ProQR Announces Encouraging AX-0810 Phase 1 Safety and PK Data, Development Candidate Selections, and 2026 Outlook

Core Insights - ProQR Therapeutics announced initial safety and pharmacokinetic (PK) data from its Phase 1 trial of AX-0810, indicating a positive early milestone for the company [2][5] - The company is advancing its pipeline with new development candidates for AX-2402 and AX-2911, targeting Rett syndrome and metabolic-associated steatohepatitis (MASH) respectively [7][12] AX-0810 Phase 1 Study - AX-0810 is a lead investigational RNA editing oligonucleotide targeting NTCP for cholestatic diseases [3][16] - The Phase 1 study is a randomized, double-blind, placebo-controlled trial with up to 33 participants, assessing safety, tolerability, and pharmacokinetics [4] - Initial data from the first cohort (3 mg/kg) showed no serious adverse events and PK observations aligned with non-clinical data, supporting continued dosing [5][9] Pipeline Progress - ProQR has selected development candidates for AX-2402 targeting MECP2 for Rett syndrome and AX-2911 targeting PNPLA3 for MASH [10][12] - AX-2402 demonstrated significant functional improvements in a mouse model, supported by funding from the Rett Syndrome Research Trust [11] - AX-2911 showed over 80% reduction in hepatic fat content in a humanized mouse model, outperforming a clinical-stage therapy [13] Strategic Collaboration - The collaboration with Eli Lilly achieved $4.5 million in milestones in 2025, validating ProQR's Axiomer platform and providing non-dilutive capital [14] Corporate Outlook - ProQR plans to report target engagement data for AX-0810 in the first half of 2026 and initiate a patient cohort in the trial [6][18] - The company aims to advance AX-2402 to a first-in-human trial in the first half of 2027 and continue to strengthen its financial position [18]