ProQR submits clinical trial application for RNA editing therapy

LEIDEN/CAMBRIDGE - ProQR Therapeutics NV (NASDAQ:PRQR), a $228.59 million market cap biotechnology company that has seen an 11.34% gain in the past week, has submitted a Clinical Trial Application to the European Medicines Agency for its lead RNA editing therapy AX-0810, according to a press release statement issued Thursday.

The application seeks approval to begin a Phase 1 clinical trial evaluating AX-0810 in healthy volunteers, with initial data expected in the fourth quarter of 2025. The study will assess safety, tolerability, pharmacokinetics, and target engagement at a single site in the Netherlands, pending regulatory clearance. According to InvestingPro data, the company maintains a healthy financial position with a current ratio of 4.1, indicating strong ability to fund its clinical programs.

AX-0810 is designed to treat cholestatic liver diseases by targeting NTCP, a liver cell protein that transports bile acids. The investigational therapy aims to reduce toxic bile acid accumulation in the liver that can lead to inflammation, fibrosis, and progression toward liver failure.

"This submission marks a significant milestone for ProQR, representing the first to advance our Axiomer RNA editing platform and pipeline into clinical development," said Cristina Lopez Lopez, Chief Medical Officer of ProQR.

The therapy uses ProQR’s proprietary Axiomer technology, which employs a cell’s own editing machinery called ADAR (Adenosine Deaminase Acting on RNA) to make specific single nucleotide changes in RNA.

The company’s approach is supported by human genetics data showing that naturally occurring NTCP variants reduce bile acid reuptake, suggesting potential benefits for patients with cholestatic liver diseases.

This marks the first clinical-stage program for ProQR’s Axiomer platform, which aims to create RNA-based therapies for both rare and prevalent diseases with unmet medical needs. While the company holds more cash than debt on its balance sheet, InvestingPro analysis reveals over 10 additional key insights about ProQR’s financial health and market position, available through the comprehensive Pro Research Report covering 1,400+ US equities.

In other recent news, ProQR Therapeutics has been the focus of several analyst evaluations and strategic developments. Cantor Fitzgerald initiated coverage on ProQR with an Overweight rating and a price target of $8, highlighting the company’s strong position in RNA editing and promising clinical programs. Evercore ISI also began coverage, assigning an Outperform rating and a $5 price target, emphasizing ProQR’s potential for long-term value creation. Citizens JMP reiterated its Market Outperform rating with an $8 price target, noting the upcoming Clinical Trial Application for ProQR’s candidate AX0810, which could be pivotal for the company’s future clinical trials.

Additionally, ProQR announced key appointments to its executive team, naming Dennis Hom as Chief Financial Officer and Cristina Lopez Lopez as Chief Medical Officer. These appointments are part of the company’s strategy to advance its Axiomer™ RNA editing platform into clinical stages. The new executives bring extensive experience in financial leadership and translational R&D, respectively, which ProQR anticipates will bolster its strategic initiatives. ProQR is also preparing to update on its AX-1412 candidate by mid-2025, aiming to optimize its delivery system for treating cardiovascular diseases. These developments reflect ProQR’s ongoing efforts to expand its pipeline and strengthen its position in the RNA editing field.

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