Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 35/100

Failure Rate

0.0%

0 terminated/withdrawn out of 9 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

22%

2 trials in Phase 3/4

Results Transparency

0 of 8 completed trials have results

Key Signals

Enrollment Performance

Analytics

Phase 2

4(57.1%)

Phase 3

2(28.6%)

Phase 1

1(14.3%)

7Total

Phase 2(4)

Phase 3(2)

Phase 1(1)

Activity Timeline

Global Presence

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Clinical Trials (9)

Showing 9 of 9 trials

NCT01303146Phase 2Completed

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

Role: collaborator

NCT02478840Phase 3Completed

Evaluation of Long-term Efficacy of Treatment With Lamazym

Role: lead

NCT01268358Phase 1Completed

Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis

Role: lead

NCT00498420Completed

The Natural History of Alpha-Mannosidosis

Role: lead

NCT01681953Phase 3Completed

A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis

Role: lead

NCT00418795Phase 2Completed

Porphozym in the Treatment of Acute Attacks in AIP

Role: lead

NCT00983879Completed

The Natural History of Infantile Globoid Cell Leukodystrophy

Role: lead

NCT01681940Phase 2Completed

Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Role: lead

NCT01285700Phase 2Unknown

Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis

Role: lead

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