ALNY Alnylam Pharmaceuticals Inc.

201.3
-3.03  -1%
Previous Close 204.33
Open 205.17
52 Week Low 119.29
52 Week High 212
Market Cap $23,924,040,601
Shares 118,847,693
Float 102,229,141
Enterprise Value $23,084,526,787
Volume 177,348
Av. Daily Volume 510,745
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Upcoming Catalysts

Drug Stage Catalyst Date
VIR-2218 with pegylated interferon-alpha (PEG-IFN-α)
Hepatitis B
Phase 2
Phase 2
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Inclisiran
Hypercholesterolemia
PDUFA
PDUFA
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Fitusiran (ATLAS)
Hemophilia A/B
Phase 3
Phase 3
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Vutrisiran
ATTR amyloidosis
PDUFA
PDUFA
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ONPATTRO (patisiran) - APOLLO-B
Wild-type ATTR amyloidosis patients with cardiomyopathy
Phase 3
Phase 3
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Vutrisiran - HELIOS-B
ATTR amyloidosis with cardiomyopathy
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
ALN-TTRsc04
ATTR amyloidosis
Phase 1
Phase 1
IND due by year-end 2022.
Vutrisiran (HELIOS-A)
ATTR Amyloidosis
Phase 3
Phase 3
Positive results from subgroup analyses and exploratory endpoints compared with placebo at 9-month period. September 7, 2021
OXLUMO (lumasiran) - ILLUMINATE-C
Impaired renal function
Phase 3
Phase 3
Phase 3 top-line data released July 29, 2021.
Zilebesiran (ALN-AGT) - KARDIA-1
Hypertension
Phase 2
Phase 2
Phase 2 initiation announced June 30, 2021.
GIVLAARI (givosiran)
Acute hepatic porphyrias
Approved
Approved
FDA approval announced November 20, 2019.
ONPATTRO (patisiran)
Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR
Approved
Approved
FDA Approval announced August 10, 2018.
OXLUMO (lumasiran) - ILLUMINATE-B
Primary Hyperoxaluria Type 1
Phase 3
Phase 3
Phase 3 top-line released September 30, 2020.
OXLUMO (lumasiran)
Primary Hyperoxaluria Type 1 (PH1)
Approved
Approved
FDA approval announced November 24, 2020.
ALN-AAT02
alpha-1 anti-trypsin deficiency-associated liver disease
Phase 1/2
Phase 1/2
Phase 1/2 initial data released.

Latest News

  1. -- GIVLAARI reduces Chronic Pain, Improves Quality of Life and is the First and Only Therapy Proven to Prevent AHP Attacks --

    TORONTO, Oct. 18, 2021 /CNW/ - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is pleased to announce that GIVLAARI® (givosiran) has now received a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH). GIVLAARI® is approved by Health Canada for subcutaneous use for the treatment of acute hepatic porphyria (AHP) in adults.1

    "The Health Canada market authorization of GIVLAARI provides new hope for those living with AHP. For the first time, all patients have a treatment option specifically targeted for this rare and serious disease," says Colleen Coxson, Country General…

    -- GIVLAARI reduces Chronic Pain, Improves Quality of Life and is the First and Only Therapy Proven to Prevent AHP Attacks --

    TORONTO, Oct. 18, 2021 /CNW/ - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is pleased to announce that GIVLAARI® (givosiran) has now received a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH). GIVLAARI® is approved by Health Canada for subcutaneous use for the treatment of acute hepatic porphyria (AHP) in adults.1

    "The Health Canada market authorization of GIVLAARI provides new hope for those living with AHP. For the first time, all patients have a treatment option specifically targeted for this rare and serious disease," says Colleen Coxson, Country General Manager, Alnylam Pharmaceuticals. "We are proud of what's been accomplished and are working towards full national reimbursement so that patients across Canada can have access to a treatment that will improve their overall health and quality of life."

    AHP is an ultra-rare condition in which patients can experience debilitating attacks of severe abdominal pain, vomiting and seizures. It can be life-threatening due to the possibility of paralysis and respiratory arrest during attacks. Many patients also experience chronic symptoms, including severe pain, which continues to be present between attacks, linked to increased rates of liver cancer, kidney failure and hypertension.2, 3

    "Patients suffering from this very rare, genetic disease often require urgent healthcare assistance and hospitalization, due to serious symptoms such as porphyria attacks which can involve severe abdominal pain, vomiting, breathing problems, seizures, and can lead to long term complications like chronic pain, chronic kidney failure and liver damage," says Dr. Colin Van Zoost MD, FRCPC, Assistant Professor of Medicine Dalhousie University. "Not knowing when the next attack will can cause severe anxiety for many patients, making engagement in day-to-day life, work and social situations difficult. GIVLAARI® gives us a treatment option that can help patients avoid ever having another attack, allowing them to re-engage with society and live as close to a normal life as possible."

    The positive recommendation for reimbursement was supported by the results of the ENVISION Phase 3 Study, a randomized, double-blind, placebo-controlled, global, multicenter study to evaluate the efficacy and safety of GIVLAARI® in patients with a documented diagnosis of acute hepatic porphyria (AHP).4 The primary endpoint was reduction relative to placebo in the annualized rate of composite porphyria attacks, defined as those requiring hospitalization, urgent healthcare visit, or intravenous hemin administration at home, in patients with acute intermittent porphyria (AIP, the most common subtype of AHP) over six months.5 Full details on the recommendation is available on the CADTH website.6

    About GIVLAARI® (givosiran)

    GIVLAARI® is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of adults with acute hepatic porphyria (AHP). In the pivotal study, givosiran was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam's first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP.

    About Acute Hepatic Porphyria

    Acute hepatic porphyria (AHP) refers to a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. AHP is comprised of four subtypes: acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), variegate porphyria (VP), and ALA dehydratase-deficiency porphyria (ADP). Each type of AHP results from a genetic defect leading to a lack of certain enzymes needed to produce heme in the liver, which leads to an accumulation of porphyrins in the body to toxic amounts. AHP disproportionately impacts women of working and childbearing age, and symptoms of the disease vary widely. Severe, unexplained abdominal pain is the most common symptom, which can be accompanied by limb, back, or chest pain, nausea, vomiting, confusion, anxiety, seizures, weak limbs, constipation, diarrhea, or dark or reddish urine. AHP is life-threatening due to the possibility of paralysis and respiratory arrest during attacks. The nonspecific nature of AHP signs and symptoms can often lead to misdiagnoses of other more common conditions such as gynecological disorders, viral gastroenteritis, irritable bowel syndrome (IBS), and appendicitis. Consequently, on a global perspective, patients with AHP can wait up to 15 years for a confirmed diagnosis, with the risk of addiction problems. In addition, long-term complications and comorbidities of AHP can include hypertension, chronic kidney disease or liver disease, including hepatocellular carcinoma.

    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.

    About Alnylam Pharmaceuticals

    Alnylam (NASDAQ:ALNY) is leading the translation of RNA interference (RNAi) into a new class of innovative medicines with the potential to improve the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS) 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 discovery platform. Alnylam has a deep pipeline of investigational medicines, including five product candidates that are in Phase 3 clinical trials and one in registration. Looking forward, Alnylam will continue to execute 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 employs over 1,200 people worldwide and is headquartered in Cambridge, MA. Alnylam Canada is headquartered in Mississauga, Ontario with established operations since June 2018. 

    ______________________________

    1 GIVLAARI Product Monograph. October 8, 2020

    2 Puy, The Lancet 2010

    3 Anderson, Ann. Intern. Med. 2005.

    4 GIVLAARI Product Monograph. October 8, 2020

    5 GIVLAARI Product Monograph. October 8, 2020

    6 https://cadth.ca/givosiran

     

    SOURCE Alnylam Pharmaceuticals, Inc.

    Cision View original content: http://www.newswire.ca/en/releases/archive/October2021/18/c7841.html

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  2. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that it will report financial results for the third quarter ending September 30, 2021 on Thursday, October 28, 2021, before the U.S. financial markets open.

    Management will provide an update on the Company and discuss third quarter 2021 results as well as expectations for the future via conference call on Thursday, October 28, 2021 at 8:30 am ET. 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 1428538. A replay of the call will be available beginning at 11:30 am ET on the day of the call. To access the replay, please dial 855-859-2056 (domestic…

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that it will report financial results for the third quarter ending September 30, 2021 on Thursday, October 28, 2021, before the U.S. financial markets open.

    Management will provide an update on the Company and discuss third quarter 2021 results as well as expectations for the future via conference call on Thursday, October 28, 2021 at 8:30 am ET. 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 1428538. A replay of the call will be available beginning at 11:30 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 1428538.

    A live audio webcast of the call will be available on the Investors section of the Company's website at www.alnylam.com/events. An archived webcast will be available on the Alnylam website approximately two 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), as well as Leqvio® (inclisiran), which is 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, on LinkedIn, or on Instagram.

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  3. − Alnylam will provide a $1 Million Grant to Acumen America and will serve as an Anchor Partner of their Health Equity Portfolio –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced the launch of Alnylam Challengers, a signature community impact program that will support bold and diverse organizations in their pursuit of developing new and sustainable solutions to address challenges in health equity. Through this program, Alnylam will collaborate with social entrepreneurs, employees, and business partners to create innovative health solutions to tackle obstacles to accessing health care such as poverty, racial discrimination, and other social determinants of health.

    Alnylam's first U.S. partnership…

    − Alnylam will provide a $1 Million Grant to Acumen America and will serve as an Anchor Partner of their Health Equity Portfolio –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced the launch of Alnylam Challengers, a signature community impact program that will support bold and diverse organizations in their pursuit of developing new and sustainable solutions to address challenges in health equity. Through this program, Alnylam will collaborate with social entrepreneurs, employees, and business partners to create innovative health solutions to tackle obstacles to accessing health care such as poverty, racial discrimination, and other social determinants of health.

    Alnylam's first U.S. partnership is with Acumen America, the U.S. division of a global nonprofit that raises philanthropic dollars and invests them in social impact-driven companies, leaders and ideas that take on world poverty. Alnylam is contributing $1 million to support Acumen America in its efforts to impact 75 million people in the U.S. by 2024.

    "At Alnylam, corporate responsibility is a commitment to tackling unprecedented and complex challenges, taking courageous action, and using our business as a force for good. Alnylam Challengers is a multi-dimensional commitment for Alnylam," said Dr. Pushkal Garg, Chief Medical Officer. "We are dedicated to the partnership aspect of this program – our employees, both as experts in their fields and as volunteers, are working with organizations, through our partnership with Acumen America, to enable their work, fuel growth and drive real change for the communities they serve."

    The three focus areas of Alnylam Challengers include:

    • Social Investment Portfolio: A strategic social investment model to tackle health inequities by partnering with a diverse and boldly innovative set of social entrepreneurs, enterprises, and non-profit organizations.
    • Advisory Corps: A skills-based volunteerism initiative where Alnylam employee teams serve as subject-matter expert (SME) advisors to address health equity issues through innovative business models.
    • Convenings: A series of cross-sector forums that embed Alnylam into industry, national, and international dialogue around pressing issues of health equity and rally others to think boldly and partner on important health issues.

    "We are thrilled to be the first Alnylam Challengers partner to further our goal of empowering social entrepreneurs to address health equity," said Catherine Casey Nanda, Acumen America Director. "Our approach centers on the idea of patient capital investing, which bridges the gap between the efficiency and scale of market-based approaches and the social impact of philanthropy."

    Alnylam's Corporate Responsibility actions are organized across five interconnected, stakeholder-related focus areas: patients, science, employees, communities, and planet. Through Challengers, Alnylam is advancing its commitment to improve the health of humanity by investing in social organizations that are actively tackling the most pressing community and health equity challenges.

    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), as well as Leqvio® (inclisiran), which is 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, on LinkedIn, or on Instagram.

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  4. − Reveals Hexadecyl (C16) Lipophilic Conjugate for Potent and Effective Delivery of siRNAs in the CNS –

    − Presents Pre-clinical Data with IKARIA Platform Demonstrating Potential to Achieve Over 90% Target Knockdown with an Annual Dosing Regimen –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today new pre-clinical research results demonstrating continued RNAi therapeutics platform innovation and scientific leadership. The research was presented at the 17th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held virtually from September 26 – 29, 2021. Among the presentations from Alnylam scientists, new pre-clinical data were presented identifying a hexadecyl (C16) moiety as a…

    − Reveals Hexadecyl (C16) Lipophilic Conjugate for Potent and Effective Delivery of siRNAs in the CNS –

    − Presents Pre-clinical Data with IKARIA Platform Demonstrating Potential to Achieve Over 90% Target Knockdown with an Annual Dosing Regimen –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today new pre-clinical research results demonstrating continued RNAi therapeutics platform innovation and scientific leadership. The research was presented at the 17th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held virtually from September 26 – 29, 2021. Among the presentations from Alnylam scientists, new pre-clinical data were presented identifying a hexadecyl (C16) moiety as a lipophilic ligand by which conjugation to siRNAs enables the potential for safe, robust, and durable target knockdown in the central nervous system following intrathecal administration. In addition, pre-clinical platform data were presented highlighting the Company's extended duration platform, called IKARIA™, which has the potential to enable highly potent (i.e., >90 percent) target mRNA silencing with an annual dosing regimen.

    "At OTS this year we are pleased to share research illustrating Alnylam's continued leadership in the advancement of RNAi therapeutics. Here we show that conjugation of lipophilic C16 to siRNAs enables robust and durable target knockdown in the CNS in rats and non-human primates following intrathecal administration. We believe that if these pre-clinical findings are translated in human studies, then RNAi therapeutics could offer a meaningful new treatment option with infrequent dosing for a broad range of CNS diseases," said Kevin Fitzgerald, Ph.D., Chief Scientific Officer of Alnylam. "In addition, we are highlighting advancements in our siRNA platform with IKARIA, a proprietary design and chemistry approach that confers exquisite target specificity, deeper levels of target mRNA silencing, and potential for an annual dosing regimen. With IKARIA, we believe we can design very long-acting siRNAs with all the proven pharmacological advantages and established human experience of existing RNAi therapeutics, with the potential to deliver meaningful advances for patients."

    New pre-clinical data were presented from Alnylam's CNS-targeting platform. Various fatty acid ligands were conjugated to SOD1-targeting siRNAs and evaluated in rats. A hexadecyl (C16)-conjugated siRNA was shown to yield robust and durable target knockdown throughout the brain and spinal cord following intrathecal administration. Combining a 5'-vinyl phosphonate (VP) modification with C16 provided the greatest level of potency. This chemistry was therefore selected for further evaluation. In rat studies, single or multiple intrathecal doses of a SOD1-targeting siRNA led to robust, dose-dependent, and durable silencing of SOD1 throughout the brain. Further, in toxicology studies performed in rats and NHPs, no test article-related findings were seen for C16 conjugates, suggesting the potential for a clean safety and tolerability profile.

    Additional pre-clinical data were presented with an siRNA targeting amyloid precursor protein (APP). Alnylam is developing ALN-APP as a potential treatment for early onset Alzheimer's Disease (AD) and cerebral amyloid angiopathy (CAA), with a planned Clinical Trial Application (CTA) filing in late 2021. An APP siRNA was shown in vitro to cause greater intracellular reductions in soluble APP beta (sAPPβ) fragments compared to β-site APP cleaving enzyme (BACE) inhibitors. Moreover, in an experiment using induced pluripotent stem cell (iPSC)-derived neurons, treatment with APP siRNA was able to reduce the size of Rab5+ early endosomes back to wild-type levels within two weeks of treatment compared to control treated cells. This result shows that siRNA-mediated APP lowering corrects an early intracellular phenotype known to be linked to neurotoxicity in AD patients. Finally, in a transgenic mouse model of human sAPP, siRNA-mediated silencing of APP led to phenotypic improvements. Further, in CTA-enabling GLP toxicology studies in rats and NHPs there were no test-article related findings with ALN-APP, revealing an encouraging non-clinical tolerability profile in support of upcoming human studies.

    In a separate presentation, initial pre-clinical results were presented on the Company's IKARIA platform. Using the proprietary IKARIA design and chemistry, RNA sequencing analysis revealed that a transthyretin (TTR)-targeting siRNA delivered to hepatocytes, ALN-TTRsc04, achieved exquisite specificity. The IKARIA-derived ALN-TTRsc04 molecule also demonstrated comparable potency and durability with an analog of vutrisiran in NHP studies. Computational modeling data suggest that a single 300 mg subcutaneous dose of ALN-TTRsc04 may result in trough TTR reduction greater than 90% that is durable for approximately one year. ALN-TTRsc04 is planned to enter clinical development at or around year-end 2022 with an investigational new drug (IND) application filing.

    Additional OTS presentations by Alnylam included previously shown results on:

    • Development of lumasiran for the treatment of primary hyperoxaluria type 1
    • Interim results from a Phase 1 study of zilebesiran (ALN-AGT), an investigational RNAi therapeutic in development for the treatment of hypertension.

    For the full breadth of results presented by the Company at OTS please visit the Capella section of the Alnylam website.

    Finally, Alnylam is also pleased to announce that its publication in Nucleic Acids Research entitled Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates* has been chosen by the Oligonucleotide Therapeutics Society Awards Committee as a winner of the Paper of the Year – Basic Research category. This award is designed to honor the year's most impactful paper in the field of oligonucleotide therapeutics.

    * Brown et al., Nucleic Acids Res. 2020 Dec 2;48(21):11827-11844.

    About hATTR Amyloidosis

    Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and fatal disease caused by variants (i.e., mutations) in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Variants 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 IKARIA™ Platform

    Alnylam's IKARIA platform takes advantage of more than two decades of experience in developing RNAi therapeutics. IKARIA enables an extended duration of activity in preclinical studies, with potential for annual dosing in humans, and has design features which provide exquisite specificity, further widening the potential therapeutic index, with enhanced target reduction levels.

    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), as well as Leqvio® (inclisiran), which is 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, on LinkedIn, or on Instagram.

    Alnylam Forward Looking Statements

    Various statements in this release concerning Alnylam's expectations, plans and prospects, including without limitation, the potential of lipophilic C16 conjugated siRNAs to enable robust and durable target knockdown in the CNS in humans, resulting in the possibility of a meaningful new treatment option with infrequent dosing for a broad range of CNS diseases, the potential of its IKARIA platform to enable highly potent target mRNA silencing with an annual dosing regimen, the potential of ALN-APP as a treatment for early onset AD and for CAA, and the expected timing of a CTA filing for ALN-APP, the potential of ALN-TTRsc04 to result in trough TTR reduction greater than 90% that is durable for approximately one year and the expected timing for an IND filing, its aspirations to become a leading biotech company, and the planned achievement of its "Alnylam P5x25" strategy, 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 on Alnylam's business, results of operations and financial condition and the effectiveness or timeliness of Alnylam's efforts to mitigate the impact of the pandemic; Alnylam's ability to discover and develop novel drug candidates and delivery approaches, including C16 and IKARIA, and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for its product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling its approved products globally; delays, interruptions or failures in the manufacture and supply of its product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to successfully expand the indication for ONPATTRO (or vutrisiran, if approved) in the future; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products, including Novartis, Regeneron and Vir; the outcome of litigation; the potential impact of a current government investigation and the risk of future 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 its other SEC filings. 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.

    This release is not intended to convey conclusions about efficacy or safety as to any investigational uses or dosing regimens of any investigational RNAi therapeutics. There is no guarantee that any investigational therapeutics or dosing regimens for such therapeutics will successfully complete clinical development or gain health authority approval.

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  5. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Chardan Virtual 5th Annual Genetic Medicines Conference on Tuesday, October 5, 2021 at 2:30 pm ET.

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

    About Alnylam

    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…

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Chardan Virtual 5th Annual Genetic Medicines Conference on Tuesday, October 5, 2021 at 2:30 pm ET.

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

    About Alnylam

    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 potential 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 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, on LinkedIn, or on Instagram.

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