Biotech and healthcare stocks saw notable activity in Friday's after-hours trading session, with several names posting sharp moves on the back of corporate updates and investor anticipation. Here are the highlights from November 21, 2025.Butterfly Network, Inc. (BFLY) shares climbed in extended trading, rising 6.04% to $2.81 after adding $0.16. At the close, the stock had already surged 14.22% to $2.65. The digital company announced that CEO Joseph DeVivo and Interim CFO Megan Carlson will participate in a ...

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Received CTA authorization for Phase 1 trial of lead program AX-0810, targeting NTCP for cholestatic diseasesInitiating Phase 1 study of AX-0810 in healthy volunteers, with initial safety, tolerability, and PK data from Cohort 1 expected by year-end, and target engagement data from all cohorts in H1 2026€ 106.9 million cash and cash equivalents as of end Q3 – providing runway into mid-2027 LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 06, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR ...

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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.

Agenda Agenda | | | Corporate Outlook Dennis Hom Q&A ENTERING THE CLINIC WITH AX-0810 Establishing safety, PK, and the biomarker roadmap for proof of target engagement November 3, 2025 Strategic Overview Daniel A. de Boer AX-0810 Lead Pipeline Program in Liver Cristina Lopez Lopez Henkjan Verkade Daniel A. de Boer Cristina Lopez Lopez Gerard Platenburg Dennis Hom Cristina Lopez Lopez, MD, PhD Chief Medical Officer Dennis Hom Chief Financial Officer Gerard Platenburg Chief Scientific Officer Speakers Invited ...

Summary of ProQR Therapeutics FY Conference Call Company and Industry Overview - **Company**: ProQR Therapeutics (NasdaqCM:PRQR) - **Industry**: RNA editing and therapeutics, specifically targeting alpha-1 antitrypsin deficiency (AATD) and cholestatic diseases Key Points and Arguments Alpha-1 Antitrypsin Deficiency (AATD) Treatment - ProQR's lead program focuses on AATD, a validated target caused by a single point mutation with measurable biomarkers [2][4] - The company aims to transition patients from a ZZ phenotype (high risk of lung and liver disease) to an MZ phenotype (low risk) through RNA editing [5][6] - Clinical data showed a shift from 0% M protein to 44% after a single dose, with levels reaching 65% in multi-dose cohorts [7][8] - The treatment demonstrated the ability to produce more protein than previously seen with inhibitors, indicating a durable response [9][10] Mechanism of Action - The editing approach corrects mutations at the transcript level, allowing patients to produce functional proteins during acute phase responses [6][9] - The drug is designed to be stable and effective, utilizing GalNAc-conjugation for subcutaneous delivery [8][10] Clinical Development and Future Steps - ProQR is preparing for a 400 mg cohort study in Q1, aiming to assess the durability and efficiency of the editing process [9][10] - The company plans to engage with regulatory agencies to discuss approval thresholds, focusing on the MZ phenotype and its clinical outcomes [16][14] Other Company Insights - ProQR's AX-0810 program targets cholestatic diseases by editing NTCP to block bile acid transport into the liver, with a phase one trial recently cleared to initiate [27][28] - Preclinical data indicated a 15% editing level translating to a two-fold increase in serum bile acids, supporting the therapeutic potential [30] Industry Context and Comparisons - The RNA editing field is evolving rapidly, with ProQR and other companies focusing on GalNAc delivery to the liver [45][46] - The panel discussed the importance of selecting appropriate indications for RNA editing technologies, emphasizing the need for clear clinical benefits [68][69] Challenges and Considerations - There are misconceptions regarding the efficacy of RNA editing compared to DNA editing, particularly concerning the expected levels of protein production [68] - The need for a clear understanding of the relationship between editing levels and therapeutic benefits is crucial for gaining investor confidence [54][68] Conclusion - The conference highlighted the advancements in RNA editing technologies, particularly in treating AATD and cholestatic diseases, with ProQR at the forefront of clinical development [70][72] - The discussion underscored the importance of translating preclinical data to human clinical outcomes and the potential for RNA editing to provide meaningful therapeutic benefits [61][70]

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, an investigational RNA editing oligonucleotide targeting NTCP, aimed at treating cholestatic diseases such as primary sclerosing cholangitis and biliary atresia [1][2] Company Overview - ProQR Therapeutics is focused on developing transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform, which utilizes the body's endogenous ADAR enzymes for RNA editing [1][8] - The company aims to create a new class of medicines for both rare and prevalent diseases with unmet medical needs [8] 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][5] - AX-0810 is the first program from ProQR's Axiomer™ RNA editing pipeline to enter clinical development, with the trial being conducted in the Netherlands [2][5] Upcoming Events - ProQR will host a virtual Investor and Analyst Event on November 3, 2025, to discuss the clinical trial and present findings related to AX-0810 [2][6] - The event will feature presentations from ProQR management and Professor Henkjan J. Verkade, a key opinion leader in pediatric gastroenterology/hepatology [2][3] Research Focus - Professor Verkade's research interests include pediatric cholestatic liver diseases, mechanisms of bile acid transport, and the development of biomarkers and novel therapies for rare inherited liver disorders [4] - He has authored over 300 peer-reviewed publications, contributing significantly to the field [4] Technology Platform - Axiomer™ technology allows for specific single nucleotide changes in RNA, potentially correcting disease-causing mutations and modulating protein expression [6][7] - This innovative approach aims to reduce toxic bile acid accumulation in the liver, addressing the underlying causes of cholestatic diseases [5][7]

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]