Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 40/100

Failure Rate

13.0%

3 terminated/withdrawn out of 23 trials

Success Rate

84.2%

-2.3% vs industry average

Late-Stage Pipeline

17%

4 trials in Phase 3/4

Results Transparency

63%

10 of 16 completed trials have results

Key Signals

2 recruiting10 with results

Enrollment Performance

Analytics

Phase 1

7(36.8%)

Phase 2

7(36.8%)

Phase 3

3(15.8%)

N/A

1(5.3%)

Phase 4

1(5.3%)

19Total

Phase 1(7)

Phase 2(7)

Phase 3(3)

N/A(1)

+1 more

Activity Timeline

Global Presence

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Clinical Trials (23)

Showing 20 of 23 trials

NCT04850105Recruiting

A Non-interventional Cohort Safety Study of Patients With hATTR-PN

Role: lead

NCT04270058Not Yet Recruiting

TEGSEDI Pregnancy Surveillance Program

Role: lead

NCT04306510Phase 4Terminated

A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN

Role: lead

NCT05577819Not ApplicableRecruiting

Prevalence and Prediction of ATTR in Ambulatory Patients With HFpEF

Role: collaborator

NCT03385239Phase 2Completed

Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Participants With Hypertriglyceridemia and Established Cardiovascular Disease (CVD)

Role: lead

NCT02211209Phase 3Completed

The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome

Role: collaborator

NCT02300233Phase 3Completed

The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia

Role: collaborator

NCT02910635Phase 1Completed

A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers

Role: collaborator

NCT02527343Phase 2Terminated

The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy

Role: lead

NCT03544060Unknown

Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Role: lead

NCT02658175Phase 3Completed

The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome

Role: lead

NCT03514420Phase 2Completed

Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)

Role: lead

NCT03371355Phase 2Completed

Study of ISIS 703802 in Participants With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease

Role: lead

NCT03360747Phase 2Completed

Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)

Role: lead

NCT02709850Phase 1Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia

Role: collaborator

NCT03070782Phase 2Completed

Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease

Role: lead

NCT04223908Completed

InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia

Role: collaborator

NCT03506854Phase 1Completed

Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy Patients

Role: lead

NCT02414594Phase 1Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)

Role: collaborator

NCT03455777Phase 2Withdrawn

Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

Role: lead