Biotech · Public company · Carlsbad, CA, US
Ionis Pharmaceuticals Inc
Ticker IONS · SEC CIK 0000874015
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Clinical trials2521 recruiting or active
Pipeline programs19From trials and the company's site
Disclosed deals0None found yet
Cash runwayNot availableNo recent cash burn filed
Pipeline19
- DonidalorsenPhase 3 · Hereditary AngioedemaClinicalTrials.gov · fetched Nov 12, 2026
- DonidalorsenPhase 3 · Hereditary Angioedema (HAE)ClinicalTrials.gov · fetched Nov 12, 2026
- EplontersenPhase 3 · Hereditary Transthyretin-Mediated Amyloid PolyneuropathyClinicalTrials.gov · fetched Nov 12, 2026
- EplontersenPhase 3 · Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)ClinicalTrials.gov · fetched Nov 12, 2026
- ION363Phase 3 · Amyotrophic Lateral SclerosisClinicalTrials.gov · fetched Nov 12, 2026
- obudanersenPhase 3 · Angelman SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- OlezarsenPhase 3 · Familial Chylomicronemia SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- OlezarsenPhase 3 · Severe HypertriglyceridemiaClinicalTrials.gov · fetched Nov 12, 2026
- zilganersenPhase 3 · Alexander DiseaseClinicalTrials.gov · fetched Nov 12, 2026
- ION337Phase 2 · Dravet SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- ION440Phase 2 · Methyl CpG Binding Protein 2 (MECP2) Duplication SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- ION582Phase 2 · Angelman SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- ION717Phase 2 · Prion DiseaseClinicalTrials.gov · fetched Nov 12, 2026
- ION775Phase 2 · HypertriglyceridemiaClinicalTrials.gov · fetched Nov 12, 2026
- sapablursenPhase 2 · Phlebotomy Dependent Polycythemia VeraClinicalTrials.gov · fetched Nov 12, 2026
- Sham procedurePhase 2 · Methyl CpG Binding Protein 2 (MECP2) Duplication SyndromeClinicalTrials.gov · fetched Nov 12, 2026
- ION356Phase 1 · Pelizaeus-Merzbacher DiseaseClinicalTrials.gov · fetched Nov 12, 2026
- Cardiac MRITransthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)ClinicalTrials.gov · fetched Nov 12, 2026
- Scintigraphy scanTransthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)ClinicalTrials.gov · fetched Nov 12, 2026
Clinical trials25
- CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)PHASE3 · RECRUITING · started Sep 30, 2026ClinicalTrials.gov NCT07782827 · fetched Nov 12, 2026
- A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric ParticipantsPHASE3 · RECRUITING · started Jul 31, 2026ClinicalTrials.gov NCT07727538 · fetched Nov 12, 2026
- ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet SyndromePHASE1_PHASE2 · RECRUITING · started May 21, 2026ClinicalTrials.gov NCT07531745 · fetched Nov 12, 2026
- A Study to Assess the Safety, Tolerability and Efficacy of ION775PHASE2 · RECRUITING · started May 12, 2026ClinicalTrials.gov NCT07604974 · fetched Nov 12, 2026
- Donidalorsen Treatment in Children With Hereditary AngioedemaPHASE3 · RECRUITING · started Apr 21, 2026ClinicalTrials.gov NCT07298447 · fetched Nov 12, 2026
- REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromePHASE3 · ACTIVE_NOT_RECRUITING · started Jun 10, 2025ClinicalTrials.gov NCT06914609 · fetched Nov 12, 2026
- 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)PHASE1_PHASE2 · RECRUITING · started Oct 21, 2024ClinicalTrials.gov NCT06430385 · fetched Nov 12, 2026
- Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)PHASE1 · RECRUITING · started Apr 10, 2024ClinicalTrials.gov NCT06150716 · fetched Nov 12, 2026
- PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717PHASE1_PHASE2 · ACTIVE_NOT_RECRUITING · started Jan 4, 2024ClinicalTrials.gov NCT06153966 · fetched Nov 12, 2026
- The CARDIO-TTRansform Scintigraphy Sub-studyCOMPLETED · started Apr 4, 2023ClinicalTrials.gov NCT06073587 · fetched Nov 12, 2026
- CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)PHASE3 · ACTIVE_NOT_RECRUITING · started Dec 13, 2022ClinicalTrials.gov NCT05681351 · fetched Nov 12, 2026
- An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)PHASE3 · ENROLLING_BY_INVITATION · started Nov 30, 2022ClinicalTrials.gov NCT05667493 · fetched Nov 12, 2026
- Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher DiseaseRECRUITING · started Oct 3, 2022ClinicalTrials.gov NCT05659901 · fetched Nov 12, 2026
- A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)PHASE3 · ACTIVE_NOT_RECRUITING · started Jul 13, 2022ClinicalTrials.gov NCT05392114 · fetched Nov 12, 2026
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With VolanesorsenPHASE3 · ACTIVE_NOT_RECRUITING · started Feb 25, 2022ClinicalTrials.gov NCT05185843 · fetched Nov 12, 2026
- 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 PolyneuropathyPHASE3 · ACTIVE_NOT_RECRUITING · started Jan 4, 2022ClinicalTrials.gov NCT05071300 · fetched Nov 12, 2026
- A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia VeraPHASE2 · COMPLETED · started Dec 30, 2021ClinicalTrials.gov NCT05143957 · fetched Nov 12, 2026
- HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman SyndromePHASE1_PHASE2 · ACTIVE_NOT_RECRUITING · started Dec 22, 2021ClinicalTrials.gov NCT05127226 · fetched Nov 12, 2026
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)PHASE3 · ACTIVE_NOT_RECRUITING · started Nov 18, 2021ClinicalTrials.gov NCT05130450 · fetched Nov 12, 2026
- 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)PHASE3 · ACTIVE_NOT_RECRUITING · started Jun 14, 2021ClinicalTrials.gov NCT04768972 · fetched Nov 12, 2026
- A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)PHASE3 · ACTIVE_NOT_RECRUITING · started Jun 1, 2021ClinicalTrials.gov NCT04849741 · fetched Nov 12, 2026
- CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-studyCOMPLETED · started Mar 31, 2021ClinicalTrials.gov NCT06073574 · fetched Nov 12, 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)PHASE3 · COMPLETED · started Mar 13, 2020ClinicalTrials.gov NCT04136171 · fetched Nov 12, 2026
- The Myelin Disorders Biorepository ProjectRECRUITING · started Dec 8, 2016ClinicalTrials.gov NCT03047369 · fetched Nov 12, 2026
- Evaluation of Outcome Metrics in Alexander DiseaseRECRUITING · started Jan 26, 2016ClinicalTrials.gov NCT02714764 · fetched Nov 12, 2026
SEC filings12
- 10-Q · QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(D)SEC EDGAR · filed Nov 9, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Nov 9, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Oct 23, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Oct 21, 2026 · fetched Nov 12, 2026
- 8-K · CURRENT REPORT FILINGSEC EDGAR · filed Sep 23, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Sep 4, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Sep 4, 2026 · fetched Nov 12, 2026
- 10-Q · IONIS PHARMACEUTICALS, INC. 10-Q 06-30-2026SEC EDGAR · filed Jul 29, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Jul 29, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Jul 10, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Jul 9, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Jun 24, 2026 · fetched Nov 12, 2026