Ionis Pharmaceuticals
Open enrollment with an early-phase pipeline tilt
According to ClinicalTrials.gov-derived indexes, Ionis Pharmaceuticals sits in the top decile of US sponsors by registered trial count (#286 of 15,742 sponsors by registered trial count).
Ionis Pharmaceuticals: 76 sponsored US clinical trials, 9 recruiting.
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.
Top-decile sponsor footprint
Ionis Pharmaceuticals ranks in the top decile of sponsors by registered trial count (#286 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.
Trial completion reliability score
52 of 55 decided trials (Completed vs. Terminated on ClinicalTrials.gov) reached their planned completion (94.5%), vs. national p10 68.4% / p90 100% among 1,234 sponsors with at least 10 decided trials. Still-open trials (Recruiting, Active-not-recruiting, Not-yet-recruiting) are excluded since they have not reached an outcome yet.
Open and recent trials
76 total, page 1 of 2
Donidalorsen Treatment in Children With Hereditary Angioedema
NCT07298447
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
NCT07531745
A Study to Assess the Safety, Tolerability and Efficacy of ION775
NCT07604974
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
NCT06914609
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
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
NCT06150716
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
NCT06153966
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
NCT05659901
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
NCT05127226
Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
NCT06014541
The CARDIO-TTRansform Scintigraphy Sub-study
NCT06073587
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
NCT05681351
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
NCT05392114
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
NCT05185843
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
A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
NCT05143957
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
NCT05130450
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
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
NCT04849741
CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study
NCT06073574
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
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
A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia
NCT05610280
A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants
NCT05579860
A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia
NCT05552326
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
A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled Hypertension
NCT05314439
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
A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia
NCT05079919
A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224
NCT04932512
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection Fraction
NCT04836182
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood Pressure
NCT04714320
A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma
NCT04398485
A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors
NCT04659096
A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly
NCT04522180
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
NCT04568434
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
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
NCT04307381
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
NCT04030598
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
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx
NCT04083222
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
NCT03976349
Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
NCT03967249
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
Natural History and Functional Status Study of Patients With Lafora Disease
NCT03876522
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
A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants
NCT05337878
Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
NCT03548415
A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers
NCT02910635
Safety, Tolerability and Efficacy of ISIS-GCGRRx in Patients With Type 2 Diabetes
NCT02583919
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 20 |
| Phase 2 | 28 |
| Phase 3 | 21 |
Therapeutic Areas
What is open in the Ionis Pharmaceuticals pipeline
9 recruiting of 76 indexed (12% open); 52 completed (68%).
Phase mix: 21 late (III/IV) vs 48 early (I/II) (early-phase heavy).
Top condition: Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) (3 trials), 9 others listed.
How open-pipeline share is computed
Recruiting share is recruiting_count divided by trial_count for this sponsor in the public registry export. Status can change between pulls; treat the figure as a research-attention snapshot, not product availability.
Ionis Pharmaceuticals: open-pipeline peers nationwide
Peers matched on 76 registered trials and 12% open enrollment, not the therapeutic-area sidebar.
Ionis Pharmaceuticals sits at or above the median trial volume and below the median recruiting share among these 9 nationwide peers -- the two do not always move together.
Sponsors near 76 trials (± recruiting share)
- MetroHealth Medical Center · 76 trials · 13% recruiting
- Texas Tech University Health Sciences Center · 76 trials · 13% recruiting
- University of New Mexi · 76 trials · 30% recruiting
- University of Hawaii · 77 trials · 17% recruiting
Sponsors near 12% open enrollment (± trial volume)
- United States Army Research Institute of Environmental Medicine · 12% recruiting · 59 trials
- Fred Hutchinson Cancer Center · 12% recruiting · 483 trials
- Cochlear · 12% recruiting · 34 trials
- Erchonia Corporation · 12% recruiting · 34 trials
Related
What to do with this Ionis Pharmaceuticals page
76 registered trials is a research-attention snapshot, not an endorsement of Ionis Pharmaceuticals or any single study.
- 9 of Ionis Pharmaceuticals's trials are currently recruiting, check each one's own eligibility criteria before contacting a site. Browse recruiting trials
- Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) is the condition Ionis Pharmaceuticals studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Ionis Pharmaceuticals pipeline card: 76 registered trials from official public datasets.Report a data error.