Earnings release
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Source: ProQR Therapeutics N.V. November 06, 2025 07:00 ET ProQR Announces Third Quarter2025 Operating and FinancialResults 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 fromCohort 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 itsproprietary Axiomer™ RNA editing technology platform, today reported its nancial and operating results for thethird 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 rst-in-humanstudy for AX-0810, marking a signi cant step forward for both this program and our Axiomer RNA editing platform,”said Daniel A. de Boer, Founder and Chief Executive Of cer of ProQR. “This study represents the rst clinicalevaluation 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 rst half of 2026. Supported by our strong cash position providingrunway into mid-2027, we continue to advance our broader pipeline, platform, and partnership activities, executingon 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 thePhase 1 trial design and target engagement/biomarker strategy. AX-0810 uses Axiomer’s ability to modulate protein function by targeting NTCP for the treatment ofcholestatic diseases. In October AX-0810 received CTA authorization and a rst-in-human (FIH) Phase 1 study isbeing initiated in healthy volunteers. Initial safety, tolerability, and PK data for Cohort 1 is expected toward theend of 2025 with target engagement data for all healthy volunteer cohorts in the rst half of 2026. In parallel,activities are underway to include a patient cohort in this FIH following completion of the healthy volunteercohorts. Beyond AX-0810, ProQR’s Axiomer pipeline demonstrates the broad potential of RNA editing across three keyapplications – modulate, correct, and protect – with updates expected across these earlier-stage programs inthe coming months.AX-2402 applies Axiomer RNA editing to correct disease-causing mutations, targeting the R270Xnonsense 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 variantassociated with signi cantly reduced cardiovascular risk.
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In October, the Company presented at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting inBudapest, 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 andmilestone income from the ongoing collaboration. Financial Highlights At September 30, 2025, ProQR held cash and cash equivalents of approximately € 106.9 million, compared to € 149.4million 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 rst nine months of 2025, the Companyachieved 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, 2025compared 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 comparedto € 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 nancial informationfor the period ended September 30, 2025, please refer to the Q3 nancial report 6-K ling. 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 speci c 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 istranslated 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 ortreat 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 speci c single nucleotide edits in RNA to reverse a mutation or modulate protein expression andcould potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on ourunique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones inmind. 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 areforward-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, statementsregarding our business, technology, strategy, preclinical and clinical model data, our initial pipeline targets and theupcoming strategic priorities and milestones related thereto, the continued advancement of our lead developmentpipeline programs, including ongoing and planned clinical trials, the planned Phase 1 clinical study of for AX-0810 inNTCP 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; expectationsregarding the safety and therapeutic bene ts of AX-0810, including the planned dosing levels and their ef cacy; theanticipated timing of initial Phase 1 clinical data for our lead program, AX-0810, and other programs, the continueddevelopment and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editingoligonucleotides and product candidates, the timing, progress and results of our preclinical studies and otherdevelopment and pipeline activities, including the release of data related thereto, our patent estate, including ouranticipated strength and our continued investment in it, as well as the timing of our clinical development, thepotential of our technologies and product candidates, the collaboration with Lilly and the intended bene ts thereof,including timing for data updates, potential milestones, exercise of an option to expand targets and the receipt ofan opt-in payment, our ability to selectively form new partnerships and enter into future collaborations, and our nancial position and cash-runway. Forward-looking statements are based on management's beliefs andassumptions and on information available to management only as of the date of this press release. Our actualresults could differ materially from those expressed or implied by these forward-looking statements for manyreasons, including, without limitation, the risks, uncertainties and other factors in our lings made with theSecurities and Exchange Commission, including certain sections of our most recent annual report led on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical
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trials and other development activities by us and our collaborative partners whose operations and activities may beslowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions andinternational tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelinesprovided and reliance on our contract research organizations and predictability of timely enrollment of subjects andpatients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers tosupply 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 ofthe duration and results of the regulatory review of applications or clearances that are necessary to initiate andcontinue to advance and progress our clinical programs; the ability to secure, maintain and realize the intendedbene ts of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability toobtain, and costs to obtain intellectual property rights; possible safety or ef cacy concerns that could emerge asnew data are generated in research and development; general business, operational, nancial and accounting risks,and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions andmarket volatility resulting from global economic developments, geopolitical events and con icts, high in ation,rising interest rates, tariffs and potential for signi cant changes in U.S. policies and regulatory environment. Giventhese risks, uncertainties and other factors, you should not place undue reliance on these forward-lookingstatements, and we assume no obligation to update these forward-looking statements, even if new informationbecomes 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 7579pkelleher@lifesciadvisors.com Financial Tables PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Financial Position September 30, December 31, 2025 2024 €1,000 €1,000 Assets Property, plant and equipment 13,018 14,113 Investments in nancial assets — — Non-current assets 13,018 14,113 Cash and cash equivalents 106,883 149,408 Prepayments and other receivables 3,872 3,747 Other taxes 576 690 Current assets 111,331 153,845 Total assets 124,349 167,958 Equity and liabilities Equity Equity attributable to owners of the Company 57,071 88,560 Total equity 57,071 88,560 Liabilities Borrowings — — Lease liabilities 10,017 11,067 Deferred income 24,125 29,429 Non-current liabilities 34,142 40,496 Borrowings 4,800 4,582 Lease liabilities 1,674 1,567
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Derivative nancial instruments 292 468 Trade payables 305 16 Social securities and other taxes 121 1,478 Deferred income 17,394 21,942 Other current liabilities 8,550 8,849 Current liabilities 33,136 38,902 Total liabilities 67,278 79,398 Total equity and liabilities 124,349 167,958 PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Pro t or Loss and Other Comprehensive Income(€ in thousands, except share and per share data) Three month period Nine month period ended September 30, ended September 30, 2025 2024 2025 2024 €1,000 €1,000 €1,000 €1,000 Revenue 2,882 3,843 11,218 14,598 Other income 33 138 413 504 Research and development costs (11,080) (9,414) (34,811) (25,745) General and administrative costs (3,119) (3,283) (11,169) (9,748) Total operating costs (14,199) (12,697) (45,980) (35,493) Operating result (11,284) (8,716) (34,349) (20,391) Finance income and expense 280 469 927 1,470 Results related to nancial liabilitiesmeasured at fair value throughpro t or loss (2) 139 176 266 Result before corporate incometaxes (11,006) (8,108) (33,246) (18,655) Income taxes — — (18) 197 Result for the period (11,006) (8,108) (33,264) (18,458) Other comprehensive income(foreign exchange differences onforeign operation) (23) (377) (1,076) (101) Total comprehensive income (11,029) (8,485) (34,340) (18,559) Result attributable to Owners of the Company (11,006) (8,108) (33,264) (18,458) Non-controlling interests — — — — (11,006) (8,108) (33,264) (18,458) Total comprehensive incomeattributable to Owners of the Company (11,029) (8,485) (34,340) (18,559) Non-controlling interests — — — — (11,029) (8,485) (34,340) (18,559) Share information Weighted average number of shares outstanding1 105,344,996 81,682,296 105,328,752 81,639,533 Earnings per share attributable toowners of the Company (Euro pershare)
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Basic loss per share1 (0.10) (0.10) (0.32) (0.23) Diluted loss per share1 (0.10) (0.10) (0.32) (0.23) . For these periods the potential exercise of share options is not included in the diluted earnings per share asthe Company was loss-making. Due to the anti-dilutive nature of the outstanding options, basic and dilutedearnings 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 SharePremiumEquitysettledEmployeeBene tReserve TranslationReserve AccumulatedDe cit TotalEquity €1,000 €1,000 €1,000 €1,000 €1,000 €1,000 Balance at January1, 2024 84,248,384 3,370 412,894 25,159 817 (400,850) 41,390 Result for theperiod — — — — — (18,458) (18,458) Othercomprehensiveincome — — — — (101) — (101) Recognition ofshare-basedpayments — — — 1,976 — — 1,976 Treasury sharestransferred (329,675) — — — — — — Share optionslapsed — — — (994) — 994 — Share optionsexercised / RSUsvested 328,597 — 175 (291) — 291 175 Balance atSeptember 30, 2024 84,247,306 3,370 413,069 25,850 716 (418,023) 24,982 Balance at January1, 2025 107,710,916 4,308 483,812 26,248 1,350 (427,158) 88,560 Result for theperiod — — — — — (33,264) (33,264) Othercomprehensiveincome — — — — (1,076) — (1,076) Recognition ofshare-basedpayments — — — 2,784 — — 2,784 Treasury sharestransferred (132,665) — — — — — — Share optionslapsed — — — (1,532) — 1,532 — Share optionsexercised / RSUsvested 132,665 — 67 (184) — 184 67 Balance atSeptember 30, 2025 107,710,916 4,308 483,879 27,316 274 (458,706) 57,071 . PROQR THERAPEUTICS N.V.Unaudited Condensed Consolidated Statement of Cash Flows
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Three month period Nine month period ended September 30, ended September 30, 2025 2024 2025 2024 €1,000 €1,000 €1,000 €1,000 Cash ows from operating activities Net result (11,006) (8,108) (33,264) (18,458) Adjustments for: — Other income (33) — (413) — — Depreciation 671 688 2,024 2,090 — Share-based compensation 1,117 612 2,784 1,976 — Financial income and expenses (280) (469) (927) (1,470) — Results related to nancial liabilitiesmeasured at fair value through pro tor loss 2 (139) (176) (266) — Income tax expenses — — 18 (197) Changes in working capital (3,074) 1,117 (10,974) (12,721) Cash used in operations (12,603) (6,299) (40,928) (29,046) Corporate income tax received / (paid) — 1 (18) 197 Interest received 519 860 1,924 2,402 Interest paid (151) (219) (412) (598) Net cash used in operating activities (12,235) (5,657) (39,434) (27,045) Cash ow from investing activities Increase in nancial asset - current — — — (17,000) Decrease in nancial asset - current — 17,000 — 17,000 Purchases of property, plant andequipment (169) (286) (494) (1,285) Net cash (used in) / generated byinvesting activities (169) 16,714 (494) (1,285) Cash ow from nancing activities Proceeds from exercise of shareoptions — 1 67 175 Repayment of lease liability (444) (454) (1,304) (1,329) Net cash used in nancing activities (444) (453) (1,237) (1,154) Net (decrease) / increase in cash andcash equivalents (12,848) 10,604 (41,165) (29,484) Currency effect cash and cashequivalents (34) (173) (1,360) (40) Cash and cash equivalents, atbeginning of the period 119,765 78,970 149,408 118,925 Cash and cash equivalents at the endof the period 106,883 89,401 106,883 89,401