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ProQR Announces Fourth Quarter and Full Year 2020 Operating and Financial Results

Written by Customer Service on . Posted in Public Companies.

Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022; Brighten pediatric trial of sepofarsen to begin in 2021; additional data from Phase 1/2 InSight extension study to be reported in H2 2021QR-421a Stellar Phase 1/2 trial for Usher syndrome and non-syndromic retinitis pigmentosa interim analysis on track for late Q1/early Q2 2021Further pipeline progress anticipated with first clinical data in 2021 from QR-1123 program for autosomal dominant retinitis pigmentosa and QR-504a clinical study in Fuchs endothelial corneal dystrophy expected to start in H1 2021Cash runway into 2023LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 25, 2021 (GLOBE NEWSWIRE) — ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today reported its financial and operating results for the fourth quarter and full year ended December 31, 2020 and provided a business update.“During 2020, we made progress across our pipeline, capped off with completing enrollment in our pivotal trial of sepofarsen for LCA10 last month,” said Daniel A. de Boer, Founder and CEO of ProQR. “On the basis of this performance, we begin 2021 in a strong position to deliver on a number of important catalysts across our pipeline, including the next interim analysis for QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa, which is on track for late Q1/early Q2. With the findings from this IA, in addition to confirming the safety profile and biological activity, we expect to be able to clearly inform the design of the next study of QR-421a, including the dose to take forward, population to enrich for, and endpoint selection. In the year ahead, we also plan to share the first clinical data from QR-1123 for adRP, data from the Phase 1/2 extension study of sepofarsen, and to start dosing patients with QR-504a for FECD.”De Boer continued, “In 2020, we also advanced the business operationally on several fronts. We strengthened our Scientific Advisory Board with the addition of leading experts in IRDs and RNA therapies. Naveed Shams, MD, PhD, a proven leader with deep ophthalmology experience including multiple product approvals globally, joined us as Chief Scientific Officer. And, we extended our cash runway into 2023 through a strategic convertible debt facility, increasing our financial flexibility as we continue to advance our pipeline and build toward commercialization.”Business Operations and Program Updates

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