ProQR has announced the completion of an in-depth strategic review designed to deliver on our commitment to advance RNA therapies for diseases with high unmet need. We are also sharing additional analyses of the sepofarsen Illuminate trial.
… ProQR Announces Publication in Nature Medicine for Sepofarsen in Leber Congenital Amaurosis 10 LEIDEN, … after a single treatment with antisense oligonucleotide sepofarsen: a case report ” describing durable vision …
Study did not meet primary endpoint nor notable secondary endpoints – no benefit observed in either treatment arm versus shamAdditional analyses to be conducted and presented at a future medical congressSepofarsen continues to be generally well tolerated
Agreement provides ProQR with initial payment of €12.5M and up to €135M in further payments, as well as potential additional earn outs based on commercial sales in the US and EU
Divestment of sepofarsen and ultevursen supports ProQR’s strategic focus on the Axiomer®&
Top-line results expected H1 2022
Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan.
… ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Extension Trial Data at the Association for … from the Company’s Phase 1/2 InSight extension trial of sepofarsen and live Q&A session at the Association for …
Call on July 20 will include a snapshot from the ongoing InSight open-label extension study of sepofarsen, including preliminary second eye treatment data
Top-line data now expected to be announced in Q1 2022Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
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
