Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Low Risk

Score: 15/100

Failure Rate

9.4%

3 terminated/withdrawn out of 32 trials

Success Rate

88.9%

+2.4% vs industry average

Late-Stage Pipeline

34%

11 trials in Phase 3/4

Results Transparency

58%

14 of 24 completed trials have results

Key Signals

14 with results

Enrollment Performance

Analytics

Phase 3

9(37.5%)

Phase 2

8(33.3%)

Phase 1

5(20.8%)

Phase 4

2(8.3%)

24Total

Phase 3(9)

Phase 2(8)

Phase 1(5)

Phase 4(2)

Activity Timeline

Global Presence

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Clinical Trials (32)

Showing 20 of 32 trials

NCT06713135Phase 4Active Not Recruiting

A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy

Role: lead

NCT03863119Unknown

Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Role: lead

NCT06689527Phase 1Completed

Evaluation of Vamorolone CYP3A4 Induction on Midazolam (a Sensitive CYP 3A4 Substrate) Pharmacokinetics

Role: lead

NCT05185622Phase 2Completed

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Role: lead

NCT05166109Phase 2Completed

A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)

Role: collaborator

NCT06649409Phase 1Completed

Evaluation of Vamorolone Mineralocorticoid Receptor Antagonism in Healthy Subjects

Role: lead

NCT00537680Phase 3Completed

Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia

Role: lead

NCT00697073Phase 3Completed

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Role: lead

NCT03433807Unknown

Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)

Role: lead

NCT04381091Unknown

Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the LEROS Study

Role: lead

NCT02774005Phase 4Completed

Study to Assess the Efficacy and Safety of Raxone in LHON Patients

Role: lead

NCT02814019Phase 3Terminated

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

Role: lead

NCT03603288Phase 3Terminated

Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

Role: lead

NCT03748199Phase 1Completed

Clinical Study to Investigate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of POL6014 in Patients With CF

Role: lead

NCT01805024Phase 1Completed

Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)

Role: lead

NCT02771379Completed

Post Authorisation Safety Study With Raxone in LHON Patients

Role: lead

NCT02796274Completed

Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)

Role: lead

NCT03680365Completed

Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families

Role: collaborator

NCT00993967Phase 3Completed

Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

Role: lead

NCT02887443Phase 1Completed

A Study to Assess the Potential for Pre-systemic Inhibition of CYP3A by Idebenone Using Midazolam as a Substrate

Role: lead