Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 55/100

Failure Rate

20.0%

2 terminated/withdrawn out of 10 trials

Success Rate

33.3%

-53.2% vs industry average

Late-Stage Pipeline

30%

3 trials in Phase 3/4

Results Transparency

100%

1 of 1 completed trials have results

Key Signals

4 recruiting1 with results

Enrollment Performance

Analytics

Phase 1

5(62.5%)

Phase 3

3(37.5%)

8Total

Phase 1(5)

Phase 3(3)

Activity Timeline

Global Presence

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Clinical Trials (10)

Showing 10 of 10 trials

NCT06672237Phase 3Recruiting

A Phase 3 Study of NTLA-2001 in ATTRv-PN

Role: lead

NCT06379789Phase 1Recruiting

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

Role: collaborator

NCT06128629Phase 3Recruiting

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Role: lead

NCT05120830Phase 1Active Not Recruiting

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

Role: lead

NCT04601051Phase 1Completed

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Role: lead

NCT06634420Phase 3Active Not Recruiting

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

Role: lead

NCT06262399Enrolling By Invitation

Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002

Role: lead

NCT05697861Recruiting

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Role: lead

NCT06622668Phase 1Withdrawn

NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease