Ionis Pharmaceuticals Inc. — Sponsor Intelligence | 23 EU Trials

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23 Registered trials

9 Recruiting

13 Completed trials

22 Countries active

11 Unique IMPs

100% Results reported

Phase III

Phase I

Phase I (FiH)

Phase III: 12 Phase II/III: 1 Phase II: 2 Phase I: 3 Phase I (FiH): 5

100%

13 of 13 completed trials have published summary results (EU CTR Article 37 — 12-month obligation).

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Top indications

Familial hypertriglyceridaemia 3 Hereditary angioedema 3 Prion agent test positive 1 Alexander disease 1 Pelizaeus-Merzbacher disease 1 Angelman´s syndrome 1 Angelman's syndrome 1 Multiple system atrophy 1 Amyotrophic lateral sclerosis 1

Top IMPs

ISIS 678354 SODIUM SALT 6 DONIDALORSEN 3 EPLONTERSEN 3 2'-O-(2-METHOXYETHYL) MODIFIED ANTISENSE OLIGONUCLEOTIDE TARGETING UBE3A ANTISENSE TRANSCRIPT RNA 2 2'-O-(2-METHOXYETHYL) MODIFIED ANTISENSE OLIGONUCLEOTIDE TARGETING PLP1 PRE-MRNA 1 2'-O-(2-METHOXYETHYL)-D-RIBOSE ANTISENSE OLIGONUCLEOTIDE TARGETING GLIAL FIBRILLARY ACIDIC PROTEIN MESSENGER RIBONUCLEIC ACID 1 ERGOCALCIFEROL BP, NICOTINAMIDE BP, RIBOFLAVIN BP, ALL-RAC-ALPHA-TOCOPHERYL ACETATE BP, CALCIUM PANTOTHENATE BP, THIAMINE NITRATE BP, VITAMIN A BP, ASCORBIC ACID BP, PYRIDOXINE HYDROCHLORIDE BP 1 ION337 1 ION440 1 SAPABLURSEN 1 ULEFNERSEN 1

All trials

23 total

Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome Pending

2025-523835-20-00 Phase I (FiH) 5 countries ION337
A Phase 1-2, Double-Blind, MAD Study of ION440 in MDS Recruiting

2023-507192-22-00 Phase I (FiH) 3 countries ION440
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome Recruiting

2024-519711-33-01 Phase III 4 countries 2'-O-(2-METHOXYETHYL) MODIFIED ANTISENSE OLIGONUCLEOTIDE TARGETING UBE3A ANTISEN…
Donidalorsen Treatment in Children with Hereditary Angioedema Recruiting

2025-523499-22-00 Phase III 3 countries DONIDALORSEN
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients with An… Recruiting

2024-512589-32-00 Phase I (FiH) 2 countries 2'-O-(2-METHOXYETHYL) MODIFIED ANTISENSE OLIGONUCLEOTIDE TARGETING UBE3A ANTISEN…
Phase 1b Study of ION356 to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics in Patients with Pelizaeus-Merzbacher Disease Recruiting

2022-502432-39-00 Phase I 3 countries 2'-O-(2-METHOXYETHYL) MODIFIED ANTISENSE OLIGONUCLEOTIDE TARGETING PLP1 PRE-MRNA
A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants with Severe Hypertriglyceridemia (SHTG) Recruiting

2022-501999-26-00 Phase III 19 countries ISIS 678354 SODIUM SALT
Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Recruiting

2024-511201-32-00 Phase III 7 countries EPLONTERSEN
An Open-Label Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS) Previously Tre… Recruiting

2023-508815-22-00 Phase III 1 country ISIS 678354 SODIUM SALT
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON) Recruiting

2024-512528-13-00 Phase I 4 countries
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patie… Ended

2023-503355-98-00 Phase I (FiH) 4 countries
An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) Ended

2022-502415-11-00 Phase III 12 countries EPLONTERSEN; ERGOCALCIFEROL BP, NICOTINAMIDE BP, RIBOFLAVIN BP, ALL-RAC-ALPHA-TO…
An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) Ended

2023-509201-77-00 Phase III 8 countries DONIDALORSEN
An Open-Label Extension Study of AKCEA-APOCIII-LRX Administered to Patients with Familial Chylomicronemia Syndrome (FCS) Ended

2023-509029-29-00 Phase III 7 countries ISIS 678354 SODIUM SALT
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With… Ended

2024-512163-31-00 Phase II/III 8 countries ULEFNERSEN
Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexande… Ended

2024-510603-11-00 Phase I (FiH) 2 countries 2'-O-(2-METHOXYETHYL)-D-RIBOSE ANTISENSE OLIGONUCLEOTIDE TARGETING GLIAL FIBRILL…
A global trial to determine the effectiveness and safety of ION-682884 in patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-… Ended

2024-514434-20-00 Phase III 12 countries EPLONTERSEN
Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) of ION269 in Participants With Down Syndrome (DS) at Risk for … Ended

2023-509257-31-00 Phase I 1 country
A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of sapablurs… Ended

2024-512482-14-00 Phase II 1 country SAPABLURSEN
A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hype… Ended

2022-503022-13-00 Phase III 13 countries ISIS 678354 SODIUM SALT
A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia Ended

2022-501420-20-00 Phase III 15 countries ISIS 678354 SODIUM SALT
A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia. Ended

2024-510696-38-00 Phase III 15 countries ISIS 678354 SODIUM SALT
ISIS 721744-CS3: An Open-Label Extension Study of ISIS 721744 in Patients with Hereditary Angioedema Ended

2024-517249-15-00 Phase II 1 country DONIDALORSEN

Showing 1–23 of 23 trials

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