Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 50/100

Failure Rate

14.3%

1 terminated/withdrawn out of 7 trials

Success Rate

75.0%

-11.5% vs industry average

Late-Stage Pipeline

0 trials in Phase 3/4

Results Transparency

67%

2 of 3 completed trials have results

Key Signals

2 with results

Enrollment Performance

Analytics

Phase 1

5(71.4%)

Phase 2

2(28.6%)

7Total

Phase 1(5)

Phase 2(2)

Activity Timeline

Global Presence

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Clinical Trials (7)

Showing 7 of 7 trials

NCT05052554Phase 1Withdrawn

Study With QR-504a to Evaluate Safety, Tolerability & Corneal Endothelium Molecular Biomarker(s) in Subjects With FECD3

Role: lead

NCT04123626Phase 1Active Not Recruiting

A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene

Role: lead

NCT03780257Phase 1Completed

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

Role: lead

NCT04855045Phase 2Unknown

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

Role: lead

NCT03913143Phase 2Active Not Recruiting

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Role: lead

NCT02564354Phase 1Completed

Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

Role: lead

NCT02532764Phase 1Completed

Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

Role: lead

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