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
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators in Q3
LEIDEN, Netherlands & CAMBRIDGE, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced a virtual presentation of data fro
ProQR will focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas. This comes following feedback from the European Medicines Agency (EMA) related to ProQR’s sepofarsen for Leber Congenital Amaurosis type 10 (LCA10).
ProQR to host a conference call to discuss the top-line results of the Phase 2/3 Illuminate clinical trial of investigational RNA therapy sepofarsen in CEP20-mediated LCA10.
Access based on positive interim analysis of clinical data as well as preclinical data to date PRIME designation provides a pathway for frequent and early interactions with the EMA aimed at supporting accelerated evaluation and approval
Divestment of sepofarsen and ultevursen completed – Théa to continue development of sepofarsen and ultevursen for patients with LCA10 and Usher syndrome
