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LEIDEN, the Netherlands & CAMBRIDGE, Mass.—ProQR Therapeutics N.V., which is working on RNA therapies for severe, rare genetic diseases, recently announced 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 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 Dr. David Rodman, 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 percent 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.”
Key initial findings include the following:
Based on the safety profile and early evidence of efficacy observed to date, ProQR plans to take advantage of the adaptive design, and expand the 100 µg cohort with additional subjects who are homozygous for exon 13 mutations. Dose escalation to 200 µg (“high dose”) is planned to occur in parallel. An interim analysis of dose- and gene copy-dependent safety and efficacy will be planned once all additional subjects have reached at least three months of treatment.