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 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