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 ...

ProQR Therapeutics Conference Call Summary Company Overview - ProQR Therapeutics is a biopharmaceutical company based in the Netherlands, focused on developing RNA editing medicines to treat genetic and common diseases by modifying human messenger RNA [1][2]. Core Technology and Partnerships - The company has developed a proprietary RNA editing technology over the past 10 years and controls all foundational intellectual property [2]. - In 2021, ProQR entered a $4 billion collaboration with Eli Lilly, receiving $125 million upfront and the potential for $250 million in milestone payments for each of the 15 targets [2]. Pipeline and Clinical Programs - ProQR is pursuing a wholly owned pipeline focusing on liver and CNS indications, with the lead program AX-0810 targeting cholestatic diseases [3]. - The company has initiated a clinical study for AX-0810, expecting first human clinical data in the first half of next year [3][11]. Trial Design and Expectations - The AX-0810 trial will enroll 33 healthy volunteers across three cohorts, with a randomized, placebo-controlled, and double-blinded design [10]. - Initial safety and pharmacokinetic data are expected by the end of this year, while pharmacodynamic data will be available in the first half of next year [11]. Unmet Medical Needs - The target diseases, Primary Sclerosing Cholangitis (PSC) and Biliary Atresia (BA), have no approved therapies and represent a significant unmet medical need [7][14]. - The company aims to develop a medicine that addresses this need while generating a robust dataset to validate its technology [8]. Safety and Efficacy Considerations - ProQR believes that pruritus (itching) associated with high bile acid levels is driven by inflammation rather than bile acids themselves, supported by data from healthy individuals with high bile acid levels who do not experience pruritus [17][22]. - The company aims for a twofold increase in bile acid levels in serum to halt disease progression, with a linear correlation between editing percentage and bile acid concentration [23][25]. Future Development Plans - Following the healthy volunteer study, ProQR plans to conduct a Phase 1B study in PSC patients, with results expected before the end of next year [13]. - The company is exploring accelerated development plans for potential approval based on efficacy data [31]. Rett Syndrome Program - ProQR is also developing a program for Rett syndrome, a neurodevelopmental disease caused by the absence of the MECP2 protein, with the potential to restore normal function through RNA editing [34]. - The program is co-financed by the Rett Syndrome Research Trust, and a clinical candidate is expected to be selected soon [35]. Differentiation from Gene Therapy - The RNA editing approach allows for precise restoration of the MECP2 protein without the risk of overexpression, which is a concern in gene therapy [37]. - ProQR plans to target specific mutations, particularly stop codon mutations, which affect approximately 5,000 patients each [38]. Conclusion - ProQR Therapeutics is positioned to address significant unmet medical needs in liver diseases and Rett syndrome through innovative RNA editing technologies, with ongoing clinical trials and strategic partnerships enhancing its development pipeline [1][2][34].

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 01, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary ADAR-mediated Axiomer™ RNA editing technology platform, today announced that the Company will participate in a fireside chat at the 8th Annual Evercore Healthcare Conference on Thursday, December 4, 2025 at 8:20am ET. Webcast details will be accessible from the “Investors & Media” section ...

Core Insights - Biotech and healthcare stocks experienced significant movements in after-hours trading, driven by corporate updates and investor anticipation [1] Company Updates - Butterfly Network, Inc. (BFLY) shares increased by 6.04% to $2.81 after a 14.22% rise to $2.65 at market close, following the announcement of CEO Joseph DeVivo and Interim CFO Megan Carlson's participation in the 8th Annual Evercore Healthcare Conference [2] - Citius Pharmaceuticals Inc. (CTXR) saw a 7.90% increase to $1.23 after a 15.15% rally to $1.14, attributed to a deeper collaboration with Verix for the commercialization of LYMPHIR, an FDA-approved immunotherapy expected to launch in Q4 2025 [3] - ProQR Therapeutics N.V. (PRQR) rose 6.80% to $2.20 after a flat close at $2.06, with no specific news indicating that the movement may be due to investor sentiment [4] - Perrigo Company plc (PRGO) advanced 3.97% to $13.14, building on a 3.10% gain at the close, reflecting steady investor interest despite no new announcements [4] - Tempest Therapeutics, Inc. (TPST) added 4.86% to $3.67 after reversing a decline, following the announcement of plans to acquire CAR-T programs from Factor Bioscience, expected to close in early 2026 [5] - Iterum Therapeutics plc (ITRM) rose 2.77% to $0.3969 after a 4.38% gain at the close, with no new updates released [6] - Processa Pharmaceuticals, Inc. (PCSA) edged up 1.86% to $0.26 despite a steep decline at the close, with no news issued [6]

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Exhibit 99.1 Table of Contents PROQR THERAPEUTICS N.V. Index to Unaudited Condensed Consolidated Financial Statements | PAGE | | | --- | --- | | Unaudited Condensed Consolidated Statement of Financial Position at September 30, 2025 and December 31, | | | 2024 | 1 | | Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income for the | | | Three and Nine Month Periods ended September 30, 2025 and 2024 | 2 | | Unaudited Condensed Consolidated Statement of Changes in Equity for ...

Core Insights - ProQR Therapeutics reported its financial and operational results for Q3 2025, highlighting significant advancements in its RNA editing technology platform, Axiomer, and the initiation of a first-in-human study for its lead program AX-0810 [1][2]. Financial Highlights - As of September 30, 2025, ProQR held cash and cash equivalents of approximately €106.9 million, a decrease from €149.4 million at the end of 2024 [4]. - The net cash used in operating activities for the nine-month period ended September 30, 2025, was €39.4 million, compared to €27.0 million for the same period in 2024 [4][6]. - The net loss for the nine-month period was €33.3 million, or €0.32 per diluted share, compared to a net loss of €18.5 million, or €0.23 per diluted share, for the same period last year [7][16]. Recent Progress and Upcoming Events - The company received CTA authorization for the Phase 1 trial of AX-0810, targeting NTCP for cholestatic diseases, and is initiating the study in healthy volunteers [4][5]. - Initial safety, tolerability, and pharmacokinetic (PK) data from Cohort 1 are expected by year-end 2025, with target engagement data anticipated in the first half of 2026 [5]. - ProQR's Axiomer pipeline includes programs targeting various diseases, such as AX-2402 for Rett syndrome and AX-2911 for fatty liver disease, with updates expected in the coming months [5]. Collaboration and Milestones - The company achieved certain milestones in its collaboration with Eli Lilly, amounting to $2.0 million (~€1.8 million) [6]. - ProQR continues to execute its partnership with Eli Lilly, with potential data updates and milestone income from the ongoing collaboration [5]. Research and Development Costs - Research and development costs for the nine-month period ended September 30, 2025, were €34.8 million, compared to €25.7 million for the same period last year [6]. - General and administrative costs were €11.2 million for the same period, up from €9.7 million in 2024 [6].

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Core Points - ProQR Therapeutics hosted a virtual investor and analyst event to discuss company updates and future prospects [1] Group 1 - The event was led by Sarah Kiely, Vice President of Investor Relations and Corporate Affairs at ProQR Therapeutics [1]

ProQR 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.