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Alnylam Pharmaceuticals, Inc. (ALNY) Alnylam is a biopharmaceutical company seeking to develop and commercialize new drugs that work through a recently discovered system in cells known as RNA interference, or RNAi. We believe that drugs that work through RNA interference, or RNAi therapeutics, have the potential to become a major class of drugs, like small molecule, protein and antibody drugs. Using our intellectual property and the expertise we have built in RNAi, we are developing a set of biological and chemical methods and know how that we expect to apply in a systematic way to develop RNAi therapeutics for a variety of diseases. We refer to these methods and their systematic application as our “product engine”. Using our current capabilities, we have initiated programs to develop RNAi therapeutics that will be administered directly to diseased parts of the body, which we refer to as Direct RNAi therapeutics. We believe there are multiple opportunities for Direct RNAi therapeutics. Our current Direct RNAi programs are focused on an eye disease known as age-related macular degeneration, or AMD, and on a central nervous system disorder known as Parkinson’s disease, or PD. We expect to initiate a clinical trial for our lead AMD product candidate in 2005. We are also working to extend our capabilities to enable the development of RNAi therapeutics that travel through the blood stream to reach diseased parts of the body, which we refer to as Systemic RNAi therapeutics. We believe Systemic RNAi will be used to treat a broad range of diseases. RNAi is a recently discovered natural mechanism for selectively silencing genes. Genes provide cells with coded instructions for making proteins, and silencing a gene refers to stopping or reducing production of the protein specified, or encoded, by that gene. Our goal is to develop new drugs that use the RNAi mechanism to selectively silence genes encoding proteins that play harmful roles in disease. We intend to develop drugs based on a type of molecule known as small interfering RNA, or siRNA. siRNAs are the molecules within cells that directly trigger RNAi. We expect that our RNAi therapeutics will consist of chemically modified siRNAs designed to silence specific genes. Given the recent availability of the base sequence of the entire human genome, RNAi therapeutics can be designed, in theory, to silence any gene that encodes a protein involved in disease, even if currently this protein cannot be adequately controlled by conventional drugs. The scientific evidence to support the feasibility of developing drugs based on RNAi technology is both preliminary and limited. siRNAs do not naturally possess many of the properties required of drugs, such as the ability to survive in the blood stream and penetrate into diseased tissues. The methods we are developing for our product engine need to introduce these drug-like properties into siRNAs without making these siRNAs unsafe or ineffective. We do not currently know whether we will be successful in developing these methods. Very few drug candidates based on RNAi technology have been tested in animals, and none has been tested in humans. We believe that we have a strong intellectual property position relating to the development and commercialization of siRNAs as therapeutics, consisting of: • a concentration of intellectual property rights claiming fundamental features of siRNAs and their use as therapeutics, which includes our ownership of, or exclusive rights to, several issued patents and pending patent applications; • a broad portfolio of intellectual property relating to chemical modifications of siRNAs, including over 150 patents licensed from Isis Pharmaceuticals, Inc.; and • a number of pending patent applications claiming siRNAs directed to specific targets as treatments for particular diseases. We have filed or licensed over 200 patents and patent applications in the RNAi field. Nevertheless, we may need to acquire additional intellectual property rights in order to develop our products. In addition, our intellectual property rights may not be sufficient to prevent other companies from developing products that compete with any products we may develop. Our goal is to develop and commercialize RNAi therapeutics. To access the substantial funding and expertise required to develop and commercialize RNAi therapeutics, we intend to form strategic collaborations with pharmaceutical companies. In the collaborations we form in the near term, we expect to take the lead role in discovery and early preclinical development of specific RNAi therapeutics, and to share responsibilities with our collaborators in later-stage development and commercialization of these RNAi therapeutics. We expect that our collaborators will provide us with significant funding for the work we perform, access to their development and commercial capabilities, and a share of revenues from products we originate. Our first such alliance is with Merck & Co., Inc. Over time, as we expand our capabilities and resources, we expect the nature of the collaborations we form will evolve, so that we take on progressively more responsibility for development and commercialization of products we originate, and retain a greater share of the revenues these products generate. In the longer term, we expect to develop and commercialize RNAi therapeutics independently. Our ability to form appropriate collaborations with pharmaceutical companies will be crucial for our success. The development and commercialization of drugs often takes a decade or more to complete and requires significant expenditures. It is unlikely that we will be able to afford the time or expense involved in the development and commercialization of drugs unless we secure suitable collaborations with pharmaceutical companies. If we are unable to form or to maintain such collaborations, or our collaborators do not perform as we expect them to, we may not be able to develop and commercialize RNAi therapeutics. Moreover, the pharmaceutical marketplace is highly competitive, with hundreds of companies pursuing the same or very similar product opportunities. Even if we do succeed in developing RNAi therapeutics, we may not be able to compete effectively in the marketplace. Our scientific founders were among the first scientists to publish significant discoveries in the field of RNAi. In addition, we have assembled an experienced management team to implement our business plan. Members of our management team have led discovery, development and commercialization programs for a number of marketed drugs, including Aranesp, Neulasta, Angiomax and Velcade. Potential for RNAi Therapeutics Proteins perform many of the vital functions of the cell and of the human body. Although the roles they play are generally beneficial, in certain circumstances, proteins can be harmful. Many human diseases are caused by the inappropriate behavior of proteins. A particular protein may, for example, be present in too great a quantity, be too active, or appear in the wrong place or at the wrong time. In these circumstances, the ability to stop or reduce production of the protein by selectively silencing the gene that directs its synthesis could be very beneficial in the treatment of the disease. Beginning in 1999, our scientific founders were the first to describe and provide evidence that the RNAi mechanism occurs in mammalian cells and that its immediate trigger is a type of molecule known as small interfering RNA, or siRNA. They showed that laboratory-synthesized siRNAs could be introduced into the cell and suppress production of specific target proteins. Because it is possible, in theory, to design and synthesize siRNAs specific for any gene of interest, we believe that RNAi therapeutics have the potential to become a broad new class of drugs. We believe that RNAi therapeutics could offer the following benefits: • Ability to treat a broad range of diseases. Given the availability of the base sequence of the entire human genome, in theory, it should be possible to design siRNAs to suppress the production of virtually any human protein whose presence or activity causes disease. • Ability to target proteins that cannot be targeted effectively by existing drug classes. Many proteins that play important roles in disease cannot be targeted effectively with small molecules and therapeutic proteins, including monoclonal antibodies. In theory, siRNAs should not face the same limitations as these drug classes and, therefore, we believe RNAi therapeutics will be able to target proteins that small molecule and protein drugs cannot currently target. • An inherently potent mechanism of action. Each siRNA molecule can trigger destruction of multiple messenger RNA molecules, each of which could otherwise direct the synthesis of many protein molecules. As a result, one siRNA molecule may effectively be able to block the activity of many protein molecules. This inherent potency of the RNAi mechanism suggests a potentially high degree of potency for RNAi therapeutics. • Simplified discovery of drug candidates. Identification of siRNA drug candidates has the potential to be much simpler and take considerably less time than other drug classes because, in theory, it will involve a relatively standard process that can be applied in a similar fashion to many successive product candidates. None of these potential benefits has yet been proven. If we are unable to design siRNAs with sufficient stability and the ability to enter tissues and cells, we will not be able to develop safe and effective RNAi therapeutics. Our Business Strategy Our strategy is to use our strong intellectual property position and our expertise in RNAi to develop and commercialize RNAi therapeutics. The key elements of our business strategy are as follows: • Pursue product opportunities in a phased approach based on the evolving capabilities of our product engine. We are implementing a phased approach to product development that we believe will allow us to initiate product development activities in three main phases: — Direct RNAi Therapeutics: We intend to utilize the current capabilities of our product engine by focusing our efforts on developing RNAi therapeutics that can be administered directly at diseased parts of the body, such as the eye or the brain. As part of this phase, we have initiated Direct RNAi programs focused on AMD and PD. — Systemic RNAi Therapeutics for liver-based diseases: As we extend the capabilities of our product engine, we intend to develop RNAi therapeutics for liver-based diseases because it appears, on the basis of early preclinical evidence, that the liver takes up siRNAs more readily than other tissues. — Systemic RNAi Therapeutics for other diseases: As a third phase of our product development strategy, we will seek to develop RNAi therapeutics that exert their effects in tissues other than the liver. We believe achievement of this objective could permit us to develop Systemic RNAi drugs for a broad range of diseases, such as cancer and autoimmune diseases. • Maintain our strong intellectual property position in the RNAi field. We believe we have a strong intellectual property position relating to the development and commercialization of siRNAs as therapeutics. To build upon our existing intellectual property position, we are focusing on patents and patent applications covering fundamental aspects of siRNAs, chemical modifications to siRNAs and specific targets for RNAi therapeutics. • Capitalize on our expertise in RNAi and our intellectual property position to gain access to additional resources to develop and commercialize RNAi therapeutics. We believe that we can use our expertise and the strength of our intellectual property to drive the formation of strategic alliances that will provide us with funding and access to important additional resources. We intend to take an active role in these alliances, including maintaining certain development and commercialization rights. We also intend to use our early alliances to expand our own capabilities so that in the future we will be able to develop and commercialize our therapeutic products independently. • Leverage our intellectual property position by licensing our technology to generate revenues. We intend to generate revenues by granting licenses to our intellectual property to third parties for the development of therapeutics outside our areas of focus and for the development of research reagents and services. Our Product Engine To realize the potential of RNAi therapeutics as a broad new class of drugs, we are developing a set of biological and chemical methods and know how that we expect to apply in a systematic way to develop RNAi therapeutics for a variety of diseases. We refer to these methods and their systematic application as our product engine. We believe that our product engine will provide a systematic approach for identifying siRNA drug candidates, using the following steps: • Sequence selection — the use of computational tools to design siRNA sequences likely to be selective for the gene that is to be silenced, • Potency selection — the synthesis and comparison of different siRNAs to determine which are most potent in silencing the target gene, • Stabilization — the introduction of chemical modifications into potent siRNAs to make them more stable within the body, and • Improvement of biodistribution — the addition of further chemical groups to stabilized siRNAs to improve their ability to reach different parts of the body. Our Development Programs Using the current capabilities of our product engine, we have initiated two programs to identify specific siRNAs for potential further development as Direct RNAi drug candidates. We expect to initiate additional programs as the capabilities of our product engine evolve. Our current programs are focused on AMD and PD. Age-Related Macular Degeneration AMD can cause severe deterioration of vision and may ultimately cause blindness. The National Eye Institute estimates that over 1.6 million adults over 50 in the United States suffer from advanced AMD. The siRNAs we are exploring would treat wet AMD, a subtype of AMD often associated with severe vision loss. AMD Alliance International estimates that approximately 200,000 new cases of wet AMD are diagnosed in North America each year and 500,000 new cases of wet AMD are diagnosed worldwide each year. We are developing a RNAi therapeutic intended to treat wet AMD by suppressing production of the protein known as vascular endothelial growth factor, or VEGF. VEGF is believed to play a key role in wet AMD by stimulating the growth and leakage of blood vessels that disrupt the retina. We are currently evaluating several siRNAs designed to inhibit the production of VEGF in animal models and expect to begin a clinical trial for an AMD product candidate in 2005. Any siRNA we develop for AMD will be a Direct RNAi drug administered by injection into the eye, the mode of administration used for two drug candidates currently in late stage clinical trials by other companies. We have filed patent applications relating to the use of siRNA to suppress VEGF production for therapeutic purposes. Parkinson’s Disease PD is a disorder of the nervous system that the American Parkinson Disease Association estimates afflicts more than 1.5 million people in the United States, and the World Health Organization estimates afflicts approximately four million people worldwide. Current treatments for PD focus on treating the symptoms of the disease, which are caused by the shortage of a substance known as dopamine in the brain. There are no drugs currently approved to treat the cause of the disease. The siRNAs we have started to explore as potential treatments for PD are based on treating the suspected cause of PD rather than the symptoms. Instead of replacing the function of missing dopamine in persons with PD, our approach would attempt to prevent the death of dopamine-producing cells that causes this shortage. Based on recent scientific findings, we believe that it may be possible to prevent the death of these cells by using an siRNA to suppress production of a protein known as alpha-synuclein. We recently entered into a collaboration with the Mayo Foundation for Medical Education and Research and the Mayo Clinic Jacksonville, to explore this possibility in animal models of PD. Early Stage Company We are an early stage company and the approach we are taking to discover and develop drugs is novel and unproven. Our potential product candidates are in early stages of preclinical development where failure is common. Neither we nor any other company has received regulatory approval to market therapeutics utilizing siRNAs. We do not expect any of our product candidates, if successfully developed, to receive regulatory approval for commercial sale for at least several years. See “Risk Factors” beginning on page 11 for risks related to an investment in our common stock. Corporate Information Alnylam Pharmaceuticals, Inc. was incorporated in Delaware in May 2003. Alnylam Europe, AG, which was incorporated in Germany in June 2000 under the name Ribopharma AG, and Alnylam U.S., Inc., which was incorporated in Delaware in June 2002, are wholly owned subsidiaries of Alnylam Pharmaceuticals, Inc. Alnylam Pharmaceuticals, Inc. acquired Alnylam Europe, AG in July 2003. Our principal executive office is located at 790 Memorial Drive, Cambridge, Massachusetts 02139, and our telephone number is (617) 252-0700. Our internet address is www.alnylam.com. The information on our web site is not incorporated by reference into this prospectus and should not be considered to be a part of this prospectus. Our web site address is included in this prospectus as an inactive technical reference only. | ||||||||||||||
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