Business
ProQR Announces Recent Progress and Financial Results for the Third Quarter of 2019
Encouraging clinical data reported from Phase 1/2 trial of sepofarsen for LCA10Initial clinical data from Phase 1/2 trial of QR-421a for Usher syndrome type 2
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"Encouraging clinical data reported from Phase 1/2 trial of sepofarsen for LCA10Initial clinical data from Phase 1/2 trial of QR-421a for Usher syndrome type 2 on track for Q1 2020QR-1123 Investigational New Drug application active for autosomal dominant retinitis pigmentosa€49.3 million net proceeds from public offering extends cash runway into 2022 LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 06, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced results for the third quarter of 2019.\n “During the third quarter we made great progress towards our mission to bring novel RNA therapies to patients and we recently strengthened our capital position,” said Daniel A. de Boer, chief executive officer of ProQR. “In October we announced that LCA10 patients experienced a significant improvement in vision after treatment with sepofarsen in a Phase 1/2 trial. These results strengthen our confidence in the design of the ongoing pivotal Illuminate Phase 2/3 trial and our broader inherited retinal diseases pipeline. The Phase 1/2 trial of QR-421a in Usher syndrome is on track to deliver first clinical data in Q1 2020 and we are about to dose a first patient in a Phase 1/2 trial of QR-1123 for adRP. We strengthened our financial position with a public offering in October that will allow us to fund operations into 2022, well beyond the expected top-line readout of Illuminate.” Corporate Highlights and Business Update Sepofarsen (QR-110) for Leber’s congenital amaurosis 10 (LCA10) Presented positive top-line results from the Phase 1/2 clinical trial. In the trial, sepofarsen was observed to be well tolerated with rapid, significant and durable improvements in vision observed at month twelve. The target registration dose for the ongoing Phase 2/3 Illuminate trial showed a favorable benefit/risk profile in the Phase 1/2 trial.The top-line results from the Phase 1/2 trial support confidence in the design of the ongoing Phase 2/3 Illuminate trial that could be the sole registration trial for the program. Top-line data from Illuminate are expected during the first half of 2021.Received Rare Pediatric Disease designation from the Food and Drug Administration (FDA) for the treatment of...