Business
ProQR Announces First Quarter 2023 Operating and Financial Results
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1Development of
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1Development of Axiomer® RNA base editing technology platform continues across multiple therapeutic areasStrength of intellectual property estate and leading IP position supported with successful defense of opposition against a key Axiomer patent protecting ADAR-mediated RNA editingWell-capitalized with €139 M, in cash and cash equivalents supporting runway into mid-2026 LEIDEN, Netherlands & CAMBRIDGE, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today reported its financial and operating results for the first quarter ended March 31, 2023, and provided a business update. “During our R&D event in Q1, we provided a comprehensive update on how we’re executing against our strategy to accelerate the development of Axiomer,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “We believe this platform has broad applicability across multiple therapeutic areas and, based on the encouraging initial in vivo data we’ve generated, look forward to advancing initial programs for diseases that originate in the liver. Over the next 12-18 months, we anticipate presenting and publishing additional preclinical data, including further non-human primate data, to expand on the potential of Axiomer to address diseases with high unmet medical need.” Recent Progress In March, ProQR announced AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 as initial pipeline programs. These programs share several key characteristics including a deep rooting in human genetics, the potential to have a major impact in indications with high unmet medical need, the ability to leverage the existing proven delivery technology to the liver, the opportunity to monitor early biomarkers to establish target engagement in Phase I trials for human proof of concept, and the availability of well-defined clinical endpoints. AX-0810 for Cholestatic Diseases targeting NTCP is designed to introduce a loss of function (LOF) variant that has been found in human genetics to prevent re-uptake of bile acids in liver. ...