ProQR Shares Crater After Disappointing Sepofarsen Data In Inherited Vision Loss Disorder

ProQR Therapeutics N.V. PRQR shares are plunging after disappointing data from its pivotal Phase 2/3 Illuminate trial of sepofarsen for CEP290-mediated Leber congenital amaurosis 10 (LCA10).

• The trial failed to meet its primary endpoint of Best Corrected Visual Acuity (BCVA) at Month 12.
• Illuminate trial enrolled 36 participants aged eight years or older with genetically confirmed LCA10. 
• Participants were randomized to three equal groups - sepofarsen (160 μg/80 μg loading dose/maintenance doses), a low dose of sepofarsen for masking (80 μg/40 μg loading dose/maintenance doses), or sham procedure.
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• At Month 12, the mean change from baseline in BCVA in the 160/80 μg dose group was -0.11 logMAR (p=0.96), in the 80/40 μg group -0.13 logMAR (p=0.97), and the sham group -0.12 logMAR. 
• For secondary endpoints full-field stimulus test and mobility, there was also no difference between the treated and sham groups in the top-line analysis.
• Sepofarsen was observed to be generally well-tolerated. 
• Price Action: PRQR shares are down 71.86% at $1.60 during the premarket session on the last check Friday.