ProQR Announces Positive Interim Results from Phase I/II Clinical Trial of QR-110 in LCA10 Patients, and Plans to Start a Phase II/III Pivotal Trial

Posted on: Wednesday, September 5th, 2018



ProQR Therapeutics, developers of transformative medicines for the treatment of rare genetic diseases, today announced results from its Phase I/II trial of therapy QR-110 in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene.

LCA10 typically leads to childhood blindness and has no available treatment options. In the trial, QR-110 demonstrated rapid and sustained improvement in vision in patients with LCA10, as measured by visual acuity and the mobility course performance, as well as being well-tolerated with no serious adverse events recorded. Results from this interim analysis were presented earlier today at the Retinal Degeneration 2018 meeting in Killarney, Ireland.

“The results of this interim analysis are encouraging and met our decision criteria to stop enrolment in this study and progress to a pivotal Phase II/III trial,” said David Rodman, MD, executive vice president of research and development of ProQR. “We observed a clinically meaningful improvement in vision in the treated eye as measured by both mechanistic and potential registration endpoints. Consistent with predictions based on our patient derived optic-cup models, improvement in visual function was observed as early as two months after treatment and was maximal and stable by three months and thereafter. We are very grateful to the study participants, their caregivers, and the investigators and their staff for the support in the development of QR-110 in this trial.”

Thaddeus P. Dryja, M.D., professor of ophthalmology at Harvard Medical School and Massachusetts Eye and Ear and member of the National Academy of Sciences, commented, “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. 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.”

Based on the emerging findings from the Phase I/II trial, the Company agreed with the FDA to submit a protocol to progress to a pivotal Phase II/III trial. In light thereof, the originally planned interim analysis at six months treatment was accelerated to the point when eight patients had reached three months of treatment. Given comparable activity was observed in the first two dose levels, the trial did not escalate up to the high dose and trial enrolment has stopped, in anticipation of the start of a Phase II/III trial.