Ionis Pharmaceuticals, Inc.

IONS

NASDAQBiotechnology🇺🇸North America

Drugs in Pipeline

Phase 3 Programs

Upcoming Catalysts

Next Catalyst

Apr 15, 2026

Drug Pipeline

Drug Name	Phase	Indication	Designations
ION582	Phase 3	Angelman Syndrome	-
ION363	Phase 3	Amyotrophic Lateral Sclerosis	-
Eplontersen	Phase 3	Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)	-
ISIS 3521	Phase 3	Lung Cancer	-
Inotersen	Phase 3	FAP	-
Olezarsen	Phase 3	Familial Chylomicronemia Syndrome	-
zilganersen	Phase 3	Alexander Disease	-
Volanesorsen	Phase 3	Familial Chylomicronemia Syndrome	-
Alicaforsen	Phase 2	Ulcerative Colitis	-
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	-
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	-
Donidalorsen	Phase 2	Hereditary Angioedema	-
Paclitaxel	Phase 2	Non-small Cell Lung Cancer	-
IONIS-STAT3Rx	Phase 2	Advanced Cancers	-
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)	-
ISIS CRP Rx or Placebo	Phase 2	Rheumatoid Arthritis	-
ION224	Phase 2	Steatohepatitis, Nonalcoholic	-
ISIS 443139 10 mg	Phase 2	Huntington's Disease	-
ISIS 113715	Phase 2	Type 2 Diabetes Mellitus	-
Placebo	Phase 2	Hypertension	-
ISIS-GCCRRx	Phase 2	Type 2 Diabetes Mellitus	-

Catalyst Timeline

2 upcoming, 1 past

⭐Phase 3Next

Eplontersen Phase 3 Results Expected

April 2026~Eplontersen

Primary completion for Eplontersen trial (NCT04136171) in Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Source

⭐Phase 3

ION363 Phase 3 Results Expected

June 2026~ION363

Primary completion for ION363 trial (NCT04768972) in Amyotrophic Lateral Sclerosis

Source

Regulatory Approvals

IONS News

Ionis Pharmaceuticals, Inc.

IONS

NASDAQBiotechnology🇺🇸North America

Drugs in Pipeline

Phase 3 Programs

Upcoming Catalysts

Next Catalyst

Apr 15, 2026

Drug Pipeline

Drug Name	Phase	Indication	Designations
ION582	Phase 3	Angelman Syndrome	-
ION363	Phase 3	Amyotrophic Lateral Sclerosis	-
Eplontersen	Phase 3	Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)	-
ISIS 3521	Phase 3	Lung Cancer	-
Inotersen	Phase 3	FAP	-
Olezarsen	Phase 3	Familial Chylomicronemia Syndrome	-
zilganersen	Phase 3	Alexander Disease	-
Volanesorsen	Phase 3	Familial Chylomicronemia Syndrome	-
Alicaforsen	Phase 2	Ulcerative Colitis	-
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	-
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	-
Donidalorsen	Phase 2	Hereditary Angioedema	-
Paclitaxel	Phase 2	Non-small Cell Lung Cancer	-
IONIS-STAT3Rx	Phase 2	Advanced Cancers	-
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)	-
ISIS CRP Rx or Placebo	Phase 2	Rheumatoid Arthritis	-
ION224	Phase 2	Steatohepatitis, Nonalcoholic	-
ISIS 443139 10 mg	Phase 2	Huntington's Disease	-
ISIS 113715	Phase 2	Type 2 Diabetes Mellitus	-
Placebo	Phase 2	Hypertension	-
ISIS-GCCRRx	Phase 2	Type 2 Diabetes Mellitus	-

Catalyst Timeline

2 upcoming, 1 past

⭐Phase 3Next

Eplontersen Phase 3 Results Expected

April 2026~Eplontersen

Primary completion for Eplontersen trial (NCT04136171) in Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Source

⭐Phase 3

ION363 Phase 3 Results Expected

June 2026~ION363

Primary completion for ION363 trial (NCT04768972) in Amyotrophic Lateral Sclerosis

Source

Regulatory Approvals

IONS News