ProQR Reports Q2 2024 Financial and Operating Results

ProQR Therapeutics NV, a company committed to developing transformative RNA therapies based on its Axiomer™ RNA editing technology, released its financial and operational performance for the second quarter ending June 30, 2024.

Founder and CEO Daniel A. de Boer emphasized the progress in advancing the company's first RNA editing programs to the clinical stage. The AX-0810 program, targeting NTCP for cholestatic diseases, and the AX-1412 program, targeting B4GALT1 for cardiovascular diseases, are on schedule to enter clinical trials by late 2024 or early 2025. De Boer highlighted the in vivo proof of target engagement shown at the American Society of Cell and Gene Therapy (ASGCT) Annual Meeting in May, which demonstrated significant changes in disease-related biomarkers in non-human primates for the NTCP program. ProQR plans to announce more translational data and detailed clinical plans for AX-0810 later this year, along with preclinical proof of concept data for AX-1412.

De Boer also expressed satisfaction with the progress of their partnership with Eli Lilly, noting multiple milestone achievements in the first half of the year. He credited the successful collaboration, the company's genetic-based pipeline strategy, an extensive intellectual property portfolio, and a strong financial position with runway into mid-2026 as key factors for future advancements.

Recent developments include ProQR's presentations at the RNA Editing Summit in June and the ASGCT Annual Meeting in May, showcasing their Axiomer platform and preclinical data for AX-0810. The company also demonstrated for the first time in vivo RNA editing proof of target engagement with significant biomarker changes in non-human primates.

Anticipated events for the latter half of 2024 include reporting clinical development candidate translational data and clinical trial design for AX-0810, as well as preclinical proof of concept data for AX-1412. ProQR also expects to advance these programs to clinical trials by late 2024 or early 2025. The company may announce additional new pipeline targets and will continue to focus on its partnership with Eli Lilly, which could include further data updates and potential milestone income. ProQR may form new partnerships, including multi-target discovery alliances or product-specific collaborations.

Financially, ProQR reported holding €96.2 million in cash and cash equivalents at the end of Q2 2024, down from €118.9 million at the end of 2023. The net cash used in operating activities during Q2 2024 was €6.3 million, compared to €10.3 million in the same period last year. The company achieved $4.5 million in milestones in the first half of 2024 through its collaboration with Eli Lilly.

Research and development expenses for Q2 2024 were €7.1 million, up from €5.9 million in the same period last year. General and administrative costs were lower, at €3.0 million in Q2 2024 compared to €4.1 million in Q2 2023. The net loss for Q2 2024 was €2.7 million, translating to €0.03 per diluted share, a significant improvement compared to a net loss of €8.0 million, or €0.10 per diluted share, for the same period last year.

ProQR's Axiomer™ technology represents a potential breakthrough in RNA base editing, offering a new class of treatments for various diseases. This technology utilizes the endogenous ADAR (Adenosine Deaminase Acting on RNA) present in human cells to modify RNA, potentially correcting disease-causing mutations or modulating protein expression to treat diseases.

Cholestatic diseases such as biliary atresia (BA) and primary sclerosing cholangitis (PSC) involve the buildup of toxic bile acids in the liver, leading to liver damage and various debilitating symptoms. With no approved therapies for BA and PSC, ProQR's AX-0810 program offers hope for disease-modifying treatments by targeting NTCP.

Cardiovascular diseases (CVDs) remain the leading cause of death globally. ProQR's AX-1412 program aims to address CVD risks by introducing a protective variant into B4GALT1 RNA, potentially providing a novel treatment approach for reducing the residual risk of developing cardiovascular diseases.

ProQR Therapeutics is committed to advancing RNA therapies and aims to make significant strides in treating rare and prevalent diseases through its innovative RNA editing technology, Axiomer™. The company remains focused on executing its pipeline programs and leveraging strategic partnerships to bring transformative treatments to patients in need.