BioCentury
ARTICLE | Clinical News

ProQR planning pivotal trial of vision loss candidate

September 7, 2018 6:14 PM UTC

ProQR Therapeutics N.V. (NASDAQ:PRQR) said it plans to start the pivotal Phase II/III ILLUMINATE trial of QR-110 next half to treat Leber congenital amaurosis type 10 (LCA10) caused by the p.Cys998X mutation in the centrosomal protein 290kDa (CEP290) gene. The decision was based on newly released interim data from the Phase I/II PQ-110-001 trial showing that the candidate led to rapid and sustained visual improvements at three months.

In the U.S. and Belgian PQ-110-001 study, QR-110 improved mean best corrected visual acuity (BCVA) at three months by logMAR 0.67 vs. a worsening of logMAR 0.02 for the untreated eye as measured by the Berkeley Rudimentary Vision Test (BRVT) and the Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart. The candidate also improved functional visual performance per a series of mobility courses of increasing difficulty and multiple light intensities by a mean of 2.6 levels vs. 1.36 levels for the untreated eye...

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