IONIS PHARMACEUTICALS INC (IONS)
A+
Pipeline Grade
Financial Position (from SEC Filings)
$173.4M
Cash & Equivalents
$792.8M
Quarterly Burn Rate
1 mo
Est. Cash Runway
$9,627M
Market Cap
Estimated from most recent SEC quarterly filing. View SEC filings
Drug Pipeline (2 Assets)
| Drug | Phase | Indications |
|---|---|---|
|
OLEZARSEN SODIUM
TRYNGOLZA (AUTOINJECTOR) |
APPROVED | 4H Syndrome, Abnormalities, Cardiovascular, Acromegaly, ADLD, Adrenoleukodystrophy, Adrenomyeloneuropathy, Advanced Adult Hepatocellular Carcinoma, Advanced Cancers, Advanced Solid Tumors, Age Related Macular Degeneration, AGS, Aicardi Goutieres Syndrome, ALD, ALD (Adrenoleukodystrophy), ALD Gene Mutation, Alexander Disease, Alexanders Leukodystrophy, Allan-Herndon-Dudley Syndrome, ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation, ALSP, Alzheimer Disease, AMN, Amyloidosis, Amyloidosis, Hereditary, Amyotrophic Lateral Sclerosis, Angelman Syndrome, Atherosclerosis, Atherosclerotic Cardiovascular Disease, AxD, Beta Thalassemia Intermedia, BPAN, Cadasil, Canavan Disease, Cardiovascular Disease, Cardiovascular Diseases, Castrate Resistant Prostate Cancer, Cerebrotendinous Xanthomatoses, Charcot-Marie-Tooth, Chronic Bronchitis, Chronic Heart Failure With Reduced Ejection Fraction, Chronic Hepatitis B Atypical, Chronic Migraine, Chronic Obstructive Pulmonary Disease, CMT, Cockayne Syndrome, Congenital Abnormalit |
|
DONIDALORSEN SODIUM
DAWNZERA (AUTOINJECTOR) |
APPROVED | — |
Upcoming Catalysts
| Date | Event | Drug |
|---|---|---|
| 2026-09-22 | Pdufa | — |
| 2026-12-01 | Phase3 Readout | — |
| 2027-02-01 | Phase2 Readout | — |
| 2027-03-01 | Phase3 Readout | — |
| 2027-06-01 | Phase3 Readout | — |
Active Clinical Trials (21)
| NCT # | Title | Phase | Status | Enrollment |
|---|---|---|---|---|
| NCT06073574 | CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study | OTHER | Active Not Recruiting | 150 |
| NCT06073587 | The CARDIO-TTRansform Scintigraphy Sub-study | OTHER | Active Not Recruiting | 150 |
| NCT02714764 | Evaluation of Outcome Metrics in Alexander Disease | OTHER | Recruiting | 200 |
| NCT05659901 | Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disea... | OTHER | Recruiting | 32 |
| NCT04849741 | A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) | PHASE3 | Active Not Recruiting | 54 |
| NCT04136171 | CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-L... | PHASE3 | Active Not Recruiting | 1,438 |
| NCT05185843 | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome... | PHASE3 | Active Not Recruiting | 24 |
| NCT05130450 | A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) | PHASE3 | Active Not Recruiting | 60 |
| NCT07298447 | Donidalorsen Treatment in Children With Hereditary Angioedema | PHASE3 | Recruiting | 20 |
| NCT05681351 | CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemi... | PHASE3 | Active Not Recruiting | 885 |
| NCT05667493 | An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopath... | PHASE3 | Enrolling By Invitation | 1,400 |
| NCT05071300 | A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCE... | PHASE3 | Active Not Recruiting | 151 |
| NCT04768972 | FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral... | PHASE3 | Active Not Recruiting | 89 |
| NCT05392114 | A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioede... | PHASE3 | Active Not Recruiting | 154 |
| NCT06153966 | PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717 | PHASE2 | Recruiting | 76 |
| NCT06430385 | ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administer... | PHASE2 | Recruiting | 48 |
| NCT07531745 | ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome | PHASE2 | Recruiting | 32 |
| NCT07604974 | A Study to Assess the Safety, Tolerability and Efficacy of ION775 | PHASE2 | Recruiting | 72 |
| NCT05127226 | HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Part... | PHASE2 | Recruiting | 70 |
| NCT06150716 | Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356... | PHASE1 | Recruiting | 24 |
| NCT04165486 | Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System A... | PHASE1 | Recruiting | 40 |
Source: ClinicalTrials.gov. Trial status last updated by sponsor — may not reflect current state.