ALNY Alnylam Pharmaceuticals Inc.

138.23
-4.27  -3%
Previous Close 142.5
Open 141.94
52 Week Low 110.74
52 Week High 178.41
Market Cap $16,217,161,155
Shares 117,320,127
Float 100,701,575
Enterprise Value $15,364,912,097
Volume 419,941
Av. Daily Volume 589,417
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Upcoming Catalysts

Drug Stage Catalyst Date
Vutrisiran - HELIOS-A
ATTR amyloidosis
Phase 3
Phase 3
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Lumasiran (ALN-GO1) ILLUMINATE-C
Impaired renal function
Phase 3
Phase 3
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Inclisiran
Hypercholesterolemia
NDA Filing
NDA Filing
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Fitusiran (ATLAS)
Hemophilia A/B
Phase 3
Phase 3
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ALN-CC5 (cemdisiran)
IgA nephropathy
Phase 2
Phase 2
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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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Drug Pipeline

Drug Stage Notes
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. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the 20th Annual Needham Virtual Healthcare Conference on Tuesday, April 13, 2021 at 1:30 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…

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the 20th Annual Needham Virtual Healthcare Conference on Tuesday, April 13, 2021 at 1:30 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 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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  2. - ILLUMINATE-A Phase 3 Study Evaluated the Efficacy and Safety of Lumasiran in Adult and Pediatric Patients with Primary Hyperoxaluria Type 1 (PH1) -

    - Lumasiran Demonstrated a Clinically Significant Reduction in Urinary Oxalate, a Primary Determinant of Progression to Renal Failure in PH1, Compared to Placebo -

    - Manuscript Marks the Tenth Publication of Clinical Trial Results for Alnylam Programs in The New England Journal of Medicine, Highlighting the Impact of RNAi Therapeutics as a New Class of Medicines -

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1)

    - ILLUMINATE-A Phase 3 Study Evaluated the Efficacy and Safety of Lumasiran in Adult and Pediatric Patients with Primary Hyperoxaluria Type 1 (PH1) -

    - Lumasiran Demonstrated a Clinically Significant Reduction in Urinary Oxalate, a Primary Determinant of Progression to Renal Failure in PH1, Compared to Placebo -

    - Manuscript Marks the Tenth Publication of Clinical Trial Results for Alnylam Programs in The New England Journal of Medicine, Highlighting the Impact of RNAi Therapeutics as a New Class of Medicines -

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1), were published online in The New England Journal of Medicine (NEJM). In November 2020, OXLUMO™ (lumasiran) was approved by the U.S. Food and Drug Administration for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients, and received marketing authorization from the European Commission for the treatment of PH1 in all age groups. OXLUMO is the first-ever treatment approved for PH1 and the first RNAi therapeutic evaluated in both children and adults. The full manuscript titled "Phase 3 Trial of Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1," will appear in the April 1, 2021 issue of NEJM.

    The data reported in the ILLUMINATE-A Phase 3 study publication demonstrated that RNAi-mediated targeting of liver GO by lumasiran led to substantial and sustained reductions in urinary oxalate—the toxic metabolite responsible for the debilitating and life-threatening clinical manifestations of PH1. Relative to placebo, treatment with lumasiran resulted in a clinically significant (53.5 percent) reduction in 24-hour urinary oxalate excretion from baseline to month 6 – the primary endpoint of the study.

    Improvements were also observed in a number of secondary endpoints, including the proportion of patients achieving normala or near-normalb levels of urinary oxalate, with 84 percent of lumasiran-treated patients meeting this endpoint compared with no patients (0 percent) on placebo. Patients treated with lumasiran also experienced favorable effects on exploratory endpoints related to nephrocalcinosis and the rate of renal stone eventsc compared with placebo.

    Lumasiran administration was associated with an encouraging safety and tolerability profile, with no serious or severe adverse events (AEs). The most common AEs that occurred more frequently with lumasiran than placebo were injection site reactions (38 versus 0 percent). All injection site reactions were mild and transient and did not result in discontinuation of treatment.

    "PH1 often presents in early life, with kidney stones, nephrocalcinosis, renal failure and, in advanced stages, systemic spread of oxalate throughout the body with life-threatening consequences. Oxalate drives disease manifestations and progression, and is the toxic mediator of end-organ damage in PH1," said Prof. Yaacov Frishberg, M.D., Head of Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem, Israel, and lead co-author on the manuscript. "We believe the publication of the ILLUMINATE-A Phase 3 study results in the NEJM is a testament to lumasiran as an oxalate-lowering therapy which is expected to confer significant clinical benefit to children and adults living with this disease."

    "Publication of the ILLUMINATE-A results in NEJM is an exciting achievement, highlighting the tremendous unmet need for novel therapies for this devastating disease and the role lumasiran is playing to fill this need. We are thrilled by the fact that this is now the tenth publication in NEJM on results from clinical studies of Alnylam's RNAi therapeutics, a noteworthy milestone underscoring the impact of RNAi on clinical research and the practice of medicine," said Pritesh J. Gandhi, PharmD., Vice President and General Manager, Lumasiran Program at Alnylam. "We look forward to reporting and publishing additional data from the ILLUMINATE program including in patients under the age of six and those with impaired kidney function later this year."

    A total of 38d of 39 patients completed the ILLUMINATE-A 6-month primary analysis period and all eligible patients transitioned to the study extension period. Results from the 12-Month extension period were presented at the American Society of Nephrology (ASN) virtual congress in October 2020 and demonstrated sustained efficacy with no safety findings leading to discontinuation in the study.

    IMPORTANT SAFETY INFORMATION

    Adverse Reactions

    The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling.

    Pregnancy and Lactation

    No data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production. Consider the developmental and health benefits of breastfeeding along with the mother's clinical need for OXLUMO and any potential adverse effects on the breastfed child from OXLUMO or the underlying maternal condition.

    For additional information about OXLUMO, please see the full Prescribing Information.

    About OXLUMO™ (lumasiran)

    OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. OXLUMO works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. Injection site reactions (ISRs) were the most common drug-related adverse reaction. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. OXLUMO utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology designed to increase potency and durability. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly thereafter at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMO should be administered by a healthcare professional. For more information about OXLUMO, visit OXLUMO.com.

    About ILLUMINATE-A Phase 3 Study

    ILLUMINATE-A (NCT03681184) is a six-month randomized, double-blind, placebo-controlled, global, multicenter Phase 3 study (with a 54-month extension period) to evaluate the efficacy and safety of lumasiran in 39 patients, age six and older, with a documented diagnosis of PH1. Patients were randomized 2:1 to receive three monthly doses of lumasiran or placebo followed by quarterly doses at 3 mg/kg. The primary endpoint was the percent change in 24-hour urinary oxalate excretion from baseline to the average of months 3 to 6 in the patients treated with lumasiran as compared to placebo. Treatment arms were stratified at randomization based upon mean 24-hour urinary oxalate during screening (≤1.7 or >1.7 mmol/24hr/1.73m2). Key secondary and exploratory endpoints were designed to evaluate additional measures of urinary oxalate, plasma oxalate, estimated glomerular filtration rate (eGFR), nephrocalcinosis, renal stone events, safety and tolerability.

    About Primary Hyperoxaluria Type 1 (PH1)

    PH1 is an ultra-rare genetic disease that affects an estimated one to three individuals per million in the United States and Europe. PH1 is characterized by oxalate overproduction in the liver. The excess oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones and nephrocalcinosis. Renal damage is caused by a combination of tubular toxicity from oxalate, calcium oxalate deposition in the kidneys, and urinary obstruction by calcium oxalate stones. PH1 is associated with a progressive decline in kidney function, which exacerbates the disease as the excess oxalate can no longer be effectively excreted, resulting in subsequent accumulation and deposition of oxalate in bones, eyes, skin, and heart, leading to severe illness and death. Management options to date were limited to hyperhydration, crystallization inhibitors and, in a minority of patients with a specific genotype, pyridoxine (vitamin B6). These measures do not adequately address oxalate overproduction but instead help to delay inevitable progression to kidney failure and the need for intensive dialysis as a bridge to a dual or sequential liver/kidney transplant. Liver transplantation is the only intervention that addresses the underlying metabolic defect, but is associated with high morbidity and mortality, and life-long immunosuppression. Until today, there were no approved pharmaceutical therapies for PH1.

    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), 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.

    Alnylam Forward Looking Statements

    Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam's views with respect to the safety and efficacy of OXLUMO as demonstrated in the ILLUMINATE-A and ILLUMINATE-B Phase 3 studies, the role of OXLUMO as an oxalate-lowering therapy which is expected to confer significant clinical benefit to children and adults living with PH1 and its ability to meet an unmet need for novel therapies for the treatment of PH1, and Alnylam's ability to achieve 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 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 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 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 Annual Report on Form 10-K 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.

    Footnotes:

    a Normal is defined as urinary oxalate levels at or below the upper limit of normal (ULN; ≤ 0.514 mmol/24 hr/1.73 m2); b near-normal is defined as urinary oxalate levels at or below 1.5 x ULN (≤ 0.771 mmol/24 hr/1.73 m2); ca renal stone event was defined as an event which includes at least one of the following: visit to healthcare provider because of a renal stone, medication for renal colic, stone passage, macroscopic hematuria due to a renal stone; done patient discontinued study drug after receiving a single dose and withdrew from the study after Month 3. Parent/guardian stopped participation due to patient's inability to comply with protocol-specific testing.

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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 Stifel 3rd Annual CNS Day on Thursday, April 1, 2021 at 11:00 am 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…

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Stifel 3rd Annual CNS Day on Thursday, April 1, 2021 at 11:00 am 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 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.

    View Full Article Hide Full Article
  4. – Alnylam to Share Additional Data From its RNAi Product Portfolio Including ATTR Amyloidosis and Acute Hepatic Porphyria –

    –Company to Host Conference Call April 19th at 4:00 pm ET to Discuss HELIOS-A Results –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that the Company will present full 9-month results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy of hereditary ATTR (hATTR) amyloidosis, at the American Academy of Neurology (AAN) Virtual Annual Meeting, being held April 17-22. The Company previously announced positive topline 9-month results from the HELIOS-A study in January. Additional data…

    – Alnylam to Share Additional Data From its RNAi Product Portfolio Including ATTR Amyloidosis and Acute Hepatic Porphyria –

    –Company to Host Conference Call April 19th at 4:00 pm ET to Discuss HELIOS-A Results –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that the Company will present full 9-month results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy of hereditary ATTR (hATTR) amyloidosis, at the American Academy of Neurology (AAN) Virtual Annual Meeting, being held April 17-22. The Company previously announced positive topline 9-month results from the HELIOS-A study in January. Additional data from Alnylam's ATTR amyloidosis program, including updates from the ongoing open-label extension study of ONPATTRO (patisiran) in patients with hATTR amyloidosis with polyneuropathy, will also be presented at AAN, along with data from the Phase 3 ENVISION study of GIVLAARI in patients with acute hepatic porphyria (AHP).

    "The depth and breadth of data that will be presented at AAN from across our RNAi product and pipeline portfolio reinforce the tremendous progress we have made with developing potentially transformative medicines for patients with rare diseases," said Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam. "In January we announced positive topline results from the HELIOS-A Phase 3 study of vutrisiran, the fifth of our investigational RNAi medicines to reach that significant milestone. We are excited for the upcoming presentation of the full 9-month HELIOS-A results as we believe in the potential of vutrisiran, as a low-dose, once quarterly, subcutaneously administered treatment option for patients living with a progressive, life-threatening, multi-system disease. We look forward to submitting our U.S. regulatory filing for marketing approval of vutrisiran in early 2021 as we continue to make progress towards building what we believe will be an industry-leading franchise of medicines for the treatment of ATTR amyloidosis."

    hATTR Amyloidosis

    • HELIOS-A: 9-month Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

      Oral Presentation

      Monday, April 19, 2021 at 2:00 pm ET

      Lead Author: David Adams
    • Global Open-label Extension: 24-month Data in Patients with hATTR Amyloidosis

      Oral Presentation

      Wednesday, April 21, 2021 at 4:00 pm ET

      Lead Author: David Adams
    • Impact of Patisiran on Activities of Daily Living and Functional Status in hATTR Amyloidosis

      Oral Presentation

      Wednesday, April 21, 2021 at 4:08 pm ET

      Lead Author: Amanda Peltier
    • Neurofilament Light Chain (NfL) as a Potential Biomarker of Treatment Response in Hereditary Transthyretin-Mediated Amyloidosis: Data from the Patisiran Global OLE Study

      Poster Presentation

      Available April 17

      Lead Author: Michael Polydefkis
    • Open-label Study of Patisiran in Patients with hATTR Amyloidosis Post-Orthotopic Liver Transplant

      Poster Presentation

      Available April 17

      Lead Author: Seth Arum
    • Evaluation of Patisiran With Concomitant or Prior Use of Transthyretin Stabilizers

      Poster Presentation

      Available April 17

      Lead Author: Madeline Merkel

    Acute Hepatic Porphyria

    • Reduction in Pain During and Between Attacks in Patients with Acute Hepatic Porphyria Treated with Givosiran: A Post-Hoc Analysis of the Phase 3 ENVISION Study

      Oral Presentation

      Tuesday, April 20, 2021 at 4:24 pm ET

      Lead Author: Susana Monroy
    • Disease Burden and Healthcare Utilization Among Patients with Acute Intermittent Porphyria Experiencing Chronic Neuropathy: Analyses from a National Healthcare Database

      Poster Presentation

      Available April 17

      Lead Author: Angelika Erwin

    Conference Call

    Alnylam management will discuss the full 9-month results from the HELIOS-A Phase 3 clinical trial via conference call on Monday, April 19th at 4:00 pm 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 9580096. A replay of the call will be available beginning at 7:00 pm 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 9580096.

    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 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 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 ONPATTRO® (patisiran)

    ONPATTRO is an RNAi therapeutic that was approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO blocks the production of TTR in the liver, reducing its accumulation in the body's tissues in order to halt or slow down the progression of the polyneuropathy associated with the disease. For more information about ONPATTRO, including full prescribing information and important safety information, visit ONPATTRO.com.

    About GIVLAARI® (givosiran)

    GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of adults and adolescents 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. For more information about GIVLAARI, including full prescribing information and important safety information, visit GIVLAARI.com.

    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), 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 expectations, plans, aspirations, and goals, including those related to vutrisiran and its potential as a low-dose, once quarterly, subcutaneously administered treatment option for patients, the expected timing of the filing of a NDA for vutrisiran, building an industry-leading franchise in medicines for the treatment of ATTR amyloidosis, becoming a leading biotech company, and the 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 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 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 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 Annual Report on Form 10-K 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 RNAi therapeutics or investigational uses previously approved therapeutics. There is no guarantee that any investigational therapeutics or expanded uses of commercial products will successfully complete clinical development or gain health authority approval.

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  5. New Strategic Framework Highlights Alnylam's Approach to Corporate Responsibility, Establishing the Foundation for Continuous Tracking of Initiatives and Progress —

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today issued its first Corporate Responsibility (CR) Summary, formalizing the management, tracking and communication of its environmental, social and governance (ESG) standards worldwide. The summary highlights Alnylam's commitments to sustainable business practices and demonstrated focus on its people, the communities it intends to serve, and the planet. The summary, available online at Alnylam.com, features case studies and analyses that illustrate the company's CR-driven philosophy, accomplishments…

    New Strategic Framework Highlights Alnylam's Approach to Corporate Responsibility, Establishing the Foundation for Continuous Tracking of Initiatives and Progress —

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today issued its first Corporate Responsibility (CR) Summary, formalizing the management, tracking and communication of its environmental, social and governance (ESG) standards worldwide. The summary highlights Alnylam's commitments to sustainable business practices and demonstrated focus on its people, the communities it intends to serve, and the planet. The summary, available online at Alnylam.com, features case studies and analyses that illustrate the company's CR-driven philosophy, accomplishments, aspirations and areas of future focus.

    "Since its founding, Alnylam has been purpose-driven and socially conscious. Our culture prioritizes people, communities – both patient communities and those in which we have operations - and the planet to stay at the forefront of transformational solutions that improve lives," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "In this new summary we are providing a snapshot of how Alnylam's core values are a compass for corporate responsibility, how this foundation has enabled us to respond to our world in new ways, and how it will keep us accountable while growing our business towards becoming a top 5 biotech company. This marks an important first step in our ongoing commitment to being a responsible business and to set goals that will measure and propel that progress."

    New Strategic Framework for Corporate Responsibility

    Alnylam's CR theme – Accepting Challenges to Improve the Health of Humanity – describes the Company's ongoing commitment to tackling unprecedented and complex challenges, taking courageous action, and using its business as a force of good. The Company's CR efforts are organized into five interconnected, stakeholder-related focus areas: patients, science, employees, communities, and planet.

    A guiding imperative, leadership team and set of accountabilities to track and manage Alnylam's impact are being established for each area. Alnylam has also implemented an enhanced governance structure to support its new CR framework, led by a new cross-functional CR Committee with direction from the Management Board and oversight from Alnylam's Board of Directors.

    Key initial priorities outlined in the CR summary include:

    • Increasing participation of minority populations in Alnylam clinical trials;
    • Launching new programs focused on female leadership and health equity in the U.S. and other regions;
    • Increasing the overall number of Black and Latinx individuals that make up the Company's U.S. employee base at all levels by 20%, in coordination with Alnylam's new Chief Diversity, Equity and Inclusion (DE&I) Officer;
    • Expanding Alnylam's existing programs to support employees, including COVID response, workplace safety, and mental health; and
    • Sustainability of Alnylam office space and operations, with the implementation of baseline metrics for solid waste worldwide, use of plastics and energy, and greenhouse gas emissions by 2022.

    Accepting Challenges to Improve the Health of Humanity builds upon Alnylam's decade long tradition of creating and meeting ambitious goals. Prior to its CR commitment, Alnylam made a similar commitment with its 2017 Patient Access Philosophy, for which it has annually measured the company's progress on education, advocacy and assistance, commercial and clinical access.

    To read more about Alnylam's CR summary, framework and key priorities, download Corporate Responsibility at Alnylam here.

    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 expectations, plans, aspirations, goals and priorities, including those related to CR and ESG matters, becoming a top 5 biotech company and the achievement of its "Alnylam P5x25" strategy, as well as Alnylam's general expectations of meeting ambitious goals 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 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 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 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 Annual Report on Form 10-K 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 RNAi therapeutics or investigational uses previously approved therapeutics. There is no guarantee that any investigational therapeutics or expanded uses of commercial products will successfully complete clinical development or gain health authority approval.

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