PRQR - ProQR says trial for sepofarsen in Leber congenital amaurosis did not meet main goal

ProQR Therapeutics (NASDAQ:PRQR) announced on Friday that the company’s pivotal Phase 2/3 Illuminate trial for RNA therapy sepofarsen in EP290-mediated Leber congenital amaurosis 10 (LCA10) did not meet the primary endpoint of Best Corrected Visual Acuity (BCVA). ProQR (PRQR) is currently on hold for trading pending news. According to the topline analysis at 12 months, the mean BCVA change in the 160/80 ?g dose group and 80/40 ?g group dose groups stood at -0.11 logMAR (p=0.96) and -0.13 logMAR (p=0.97), compared to -0.12 logMAR in the sham group. Therefore, no benefit was found in either treatment arm vs. sham. There was also no difference between the treated and sham groups in terms of secondary endpoints full-field stimulus test (FST) and mobility. However, sepofarsen was generally well tolerated during the study with findings consistent with the Phase 1/2 trial. Announcing an update on its liquidity, ProQR (PRQR) said estimated a cash

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ProQR says trial for sepofarsen in Leber congenital amaurosis did not meet main goal