Ionis Pharmaceuticals

76 total trials 9 currently recruiting 52 completed

Ionis Pharmaceuticals sponsors 76 registered US clinical trials on ClinicalTrials.gov, 9 of them currently recruiting, and 52 completed. 21 of these trials are in Phase 3-4 (later-stage) and 48 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM), with 3 trials.

Trial Pipeline

76 total, page 1 of 2

RECRUITING Phase 3

Donidalorsen Treatment in Children With Hereditary Angioedema

NCT07298447

RECRUITING Phase 1

ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome

NCT07531745

RECRUITING Phase 2

A Study to Assess the Safety, Tolerability and Efficacy of ION775

NCT07604974

RECRUITING Phase 3

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

NCT06914609

RECRUITING Phase 1

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

NCT06430385

RECRUITING Phase 1

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

NCT06150716

RECRUITING Phase 1

PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717

NCT06153966

RECRUITING

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

NCT05659901

RECRUITING Phase 1

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

NCT05127226

ACTIVE NOT RECRUITING

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

NCT06014541

ACTIVE NOT RECRUITING

The CARDIO-TTRansform Scintigraphy Sub-study

NCT06073587

ACTIVE NOT RECRUITING Phase 3

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

NCT05681351

ACTIVE NOT RECRUITING Phase 3

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

NCT05392114

ACTIVE NOT RECRUITING Phase 3

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

NCT05185843

ACTIVE NOT RECRUITING Phase 3

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

NCT05071300

ACTIVE NOT RECRUITING Phase 2

A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera

NCT05143957

ACTIVE NOT RECRUITING Phase 3

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

NCT05130450

ACTIVE NOT RECRUITING Phase 3

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)

NCT04768972

ACTIVE NOT RECRUITING Phase 3

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

NCT04849741

ACTIVE NOT RECRUITING

CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study

NCT06073574

ACTIVE NOT RECRUITING Phase 3

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)

NCT04136171

TERMINATED Phase 1

Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD)

NCT06673069

COMPLETED Phase 3

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

NCT05610280

COMPLETED Phase 1

A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants

NCT05579860

COMPLETED Phase 3

A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia

NCT05552326

COMPLETED Phase 2

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia

NCT05355402

COMPLETED Phase 2

A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled Hypertension

NCT05314439

COMPLETED Phase 3

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

NCT05139810

COMPLETED Phase 3

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

NCT05079919

COMPLETED Phase 2

A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224

NCT04932512

COMPLETED Phase 2

A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection Fraction

NCT04836182

COMPLETED Phase 2

A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood Pressure

NCT04714320

COMPLETED Phase 1

A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma

NCT04398485

COMPLETED Phase 1

A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors

NCT04659096

COMPLETED Phase 2

A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly

NCT04522180

COMPLETED Phase 3

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

NCT04568434

COMPLETED Phase 1

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants

NCT04302064

COMPLETED Phase 2

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

NCT04307381

COMPLETED Phase 2

A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

NCT04030598

COMPLETED Phase 3

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

NCT04136184

COMPLETED Phase 2

A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx

NCT04083222

COMPLETED Phase 1

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease

NCT03976349

COMPLETED Phase 2

Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands

NCT03967249

COMPLETED Phase 2

GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)

NCT03815825

COMPLETED

Natural History and Functional Status Study of Patients With Lafora Disease

NCT03876522

COMPLETED Phase 2

A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx, an Antisense Inhibitor Administered Subcutaneously to Hypertensive Participants With Controlled Blood Pressure

NCT03714776

COMPLETED Phase 1

A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants

NCT05337878

COMPLETED Phase 2

Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands

NCT03548415

COMPLETED Phase 1

A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers

NCT02910635

COMPLETED Phase 2

Safety, Tolerability and Efficacy of ISIS-GCGRRx in Patients With Type 2 Diabetes

NCT02583919

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What the Pipeline for Ionis Pharmaceuticals Shows

According to the ClinicalTrials.gov registry, Ionis Pharmaceuticals is linked to 76 US clinical trials across every stage of research activity. Of those, 9 studies are currently recruiting, about 12% of the sponsor's indexed portfolio, and 52 are already marked complete, representing roughly 68% of the total.

The phase mix for Ionis Pharmaceuticals reports 21 late-stage studies (Phase 3 and Phase 4 combined) and 48 earlier-phase studies (Phase 1 and Phase 2).

The top therapeutic focus area indexed for Ionis Pharmaceuticals is Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) with 3 linked trials, and 9 other condition areas appear in the top list above.

How to read these pipeline numbers

Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.

A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work, safety, dosing, and early signal detection, and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.

That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice, verify current details directly with ClinicalTrials.gov before relying on any figure here.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.