QR-110, an RNA-based oligonucleotide treatment candidate for Leber’s congenital amaurosis 10, showed rapid and sustained improvement in vision in a phase 1/2 clinical trial, according to a press release from ProQR.
An interim analysis of the trial, in which 10 patients with Leber’s congenital amaurosis 10 (LCA10) were dosed with either 80 µg or 160 µg of QR-110 in one eye, found 60% of subjects had a clinically meaningful response in visual acuity and mobility course endpoints at 3 months of treatment, the release said.
An overall improvement in best corrected visual acuity was measured in a majority of patients, with a mean improvement of –0.67 logMAR after 3 months, and 62.5% of patients showed more than –0.3 logMAR improvement from baseline.
The mean improvement in navigating the mobility course was 2.6 levels, with 57.1% of subjects showing improvement of more than 2 levels, the release said.
LCA10, caused by a mutation in the CEP290 gene, typically leads to childhood blindness, and currently there are no available treatments.
“These results are the first human data to evaluate the clinical utility of RNA-based therapeutics in a human photoreceptor disease, particularly one with a severe unmet medical need,” Thaddeus P. Dryja, MD, professor of ophthalmology at Harvard Medical School and Massachusetts Eye and Ear, said in the release. “While a confirmatory trial will be required to establish the full potential of QR-110 in LCA10, these results suggest that therapeutic oligonucleotides have the potential to be broadly applicable to a wide spectrum of inherited retinal disorders.”
ProQR is planning to begin a phase 2/3 pivotal trial in the first half of 2019 based on the findings of this study.