ProQR: QR-110 Phase 1/2 Clinical Trial Positive; To Progress To Phase 2/3 Trial

(RTTNews) - ProQR Therapeutics N.V. (PRQR), which creates transformative RNA medicines for the treatment of severe genetic rare diseases, announced Wednesday positive results from a planned interim analysis of its Phase 1/2 trial of QR-110 in patients with Leber's congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene. The company said QR-110 was well-tolerated with no serious adverse events.

Based on the emerging findings from the Phase 1/2 trial, the Company agreed with the FDA to submit a protocol to progress to a pivotal Phase 2/3 trial, which is expected to start in the first half of 2019.

In pre-market activity on Nasdaq, ProQR shares were trading 3.8 percent higher at $8.25.

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 at the Retinal Degeneration 2018 meeting in Killarney, Ireland.

David Rodman, M.D., executive vice president of research and development of ProQR, said, "The results of this interim analysis are encouraging and met our decision criteria to stop enrollment in this study and progress to a pivotal Phase 2/3 trial. ...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."

The company said 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 enrollment has stopped, in anticipation of the start of a Phase 2/3 trial.