Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 100/100

Failure Rate

40.0%

2 terminated/withdrawn out of 5 trials

Success Rate

33.3%

-53.2% vs industry average

Late-Stage Pipeline

0%

0 trials in Phase 3/4

Results Transparency

0%

0 of 1 completed trials have results

Key Signals

2 recruiting

Enrollment Performance

Analytics

Phase 2
4(80.0%)
Phase 1
1(20.0%)
5Total
Phase 2(4)
Phase 1(1)

Activity Timeline

Global Presence

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Clinical Trials (5)

Showing 5 of 5 trials
NCT06667453Phase 2Recruiting

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

Role: lead

NCT07220603Phase 2Recruiting

An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

Role: lead

NCT06204809Phase 1Completed

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

Role: lead

NCT06079736Phase 2Terminated

A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment

Role: lead

NCT06833931Phase 2Withdrawn

A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Role: lead

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