Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 60/100

Failure Rate

28.6%

2 terminated/withdrawn out of 7 trials

Success Rate

50.0%

-36.6% vs industry average

Late-Stage Pipeline

43%

3 trials in Phase 3/4

Results Transparency

100%

2 of 2 completed trials have results

Key Signals

2 recruiting2 with results

Enrollment Performance

Analytics

Phase 3

2(50.0%)

Phase 4

1(25.0%)

Phase 2

1(25.0%)

4Total

Phase 3(2)

Phase 4(1)

Phase 2(1)

Activity Timeline

Global Presence

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Clinical Trials (7)

Showing 7 of 7 trials

NCT06502990Phase 3Terminated

Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy

Role: lead

NCT02135705Completed

LOWER: Lomitapide Observational Worldwide Evaluation Registry

Role: lead

NCT06423573Recruiting

A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez

Role: lead

NCT04681170Phase 3Completed

Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)

Role: lead

NCT06484868Phase 4Recruiting

Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

Role: lead

NCT05351164Phase 2Active Not Recruiting

Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)

Role: collaborator

NCT02399839Terminated

Global Lomitapide Pregnancy Exposure Registry

Role: lead

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