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Investing.com - ProQR Therapeutics N.V. (NASDAQ:PRQR) stock rose Tuesday, extending its impressive 11.92% gain over the past week, after JMP Securities reiterated its Market Outperform rating and $8.00 price target on the RNA editing company. According to InvestingPro data, the company’s stock has delivered a robust 30.91% return over the past year.
JMP highlighted ProQR’s on-schedule submission of a Clinical Trial Application (CTA) for AX-0810, calling it "fundamentally derisking" as the first candidate from the company’s Axiomer RNA editing platform to enter clinical testing. The company maintains a strong financial position with more cash than debt on its balance sheet, though InvestingPro analysis indicates rapid cash burn rates.
The upcoming Phase 1 trial in healthy volunteers will assess AX-0910, which introduces a loss of function variant in SLC10A1 RNA to prevent bile acid re-uptake in the liver via the sodium-taurocholate co-transporting polypeptide (NTCP), potentially offering disease-modifying treatment for multiple cholestatic diseases.
Key endpoints in the Phase 1 study will include measurements of serum bile acids, with ProQR targeting a two-fold increase as evidence of target engagement, along with a concordant increase in urinary bile acid levels and analysis of conjugated versus unconjugated bile acids.
JMP expressed continued confidence in ProQR’s position in the RNA-editing field, noting its "differentiated development strategy" and anticipating initial clinical data from the Axiomer platform in the near term. With a market capitalization of $228.59 million, ProQR shows promising momentum despite current unprofitability. Discover more insights and 8 additional ProTips with InvestingPro’s comprehensive analysis and research reports.
In other recent news, ProQR Therapeutics has submitted a Clinical Trial Application to the European Medicines Agency for its RNA editing therapy, AX-0810. This application marks a significant milestone as it seeks approval to commence a Phase 1 clinical trial aimed at treating cholestatic liver diseases. The trial will focus on evaluating the safety and effectiveness of the therapy in healthy volunteers, with initial data expected in the fourth quarter of 2025. Additionally, Cantor Fitzgerald has initiated coverage on ProQR with an Overweight rating and a price target of $8.00, citing the company’s promising clinical program and leadership in RNA editing. Evercore ISI also began coverage, assigning an Outperform rating with a $5.00 price target, highlighting ProQR’s potential for long-term value creation. Citizens JMP reaffirmed its Market Outperform rating and $8.00 price target, noting the anticipated filing of the Clinical Trial Application as a key step forward. Furthermore, ProQR announced new appointments to its executive team, naming Dennis Hom as Chief Financial Officer and Cristina Lopez Lopez as Chief Medical (TASE:BLWV) Officer, to support the advancement of its Axiomer RNA editing platform. These developments underscore ProQR’s commitment to leveraging its RNA-editing technology across various therapeutic areas.
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