ProQR Therapeutics N.V. (NASDAQ: PRQR) shares rose Wednesday after the company disclosed positive interim results from Phase 1/2 clinical trial of QR-110 in LCA10 patients and plans to start a Phase 2/3 pivotal trial.
The Dutch-based company, with American headquarters in Cambridge, Mass., points out that 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.
"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," said David Rodman, M.D., 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."
The company added that approximately 60% of subjects showed a clinically meaningful response in visual acuity and mobility course endpoints at three months of treatment and there was general concordance across the endpoints. Efficacy signals were observed within two months with maximal benefits seen within two to three months post treatment initiation.
Shares in the company ballooned $44.40, or 55.4%, to $12.35
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