ProQR Therapeutics N.V. (NASDAQ:PRQR) earns a spot in our list of the top 10 stocks under $5 that could triple. Growing Optimism About ProQR Therapeutics N.V. (PRQR)’s RNA Editing Platform and Future Clinical Catalysts As of March 19, 2026, analysts remain confident in ProQR Therapeutics N.V. (NASDAQ:PRQR), with 100% maintaining “Buy” ratings. The consensus price target of $9.44 implies a 448.56% upside, reflecting growing optimism about the company’s RNA editing platform and future clinical catalysts. ...

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 25, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will host a virtual Investor and Analyst Event highlighting its pipeline entitled: “Expanding the Axiomer™ RNA Editing Opportunity Beyond AX-0810” Ahead of the anticipated target engagement data for AX-0810 in the first hal ...

Question-and-Answer SessionDennis HomChief Financial Officer Sure. So the company has evolved over time, as you alluded to. But since 2022, we've been exclusively focused on developing our Axiomer technology, which is our version of RNA editing. So the strategy we're taking is fairly straightforward. We're developing our own, wholly-owned pipeline of programs based on Axiomer and we continue to innovate on that technology. I would mention that we are the pioneers of RNA editing. So ADAR-mediated RNA editing ...

Financial Performance - The company reported net losses of €42,184,000, €27,763,000, and €27,735,000 for the years ended December 31, 2025, 2024, and 2023, respectively, with an accumulated deficit of €467,506,000 as of December 31, 2025[40]. - The company has not generated, and does not expect to generate, any product revenue for the foreseeable future, relying on government research grants and collaboration agreements for income[42]. - The company anticipates continuing to incur significant operating losses due to research and development costs associated with its RNA editing platform and product candidates[43]. - The company may face substantial liabilities from product liability claims, which could adversely affect its financial condition and share price[197]. - The company has a risk of losing a material portion of its net operating losses due to regulatory changes, even if it attains profitability[199]. - The trading price of the company's ordinary shares reached a high of $24.99 per share on March 10, 2015, but decreased to as low as $0.56 per share on May 11, 2022[204]. - The company may face potential volatility in its share price due to various factors, including clinical trial results and regulatory actions[204]. - The company has never declared or paid cash dividends on its ordinary shares, and capital appreciation will be the sole source of potential gain for shareholders in the foreseeable future[219]. Research and Development - The company has focused its resources exclusively on the development of its RNA editing platform since August 2022, ceasing clinical development of sepofarsen and ultevursen, which were sold to Laboratoires Théa S.A.S. in December 2023[40]. - The company has allocated significant resources to the phase 1 clinical development of its lead product candidate AX-0810, targeting na-taurocholate cotransporting polypeptide (NTCP) for cholestatic diseases[40]. - The company’s lead program, AX-0810, targets cholestatic diseases and has received clinical trial authorization for a Phase 1 study, with the first healthy volunteer dosed in December 2025[63]. - The development of drug candidates is lengthy, uncertain, and expensive, with a high historical failure rate for product candidates due to various factors[69]. - The company has faced setbacks in its previous clinical trials, including the Phase 2/3 trial of sepofarsen, which did not meet its primary endpoint[59]. - The company may need to conduct additional clinical trials to obtain regulatory approval, which could further delay commercialization[75]. - The company is developing an RNA editing platform using proprietary Axiomer RNA editing technology for genetic disorders with unmet needs, but the scientific research is still preliminary and not clinically validated[78]. Regulatory and Compliance Risks - Regulatory approval processes for product candidates are lengthy and unpredictable, requiring extensive preclinical and clinical trials before marketing applications can be submitted[65]. - The company may incur significant additional costs to comply with potential new SEC climate-related disclosure rules, which could impact operational expenses[57]. - The company is subject to extensive regulations regarding the distribution of biopharmaceutical products, which include record-keeping and licensing requirements[102]. - Non-compliance with healthcare laws could result in significant penalties, including civil and criminal liabilities, which may adversely affect the company's operations and financial results[104]. - The company is subject to complex tax laws and regulations that may change, potentially leading to increased tax liabilities and operational costs[161]. - The company is subject to extensive ongoing regulatory requirements post-approval, which may impose additional costs and operational challenges[151]. - The company may face challenges in international markets due to varying reimbursement and healthcare payment systems, which could restrict its ability to commercialize products[148]. Funding and Capital Requirements - As of December 31, 2025, the company had €92,413,000 in cash and cash equivalents, which is expected to fund operations into mid-2027[49]. - Future capital requirements may vary significantly, and the company may need to seek additional funding sooner than planned due to uncertainties in development timelines[49]. - The company has received funding of $6.8 million from Foundation Fighting Blindness to advance ultevursen into the clinic, with repayments settled upon the sale of ultevursen[47]. Collaborations and Partnerships - The company has entered into a licensing and research collaboration agreement with Lilly, focusing on potential new medicines in the peripheral nervous system and metabolic diseases[87]. - The company announced a new research partnership with the Rett Syndrome Research Trust to develop editing oligonucleotides targeting a genetic variant causing Rett syndrome, included in the pipeline as AX-2402[87]. - The company relies on Théa for the development and commercialization of sepofarsen and ultevursen, and any failure by collaborators could significantly reduce future revenue potential[90]. - Collaborations are subject to risks, including collaborators' discretion in resource allocation and potential delays in clinical trials, which could negatively impact financial results[91]. Intellectual Property Risks - The company faces risks related to intellectual property, including potential litigation that could adversely affect its business and financial performance[110]. - The company must comply with various procedural requirements to maintain patent protection, and failure to do so could result in loss of patent rights[111]. - The company may need to litigate to protect its intellectual property rights, which could be costly and time-consuming[112]. - Third-party claims of patent infringement could lead to significant legal challenges and impact the company's ability to commercialize its products[115]. - Protecting intellectual property rights globally is expensive, and competitors may exploit technologies in jurisdictions without patent protection[121]. Operational Risks - The company faces risks related to dependence on third-party collaborators, which could delay or terminate the development and commercialization of product candidates[87]. - The company relies on third-party manufacturers for clinical and commercial supplies, and any disruptions in supply could hinder product development and commercialization[93]. - Delays in preclinical and clinical studies due to third-party performance issues could have materially adverse effects on the company's financial condition and prospects[96]. - The company faces risks related to outsourcing functions to third parties, including potential misappropriation of proprietary information and limited internal resources to manage these relationships[98]. - The company is dependent on attracting and retaining qualified key management and technical personnel, with increased competition for talent in specialized fields[166]. Market and Competitive Risks - The company faces significant competition from larger entities with more resources, which could impact its ability to develop and commercialize products[126]. - Reimbursement from third-party payors is essential for the commercial success of any approved product, and uncertainty exists regarding coverage and reimbursement levels[127]. - Legislative changes, such as the Affordable Care Act, may negatively impact the company's ability to profitably sell its product candidates[135]. - Market acceptance of product candidates will depend on various factors, including safety, effectiveness, and pricing compared to existing treatments[136]. Cybersecurity and Data Protection Risks - The company faces risks from cybersecurity incidents and data breaches, which could lead to regulatory fines and damage to its reputation[180]. - The company relies on technology systems for data processing and regulatory compliance, making it vulnerable to cyberattacks and data breaches[178]. - The company must comply with various international data protection laws, which may impose additional operational and compliance costs[173]. Economic and Macroeconomic Risks - Unstable macroeconomic conditions may adversely affect the company's business operations and financial performance[210]. - Changes in U.S. federal policies and tariffs could negatively impact the company's business and market conditions[211].

Exhibit 99.1 ProQR Announces Year End 2025 Operating and Financial Results LEIDEN, Netherlands & CAMBRIDGE, Mass., March 12, 2026 – ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2025, and provided a business update. "2025 was a year of meaningful clinical progress for ProQR as we adva ...

Core Insights - ProQR Therapeutics reported significant clinical advancements in 2025, particularly with AX-0810 for cholestatic diseases entering Phase 1 trials, and aims to provide target engagement data in the first half of 2026 [2][3] - The company has strengthened its pipeline with new development candidates AX-2402 for Rett syndrome and AX-2911 for MASH, alongside a successful collaboration with Eli Lilly, achieving $4.5 million in milestones [2][8] Financial Highlights - As of December 31, 2025, ProQR held cash and cash equivalents of approximately €92.4 million, down from €149.4 million in 2024 [11] - The net cash used in operating activities for 2025 was €52.8 million, compared to €36.4 million in 2024 [11] - The net loss for 2025 was €42.2 million, or €0.40 per diluted share, an increase from €27.8 million, or €0.32 per diluted share, in 2024 [12] Clinical Development - AX-0810 is currently in a Phase 1 multiple-dose escalation study in healthy volunteers, with initial safety and pharmacokinetic data reported [6] - The company plans to include a PSC patient cohort in the ongoing Phase 1 trial after completing the healthy volunteer cohorts [6] - AX-2402 has shown promising preclinical results in a Rett syndrome mouse model, with plans to initiate first-in-human clinical testing in the first half of 2027 [6][12] Pipeline Expansion - AX-2402 targets the R270X mutation in MECP2 to restore normal protein function for Rett syndrome [4] - AX-2911 aims to address the I148M mutation in PNPLA3, a significant genetic risk factor for fatty liver disease, demonstrating over 80% reduction in hepatic fat content in preclinical studies [5][7] Strategic Collaborations - The collaboration with Eli Lilly is expected to continue in 2026, with potential data updates and additional milestone payments anticipated [8]

Core Insights - ProQR Therapeutics N.V. is focused on developing transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform [1][4] - The company will present at the Citizens Life Sciences Conference on March 11, 2026, at 3:25pm ET [1] - Axiomer technology enables specific single nucleotide changes in RNA, potentially leading to new medicines for various diseases [3][4] Company Overview - ProQR is dedicated to creating RNA therapies that can reverse mutations or modulate protein expression, addressing both rare and prevalent diseases with unmet medical needs [4] - The Axiomer platform utilizes the ADAR (Adenosine Deaminase Acting on RNA) machinery found in human cells to facilitate precise RNA edits [3][4] - The company aims to grow its pipeline with a focus on patient needs and innovative solutions [4]

Core Viewpoint - ProQR Therapeutics is undergoing changes in its Board composition as part of its commitment to corporate governance and long-term succession planning, with two co-founders rotating off the Board at the upcoming Annual General Meeting [1][2]. Group 1: Board Changes - Dinko Valerio and Alison Lawton will step down from the Board at the next AGM as their terms conclude [1]. - An executive search firm has been engaged to identify new candidates for the Board to support long-term value creation [2]. - The Board regularly reviews its composition to ensure a balance of skills and experience, having appointed two new members in the last three years [3]. Group 2: Axiomer Technology - ProQR is pioneering a next-generation RNA base editing technology called Axiomer, which aims to create a new class of medicines for various diseases [4][5]. - Axiomer Editing Oligonucleotides (EONs) facilitate specific single nucleotide changes in RNA, potentially correcting disease-causing mutations and modulating protein expression [4]. Group 3: Company Mission - ProQR is dedicated to transforming lives through innovative RNA therapies, focusing on unmet medical needs for both rare and prevalent diseases [5].

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]

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].