Business
ProQR Announces Fourth Quarter and Full Year 2021 Operating and Financial Results
Additional analyses ongoing from Phase 2/3 Illuminate trial of sepofarsen in LCA10 with updates on any potential next steps with this program and overall
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"Additional analyses ongoing from Phase 2/3 Illuminate trial of sepofarsen in LCA10 with updates on any potential next steps with this program and overall strategy anticipated in Q2 2022Phase 2/3 Sirius and Celeste clinical trials of ultevursen (QR-421a) in Usher syndrome and retinitis pigmentosa underway; interim data readout from Helia extension study of ultevursen planned by year-end 2022Repeated dose study planned for QR-1123 for autosomal dominant retinitis pigmentosa and initial data from molecular proof-of concept biomarker study for QR-504a for Fuchs endothelial corneal dystrophy to be reported in 2022Cash runway into mid-to-late 2024 LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 24, 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 reported its financial and operating results for the fourth quarter and full year ended December 31, 2021, and provided a business update. “Following the disappointing readout of top-line results from the Illuminate trial of sepofarsen, we are conducting a comprehensive analysis of the data with the goal of providing an update on any potential next steps with this program and our overall strategy in Q2,” said Daniel A. de Boer, Founder and CEO of ProQR. “Based on the previous data we have reported, we remain very confident in our RNA approach and will continue to follow the data to determine how to best position ProQR for success.” De Boer continued, “In the coming year, we anticipate sharing data from our Helia extension trial of ultevursen, as well as the initial data from our QR-504a biomarker study. In parallel, our Sirius and Celeste trials of ultevursen are enrolling patients with USH2A-mediated Usher syndrome and retinitis pigmentosa. Beyond our clinical pipeline, we are excited by the potential of our next-generation Axiomer RNA editing technology. In addition to using this platform to develop therapies for our wholly-owned pipeline, we anticipate continuing to develop collaborations on a target-by-target basis, similar to the partnership with Lilly we announced last fall. ProQR is in a strong financial position with a cash runway into mid-to-late 2024 and we will continue to be diligent with our capital, as we work to advance RNA ther...