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ProQR Announces Last Patient Has Completed 12 Month Visit in Phase 2/3 Illuminate Trial of Sepofarsen
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
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"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 LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan. 04, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases today announced that the last patient completed their last visit (Month 12) in the Phase 2/3 Illuminate trial of sepofarsen for CEP290-mediated Leber Congenital Amaurosis 10 (LCA10) due to the p.Cys998X mutation, also known as c.2991+1655A>G. “The last patient having completed their 12 Month visit is an important milestone toward the top-line results from the Phase 2/3 Illuminate trial of our lead program sepofarsen for LCA10,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “Based on this event, we now anticipate sharing the top-line results in the first quarter of 2022. We are grateful to those who have supported the execution of this trial, including our investigators, patients, and caregivers, and look forward to sharing the top-line results this quarter.” The Illuminate trial completed enrollment in January 2021 following randomization of 36 patients aged 8 years or older to receive either sepofarsen at the target registration dose, a low dose, or sham treatment. The primary endpoint for Illuminate is mean change from baseline in best-corrected visual acuity (BCVA) at Month 12. The Illuminate trial was initiated based on data from a Phase 1/2 study, which indicated that at Month 12, patients treated with sepofarsen had an improvement in visual acuity, as measured by BCVA. In a subset of patients (n=6) who were treated at the target registration dose, the mean change from baseline for BCVA at Month 12 was -0.93 logMAR, equivalent to approximately 9 lines improvement (or 45 letters) on the ETDRS chart. In the Phase 1/2 study, concordant improvements in measures of full-field stimulus testing (FST) and mobility were also observed, which are secondary endpoints in the Illuminate trial. About Leber Congenital Amaurosis 10 (LCA10) Leber congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children. It consists of a group of diseases of which...