Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 60/100

Failure Rate

37.5%

3 terminated/withdrawn out of 8 trials

Success Rate

57.1%

-29.4% vs industry average

Late-Stage Pipeline

38%

3 trials in Phase 3/4

Results Transparency

125%

5 of 4 completed trials have results

Key Signals

5 with results

Enrollment Performance

Analytics

Phase 3

3(50.0%)

Phase 2

3(50.0%)

6Total

Phase 3(3)

Phase 2(3)

Activity Timeline

Global Presence

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Clinical Trials (8)

Showing 8 of 8 trials

NCT02612129Phase 2Completed

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

Role: lead

NCT04316637Unknown

Early Access Program With Arimoclomol in US Patients With NPC

Role: lead

NCT04049097Phase 3Terminated

Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

Role: lead

NCT03491462Phase 3Completed

Arimoclomol in Amyotropic Lateral Sclerosis

Role: lead

NCT03836716Phase 3Terminated

Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial

Role: lead

NCT02753530Phase 2Completed

Study of Arimoclomol in Inclusion Body Myositis (IBM)

Role: lead

NCT03746587Phase 2Terminated

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Role: lead

NCT02435030Completed

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

Role: lead

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