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Ionis Pharmaceuticals, Inc. is a biotechnology company that specializes in discovering and developing antisense therapy, as well as RNA interference and CRISPR therapeutics. The company was founded in 1989 is based in Carlsbad, California. The company was previously known as Isis Pharmaceuticals until December 2015.
Drugs in Pipeline
44
Phase 3 Programs
11
Upcoming Catalysts
3
Next Catalyst
Apr 15, 2026
11wStock performance and market intelligence
4 upcoming, 1 past
For transthyretin-mediated amyloidosis. NDA filing. Extracted from SEC filing: 8-K
SourcePrimary completion for Eplontersen trial (NCT04136171) in Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
SourcePrimary completion for ION363 trial (NCT04768972) in Amyotrophic Lateral Sclerosis
SourcePrimary completion for Donidalorsen trial (NCT05392114) in Hereditary Angioedema
SourceMachine learning insights and smart money movement
Pending NDA/BLA submissions with predicted PDUFA dates
Clinical development programs and drug candidates
Alicaforsen
Ulcerative Colitis
Donidalorsen
Hereditary Angioedema
Paclitaxel
Non-small Cell Lung Cancer
Olezarsen
Familial Chylomicronemia Syndrome
zilganersen
Alexander Disease
Inotersen
FAP
ISIS 3521
Lung Cancer
ION582
Angelman Syndrome
Eplontersen
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
ION363
Amyotrophic Lateral Sclerosis
Volanesorsen
Familial Chylomicronemia Syndrome
ION904
Hypertension
ISIS 104838
Rheumatoid Arthritis
IONIS-DMPKRx
Myotonic Dystrophy Type 1
Fomivirsen sodium
Cytomegalovirus Retinitis
ISIS apoC-III Rx
Hypertriglyceridemia
ION-682884
Healthy Volunteers
ISIS 14803
Hepatitis C, Chronic
ISIS-GCGRRx- Dose Level 1
Type 2 Diabetes
ISIS-FGFR4RX
Obesity
ISIS-GCGRRx - Dose Level 1
Type 2 Diabetes Mellitus
ISIS CRP Rx
Paroxysmal Atrial Fibrillation
IONIS DGAT2Rx
Hepatic Steatosis
sapablursen
Phlebotomy Dependent Polycythemia Vera
IONIS-FB-LRx
Macular Degeneration
IONIS-GHR-LRx
Acromegaly
IONIS-STAT3Rx
Advanced Cancers
IONIS-AGT-LRx
Chronic Heart Failure With Reduced Ejection Fraction
IONIS GHR-LRx
Acromegaly
GHR-LRX
Acromegaly
ISIS-FXIRx Dose #2
Venous Thromboembolism
ISIS 14803, peginterferon alfa, ribavirin
Hepatitis C, Chronic
ION440
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
ISIS-GCGRRx
Type 2 Diabetes
ISIS-APO(a)Rx
Elevated Lipoprotein(a)
ION224
Steatohepatitis, Nonalcoholic
ISIS 443139 10 mg
Huntington's Disease
ISIS 113715
Type 2 Diabetes Mellitus
ISIS-GCCRRx
Type 2 Diabetes Mellitus
ISIS EIF4E Rx
Castrate-Resistant Prostate Cancer
ISIS 757456
Mild Hypertension
ISIS 416858
End-stage Renal Disease (ESRD)
ION717
Prion Disease
ISIS-PTP1BRx
Type 2 Diabetes Mellitus
| Drug Name | Phase | Indication | Designations |
|---|---|---|---|
Alicaforsen | Phase 3 | Ulcerative Colitis | - |
Donidalorsen | Phase 3 | Hereditary Angioedema | - |
Paclitaxel | Phase 3 | Non-small Cell Lung Cancer | - |
Olezarsen | Phase 3 | Familial Chylomicronemia Syndrome | - |
zilganersen | Phase 3 | Alexander Disease | - |
Inotersen | Phase 3 | FAP | - |
ISIS 3521 | Phase 3 | Lung Cancer | - |
ION582 | Phase 3 | Angelman Syndrome | - |
Eplontersen | Phase 3 | Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) | - |
ION363 | Phase 3 | Amyotrophic Lateral Sclerosis | - |
Volanesorsen | Phase 3 | Familial Chylomicronemia Syndrome | - |
ION904 | Phase 2 | Hypertension | - |
ISIS 104838 | Phase 2 | Rheumatoid Arthritis | - |
IONIS-DMPKRx | Phase 2 | Myotonic Dystrophy Type 1 | - |
Fomivirsen sodium | Phase 2 | Cytomegalovirus Retinitis | - |
ISIS apoC-III Rx | Phase 2 | Hypertriglyceridemia | - |
ION-682884 | Phase 2 | Healthy Volunteers | - |
ISIS 14803 | Phase 2 | Hepatitis C, Chronic | - |
ISIS-GCGRRx- Dose Level 1 | Phase 2 | Type 2 Diabetes | - |
ISIS-FGFR4RX | Phase 2 | Obesity | - |
ISIS-GCGRRx - Dose Level 1 | Phase 2 | Type 2 Diabetes Mellitus | - |
ISIS CRP Rx | Phase 2 | Paroxysmal Atrial Fibrillation | - |
IONIS DGAT2Rx | Phase 2 | Hepatic Steatosis | - |
sapablursen | Phase 2 | Phlebotomy Dependent Polycythemia Vera | - |
IONIS-FB-LRx | Phase 2 | Macular Degeneration | - |
IONIS-GHR-LRx | Phase 2 | Acromegaly | - |
IONIS-STAT3Rx | Phase 2 | Advanced Cancers | - |
IONIS-AGT-LRx | Phase 2 | Chronic Heart Failure With Reduced Ejection Fraction | - |
IONIS GHR-LRx | Phase 2 | Acromegaly | - |
GHR-LRX | Phase 2 | Acromegaly | - |
ISIS-FXIRx Dose #2 | Phase 2 | Venous Thromboembolism | - |
ISIS 14803, peginterferon alfa, ribavirin | Phase 2 | Hepatitis C, Chronic | - |
ION440 | Phase 2 | Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | - |
ISIS-GCGRRx | Phase 2 | Type 2 Diabetes | - |
ISIS-APO(a)Rx | Phase 2 | Elevated Lipoprotein(a) | - |
ION224 | Phase 2 | Steatohepatitis, Nonalcoholic | - |
ISIS 443139 10 mg | Phase 2 | Huntington's Disease | - |
ISIS 113715 | Phase 2 | Type 2 Diabetes Mellitus | - |
ISIS-GCCRRx | Phase 2 | Type 2 Diabetes Mellitus | - |
ISIS EIF4E Rx | Phase 2 | Castrate-Resistant Prostate Cancer | - |
ISIS 757456 | Phase 2 | Mild Hypertension | - |
ISIS 416858 | Phase 2 | End-stage Renal Disease (ESRD) | - |
ION717 | Phase 2 | Prion Disease | - |
ISIS-PTP1BRx | Phase 2 | Type 2 Diabetes Mellitus | - |
Approvals, filings, and latest developments