ProQR Reports Q1 2024 Results

ProQR Therapeutics NV, a company committed to developing transformative RNA therapies, recently disclosed its financial and operational results for the first quarter ending March 31, 2024. The company highlighted key advancements and provided a business update emphasizing its proprietary Axiomer™ RNA editing technology.

ProQR's CEO, Daniel A. de Boer, underscored a significant milestone achieved with the presentation of preclinical proof of concept data for their NTCP program at the ASGCT 27th Annual Meeting. This data demonstrated, for the first time in the ADAR RNA editing field, in vivo proof of target engagement with meaningful biomarker changes in non-human primates (NHPs). This achievement bolsters confidence in ProQR's Axiomer technology. The company anticipates further milestones this year as they advance their first editing oligonucleotide pipeline programs to the clinic by late 2024 or early 2025.

In May, ProQR presented compelling preclinical data for AX-0810, targeting NTCP for cholestatic diseases, at the ASGCT Annual Meeting. The data revealed that Axiomer EONs (Editing Oligonucleotides) can specifically modulate NTCP protein bile acids reuptake function while preserving protein expression. A strong correlation was observed between editing levels of NTCP and changes in serum bile acids in NHPs. The early generation of Axiomer EONs achieved up to 29% editing of NTCP in the liver of NHPs after a single dose, resulting in an eight-fold change in serum bile acids 72 hours post-treatment. Further optimizations have led to up to 60% editing in vitro. The in vivo results in NHPs confirmed findings from in vitro models, demonstrating translatability across models.

ProQR, in collaboration with Eli Lilly, also presented preclinical data at the ASGCT Annual Meeting, highlighting the complex metabolism and prolonged PK/PD of a GalNAc-conjugated EON in mice. This partnership continues to show the potential for Axiomer technology in various applications.

In April, ProQR announced the nomination of Martin Maier, PhD, to its Board of Directors, with the Annual General Meeting scheduled for May 22, 2024. Dr. Maier brings extensive expertise in RNA platforms and delivery technology, having contributed to multiple RNA product approvals during his tenure at Alnylam Pharmaceuticals.

Additionally, ProQR successfully defended a challenge to its Axiomer IP portfolio filed with the European Patent Office. This defense underscores the strength of ProQR's leading IP estate around ADAR-mediated RNA editing.

Looking ahead, ProQR plans to report translational data and share clinical trial designs for AX-0810 in the second half of 2024. They will also present preclinical proof of concept and translational data for AX-1412, targeting B4GALT1 for cardiovascular diseases, with the program expected to advance to the clinic in late 2024 or early 2025. ProQR may announce additional pipeline targets and continue to execute their partnership with Eli Lilly, potentially expanding the deal to 15 targets, which could result in a $50 million opt-in payment.

Financially, ProQR reported holding €102.7 million in cash as of March 31, 2024, providing a runway into mid-2026. During the first quarter, the company earned $1.0 million from a milestone in their collaboration with Eli Lilly. Research and development costs for the quarter were €9.3 million, compared to €6.1 million last year, while general and administrative costs were €3.5 million, down from €4.0 million. The net loss for the quarter was €7.7 million, or €0.09 per diluted share, compared to a net loss of €8.9 million, or €0.11 per diluted share, for the same period last year.

ProQR continues to pioneer Axiomer™, an RNA base editing technology that employs the cell's own editing machinery to make specific single nucleotide edits in RNA, potentially creating a new class of medicines for various diseases. This technology could reverse mutations or modulate protein expression, addressing both rare and prevalent diseases with unmet medical needs.