Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 65/100

Failure Rate

20.0%

1 terminated/withdrawn out of 5 trials

Success Rate

80.0%

-6.5% vs industry average

Late-Stage Pipeline

20%

1 trials in Phase 3/4

Results Transparency

25%

1 of 4 completed trials have results

Key Signals

1 with results

Enrollment Performance

Analytics

Phase 2

3(60.0%)

Phase 1

1(20.0%)

Phase 3

1(20.0%)

5Total

Phase 2(3)

Phase 1(1)

Phase 3(1)

Activity Timeline

Global Presence

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Clinical Trials (5)

Showing 5 of 5 trials

NCT04226924Phase 2Withdrawn

Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose

Role: lead

NCT02328482Phase 3Completed

Continuation Protocol to Protocol BBCO-001

Role: lead

NCT02015481Phase 2Completed

Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

Role: lead

NCT02147886Phase 2Completed

Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease

Role: lead

NCT02725957Phase 1Completed

A Study to Establish Safety and Maximum Tolerated Dose of IV Trehalose in Healthy Subjects

Role: lead

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