PRQR

First clinical validation of Axiomer™ RNA editing established through NTCP modulation with positive AX-0810 clinical target engagement data; full Phase 1 dataset to be presented at a medical or scientific conference later this year Multiple clinical milestones, including initial AX-0811 Phase 1 data expected by year-end 2026, followed in H1 2027 by: Strengthened balance sheet through $59.2 million financing during the quarter; ended Q2 2026 with € 117.1 million cash and cash equivalents, supporting runway through mid-2028 LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 13, 2026 (GLOBE NEWSWIRE) --  ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage 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 second quarter ended June 30, 2026, and provided a business update. “The second quarter marked an important inflection point for ProQR as we established the first clinical validation of our Axiomer RNA editing platform by modulating NTCP with AX-0810,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “We believe these data, together with the significant unmet need that remains in cholestatic liver diseases, including biliary atresia, support the continued advancement of our NTCP franchise. Combined with our recent financing, we are well positioned to deliver a series of important clinical milestones, including presentation of the full AX-0810 Phase 1 dataset later this year, initial Phase 1 data from AX-0811 by year end, initiation of our biliary atresia clinical program and initial data in the first half of 2027, and continued advancement of our MPS I Hurler syndrome and MASH programs. Together, these milestones reflect ProQR’s evolution into a multi-program clinical-stage RNA editing company, strengthen the growing body of evidence supporting the broader potential of our Axiomer platform, and bring us closer to delivering transformative RNA editing therapies for patients.” Recent Progress and Anticipated Upcoming Events First Clinical Validation of Axiomer and Next Milestones with NTCP Franchise ProQR’s NTCP RNA editing approach is designed to modulate NTCP and reduce bile acid uptake directly in hepatocytes, with the goal of reducing intrahepatic cholestasis. In June, ProQR announced positive AX-0810 Phase 1 target engagement data from healthy volunteers, establishing the first clinical validation of the Company’s proprietary Axiomer RNA editing platform. Key findings included: Up to 8-fold increase in total serum bile acids (6 mg/kg) exceeding the Company’s pre-defined 2-fold threshold for meaningful NTCP modulation; Dose-dependent changes in conjugated bile acids, together with concordant changes in total serum bile acids and TUDCA, supporting the therapeutic rationale that modulating NTCP reduces the accumulation of toxic bile acids in the liver; Favorable safety and tolerability profile observed with AX-0810 to date, with no serious adverse events or pruritus reported; pharmacokinetic findings to date support sustained target engagement, including a half-life of eight weeks; and Data supporting advancement into patient study. ProQR plans to present the full dataset from the Phase 1 study of AX-0810, including Cohort 3, at a medical or scientific conference later this year. In July, the Company submitted a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of AX-0811, its next-generation editing oligonucleotide (EON) targeting NTCP for the treatment of cholestatic diseases. AX-0811 is generated by ProQR’s AI-enabled discovery engine and demonstrated higher potency and longer durability than AX-0810 preclinically, which will lead to lower dose levels and less frequent dosing in the clinic. Pending CTA authorization, initial data with AX-0811 is anticipated by year end 2026. ProQR continues preparations for an investigator-initiated trial (IIT) in pediatric biliary atresia in China, with initial clinical data expected in the first half of 2027. Strengthened Financial Position In June, ProQR closed an underwritten registered direct offering of $50.0 million, alongside a concurrent private placement to an existing shareholder and strategic partner Eli Lilly and Company (Lilly) of $9.2 million, for total gross proceeds of approximately $59.2 million. The financing further strengthened the Company’s balance sheet providing runway into mid-2028, and is expected to support the continued advancement of its wholly owned Axiomer RNA editing pipeline, including multiple anticipated clinical milestones. Platform and Research Progress ProQR continues to strengthen the scientific foundation of its Axiomer RNA editing platform through ongoing research and publication of preclinical findings. During the quarter, the Company’s preclinical research describing RNA editing of B4GALT1 was published online as a peer-reviewed Journal Pre-proof in Molecular Therapy – Nucleic Acids. The publication further expands the body of evidence supporting the breadth and versatility of the Axiomer platform beyond the Company’s clinical pipeline. Upcoming anticipated milestones NTCP franchise AX-0810 data from the 9 mg/kg cohort and 12-week follow up from the ongoing Phase 1 study expected by year-end 2026 AX-0811 initial data in healthy volunteers expected by year-end 2026, pending CTA authorization IIT in pediatric biliary atresia in China, with initial data targeted for first half of 2027 Potentially registration-enabling Phase 2 program, subject to regulatory interactions, expected to start in mid-2027 with first interim analysis data expected by mid-2028 Other pipeline candidates AX-0422 for MPS I Hurler Syndrome (IDUA) CTA filing for a first-in-patient study anticipated in early 2027 and initial data expected in the first half of 2027 AX-2911(PNPLA3) FIH IIT in China expected in the first half of 2027 Partnership Continue to execute on Lilly collaboration, with potential data updates and milestone payments Financial Highlights At June 30, 2026, ProQR held cash and cash equivalents of approximately € 117.1 million, compared to € 92.4 million at December 31, 2025. In June 2026, the Company closed an underwritten registered direct offering of 27,624,310 ordinary shares and a concurrent private placement of 5,100,780 ordinary shares with Lilly for gross proceeds totaling approximately $59.2 million. Net cash used in operating activities during the second quarter ended June 30, 2026 was € 12.3 million, compared to € 11.4 million used for the same period in 2025. Research and development (R&D) costs for the quarter ended June 30, 2026 were € 12.7 million, compared to € 11.4 million for the same period last year. General and administrative costs for the quarter ended June 30, 2026 were € 4.9 million, compared to € 4.8 million for the same period in 2025. Net loss for the quarter ended June 30, 2026 was € 8.7 million, or € 0.08 per diluted share, compared to € 12.2 million, or € 0.12 per diluted share, for the same period in 2025. For further financial information for the period ended June 30, 2026, please refer to our Q2 financial report filing available on our website, www.proqr.com under Financials and Filings. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About ProQR ProQR Therapeutics is a clinical-stage company dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. Forward Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical and clinical model data; our initial pipeline targets and the upcoming strategic priorities and milestones related thereto; the continued advancement of our lead development pipeline programs, including ongoing and planned clinical trials; the ongoing Phase 1 clinical study of AX-0810 in NTCP for cholestatic diseases, including the timing and presentation of the full Phase 1 dataset, including Cohort 3 and 12-week follow-up data, and the potential advancement into patient studies; our expectations regarding the safety, tolerability, target engagement and potential therapeutic benefits of AX-0810; our ability to collaborate with investigators to initiate, conduct and recruit for an IIT of our NTCP program in China in pediatric participants with biliary atresia and to generate meaningful data therefrom, including the anticipated timing of initial data readout in H1 2027; the anticipated initiation of our registration-enabling Phase 2 program in mid-2027 with first interim analysis data expected by mid-2028, subject to regulatory interactions; our pipeline targets, including the planned Phase 1 clinical trial of AX-0811 in healthy volunteers; our ability to obtain authorization for, initiate, enroll, and complete a Phase 1 clinical trial for AX-0811 and the anticipated timing of initial data readout by year end 2026, pending CTA authorization; the anticipated first-in-human study of AX-0422 targeting IDUA for MPS I Hurler syndrome, with a CTA filing expected in early 2027 and anticipated initial clinical data readout in H1 2027; the anticipated IIT in China of AX-2911 targeting PNPLA3 for MASH in H1 2027; our expectations regarding clinical updates across multiple programs in 2026 and 2027; the potential design, initiation and timing of a potentially registration-enabling Phase 2 program for the NTCP franchise, subject to regulatory interactions; the therapeutic potential and development timeline regarding AX-0810, AX-0811, AX-0422, and AX-2911;our participation at upcoming scientific conferences; the continued development and advancement of our Axiomer platform; the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates; the timing, progress and results of our preclinical studies and other development and pipeline activities, including the release of data related thereto; our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development; the potential of our technologies and product candidates; the collaboration with Lilly and the intended benefits thereof, including timing for data updates, and potential milestones; our ability to selectively form new partnerships and enter into future collaborations; our financial position and expected cash-runway to fund our operations through mid 2028. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, risks and uncertainties associated with conducting IITs in China, including evolving regulatory requirements, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted due to shortage and pressure on supply chains and logistics in the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and our reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical events and conflicts, inflationary pressures, fluctuating interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor and media contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 [email protected] contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 [email protected] PROQR THERAPEUTICS N.V.Unaudited Interim Condensed Consolidated Statement of Financial Position PROQR THERAPEUTICS N.V.Unaudited Interim Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income(€ in thousands, except share and per share data) For these periods the potential exercise of share options is not included in the diluted earnings per share as the Company was loss-making. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal. PROQR THERAPEUTICS N.V.Unaudited Interim Condensed Consolidated Statement of Changes in Equity PROQR THERAPEUTICS N.V.Unaudited Interim Condensed Consolidated Statement of Cash Flows

Kamada (KMDA) came out with quarterly earnings of $0.07 per share, missing the Zacks Consensus Estimate of $0.12 per share. This compares to earnings of $0.07 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -40.02%. A quarter ago, it was expected that this biopharmaceutical would post earnings of $0.09 per share when it actually produced earnings of $0.06, delivering a surprise of -33.33%. Over the last four quarters, the company has surpassed consensus EPS estimates just once. Kamada, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $45.24 million for the quarter ended March 2026, missing the Zacks Consensus Estimate by 2.63%. This compares to year-ago revenues of $44.02 million. The company has topped consensus revenue estimates just once over the last four quarters. The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call. Kamada shares have added about 19.4% since the beginning of the year versus the S&P 500's gain of 8.1%. While Kamada has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately. Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions. Ahead of this earnings release, the estimate revisions trend for Kamada was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is $0.12 on $50.13 million in revenues for the coming quarter and $0.52 on $201.77 million in revenues for the current fiscal year. Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the bottom 41% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1. Another stock from the same industry, ProQR (PRQR), has yet to report results for the quarter ended March 2026. This Dutch drugmaker is expected to post quarterly loss of $0.15 per share in its upcoming report, which represents a year-over-year change of -36.4%. The consensus EPS estimate for the quarter has been revised 7% lower over the last 30 days to the current level. ProQR's revenues are expected to be $5.53 million, up 10.7% from the year-ago quarter. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Kamada Ltd. (KMDA) : Free Stock Analysis Report ProQR Therapeutics N.V. (PRQR) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

AX-0810 target engagement data in healthy volunteers on track for Q2 2026; biliary atresia selected as initial Phase 2 indication Pipeline expansion with advancement of AX-0811 (NTCP) and AX-0422 (IDUA) toward the clinic, supporting multiple upcoming clinical catalysts Continued advancement of Axiomer platform, including AI-enabled discovery capabilities, partnership with Ginkgo Bioworks, and formation of AI Advisory Board Ended Q1 2026 with € 81.1 million cash and cash equivalents, supporting runway into mid-2027 LEIDEN, Netherlands & CAMBRIDGE, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage 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 quarter ended March 31, 2026, and provided a business update. “We are on track to deliver multiple clinical readouts within our current runway, including target engagement data for AX-0810 in the second quarter,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “We have refined our initial development strategy for AX-0810 to prioritize biliary atresia, where we believe we can generate the most meaningful clinical data in this target patient population, while maintaining flexibility to explore additional indications, including PSC, as the program advances. We also continue to advance our platform more broadly to bring RNA editing therapies to patients.” Recent Progress and Anticipated Upcoming Events AX-0810 Advancing Toward Target Engagement Data AX-0810, ProQR’s lead RNA editing program targeting NTCP, remains on track to report target engagement data from healthy volunteers in the second quarter of 2026. The Company has selected biliary atresia (BA) as the initial indication for Phase 2 development, based on strong biological rationale and high unmet need. As part of this strategy, ProQR plans to conduct an investigator-initiated trial (IIT) in pediatric participants with BA in China instead of the previously planned adult participant PSC cohort in its Phase 1 program. While PSC remains of interest as a potential setting for broader development, the Company is prioritizing a BA-focused IIT to provide a more direct path to early, data generation in the target patient population, prior to initiation of a Phase 2 study. ProQR is working with investigators to initiate the IIT, with initial clinical data targeted for H1 2027. Pipeline Expansion and Upcoming Catalysts ProQR continues to expand its pipeline, with multiple programs advancing toward the clinic: AX-0811, a next-generation NTCP program for cholestatic diseases generated using ProQR’s AI-enabled discovery engine, with a clinical trial application (CTA) filing expected in mid-2026 and initial clinical data anticipated by year-end 2026; AX-0422, targeting IDUA for Hurler syndrome, with a CTA filing expected in early 2027 and initial clinical data targeted in the first half of 2027; AX-2911, targeting PNPLA3 for MASH, anticipating a first-in-human (FIH) IIT in China in the first half of 2027. Scientific Conference Presentations ProQR is presenting multiple presentations at scientific conferences in May, including TIDES USA, the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, the RNA Society Annual Meeting, and the European Association for the Study of the Liver (EASL) Congress. Collectively, these presentations expand the body of evidence supporting the breadth and versatility of the Company’s Axiomer RNA editing platform including applications across multiple tissues and disease areas. Advancing the Axiomer™ Platform ProQR continues to enhance its Axiomer platform through the integration of AI-enabled discovery and high-throughput screening to accelerate the design and optimization of RNA editing therapeutics. During the quarter, the Company announced a strategic partnership with Ginkgo Bioworks, providing access to Ginkgo’s autonomous laboratory infrastructure to enable high-throughput data generation. This capability is expected to increase the scale and speed of experimental data generation, supporting more efficient discovery timelines and improving predictive performance of ProQR’s AI models. In addition, ProQR established an AI Advisory Board comprising leaders from industry and academia to support the Company’s AI strategy. The Advisory Board provides guidance on best practices, emerging technologies, and applications of AI in drug discovery, helping to further advance Axiomer-based innovation. Board Nomination and Annual General Meeting Dr. Lykke Hinsch Gylvin, Chief Medical Officer and Head of Global Medicine at Boehringer Ingelheim, has been nominated for appointment to the ProQR Board of Directors, subject to shareholder approval at the upcoming Annual General Meeting, scheduled for June 2, 2026. Dr. Hinsch Gylvin is a seasoned pharmaceutical executive with more than 20 years of global leadership experience spanning all phases of drug development in a broad range of therapeutic areas. Summary of Anticipated Milestones AX-0810 (NTCP) Phase 1 target engagement data in healthy volunteers, second quarter of 2026 IIT in pediatric biliary atresia in China, with initial data targeted for first half of 2027 AX-0811 (NTCP, next gen Axiomer) CTA filing in mid 2026, with initial data in healthy volunteers by year-end 2026 AX-0422 (IDUA) CTA filing in early 2027, with initial data in patients targeted for the first half of 2027 AX-2911(PNPLA3) FIH IIT in the first half of 2027 Continue to execute on Lilly collaboration, with potential data updates and milestone payments Financial Highlights At March 31, 2026, ProQR held cash and cash equivalents of approximately € 81.1 million, compared to € 92.4 million at December 31, 2025. Net cash used in operating activities during the first quarter ended March 31, 2026 was € 11.1 million, compared to € 15.8 million used for the same period in 2025. Research and development (R&D) costs for the quarter ended March 31, 2026 were € 11.8 million, compared to € 12.3 million for the same period last year. General and administrative costs for the quarter ended March 31, 2026 were € 3.9 million, compared to € 3.2 million for the same period in 2025. Net loss for the quarter ended March 31, 2026 was € 13.4 million, or € 0.13 per diluted share, compared to € 10.1 million, or € 0.10 per diluted share, for the same period in 2025. For further financial information for the period ended March 31, 2026, please refer to our Q1 financial report filing available on our website, www.proqr.com under Financials and Filings. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. Forward Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical and clinical model data; our initial pipeline targets and the upcoming strategic priorities and milestones related thereto; the continued advancement of our lead development pipeline programs, including ongoing and planned clinical trials; the ongoing Phase 1 clinical study of AX-0810 in NTCP for cholestatic diseases, including the anticipated timing of initial Phase 1 target engagement data from the first cohort of healthy volunteers in the second quarter of 2026; our expectations regarding the safety and therapeutic benefits of AX-0810, including the planned dosing levels and their efficacy; our ability to collaborate with investigators to execute and recruit for an investigator-initiated trial of AX-0810 in China in pediatric participants with biliary atresia and to generate meaningful data therefrom, including the anticipated timing of initial data readout in H1 2027; risks and uncertainties associated with conducting clinical trials in China, including evolving regulatory requirements; our pipeline targets, including the planned Phase 1 clinical trial of AX-0811 for cholestatic diseases; our ability to recruit for and complete a Phase 1 clinical trial for AX-0811, including the anticipated timing of a CTA filing in mid 2026 and initial data readout by year end 2026; the anticipated first-in-human study of AX-0422 targeting IDUA for Hurler syndrome, with a CTA filing expected in early 2027 and anticipated initial clinical data readout in H1 2027; the anticipated investigator-initiated study in China of AX-2911 targeting PNPLA3 for MASH in H1 2027; our expectations regarding clinical updates across multiple programs in 2026 and 2027; the therapeutic potential and development timeline regarding AX-0810, AX-0811, AX-0422, and AX-2911; the anticipated benefits from our partnership with Ginkgo Bioworks; the role and expected contributions of our AI Advisory Board; our participation at upcoming scientific conferences; the continued development and advancement of our Axiomer platform; the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates; the timing, progress and results of our preclinical studies and other development and pipeline activities, including the release of data related thereto; our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development; the potential of our technologies and product candidates; the collaboration with Lilly and the intended benefits thereof, including timing for data updates, potential milestones, exercise of an option to expand targets and the receipt of an opt-in payment; our ability to selectively form new partnerships and enter into future collaborations; our financial position and cash-runway to fund our operations into mid 2027, and the anticipated changes to our Board composition, including the nomination of Dr. Lykke Hinsch Gylvin, subject to shareholder approval. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted due to shortage and pressure on supply chains and logistics in the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and our reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical events and conflicts, inflationary pressures, fluctuating interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor and media contact: Sarah Kiely ProQR Therapeutics N.V. T: +1 617 599 6228 [email protected] or Investor contact: Peter Kelleher LifeSci Advisors T: +1 617 430 7579 [email protected] PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Financial Position PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income (€ in thousands, except share and per share data) PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Changes in Equity PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Cash Flows

Wall Street expects a year-over-year decline in earnings on higher revenues when ProQR (PRQR) reports results for the quarter ended March 2026. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates. The earnings report might help the stock move higher if these key numbers are better than expectations. On the other hand, if they miss, the stock may move lower. While management's discussion of business conditions on the earnings call will mostly determine the sustainability of the immediate price change and future earnings expectations, it's worth having a handicapping insight into the odds of a positive EPS surprise. This Dutch drugmaker is expected to post quarterly loss of $0.15 per share in its upcoming report, which represents a year-over-year change of -36.4%. Revenues are expected to be $5.53 million, up 10.8% from the year-ago quarter. The consensus EPS estimate for the quarter has been revised 6.98% lower over the last 30 days to the current level. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period. Investors should keep in mind that the direction of estimate revisions by each of the covering analysts may not always get reflected in the aggregate change. Price, Consensus and EPS Surprise Estimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. This insight is at the core of our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction). The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier. Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only. A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP. Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell). For ProQR, the Most Accurate Estimate is lower than the Zacks Consensus Estimate, suggesting that analysts have recently become bearish on the company's earnings prospects. This has resulted in an Earnings ESP of -20.00%. On the other hand, the stock currently carries a Zacks Rank of #3. So, this combination makes it difficult to conclusively predict that ProQR will beat the consensus EPS estimate. Analysts often consider to what extent a company has been able to match consensus estimates in the past while calculating their estimates for its future earnings. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number. For the last reported quarter, it was expected that ProQR would post a loss of$0.08 per share when it actually produced a loss of -$0.09, delivering a surprise of -12.50%. The company has not been able to beat consensus EPS estimates in any of the last four quarters. An earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss. That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported. ProQR doesn't appear a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release. Another stock from the Zacks Medical - Biomedical and Genetics industry, Arvinas, Inc. (ARVN), is soon expected to post loss of $0.95 per share for the quarter ended March 2026. This estimate indicates a year-over-year change of -183.3%. Revenues for the quarter are expected to be $15.72 million, down 91.7% from the year-ago quarter. The consensus EPS estimate for Arvinas has been revised 3.3% lower over the last 30 days to the current level. However, a lower Most Accurate Estimate has resulted in an Earnings ESP of -1.32%. When combined with a Zacks Rank of #3 (Hold), this Earnings ESP makes it difficult to conclusively predict that Arvinas will beat the consensus EPS estimate. Over the last four quarters, the company surpassed consensus EPS estimates three times. Stay on top of upcoming earnings announcements with the Zacks Earnings Calendar. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report ProQR Therapeutics N.V. (PRQR) : Free Stock Analysis Report Arvinas, Inc. (ARVN) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

AX-0810 Phase I target engagement data due this quarter — ProQR expects readout from its healthy-volunteer study later this quarter after first-cohort results showed favorable safety and PK, with a planned PSC patient cohort and Phase II targeted at biliary atresia to follow. AX-0811 is a next‑generation NTCP candidate driven by AI — ProQR says its AI-enabled discovery cut timelines dramatically and produced AX-0811 with much higher editing efficiency (~60% in humanized mice); management indicated a CTA and early clinical data are planned (timing described variably across presentations). New programs and funding runway — ProQR regained rights to AX-0422 for Hurler syndrome (CTA enabling now; CTA expected early 2027 with initial data H1 2027) and is advancing AX-2911 for PNPLA3-driven MASH via a China IIT, and the company says it is funded into mid-2027. Interested in ProQR Therapeutics N.V.? Here are five stocks we like better. ProQR Therapeutics (NASDAQ:PRQR) outlined multiple pipeline updates and development priorities during its virtual investor and analyst event, highlighting upcoming clinical readouts for its lead RNA editing program AX-0810, a next-generation follow-on candidate (AX-0811), and the addition of two programs, AX-0422 for Hurler syndrome and AX-2911 for metabolic dysfunction-associated steatohepatitis (MASH). Chief Executive Officer Daniel de Boer said ProQR remains on track to report target engagement data from its ongoing Phase I healthy volunteer study of AX-0810 “later this quarter,” describing it as a key milestone designed to demonstrate engagement on bile acid transport. Chief Medical Officer Dr. Cristina López-López said initial data from the first cohort showed a “favorable safety and tolerability profile,” with pharmacokinetics consistent with non-clinical observations. → Levi Strauss Gains as DTC Continues to Fuel Revenue Growth AX-0810 targets NTCP, a bile acid transporter responsible for uptake from the bloodstream into the liver. López-López said AX-0810 is designed to convert wild-type NTCP into the protective Q68R variant to limit bile acid re-uptake into hepatocytes, aiming to reduce toxic bile acid accumulation that drives cholestatic liver diseases. The Phase I study’s biomarker-based target engagement strategy includes three readouts, according to López-López: Total bile acids in serum (with a two-fold increase described as a meaningful indicator of NTCP modulation) Bile acid profile, with focus on changes in conjugated bile acids A TUDCA challenge test as a functional readout of NTCP-mediated transport → Why PriceSmart’s Discount May Not Last Much Longer In the Q&A, López-López clarified that the two-fold threshold for total bile acids would be evaluated “both compared to baseline as well as compared to placebo.” Management also emphasized the trial remains double-blind and placebo-controlled, and the company has not reviewed unblinded data. Following healthy volunteer assessments, López-López said ProQR plans to open a patient cohort in primary sclerosing cholangitis (PSC), with data expected “at the end of this year.” She said the PSC cohort is intended to help bridge translation from healthy volunteers to disease context and inform dose frequency and expected effect size as the company advances toward pediatric development in biliary atresia. → 3 Surprising S&P 500 Outperformers of 2026 López-López announced ProQR has selected biliary atresia as the initial indication for Phase II development of AX-0810. She described biliary atresia as a severe pediatric cholestatic disease affecting “approximately 20,000 patients worldwide,” with no approved pharmacological therapies that modify disease progression. She added that biliary atresia accounts for “approximately 45% of all pediatric liver transplants,” and that “60%-80% of patients ultimately require liver transplantation before adulthood” even with Kasai portoenterostomy. In response to analyst questions, López-López said the company is “not moving away from PSC,” but is prioritizing biliary atresia based on the combination of unmet need, biological clarity, and development feasibility. On development strategy, she said the company expects to pursue an accelerated pathway using liver biomarkers and measures of liver health and stiffness, with longer-term follow-up as a post-market commitment, pending regulatory discussions. She also noted ProQR expects to treat “different stages of the disease post Kasai procedure,” with Phase II design details still to come. De Boer and management positioned AX-0811 as a next-generation exomer editing oligonucleotide targeting NTCP, generated using ProQR’s AI-enabled discovery engine. De Boer said ProQR expects to file a CTA for AX-0811 in “mid-2026,” with initial clinical data expected “by the end of this year.” (Later in the event, management also described a CTA filing “this year” with initial healthy-volunteer data before year-end.) López-López highlighted platform-driven improvements, stating ProQR’s AI-driven high-throughput screening model has reduced discovery timelines from up to three years to roughly six months, and can produce editing oligonucleotides with up to sixfold increases in editing efficiency compared to earlier designs. For AX-0811 specifically, she said humanized mouse models showed editing efficiencies “around 60%,” a roughly threefold increase versus the prior generation, with improved stability. In Q&A, management attributed gains to AI-guided optimization across factors including sequence length and chemical modifications, without disclosing specifics. ProQR introduced AX-0422 (also referred to as AXS-422 during the event), an RNA editing therapy targeting IDUA for Hurler syndrome (MPS I). De Boer said the program originated from internal discovery and was advanced under the company’s partnership with Eli Lilly, but following a portfolio review ProQR will regain full rights, allowing it to advance AX-0422 as a wholly owned asset. De Boer said the broader Lilly collaboration continues “without any change in priority,” with multiple programs progressing. López-López said AX-0422 is designed to correct the Trp402X nonsense variant—described as the most common severe mutation in IDUA—back to wild type at the RNA level. She presented preclinical data showing approximately 20% of wild-type IDUA enzymatic activity restored in liver at eight weeks after subcutaneous delivery, along with dose-dependent reductions in glycosaminoglycans (GAGs), including “almost 90%” reduction in urine versus control. She also cited head-to-head animal model data comparing RNA editing to enzyme replacement therapy (ERT), describing urinary GAG reductions of “over 90%” with RNA editing versus “around 60%” with ERT, and improvement in a motor skill test. Beyond systemic effects, López-López said preclinical brain delivery showed restoration of enzymatic activity across multiple brain regions. Management described a two-step clinical approach: liver-directed editing first, followed by intrathecal administration to address CNS manifestations. In Q&A, López-López said ProQR anticipates intrathecal dosing “once every 9-12 months” and subcutaneous dosing “once every 3-6 months,” noting CNS dosing durability is generally longer than liver residence time for oligonucleotides. On timing, management said CTA-enabling activities are ongoing, with a CTA filing expected in early 2027 and initial clinical data anticipated in the first half of 2027. Gerard Platenburg, ProQR’s Chief Scientific Officer, said the literature suggests restoration of approximately “1%-15%” of normal IDUA enzymatic function can improve disease phenotype, and said the company observed ~21% restoration associated with near normalization of urinary GAGs in its data. ProQR also highlighted AX-2911, targeting the PNPLA3 I148M variant, which López-López called the “strongest known genetic risk factor” for liver disease progression in MASH and said is carried by about half of MASH patients. She estimated homozygous carriers represent “approximately 8 million individuals in the U.S. and EU.” Discussing preclinical proof-of-concept, López-López said ProQR used a humanized model incorporating primary human hepatocytes carrying the homozygous variant. In the comparison presented, an antisense oligonucleotide (ASO) knockdown approach achieved ~90% PNPLA3 mRNA knockdown and a 36% reduction in lipid droplets, while AX-2911 achieved 23% RNA editing with an approximately 80% reduction in lipid droplets. Platenburg said ProQR believes correcting the mutation to restore a functional protein is a key differentiator versus knockdown. On development strategy, López-López said ProQR declared a development candidate earlier this year and plans to advance AX-2911 through an investigator-initiated trial (IIT) in China to generate early clinical data while maintaining “capital discipline and flexibility.” She said the study is designed to assess safety, pharmacokinetics, and exploratory biomarkers related to liver fat and disease activity. In closing remarks, management reiterated expectations for multiple data catalysts within the company’s cash runway, stating it remains funded into “mid-2027,” consistent with prior guidance. De Boer also highlighted platform initiatives, including an AI model built over the last 18 months, a strategic partnership with Ginkgo Bioworks to enable roboticized high-throughput data generation, and the creation of an AI advisory board to support AI and machine learning efforts. ProQR Therapeutics N.V. is a clinical-stage biotechnology company dedicated to developing RNA-based therapies for severe genetic diseases. Utilizing its proprietary Axiomer® RNA editing platform, ProQR aims to correct disease-causing mutations directly at the messenger RNA level. The company's pipeline features several investigational candidates, including sepofarsen (formerly QR-110) for Leber congenital amaurosis type 10, QR-421a targeting Usher syndrome and certain forms of retinitis pigmentosa, and QR-313 for dystrophic epidermolysis bullosa. Founded in 2012 and headquartered in Leiden, the Netherlands, ProQR maintains a significant presence in Cambridge, Massachusetts, to support its clinical research and regulatory initiatives. The article "ProQR Therapeutics Unveils New RNA Editing Programs; AX-0810 Phase 1 Target Data Due This Quarter" was originally published by MarketBeat.

Advancing AX-0810 through ongoing Phase 1 dosing, with target engagement data expected 1H 2026 Selected Development Candidates AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3) Ended 2025 with € 92.4 million cash and cash equivalents and achieved $4.5 million in Eli Lilly collaboration milestones in 2025, supporting runway into mid-2027 LEIDEN, Netherlands & CAMBRIDGE, Mass., March 12, 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 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 advanced AX-0810 for Cholestatic Diseases into a Phase 1 trial and continued to expand the reach of our Axiomer RNA editing platform and pipeline,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “With target engagement data for AX-0810 expected in the first half of 2026, we are approaching an important milestone for the Company and an important step toward bringing a new treatment closer to patients with cholestatic diseases.” De Boer continued, “In parallel to advancing AX-0810, we strengthened our pipeline with the selection of Development Candidates for AX-2402 in Rett syndrome and AX-2911 in MASH, and continued to advance our collaboration with Lilly, achieving $4.5 million in milestones during the year. As we enter 2026, our focus remains on delivering the AX-0810 target engagement data in the first half the year and advancing our innovative RNA editing therapies for patients with significant unmet need.” Recent Progress and Anticipated Upcoming Events AX-0810 – Phase 1 Progress and Upcoming Target Engagement Data AX-0810 uses Axiomer’s ability to modulate protein function by editing NTCP RNA for the treatment of cholestatic diseases, such as primary sclerosing cholangitis (PSC) and biliary atresia. AX-0810 is in a Phase 1 multiple-dose escalation study ongoing in healthy volunteers in the Netherlands. ProQR reported encouraging initial 4-week safety and pharmacokinetic data from the first cohort of healthy volunteers. Target engagement data in healthy volunteer cohorts is on track for the first half of 2026. Preparations are underway to include a PSC patient cohort in the ongoing Phase 1 trial, following completion of healthy volunteer cohorts. Advancing the Broader Axiomer Pipeline AX-2402 applies Axiomer RNA editing to correct disease-causing mutations, targeting the R270X nonsense mutation in MECP2 to restore normal protein function for the treatment of Rett syndrome. The Company has selected a Development Candidate based on a robust preclinical profile. Non-clinical proof-of-concept data demonstrated statistically significant and clinically relevant functional improvements in a Rett syndrome mouse model. These effects included improvement in cumulative Bird score, driven in part by robust improvements in hindlimb clasping score. Development activities are underway with the objective to initiate first-in-human clinical testing in the first half of 2027. Development of AX-2402 is supported by funding of up to $9.2 million from the Rett Syndrome Research Trust. AX-2911 leverages Axiomer’s ability to modulate protein activity by editing the I148M mutation in PNPLA3, the strongest known genetic risk factor for fatty liver disease. AX-2911 is positioned as a potentially disease-modifying approach for genetically defined MASH populations. A Development Candidate was selected following strong preclinical proof-of-concept data demonstrating >80% reduction in hepatic fat content in a humanized PNPLA3-148M mouse model in head-to-head comparison studies. Strategic Collaboration with Eli Lilly ProQR’s collaboration with Eli Lilly and Company (Lilly) continued to progress in 2025, resulting in $4.5 million in milestones achieved during the year. The Company expects continued execution under the partnership in 2026, including potential data updates and additional milestone payments. Planned Changes to Board Composition In February, the Company announced planned changes to its Board composition. Dinko Valerio, co-founder of the Company, and Alison Lawton will rotate off the Board at the Company’s next Annual General Meeting as their terms conclude. An executive search firm has been engaged to identify new Board candidates aligned with ProQR’s continued clinical-stage evolution and long-term strategy. Summary of Anticipated Upcoming Milestones Report AX-0810 Phase 1 target engagement data in healthy volunteers (1H 2026) Initiate patient cohort in AX-0810 Phase 1 trial following healthy volunteers Advance AX-2402 for Rett syndrome to first-in-human study (1H 2027) Disclose additional preclinical data across earlier-stage programs Continue to execute on Lilly collaboration, with potential data updates and milestone payments Financial Highlights At December 31, 2025, ProQR held cash and cash equivalents of approximately € 92.4 million, compared to € 149.4 million at December 31, 2024. Net cash used in operating activities during the full year ended December 31, 2025 was € 52.8 million, compared to € 36.4 million used for the same period in 2024. During 2025, the Company achieved certain milestones in the collaboration agreement with Lilly amounting to $4.5 million (~€ 3.9 million). Research and development (R&D) costs for the year ended December 31, 2025 were € 44.7 million, compared to € 36.4 million for the same period last year. General and administrative costs for the year ended December 31, 2025 were € 15.1 million, compared to € 13.7 million for the same period in 2024. Net loss for the year ended December 31, 2025 was € 42.2 million, or € 0.40 per diluted share, compared to € 27.8 million, or € 0.32 per diluted share, for the year ended December 31, 2024. For further financial information for the period ended December 31, 2025, please refer to our 2025 Annual Report on Form 20-F and our Statutory Annual Report, which will be available on our website, www.proqr.com under Financials and Filings. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. Forward Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical and clinical model data; our initial pipeline targets and the upcoming strategic priorities and milestones related thereto; the continued advancement of our lead development pipeline programs, including ongoing and planned clinical trials; the ongoing Phase 1 clinical study of AX-0810 in NTCP for cholestatic diseases, including the anticipated timing of initial Phase 1 target engagement data from the first cohort of healthy volunteers in the first half of 2026, our ability to recruit for and complete a Phase 1 clinical trial for AX-0810 in healthy volunteers, and our expectations regarding initiation of the planned PSC patient cohort following completion of healthy volunteer cohorts; our expectations regarding the safety and therapeutic benefits of AX-0810, including the planned dosing levels and their efficacy; the continued development and advancement of our Axiomer platform; the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates; the timing, progress and results of our preclinical studies and other development and pipeline activities, including the release of data related thereto; our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development; the potential of our technologies and product candidates; the anticipated advancement of AX-2402 for Rett syndrome to first-in-human study in the first half of 2027; the collaboration with Lilly and the intended benefits thereof, including timing for data updates, potential milestones, exercise of an option to expand targets and the receipt of an opt-in payment; our ability to selectively form new partnerships and enter into future collaborations; our financial position and cash-runway, and the anticipated changes to our Board composition. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical events and conflicts, high inflation, rising interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor and media contact: Sarah Kiely ProQR Therapeutics N.V. T: +1 617 599 6228 [email protected] or Investor contact: Peter Kelleher LifeSci Advisors T: +1 617 430 7579 [email protected]

Kamada (KMDA) came out with quarterly earnings of $0.06 per share, missing the Zacks Consensus Estimate of $0.09 per share. This compares to earnings of $0.07 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -30.80%. A quarter ago, it was expected that this biopharmaceutical would post earnings of $0.1 per share when it actually produced earnings of $0.09, delivering a surprise of -10%. Over the last four quarters, the company has surpassed consensus EPS estimates just once. Kamada, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $44.68 million for the quarter ended December 2025, missing the Zacks Consensus Estimate by 2.29%. This compares to year-ago revenues of $39.01 million. The company has topped consensus revenue estimates two times over the last four quarters. The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call. Kamada shares have added about 26.9% since the beginning of the year versus the S&P 500's decline of 0.9%. While Kamada has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately. Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions. Ahead of this earnings release, the estimate revisions trend for Kamada was favorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #2 (Buy) for the stock. So, the shares are expected to outperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is $0.11 on $46.21 million in revenues for the coming quarter and $0.48 on $200.56 million in revenues for the current fiscal year. Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the bottom 42% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1. ProQR (PRQR), another stock in the same industry, has yet to report results for the quarter ended December 2025. This Dutch drugmaker is expected to post quarterly loss of $0.08 per share in its upcoming report, which represents a year-over-year change of +20%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. ProQR's revenues are expected to be $8.55 million, up 86.2% from the year-ago quarter. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Kamada Ltd. (KMDA) : Free Stock Analysis Report ProQR Therapeutics N.V. (PRQR) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

Puma Biotech (PBYI) came out with quarterly earnings of $0.29 per share, beating the Zacks Consensus Estimate of $0.24 per share. This compares to earnings of $0.43 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of +20.83%. A quarter ago, it was expected that this biopharmaceutical company would post earnings of $0.09 per share when it actually produced earnings of $0.21, delivering a surprise of +133.33%. Over the last four quarters, the company has surpassed consensus EPS estimates four times. Puma Biotech, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $75.5 million for the quarter ended December 2025, surpassing the Zacks Consensus Estimate by 11.26%. This compares to year-ago revenues of $59.1 million. The company has topped consensus revenue estimates four times over the last four quarters. The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call. Puma Biotech shares have added about 29.1% since the beginning of the year versus the S&P 500's gain of 1.5%. While Puma Biotech has outperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock? There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately. Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions. Ahead of this earnings release, the estimate revisions trend for Puma Biotech was unfavorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #4 (Sell) for the stock. So, the shares are expected to underperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is $0.11 on $48.02 million in revenues for the coming quarter and $0.55 on $235.69 million in revenues for the current fiscal year. Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 36% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1. Another stock from the same industry, ProQR (PRQR), has yet to report results for the quarter ended December 2025. This Dutch drugmaker is expected to post quarterly loss of $0.08 per share in its upcoming report, which represents a year-over-year change of +20%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. ProQR's revenues are expected to be $8.55 million, up 86.2% from the year-ago quarter. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Puma Biotechnology, Inc. (PBYI) : Free Stock Analysis Report ProQR Therapeutics N.V. (PRQR) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

Received CTA authorization for Phase 1 trial of lead program AX-0810, targeting NTCP for cholestatic diseases Initiating 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), 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 third quarter ended September 30, 2025, and provided a business update. “Last month we achieved an important milestone with the CTA authorization and are initiating our first-in-human study for AX-0810, marking a significant step forward for both this program and our Axiomer RNA editing platform,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “This study represents the first clinical evaluation of an Axiomer editing oligonucleotide and positions us to deliver initial safety and PK data before year-end, followed by target engagement data in the first half of 2026. Supported by our strong cash position providing runway into mid-2027, we continue to advance our broader pipeline, platform, and partnership activities, executing on our strategy to deliver transformative RNA editing therapies for diseases with high unmet need.” Recent Progress and Anticipated Upcoming Events In November, the Company hosted a virtual analyst and investor event focused on AX-0810, highlighting the Phase 1 trial design and target engagement/biomarker strategy. AX-0810 uses Axiomer’s ability to modulate protein function by targeting NTCP for the treatment of cholestatic diseases. In October AX-0810 received CTA authorization and a first-in-human (FIH) Phase 1 study is being initiated in healthy volunteers. Initial safety, tolerability, and PK data for Cohort 1 is expected toward the end of 2025 with target engagement data for all healthy volunteer cohorts in the first half of 2026. In parallel, activities are underway to include a patient cohort in this FIH following completion of the healthy volunteer cohorts. Beyond AX-0810, ProQR’s Axiomer pipeline demonstrates the broad potential of RNA editing across three key applications – modulate, correct, and protect – with updates expected across these earlier-stage programs in the coming months. AX-2402 applies Axiomer RNA editing to correct disease-causing mutations, targeting the R270X nonsense mutation in MECP2 to restore normal protein function for the treatment of Rett syndrome. AX-2911 leverages Axiomer’s ability to modulate protein activity by editing the I148M mutation in PNPLA3, the strongest known genetic risk factor for fatty liver disease. AX-1412 demonstrates Axiomer’s protective application, mimicking a naturally occurring B4GALT1 variant associated with significantly reduced cardiovascular risk. In October, the Company presented at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting in Budapest, highlighting applications of its Axiomer RNA editing platform technology. ProQR continues to execute on partnership with Eli Lilly and Company (Lilly), with potential data updates and milestone income from the ongoing collaboration. Financial Highlights At September 30, 2025, ProQR held cash and cash equivalents of approximately € 106.9 million, compared to € 149.4 million at December 31, 2024. Net cash used in operating activities during the nine-month period ended September 30, 2025 was € 39.4 million, compared to € 27.0 million used for the same period last year. During the first nine months of 2025, the Company achieved certain milestones in the collaboration agreement with Eli Lilly amounting to $2.0 million (~€ 1.8 million). Research and development (R&D) costs were € 34.8 million for the nine month period ended September 30, 2025 compared to € 25.7 million for the same period last year. General and administrative costs were € 11.2 million for the nine month period ended September 30, 2025 compared to € 9.7 million for the same period in 2024. Net loss for the nine-month period ended September 30, 2025 was € 33.3 million, or € 0.32 per diluted share, compared to € 18.5 million, or € 0.23 per diluted share, for the same period last year. For further financial information for the period ended September 30, 2025, please refer to the Q3 financial report 6-K filing. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. Forward Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical and clinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, the continued advancement of our lead development pipeline programs, including ongoing and planned clinical trials, the planned Phase 1 clinical study of for AX-0810 in NTCP for cholestatic diseases, including the planned trial design, implementation and initiation in the Netherlands, and our ability to recruit for and complete a Phase 1 clinical trial for AX-0810 in healthy volunteers; expectations regarding the safety and therapeutic benefits of AX-0810, including the planned dosing levels and their efficacy; the anticipated timing of initial Phase 1 clinical data for our lead program, AX-0810, and other programs, the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates, the timing, progress and results of our preclinical studies and other development and pipeline activities, including the release of data related thereto, our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration with Lilly and the intended benefits thereof, including timing for data updates, potential milestones, exercise of an option to expand targets and the receipt of an opt-in payment, our ability to selectively form new partnerships and enter into future collaborations, and our financial position and cash-runway. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical events and conflicts, high inflation, rising interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor and media contact: Sarah Kiely ProQR Therapeutics N.V. T: +1 617 599 6228 [email protected] or Investor contact: Peter Kelleher LifeSci Advisors T: +1 617 430 7579 [email protected] Financial Tables PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Financial Position PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income (€ in thousands, except share and per share data) PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Changes in Equity . PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Cash Flows