PNPLA3

Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseasesAdvancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett SyndromeHosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 Phase 1 trial design and 2025 data expectations, plus updates across differentiated liver and CNS pipeline€ 119.8 million cash and cash equivalents as of end Q2 – providing runway into mid-2027, not including additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 07, 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 second quarter ended June 30, 2025, and provided a business update. “We continued to execute across our pipeline in the second quarter. Notably, we submitted the Clinical Trial Application for AX-0810, our lead RNA editing program targeting NTCP for cholestatic diseases, and expect to share initial data from the trial later this year,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “As AX-0810 advances and we progress additional pipeline programs, including our first CNS program AX-2402 targeting MECP2 for Rett syndrome, we remain committed to delivering value by addressing high unmet need patient populations with innovative RNA editing therapies. We look forward to providing additional updates on our Axiomer pipeline and platform progress at our Analyst and Investor Event this fall.” Recent Progress In July, the Company presented at the RNA Editing Summit in Boston, highlighting applications of its Axiomer RNA editing platform technology in CNS. In June, ProQR announced it submitted at Clinical Trial Application (CTA) to the European Medicines Agency (EMA) for a first-in-human Phase 1 study of AX-0810, targeting NTCP in healthy volunteers. This milestone marks the first clinical entry for ProQR’s Axiomer RNA editing platform. Pending regulatory clearance, the study is expected to commence at a single site in the Netherlands with initial data anticipated in Q4 2025. The trial will assess safety, tolerability, pharmacokinetics, and target engagement. In May, ProQR showcased its scientific leadership in RNA editing through multiple presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting and the Oligonucleotide and Peptide Therapeutics Conferenece (TIDES USA), highlighting advances in its Axiomer RNA editing platform, including novel applications.In April, ProQR announced the appointments of Dennis Hom as Chief Financial Officer and Cristina Lopez Lopez, MD, PhD as Chief Medical Officer. These key leadership appointments support the advancement of the Company’s Axiomer platform technology and pipeline of RNA editing programs as it enters the clinical stage. Anticipated Upcoming Events ProQR will host a virtual Analyst and Investor Event in the fall to highlight a detailed overview of the AX-0810 Phase 1 trial design, set expectations for initial data in 2025, and provide broader pipeline updates, including for the first CNS program, AX-2402 for Rett syndrome. Additional information, including date and registration details for this event will be shared in a future announcement. Pipeline programs Program Target Indication Upcoming milestone AX-0810 NTCP Cholestatic diseases Q4 2025 initial Phase 1 data in healthy volunteers AX-2402 MECP2 Rett Syndrome (R270X) 2025 clinical candidate selection AX-2911 PNPLA3 MASH 2025 clinical candidate selection AX-1412 B4GALT1 Cardiovascular disease 2025 update on optimization for GalNAc delivery Continue to execute on partnership with Eli Lilly and Company (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. Financial Highlights At June 30, 2025, ProQR held cash and cash equivalents of approximately € 119.8 million, compared to € 149.4 million at December 31, 2024. Net cash used in operating activities during the six-month period ended June 30, 2025 was € 27.2 million, compared to € 21.4 million used for the same period last year. During the first half 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 € 23.7 million for the six month period ended June 30, 2025 compared to € 16.3 million for the same period last year. General and administrative costs were € 8.1 million for the six month period ended June 30, 2025 compared to € 6.5 million for the same period in 2024. Net loss for the six-month period ended June 30, 2025 was € 22.3 million, or € 0.21 per diluted share, compared to € 10.4 million, or € 0.13 per diluted share, for the same period last year. For further financial information for the period ended June 30, 2025, please refer to the Q2 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, expectations regarding regulatory feedback and the potential registrational pathway for AX-0810 in NTCP for cholestatic diseases, the anticipated timing of initial Phase 1 clinical data for our lead program, AX-0810, in Q4 2025, and clinical updates across multiple programs in 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 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 KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorInvestor contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.com Financial Tables PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Financial Position June 30, December 31, 2025 2024 €1,000 €1,000 Assets Property, plant and equipment 13,258 14,113 Investments in financial assets — — Non-current assets 13,258 14,113 Cash and cash equivalents 119,765 149,408 Prepayments and other receivables 3,931 3,747 Other taxes 583 690 Current assets 124,279 153,845 Total assets 137,537 167,958 Equity and liabilities Equity Equity attributable to owners of the Company 66,983 88,560 Total equity 66,983 88,560 Liabilities Borrowings — — Lease liabilities 10,481 11,067 Deferred income 26,985 29,429 Non-current liabilities 37,466 40,496 Borrowings 4,727 4,582 Lease liabilities 1,654 1,567 Derivative financial instruments 290 468 Trade payables 1,283 16 Social securities and other taxes 281 1,478 Deferred income 17,450 21,942 Other current liabilities 7,403 8,849 Current liabilities 33,088 38,902 Total liabilities 70,554 79,398 Total equity and liabilities 137,537 167,958 PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income (€ in thousands, except share and per share data) Three month period Six month period ended June 30, ended June 30, 2025 2024 2025 2024 €1,000 €1,000 €1,000 €1,000 Revenue 3,817 6,305 8,336 10,755 Other income 158 156 380 366 Research and development costs (11,408) (7,048) (23,731) (16,331) General and administrative costs (4,816) (3,013) (8,050) (6,465) Total operating costs (16,224) (10,061) (31,781) (22,796) Operating result (12,249) (3,600) (23,065) (11,675) Finance income and expense 192 513 647 1,001 Results related to financial liabilities measured at fair value through profit or loss (104) 195 178 127 Result before corporate income taxes (12,161) (2,892) (22,240) (10,547) Income taxes (18) 200 (18) 197 Result for the period (12,179) (2,692) (22,258) (10,350) Other comprehensive income (foreign exchange differences on foreign operation) (682) 85 (1,053) 276 Total comprehensive income (12,861) (2,607) (23,311) (10,074) Result attributable to Owners of the Company (12,179) (2,692) (22,258) (10,350) Non-controlling interests — — — — (12,179) (2,692) (22,258) (10,350) Total comprehensive income attributable to Owners of the Company (12,861) (2,607) (23,311) (10,074) Non-controlling interests — — — — (12,861) (2,607) (23,311) (10,074) Share information Weighted average number of shares outstanding1 105,343,897 81,665,565 105,320,495 81,618,038 Earnings per share attributable to owners of the Company (Euro per share) Basic loss per share1 (0.12) (0.03) (0.21) (0.13) Diluted loss per share1 (0.12) (0.03) (0.21) (0.13) 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 TotalEquity €1,000 €1,000 €1,000 €1,000 €1,000 €1,000 Balance at January 1, 2024 84,248,384 3,370 412,894 25,159 817 (400,850) 41,390 Result for the period — — — — — (10,350) (10,350) Other comprehensive income — — — — 276 — 276 Recognition of share-based payments — — — 1,364 — — 1,364 Treasury shares transferred (326,455) — — — — — — Share options lapsed — — — (359) — 359 — Share options exercised / RSUs vested 326,455 — 174 (288) — 288 174 Balance at June 30, 2024 84,248,384 3,370 413,068 25,876 1,093 (410,553) 32,854 Balance at January 1, 2025 107,710,916 4,308 483,812 26,248 1,350 (427,158) 88,560 Result for the period — — — — — (22,258) (22,258) Other comprehensive income — — — — (1,053) — (1,053) Recognition of share-based payments — — — 1,667 — — 1,667 Treasury shares transferred (131,525) — — — — — — Share options lapsed — — — (1,462) — 1,462 — Share options exercised / RSUs vested 131,525 — 67 (181) — 181 67 — Balance at June 30, 2025 107,710,916 4,308 483,879 26,272 297 (447,773) 66,983 PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Cash Flows Three month period Six month period ended June 30, ended June 30, 2025 2024 2025 2024 €1,000 €1,000 €1,000 €1,000 Cash flows from operating activities Net result (12,179) (2,692) (22,258) (10,350) Adjustments for: — Other income (158) — (380) — — Depreciation 675 711 1,353 1,402 — Share-based compensation 909 628 1,667 1,364 — Financial income and expenses (139) (513) (647) (1,001) — Results related to financial liabilities measured at fair value through profit or loss 104 (195) (178) (127) — Income tax expenses 18 (200) 18 (197) Changes in working capital (1,178) (4,614) (7,900) (13,838) Cash used in operations (11,948) (6,875) (28,325) (22,747) Corporate income tax (paid)/received (18) 199 (18) 196 Interest received 617 610 1,405 1,542 Interest paid (52) (190) (261) (379) Net cash used in operating activities (11,401) (6,256) (27,199) (21,388) Cash flow from investing activities Increase in financial asset - current — — — (17,000) Purchases of property, plant and equipment (101) (267) (325) (999) Net cash used in investing activities (101) (267) (325) (17,999) Cash flow from financing activities Proceeds from exercise of share options — 12 67 174 Repayment of lease liability (293) (294) (860) (875) Net cash used in financing activities (293) (282) (793) (701) Net decrease in cash and cash equivalents (11,795) (6,805) (28,317) (40,088) Currency effect cash and cash equivalents (854) 62 (1,326) 133 Cash and cash equivalents, at beginning of the period 132,414 85,713 149,408 118,925 Cash and cash equivalents at the end of the period 119,765 78,970 119,765 78,970

Dosing complete in the first two cohorts of INLIGHT trial in obesity of WVE-007 (INHBE siRNA), designed to induce healthy weight loss by reducing fat without impacting muscle; clinical data on track for 2H 2025 Dosing underway in second single dose cohort (400 mg) and multidosing (200 mg) ongoing in RestorAATion-2 clinical trial of WVE-006 in individuals with PiZZ AATD; data from the complete 200 mg multidose and single dose cohorts expected in 3Q 2025; data from complete 400 mg single dose cohort expected in the fall of 2025 Delivered positive data from FORWARD-53 clinical trial of WVE-N531 in exon 53 amenable DMD including statistically significant and clinically meaningful improvement in TTR, substantial improvements in muscle health; NDA submission for accelerated approval with monthly dosing planned for 2026 IND submission expected 2H 2025 for potentially registrational WVE-003 Phase 2/3 study in HD with caudate atrophy as a primary endpoint Cash and cash equivalents of $243.1 million as of March 31, 2025 , with runway expected into 2027 Investor conference call and webcast at 8:30 a.m. ET today CAMBRIDGE, Mass. , May 08, 2025 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced financial results for the first quarter ended March 31, 2025 , and provided a business update. “We’ve continued our consistent execution across modalities as we completed dosing in the first two cohorts of our INLIGHT trial in obesity, advanced our RestorAATion-2 trial in AATD, and delivered positive data from our FORWARD-53 clinical trial in DMD. We are on track to deliver multiple clinical datasets this year that will further demonstrate our broad capabilities across modalities and our leadership in RNA medicines,” said Paul Bolno , MD, MBA, President and Chief Executive Officer at Wave Life Sciences . “Our RestorAATion-2 trial of WVE-006, a subcutaneously-dosed GalNAc-conjugated RNA editing oligonucleotide, continues to advance and we are on track to deliver data from multiple dose cohorts this year, which will inform the therapeutic potential of WVE-006 and our pipeline of wholly-owned GalNAc-RNA editing programs. In obesity, we are evaluating WVE-007, our INHBE GalNAc-siRNA in our ongoing INLIGHT clinical trial, and are on track to deliver the first clinical data in the second half of this year. This program has potential to transform the obesity treatment paradigm with healthy weight loss, preservation of muscle mass, and infrequent dosing of once or twice a year.” Dr. Bolno continued, “In DMD, we delivered the first-ever substantial improvements in muscle health with an exon skipping therapy and showed statistically significant and clinically meaningful functional data from our FORWARD-53 trial of WVE-N531 in March. We have been engaged with the community in discussing our recent clinical results and are excited by the potential to bring a meaningful new potential treatment option to boys with DMD. In HD, our WVE-003 program has industry-leading CSF mutant lowering, and remains the only program to have successfully demonstrated allele-selective knockdown with wild-type HTT preservation in the clinic. We are actively engaged with both the HD community and prospective strategic partners, as we continue to prepare for our potentially registrational Phase 2/3 study.” Recent Business Highlights and Expected Milestones Obesity WVE-007 is a GalNAc-conjugated small interfering RNA (GalNAc-siRNA) designed to silence INHBE mRNA, an obesity target with strong evidence from human genetics. WVE-007 is Wave’s first siRNA candidate to enter clinical development and uses Wave’s best-in-class proprietary oligonucleotide chemistry. INLIGHT is an ongoing, first-in-human, placebo-controlled, clinical trial evaluating WVE-007 in adults living with overweight or obesity and assesses safety, tolerability, pharmacokinetics, biomarkers for target engagement, body weight and composition, and metabolic health. Today, Wave announced that it has completed dosing in the first and second single dose cohorts of INLIGHT. Next week, in oral presentations at the 32nd European Congress on Obesity (ECO) and the American Society of Gene and Cell Therapies (ASGCT) 28th Annual Meeting, Wave will highlight its preclinical data supporting WVE-007’s potential in multiple treatment settings with potential for dosing once or twice a year, including: A single dose of Wave’s INHBE siRNA led to weight loss on par with semaglutide, but with no muscle loss. When administered as an add-on to semaglutide, a single dose of Wave’s INHBE siRNA doubled the amount of weight loss. Wave’s INHBE siRNA curtailed rebound weight gain when semaglutide treatment was discontinued, highlighting its potential as an off-ramp and maintenance treatment following GLP-1 treatment. Expected milestones: Wave expects to deliver clinical data from INLIGHT in the second half of 2025, including safety, tolerability and biomarkers reflective of healthy weight loss. A single dose of Wave’s INHBE siRNA led to weight loss on par with semaglutide, but with no muscle loss. When administered as an add-on to semaglutide, a single dose of Wave’s INHBE siRNA doubled the amount of weight loss. Wave’s INHBE siRNA curtailed rebound weight gain when semaglutide treatment was discontinued, highlighting its potential as an off-ramp and maintenance treatment following GLP-1 treatment. AATD (Alpha-1 antitrypsin deficiency) WVE-006 is a GalNAc-conjugated, subcutaneously delivered, A-to-I RNA editing oligonucleotide (AIMer) that is uniquely designed to address alpha-1 antitrypsin deficiency (AATD)-related lung disease, liver disease, or both. RestorAATion clinical program: Multi-dosing is complete in RestorAATion-1 (healthy volunteers) at a dose level greater than those planned for any cohort in its ongoing RestorAATion-2 study. RestorAATion-2 is a Phase 1b/2a open-label study with both single and multiple ascending dose portions, which is evaluating the safety, tolerability, pharmacodynamics and pharmacokinetics of WVE-006 in individuals with AATD who have the homozygous Pi*ZZ mutation. Multi-dosing is ongoing in the first cohort of RestorAATion-2, where patients are receiving 200 mg subcutaneous doses every two weeks. Dosing is also underway in the second single dose cohort at 400 mg. In October 2024 , Wave delivered proof-of-mechanism data from a single, lowest dose of WVE-006 from the first two patients in the ongoing RestorAATion-2 clinical study, representing the first-ever clinical demonstration of RNA editing in humans. Circulating wild-type M-AAT protein in plasma reached a mean of 6.9 micromolar, representing more than 60% of total AAT. Mean total AAT protein increased to 10.8 micromolar, meeting the level that has been the basis for regulatory approval for AAT augmentation therapies. Expected milestones: Wave expects to share data from the complete 200 mg multidose and single dose cohorts of RestorAATion-2 in the third quarter of 2025, and data from the complete 400 mg single dose cohort in the fall of 2025. Emerging wholly owned siRNA and RNA editing pipeline Wave is advancing new targets across multiple disease areas to expand its pipeline of wholly owned programs in both rare and common diseases. Wave’s pipeline of preclinical candidates utilize Wave’s proprietary chemistry to achieve best-in-class silencing using siRNA and RNA editing in a variety of hepatic and extrahepatic tissues, including in the CNS with multiple AIMers such as MECP2. Within RNA editing, Wave has demonstrated the ability to correct single variants to restore wild-type protein function and to increase the stability of the mRNA transcript to upregulate protein levels. Wave’s wholly owned RNA editing pipeline includes programs that use GalNAc conjugation and have efficient clinical paths to proof-of-concept. These include PNPLA3 mRNA correction to potentially address the nine million homozygous I148M carriers in the US and Europe at risk for a variety of liver diseases, and mRNA upregulation (LDLR) and mRNA correction (APOB), which together would address approximately one million people living with heterozygous familial hypercholesterolemia (HeFH) in the US and Europe . Next week, in an oral presentation at the ASGCT 28th Annual Meeting, Wave plans to share preclinical data demonstrating proof-of-principle for the use of AIMers in lung indications, including cystic fibrosis (CF). Available therapies for CF cannot address stop codon mutations in the CFTR gene. In human bronchial epithelial cells with CFTR mutation W1282X, CFTR AIMers increased expression of CFTR mRNA 3-fold and restored up to 50% of functional wild-type CFTR protein levels. Expected milestones: Wave plans to share new preclinical data from hepatic and extra-hepatic RNA editing programs in 2025 and to initiate clinical development of additional RNA editing programs, including PNPLA3, LDLR, and APOB, in 2026. DMD (Duchenne muscular dystrophy) WVE-N531 is an exon skipping oligonucleotide being developed as a disease modifying treatment for boys with Duchenne muscular dystrophy amenable to exon 53 skipping. WVE-N531 was designed using Wave’s best-in-class oligonucleotide chemistry modifications, including PN backbone chemistry. WVE-N531 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food & Drug Administration . In March 2025 , Wave announced positive 48-week data from its Phase 2, open-label FORWARD-53 clinical trial of WVE-N531, which included: Statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise vs. natural history with largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks; additional functional benefits observed in other outcome measures including NSAA. First-ever demonstration of substantial improvements in muscle health with exon skipping – statistically significant reduction in fibrosis driven by decreases in inflammation and necrosis, coupled with transition from regenerative to mature muscle; decreases in creatine kinase and circulating inflammatory biomarkers. Dystrophin expression stabilized between 24 and 48 weeks and averaged 7.8%, with 88% of boys above 5% average dystrophin. WVE-N531 remains safe and well-tolerated with no Serious Adverse Events. All participants in FORWARD-53 elected to advance to the extension portion of the clinical trial, which is currently ongoing with boys receiving monthly doses of WVE-N531. To augment monthly data and ensure a monthly regimen at a potential launch, Wave is also expanding FORWARD-53 to include additional boys who will be dosed monthly. Also in March 2025 , Wave announced that the company met with the U.S. Food and Drug Administration (FDA) on WVE-N531 to discuss its interim 24-week data and initial plans for the confirmatory trial, where the Agency confirmed that the accelerated approval pathway using dystrophin expression as a surrogate endpoint remains open. Expected milestones: Wave plans to file a New Drug Application (NDA) in 2026 to support accelerated approval of WVE-N531 with monthly dosing. Wave expects to submit clinical trial applications (CTAs) for additional exon skipping programs in 2026. Statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise vs. natural history with largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks; additional functional benefits observed in other outcome measures including NSAA. First-ever demonstration of substantial improvements in muscle health with exon skipping – statistically significant reduction in fibrosis driven by decreases in inflammation and necrosis, coupled with transition from regenerative to mature muscle; decreases in creatine kinase and circulating inflammatory biomarkers. Dystrophin expression stabilized between 24 and 48 weeks and averaged 7.8%, with 88% of boys above 5% average dystrophin. WVE-N531 remains safe and well-tolerated with no Serious Adverse Events. HD (Huntington’s disease) WVE-003 is a first-in-class, allele-selective oligonucleotide for the treatment of Huntington’s disease (HD). In the SELECT-HD clinical trial, data demonstrated the first-ever allele-selective reduction in CSF mHTT protein and preservation of healthy, wtHTT with multiple doses of WVE-003, as well as a statistically significant correlation between mHTT reduction and slowing of caudate atrophy. By reducing mHTT at the mRNA and protein level, WVE-003 addresses underlying drivers of neurodegeneration. In addition, by sparing wtHTT protein, which is critical to the health of the central nervous system, WVE-003 is uniquely positioned to address presymptomatic HD patients, as well as symptomatic patients. Wave has received supportive initial feedback from FDA, who recognize the severity of HD and are receptive to and engaged with Wave regarding a potential pathway to accelerated approval. Preparation is ongoing for a potentially registrational, global Phase 2/3 study of WVE-003 in adults with SNP3 and HD using caudate atrophy as a primary endpoint. Expected milestones: Wave expects to submit an Investigational New Drug (IND) application for a potentially registrational Phase 2/3 study of WVE-003 in HD in the second half of 2025. Financial Highlights Cash and cash equivalents were $243.1 million as of March 31, 2025 , compared to $302.1 million as of December 31, 2024 . Wave expects that its current cash and cash equivalents will be sufficient to fund operations into 2027. Potential future milestones and other payments to Wave under its GSK collaboration are not included in its cash runway. Revenue recognized was $9.2 million for the first quarter of 2025 as compared to $12.5 million in the prior year quarter. Research and development expenses were $40.6 million in the first quarter of 2025 as compared to $33.4 million in the same period in 2024. General and administrative expenses were $18.4 million in the first quarter 2025 as compared to $13.5 million in the same period in 2024. Net loss was $46.9 million for the first quarter of 2025 as compared to $31.6 million in the prior year quarter. Investor Conference Call and WebcastWave will host an investor conference call today at 8:30 a.m. ET to review the first quarter 2025 financial results and pipeline updates. A webcast of the conference call can be accessed by visiting “Investor Events” on the investor relations section of the Wave Life Sciences website: https://ir.wavelifesciences.com/events-publications/events. Analysts planning to participate during the Q&A portion of the live call can join the conference call by dialing (833) 630-1956 (domestic) or (412) 317-1837 (international). Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website. About Wave Life Sciences Wave Life Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave’s RNA medicines platform, PRISM®, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and common disorders. Its toolkit of RNA-targeting modalities includes editing, splicing, RNA interference and antisense silencing, providing Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave’s diversified pipeline includes clinical programs in Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington’s disease, and Obesity, as well as several preclinical programs utilizing the company’s broad RNA therapeutics toolkit. Driven by the calling to “Reimagine Possible”, Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in Cambridge, MA. For more information on Wave’s science, pipeline and people, please visit www.wavelifesciences.com and follow Wave on X (formerly Twitter) and LinkedIn. Forward-Looking StatementsThis press release contains forward-looking statements concerning our goals, beliefs, expectations, strategies, objectives and plans, and other statements that are not necessarily based on historical facts, including statements regarding the following, among others: the anticipated initiation, site activation, patient recruitment, patient enrollment, dosing, generation and reporting of data and completion of our clinical trials, including interactions with regulators and any potential registration based on these data, and the timing and announcement of such events; the protocol, design, endpoints, dose levels and dosing frequency for our investigational therapeutics in clinical trials; the future performance and results of our programs in clinical trials; our expectations with respect to how our clinical data successes to date may predict success for our future therapeutic candidates and data readouts and may further validate our platform; preclinical activities and programs and their potential to transition into clinical-stage programs; the potential of our preclinical data to predict the behavior of our compounds in humans; regulatory submissions and timing for regulatory feedback; the submission of marketing approval applications to regulators, approval thereof, and the potential commercialization of our late-stage programs; the progress and potential benefits of collaborations and strategic partnerships; the potential achievement of milestones under any collaborations; our identification of future product candidates and their therapeutic potential; the anticipated benefits of our therapeutic candidates and pipeline compared to our competitors; the potential unmet medical needs and addressable patient population estimates related to our therapeutic candidates; our ability to design compounds using the most appropriate of our multiple modalities and the anticipated benefits of that approach; the breadth and versatility of our drug discovery and development platform; the expected benefits of our stereopure oligonucleotides compared with stereorandom oligonucleotides; the potential benefits of our RNA editing capability, including our AIMers, compared to others; the potential for certain of our programs to be best-in-class or first-in-class or to change the existing treatment paradigm or show substantial benefits over existing standards of care; the status and progress of our programs relative to potential competitors; anticipated benefits of our proprietary manufacturing processes and our internal manufacturing capabilities; the benefits of RNA medicines generally; the strength of our intellectual property and the data that support our IP; the anticipated duration of our cash runway and our ability to fund future operations; our intended uses of capital; and our expectations regarding the impact of any potential global macro events on our business. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the following: our ability to finance our drug discovery and development efforts and to raise additional capital when needed; the ability of our preclinical programs to produce data sufficient to support our clinical trial applications and the timing thereof; the clinical results of our programs and the timing thereof, which may not support further development of our product candidates; actions of regulatory authorities and their receptiveness to our trial designs and accelerated approval pathways, which may affect the initiation, timing and progress of clinical trials; our effectiveness in managing interactions with regulatory authorities; the effectiveness of our drug discovery and development platform; the effectiveness of our RNA editing capability and our AIMers; our ability to demonstrate the therapeutic benefits of our candidates in clinical trials, including our ability to develop candidates across multiple therapeutic modalities; our dependence on third parties, including contract research organizations, contract manufacturing organizations, collaborators and partners; our ability to manufacture or contract with third parties to manufacture drug material to support our programs and growth; our ability to obtain, maintain and protect our intellectual property; our ability to enforce our patents against infringers and defend our patent portfolio against challenges from third parties; competition from others developing therapies for the indications we are pursuing; our ability to maintain the company infrastructure and personnel needed to achieve our goals; and the information under the caption “Risk Factors” contained in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) and in other filings we make with the SEC from time to time. We undertake no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances. Contact: Kate Rausch VP, Corporate Affairs and Investor Relations+1 617-949-4827 Investors:InvestorRelations@wavelifesci.com Media:MediaRelations@wavelifesci.com Source: Wave Life Sciences USA, Inc.

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