Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; Phase 1 enrollment and dosing in healthy volunteers ongoing with target engagement data expected in H1 2026, followed by inclusion of a patient cohortDevelopment Candidates selected for pipeline programs AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3)Strategic collaboration with Eli Lilly achieved $4.5 million in milestones in 2025, contributing to strong financi ...

Company Overview - ProQR is pioneering ADAR-mediated RNA editing with its Axiomer platform, moving from invention to clinical trials [11, 17] - The company has a strong intellectual property estate and strategic partnerships with Eli Lilly and Rett Syndrome Research Trust [17, 135] - ProQR's cash and cash equivalents were €119.8 million as of the end of Q2 2025, providing a runway into mid-2027 [18, 136] Pipeline and Clinical Development - AX-0810, targeting NTCP for cholestatic diseases, has received CTA authorization and is entering a Phase 1 clinical trial [17, 21] - The Phase 1 trial aims to establish safety, PK, and target engagement in healthy volunteers [21] - AX-2402, for Rett syndrome, is rapidly advancing, positioning ProQR at the forefront of neurological RNA editing medicines [21] AX-0810 and Cholestatic Diseases - Cholestatic diseases, including Primary Sclerosing Cholangitis (PSC) affecting approximately 80,000 adults and Congenital Biliary Atresia affecting approximately 20,000 pediatrics, have high unmet medical needs [24, 38] - AX-0810 is a first-in-class RNA editing therapy designed to modulate the NTCP channel, limiting bile acid uptake while preserving other channel functions [31] - The therapeutic goal of AX-0810 is to reduce inflammation and fibrosis, alleviate symptoms in PSC and BA, and prevent or delay cirrhosis, organ failure, and transplant [36] AX-0810 Clinical Trial Design - The Phase 1 trial is a multiple ascending dose (MAD) study with N=33 participants (24 on treatment, 9 on placebo) across three cohorts: 3mg/kg, 6mg/kg, and 9mg/kg (TBC) [103, 104] - The trial will assess safety, tolerability, and PK of AX-0810, and confirm target engagement as measured by changes in bile acid levels, bile acid profile, and TUDCA challenge [107] - Initial Cohort 1 safety, tolerability, and PK data are expected toward the end of 2025, with target engagement data on all cohorts anticipated in H1 2026 [107, 131]

Core Insights - ProQR Therapeutics has received authorization from the Central Committee on Research Involving Human Subjects (CCMO) for a Phase 1 Clinical Trial Application (CTA) for AX-0810, targeting cholestatic diseases [1][2] - AX-0810 is the first investigational RNA editing oligonucleotide (EON) from ProQR's Axiomer™ platform, aimed at reducing toxic bile acid accumulation in the liver [6][10] Company Overview - ProQR Therapeutics is focused on developing transformative RNA therapies using its proprietary Axiomer technology, which utilizes the body's own ADAR enzymes for RNA editing [1][10] - The company aims to create a new class of medicines for both rare and prevalent diseases with unmet medical needs [10] Clinical Development - The Phase 1 study of AX-0810 will assess safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) through biomarkers to establish proof of target engagement [2][6] - The trial will be conducted in the Netherlands, and dosing is set to begin following the CTA approval [2] Upcoming Events - ProQR will host a virtual Investor and Analyst Event on November 3, 2025, to discuss the clinical development of AX-0810 [2][8] - The event will feature presentations from ProQR management and Professor Henkjan J. Verkade, a key opinion leader in pediatric gastroenterology [2][4]

Core Insights - ProQR Therapeutics is focused on developing transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform [1][3] - The company will participate in several upcoming investor conferences, providing opportunities for engagement with investors [1][5] Company Overview - ProQR Therapeutics is dedicated to creating RNA therapies that can address both rare and prevalent diseases with unmet medical needs [3] - The Axiomer technology utilizes the human cell's own ADAR machinery to make precise single nucleotide edits in RNA, potentially reversing mutations or modulating protein expression [2][3] Upcoming Events - ProQR will present at the H.C. Wainwright Genetic Medicines Virtual Conference on October 14-15, 2025, with presentations available on demand starting October 14 [5] - The company will also participate in the H.C. Wainwright Liver Disease Virtual Conference on October 21-22, 2025, and the Chardan's 9th Annual Genetic Medicines Conference on October 21, 2025 [5]