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# ProQR Trial in Eye Disease Fails
- URL: https://www.fdaweb.com/proqr-trial-in-eye-disease-fails/
- Published: 2022-02-14T12:00:00.000Z
- Updated: 2026-09-14T17:32:32.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5151155

ProQR Therapeutics says a pivotal Phase 2-3 trial (Illuminate) evaluating sepofarsen for treating genetic eye disease CEP290-mediated Leber congenital amaurosis 10 (LCA10) did not meet its primary endpoint or notable secondary endpoints. The company said there was no benefit observed in either treatment arm versus sham against the primary endpoint of best corrected visual acuity at Month 12\. LCA10 is described by ProQR as a difficult-to-treat retinal disease resulting in blindness, with no approved therapies. The company did find that sepofarsen was observed to be generally well-tolerated, adding that additional analyses will be conducted and presented at a future medical congress.