Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 80/100

Failure Rate

50.0%

2 terminated/withdrawn out of 4 trials

Success Rate

50.0%

-36.5% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

150%

3 of 2 completed trials have results

Key Signals

3 with results

Enrollment Performance

Analytics

Phase 1
3(75.0%)
Phase 2
1(25.0%)
4Total
Phase 1(3)
Phase 2(1)

Activity Timeline

Global Presence

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Clinical Trials (4)

Showing 4 of 4 trials
NCT03333590Phase 1Completed

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

Role: lead

NCT04179409Phase 2Completed

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

Role: lead

NCT03777319Phase 1Terminated

Spironolactone Versus Prednisolone in DMD

Role: lead

NCT02704325Phase 1Withdrawn

Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2

Role: lead

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