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On Wednesday, Citizens JMP reaffirmed its Market Outperform rating and a price target of $8.00 on ProQR Therapeutics N.V. (NASDAQ:PRQR), a $142 million market cap biotechnology company currently trading at $1.35. According to InvestingPro analysis, the stock appears undervalued based on its Fair Value calculations. The company, which specializes in RNA-editing technology, is anticipated to file a Clinical Trial Application (CTA) for its novel candidate AX0810 within the current quarter. This step is crucial as it paves the way for the first-in-human trials.
The focus is on AX0810’s potential to treat cholestatic diseases, a group of conditions that impair bile flow from the liver. The upcoming trial aims to gather initial data from healthy volunteers, which will help determine the drug’s target engagement and biomarkers. These early indicators are expected to shed light on the treatment’s possible effectiveness. InvestingPro data shows the company maintains a strong liquidity position with a current ratio of 3.95, though it’s currently experiencing rapid cash burn - a crucial factor for biotechnology companies in the development phase.
ProQR Therapeutics is also progressing with its research in other areas. The company is preparing to select candidates for its programs targeting Rett Syndrome and Metabolic Associated Steatohepatitis (MASH) within this year, with initial data anticipated in 2026. These developments are part of the company’s broader efforts to leverage its RNA-editing platform across various diseases.
Moreover, ProQR is set to provide an update by mid-2025 on another of its candidates, AX-1412. This drug is designed to target B4GALT1, a gene associated with cardiovascular disease, and the update will include details on the optimization of its delivery system.
Citizens JMP’s analyst expressed optimism about the company’s position in the RNA-editing field and the upcoming milestones that could potentially de-risk its pipeline. The fourth quarter of 2025 is particularly significant as it is when de-risking data for AX0810 is expected to become available. Analyst targets range from $4 to $15 per share, reflecting significant upside potential. For deeper insights into ProQR’s financial health and growth prospects, investors can access the comprehensive Pro Research Report, available exclusively on InvestingPro, which covers this and 1,400+ other US equities with detailed analysis and actionable intelligence.
In other recent news, ProQR Therapeutics has made significant strides in its executive team and research initiatives. The company announced the appointment of Dennis Hom as Chief Financial Officer and Cristina Lopez Lopez, MD, PhD, as Chief Medical (TASE:BLWV) Officer. These strategic hires aim to advance ProQR’s Axiomer™ RNA editing platform into clinical stages. Meanwhile, H.C. Wainwright raised ProQR’s stock target to $12, maintaining a Buy rating, citing optimism in the company’s lead program, AX-0810, for Primary Sclerosing Cholangitis (PSC). Raymond (NSE:RYMD) James also maintained a Strong Buy rating with a $14 target, following ProQR’s solid financials and clinical progress, including collaboration revenue of €4.3 million and cash reserves of €149.4 million. Citi upgraded ProQR’s rating to Buy with a $4 target, highlighting the market’s undervaluation of AX-0810. Additionally, Jones Trading initiated coverage with a Buy rating and an $11 target, emphasizing the potential of ProQR’s RNA editing programs for both rare and common diseases. These developments reflect ProQR’s ongoing efforts to strengthen its leadership and expand its research capabilities.
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