ProQR Announces First Patient Dosed in Phase 1/2 Aurora Trial of QR-1123 for Autosomal Dominant Retinitis Pigmentosa

ProQR Therapeutics announced the first patient dosed in the phase 1/2 Aurora clinical trial of QR-1123 in patients with autosomal dominant retinitis pigmentosa (adRP). Initial data from the study are expected in 2021.

“From previous clinical trials we have seen that RNA therapies can be a promising approach for patients with inherited retinal diseases and there is a strong need for novel approaches in diseases that currently have no treatments,” David G. Birch, PhD, Principal Investigator of Aurora and Scientific Director of the Retina Foundation of the Southwest in Dallas, said in a company news release. “QR-1123 has shown encouraging results in preclinical models and we hope to see that translated to the clinic in this first-in-human trial in patients with adRP.”

“QR-1123 aims to block expression of the toxic mutated rhodopsin protein in the retina thereby targeting the underlying cause of the vision loss associated with adRP due to the P23H mutation. We are excited to have started a clinical trial for the third RNA therapy in our inherited retinal disease pipeline,” David Rodman, MD, Executive Vice President of Research & Development of ProQR, said in the news release.

Aurora, or PQ-1123-001, is a first-in-human study that will initially include up to 35 adults with adRP due to the P23H mutation in the rhodopsin (RHO) gene. The trial will include single-dose escalation (open label) groups and multiple-dose escalation (double-masked) groups in which intravitreal injections of QR-1123 or sham procedures will be given in one eye. The objectives of the trial include evaluation of safety and tolerability. Efficacy as measured by improvement of visual function and retinal structure will be assessed through ophthalmic endpoints such as visual acuity, visual field and optical coherence tomography. The trial will be conducted at expert sites in North America.