Additional analyses ongoing from Phase 2/3 Illuminate trial of sepofarsen in LCA10 with updates on any potential next steps with this program and overall strategy anticipated in Q2 2022
Phase 2/3 Sirius and Celeste clinical trials of ultevur
Top-line data from pivotal Phase 2/3 Illuminate trial of sepofarsen for CEP290-mediated LCA10 anticipated late Q1/early Q2 2022
Five-target collaboration with Lilly highlights significant potential of ProQR’s Axiomer® RNA base-editing pla
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022
Positive data reported from the Phase 1/2 clinical trial of QR-421a for the treatment of Usher syndrome and non-syndromic retini
Illuminate Phase 2/3 trial of sepofarsen expected to complete enrollment in Q1 2021; additional data from Phase 1/2 InSight extension study to be reported in H2 2021
Enrollment completed for QR-421a dosing cohorts and data from next Phase 1/2 interim
In line with corporate strategy update in April, ProQR to focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas; update on initial pipeline targets expected in late 2022/early 2023
Additi
Reported rapid, significant and durable improvements in vision at twelve months
Concordant improvement in key secondary outcome measures
The target registration dose of sepofarsen was well-tolerated with a favorable benefit/risk profile
Strengthens confidence