\n\u201cFive years ago, we charted our Alnylam 2020<\/i> goals of building a multi-product, global commercial company with a deep clinical pipeline for future growth and an organic product engine for sustainable innovation. We have now exceeded those ambitious goals with four marketed products and 12 organically derived clinical programs across four strategic therapeutic areas,\u201d said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.\u201cIn 2021, across our portfolio of partnered and proprietary programs, we aim to deliver performance on four commercial brands, file one new NDA, and report topline results on two Phase 3 programs, amongst other objectives. In the coming years, we believe Alnylam is positioned to emerge as a leading, top-five biotech company, driven by continuous global commercial execution and top-line growth, advancement of a robust clinical development pipeline, continued leverage of our organic product engine as a source of sustainable innovation and transformational medicines, and achievement of a self-sustainable financial profile.\u201d\n<\/p>\n
2021 Product and Pipeline Goals<\/b><\/p>\n
ONPATTRO\u00ae<\/sup> (patisiran)<\/b>, a commercial-stage RNAi therapeutic targeting transthyretin (TTR) for the treatment of polyneuropathy in patients with hATTR amyloidosis. Alnylam plans to:\n<\/p>\n GIVLAARI\u00ae<\/sup> (givosiran)<\/b>, a commercial-stage RNAi therapeutic for the treatment of adults with acute hepatic porphyria (AHP). Alnylam plans to:\n<\/p>\n OXLUMO\u2122 (lumasiran)<\/b>, a commercial-stage RNAi therapeutic for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients. Alnylam plans to:\n<\/p>\n Vutrisiran<\/b>, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. Alnylam plans to:\n<\/p>\n ALN-AGT<\/b>, an investigational RNAi therapeutic in development for the treatment of hypertension. Alnylam plans to:\n<\/p>\n \nAlnylam also plans to support, as needed, Novartis\u2019 continued efforts with respect to Leqvio\u00ae (inclisiran)<\/b>, the first and only siRNA therapy (or RNAi therapeutic) for the treatment of adults with hypercholesterolemia or mixed dyslipidemia, now approved in Europe. Approval of inclisiran by the FDA is anticipated by end of 2020, and Novartis plans to:\n<\/p>\n \nAlnylam also plans to support, as needed, Sanofi\u2019s continued efforts in advancing fitusiran<\/b>, an investigational RNAi therapeutic in development for the treatment of hemophilia, for which Sanofi intends to resume dosing in the ATLAS Phase 3 studies.\u00a0\n<\/p>\n \nIn addition, the Company plans to continue advancement of its additional mid- and early-stage clinical pipeline programs.\n<\/p>\n \n\u201cAt Alnylam, we are excited about the opportunity for RNAi therapeutics in rare and common diseases with liver and extra-hepatic delivery to realize the greatest potential for this new class of medicines in human health,\u201d said Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam. \u201cOur annual R&D Day will feature updates across our pipeline, including deep dive discussions on RNAi therapeutic opportunities in wild-type ATTR amyloidosis, hypertension, and NASH. Notably, new data from our ALN-AGT program, with an over 15 mm Hg mean reduction in systolic blood pressure as monotherapy, highlight the opportunity to reimagine the management of hypertension. In addition, we\u2019ll highlight a new program opportunity in recurrent renal stones for lumasiran, and new programs in gout, metabolic syndrome, and type 2 diabetes that are directed to highly prevalent diseases where RNAi therapeutics could have transformative potential.\u201d\n<\/p>\n Expanding Opportunities in Wild-type ATTR Amyloidosis<\/b><\/p>\n \nAlnylam will discuss the opportunity for its ATTR franchise programs with patisiran and vutrisiran in wild-type ATTR amyloidosis, a disease caused by TTR via the same pathogenic mechanism as in hereditary ATTR amyloidosis. Wild-type ATTR amyloidosis affects 200-300,000 patients worldwide representing a substantial expansion opportunity; the company will present data supporting the potential for its investigational RNAi therapeutics in this indication. Alnylam expects completion of enrollment in APOLLO-B with patisiran in early 2021 with topline data in mid-2022, while the HELIOS-B study with vutrisiran continues to enroll. Alnylam also plans to initiate in the next 12-18 months the HELIOS-C study which is aimed at evaluating vutrisiran for prevention of disease manifestations associated with ATTR amyloidosis.\n<\/p>\n \nFinally, Alnylam remains on track to report topline results from the HELIOS-A study of vutrisiran in early 2021. The HELIOS-A study includes a number of exploratory endpoints related to cardiac manifestations in patients with hATTR amyloidosis with polyneuropathy; the Company intends to present these data as part of the 18-month data readout in late 2021.\n<\/p>\n Harnessing the Transformative Potential of RNAi in Hypertension<\/b><\/p>\n \nRNAi therapeutics represent an opportunity to transform the treatment of hypertension with a once quarterly or bi-annually administered medicine that improves adherence and optimizes outcomes due to tonic control of blood pressure. Uncontrolled hypertension in potential target populations under evaluation comprise approximately 60 million patients in the U.S. alone.\n<\/p>\n \nThe Company will present new clinical data from the ongoing Phase 1 study of ALN-AGT, an investigational RNAi therapeutic targeting angiotensinogen in development for the treatment of hypertension. The Phase 1 study was performed in mild-to-moderate hypertension patients who were na\u00efve or who had washed out their anti-hypertensive medication; ALN-AGT was administered as monotherapy. In the new update, patients receiving a single dose of 400 mg or 800 mg ALN-AGT demonstrated mean serum AGT reductions of 97.5% at 4 weeks post-dose. Further, patients at the top dose demonstrated mean reductions in 24-hour systolic blood pressure (SBP) and diastolic blood pressure (DBP) of 16.8 +\/- 16.3 mm Hg and 9.1 +\/- 9.5 mm Hg, respectively. The safety and tolerability of ALN-AGT was consistent with data previously presented at the American Heart Association (AHA) Scientific Sessions in November 2020, with an acceptable safety profile for continued development.\n<\/p>\n Addressing NASH with the Power of Human Genetics<\/b><\/p>\n \nNASH \u2013 a leading cause of liver fibrosis, cirrhosis, and hepatocellular carcinoma \u2013 remains an area of enormous unmet need with approximately 15 million patients in the U.S. alone. Today, existing investigational drugs in development for NASH are directed toward molecular targets lacking genetic validation. Alnylam will present an update on its lead NASH program ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 \u2013 a genetically validated target \u2013 for the treatment of NASH. The Company will review the ongoing Phase 1 study and expected timelines for human proof-of-concept in 2022. In addition, Alnylam will announce a new NASH program targeting PNPLA3, another genetically validated target. Alnylam is partnered with Regeneron in a 50-50 collaboration for the advancement of RNAi therapeutics for NASH.\n<\/p>\n Advancing the Next Wave of RNAi Therapeutics for Prevalent Diseases<\/b><\/p>\n \nDuring its R&D Day event, Alnylam management will introduce multiple newly disclosed, additional programs that the Company believes may comprise the \u201cNext Wave\u201d of investigational RNAi therapeutics for prevalent diseases. These programs include:\n<\/p>\n Delivering on the Potential for RNAi Therapeutics Beyond the Liver<\/b><\/p>\n \nFinally, Alnylam will present an update on its programs involving extra-hepatic delivery of investigational RNAi therapeutics, including opportunities for CNS, ocular and pulmonary diseases. These programs include:\n<\/p>\n \nAlnylam is advancing its CNS and ocular disease programs in a 50-50 collaboration with Regeneron. Alnylam is partnered with Vir on ALN-COV.\n<\/p>\n R&D Day Webcast Information<\/b><\/p>\n \nThe Company\u2019s R&D Day event will be held on Tuesday and Wednesday, December 15 and 16, 2020 from 9:00 am to 12:00 pm ET each day and will include a live video stream on the Investors section of the Company\u2019s website, www.alnylam.com<\/a>. Replays will be available on the Alnylam website within 48 hours after each event. Presentations showcased during the event will be featured on Capella (www.alnylam.com\/capella<\/a>).\n<\/p>\n About RNAi <\/b><\/p>\n \nRNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today\u2019s medicines by potently silencing messenger RNA (mRNA) \u2013 the genetic precursors \u2013 that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.\n<\/p>\n About Alnylam Pharmaceuticals<\/b><\/p>\n \nAlnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)\/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam\u2019s commercial RNAi therapeutic products are ONPATTRO\u00ae<\/sup> (patisiran), GIVLAARI\u00ae<\/sup> (givosiran), and OXLUMO\u2122 (lumasiran). Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam has been executing on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com<\/a> and engage with us on Twitter at @Alnylam<\/a> or on LinkedIn<\/a>.\n<\/p>\n Alnylam Forward Looking Statements<\/b><\/p>\n \nVarious statements in this release concerning Alnylam’s future expectations, plans and prospects, including, without limitation, expectations regarding the direct or indirect effects on Alnylam\u2019s business, activities and prospects as a result of the COVID-19 pandemic, or delays or interruptions resulting therefrom and the success of Alnylam\u2019s mitigation efforts, Alnylam’s views and plans with respect to its product and pipeline goals for 2021 and the potential for RNAi therapeutics, including ONPATTRO, GIVLAARI, OXLUMO, Leqvio, patisiran, vutrisiran, fitusiran, ALN-AGT, ALN-HSD, ALN-XDH, ALN-KHK, ALN-APP, ALN-HTT and ALN-COV, its plans for additional global regulatory filings and the continuing product launches of ONPATTRO, GIVLAARI and OXLUMO, the achievement of additional pipeline milestones, including relating to the timing of topline data and potential regulatory filings for vutrisiran and the initiation of a Phase 2 study of ALN-AGT, the implications of the Phase 1 data for ALN-AGT and its potential to transform the treatment of hypertension, its expectations regarding the commercialization of Leqvio by Novartis and the continued clinical advancement of fitusiran by Sanofi, Alnylam\u2019s views with respect to the opportunity for patisiran and vutrisiran in wild-type ATTR amyloidosis, its expectations regarding the ongoing Phase 1 study of ALN-HSD and the timing for human proof-of-concept, the potential for RNAi therapeutics in prevalent diseases and to treat CNS and ocular diseases, and its belief that it has exceeded its \u201cAlnylam 2020\u201d guidance for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation: the direct or indirect impact of the COVID-19 global pandemic or any future pandemic, such as the scope and duration of the outbreak, government actions and restrictive measures implemented in response, material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by Alnylam products, or in patient enrollment in clinical trials, potential supply chain disruptions, and other potential impacts to Alnylam\u2019s business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam\u2019s ability to execute business continuity plans to address disruptions caused by the COVID-19 or any future pandemic; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all; actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and\/or progress of clinical trials or result in the need for additional pre-clinical and\/or clinical testing; delays, interruptions or failures in the manufacture and supply of its product candidates or its or its partner Novartis\u2019 marketed products, including ONPATTRO, GIVLAARI, OXLUMO and Leqvio; obtaining, maintaining and protecting intellectual property; intellectual property matters including potential patent litigation relating to its platform, products or product candidates; obtaining regulatory approval for its product candidates, and maintaining regulatory approval and obtaining pricing and reimbursement for its products, including ONPATTRO, GIVLAARI, and OXLUMO; progress in continuing to establish an ex-United States infrastructure; successfully launching, marketing and selling its approved products globally, including ONPATTRO, GIVLAARI, and OXLUMO, and achieving net product revenues for ONPATTRO within its revised expected range during 2020; Alnylam\u2019s ability to successfully expand the indication for ONPATTRO in the future; competition from others using technology similar to Alnylam’s and others developing products for similar uses; Alnylam’s ability to manage its growth and operating expenses within the ranges of guidance provided by Alnylam through the implementation of further discipline in operations to moderate spend and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam\u2019s ability to establish and maintain strategic business alliances and new business initiatives; Alnylam’s dependence on third parties, including Novartis for the continued development and commercialization of Leqvio, Regeneron for development, manufacture and distribution of certain products, including eye and CNS products, and Vir for the development of ALN-COV and other potential RNAi therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2; the outcome of litigation; the risk of government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. 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 explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.\n<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20201215005376\/en\/<\/a><\/span><\/p>\n\n
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