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ALNYLAM PHARMACEUTICALS, INC. (ALNY)

Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company primarily dedicated to the discovery, development, and commercialization of innovative therapeutic solutions leveraging ribonucleic acid interference (RNAi) technology. Its robust pipeline of RNAi-based treatments addresses a range of critical therapeutic areas, including inherited genetic disorders, cardio-metabolic conditions, hepatic infectious diseases, and central nervous system (CNS) and ocular disorders. Currently, Alnylam offers several approved therapies: ONPATTRO (patisiran) for adults suffering from polyneuropathy associated with hereditary transthyretin-mediated amyloidosis; GIVLAARI for adult patients with acute hepatic porphyria (AHP); and OXLUMO (lumasiran) for primary hyperoxaluria type 1 (PH1). Beyond its commercial portfolio, the company maintains an active development pipeline. Key investigational therapies include givosiran, aimed at adolescent patients with AHP; patisiran, being explored for transthyretin amyloidosis (ATTR) with cardiomyopathy; cemdisiran for complement-mediated disorders; ALN-AAT02 for AAT deficiency-associated liver disease; ALN-HBV02 for chronic hepatitis B virus infection; Zilebesiran for hypertension; and ALN-HSD for non-alcoholic steatohepatitis (NASH). Additionally, other candidates such as Fitusiran for hemophilia and bleeding disorders, Inclisiran for hypercholesterolemia, an expanded indication for lumasiran for advanced PH1 and recurrent kidney stones, and vutrisiran for ATTR amyloidosis (currently in Phase 3 clinical trials) are also progressing. Alnylam also engages in strategic alliances with other pharmaceutical leaders. Notable collaborations include those with Regeneron Pharmaceuticals, Inc., focused on discovering and developing RNAi therapeutics for ocular and CNS targets, and with Sanofi Genzyme for broader RNAi therapeutic development and commercialization efforts. Further licensing and partnership agreements are in place with entities such as Novartis AG, Vir Biotechnology, Inc., Dicerna Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., and PeptiDream, Inc. Established in 2002, Alnylam Pharmaceuticals maintains its corporate headquarters in Cambridge, Massachusetts.

Sector: Pharmaceutical Preparations

Industry: Biotechnology

Alnylam Pharmaceuticals, Inc. logo

Alnylam Pharmaceuticals, Inc.

NASDAQ:ALNYHealthcare
-5.17%$165.00
AH$165.88+0.53%vs close
Mkt Cap$22.1B
Float133.61M
O/S133.94M
Vol
$ Vol
RVOL
Inst Own99.1%
SI4.9%
Earnings
Dilution
Alnylam Pharmaceuticals, Inc. logoNASDAQ:ALNY$165.00-5.17%
Upcoming Catalysts
Phase Distribution
84 lead-sponsored trials·26 active·43 completed
Phase 124
Phase 1/210
Phase 220
Phase 315
Not applicable15
(11 more as collaborator)

Trials need funding. See this company's dilution risk and cash position.

Clinical Trials
Sponsor role
Status
Phase
  • Phase 3
    Completed

    APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

    patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)

    TTR-mediated Amyloidosis, Amyloidosis, Hereditary +4

    Enrollment
    225
    Start
    Nov 2013
    Primary Completion
    Aug 2017
    Completion
    Aug 2017
    NCT01960348
  • Phase 3
    Completed

    ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

    Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)

    Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary +4

    Enrollment
    206
    Start
    Dec 2014
    Primary Completion
    Mar 2017
    Completion
    Mar 2017
    NCT02319005
  • Phase 3
    Completed

    The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

    Patisiran

    Amyloidosis

    Enrollment
    211
    Start
    Jul 2015
    Primary Completion
    Nov 2022
    Completion
    Nov 2022
    NCT02510261
  • Phase 3
    Completed

    ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

    Givosiran, Placebo

    Acute Hepatic Porphyria, Acute Intermittent Porphyria +5

    Enrollment
    94
    Start
    Nov 2017
    Primary Completion
    Jan 2019
    Completion
    May 2021
    NCT03338816
  • Phase 3
    Completed

    A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

    Placebo, Lumasiran

    Primary Hyperoxaluria Type 1 (PH1)

    Enrollment
    39
    Start
    Nov 2018
    Primary Completion
    Nov 2019
    Completion
    Jan 2024
    NCT03681184
  • Phase 3
    Completed

    HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

    Patisiran, Vutrisiran

    Amyloidosis, Hereditary, Transthyretin Amyloidosis

    Enrollment
    164
    Start
    Feb 2019
    Primary Completion
    Nov 2020
    Completion
    Nov 2025
    NCT03759379
  • Phase 3
    Completed

    Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

    Patisiran

    Amyloidosis, Familial, Transthyretin Amyloidosis

    Enrollment
    24
    Start
    Mar 2019
    Primary Completion
    Oct 2020
    Completion
    Oct 2020
    NCT03862807
  • Phase 3
    Completed

    A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

    Lumasiran

    Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)

    Enrollment
    18
    Start
    Apr 2019
    Primary Completion
    Jun 2020
    Completion
    Jul 2024
    NCT03905694
  • Phase 3
    Completed

    APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

    Placebo, Patisiran

    Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

    Enrollment
    360
    Start
    Sep 2019
    Primary Completion
    Jun 2022
    Completion
    Dec 2025
    NCT03997383
  • Phase 3
    Completed

    A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

    Lumasiran

    Primary Hyperoxaluria Type 1, Primary Hyperoxaluria

    Enrollment
    21
    Start
    Jan 2020
    Primary Completion
    May 2021
    Completion
    Jun 2025
    NCT04152200
  • Phase 3
    Active, not recruiting

    HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

    Vutrisiran, Sterile Normal Saline (0.9% NaCl)

    Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

    Enrollment
    655
    Start
    Nov 2019
    Primary Completion
    May 2024
    Completion
    Dec 2026est.
    NCT04153149
  • Phase 3
    Enrolling by invitation

    A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

    Vutrisiran

    Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

    Enrollment
    700
    Start
    Dec 2024
    Primary Completion
    Mar 2028est.
    Completion
    Mar 2028est.
    NCT06679946
  • Phase 3
    Recruiting

    TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

    Nucresiran, Sterile Normal Saline (0.9% NaCl)

    Transthyretin Amyloidosis With Cardiomyopathy

    Enrollment
    1,750
    Start
    Jul 2025
    Primary Completion
    May 2030est.
    Completion
    Nov 2032est.
    NCT07052903
  • Phase 3
    Recruiting

    Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease

    Zilebesiran, Placebo

    High Risk Cardiovascular Disease, Hypertension +1

    Enrollment
    11,000
    Start
    Sep 2025
    Primary Completion
    Sep 2030est.
    Completion
    Sep 2030est.
    NCT07181109
  • Phase 3
    Recruiting

    TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

    Nucresiran, Vutrisiran

    Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN

    Enrollment
    125
    Start
    Feb 2026
    Primary Completion
    Dec 2027est.
    Completion
    Jun 2031est.
    NCT07223203
  • Phase 2
    Completed

    Intranasal ALN-RSV01 Administered to Adult Volunteers Experimentally Inoculated With Respiratory Syncytial Virus

    ALN-RSV01

    Respiratory Syncytial Virus Infections

    Enrollment
    88
    Start
    Jul 2007
    Primary Completion
    -
    Completion
    Nov 2007
    NCT00496821
  • Phase 2
    Completed

    Phase 2 Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)

    ALN-RSV01, normal saline

    Respiratory Syncytial Virus Infections

    Enrollment
    24
    Start
    Apr 2008
    Primary Completion
    Apr 2009
    Completion
    Jun 2009
    NCT00658086
  • Phase 2
    Completed

    Phase 2b Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)

    ALN-RSV01, Normal Saline

    Respiratory Syncytial Virus Infections

    Enrollment
    87
    Start
    Feb 2010
    Primary Completion
    Apr 2012
    Completion
    May 2012
    NCT01065935
  • Phase 2
    Completed

    Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

    Patisiran

    TTR-mediated Amyloidosis

    Enrollment
    29
    Start
    May 2012
    Primary Completion
    Oct 2013
    Completion
    Jan 2014
    NCT01617967
  • Phase 2
    Completed

    The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

    ALN-TTR02 (patisiran) administered by intravenous (IV) infusion

    TTR-mediated Amyloidosis

    Enrollment
    27
    Start
    Oct 2013
    Primary Completion
    Jul 2016
    Completion
    Aug 2016
    NCT01961921
  • Phase 2
    Completed

    Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

    ALN-TTRSC (revusiran) for subcutaneous administration

    TTR-mediated Amyloidosis

    Enrollment
    26
    Start
    Dec 2013
    Primary Completion
    Oct 2014
    Completion
    Jan 2015
    NCT01981837
  • Phase 2
    Completed

    A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

    Revusiran (ALN-TTRSC)

    TTR-mediated Amyloidosis

    Enrollment
    25
    Start
    Oct 2014
    Primary Completion
    Feb 2017
    Completion
    Feb 2017
    NCT02292186
  • Phase 2
    Completed

    The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

    Revusiran

    Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP) +2

    Enrollment
    12
    Start
    Oct 2015
    Primary Completion
    Feb 2017
    Completion
    Feb 2017
    NCT02595983
  • Phase 2
    Withdrawn

    A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome

    Cemdisiran

    Atypical Hemolytic Uremic Syndrome

    Enrollment
    0
    Start
    Sep 2017
    Primary Completion
    Sep 2018
    Completion
    Sep 2018
    NCT03303313
  • Phase 2
    Completed

    An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1

    Lumasiran

    PH1, Primary Hyperoxaluria +3

    Enrollment
    20
    Start
    Apr 2018
    Primary Completion
    Feb 2023
    Completion
    Feb 2023
    NCT03350451

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