B4GALT1

Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expects up to four clinical data readouts in 2025 and 2026 across multiple programs, highlighted by first clinical data for AX-0810 anticipated in Q4 2025€ 149.4 million cash and cash equivalents as of end of 2024, providing runway into mid-2027, plus additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., March 13, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (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, 2024, and provided a business update. “As we continue to advance our Axiomer ADAR-mediated RNA editing platform and pipeline, ProQR is entering a pivotal phase of growth and innovation,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “With up to four clinical trial data readouts anticipated in 2025 and 2026, including topline data for our NTCP program later this year, we believe we are poised to demonstrate the broad therapeutic potential of RNA editing. Our growing discovery pipeline, now including CNS applications, leading intellectual property portfolio, and strategic partnerships – such as our $3.9 billion collaboration with Eli Lilly and partnership with the Rett Syndrome Research Trust – reinforce our leadership in the field.” De Boer continued, “Our successful financing in October 2024 further strengthens our financial foundation, providing cash runway into mid-2027. With this strong position, we believe we are well equipped to advance key upcoming milestones, including submission of the CTA for our first RNA editing pipeline program, AX-0810, on track for Q2 2025 and there following our first clinical data readout in Q4 of this year, as well as continued progress across our pipeline and partnered programs. We remain committed to driving forward the next era of transformative RNA editing therapeutics for patients in need.” Recent Progress In December 2024, the Company hosted a comprehensive virtual Analyst and Investor event to highlight pipeline programs and progress with its proprietary Axiomer RNA editing technology platform.In December 2024, ProQR expanded its collaboration with the Rett Syndrome Research Trust, securing additional funding for a total of up to $9.2 million to support the advancement of AX-2402, an RNA editing therapy targeting the R270X mutation in the MECP2 gene associated with Rett Syndrome, into clinical trials.In December 2024, ProQR appointed Dr. Peter A. Beal, as Chief ADAR Scientist. Dr. Beal is a leading expert in ADAR and RNA biology from the University of California, Davis, and member of ProQR’s Scientific Advisory Board. As Chief ADAR Scientist, he drives the development and optimization of the Company’s Axiomer ADAR-mediated RNA editing platform.In October 2024, ProQR closed an underwritten public offering of 18,000,000 ordinary shares and a concurrent private placement of 3,523,538 ordinary shares with Eli Lilly and Company (Lilly), as well as exercise of the underwriters’ option for 1,940,072 additional shares for gross proceeds totaling approximately $82.1 million. Anticipated Upcoming Events AX-0810 targeting NTCP for Cholestatic diseases Q2 2025 Clinical Trial Application (CTA) submissionQ4 2025 first clinical target engagement and biomarker data in healthy volunteers AX-2402 targeting MECP2 (R270X) for Rett Syndrome 2025 clinical candidate selection 2026 clinical trial initiation and topline data readout AX-2911 targeting PNPLA3 for MASH 2025 clinical candidate selection 2026 clinical trial initiation and topline data readout AX-1412 targeting B4GALT1 for Cardiovascular diseases mid-2025 update on optimization for GalNAc delivery Continue to execute on partnership with Lilly, with potential data updates, milestone income from the existing partnership, and an option to exercise for an additional five targets for expansion to a total of 15 targets, which would result in a $50 million opt-in payment to ProQR.ProQR may selectively form new partnerships, which could include multi-target discovery alliances, or product alliances on specific programs. Year End 2024 Financial Highlights At December 31, 2024, ProQR held cash and cash equivalents of € 149.4 million, compared to € 118.9 million at December 31, 2023. Net cash used in operating activities during the full year ended December 31, 2024 was € 36.4 million, compared to € 21.5 million net cash generated by operating activities for the same period in 2023. The Company experienced a net positive cash flow from operating activities in 2023 primarily due to the receipt of the Lilly up-front payment of $60 million in February 2023. Research and development costs for the year ended December 31, 2024 were € 36.4 million, compared to € 25.1 million for the same period in 2023. The increase in research and development costs for the year ended December 31, 2024 compared to 2023 was due primarily to increased investments in the platform and advancing pipeline programs. General and administrative costs for the year ended December 31, 2024 were € 13.7 million, compared to € 16.2 million for the same period in 2023. Net loss for the year ended December 31, 2024 was € 27.8 million or € 0.32 per diluted share, compared to € 27.7 million, or € 0.35 per diluted share for the year ended December 31, 2023. For further financial information for the period ended December 31, 2024, please refer to our 2024 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 anticipated timing of initial clinical data readouts across multiple programs in 2025 and 2026, and the filing of a CTA application for our lead program, AX-0810, in Q2 2025, our Axiomer™ platform, including 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 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 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 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 instability and conflicts. 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 6228skiely@proqr.comorInvestor contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.com

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 05, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced that the Company will host a virtual Analyst and Investor Event on Wednesday, December 11, 2024 from 10:00 am until approximately 12:30 pm EST, including an Analyst Q&A session with members of the ProQR Management Team. During the event, ProQR will highlight its proprietary Axiomer™ ADAR-mediated RNA editing platform, along with updates on its pipeline of development candidates including data updates and next steps on its programs for NTCP and B4GALT1, AX-0810 and AX-1412. Presentations will feature members of the Management Team including Daniel A. de Boer, Founder and CEO, and Gerard Platenburg, Chief Scientific Officer; René Beukema, Chief Corporate Development Officer will also join the analyst Q&A session with the Management Team. Additionally, the following speakers will present at the event: Peter Beal, PhD, will share his perspectives on driving innovation in the ADAR field. Dr. Beal is a Professor in the Department of Chemistry at the University of California at Davis and Director of the NIH-funded UC Davis Chemical Biology Graduate Program and member of the Scientific Advisory Board at ProQR.Prof. Gideon Hirschfield, MA(Oxon) MB BChir (Cantab) FRCP PhD, a key opinion leader, will share his views on the unmet need in the field and the opportunity for AX-0810. He is Professor of Gastroenterology and Hepatology, Toronto Centre for Liver Disease. More detailed biographies are available below. Event Registration Details To register for the Investor and Analyst webcast (December 11, 2024), please click here. A live webcast of the event will be available under “Events” in the “Investors & Media” section of ProQR’s website at https://www.proqr.com/events. To join via phone, participants may preregister to receive dedicated dial-in details to access the call via the following website: https://register.vevent.com/register/BI219c7b4f854c4c718b45b428b613d113 It is suggested that participants dial into the conference call 15 minutes prior to the scheduled start time to avoid any delays in attendance. An archived version of the webcast will be available for replay via ProQR’s website for approximately 30 days following the event. Speaker Biographies Peter A. Beal, PhD Peter A. Beal is a Professor in the Department of Chemistry at the University of California at Davis and current Director of the NIH-funded UC Davis Chemical Biology Graduate Program. Research from the Beal laboratory has advanced understanding of the structures and mechanism of action for ADAR enzymes responsible for adenosine to inosine RNA editing in humans. Recently, this knowledge has been applied to the design of guiding oligonucleotides capable of directing ADARs to make edits that correct disease-causing mutations in the transcriptome. Targets include mutations in mRNAs associated with Rett syndrome, Alzheimer’s disease and Parkinson’s disease. Dr. Beal teaches organic chemistry at the undergraduate level and several classes in nucleic acids chemistry and chemical biology at the graduate level. Prof. Gideon Hirschfield, MA(Oxon) MB BChir (Cantab) FRCP PhD Prof. Gideon M. Hirschfield is a distinguished clinician-scientist specializing in autoimmune liver diseases. He holds the Lily and Terry Horner Chair in Autoimmune Liver Disease Research at the Toronto Centre for Liver Disease, Toronto General Hospital, and serves as a Professor of Medicine in the Division of Gastroenterology at the University of Toronto. Prof. Hirschfield earned his medical degree from the University of Cambridge in 1996 and completed his undergraduate studies at the University of Oxford in 1994. He was awarded a PhD from the University of London in 2006. His postgraduate training included positions at Hammersmith Hospital and Royal Brompton Hospital in London, as well as a fellowship in Toronto, Canada. An internationally recognized expert, Prof. Hirschfield has published over 200 peer-reviewed articles, including lead authorship in high-impact journals such as the New England Journal of Medicine, The Lancet, and Nature Genetics. His research focuses on advancing therapies for inflammatory liver diseases to prevent the need for transplantation 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, express and implied statements regarding this event, the potential of our technologies and product candidates, our business, technology, strategy, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our Axiomer™ platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates, our development programs, including the timing, progress and results of our preclinical studies, clinical trials and other development activities, including the release of data related thereto, our business operations, as well as the timing of our clinical development. 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 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; 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 Eli Lilly and Company; 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 instability and conflicts. 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 contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorPeter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.com Media contact:Robert StanislaroFTI ConsultingT: +1 212 850 5657robert.stanislaro@fticonsulting.com

AX-0810 targeting NTCP for cholestatic diseases to be first ProQR Axiomer™ RNA editing program to enter the clinic Company to host virtual Investor and Analyst Event in December to highlight translational data and clinical plans for AX-0810, as well as AX-1412, targeting B4GALT1 for cardiovascular diseases, and progress made across the Axiomer platform€89.4 million cash and cash equivalents as of end of Q3, plus $82.1 million gross proceeds from October financing providing runway into mid-2027 LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 07, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (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, 2024, and provided a business update. “We’re grateful for the strong support from investors and our partner Lilly in our $82.1 million equity financing last month, which further strengthens our financial position and extends our runway into mid-2027,” said Daniel A. de Boer, Founder and CEO of ProQR Therapeutics. “As we approach the clinic with AX-0810 for cholestatic diseases, we look forward to sharing updates on this program, including preclinical data, clinical trial design, and biomarkers that will be assessed, along with pipeline highlights at our Investor and Analyst Event in December. With our Axiomer™ RNA editing platform, a differentiated pipeline, a productive partnership, and the leading IP position in the field, we remain focused on advancing transformative therapies for the unmet needs of patients.” Recent Progress and Anticipated Upcoming Events In October, ProQR closed an underwritten public offering of 18,000,000 ordinary shares and a concurrent private placement of 3,523,538 ordinary shares with Eli Lilly, as well as exercise of the underwriter’s option for 1,940,072 additional shares for gross proceeds totaling approximately $82.1 million. In October, ProQR presented at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society in Montreal, highlighting its Axiomer technology platform and AX-0810, the Company’s program targeting NTCP for cholestatic diseases. ProQR will hold a virtual Analyst and Investor Event in December to highlight translational non-clinical data, delivery data, clinical trial design and timelines, and details on biomarkers for AX-0810 targeting NTCP for cholestatic diseases. Additional data will be shared related to AX-1412 targeting B4GALT1 for cardiovascular disease and other updates will highlight advancements with the platform. A webcast announcement with additional information, including date and registration details for this event will be shared in the coming weeks. Financial Highlights At September 30, 2024, ProQR held cash and cash equivalents of €89.4 million, compared to €118.9 million cash and cash equivalents at December 31, 2023. Net cash used in operating activities during the three-month period ended September 30, 2024 was €5.7 million, compared to €7.5 million used for the same period last year. During the first three quarters of 2024, the Company achieved certain milestones in the collaboration agreement with Eli Lilly amounting to $5.5 million. In October, the Company closed an underwritten public offering of 18,000,000 ordinary shares and a concurrent private placement of 3,523,538 ordinary shares with Eli Lilly, as well as exercise of the underwriter’s option for 1,940,072 additional shares for gross proceeds totaling approximately $82.1 million. Research and development (R&D) costs were €9.4 million for the quarter ended September 30, 2024 compared to €5.4 million for the same period last year. General and administrative costs were €3.3 million for the quarter ended September 30, 2024 in line with €3.3 million for the same period last year. Net loss for the three-month period ended September 30, 2024 was €8.1 million, or €0.10 per diluted share, compared to €5.4 million, or €0.07 per diluted share, for the same period last year. For further financial information for the period ended September 30, 2024, please refer to the Q3 financial report 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 Biliary Atresia (BA) and Primary Sclerosing Cholangitis (PSC) Cholestatic disorders refer to a group of diseases presenting excessive and toxic buildup of bile acids in the liver due to bile ducts dysfunction. This leads to liver damage and a range of debilitating symptoms. Without treatment, liver damage can progress through various stages, ultimately leading to liver failure and elevated risk of liver malignancy, affecting life expectancy. Cholestatic diseases remain leading causes of liver transplantation. There are no approved therapies for primary sclerosing cholangitis (PSC) for adults and biliary atresia (BA) for pediatrics. It is estimated that 80,000 and 20,000 individuals have PSC and BA, respectively, in North America and in Europe. About AX-0810 targeting NTCP The majority of the bile acids present in the liver cells originate from the enterohepatic reuptake cycle. The key transporter responsible for hepatic uptake of bile acids from portal circulation is the sodium (Na+)-taurocholate cotransporting polypeptide (NTCP, SLC10A1 gene) expressed in the liver. AX-0810 is designed to introduce a loss of function variant in SLC10A1 RNA that has been found in human genetics to prevent re-uptake of bile acids in liver via NTCP. Based on its mechanism of action, AX-0810 has the potential to become a disease modifying treatment for PSC and BA primarily among other cholestatic diseases. About Cardiovascular Diseases Cardiovascular diseases (CVDs) are a group of health conditions that affect the heart and blood vessels, such as atherosclerosis which can lead to severe problems like heart attacks, heart failure, and stroke. CVDs represent the leading cause of disability and death in the world. Approximately 18 million people die every year from CVDs representing one third of all the global deaths. Despite available lipid lowering therapies and hypertension medications, the risk of CVDs is still projected to increase rapidly over the coming years. About AX-1412 targeting B4GALT1 Gene–based analysis of rare beta-1,4-galactosyltransferase 1 (B4GALT1) missense variant (p.Asn352Ser) is known to lead to B4GALT1 protein loss of function and showed an association with decreased coronary artery disease. These beneficial effects are mediated by hypo-galactosylation of the apolipoprotein B100 and fibrinogen, known – independent – drivers of increased risk of CVDs. AX-1412 introduces a protective variant into B4GALT1 RNA to address the remaining residual risk of developing cardiovascular diseases. ProQR intends to advance AX-1412 targeting B4GALT1 to early clinical proof of concept stage, then would seek to partner this program. 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 model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our Axiomer™ platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and our ability to expand preclinical in vivo and in vitro data, the timing, progress and results of our preclinical studies and other development 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 and potential benefits thereof, including the receipt of milestone and royalty payments from commercial product sales, if any, from the products covered by the collaboration, 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 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; 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 instability and conflicts. 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 contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorPeter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.com Media contact:Robert StanislaroFTI ConsultingT: +1 212 850 5657robert.stanislaro@fticonsulting.com Financial Tables PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Financial Position September 30, December 31, 2024 2023 €1,000 €1,000 Assets Property, plant and equipment 14,437 16,897 Investments in financial assets — — Non-current assets 14,437 16,897 Other taxes 535 523 Prepayments and other receivables 2,478 1,538 Cash and cash equivalents 89,401 118,925 Financial asset - current — — Current assets 92,414 120,986 Total assets 106,851 137,883 Equity and liabilities Equity Equity attributable to owners of the Company 24,982 41,390 Total equity 24,982 41,390 Liabilities Borrowings — 4,292 Lease liabilities 11,501 13,828 Deferred income 30,728 44,170 Non-current liabilities 42,229 62,290 Borrowings 4,510 — Lease liabilities 1,386 1,614 Derivative financial instruments 45 311 Trade payables 31 1,541 Social securities and other taxes 1,369 1,659 Deferred income 24,860 20,569 Other current liabilities 7,439 8,509 Current liabilities 39,640 34,203 Total liabilities 81,869 96,493 Total equity and liabilities 106,851 137,883 PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Profit or Loss and OCI (€ in thousands, except share and per share data) Three month period Nine month period ended September 30, ended September 30, 2024 2023 2024 2023 €1,000 €1,000 €1,000 €1,000 Revenue 3,843 1,370 14,598 3,230 Other income 138 — 504 80 Research and development costs (9,414) (5,446) (25,745) (17,415) General and administrative costs (3,283) (3,315) (9,748) (11,486) Total operating costs (12,697) (8,761) (35,493) (28,901) Operating result (8,716) (7,391) (20,391) (25,591) Finance income and expense 469 363 1,470 289 Results related to associates and financial assets — — — — Result on derecognition of subsidiary — 92 — 92 Results related to financial liabilities measured at fair value through profit or loss 139 118 266 1,009 Results on derecognition of financial liabilities — 1,357 — 1,866 Result before corporate income taxes (8,108) (5,461) (18,655) (22,335) Corporate income taxes — 41 197 83 Result for the period (8,108) (5,420) (18,458) (22,252) Other comprehensive income Items that will not be reclassified subsequently to profit or loss Fair value loss on investment in financial asset designated as at FVTOCI — (621) — (621) Items that may be reclassified subsequently to profit or loss Foreign exchange differences on translation of foreign operations (377) 286 (101) 74 Total comprehensive loss (8,485) (5,755) (18,559) (22,799) Result attributable to Owners of the Company (8,108) (5,710) (18,458) (22,636) Non-controlling interests — 290 — 384 (8,108) (5,420) (18,458) (22,252) Total comprehensive income attributable to Owners of the Company (8,485) (6,045) (18,559) (23,183) Non-controlling interests — 290 — 384 (8,485) (5,755) (18,559) (22,799) Share information Weighted average number of shares outstanding1 81,682,296 81,000,320 81,639,533 80,942,881 Earnings per share attributable to owners of the Company (Euro per share) Basic loss per share1 (0.10) (0.07) (0.23) (0.28) Diluted loss per share1 (0.10) (0.07) (0.23) (0.28) 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 Condensed Consolidated Statement of Changes in Equity Attributable to owners of the Company Numberof shares ShareCapital SharePremium Equity settledEmployeeBenefitReserve TranslationReserve AccumulatedDeficit Total Non-controllinginterests TotalEquity €1,000 €1,000 €1,000 €1,000 €1,000 €1,000 €1,000 €1,000 Balance at January 1, 2023 84,246,967 3,370 412,540 29,052 1,212 (379,110) 67,064 (384) 66,680 Result for the period — — — — — (22,636) (22,636) 384 (22,252) Other comprehensive income — — — — 74 (621) (547) — (547) Recognition of share-based payments — — — 2,304 — — 2,304 — 2,304 Treasury shares transferred (341,492) — — — — — — — — Share options lapsed — — — (6,209) — 6,209 — — — Share options exercised 341,492 — 155 (426) — 426 155 — 155 Balance at September 30, 2023 84,246,967 3,370 412,695 24,721 1,286 (395,732) 46,340 — 46,340 Balance at January 1, 2024 84,248,384 3,370 412,894 25,159 817 (400,850) 41,390 — 41,390 Result for the period — — — — — (18,458) (18,458) — (18,458) Other comprehensive income — — — — (101) — (101) — (101) Recognition of share-based payments — — — 1,976 — — 1,976 — 1,976 Treasury shares transferred (329,675) — — — — — — — — Share options lapsed — — — (994) — 994 — — — Share options exercised / RSUs vested 328,597 — 175 (291) — 291 175 — 175 Balance at September 30, 2024 84,247,306 3,370 413,069 25,850 716 (418,023) 24,982 — 24,982 PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Cash Flows Three month period Nine month period ended September 30, ended September 30, 2024 2023 2024 2023 €1,000 €1,000 €1,000 €1,000 Cash flows from operating activities Net result (8,108) (5,420) (18,458) (22,252) Adjustments for: — Depreciation 688 642 2,090 1,785 — Share-based compensation 612 444 1,976 2,304 — Financial income and expenses (469) (363) (1,470) (289) — Results on derecognition of subsidiary — (131) — (131) — Results on derecognition of financial liabilities — (1,357) — (1,866) — Results related to financial liabilities measured at fair value through profit or loss (139) (117) (266) (1,008) — Income tax expenses — (83) (197) (83) Changes in working capital 1,117 (2,008) (12,721) 46,660 Cash (used in) / generated by operations (6,299) (8,393) (29,046) 25,120 Corporate income tax received 1 83 197 83 Interest received 860 802 2,402 1,667 Interest paid (219) — (598) — Net cash (used in) / generated by operating activities (5,657) (7,508) (27,045) 26,870 Cash flow from investing activities Increase in financial asset - current — — (17,000) — Decrease in financial asset - current 17,000 — 17,000 — Purchases of property, plant and equipment (286) (339) (1,285) (769) Proceeds from sale of property, plant and equipment — — — 47 Net cash generated by / (used in) investing activities 16,714 (339) (1,285) (722) Cash flow from financing activities Proceeds from exercise of share options 1 151 175 155 Repayment of lease liability (454) (432) (1,329) (1,338) Net cash used in financing activities (453) (281) (1,154) (1,183) Net increase / (decrease) in cash and cash equivalents 10,604 (8,128) (29,484) 24,965 Currency effect cash and cash equivalents (173) 118 (40) 812 Cash and cash equivalents, at beginning of the period 78,970 128,562 118,925 94,775 Cash and cash equivalents at the end of the period 89,401 120,552 89,401 120,552