Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 80/100

Failure Rate

44.4%

4 terminated/withdrawn out of 9 trials

Success Rate

55.6%

-31.0% vs industry average

Late-Stage Pipeline

11%

1 trials in Phase 3/4

Results Transparency

100%

5 of 5 completed trials have results

Key Signals

5 with results

Enrollment Performance

Analytics

Phase 1

4(57.1%)

Phase 2

2(28.6%)

Phase 3

1(14.3%)

7Total

Phase 1(4)

Phase 2(2)

Phase 3(1)

Activity Timeline

Global Presence

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Clinical Trials (9)

Showing 9 of 9 trials

NCT05072912Terminated

Screening Study to Identify Patients With Complement Factor I Deficiencies

Role: lead

NCT05095623Terminated

A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease

Role: lead

NCT04548791Phase 1Terminated

Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders

Role: lead

NCT04489537Phase 3Terminated

Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B

Role: lead

NCT03407651Phase 2Completed

Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B

Role: lead

NCT04072237Phase 1Completed

Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia

Role: lead

NCT03995784Phase 2Completed

Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B

Role: lead

NCT03186677Phase 1Completed

Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients

Role: collaborator

NCT01439971Phase 1Completed

Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia

Role: lead

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