ProQR Therapeutics N.V. (NASDAQ: PRQR) shares made a massive gain early on Wednesday after the firm announced positive interim results from its Phase 1/2 clinical trial of QR-110 in patients with Leber’s congenital amaurosis 10 (LCA10).
For some quick background: 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.
As for the results, roughly 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. Also, there was a substantive overall improvement in best corrected visual acuity, with 62.5% of subjects showing an improvement.
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.
David Rodman, M.D., executive vice president of research and development of ProQR, commented:
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. 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.
Shares of ProQR closed Tuesday at $7.95, with a consensus analyst price target of $16.33 and a 52-week range of $2.75 to $7.95. Following the announcement, the stock was up about 60% at $12.70 in early trading indications Wednesday.