Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

0.0%

0 terminated/withdrawn out of 5 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

0 trials in Phase 3/4

Results Transparency

40%

2 of 5 completed trials have results

Key Signals

2 with results

Enrollment Performance

Analytics

Phase 2

2(66.7%)

Phase 1

1(33.3%)

3Total

Phase 2(2)

Phase 1(1)

Activity Timeline

Global Presence

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Clinical Trials (5)

Showing 5 of 5 trials

NCT02047461Phase 2Completed

Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP

Role: lead

NCT02629393Phase 2Completed

Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A

Role: lead

NCT01735188Completed

A Natural History Study of Molybdenum Cofactor and Isolated Sulfite Oxidase Deficiencies

Role: lead

NCT01640717Completed

A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)

Role: lead

NCT01894165Phase 1Completed

Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

Role: lead

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