- Thinly traded micro cap ProQR Therapeutics (NASDAQ:PRQR -8.5%) is down on almost double normal volume. Shares have retreated ~30% this week in apparent response to its timeline for sepofarsen (QR-110) for the treatment of a certain type of Leber’s congenital amaurosis 10 (LCA10), an inherited disorder that causes childhood blindness.
- On Monday, it announced that the FDA signed off on the design of a 30-subject Phase 2/3 clinical trial, ILLUMINATE, in LCA10 sufferers who harbor a mutation in the CEP290 gene called p.Cys998X. The primary endpoint will be the change from baseline in visual acuity compared to control at month 12.
- Patients in the control arm will have the option to cross over to active treatment. All participants will continue to receive sepofarsen for another 12 months. Topline data should be available by year-end 2020.
- Another factor that may be depressing shares is the longer-term safety profile of PQ-110-001, in Phase 1/2 development for the same patient population. 11 patients have been dosed thus far. Two developed cystoid macular edema which responded to standard-of-care treatment and six experienced lens opacities, three who had their corrective lenses replaced. The events were determined to be likely related to PQ-110-001. There were no discontinuations.