ProQR Therapeutics (NASDAQ:PRQR) announces positive topline results from a Phase 1/2 clinical trial
evaluating sepofarsen (QR-110) in patients with Leber congenital
amaurosis type 10 (LCA10) due to a certain mutation in the CEP290 gene.
The target dose (80 µg with a 160 µg loading dose)
was associated with a clinically meaningful and statistically
significant improvement in vision with a favorable benefit/risk profile.
Durable improvements in vision were observed after six months of
treatment.
A Phase 2/3 trial is in process.
Management will host a conference call this morning at 8:00 ET to discuss the results.
Shares are up 7% premarket on light volume.
https://seekingalpha.com/news/3504920-proqrs-sepofarsen-shows-positive-action-phase-1-2-study-lca10
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