Business
ProQR Announces First Quarter 2020 Operating and Financial Results
Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing with dose expansion and escalation planned;Updated data from the Phase 1/2 InSight extension study of sepofarsen, including data from contralateral eye treatment, on track to be reported in H2 2020;Three clinical stage RNA therapies in development for inherited retinal diseases, with fourth ophthalmic program slated to enter mechanistic proof-of-concept studies;ProQR anticipates its cash runway will fund operations into H2 2022 LEIDEN, Netherlands & CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- 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 reported its financial results for the first quarter ended March 31, 2020 and provided a business update.\n “At the end of the first quarter we shared positive findings from the interim analysis of the Phase 1/2 Stellar trial of QR-421a for Usher’s syndrome, which adds to the growing body of evidence further validating the potential of our platform,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “Based on these findings, we are continuing the trial as designed, with dose expansion and escalation cohorts planned. We are working closely with our clinical trial sites to monitor the evolving COVID-19 situation and preparing to rapidly ramp up enrollment once it is deemed safe to do so. In the second half of 2020 we look forward to sharing updated data from the Phase 1/2 InSight extension study of sepofarsen for LCA10, including data from the contralateral eye treatment. We are confident in our fundamentals – we have a productive platform, a deep pipeline, and are well capitalized – as we continue our work to bring novel RNA therapies to patients.” Business Operations and Program Updates Ongoing clinical studies of sepofarsen for LCA10, QR-421a for Usher syndrome and nsRP, and QR-1123 for adRP are all currently active, but the effects of the COVID-19 pandemic are resulting in disruptions to patient enrollment across these programs. In consultation with clinical trial sites, ProQR is implementing mitigation procedures that support a rapid ramp up in enrollment as soon as the disruption all...