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On Monday, H.C. Wainwright analyst Andrew Fein adjusted the price target for ProQR Therapeutics N.V. (NASDAQ:PRQR) to $12.00, an increase from the previous $10.00, while keeping a Buy rating on the stock. Currently trading at $1.69, significantly below its 52-week high of $4.62, the stock has garnered strong analyst support with a highly bullish consensus rating. According to InvestingPro data, analyst price targets range from $4 to $15, suggesting substantial upside potential from current levels. Fein’s optimism is rooted in the potential of ProQR’s Axiomer technology, particularly as it pertains to the company’s lead program AX-0810 for Primary Sclerosing Cholangitis (PSC), which is currently entering a Phase 1 study. Topline data from this study is anticipated in the fourth quarter of 2025. InvestingPro analysis shows the company maintains a strong financial position with more cash than debt and a healthy current ratio of 3.95, though investors should note the company is currently burning through cash rapidly.
ProQR’s AX-0810 is being closely monitored as it targets PSC, a disease with no approved treatments and a significant unmet medical need. Fein highlighted several factors contributing to his positive outlook, including the recognition of bile acids’ importance and toxicity in PSC, supporting the RNA editing approach that ProQR is employing. Additionally, preclinical data and other scientific findings provide evidence that supports the therapeutic potential of AX-0810.
Fein pointed out that improvements in liver fibrosis and liver enzymes have been observed in the absence of virologic response in clinical data from Bulevirtide, an inhibitor of NTCP, which is also the target for PSC. Human genetics data has shown that a variant of NTCP can limit bile acid uptake, which further supports the potential efficacy of ProQR’s approach.
The analyst also noted the promising preclinical data from humanized mice and non-human primates (NHPs) that demonstrated significant target engagement and a potential safety profile for AX-0810. The Phase 1 study will focus on changes in bile acid levels in plasma, bolstered by known target engagement profiles in NHPs and effects on bile acid levels in engineered mice.
Fein’s reevaluation of the market penetration assumptions for PSC has led to the increased price target for ProQR. The firm’s confidence in the RNA editing modality and the comprehensive data supporting AX-0810’s development for PSC treatment have contributed to this optimistic assessment. With a market capitalization of $177.81 million and significant revenue growth potential, InvestingPro analysis suggests the stock is currently undervalued. Discover more insights about PRQR and access comprehensive analysis of 1,400+ stocks through InvestingPro’s detailed research reports, which transform complex financial data into actionable intelligence for smarter investment decisions.
In other recent news, ProQR Therapeutics N.V. has reported its fourth-quarter earnings for 2024, highlighting collaboration revenue of €4.3 million and operating expenses of €14.5 million, resulting in an earnings per share (EPS) of €(0.09). The company maintains a strong financial position with cash and cash equivalents totaling €149.4 million, bolstered by recent financing. Raymond (NSE:RYMD) James reiterated a Strong Buy rating for ProQR, setting a price target of $14.00, citing promising clinical developments and solid financials. Citi upgraded ProQR’s stock from Neutral to Buy, with a price target of $4.00, emphasizing the potential of its lead drug candidate, AX-0810, for treating cholestatic diseases. Jones Trading initiated coverage with a Buy rating and an $11.00 price target, noting the company’s focus on RNA editing and its pipeline for rare and common diseases. ProQR is expected to submit a Clinical Trial Application for AX-0810 in the second quarter of 2025, with top-line data anticipated in the fourth quarter. The company has also expanded its collaboration with the Rett Syndrome Research Trust, securing up to $9.2 million to support the development of AX-2402. Additionally, ProQR’s partnership with Eli Lilly (NYSE:LLY) underscores the platform’s value and potential for future milestone payments.
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