133
Trials as lead sponsor
Most active programmes
The drugs appearing most often across Ionis Pharmaceuticals's recently active trials. Comparator arms are excluded.
- Olezarsen11 trials
- Donidalorsen7 trials
- Eplontersen5 trials
- Volanesorsen5 trials
- Alicaforsen4 trials
- Inotersen3 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 Obudanersen (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
- Aug 2030A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric P…Phase 3
Top therapeutic areas
Conditions studied most often across Ionis Pharmaceuticals's recently active trials.
- Hypertriglyceridemia8 trials
- Familial Chylomicronemia Syndrome8 trials
- Hereditary Angioedema5 trials
- Severe Hypertriglyceridemia5 trials
- Angelman Syndrome3 trials
- Hypertension3 trials
Ionis Pharmaceuticals pipeline by phase
The main study drugs across 19 active trials Ionis Pharmaceuticals leads on ClinicalTrials.gov. Each drug sits in the highest phase among its currently active trials, which can be lower than a phase it has already completed.
Phase 36 drugs
| Drug | Trials | Recruiting | Next readout | Sample trials |
|---|---|---|---|---|
| Olezarsen | 4 | 1 | Mar 2027 est. | NCT07727538 · NCT05681351 · NCT05185843 |
| Donidalorsen | 2 | 1 | Dec 2026 est. | NCT07298447 · NCT05392114 |
| Eplontersen | 2 | — | Aug 2029 est. | NCT05667493 · NCT05071300 |
| obudanersen | 2 | 1 | Aug 2027 est. | NCT07782827 · NCT06914609 |
| ION363 | 1 | — | — | NCT04768972 |
| zilganersen | 1 | — | — | NCT04849741 |
Phase 21 drug
| Drug | Trials | Recruiting | Next readout | Sample trials |
|---|---|---|---|---|
| ION775 | 1 | 1 | Sept 2027 est. | NCT07604974 |
Phase 16 drugs
| Drug | Trials | Recruiting | Next readout | Sample trials |
|---|---|---|---|---|
| ION337 | 1 | 1 | Dec 2030 est. | NCT07531745 |
| ION356 | 1 | 1 | Jun 2028 est. | NCT06150716 |
| ION440 | 1 | 1 | Sept 2027 est. | NCT06430385 |
| ION464 | 1 | 1 | Sept 2027 est. | NCT04165486 |
| ION582 | 1 | — | Mar 2029 est. | NCT05127226 |
| ION717 | 1 | — | Apr 2027 est. | NCT06153966 |
Derived from 19 active trials Ionis Pharmaceuticals leads; 19 name an intervention, folding into 13 phased drugs. This is ComplyRx's reading of the registry, not Ionis Pharmaceuticals's own pipeline disclosure.
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 |
| 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 |
| PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717NCT06153966ION717 | Phase 1 | Locked · track to reveal | OngoingActive, not recruiting | 85 est. | Apr 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 Obudanersen (ION582) in Angelman SyndromeNCT06914609obudanersen | 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 |
| CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)NCT07782827obudanersen | Phase 3 | Not yet scored | OngoingNot yet recruiting | 30 est. | May 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 |
| Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher DiseaseNCT05659901 | — | Not yet scored | OngoingRecruiting | 32 est. | Mar 2029 |
| 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 | OngoingActive, not recruiting | 71 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 |
| A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric ParticipantsNCT07727538Olezarsen | Phase 3 | Locked · track to reveal | OngoingRecruiting | 12 est. | Aug 2030 |
| 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 | OngoingActive, not recruiting | 202 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 | Completed | 1,438 est. | 25 Mar 2026 |
| The CARDIO-TTRansform Scintigraphy Sub-studyNCT06073587Scintigraphy scan | — | Not yet scored | Completed | 163 est. | 25 Mar 2026 |
| CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-studyNCT06073574Cardiac MRI | — | Not yet scored | Completed | 160 est. | 13 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 |