ProQR Reports Q3 2024 Results

ProQR Therapeutics NV, a biotechnology firm specializing in transformative RNA therapies, recently announced its financial and operational results for the third quarter ending September 30, 2024. ProQR is advancing its proprietary Axiomer™ RNA editing technology, which aims to address various genetic diseases through precise RNA modifications.

The company successfully completed an $82.1 million equity financing in October 2024, bolstering its financial position and extending its operational runway to mid-2027. ProQR's CEO, Daniel A. de Boer, expressed gratitude for the unwavering support from investors and their partner, Eli Lilly, emphasizing ProQR's focus on developing transformative therapies for unmet medical needs.

ProQR plans to initiate clinical trials for its leading RNA editing program, AX-0810, which targets NTCP for cholestatic diseases. The company intends to share further details on this program, including preclinical data, clinical trial design, and the biomarkers to be assessed, during a virtual Investor and Analyst Event scheduled for December. Additionally, updates will be provided on AX-1412, targeting B4GALT1 for cardiovascular diseases, and other advancements within the Axiomer platform.

Financially, ProQR reported having €89.4 million in cash and cash equivalents as of September 30, 2024, a decrease from €118.9 million at the end of 2023. The net cash used in operating activities during the third quarter of 2024 was €5.7 million, an improvement compared to €7.5 million for the same period in the previous year. The company achieved certain milestones in its collaboration with Eli Lilly, amounting to $5.5 million.

Research and development (R&D) expenses for the quarter increased to €9.4 million compared to €5.4 million in the same period last year. General and administrative costs remained stable at €3.3 million. The net loss for the quarter was €8.1 million, or €0.10 per diluted share, compared to a net loss of €5.4 million, or €0.07 per diluted share, in the same period the previous year.

ProQR highlighted its progress and future plans for the Axiomer platform at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society in Montreal, with a focus on AX-0810 and its potential to address cholestatic diseases by targeting NTCP. The upcoming virtual event in December will further outline translational non-clinical data, delivery data, clinical trial design, timelines, and details on biomarkers for AX-0810. Additional updates on AX-1412 for cardiovascular disease and other platform advancements will also be shared.

Cholestatic disorders result in an excessive and toxic buildup of bile acids in the liver, leading to liver damage and severe symptoms. Without effective treatment, these conditions can progress to liver failure and increase the risk of liver malignancy. Currently, no approved therapies exist for primary sclerosing cholangitis (PSC) in adults or biliary atresia (BA) in pediatric patients, which respectively affect approximately 80,000 and 20,000 individuals in North America and Europe.

AX-0810 is designed to inhibit the NTCP transporter, which is responsible for the reuptake of bile acids in the liver. This approach has the potential to modify the disease course for PSC and BA. In the cardiovascular field, AX-1412 targets the B4GALT1 gene, introducing a protective variant to reduce the risk of cardiovascular diseases by addressing residual risk factors such as hypo-galactosylation of apolipoprotein B100 and fibrinogen.

ProQR, through its Axiomer™ technology, aims to pioneer RNA-based therapies capable of making precise single nucleotide edits in RNA. This technology leverages the cell's own editing machinery, ADAR, to correct disease-causing mutations or modulate protein expression, potentially creating a new class of medicines for various diseases with unmet medical needs. The company continues to expand its pipeline, guided by the goal of improving patient outcomes and addressing significant medical challenges.