IONIS PHARMACEUTICALS

Latest clinical program signals indexed for IONIS PHARMACEUTICALS.

1. ION373-CS1 — Phase 2

   Jun 8, 2026
   Clinical update →

2. ISIS 678354-CS5 — Phase 3

   Jun 8, 2026
   Clinical update →

3. ISIS 678354-CS13 — Phase 3

   Jun 8, 2026
   Clinical update →

4. ION363-CS1 — Phase 3

   Jun 8, 2026
   Clinical update →

5. ION269-CS1 — Phase 1

   Jun 8, 2026
   Clinical update →

6. ION-682884-CS2 — Phase 3

   Jun 8, 2026
   Clinical update →

7. 262SP101(ION464-CS1) — Phase 1

   Jun 8, 2026
   Clinical update →

8. ISIS 14803-CS3 — Phase 2

   Jun 8, 2026
   Clinical update →

1. 2023-507192-22-00

   ION440

   Phase 2 · active · small_molecule

   Methyl CpG binding protein 2 (MECP2) Duplication Syndrome (MDS)

   CTIS ↗
2. 2023-508815-22-00

   ISIS 678354

   Phase 3 · active · small_molecule

   Familial Chylomicronemia Syndrome

   CTIS ↗
3. 2023-509029-29-00

   ISIS 678354-CS13

   Phase 3 · active · small_molecule

   Familial Chylomicronemia Syndrome (FCS)

   CTIS ↗
4. 2023-509201-77-00

   ISIS 721744

   Phase 3 · active · small_molecule

   Hereditary Angioedema

   CTIS ↗
5. 2023-509257-31-00

   ION269-CS1

   Phase 1 · active · other

   Alzheimer’s Disease in Individuals with Down Syndrome

   CTIS ↗
6. 2024-510603-11-00

   ION373-CS1

   Phase 2 · active · small_molecule

   Alexander Disease

   CTIS ↗
7. 2024-510696-38-00

   ISIS 678354-CS5

   Phase 3 · active · small_molecule

   Severe Hypertriglyceridemia.

   CTIS ↗
8. 2024-511201-32-00

   ION 682884, ION 682884

   Phase 3 · active · small_molecule

   Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

   CTIS ↗
9. 2024-512163-31-00

   ION363-CS1

   Phase 3 · active · small_molecule

   Amyotrophic Lateral Sclerosis with Fused in Sarcoma mutations

   CTIS ↗
10. 2024-512482-14-00

    ISIS 702843

    Phase 2 · active · small_molecule

    Polycythemia Vera

    CTIS ↗
11. 2024-512528-13-00

    262SP101(ION464-CS1)

    Phase 1 · active · other

    Multiple System Atrophy

    CTIS ↗
12. 2024-512589-32-00

    ION582

    Phase 2 · active · small_molecule

    Angelman syndrome

    CTIS ↗
13. 2024-514434-20-00

    ION-682884-CS2

    Phase 3 · active · small_molecule

    Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

    CTIS ↗
14. 2024-517249-15-00

    ISIS 721744

    Phase 2 · active · small_molecule

    Hereditary Angioedema

    CTIS ↗
15. 2024-519711-33-01

    ION582

    Phase 3 · active · small_molecule

    Angelman syndrome

    CTIS ↗
16. 2025-523499-22-00

    ISIS 721744, ISIS 721744

    Phase 3 · active · small_molecule

    Hereditary Angioedema (HAE)

    CTIS ↗
17. NCT00002156

    Fomivirsen sodium

    Phase 2 · completed · small_molecule

    Cytomegalovirus Retinitis

    CT.gov ↗
18. NCT00002187

    Fomivirsen sodium

    Phase 2 · completed · small_molecule

    Cytomegalovirus Retinitis

    CT.gov ↗
19. NCT00002355

    Fomivirsen sodium

    Phase 2 · completed · small_molecule

    Cytomegalovirus Retinitis

    CT.gov ↗
20. NCT00002356

    Fomivirsen sodium

    Phase 2 · completed · small_molecule

    Cytomegalovirus Retinitis

    CT.gov ↗
21. NCT00005594

    ISIS 2503

    Phase 2 · completed · small_molecule

    Pancreatic Cancer

    CT.gov ↗
22. NCT00017407

    ISIS 3521

    Phase 3 · completed · mab

    Lung Cancer

    CT.gov ↗
23. NCT00035945

    ISIS 14803

    Phase 2 · completed · small_molecule

    Hepatitis C, Chronic

    CT.gov ↗
24. NCT00048113

    Alicaforsen

    Phase 3 · completed · small_molecule

    Crohn's Disease

    CT.gov ↗
25. NCT00048295

    Alicaforsen

    Phase 3 · completed · small_molecule

    Crohn's Disease

    CT.gov ↗
26. NCT00048321

    ISIS 104838

    Phase 2 · completed · small_molecule

    Rheumatoid Arthritis

    CT.gov ↗
27. NCT00062816

    ISIS 14803-CS3

    Phase 2 · completed · small_molecule

    Hepatitis C, Chronic

    CT.gov ↗
28. NCT00063414

    Alicaforsen

    Phase 2 · completed · small_molecule

    Ulcerative Colitis

    CT.gov ↗
29. NCT00063830

    Alicaforsen

    Phase 2 · completed · small_molecule

    Ulcerative Colitis

    CT.gov ↗
30. NCT00216463

    ISIS 301012 or Placebo

    Phase 2 · completed · small_molecule

    Hypercholesterolemia

    CT.gov ↗
31. NCT00231569

    ISIS 301012 or Placebo

    Phase 2 · completed · small_molecule

    Hypercholesterolemia

    CT.gov ↗
32. NCT00280995

    ISIS 301012

    Phase 2 · completed · small_molecule

    Hypercholesterolemia, Familial

    CT.gov ↗
33. NCT00281008

    ISIS 301012 or Placebo

    Phase 2 · completed · small_molecule

    Hypercholesterolemia, Familial

    CT.gov ↗
34. NCT00327626

    ISIS 113715

    Phase 2 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
35. NCT00330200

    ISIS 113715

    Phase 2 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
36. NCT00330330

    ISIS 113715

    Phase 2 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
37. NCT00362180

    mipomersen

    Phase 3 · completed · small_molecule

    Lipid Metabolism, Inborn Errors

    CT.gov ↗
38. NCT00365781

    ISIS 113715

    Phase 2 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
39. NCT00455598

    ISIS 113715

    Phase 2 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
40. NCT00477594

    mipomersen sodium

    Phase 3 · completed · small_molecule

    Lipid Metabolism, Inborn Errors

    CT.gov ↗
41. NCT00519727

    ISIS 325568

    Phase 1 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
42. NCT00607373

    mipomersen

    Phase 3 · completed · small_molecule

    Lipid Metabolism, Inborn Errors

    CT.gov ↗
43. NCT00694109

    mipomersen sodium

    Phase 3 · completed · small_molecule

    Lipid Metabolism, Inborn Errors

    CT.gov ↗
44. NCT00706849

    mipomersen sodium

    Phase 3 · completed · small_molecule

    Heterozygous Familial Hypercholesterolemia

    CT.gov ↗
45. NCT00707746

    mipomersen

    Phase 2 · completed · small_molecule

    Metabolic Diseases

    CT.gov ↗
46. NCT00734240

    ISIS 353512

    Phase 1 · completed · small_molecule

    Inflammatory Diseases

    CT.gov ↗
47. NCT00770146

    Mipomersen sodium

    Phase 3 · completed · small_molecule

    Hypercholesterolemia

    CT.gov ↗
48. NCT00794664

    Mipomersen

    Phase 3 · completed · small_molecule

    Hypercholesterolemia

    CT.gov ↗
49. NCT00836225

    ISIS 388626

    Phase 1 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
50. NCT01041222

    ISIS 333611

    Phase 1 · completed · small_molecule

    Familial Amyotrophic Lateral Sclerosis

    CT.gov ↗
51. NCT01061814

    mipomersen

    Phase 1 · completed · small_molecule

    Healthy Volunteer

    CT.gov ↗
52. NCT01090661

    mipomersen sodium

    Phase 1 · completed · small_molecule

    Healthy

    CT.gov ↗
53. NCT01133366

    warfarin sodium

    Phase 1 · completed · small_molecule

    Healthy

    CT.gov ↗
54. NCT01234025

    ISIS EIF4E Rx

    Phase 2 · completed · small_molecule

    Castrate-Resistant Prostate Cancer

    CT.gov ↗
55. NCT01234038

    ISIS EIF4E Rx

    Phase 2 · completed · small_molecule

    Non-small Cell Lung Cancer

    CT.gov ↗
56. NCT01299298

    mipomersen

    Phase 1 · completed · small_molecule

    Healthy Volunteer

    CT.gov ↗
57. NCT01414101

    ISIS CRP Rx or Placebo

    Phase 2 · completed · small_molecule

    Rheumatoid Arthritis

    CT.gov ↗
58. NCT01414881

    mipomersen

    Phase 1 · completed · small_molecule

    Healthy Volunteer

    CT.gov ↗
59. NCT01529424

    ISIS apoC-III Rx

    Phase 2 · completed · small_molecule

    Hypertriglyceridemia

    CT.gov ↗
60. NCT01563302

    IONIS-STAT3Rx

    Phase 2 · completed · small_molecule

    Advanced Cancers

    CT.gov ↗
61. NCT01647308

    ISIS-APOCIIIRX

    Phase 2 · terminated · small_molecule

    Type 2 Diabetes

    CT.gov ↗
62. NCT01710852

    ISIS CRP Rx

    Phase 2 · completed · small_molecule

    Paroxysmal Atrial Fibrillation

    CT.gov ↗
63. NCT01713361

    ISIS-FXIRx Dose #2

    Phase 2 · completed · small_molecule

    Venous Thromboembolism

    CT.gov ↗
64. NCT01737398

    Inotersen

    Phase 3 · completed · small_molecule

    FAP

    CT.gov ↗
65. NCT01839604

    AZD9150

    Phase 1 · completed · small_molecule

    Advanced Adult Hepatocellular Carcinoma

    CT.gov ↗
66. NCT01885260

    ISIS-GCGRRx - Dose Level 1

    Phase 2 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
67. NCT01918865

    ISIS-PTP1BRx

    Phase 2 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
68. NCT01968265

    ISIS-GCCRRx

    Phase 2 · completed · small_molecule

    Type 2 Diabetes Mellitus

    CT.gov ↗
69. NCT02160899

    ISIS-APO(a)Rx

    Phase 2 · completed · small_molecule

    Elevated Lipoprotein(a)

    CT.gov ↗
70. NCT02175004

    Inotersen

    Phase 3 · completed · small_molecule

    FAP

    CT.gov ↗
71. NCT02211209

    Volanesorsen

    Phase 3 · completed · small_molecule

    Familial Chylomicronemia Syndrome

    CT.gov ↗
72. NCT02300233

    Volanesorsen

    Phase 3 · completed · small_molecule

    Hypertriglyceridemia

    CT.gov ↗
73. NCT02312011

    IONIS-DMPKRx

    Phase 2 · completed · small_molecule

    Myotonic Dystrophy Type 1

    CT.gov ↗
74. NCT02414594

    IONIS-APO(a)-LRx

    Phase 1 · completed · small_molecule

    Elevated Lipoprotein(a)

    CT.gov ↗
75. NCT02476019

    ISIS-FGFR4RX

    Phase 2 · completed · small_molecule

    Obesity

    CT.gov ↗
76. NCT02519036

    ISIS 443139 10 mg

    Phase 2 · completed · small_molecule

    Huntington's Disease

    CT.gov ↗
77. NCT02527343

    volanesorsen

    Phase 3 · terminated · small_molecule

    Familial Partial Lipodystrophy

    CT.gov ↗
78. NCT02553889

    ISIS 416858

    Phase 2 · completed · small_molecule

    End-stage Renal Disease (ESRD)

    CT.gov ↗
79. NCT02583919

    ISIS-GCGRRx- Dose Level 1

    Phase 2 · completed · small_molecule

    Type 2 Diabetes

    CT.gov ↗
80. NCT02623699

    Tofersen

    Phase 3 · completed · small_molecule

    Amyotrophic Lateral Sclerosis

    CT.gov ↗
81. NCT02627820

    Isis 420915/GSK 299872

    Phase 2 · terminated · small_molecule

    Amyloidosis

    CT.gov ↗
82. NCT02658175

    Volanesorsen

    Phase 3 · completed · small_molecule

    Familial Chylomicronemia Syndrome

    CT.gov ↗
83. NCT02709850

    IONIS ANGPTL3-LRx

    Phase 1 · completed · small_molecule

    Hypertriglyceridemia

    CT.gov ↗
84. NCT02824003

    ISIS-GCGRRx

    Phase 2 · completed · small_molecule

    Type 2 Diabetes

    CT.gov ↗
85. NCT02900027

    APOC-III-L-Rx

    Phase 1 · completed · small_molecule

    Elevated Triglycerides (TG)

    CT.gov ↗
86. NCT02910635

    Volanesorsen

    Phase 1 · completed · small_molecule

    Abnormalities, Cardiovascular

    CT.gov ↗
87. NCT02981602

    IONIS-HBVRx

    Phase 2 · completed · small_molecule

    Hepatitis B

    CT.gov ↗
88. NCT03070119

    Tofersen

    Phase 3 · completed · small_molecule

    ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation

    CT.gov ↗
89. NCT03070782

    ISIS 681257

    Phase 2 · completed · small_molecule

    Elevated Lipoprotein(a)

    CT.gov ↗
90. NCT03101878

    Ionis AGT-LRx

    Phase 1 · completed · small_molecule

    Healthy Volunteers

    CT.gov ↗
91. NCT03108469

    IONIS-PKKRx (ISIS 546254)

    Phase 2 · completed · small_molecule

    Chronic Migraine

    CT.gov ↗
92. NCT03165864

    IONIS TMPRSS6-Lrx

    Phase 1 · completed · small_molecule

    Thalassemia

    CT.gov ↗
93. NCT03186989

    IONIS MAPTRx

    Phase 1 · completed · small_molecule

    Mild Alzheimer's Disease

    CT.gov ↗
94. NCT03263507

    Placebo

    Phase 1 · completed · small_molecule

    Healthy Volunteers

    CT.gov ↗
95. NCT03334214

    IONIS DGAT2Rx

    Phase 2 · completed · small_molecule

    Hepatic Steatosis

    CT.gov ↗
96. NCT03358030

    ISIS 416858

    Phase 2 · completed · small_molecule

    End-stage Renal Disease (ESRD)

    CT.gov ↗
97. NCT03360747

    AKCEA-ANGPTL3-LRx

    Phase 2 · completed · small_molecule

    Familial Chylomicronemia Syndrome

    CT.gov ↗
98. NCT03371355

    Placebo

    Phase 2 · completed · small_molecule

    NAFLD

    CT.gov ↗
99. NCT03385239

    ISIS 678354

    Phase 2 · completed · small_molecule

    Hypertriglyceridemia

    CT.gov ↗
100. NCT03446144

     IONIS-FB-Lrx

     Phase 2 · terminated · small_molecule

     Geographic Atrophy

     CT.gov ↗
101. NCT03455777

     AKCEA-ANGPTL3-LRX

     Phase 2 · terminated · small_molecule

     Homozygous Familial Hypercholesterolemia

     CT.gov ↗
102. NCT03506854

     ISIS 681257

     Phase 1 · completed · small_molecule

     Renal Impairment

     CT.gov ↗
103. NCT03514420

     AKCEA-ANGPTL3-LRx

     Phase 2 · completed · small_molecule

     Familial Partial Lipodystrophy

     CT.gov ↗
104. NCT03548415

     IONIS GHR-LRx

     Phase 2 · completed · small_molecule

     Acromegaly

     CT.gov ↗
105. NCT03582462

     IONIS FXI-LRx

     Phase 1 · completed · small_molecule

     Healthy Participants

     CT.gov ↗
106. NCT03647228

     IONIS-ENaCRx

     Phase 1 · completed · small_molecule

     Healthy Subjects

     CT.gov ↗
107. NCT03714776

     Placebo

     Phase 2 · completed · small_molecule

     Mild Hypertension

     CT.gov ↗
108. NCT03728634

     ION-682884

     Phase 2 · completed · small_molecule

     Healthy Volunteers

     CT.gov ↗
109. NCT03815825

     IONIS-FB-LRx

     Phase 2 · completed · small_molecule

     Macular Degeneration

     CT.gov ↗
110. NCT03967249

     IONIS GHR-LRx

     Phase 2 · completed · small_molecule

     Acromegaly

     CT.gov ↗
111. NCT03976349

     BIIB094

     Phase 1 · completed · small_molecule

     Parkinson's Disease

     CT.gov ↗
112. NCT04014335

     IONIS-FB-LRx

     Phase 2 · completed · small_molecule

     Primary IgA Nephropathy

     CT.gov ↗
113. NCT04030598

     Donidalorsen

     Phase 3 · active · small_molecule

     Hereditary Angioedema

     CT.gov ↗
114. NCT04059406

     sapablursen

     Phase 2 · terminated · small_molecule

     Beta Thalassemia Intermedia

     CT.gov ↗
115. NCT04083222

     Placebo

     Phase 2 · completed · small_molecule

     Hypertension

     CT.gov ↗
116. NCT04136171

     Eplontersen

     Phase 3 · active · small_molecule

     Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

     CT.gov ↗
117. NCT04136184

     Inotersen

     Phase 3 · completed · small_molecule

     Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

     CT.gov ↗
118. NCT04165486

     ION464

     Phase 1 · active · small_molecule

     Multiple System Atrophy

     CT.gov ↗
119. NCT04302064

     Eplontersen

     Phase 1 · completed · small_molecule

     Healthy Participants

     CT.gov ↗
120. NCT04307381

     Donidalorsen

     Phase 3 · active · small_molecule

     Hereditary Angioedema

     CT.gov ↗
121. NCT04398485

     ION251

     Phase 1 · completed · small_molecule

     Relapsed Multiple Myeloma

     CT.gov ↗
122. NCT04441788

     ION-827359

     Phase 2 · terminated · small_molecule

     Chronic Bronchitis

     CT.gov ↗
123. NCT04522180

     GHR-LRX

     Phase 2 · completed · small_molecule

     Acromegaly

     CT.gov ↗
124. NCT04549922

     ISIS 721744

     Phase 2 · completed · small_molecule

     Covid19

     CT.gov ↗
125. NCT04568434

     Olezarsen

     Phase 3 · active · small_molecule

     Familial Chylomicronemia Syndrome

     CT.gov ↗
126. NCT04659096

     ION537

     Phase 1 · completed · small_molecule

     Advanced Solid Tumors

     CT.gov ↗
127. NCT04714320

     Placebo

     Phase 2 · completed · small_molecule

     Hypertension

     CT.gov ↗
128. NCT04731623

     ION904

     Phase 1 · completed · small_molecule

     Healthy Volunteers

     CT.gov ↗
129. NCT04768972

     ION363

     Phase 3 · active · small_molecule

     Amyotrophic Lateral Sclerosis

     CT.gov ↗
130. NCT04836182

     IONIS-AGT-LRx

     Phase 2 · completed · small_molecule

     Chronic Heart Failure With Reduced Ejection Fraction

     CT.gov ↗
131. NCT04849741

     zilganersen

     Phase 3 · active · small_molecule

     Alexander Disease

     CT.gov ↗
132. NCT04932512

     ION224

     Phase 2 · completed · small_molecule

     Steatohepatitis, Nonalcoholic

     CT.gov ↗
133. NCT04934891

     ION547

     Phase 1 · completed · small_molecule

     Healthy Volunteers

     CT.gov ↗
134. NCT05071300

     Eplontersen

     Phase 3 · active · small_molecule

     Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

     CT.gov ↗
135. NCT05079919

     Olezarsen

     Phase 3 · completed · small_molecule

     Severe Hypertriglyceridemia

     CT.gov ↗
136. NCT05127226

     ION582

     Phase 3 · active · small_molecule

     Angelman Syndrome

     CT.gov ↗
137. NCT05130450

     Olezarsen

     Phase 3 · active · small_molecule

     Familial Chylomicronemia Syndrome

     CT.gov ↗
138. NCT05139810

     Donidalorsen

     Phase 3 · active · small_molecule

     Hereditary Angioedema

     CT.gov ↗
139. NCT05143957

     sapablursen

     Phase 2 · active · small_molecule

     Phlebotomy Dependent Polycythemia Vera

     CT.gov ↗
140. NCT05185843

     Olezarsen

     Phase 3 · active · small_molecule

     Familial Chylomicronemia Syndrome

     CT.gov ↗
141. NCT05314439

     ION904

     Phase 2 · completed · small_molecule

     Hypertension

     CT.gov ↗
142. NCT05337878

     Placebo

     Phase 1 · completed · small_molecule

     Healthy Participants

     CT.gov ↗
143. NCT05355402

     Olezarsen

     Phase 3 · completed · small_molecule

     Hypertriglyceridemia

     CT.gov ↗
144. NCT05392114

     Donidalorsen

     Phase 3 · active · small_molecule

     Hereditary Angioedema

     CT.gov ↗
145. NCT05552326

     Olezarsen

     Phase 3 · completed · small_molecule

     Severe Hypertriglyceridemia

     CT.gov ↗
146. NCT05579860

     Olezarsen

     Phase 1 · completed · small_molecule

     Healthy Participants

     CT.gov ↗
147. NCT05610280

     Olezarsen

     Phase 3 · completed · small_molecule

     Hypertriglyceridemia

     CT.gov ↗
148. NCT05667493

     Eplontersen

     Phase 3 · active · small_molecule

     Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

     CT.gov ↗
149. NCT05681351

     Olezarsen

     Phase 3 · completed · small_molecule

     Severe Hypertriglyceridemia

     CT.gov ↗
150. NCT06150716

     ION356

     Phase 1 · active · small_molecule

     Pelizaeus-Merzbacher Disease

     CT.gov ↗
151. NCT06153966

     ION717

     Phase 2 · active · small_molecule

     Prion Disease

     CT.gov ↗
152. NCT06430385

     ION440

     Phase 2 · active · small_molecule

     Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

     CT.gov ↗
153. NCT06673069

     ION269

     Phase 1 · terminated · small_molecule

     Alzheimer Disease

     CT.gov ↗
154. NCT06914609

     ION582

     Phase 3 · active · small_molecule

     Angelman Syndrome

     CT.gov ↗
155. NCT07298447

     Donidalorsen

     Phase 3 · active · small_molecule

     Hereditary Angioedema (HAE)

     CT.gov ↗
156. NCT07531745

     ION337

     Phase 2 · active · small_molecule

     Dravet Syndrome

     CT.gov ↗
157. NCT07604974

     ION775

     Phase 2 · active · small_molecule

     Hypertriglyceridemia

     CT.gov ↗