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Investing.com - ProQR Therapeutics N.V. (NASDAQ:PRQR) stock gained Thursday after Cantor Fitzgerald maintained its Overweight rating and $8.00 price target on the RNA editing company. The stock has shown strong momentum with an 11.92% gain over the past week, though according to InvestingPro data, the RSI suggests the stock is currently in overbought territory.
The reaffirmation follows ProQR’s announcement Thursday of its first Clinical Trial Application submission for its RNA editing Axiomer platform, which Cantor Fitzgerald described as triggering "a new, exciting chapter" for the company. InvestingPro analysis shows the company maintains a strong financial position with a current ratio of 4.1, indicating solid short-term liquidity.
ProQR remains on track to deliver initial data from the program in the fourth quarter of 2025, with the Phase 1 study designed to provide proof of concept for AX-0810 RNA editor targeting NTCP in healthy volunteers for treatment of cholestatic diseases.
Cantor Fitzgerald noted its financial model for ProQR is primarily driven by AX-0810, which introduces a naturally occurring loss of function Q68R mutation in NTCP to lower bile acid uptake into hepatocytes, with the company targeting approximately twice the increase in serum bile acid as a biomarker of AX-0810 activity.
In preclinical data with non-human primates, the first-generation candidate demonstrated up to four times increase in serum bile acid with only 20% editing, which was subsequently improved to 50%, according to the research firm. For deeper insights into ProQR’s financial health and growth prospects, including 8 additional ProTips and comprehensive analysis, check out the full research report available on InvestingPro.
In other recent news, ProQR Therapeutics has made significant strides with its RNA editing therapy, AX-0810, by submitting a Clinical Trial Application to the European Medicines Agency. This submission marks a major milestone as it is the first clinical-stage program for the company’s Axiomer platform, with initial data expected in the fourth quarter of 2025. The trial will assess safety and target engagement in healthy volunteers, focusing on treating cholestatic liver diseases. Analyst firms have taken note of ProQR’s progress, with JMP Securities reiterating a Market Outperform rating and an $8.00 price target, highlighting the company’s differentiated development strategy. Cantor Fitzgerald also initiated coverage with an Overweight rating and the same price target, citing ProQR’s promising clinical program and potential catalysts from upcoming biomarker data. Meanwhile, Evercore ISI began coverage with an Outperform rating and a $5.00 price target, emphasizing the company’s innovative approach and significant internal progress. ProQR’s pipeline includes other promising candidates, such as those targeting Rett Syndrome and Metabolic Associated Steatohepatitis (MASH), with initial data anticipated in 2026. These developments underscore ProQR’s continued focus on leveraging its RNA-editing platform for various diseases with unmet medical needs.
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