Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Low Risk

Score: 5/100

Failure Rate

0.0%

0 terminated/withdrawn out of 2 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

100%

2 trials in Phase 3/4

Results Transparency

100%

1 of 1 completed trials have results

Key Signals

1 with results

Enrollment Performance

Analytics

Phase 3
2(100.0%)
2Total
Phase 3(2)

Activity Timeline

Global Presence

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Clinical Trials (2)

Showing 2 of 2 trials
NCT06582524Phase 3Completed

Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

Role: lead

NCT05249556Phase 3Not Yet Recruiting

Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old

Role: lead

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