Pipeline — registered trials by phase
The CARDIO-TTRansform Scintigraphy Sub-study
— · Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) · Completed · est. 2026-03-25
A Comparison of ISIS 2922 Used Immediately or Later in Patients With Cytomegalovirus (CMV) of the Eyes
— · Cytomegalovirus Retinitis, HIV Infections · Completed
Natural History and Functional Status Study of Patients With Lafora Disease
— · Lafora Disease · Completed · est. 2022-04-01
A Study of ISIS 2922 in the Treatment of Advanced Cytomegalovirus Retinitis
— · Cytomegalovirus Retinitis, HIV Infections · Completed
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
— · Parkinson's Disease · Completed · est. 2024-08-12
Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy Volunteers
— · Type 2 Diabetes Mellitus · Completed · est. 2011-10
Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715
— · Type 2 Diabetes Mellitus · Completed
A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers
— · Abnormalities, Cardiovascular · Completed · est. 2016-12-20
A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis
— · Healthy Subjects, Cystic Fibrosis · Completed · est. 2020-10-13
Safety Study of ISIS 325568 in Healthy Volunteers
— · Type 2 Diabetes Mellitus · Completed · est. 2008-04
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy Volunteers
— · Healthy Volunteers · Completed · est. 2018-03-29
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
— · Pelizaeus-Merzbacher Disease · Recruiting · est. 2028-06
Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated Triglycerides
— · Elevated Triglycerides (TG) · Completed · est. 2018-04-30
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
— · Healthy Participants · Completed · est. 2020-09-10
A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of IONIS FXI-LRx in up to 84 Healthy Volunteers
— · Healthy Participants · Completed · est. 2019-04-26
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers
— · Healthy Volunteers · Completed · est. 2018-10-09
A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants
— · Healthy Participants · Completed · est. 2019-12-18
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease
— · Mild Alzheimer's Disease · Completed · est. 2022-05-12
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
— · Thalassemia · Completed · est. 2018-09-25
Safety and Tolerability of Single and Multiple Doses of ISIS 353512 in Healthy Volunteers
— · Inflammatory Diseases · Completed · est. 2010-02
A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants
— · Healthy Participants · Completed · est. 2023-05-22
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
— · Multiple System Atrophy · Recruiting · est. 2027-09
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547
— · Healthy Volunteers · Completed · est. 2022-09-14
A Study of ISIS 416858 Administered Subcutaneously to Participants With End-Stage Renal Disease (ESRD) on Hemodialysis
— · End-stage Renal Disease (ESRD) · Completed · est. 2019-07-10
A Safety, Tolerability, PK, and PD Study of Once Weekly ISIS-FGFR4RX SC in Obese Patients
— · Obesity · Completed · est. 2016-07
Placebo Controlled, Dose Escalation Study in Subjects With Type 2 Diabetes Mellitus Being Treated With Sulfonylurea
— · Type 2 Diabetes Mellitus · Completed · est. 2009-03
A Study to Assess the Safety, Tolerability and Efficacy of ION775
— · Hypertriglyceridemia, Severe Hypertriglyceridemia · Recruiting · est. 2027-09
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
— · Angelman Syndrome · Active Not Recruiting · est. 2029-03
Safety, Tolerability, and Efficacy of ISIS-PTP1BRx in Type 2 Diabetes
— · Type 2 Diabetes Mellitus, Obese · Completed · est. 2014-10
A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
— · Phlebotomy Dependent Polycythemia Vera · Completed · est. 2025-06-26
A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy
— · Primary IgA Nephropathy · Completed · est. 2024-02-08
Addition of ISIS 14803 to Therapy With Peginterferon and Ribavirin for Chronic Hepatitis C (HCV) Patients Not Responding Adequately to the Two Drugs
— · Hepatitis C, Chronic · Completed · est. 2004-01
ISIS 104838, an Inhibitor of Tumor Necrosis Factor, for Active Rheumatoid Arthritis
— · Rheumatoid Arthritis · Completed · est. 2003-02-01
A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1
— · Myotonic Dystrophy Type 1 · Completed · est. 2016-07
A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx
— · Hypertension · Completed · est. 2020-07-20
Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 Diabetes
— · Hepatic Steatosis · Completed · est. 2018-11-28
ISIS 2302-CS27, A 6-Week, Placebo-Controlled Clinical Study to Evaluate the Effectiveness of Alicaforsen (ISIS 2302) in Patients With Mild to Moderate Active Ulcerative Colitis.
— · Ulcerative Colitis · Completed · est. 2004-10-11
Safety, Tolerability and Efficacy of ISIS-GCGRRx in Type 2 Diabetes
— · Type 2 Diabetes Mellitus · Completed · est. 2014-05
Extension Study of IONIS-GHR-LRx Administered to Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands
— · Acromegaly · Completed · est. 2022-03-31
A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224
— · Steatohepatitis, Nonalcoholic · Completed · est. 2024-01-10
Safety, Tolerability, and Pharmacokinetic Study of ISIS ApoC-III Rx in Hypertriglyceridemia
— · Hypertriglyceridemia · Completed · est. 2014-01
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
— · Hereditary Angioedema · Completed · est. 2021-01-04
Safety and Tolerability Study of ISIS EIF4E Rx in Combination With Carboplatin and Paclitaxel
— · Non-small Cell Lung Cancer · Completed · est. 2012-10
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
— · Prion Disease · Active Not Recruiting · est. 2027-04
Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).
— · Healthy Volunteers, hATTR Amyloidosis · Completed · est. 2020-02-20
A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled Hypertension
— · Hypertension · Completed · est. 2023-01-31
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)
— · Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome · Recruiting · est. 2027-09
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
— · Hereditary Angioedema · Completed · est. 2025-01-24
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
— · Familial Chylomicronemia Syndrome · Active Not Recruiting · est. 2028-02
Carboplatin and Paclitaxel With or Without ISIS 3521 in Treating Patients With Non-Small Cell Lung Cancer
— · Lung Cancer · Completed · est. 2002-09-09
CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)
— · Angelman Syndrome · Recruiting · est. 2028-05
A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia
— · Severe Hypertriglyceridemia · Completed · est. 2024-10-22
Donidalorsen Treatment in Children With Hereditary Angioedema
— · Hereditary Angioedema (HAE) · Recruiting · est. 2029-06
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)
— · Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) · Completed · est. 2026-03-25
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
— · Hereditary Angioedema · Completed · est. 2023-11-09
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
— · Alexander Disease · Active Not Recruiting · est. 2025-08-22
A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia
— · Severe Hypertriglyceridemia · Completed · est. 2024-12-17
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
— · Familial Chylomicronemia Syndrome · Active Not Recruiting · est. 2027-06
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
— · Severe Hypertriglyceridemia · Active Not Recruiting · est. 2027-03
The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia
— · Hypertriglyceridemia · Completed · est. 2016-07-27
Alicaforsen (ISIS 2302) in Patients With Active Crohn's Disease
— · Crohn's Disease · Completed
Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
— · FAP, Familial Amyloid Polyneuropathy, TTR · Completed · est. 2020-09-11
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
— · Hereditary Angioedema · Active Not Recruiting · est. 2026-12
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)
— · Amyotrophic Lateral Sclerosis · Active Not Recruiting · est. 2026-06-17
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
— · Hereditary Transthyretin-Mediated Amyloid Polyneuropathy · Active Not Recruiting · est. 2029-08
No approved products listed under this sponsor in Drugs@FDA (partnered or subsidiary products may be listed elsewhere).
Source: ClinicalTrials.gov (trials where Ionis is lead sponsor; 76 active or completed) and Drugs@FDA (0 approved products). Registry data can include older or partnered programs; the company's own pipeline page is the official list.
SEC filings — insider trading forms excluded
- 8-K2026-09-23Current report (material event)
- 8-K2026-09-04Current report (material event)
- 8-K2026-09-04Current report (material event)
- S-82026-07-31Employee stock plan
- 10-Q2026-07-29Quarterly report
- 8-K2026-07-29Current report (material event)
- 8-K2026-07-10Current report (material event)
- 8-K2026-07-09Current report (material event)
- 8-K2026-06-24Current report (material event)
- 8-K2026-06-12Current report (material event)
- 8-K2026-06-08Current report (material event)
- 8-K2026-05-28Current report (material event)
- SCHEDULE 13G/A2026-05-15SCHEDULE 13G/A
- SCHEDULE 13G/A2026-05-14SCHEDULE 13G/A
- SCHEDULE 13G2026-04-30SCHEDULE 13G
- 10-Q2026-04-29Quarterly report
- 8-K2026-04-29Current report (material event)
- ARS2026-04-23Annual report to holders
- DEFA14A2026-04-23DEFA14A
- DEF 14A2026-04-23Proxy statement
- 8-K2026-04-21Current report (material event)
- SCHEDULE 13G/A2026-03-27SCHEDULE 13G/A
- 8-K2026-03-09Current report (material event)
- 10-K2026-02-26Annual report
- 8-K2026-02-25Current report (material event)
- SCHEDULE 13G/A2026-02-17SCHEDULE 13G/A
- 8-K2026-01-12Current report (material event)
- SCHEDULE 13G/A2026-01-07SCHEDULE 13G/A
- 8-K2026-01-07Current report (material event)
- 8-K2025-11-17Current report (material event)
- SCHEDULE 13G/A2025-11-14SCHEDULE 13G/A
- 8-K2025-11-10Current report (material event)
- 8-K/A2025-11-03Current report (amended)
- 10-Q2025-10-29Quarterly report
- 8-K2025-10-29Current report (material event)
- 8-K2025-09-22Current report (material event)
- 8-K2025-09-02Current report (material event)
- 8-K2025-08-21Current report (material event)
- SCHEDULE 13G/A2025-08-12SCHEDULE 13G/A
- S-82025-07-30Employee stock plan
Source: SEC EDGAR. Forms 3/4/5 and 144 (insider transactions) are intentionally left out.