Ionis Pharmaceuticals
Trial Pipeline
Donidalorsen Treatment in Children With Hereditary Angioedema
NCT07298447
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
A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia
NCT05552326
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 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 Hypertensive Participants With Uncontrolled Blood Pressure
NCT04714320
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
NCT03976349
A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers
NCT02910635
The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
NCT02211209
Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Carboplatin and Paclitaxel
NCT01234038
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 7 |
| Phase 2 | 3 |
| Phase 3 | 41 |
Therapeutic Areas
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.
Read our methodology — how this data is sourced, computed, and verified.