Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

0.0%

0 terminated/withdrawn out of 4 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 4 completed trials have results

Key Signals

Enrollment Performance

Analytics

Phase 1
2(66.7%)
Phase 2
1(33.3%)
3Total
Phase 1(2)
Phase 2(1)

Activity Timeline

Global Presence

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Clinical Trials (4)

Showing 4 of 4 trials
NCT01417533Completed

A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases

Role: collaborator

NCT01597401Phase 1Completed

A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects With Stable Sickle Cell Disease

Role: collaborator

NCT01634750Phase 1Completed

Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Role: collaborator

NCT01419691Phase 2Completed

Phase I and II Study of Auranofin in Chronic Lymphocytic Leukemia (CLL)

Role: collaborator

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