ALNY Alnylam Pharmaceuticals Inc.

166.36
+1.29  (+1%)
Previous Close 165.07
Open 165.35
52 Week Low 84.97
52 Week High 175.41
Market Cap $19,327,832,564
Shares 116,180,768
Float 84,273,475
Enterprise Value $17,823,410,564
Volume 711,647
Av. Daily Volume 659,613
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Upcoming Catalysts

Drug Stage Catalyst Date
Vutrisiran - HELIOS-A
ATTR amyloidosis
NDA Filing
NDA Filing
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Fitusiran (ATLAS)
Hemophilia A/B
Phase 3
Phase 3
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Lumasiran (ALN-GO1) ILLUMINATE-C
Impaired renal function
Phase 3
Phase 3
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VIR-2218
Chronic hepatitis B virus (HBV)
Phase 2
Phase 2
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IR-2218 with pegylated interferon-alpha (PEG-IFN-α)
Hepatitis B
Phase 2
Phase 2
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ALN-CC5 (cemdisiran)
IgA nephropathy
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
Inclisiran
Hyperlipidemia
CRL
CRL
CRL received December 18, 2020.
Patisiran APOLLO-B
Wild-type ATTR amyloidosis patients with cardiomyopathy
Phase 3
Phase 3
Phase 3 enrolment to be completed early-2021.
Lumasiran
Primary Hyperoxaluria Type 1 (PH1)
Approved
Approved
FDA approval announced November 24, 2020.
VIR-2703 / ALN-COV
COVID-19
Phase 1
Phase 1
IND filing has been delayed - announced November 5, 2020.
Lumasiran (ALN-GO1) ILLUMINATE-B
Primary Hyperoxaluria Type 1
Phase 3
Phase 3
Phase 3 top-line released September 30, 2020.
ALN-AAT02
alpha-1 anti-trypsin deficiency-associated liver disease
Phase 1/2
Phase 1/2
Phase 1/2 initial data released.
ALN-TTRsc02 (vutrisiran) - HELIOS-B
ATTR amyloidosis with cardiomyopathy
Phase 3
Phase 3
Phase 3 trial has been initiated - noted November 22, 2019.
Givosiran
Acute hepatic porphyrias
Approved
Approved
FDA approval announced November 20, 2019.
Patisiran
Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR
Approved
Approved
FDA Approval announced August 10, 2018.

Latest News

  1. – New 5-Year Strategy Represents Alnylam's Commitment to Delivering Transformative Rare and Prevalent Disease Medicines for Patients Around the World Through Sustainable Innovation and Exceptional Financial Performance Driving Profitability –

    – In Addition, Company Announces Full Year 2020 Preliminary* Global Net Product Revenues for ONPATTRO® and GIVLAARI® of Approximately $306 Million and $55 Million, Respectively, and Strong Initial U.S. Demand for OXLUMOTM

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced its new 5-year strategy "Alnylam P5x25" focused on the Company's planned transition to a top-5 biotech (measured by market capitalization) in the next 5 years through: sustainable innovation…

    – New 5-Year Strategy Represents Alnylam's Commitment to Delivering Transformative Rare and Prevalent Disease Medicines for Patients Around the World Through Sustainable Innovation and Exceptional Financial Performance Driving Profitability –

    – In Addition, Company Announces Full Year 2020 Preliminary* Global Net Product Revenues for ONPATTRO® and GIVLAARI® of Approximately $306 Million and $55 Million, Respectively, and Strong Initial U.S. Demand for OXLUMOTM

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced its new 5-year strategy "Alnylam P5x25" focused on the Company's planned transition to a top-5 biotech (measured by market capitalization) in the next 5 years through: sustainable innovation yielding transformative medicines for rare and common diseases for patients around the world and delivery of exceptional financial performance. Alnylam P5x25 extends the Company's decade-long heritage of providing longer term, 5-year business strategy guidance, the most recent of which was known as Alnylam 2020. In addition, Alnylam today reported preliminary fourth quarter and full year 2020 global net product revenues for ONPATTRO and GIVLAARI and provided additional updates on the Company's commercial launches, including initial OXLUMO demand.

    New 5-Year Strategy: P5x25

    Alnylam ended last year exceeding all metrics for its Alnylam 2020 strategy, with 4 marketed products (versus 3), 12 clinical programs (versus 10), 6 of which are in late-stage development (versus 4), across 4 strategic therapeutic areas (versus 3).

    The Company's Alnylam P5x25 strategy is aimed at Alnylam's transition to a top 5 biotech company, as measured by market capitalization, over the next 5 years.

    Specifically, the Company intends to end 2025 with the following profile**:

    • Patients: Over 0.5 million on Alnylam RNAi therapeutics globally
    • Products: 6 or more marketed products in rare and prevalent diseases
    • Pipeline: Over 20 clinical programs, with 10 or more in late stages and 4 or more INDs per year
    • Performance: ≥40% revenue CAGR through YE 2025
    • Profitability: Achieve sustainable non-GAAP profitability within the period

    "We executed well on our Alnylam 2020 strategy, exceeding all pre-set metrics and transitioning into a global, multi-product commercial company with a robust clinical pipeline and an organic product engine delivering sustainable innovation, a profile that has rarely been achieved in biotech history. It was especially gratifying to cap 2020 with positive Phase 3 HELIOS-A results for vutrisiran, which is set to become our 5th RNAi therapeutic to reach the market, if approved," said John Maraganore, Ph.D., CEO of Alnylam Pharmaceuticals. "We are now thrilled to launch our new chapter with Alnylam P5x25, which is aimed at Alnylam's planned transition to a top 5 biotech in market capitalization based on a proven and high-yielding technology for disruptive medical innovation and a foundational track record of commercial execution. Indeed, with Alnylam P5x25, we expect to sustainably and organically create and commercialize transformative rare and common disease medicines benefiting hundreds of thousands of patients around the world while delivering strong financial performance and profitability, resulting in a leading biotech profile."

    2020 Preliminary Commercial and Financial Performance*

    ONPATTRO® (patisiran), a commercial-stage RNAi therapeutic targeting transthyretin (TTR) for the treatment of polyneuropathy in adult patients with hATTR amyloidosis.

    • Preliminary global net product revenues for the fourth quarter and full year 2020 were approximately $90 million and $306 million, respectively.
      • Q4 results represent approximately 10 percent growth compared to Q3 and include 10 percent growth in the U.S. market segment driven by new patient demand.
      • Further, the full year ONPATTRO revenues reached the high end of the previously shared guidance range of $295 million - $310 million and represent over 80 percent growth from full year 2019.
    • As of year-end 2020, about 1,350 patients worldwide were receiving commercial ONPATTRO.

    GIVLAARI® (givosiran), a commercial-stage RNAi therapeutic for the treatment of adults with acute hepatic porphyria (AHP).

    • Preliminary global net product revenues for the fourth quarter and full year 2020 were approximately $22 million and $55 million, respectively.
      • These results represent greater than 30 percent quarter over quarter growth.
    • As of year-end 2020, the product's first full year of launch, over 200 patients are receiving commercial drug.

    OXLUMO™ (lumasiran), a commercial-stage RNAi therapeutic for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients.

    • For the period following European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) approval of OXLUMO, in late November 2020, strong initial U.S. demand was observed with 8 Start Forms received through year-end.
    • Preliminary global net product revenues for the fourth quarter were approximately $0.3 million representing initial patient demand in Europe

    Finally, the Company today reported that it expects its full year 2020 non-GAAP operating loss to be substantially improved relative to the prior year, marking 2019 as Alnylam's peak non-GAAP operating loss year as the Company transitions towards a self-sustainable financial profile.

    Further, at December 31, 2020, Alnylam had preliminary cash, cash equivalents, and marketable securities of approximately $1.9 billion, as compared to $1.5 billion at December 31, 2019. The Company balance sheet was strengthened by the 2020 strategic financing collaboration with Blackstone.

    "We are extremely pleased with ONPATTRO and GIVLAARI performance in the fourth quarter and cumulatively for the year, reflecting strong commercial execution, a substantial increase in demand and new patient adds, despite the ongoing COVID-19 pandemic. Additionally, we're pleased to report that strong top-line revenue growth at the upper end of our guidance range and disciplined R&D and SG&A investments have delivered on a lower non-GAAP operating loss for 2020 compared with the prior year. We believe we're now firmly on our way toward a self-sustainable financial profile with significant growth from four revenue generating assets and moderated operating expenses, and a balance sheet that supports achievement of profitability without the need to access the equity markets," said Jeff Poulton, Chief Financial Officer of Alnylam. "As we now launch our Alnylam P5x25 strategy, we're committed to delivering consistently strong financial performance driven by 40% or greater revenue CAGR over the next five years with non-GAAP profitability achieved within the period."

    Alnylam management will discuss these preliminary selected financial results and commercial updates during a webcast presentation at the 39th Annual J.P. Morgan Healthcare Conference tomorrow, Monday, January 11, 2021, at 8:20 a.m. ET.

    About RNAi

    RNAi (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's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – 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.

    About Alnylam Pharmaceuticals

    Alnylam (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's commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO™ (lumasiran) and Leqvio® (inclisiran) being developed and commercialized by Alnylam's partner Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam P5x25" strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

    Alnylam Forward Looking Statements

    Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam's views and plans with respect to its new 5-year "Alnylam P5x25" strategy, its intentions to achieve the metrics associated with this strategy including to become a profitable, top 5 biotech company, its unaudited, preliminary selected financial results for 2020, the potential for RNAi therapeutics, including ONPATTRO, GIVLAARI, OXLUMO, Leqvio (inclisiran) and vutrisiran, its plans for the continuing launch of its commercial products and the advancement of vutrisiran through regulatory review and toward the market, the achievement of additional pipeline milestones, and the continued development and commercialization of Leqvio (inclisiran) by its partner, Novartis, 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, the broad availability of safe and effective vaccine(s), 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's business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam's ability to execute business continuity plans to address disruptions caused by the COVID-19 or any future pandemic; the finalization and audit of Alnylam's fourth quarter and 2020 fiscal year financial results which could potentially result in changes or adjustments to the selected preliminary financial results presented herein; 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' marketed products; 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; successfully launching, marketing and selling its approved products globally; Alnylam's ability to successfully expand the indication for ONPATTRO in the future; the ability of Novartis to successfully obtain and maintain additional regulatory approvals and pricing and reimbursement for Leqvio (inclisiran); 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 and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam's ability to establish and maintain strategic business alliances and new business initiatives; Alnylam's dependence on third parties, including Novartis, Regeneron and Vir, for the continued development and commercialization of certain products; 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.

    * The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the Company's announcement of complete financial results in February 2021.

    **The "Alnylam P5x25" metrics on Patients, Products, and Pipeline include proprietary and partnered products and programs.

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  2. – Vutrisiran Met Primary and All Secondary Endpoints at 9 Months, with Statistically Significant Improvements in Progression of Neuropathy, Quality of Life (QOL), and Gait Speed, Relative to Placebo –

    – Majority of Patients Showed Reversal of Disease Manifestations with Improvements in Neuropathy Impairment and QOL, Relative to Baseline –

    – Vutrisiran Showed Improvements in the 9-Month Exploratory Cardiac Endpoint of NT-proBNP, Relative to Placebo –

    – In Addition, Vutrisiran Demonstrated Encouraging Safety and Tolerability Profile –

    – Alnylam Intends to Present Full 9-Month Results and File New Drug Application (NDA) in Early 2021 –

    – Alnylam to Host Conference Call Today at 8:00 am ET –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the…

    – Vutrisiran Met Primary and All Secondary Endpoints at 9 Months, with Statistically Significant Improvements in Progression of Neuropathy, Quality of Life (QOL), and Gait Speed, Relative to Placebo –

    – Majority of Patients Showed Reversal of Disease Manifestations with Improvements in Neuropathy Impairment and QOL, Relative to Baseline –

    – Vutrisiran Showed Improvements in the 9-Month Exploratory Cardiac Endpoint of NT-proBNP, Relative to Placebo –

    – In Addition, Vutrisiran Demonstrated Encouraging Safety and Tolerability Profile –

    – Alnylam Intends to Present Full 9-Month Results and File New Drug Application (NDA) in Early 2021 –

    – Alnylam to Host Conference Call Today at 8:00 am ET –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hATTR amyloidosis with polyneuropathy. The primary endpoint was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at 9 months as compared to historical placebo data from the APOLLO Phase 3 study of patisiran. The two secondary endpoints were changes in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and gait speed assessed by the timed 10-meter walk test (10-MWT) compared to historical placebo. Vutrisiran met the primary endpoint (p less than 0.001) and achieved statistically significant results (p less than 0.001) for each of the Norfolk QoL-DN and 10-MWT secondary endpoints. In addition, vutrisiran treatment showed improvement compared to placebo on the exploratory cardiac biomarker endpoint, NT-proBNP (nominal p less than 0.05). Vutrisiran also demonstrated an encouraging safety and tolerability profile.

    Based on these positive results, the Company plans to submit a New Drug Application (NDA) for vutrisiran with the U.S. Food and Drug Administration (FDA) in early 2021, and to follow with regulatory filings in additional countries, such as Brazil and Japan. The Company plans to submit a Marketing Authorisation Application (MAA) in the EU upon obtaining the results of the 18-month analysis – expected in late 2021 – as previously aligned with the European Medicines Agency (EMA).

    "We are excited to report positive topline results from the HELIOS-A study, which show that vutrisiran reduces neurologic impairment and improves quality of life in patients with hATTR amyloidosis with polyneuropathy as soon as 9 months, with an encouraging safety and tolerability profile. In addition, we're very pleased to see evidence for reversal of polyneuropathy manifestations of disease and also favorable effects on the exploratory cardiac endpoint, NT-proBNP. We believe that vutrisiran, as a low-dose, once-quarterly, subcutaneously administered therapy, has the potential to be a highly attractive therapeutic option for patients living with this progressive, life-threatening, multi-system disease. We look forward to presenting the full 9-month results from HELIOS-A at a medical meeting in early 2021 and to announcing additional 18-month results, including additional exploratory cardiac endpoint data, in late 2021," said Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam. "We would like to recognize and extend our profound gratitude to the patients, caregivers, investigators, and study staff who are participating in HELIOS-A and who, through their commitment during an especially difficult year, have helped make possible another potential advancement in the treatment of hATTR amyloidosis with polyneuropathy. We look forward to initiating our regulatory filings in early 2021 as we work to bring this investigational treatment one step closer to patients with this rare disease."

    HELIOS-A (NCT03759379) is a Phase 3 global, randomized, open-label study to evaluate the efficacy and safety of vutrisiran. The study enrolled 164 patients with hATTR amyloidosis with polyneuropathy at 57 sites in 22 countries. Patients were randomized 3:1 to receive either 25mg of vutrisiran (N=122) via subcutaneous injection once every three months or 0.3 mg/kg of patisiran (N=42) via intravenous infusion once every three weeks (as a reference comparator) for 18 months. The primary endpoint is the change from baseline in mNIS+7 score at 9 months1, relative to historical placebo. Secondary endpoints at 9 months are the change from baseline in the Norfolk QoL-DN score and the timed 10-MWT, relative to historical placebo. Changes from baseline in NT-proBNP were evaluated as an exploratory endpoint at 9 months. The efficacy results of vutrisiran in HELIOS-A are compared to historical placebo control data from the landmark APOLLO Phase 3 study, which evaluated the efficacy and safety of patisiran in a patient population similar to that studied in HELIOS-A. Additional secondary endpoints at 18 months will be evaluated in the HELIOS-A study, including change from baseline in mNIS+7, Norfolk QoL-DN, 10-MWT, modified body mass index (mBMI), Rasch-built Overall Disability Scale (R-ODS), and serum transthyretin (TTR) levels. Additional exploratory cardiac endpoint data at the 18-month time point will be evaluated, including NT-proBNP, echocardiographic measures and cardiac amyloid assessments with technetium scintigraphy imaging. Following the 18-month study period, all patients are eligible to receive vutrisiran for an additional 18 months as part of an open-label extension study. Full 9-month results will be presented at a medical conference in early 2021 and topline 18-month results, including further exploratory cardiac endpoint data, are expected to be announced in late 2021.

    Vutrisiran demonstrated an encouraging safety profile. There were two study discontinuations (1.6 percent) due to adverse events in the vutrisiran arm by Month 9, both due to deaths, neither of which was considered related to study drug. There were two serious adverse events (SAEs) deemed related to vutrisiran by the study investigator, consisting of dyslipidemia and urinary tract infection. Treatment emergent adverse events (AEs) occurring in 10 percent or more patients included diarrhea, pain in extremity, fall and urinary tract infections, with each of these events occurring at a similar or lower rate as compared with historical placebo. Injection site reactions (ISRs) were reported in five patients (4.1 percent) and were all mild and transient. There were no clinically significant changes in liver function tests (LFTs).

    "The HELIOS-A results reinforce our commitment to building an industry-leading franchise of medicines for the treatment of ATTR amyloidosis which began with the development and approval of ONPATTRO as a treatment for patients with hATTR amyloidosis with polyneuropathy. Indeed, the vutrisiran results from HELIOS-A now serve as a second example of the potential for RNAi therapeutics to have a meaningful impact for patients, showing the ability to halt and potentially even reverse polyneuropathy manifestations of the disease. Furthermore, our robust development program, including the APOLLO-B and HELIOS-B studies, investigates the potential of patisiran and vutrisiran, respectively, to treat the cardiac manifestations of disease across a broad spectrum of patients with ATTR amyloidosis," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We believe that our ATTR amyloidosis franchise will be a significant driver of Alnylam's growth in the years to come, with the potential to position Alnylam as a top tier biopharma company."

    Vutrisiran has been granted Orphan Drug Designation in the United States and the European Union for the treatment of ATTR amyloidosis. Vutrisiran has also been granted a Fast Track designation in the United States for the treatment of the polyneuropathy of hATTR amyloidosis in adults. The safety and efficacy of vutrisiran are being evaluated in the comprehensive HELIOS clinical development program and have not yet been evaluated by any health authority. The ongoing HELIOS-B Phase 3 clinical trial in patients with ATTR amyloidosis with cardiomyopathy was initiated in late 2019 and is currently enrolling at sites around the world. Together, the HELIOS-A and -B studies are intended to demonstrate the broad impact of vutrisiran across the multisystem manifestations of disease and the full spectrum of patients with ATTR amyloidosis.

    Conference Call Information

    Alnylam management will discuss the HELIOS-A results via conference call on Thursday, January 7, 2021, at 8:00 am ET. A webcast presentation will also be available on the Investors page of the Company's website, www.alnylam.com. To access the call, please dial 877-312-7507 (domestic) or +1-631-813-4828 (international) five minutes prior to the start time and refer to conference ID 4398564. A replay of the call will be available beginning at 11:00 am ET on the day of the call. To access the replay, please dial 855-859-2056 (domestic) or +1-404-537-3406 (international) and refer to conference ID 4398564.

    About hATTR Amyloidosis

    Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis, represents a major unmet medical need with significant morbidity and mortality affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients presenting with cardiomyopathy.

    About Vutrisiran

    Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that allows for infrequent subcutaneous injections. The safety and efficacy of vutrisiran have not been evaluated by the U.S. Food and Drug Administration, European Medicines Agency or any other health authority.

    About RNAi

    RNAi (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's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing 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.

    About Alnylam Pharmaceuticals

    Alnylam (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's commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO™ (lumasiran), and, in Europe, Leqvio® (inclisiran). Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam exceeded the goals first established in 2015 under 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 and engage with us on Twitter at @Alnylam or on LinkedIn.

    Alnylam Forward Looking Statements

    Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, expectations regarding the direct or indirect effects on Alnylam's business, activities and prospects as a result of the COVID-19 pandemic, or delays or interruptions resulting therefrom and the success of Alnylam's mitigation efforts, Alnylam's views and plans with respect to the potential for RNAi therapeutics, including vutrisiran and patisiran, expectations regarding the safety and efficacy of vutrisiran as a treatment for hATTR amyloidosis with polyneuropathy, and its potential to have a meaningful impact on the course of this disease, expectations regarding the potential of vutrisiran and patisiran to treat the cardiac manifestations of ATTR amyloidosis across a broad spectrum of patients, Alnylam's prospects for building an industry-leading ATTR amyloidosis franchise and to become a top-tier biopharma company, the expected timing for the filing of regulatory submissions for vutrisiran the presentation of full 9-month results and the announcement of 18-month topline results, including exploratory cardiac endpoint data, 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, the availability of safe and effective vaccine(s), 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's business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam's 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, including vutrisiran; 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' marketed products, including ONPATTRO, GIVLAARI, OXLUMO and Leqvio (in Europe); 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, including vutrisiran, and the success of its partner Novartis', in obtaining regulatory approval for inclisiran in the U.S. and elsewhere, and maintaining regulatory approval and obtaining pricing and reimbursement for its products, including ONPATTRO, GIVLAARI, and OXLUMO, as well as its partner Novartis' success obtaining pricing and reimbursement for Leqvio; 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's 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's 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.


    1 In alignment with the EMA, the primary endpoint of change from baseline in mNIS+7 will be evaluated at 18 months to support an MAA.

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  3. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2021 at 8:20 am ET. This presentation will include an update on unaudited fourth quarter and full year 2020 global net product revenues. In addition, the Company will webcast the Q&A breakout session immediately following its presentation at 8:40 am ET.

    A live audio webcast of both the presentation and breakout session will be available on the Investors section of the Company's website, www.alnylam.com/events. A replay will be available on the Alnylam website within 48 hours after the event.

    About Alnylam Pharmaceuticals

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2021 at 8:20 am ET. This presentation will include an update on unaudited fourth quarter and full year 2020 global net product revenues. In addition, the Company will webcast the Q&A breakout session immediately following its presentation at 8:40 am ET.

    A live audio webcast of both the presentation and breakout session will be available on the Investors section of the Company's website, www.alnylam.com/events. A replay will be available on the Alnylam website within 48 hours after the event.

    About Alnylam Pharmaceuticals

    Alnylam (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's commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO™ (lumasiran). Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam has executed on and believes it has now exceeded 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 and engage with us on Twitter at @Alnylam or on LinkedIn.

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  4. - Strong Commercial and Medical Team in Place to Execute on Three Ongoing Global Launches and Advancement of Innovative Pipeline of RNAi Therapeutics –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced the appointment of Tolga Tanguler to the role of Chief Commercial Officer where he will lead global marketing, sales, market access, commercial operations, training, new product commercialization and commercial strategy for the Company. Tolga will also join the Company's Management Board. In addition, Alnylam has recruited an experienced team of leaders across key functions, including: Kasha Witkos, Senior Vice President and Head of Canada, Europe, Middle East and Africa (CEMEA); Salil Patel…

    - Strong Commercial and Medical Team in Place to Execute on Three Ongoing Global Launches and Advancement of Innovative Pipeline of RNAi Therapeutics –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced the appointment of Tolga Tanguler to the role of Chief Commercial Officer where he will lead global marketing, sales, market access, commercial operations, training, new product commercialization and commercial strategy for the Company. Tolga will also join the Company's Management Board. In addition, Alnylam has recruited an experienced team of leaders across key functions, including: Kasha Witkos, Senior Vice President and Head of Canada, Europe, Middle East and Africa (CEMEA); Salil Patel, Senior Vice President and Head of Medical Affairs; and Agnieszka Gallagher, Chief Ethics and Compliance Officer.

    "After an in-depth and comprehensive search, we have selected Tolga Tanguler, a dynamic and highly skilled commercial leader to join Alnylam. With over 20 years of global pharmaceutical and biotech experience, most recently at Pfizer and Alexion, Tolga's considerable success commercializing multiple rare, specialty and common disease products positions him as an excellent choice for this key role," said John Maraganore, Ph.D., Chief Executive Officer at Alnylam. "With Tolga, Kasha, Salil and Aggie joining Alnylam, we have now rounded out our world-class leadership team across our commercial, medical and compliance expertise areas. Together, these outstanding leaders stand poised to support the ongoing global launches of our three marketed products and promising pipeline of innovative medicines addressing a range of patient needs, while we continue on our path towards profitability."

    Tolga Tanguler, Chief Commercial Officer

    Tolga Tanguler has a strong track record of success in the global pharmaceutical and biotech industries, building out commercial capabilities, leading highly functioning teams, and cultivating robust thought leader relationships across multiple specialties. He joins Alnylam from Alexion where he was Senior Vice President, Head of U.S. and launched three new products while building key functions and capabilities and driving over $3 billion in revenue with double digit growth in eight consecutive quarters. Prior to Alexion, he spent the majority of his career in progressively responsible roles at Pfizer. While at Pfizer he established their North America rare disease unit, leading nearly $1B business and launch preparation for tafamidis, as well as heading up the global Eliquis alliance with Bristol Myers Squibb (BMS), and serving as a country manager of Denmark and Iceland. Tolga has lived in four countries and conducted business on five continents.

    Tolga will join Alnylam on January 5, 2021.

    Tolga holds an MBA in Marketing and Supply Chain Management from Michigan State University, as well as a post graduate certificate in Marketing from the University of California, Santa Barbara. He received his Bachelor of Science in Finance and Economics from Istanbul University, Turkey.

    "This is a very exciting time at Alnylam, a company with an amazing portfolio of marketed products and investigational programs driven by an organic product engine for sustainable innovation, coupled with a strong track record of commercial execution evidenced to date," said Tolga Tanguler. "I look forward to bringing my experience and passion to a leading company like Alnylam with its commitment to transform the treatment of many diseases and its sights on becoming a top-five biotech company. I look forward to working with a broad range of teams and functions to drive global commercial execution and top-line growth, while Alnylam continues to advance a robust clinical development pipeline of breakthrough medicines."

    Kasha Witkos, SVP, Head of CEMEA

    Kasha has over 23 years of broad international experience in the pharmaceutical industry working across four continents (Europe, Middle East and Africa, North America and Asia Pacific), covering various healthcare systems and market archetypes from emerging to mature markets. Her experience includes P&L responsibilities, sales and marketing, patient programs and all commercial channels.

    Kasha joins Alnylam from Takeda and its legacy organizations (Baxter, Baxalta, Shire), where she held several commercial roles in rare diseases on Country, Regional and Global levels. She was a Cluster GM and then a Region Head EMEA for Immunology in Baxalta and Shire as well as a Global Rare Immunology Franchise Head at Takeda, leading a $5B portfolio. Prior to joining Takeda and its legacy organizations, Kasha spent 10 years with Merck/MSD in the United States, Singapore and Thailand, where she held various positions of increasing commercial responsibility.

    Kasha started her medical education at Medical Academy of Wroclaw, Poland before transferring to the University of Colorado at Boulder, USA, where she completed her degree in genetics. Prior to joining the pharmaceutical industry, Kasha conducted basic medical research at the University of Colorado - Boulder and the Northwestern University Medical School – Chicago.

    Salil Patel, Ph.D., Senior Vice President, Head of Medical Affairs

    Salil brings a wealth of experience in global Medical Affairs, working with cross-functional teams in Commercial, Market Access as well as R&D. He joins us from BMS where he held various roles leading medical strategy for their Oncology and Immunoscience assets. Salil served as Head of Medical for the Oncology business of BMS, supporting the launch of Opdivo in several indications for major markets globally. One of his key accomplishments in this role was to form the Global Expert Centers Initiative - a collaborative network with 21 European leading academic institutes. He was also Head of Precision Medicine, where he worked with R&D and Commercial to implement into practice disease-related biomarkers in oncology.

    Salil received his Ph.D. in Biochemistry from the University of Southampton, UK and had significant post-doctoral experience in Molecular Biology from Caltech and Immunology from Stanford University.

    Agnieszka Gallagher (Aggie), Chief Ethics and Compliance Officer

    Agnieszka (Aggie) Gallagher joined Alnylam in June 2020 as Chief Ethics and Compliance Officer. Prior to Alnylam, Aggie was at ViiV Healthcare, a highly successful joint venture amongst GSK, Pfizer and Shionogi, focused on HIV, where Aggie served as General Counsel, Chief Compliance Officer and Secretary since 2017. Prior to ViiV, Aggie served in various legal and compliance roles at GSK, Sandoz, Medtronic and Pfizer, with responsibilities during different chapters of her career in every region of the world, including US, CEMEA, Asia and LATAM and across different sectors of the pharmaceutical industry, including innovative pharma, biosimilars and complex generics, vaccines and medical devices. Aggie began her legal career at White & Case in New York and Warsaw after receiving her law degree and undergraduate degree from Rutgers University.

    About RNAi

    RNAi (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's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – 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.

    About Alnylam Pharmaceuticals

    Alnylam (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's commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO™ (lumasiran). Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam has executed on and believes it has now exceeded 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 and engage with us on Twitter at @Alnylam or on LinkedIn.

    Alnylam Forward Looking Statements

    Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, the potential for RNAi therapeutics, aspirations to develop transformational or breakthrough medicines, advance an innovative pipeline of investigational therapeutics, achieve revenue growth, and become a profitable top five biotechnology company, and its belief that it has exceeded its "Alnylam 2020" 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's business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam's 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' 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's 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's 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.

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  5. − Product and Pipeline Goals Include Execution on Four Commercial Brands, One New NDA Filing, Two Phase 3 Data Readouts, Amongst Other Milestones –

    – New Clinical Data Presented from Phase 1 Monotherapy Study of ALN-AGT Show Mean Reduction in 24-Hour Systolic Blood Pressure of Over 15 mm Hg –

    – New Highlighted Programs Include Investigational RNAi Therapeutics for Recurrent Renal Stones, Non-alcoholic Steatohepatitis (NASH), Gout, Metabolic Syndrome, and Type 2 Diabetes, Expanding RNAi Opportunities into Specialty and Prevalent Disease Markets –

    – Continued Progress in Extrahepatic Delivery Supports Pipeline Advancement in CNS, Ocular, and Pulmonary Diseases, with Initial Program Reaching Clinical Development Stage in 2021 –

    – Alnylam

    − Product and Pipeline Goals Include Execution on Four Commercial Brands, One New NDA Filing, Two Phase 3 Data Readouts, Amongst Other Milestones –

    – New Clinical Data Presented from Phase 1 Monotherapy Study of ALN-AGT Show Mean Reduction in 24-Hour Systolic Blood Pressure of Over 15 mm Hg –

    – New Highlighted Programs Include Investigational RNAi Therapeutics for Recurrent Renal Stones, Non-alcoholic Steatohepatitis (NASH), Gout, Metabolic Syndrome, and Type 2 Diabetes, Expanding RNAi Opportunities into Specialty and Prevalent Disease Markets –

    – Continued Progress in Extrahepatic Delivery Supports Pipeline Advancement in CNS, Ocular, and Pulmonary Diseases, with Initial Program Reaching Clinical Development Stage in 2021 –

    – Alnylam to Webcast its R&D Day Event Today and Tomorrow at 9:00 a.m. ET –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, is kicking off a virtual R&D Day event today. During the two-day event, the Company plans to showcase its commercial and R&D progress, including its product and pipeline goals for 2021, focused on continued commercial execution and advancement of early-, mid- and late-stage investigational programs. Alnylam will also provide an update on its progress toward achieving a self-sustainable financial profile for future growth and value creation.

    "Five years ago, we charted our Alnylam 2020 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," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "In 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."

    2021 Product and Pipeline Goals

    ONPATTRO® (patisiran), a commercial-stage RNAi therapeutic targeting transthyretin (TTR) for the treatment of polyneuropathy in patients with hATTR amyloidosis. Alnylam plans to:

    • Continue global commercial execution
    • Complete enrollment in the APOLLO-B study, which is now expected in early 2021

    GIVLAARI® (givosiran), a commercial-stage RNAi therapeutic for the treatment of adults with acute hepatic porphyria (AHP). Alnylam plans to:

    • Continue global commercial execution
    • Achieve regulatory approval of GIVLAARI in Japan in mid-2021

    OXLUMO™ (lumasiran), 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:

    • Execute on global commercial launches throughout 2021
    • Achieve regulatory approval of OXLUMO in Brazil in early 2021
    • Report topline results from the ILLUMINATE-C Phase 3 study in mid-2021

    Vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. Alnylam plans to:

    • Report topline results from the HELIOS-A Phase 3 study (9-month endpoint) in early 2021
    • File a New Drug Application (NDA) with the FDA in early 2021, assuming positive results from the HELIOS-A Phase 3 study
    • Initiate data generation in early 2021 to support a biannual dose regimen of vutrisiran
    • Report topline results from the HELIOS-A Phase 3 study (18-month endpoint, including cardiac data) in late 2021
    • Continue enrollment in the HELIOS-B Phase 3 study throughout 2021

    ALN-AGT, an investigational RNAi therapeutic in development for the treatment of hypertension. Alnylam plans to:

    • Initiate the KARDIA Phase 2 studies in mid-2021

    Alnylam also plans to support, as needed, Novartis' continued efforts with respect to Leqvio® (inclisiran), 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:

    • Execute on global commercial launches throughout 2021
    • Continue enrollment in the ORION-4 Phase 3 cardiovascular outcomes (CVOT) study in 2021

    Alnylam also plans to support, as needed, Sanofi's continued efforts in advancing fitusiran, an investigational RNAi therapeutic in development for the treatment of hemophilia, for which Sanofi intends to resume dosing in the ATLAS Phase 3 studies. 

    In addition, the Company plans to continue advancement of its additional mid- and early-stage clinical pipeline programs.

    "At 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," said Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam. "Our 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'll 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."

    Expanding Opportunities in Wild-type ATTR Amyloidosis

    Alnylam 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.

    Finally, 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.

    Harnessing the Transformative Potential of RNAi in Hypertension

    RNAi 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.

    The 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 naïve 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.

    Addressing NASH with the Power of Human Genetics

    NASH – a leading cause of liver fibrosis, cirrhosis, and hepatocellular carcinoma – 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 – a genetically validated target – 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.

    Advancing the Next Wave of RNAi Therapeutics for Prevalent Diseases

    During its R&D Day event, Alnylam management will introduce multiple newly disclosed, additional programs that the Company believes may comprise the "Next Wave" of investigational RNAi therapeutics for prevalent diseases. These programs include:

    • Lumasiran, targeting glycolate oxidase (GO) in development for the prevention of recurrent renal stones caused by oxalate nephrolithiasis. Lumasiran is approved in the EU and U.S. for the treatment of primary hyperoxaluria Type 1, and is marketed as OXLUMO™;
    • ALN-XDH, targeting xanthine dehydrogenase (XDH) in development for the treatment of gout; and
    • ALN-KHK, targeting ketohexokinase (KHK) in development for the treatment of metabolic syndrome and type 2 diabetes

    Delivering on the Potential for RNAi Therapeutics Beyond the Liver

    Finally, 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:

    • ALN-APP, targeting amyloid precursor protein (APP) in development for the treatment of autosomal dominant Alzheimer's Disease (ADAD) and cerebral amyloid angiopathy (CAA);
    • ALN-HTT, targeting huntingtin protein, including exon 1, in development for the treatment of Huntington's Disease (HD); and
    • ALN-COV, targeting the SARS-CoV-2 RNA genome in development for the prevention or treatment of COVID-19.

    Alnylam is advancing its CNS and ocular disease programs in a 50-50 collaboration with Regeneron. Alnylam is partnered with Vir on ALN-COV.

    R&D Day Webcast Information

    The Company's 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's website, www.alnylam.com. 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).

    About RNAi

    RNAi (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's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – 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.

    About Alnylam Pharmaceuticals

    Alnylam (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's commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), and OXLUMO™ (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 and engage with us on Twitter at @Alnylam or on LinkedIn.

    Alnylam Forward Looking Statements

    Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, expectations regarding the direct or indirect effects on Alnylam's business, activities and prospects as a result of the COVID-19 pandemic, or delays or interruptions resulting therefrom and the success of Alnylam's 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's 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 "Alnylam 2020" 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's business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam's 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' 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's 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's 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.

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