ProQR Reports Results from Phase 2-3 Illuminate Trial of Sepofarsen

Feb 11, 2022 By MarketDepth

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ProQR Therapeutics (NASDAQ: PRQR) has reported its Phase 2/3 Illuminate trial of sepofarsen to treat CEP290-mediated Leber congenital amaurosis 10 did not meet its primary endpoint of Best Corrected Visual Acuity 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 . “We will continue to work with ProQR to understand the data as they work to advance therapies for inherited retinal diseases. This was not the outcome we had hoped for and we share in the disappointment many are feeling in the community,” said Benjamin R. Yerxa, Chief Executive Officer at the Foundation Fighting Blindness. “We will continue to work alongside ProQR to learn more from the ongoing analyses and as they work to advance RNA therapies to potentially help children, adults, and families who are affected by rare genetic eye diseases.”

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