ProQR’s QR-110 Data for Leber’s Congenital Amaurosis 10 published in Nature

ProQR, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, announced the publication of a peer-reviewed manuscript describing the previously announced interim results of a clinical trial of QR-110 for the treatment of Leber’s congenital amaurosis 10 (LCA10) in the journal Nature Medicine.

“It was enormously gratifying to see robust improvements in visual acuity and significant augmentations in the patient’s ability to detect lights, and impressive to observe these effects within the first three months following a single injection,” said Professor Artur V. Cideciyan, Ph.D., who was one of the co-investigators at the Scheie Eye Institute of the University of Pennsylvania. “LCA10 is a severe form of childhood blindness and this is a major step forward in the treatment of these previously incurable conditions,” said Professor Samuel G. Jacobson, M.D., Ph.D, who is the ophthalmologist caring for four of the patients enrolled in the study and is also a co-investigator at the Scheie Eye Institute.

Published results detail a planned interim analysis of the ongoing PQ-110-001 first-in-human clinical study, evaluating QR-110 in patients with LCA10. The publication focused on a landmark analysis of eight subjects who reached three months of single dose treatment experience. Results demonstrate a substantive overall improvement in best corrected visual acuity (BCVA) with a mean improvement of -0.67 LogMAR (SEM 0.32) in the treated eye versus 0.02 LogMAR (SEM 0.05) in the contralateral (control) eye (p=0.011). The majority of patients showed an increase of at least -0.3 LogMAR in the treated eye, which is generally considered clinically meaningful, and equivalent to 15 letters, or three lines of improvement for individuals able to read a standard eye chart. Visual acuity improvements were also associated with concordant improvements in full-field stimulus thresholds to both blue and red light, improved mobility course navigation and ocular instability measurement.

LCA10 is a severe inherited retinal dystrophy associated with mutations in the CEP290 gene. QR-110 is an RNA-based drug candidate that has the potential to restore sight or slow down the process of vision loss in patients with LCA10 by correcting the most common mutation causing LCA10, p.Cys998X.

The ongoing Phase 1/2 PQ-110-001 study of QR-110 will be completed and in parallel a Phase 2/3 “ILLUMINATE” study is expected to be initiated in the first half of 2019.

Source: ProQR