Ionis Pharmaceuticals

55 total trials 35 currently recruiting 8 completed

Trial Pipeline

RECRUITING Phase 3

Donidalorsen Treatment in Children With Hereditary Angioedema

NCT07298447

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

COMPLETED Phase 3

A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia

NCT05552326

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 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 Hypertensive Participants With Uncontrolled Blood Pressure

NCT04714320

COMPLETED Phase 1

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

NCT03976349

COMPLETED Phase 1

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

NCT02910635

COMPLETED Phase 3

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

NCT02211209

COMPLETED Phase 1

Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Carboplatin and Paclitaxel

NCT01234038

What the Pipeline for Ionis Pharmaceuticals Shows

According to the ClinicalTrials.gov registry, Ionis Pharmaceuticals is linked to 55 US clinical trials across every stage of research activity. Of those, 35 studies are currently recruiting — about 64% of the sponsor's indexed portfolio — and 8 are already marked complete, representing roughly 15% of the total. 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.

The phase mix for Ionis Pharmaceuticals reports 41 late-stage studies (Phase 3 and Phase 4 combined) and 10 earlier-phase studies (Phase 1 and Phase 2). 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.

The top therapeutic focus area indexed for Ionis Pharmaceuticals is Familial Chylomicronemia Syndrome with 3 linked trials, and 9 other condition areas appear in the top list above. 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.

Related

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