131
Trials as lead sponsor
Most active programmes
The drugs appearing most often across Ionis Pharmaceuticals's recently active trials. Comparator arms are excluded.
- Olezarsen10 trials
- Donidalorsen7 trials
- Eplontersen5 trials
- Volanesorsen5 trials
- Inotersen4 trials
- Alicaforsen4 trials
Upcoming readouts
Next primary completion dates among Ionis Pharmaceuticals's active phase 2 and phase 3 trials. Dates are registry estimates and move often, which is exactly what the tracker watches.
- Aug 2027REVEAL: A Phase 3 Study of ION582 in Angelman SyndromePhase 3
- Sept 2027A Study to Assess the Safety, Tolerability and Efficacy of ION775Phase 2
- Jun 2029Donidalorsen Treatment in Children With Hereditary AngioedemaPhase 3
Top therapeutic areas
Conditions studied most often across Ionis Pharmaceuticals's recently active trials.
- Hypertriglyceridemia8 trials
- Familial Chylomicronemia Syndrome7 trials
- Severe Hypertriglyceridemia5 trials
- Hereditary Angioedema5 trials
- Hypertension3 trials
- Acromegaly3 trials
Ionis Pharmaceuticals trials
The 50 most significant of Ionis Pharmaceuticals's recently active trials, soonest estimated readout first.
Select any row for the full record; the change timeline unlocks with tracking.
| Trial | Phase | Stability | Status | Enrollment | Est. readout |
|---|---|---|---|---|---|
| A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)NCT05392114Donidalorsen | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 154 est. | Dec 2026 |
| PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717NCT06153966ION717 | Phase 1 | Locked · track to reveal | OngoingRecruiting | 76 est. | Feb 2027 |
| CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)NCT05681351Olezarsen | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 885 est. | Mar 2027 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With VolanesorsenNCT05185843Olezarsen | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 24 est. | Jun 2027 |
| REVEAL: A Phase 3 Study of ION582 in Angelman SyndromeNCT06914609ION582 | Phase 3 | Locked · track to reveal | OngoingRecruiting | 158 est. | Aug 2027 |
| A Study to Assess the Safety, Tolerability and Efficacy of ION775NCT07604974ION775 | Phase 2 | Locked · track to reveal | OngoingRecruiting | 72 est. | Sept 2027 |
| HORIZONStudy to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)NCT04165486ION464 | Phase 1 | Locked · track to reveal | OngoingRecruiting | 40 est. | Sept 2027 |
| 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)NCT06430385ION440 | Phase 1 | Locked · track to reveal | OngoingRecruiting | 48 est. | Sept 2027 |
| A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)NCT05130450Olezarsen | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 60 est. | Feb 2028 |
| Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)NCT06150716ION356 | Phase 1 | Locked · track to reveal | OngoingRecruiting | 24 est. | Jun 2028 |
| HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman SyndromeNCT05127226ION582 | Phase 1 | Locked · track to reveal | OngoingRecruiting | 70 est. | Mar 2029 |
| Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher DiseaseNCT05659901 | — | Not yet scored | OngoingRecruiting | 32 est. | Mar 2029 |
| Donidalorsen Treatment in Children With Hereditary AngioedemaNCT07298447Donidalorsen | Phase 3 | Locked · track to reveal | OngoingRecruiting | 20 est. | Jun 2029 |
| An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)NCT05667493Eplontersen | Phase 3 | Locked · track to reveal | OngoingEnrolling by invitation | 1,400 est. | Aug 2029 |
| 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 PolyneuropathyNCT05071300Eplontersen | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 151 est. | Aug 2029 |
| TEGSEDI Pregnancy Surveillance ProgramNCT04270058inotersen | — | Not yet scored | OngoingNot yet recruiting | 20 est. | 15 Nov 2030 |
| ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet SyndromeNCT07531745ION337 | Phase 1 | Locked · track to reveal | OngoingRecruiting | 32 est. | Dec 2030 |
| A Non-interventional Cohort Safety Study of Patients With hATTR-PNNCT04850105Data Collection | — | Not yet scored | OngoingRecruiting | 240 est. | 31 Mar 2036 |
| 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)NCT04768972ION363 | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 89 est. | 17 Jun 2026 |
| 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)NCT04136171Eplontersen | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 1,438 est. | Apr 2026 |
| The CARDIO-TTRansform Scintigraphy Sub-studyNCT06073587Scintigraphy scan | — | Not yet scored | OngoingActive, not recruiting | 150 est. | Mar 2026 |
| CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-studyNCT06073574Cardiac MRI | — | Not yet scored | OngoingActive, not recruiting | 150 est. | Mar 2026 |
| 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)NCT06673069ION269 | Phase 1 | Locked · track to reveal | StoppedTerminated | 1 est. | 3 Dec 2025 |
| Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication SyndromeNCT06014541 | — | Not yet scored | StoppedTerminated | 29 est. | 7 Oct 2025 |
| A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)NCT04849741zilganersen | Phase 3 | Locked · track to reveal | OngoingActive, not recruiting | 54 est. | 22 Aug 2025 |
| A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia VeraNCT05143957sapablursen | Phase 2 | Locked · track to reveal | Completed | 50 est. | 26 Jun 2025 |
| An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary AngioedemaNCT04307381Donidalorsen | Phase 2 | Locked · track to reveal | Completed | 20 est. | 24 Jan 2025 |
| A Study of Olezarsen Administered Subcutaneously to Participants With Severe HypertriglyceridemiaNCT05552326Olezarsen | Phase 3 | Locked · track to reveal | Completed | 446 est. | 17 Dec 2024 |
| A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe HypertriglyceridemiaNCT05079919Olezarsen | Phase 3 | Locked · track to reveal | Completed | 617 est. | 22 Oct 2024 |
| A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple MyelomaNCT04398485ION251 | Phase 1 | Locked · track to reveal | Completed | 23 est. | 30 Sept 2024 |
| REASONA Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's DiseaseNCT03976349BIIB094 | Phase 1 | Locked · track to reveal | Completed | 62 est. | 12 Aug 2024 |
| A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe HypertriglyceridemiaNCT05610280Olezarsen | Phase 3 | Locked · track to reveal | Completed | 1,478 est. | 8 Aug 2024 |
| 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)NCT03815825IONIS-FB-LRx | Phase 2 | Locked · track to reveal | Completed | 332 est. | 18 Apr 2024 |
| A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PNNCT04306510TEGSEDI | Phase 4 | Locked · track to reveal | StoppedTerminated | 8 est. | 20 Mar 2024 |
| 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 NephropathyNCT04014335IONIS-FB-LRx | Phase 2 | Locked · track to reveal | Completed | 23 est. | 8 Feb 2024 |
| A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224NCT04932512ION224 | Phase 2 | Locked · track to reveal | Completed | 160 est. | 10 Jan 2024 |
| OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)NCT05139810Donidalorsen | Phase 3 | Locked · track to reveal | Completed | 91 est. | 9 Nov 2023 |
| BALANCEA Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)NCT04568434Olezarsen | Phase 3 | Locked · track to reveal | Completed | 66 est. | 14 Jul 2023 |
| A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult ParticipantsNCT05579860Olezarsen | Phase 1 | Locked · track to reveal | Completed | 104 est. | 22 May 2023 |
| 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 PolyneuropathyNCT04136184Inotersen | Phase 3 | Locked · track to reveal | Completed | 168 est. | 11 Apr 2023 |
| Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)NCT04059406sapablursen | Phase 2 | Locked · track to reveal | StoppedTerminated | 29 est. | 28 Mar 2023 |
| 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 HypertriglyceridemiaNCT05355402Olezarsen | Phase 2 | Locked · track to reveal | Completed | 154 est. | 22 Mar 2023 |
| A Study to Assess the Safety, Tolerability, and Efficacy of Monthly Subcutaneous Administration of ION904 in Participants With Uncontrolled HypertensionNCT05314439ION904 | Phase 2 | Locked · track to reveal | Completed | 48 est. | 31 Jan 2023 |
| ASTRAAS-HFA Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection FractionNCT04836182IONIS-AGT-LRx | Phase 2 | Locked · track to reveal | Completed | 72 est. | 19 Oct 2022 |
| A Study of ION537 in Patients With Molecularly Selected Advanced Solid TumorsNCT04659096ION537 | Phase 1 | Locked · track to reveal | Completed | 15 est. | 19 Oct 2022 |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547NCT04934891ION547 | Phase 1 | Locked · track to reveal | Completed | 48 est. | 14 Sept 2022 |
| A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With AcromegalyNCT04522180GHR-LRX | Phase 2 | Locked · track to reveal | Completed | 34 est. | 15 Jul 2022 |
| ASTRAASA Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood PressureNCT04714320IONIS-AGT-LRx | Phase 2 | Locked · track to reveal | Completed | 160 est. | 29 Jun 2022 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's DiseaseNCT03186989IONIS MAPTRx | Phase 1 | Locked · track to reveal | Completed | 46 est. | 12 May 2022 |
| Natural History and Functional Status Study of Patients With Lafora DiseaseNCT03876522 | — | Not yet scored | Completed | 33 est. | 1 Apr 2022 |