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On Monday, Citi analysts upgraded shares of ProQR Therapeutics N.V. (NASDAQ:PRQR) from Neutral to Buy, setting a price target of $4.00. The upgrade comes as the company’s market capitalization stands at $197 million, closely aligning with its pro-forma cash position of approximately $150 million. According to InvestingPro data, PRQR holds more cash than debt on its balance sheet, with a healthy current ratio of 2.33x, indicating strong short-term liquidity. Analysts at Citi noted that the market is currently assigning minimal value to ProQR’s leading drug candidate, AX-0810, an RNA editing therapy aimed at treating cholestatic diseases by targeting the bile acid transporter NTCP. InvestingPro analysis shows the company’s overall financial health score is "FAIR," with analysts anticipating sales growth in the current year despite the company currently burning through cash.
ProQR is expected to submit a Clinical Trial Application (CTA) for AX-0810 in the second quarter of 2025, with top-line data for familial intrahepatic cholestasis anticipated in the fourth quarter of the same year. This marks a significant period for the company, which has been realigning its pipeline following the failure of its LCA10 treatment three years prior.
The potential market for AX-0810 is significant, with an estimated 100,000 patients in the United States and EU5 suffering from Primary Sclerosing Cholangitis and Biliary Atresia—conditions that AX-0810 aims to address. These diseases represent a multi-billion-dollar market opportunity, which Citi analysts suggest could contribute around $3 per share of their $4 price target for ProQR.
Despite a spike in ProQR’s stock in mid-October 2024, the shares have continued to decline, with InvestingPro data showing a 10.3% drop in the past week and the stock trading 59% below its 52-week high of $4.62. However, Citi analysts believe that the upcoming top-line readout later in the year could be a pivotal moment for the company’s valuation. As a result, they have reiterated their $4 price target alongside the upgrade to a Buy rating, citing valuation and the anticipated data as the basis for the positive outlook on the stock. The current analyst consensus shows strong bullish sentiment, with price targets ranging from $4 to $15 per share. Get access to 10+ additional ProTips and comprehensive financial analysis for PRQR through InvestingPro’s detailed research reports.
In other recent news, ProQR Therapeutics has received significant attention from analysts and investors due to its developments in RNA editing technology. The company announced an expanded collaboration with the Rett Syndrome Research Trust (RSRT), securing a total of $9.1 million in funding to advance its treatment for Rett Syndrome into clinical trials. This collaboration aims to optimize therapeutic candidates targeting the MECP2 gene mutation using ProQR’s proprietary Axiomer RNA editing platform.
Meanwhile, Jones Trading initiated coverage on ProQR with a Buy rating and set a price target of $11.00, citing the company’s focus on RNA editing for both rare and common diseases. The analyst highlighted ProQR’s pipeline, including treatments for primary sclerosing cholangitis (PSC) and Rett Syndrome, with the PSC program expected to deliver Phase 1 data by the fourth quarter of 2025. H.C. Wainwright also maintained a Buy rating and a price target of $10.00, expressing optimism about the company’s RNA editing capabilities and its potential to address unmet medical needs in diseases like PSC.
These recent developments underscore ProQR’s strategic focus on RNA editing, a technology that modifies mRNA without changing the genetic code, offering a promising approach for precise therapeutic interventions.
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