ProQR reports Q2 2026 results, cites first Axiomer clinical validation from AX-0810 data

ProQR Therapeutics N.V. reported financial and operating results for the second quarter ended June 30, 2026, in a release dated August 13, 2026.

The company said positive AX-0810 Phase 1 target engagement data from healthy volunteers in June marked the first clinical validation of the Company's proprietary Axiomer RNA editing platform.¹ Among the reported findings, total serum bile acids rose up to 8-fold at the 6 mg/kg dose, exceeding the company's pre-defined 2-fold threshold for meaningful NTCP modulation, with dose-dependent changes in conjugated bile acids alongside similar shifts in total serum bile acids and TUDCA, which the company said backs its theory that reducing NTCP activity lowers buildup of harmful bile acids in the liver.¹ The company also reported a favorable safety and tolerability profile with no serious adverse events or pruritus reported, along with pharmacokinetic data supporting sustained target engagement, including a half-life of eight weeks.¹ ProQR said it intends to share the complete Phase 1 results, covering Cohort 3, at a scientific or medical meeting later this year, as noted in plans to present the full dataset from the Phase 1 study of AX-0810, including Cohort 3, at a medical or scientific conference later this year.¹

In July, ProQR submitted a Clinical Trial Application to the European Medicines Agency to initiate a Phase 1 clinical trial of AX-0811, its next-generation editing oligonucleotide (EON) targeting NTCP for the treatment of cholestatic diseases.¹ Pending CTA authorization, initial data with AX-0811 is anticipated by year end 2026.¹ The company also said it continues preparations for an investigator-initiated trial (IIT) in pediatric biliary atresia in China, with initial clinical data expected in the first half of 2027.¹

On financing, in June ProQR closed an underwritten registered direct offering of $50.0 million, alongside a concurrent private placement to an existing shareholder and strategic partner Eli Lilly and Company (Lilly) of $9.2 million, for total gross proceeds of approximately $59.2 million.¹ The company ended the quarter with cash and cash equivalents of approximately €117.1 million, compared to €92.4 million at December 31, 2025,¹ which it said provided runway into mid-2028 and is expected to support continued advancement of its wholly owned Axiomer RNA editing pipeline.¹ Net loss for the quarter ended June 30, 2026 was €8.7 million, or €0.08 per diluted share, compared to €12.2 million, or €0.12 per diluted share, for the same period in 2025.¹