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ProQR Announces Positive Findings From an Interim Analysis in the Phase 1/2 trial of QR-421a for Usher Syndrome and Provides Business Update
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by translational models, further validating the platformCOVID-19 pandemic expected to impact timelines for the pipelineProQR anticipates its cash runway will fund operations into H2 2022 LEIDEN, Netherlands & CAMBRIDGE, Mass., March 31, 2020 (GLOBE NEWSWIRE) -- March 31, 2020 -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announces positive findings from a planned three-month interim analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. The Company is also providing an update on business operations in relation to the COVID-19 pandemic. Management will host a conference call today at 8:15 am ET.\n “The goal of the interim analysis of this 24 month Stellar trial of QR-421a was to assess safety and early signs of efficacy for the purpose of informing next steps in development and future trial strategy,” said David Rodman, M.D., Executive Vice President of Research and Development of ProQR. “We are pleased with the current safety profile and are very encouraged by early signals of target engagement and clinical activity supported by concordant benefit observed across multiple outcome measures for 25% of QR-421a-treated patients thus far in this trial. The findings support continuing the trial as planned, with both cohort expansion and dose escalation in order to identify a potential development path to registration. Importantly, these data represent the second program from our ophthalmology pipeline that is supported by preclinical predictions from human retinal organoids, providing further validation of our translational approach and platform technology.” Phase 1/2 Three-Month Interim Analysis The interim analysis (IA) is based on nine and three month data from the first and second dose cohorts, respectively, of the Stellar Phase 1/2 clinical trial of QR-421a, an investigational RNA therapy. The Stellar trial is a ra...