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0 terminated/withdrawn out of 2 trials

Success Rate

100.0%

+13.4% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 2 completed trials have results

Key Signals

Enrollment Performance

Analytics

Phase 2
1(100.0%)
1Total
Phase 2(1)

Activity Timeline

Global Presence

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Clinical Trials (2)

Showing 2 of 2 trials
NCT05758922Phase 2Completed

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Role: lead

NCT05109793Completed

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

Role: lead

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