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IONIS PHARMACEUTICALS INC (IONS)

Founded in 1989 and headquartered in Carlsbad, California, Ionis Pharmaceuticals, Inc. is a biopharmaceutical company specializing in the discovery and development of RNA-targeted therapies within the United States. Its commercial portfolio includes three key medications: SPINRAZA, prescribed for spinal muscular atrophy (SMA) in both children and adults; TEGSEDI, an injectable treatment targeting polyneuropathy associated with hereditary transthyretin-mediated amyloidosis in adult patients; and WAYLIVRA, which addresses familial chylomicronemia syndrome and familial partial lipodystrophy. Ionis also maintains a robust pipeline, with several drug candidates currently in Phase 3 clinical trials. These include Eplontersen, a monthly self-administered subcutaneous injection designed for all forms of TTR amyloidosis; Olezarsen, intended for individuals with severe hypertriglyceridemia (SHTG); Donidalorsen, for patients suffering from hereditary angioedema; ION363, aimed at amyotrophic lateral sclerosis (ALS); Pelacarsen, developed for those with established cardiovascular disease and elevated lipoprotein(a); and Tofersen, which works by inhibiting the production of superoxide dismutase 1. Beyond these late-stage assets, the company is actively researching treatments for metabolic, infectious, renal, and ophthalmic diseases, as well as various types of cancer. The firm engages in significant collaborations, notably a strategic partnership with Biogen Inc. Additionally, it holds licensing and collaboration agreements with AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc.

Sector: Pharmaceutical Preparations

Industry: Biotechnology

Ionis Pharmaceuticals, Inc. logo

Ionis Pharmaceuticals, Inc.

NASDAQ:IONSHealthcare
-1.96%$43.52
AH$43.60+0.18%vs close
Mkt Cap$7.23B
Float163.72M
O/S166.18M
Vol
$ Vol
RVOL
Inst Own-
SI12.6%
Earnings
Dilution
Ionis Pharmaceuticals, Inc. logoNASDAQ:IONS$43.52-1.96%
Upcoming Catalysts
  • sefaxersenREADOUT
    Cash runway at this date: 4yr
    Oct 23, 202610d agoPR
  • bepirovirsenREADOUT
    Cash runway at this date: 4yr
    Oct 20, 202613d agoPR
Phase Distribution
118 lead-sponsored trials·20 active·86 completed
Phase 126
Phase 1/211
Phase 244
Phase 2/31
Phase 325
Not applicable11
(40 more as collaborator)

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

Clinical Trials
Sponsor role
Status
Phase
  • Phase 3
    Completed

    Carboplatin and Paclitaxel With or Without ISIS 3521 in Treating Patients With Non-Small Cell Lung Cancer

    ISIS 3521, carboplatin

    Lung Cancer

    Enrollment
    -
    Start
    Oct 2000
    Primary Completion
    Sep 2002
    Completion
    Sep 2002
    NCT00017407
  • Phase 3
    Completed

    Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease

    Alicaforsen

    Crohn's Disease

    Enrollment
    150
    Start
    Jan 2001
    Primary Completion
    Apr 2002
    Completion
    Apr 2002
    NCT00048113
  • Phase 3
    Completed

    Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease

    Alicaforsen

    Crohn's Disease

    Enrollment
    150
    Start
    May 2002
    Primary Completion
    -
    Completion
    -
    NCT00048295
  • Phase 3
    Completed

    Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

    Inotersen

    FAP, Familial Amyloid Polyneuropathy +3

    Enrollment
    135
    Start
    Jun 2014
    Primary Completion
    Sep 2020
    Completion
    Jan 2021
    NCT02175004
  • Phase 3
    Completed

    The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome

    Volanesorsen, Placebo

    Familial Chylomicronemia Syndrome

    Enrollment
    67
    Start
    Dec 2014
    Primary Completion
    Dec 2016
    Completion
    Mar 2017
    NCT02211209
  • Phase 3
    Completed

    The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia

    Volanesorsen, Placebo

    Hypertriglyceridemia

    Enrollment
    114
    Start
    Feb 2015
    Primary Completion
    Jul 2016
    Completion
    Jan 2017
    NCT02300233
  • Phase 3
    Completed

    CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

    Eplontersen, Placebo

    Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

    Enrollment
    1,438
    Start
    Mar 2020
    Primary Completion
    Mar 2026
    Completion
    Aug 2026
    NCT04136171
  • Phase 3
    Completed

    NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

    Inotersen, Eplontersen

    Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

    Enrollment
    168
    Start
    Dec 2019
    Primary Completion
    Apr 2023
    Completion
    Jul 2023
    NCT04136184
  • Phase 3
    Completed

    A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)

    Olezarsen, Placebo

    Familial Chylomicronemia Syndrome

    Enrollment
    66
    Start
    Nov 2020
    Primary Completion
    Jul 2023
    Completion
    Oct 2023
    NCT04568434
  • Phase 3
    Active, not recruiting

    FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

    ION363, Placebo

    Amyotrophic Lateral Sclerosis

    Enrollment
    89
    Start
    Jun 2021
    Primary Completion
    Jun 2026
    Completion
    Mar 2028est.
    NCT04768972
  • Phase 3
    Active, not recruiting

    A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

    zilganersen, Placebo

    Alexander Disease

    Enrollment
    54
    Start
    Jun 2021
    Primary Completion
    Aug 2025
    Completion
    Jul 2034est.
    NCT04849741
  • Phase 3
    Active, not recruiting

    A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

    Eplontersen

    Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

    Enrollment
    151
    Start
    Jan 2022
    Primary Completion
    Aug 2029est.
    Completion
    Aug 2029est.
    NCT05071300
  • Phase 3
    Completed

    A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia

    Olezarsen, Placebo

    Severe Hypertriglyceridemia

    Enrollment
    617
    Start
    Oct 2021
    Primary Completion
    Oct 2024
    Completion
    Jul 2025
    NCT05079919
  • Phase 3
    Active, not recruiting

    A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

    Olezarsen

    Familial Chylomicronemia Syndrome

    Enrollment
    60
    Start
    Nov 2021
    Primary Completion
    Feb 2028est.
    Completion
    Feb 2028est.
    NCT05130450
  • Phase 3
    Completed

    OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

    Donidalorsen, Placebo

    Hereditary Angioedema

    Enrollment
    91
    Start
    Dec 2021
    Primary Completion
    Nov 2023
    Completion
    Nov 2023
    NCT05139810
  • Phase 3
    Active, not recruiting

    A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen

    Olezarsen

    Familial Chylomicronemia Syndrome

    Enrollment
    24
    Start
    Feb 2022
    Primary Completion
    Jun 2027est.
    Completion
    Jun 2027est.
    NCT05185843
  • Phase 3
    Active, not recruiting

    A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

    Donidalorsen

    Hereditary Angioedema

    Enrollment
    154
    Start
    Jul 2022
    Primary Completion
    Dec 2026est.
    Completion
    Mar 2027est.
    NCT05392114
  • Phase 3
    Completed

    A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia

    Olezarsen, Placebo

    Severe Hypertriglyceridemia

    Enrollment
    446
    Start
    Aug 2022
    Primary Completion
    Dec 2024
    Completion
    Sep 2025
    NCT05552326
  • Phase 3
    Completed

    A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia

    Olezarsen, Placebo

    Hypertriglyceridemia, Cardiovascular Diseases +1

    Enrollment
    1,478
    Start
    Nov 2022
    Primary Completion
    Aug 2024
    Completion
    Jun 2025
    NCT05610280
  • Phase 3
    Enrolling by invitation

    An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

    Eplontersen

    Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

    Enrollment
    1,400
    Start
    Nov 2022
    Primary Completion
    Aug 2029est.
    Completion
    Aug 2029est.
    NCT05667493
  • Phase 3
    Active, not recruiting

    CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)

    Olezarsen

    Severe Hypertriglyceridemia

    Enrollment
    885
    Start
    Dec 2022
    Primary Completion
    Mar 2027est.
    Completion
    Mar 2027est.
    NCT05681351
  • Phase 3
    Active, not recruiting

    REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome

    obudanersen, Placebo

    Angelman Syndrome

    Enrollment
    156
    Start
    Jun 2025
    Primary Completion
    Aug 2027est.
    Completion
    Apr 2030est.
    NCT06914609
  • Phase 3
    Recruiting

    Donidalorsen Treatment in Children With Hereditary Angioedema

    Donidalorsen

    Hereditary Angioedema (HAE)

    Enrollment
    20
    Start
    Apr 2026
    Primary Completion
    Jun 2029est.
    Completion
    Jun 2029est.
    NCT07298447
  • Phase 3
    Recruiting

    A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants

    Olezarsen

    Familial Chylomicronemia Syndrome

    Enrollment
    12
    Start
    Jul 2026
    Primary Completion
    Aug 2030est.
    Completion
    Aug 2031est.
    NCT07727538
  • Phase 3
    Recruiting

    CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)

    obudanersen

    Angelman Syndrome

    Enrollment
    30
    Start
    Sep 2026
    Primary Completion
    May 2028est.
    Completion
    May 2031est.
    NCT07782827

1-25 of 118

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