Key Highlights

Risk & Performance

Based on historical performance

Low Risk

Score: 25/100

Failure Rate

0.0%

0 terminated/withdrawn out of 5 trials

Late-Stage Pipeline

60%

3 trials in Phase 3/4

Results Transparency

0 of 4 completed trials have results

Key Signals

Phase 3

3(60.0%)

Phase 2/3

1(20.0%)

Phase 2

1(20.0%)

5Total

Phase 3(3)

Phase 2/3(1)

Phase 2(1)

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Showing 5 of 5 trials

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Role: lead

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Role: lead

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Role: lead

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Role: lead

Primary Progressive Multiple Sclerosis (PPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (PERSEUS)

Role: lead

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