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ProQR Announces Top-Line Results from Phase 2/3 Illuminate Trial of Sepofarsen in CEP290-mediated LCA10
Study did not meet primary endpoint nor notable secondary endpoints – no benefit observed in either treatment arm versus shamAdditional analyses to be
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"Study did not meet primary endpoint nor notable secondary endpoints – no benefit observed in either treatment arm versus shamAdditional analyses to be conducted and presented at a future medical congressSepofarsen continues to be generally well toleratedStrong cash position provides runway into mid- to late-2024ProQR Management to host conference call today at 8:15am EST LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 11, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced its pivotal Phase 2/3 Illuminate trial of sepofarsen for the treatment of CEP290-mediated Leber congenital amaurosis 10 (LCA10) did not meet its primary endpoint of Best Corrected Visual Acuity (BCVA) at Month 12. “Given the results observed in earlier studies of sepofarsen, the Illuminate trial results are unexpected and disappointing, especially for people living with LCA10,” said Daniel A. de Boer, Founder and CEO of ProQR Therapeutics. “ProQR was founded with the goal of developing RNA therapies for patients with high unmet medical need, and we will continue to advance our robust pipeline of therapies for genetic eye disease. We are deeply grateful to all of the participants, their supporters, and investigators who participated in the Illuminate study.” “LCA10 is a devastating, very difficult-to-treat retinal disease resulting in blindness, for which there are no therapies,” said Dr. Bart Leroy, Head of the Ophthalmology Department at Ghent University Hospital and Professor of Ophthalmology and Ophthalmic Genetics at Ghent University, Ghent, Belgium and Attending Physician for Ophthalmic Genetics and Retinal Degenerations at The Children's Hospital of Philadelphia. “We will continue to work with ProQR to understand the data as they work to advance therapies for inherited retinal diseases.” Illuminate, a randomized, sham-controlled trial, enrolled 36 participants aged eight years or older with genetically confirmed LCA10 due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene, at 14 study sites in 9 countries. Participants were randomized to three equal groups (1:1:1) of the target registration dose sepofarsen (160 μg/80 μg loading dose/maintenance doses), a low dose of sepofarsen for maskin...