect to the potential for RNAi therapeutics, including ALN-RSV01, ALN-VSP, ALN-TTR, and ALN-PCS, and its expectations with respect to the timing and success of its clinical and pre-clinical trials, including its plan to initiate clinical trials for ALN-RSV01, ALN-TTR, and ALN-PCS, the timing of regulatory filings, its expectations regarding the development of efficient delivery of RNAi therapeutics, the formation of new alliances, its cash position at the end of 2009 and 2010, and its ability to continue to generate revenue through existing and new alliances, 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; the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to enforce its patents against infringers and to defend its patent portfolio against challenges from third parties; Alnylam’s ability to obtain additional funding to support its business activities; Alnylam’s dependence on third parties for development, manufacture, marketing, sales and distribution of products; obtaining regulatory approval for products; competition from others using technology similar to Alnylam’s and others developing products for similar uses; Alnylam’s dependence on current and future collaborators; and Alnylam’s short operating history; 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

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