Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 35/100

Failure Rate

7.7%

12 terminated/withdrawn out of 155 trials

Success Rate

90.6%

+4.1% vs industry average

Late-Stage Pipeline

20%

31 trials in Phase 3/4

Results Transparency

37%

43 of 116 completed trials have results

Key Signals

11 recruiting43 with results8 terminated4 withdrawn

Enrollment Performance

Analytics

Phase 2

63(45.0%)

Phase 1

44(31.4%)

Phase 3

31(22.1%)

N/A

2(1.4%)

140Total

Phase 2(63)

Phase 1(44)

Phase 3(31)

N/A(2)

Activity Timeline

Global Presence

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Clinical Trials (155)

Showing 20 of 155 trials

NCT04307381Phase 2Completed

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Role: lead

NCT07531745Phase 1Recruiting

ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome

Role: lead

NCT06914609Phase 3Recruiting

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

Role: lead

NCT07298447Phase 3Recruiting

Donidalorsen Treatment in Children With Hereditary Angioedema

Role: lead

NCT04165486Phase 1Recruiting

Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)

Role: lead

NCT06153966Phase 1Recruiting

PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717

Role: lead

NCT05355402Phase 2Completed

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia

Role: lead

NCT05659901Recruiting

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

Role: lead

NCT05667493Phase 3Enrolling By Invitation

An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

Role: lead

NCT06014541Terminated

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Role: lead

NCT06430385Phase 1Recruiting

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

Role: lead

NCT07487389Unknown

Zilganersen Expanded Access Program for Individuals With Alexander Disease

Role: lead

NCT05311488Completed

Early Detection of Neuropathy in ATTRv

Role: collaborator

NCT06673069Phase 1Terminated

Hero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD)

Role: lead

NCT04768972Phase 3Active Not Recruiting

FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

Role: lead

NCT05079919Phase 3Completed

A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia

Role: lead

NCT05610280Phase 3Completed

A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia

Role: lead

NCT05143957Phase 2Active Not Recruiting

A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera

Role: lead

NCT02714764Recruiting

Evaluation of Outcome Metrics in Alexander Disease

Role: collaborator

NCT05130450Phase 3Active Not Recruiting

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

Role: lead