Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 80/100

Failure Rate

50.0%

4 terminated/withdrawn out of 8 trials

Success Rate

20.0%

-66.6% vs industry average

Late-Stage Pipeline

13%

1 trials in Phase 3/4

Results Transparency

500%

5 of 1 completed trials have results

Key Signals

1 recruiting5 with results

Enrollment Performance

Analytics

Phase 2

5(62.5%)

Phase 1

2(25.0%)

Phase 3

1(12.5%)

8Total

Phase 2(5)

Phase 1(2)

Phase 3(1)

Activity Timeline

Global Presence

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Clinical Trials (8)

Showing 8 of 8 trials

NCT07796646Phase 2Not Yet Recruiting

Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Role: collaborator

NCT06627179Phase 2Active Not Recruiting

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

Role: collaborator

NCT06891443Phase 3Recruiting

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Role: collaborator

NCT03140969Phase 1Completed

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Role: collaborator

NCT03913130Phase 1Terminated

Extension Study to Study PQ-110-001 (NCT03140969)

Role: collaborator

NCT05085964Phase 2Terminated

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa

Role: collaborator

NCT05176717Phase 2Terminated

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

Role: collaborator

NCT05158296Phase 2Terminated

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Role: collaborator

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