ProQR(US:PRQR)2025-11-03 16:00ProQR Therapeutics Conference Call Summary Company Overview - Company: ProQR Therapeutics (NasdaqCM: PRQR) - Event Date: November 03, 2025 - Key Focus: Update on the company's progress, particularly regarding the Axiomer RNA editing platform and the AX-0810 program for cholestatic diseases Core Points and Arguments 1. Strategic Overview: ProQR is at a significant milestone with the authorization of its Clinical Trial Application (CTA) in Europe and the initiation of a Phase I trial for AX-0810, which is expected to generate initial data by the end of the year [4][5][6] 2. Axiomer Platform: The Axiomer RNA editing platform utilizes the body's own editing enzyme, ADAR, to edit RNA without altering DNA, allowing for the repair of mutations and modulation of protein functions [4][5] 3. Pipeline Development: - AX-0810 is the lead program targeting NTCP for cholestatic diseases, with a focus on generating target engagement data [6][9] - Other programs include AX-2402 for Rett syndrome and AX-1412 for cardiovascular disease, indicating a broad therapeutic scope [6][9][49] 4. Partnerships: A notable collaboration with Eli Lilly valued at $3.9 billion focuses on Axiomer RNA editing, enhancing ProQR's development capabilities [5][8][49] 5. Financial Position: ProQR is funded into mid-2027, with approximately EUR 120 million on the balance sheet, providing a runway for multiple clinical milestones [50] Key Developments in AX-0810 1. Mechanism of Action: AX-0810 modulates NTCP to limit bile acid reuptake, addressing the toxic accumulation of bile acids in cholestatic diseases [11][12][13] 2. Target Diseases: - Primary sclerosing cholangitis (PSC) affects over 80,000 adults in the U.S. and Europe, while congenital biliary atresia (BA) impacts around 20,000 children globally [16][19] - Both conditions are severe and life-threatening, with no approved disease-modifying therapies available [16][19] 3. Clinical Trial Design: The Phase I trial will enroll 33 healthy volunteers, with a focus on safety, tolerability, and pharmacokinetics (PK) [39][40] 4. Expected Outcomes: Initial safety and PK data are anticipated by the end of the year, with target engagement data expected in the first half of 2026 [40][46] Important but Overlooked Content 1. Biomarker Assessment: The trial will assess total bile acid levels, bile acid profiles, and the effects of a TUDCA challenge to evaluate NTCP engagement and therapeutic activity [41][42][44] 2. Regulatory Review: The centralized review process for the CTA took longer than expected, reflecting the newness of the process [10] 3. Expert Insights: Professor Henk Jan Verkade provided valuable insights into cholestatic diseases and the relevance of NTCP modulation, emphasizing the high unmet medical need in this area [17][18][36] Conclusion ProQR Therapeutics is positioned for significant advancements in RNA editing therapies, particularly with AX-0810 targeting cholestatic diseases. The company's strong financial backing, innovative platform, and strategic partnerships are key drivers for future growth and value creation.