Latest news with #ProQR
Yahoo
07-08-2025
- Business
- Yahoo
ProQR Announces Second Quarter 2025 Operating and Financial Results
Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseases Advancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett Syndrome Hosting 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 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@ contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@ Financial Tables PROQR THERAPEUTICS Condensed Consolidated Statement of Financial PositionJune 30, December 31, 20252024 €1,000€1,000 Assets Property, plant and equipment13,25814,113 Investments in financial assets—— Non-current assets13,258 14,113 Cash and cash equivalents119,765149,408 Prepayments and other receivables3,9313,747 Other taxes583690 Current assets124,279 153,845 Total assets137,537 167,958 Equity and liabilities Equity Equity attributable to owners of the Company66,98388,560 Total equity66,983 88,560 Liabilities Borrowings—— Lease liabilities10,48111,067 Deferred income26,98529,429 Non-current liabilities37,466 40,496 Borrowings4,7274,582 Lease liabilities1,6541,567 Derivative financial instruments290468 Trade payables1,28316 Social securities and other taxes2811,478 Deferred income17,45021,942 Other current liabilities7,4038,849 Current liabilities33,088 38,902 Total liabilities70,554 79,398 Total equity and liabilities137,537 167,958 PROQR THERAPEUTICS Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income (€ in thousands, except share and per share data)Three month periodSix month period ended June 30, ended June 30, 20252024 20252024 €1,000€1,000€1,000€1,000 Revenue3,817 6,305 8,336 10,755 Other income158 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 expense1925136471,001 Results related to financial liabilities measured at fair value through profit or loss(104)195178127 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 outstanding1105,343,89781,665,565105,320,49581,618,038Earnings 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 Condensed Consolidated Statement of Changes in EquityAttributable 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 vested326,455—174(288)—288174 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 vested131,525—67(181)—18167 — Balance at June 30, 2025 107,710,916 4,308 483,879 26,272 297 (447,773)66,983 PROQR THERAPEUTICS Condensed Consolidated Statement of Cash FlowsThree month period Six month period ended June 30, ended June 30, 2025202420252024 €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)— — Depreciation6757111,3531,402 — Share-based compensation9096281,6671,364 — Financial income and expenses(139)(513)(647)(1,001) — Results related to financial liabilities measured at fair value through profit or loss104(195)(178)(127) — Income tax expenses18(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 received6176101,4051,542 Interest paid(52)(190)(261)(379) Cash flow from investing activities Increase in financial asset - current———(17,000) Purchases of property, plant and equipment(101)(267)(325)(999) Cash flow from financing activities Proceeds from exercise of share options—1267174 Repayment of lease liability(293)(294)(860)(875) 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 period132,41485,713149,408118,925 Cash and cash equivalents at the end of the period119,765 78,970 119,765 78,970 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
28-07-2025
- Business
- Yahoo
ProQR Announces Upcoming Presentation at RNA Editing Summit
LEIDEN, Netherlands & CAMBRIDGE, Mass., July 28, 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 announced that it will participate in the RNA Editing Summit taking place July 29-31, 2025, in Boston, MA. 'We recently achieved a key milestone with the submission of the CTA for AX-0810, targeting NTCP for cholestatic diseases, and we're building on this momentum with continued progress in CNS applications of our Axiomer technology,' said Gerard Platenburg, Chief Scientific Officer of ProQR. 'Our presentation at the RNA Editing Summit will feature preclinical NHP data in the CNS, and spotlight our Rett program targeting MECP2, supporting the potential for Axiomer to target severe neurodevelopmental diseases.' RNA Editing Summit July 29-31, 2025 | Boston, MA Title: Pioneering RNA Modification Beyond Rare Diseases by Exploring Novel Editing Technologies & Targeting Multiple Mutations to Remove the Barriers to Treating Common Diseases Presenter: Gerard Platenburg, Chief Scientific Officer Date/Time: July 31, 2025, 3:45 PM ET This presentation will include: An update on the potential of Axiomer in the CNS supported by preclinical long-term and multiple dose NHP data. An overview of the therapeutic applications of Axiomer in the CNS and liver. Presentation materials will be made available in the Publications and Presentations section of the ProQR website at 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 Forward Looking Statements for ProQR 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 participation and presentation at this conference, our business, preclinical 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, our Axiomer RNA editing technology 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, and the potential of our technologies and product candidates, 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 anticipated in 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; 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 or suppliers to supply materials for research and development and the risk of supply interruption or delays from suppliers or contract manufacturers; 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; 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. 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 "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 participation in this conference, our business, technology, strategy, our Axiomer platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides, and the potential of our technologies and product candidates. 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 anticipated in 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@ contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@
Business Upturn
26-06-2025
- Business
- Business Upturn
ProQR Announces CTA Submission for Phase 1 Study of AX-0810 Targeting NTCP
First-in-human trial of AX-0810 will evaluate safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers with initial data expected in Q4 2025 Marks the first submitted Clinical Trial Application (CTA) advancing ProQR's Axiomer™ ADAR-mediated RNA editing platform into clinical development LEIDEN, Netherlands & CAMBRIDGE, Mass., June 26, 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 announced the submission of a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of its lead pipeline program AX-0810 targeting NTCP, a liver cell protein that transports bile acids into cells. AX-0810 is an investigational ADAR-mediated RNA editing oligonucleotide (EON) designed to selectively modulate NTCP function by reducing toxic bile acid accumulation in the liver, potentially mitigating inflammation, fibrosis, and progression toward liver failure, which are common in cholestatic diseases. 'The submission of this CTA marks a significant milestone for ProQR, representing the first to advance our Axiomer RNA editing platform and pipeline into clinical development,' said Cristina Lopez Lopez, MD, PhD Chief Medical Officer of ProQR. 'This step brings us into the clinic with AX-0810 as we initiate a Phase 1 study to evaluate safety and target engagement in healthy volunteers. Cholestatic liver diseases represent an area of high unmet medical need and by targeting NTCP, AX-0810 introduces a novel therapeutic approach underpinned by human genetics and compelling preclinical data.' The proposed Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AX-0810 in healthy adult volunteers. The study will also explore biomarkers to assess early signals of target engagement. Pending regulatory clearance, the study is expected to commence at a single site in the Netherlands with initial data anticipated in Q4 2025. About AX-0810 AX-0810 is an investigational GalNac-delivered RNA editing oligonucleotide designed to treat cholestatic liver disease by targeting NTCP. These diseases are driven by toxic bile acid accumulation due to impaired bile flow, leading to progressive liver damage. AX-0810 offers a novel therapeutic approach by selectively modulating NTCP function, which decreases bile acid reuptake into the liver. This mechanism is supported by human genetics data showing that naturally occurring NTCP variants safety reduce bile acid reuptake, reinforcing the potential of NTCP modulation by ADAR-mediated RNA editing to improve liver health and alter the course of cholestatic disease. 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 Forward Looking Statements for ProQR 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, ; our Axiomer™ RNA editing technology platform, including the continued development and advancement of our Axiomer platform, the advancement of AX-0810 to clinical trials, including the timing of, and our ability to obtain regulatory clearance; our expectations regarding the initiation of the planned Phase 1 clinical study of AX-0810 in the Netherlands, including our ability to recruit for and complete the study, and the anticipated timing of initial data in Q4 2025; the plan, timing, progress and results of our preclinical studies, planned clinical trials and other development activities, including the release of data related thereto, and the potential of our technologies and product candidates. 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 anticipated in 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 or suppliers to supply materials for research and development and the risk of supply interruption or delays from suppliers or contract manufacturers; 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; 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. 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 6228 [email protected] or Investor contact: Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 [email protected]
Yahoo
12-05-2025
- Business
- Yahoo
ProQR Announces Upcoming Scientific Presentations at ASGCT and TIDES Conferences
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 12, 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 announced that it will present at two upcoming scientific conferences: the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, taking place May 13-17, 2025, in New Orleans, Louisiana and the TIDES USA 2025: Oligonucleotide and Peptide Therapeutics Conference, being held May 19-22, 2025, in San Diego, California. "We are excited to share these presentations with the scientific community, demonstrating the breadth and versatility of our Axiomer RNA editing technology," said Gerard Platenburg, Chief Scientific Officer of ProQR. "The data we will present at ASGCT and TIDES further support our vision of building a differentiated and diversified pipeline and advancing RNA editing therapies that could offer transformative benefits for patients." These presentations will highlight the Company's proprietary Axiomer RNA editing platform and pipeline programs. Details of ProQR's presentations are as follows: American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting May 13-17, 2025 | New Orleans, Louisiana, USA Oral Presentation Title: ADAR-Mediated RNA Editing of SLC10A1(NTCP) as a Therapeutic Approach to Reduce Liver Bile Acid Re-Uptake in Cholestatic Diseases (abstract 333) Presenter: Gerard Platenburg, Chief Scientific Officer Date/Time: May 16, 2025, 4:15 PM CT Session: Oligonucleotide Therapeutics II Poster Presentations Title: ADAR-Mediated RNA Editing-Based Correction of PNPLA3 I148M Functionality to Address Hepatic Steatosis (abstract 1654) Presenter: Gerard Platenburg, Chief Scientific Officer Date/Time: May 15, 2025, 5:30-7:00 PM CT Poster Number: AMA1324 Title: ADAR-Mediated RNA Editing of Premature Termination Codon Results in Functional Correction in MECP2 for Rett Syndrome (abstract 1645) Presenter: Gerard Platenburg, Chief Scientific Officer Date/Time: May 15, 2025, 5:30-7:00 PM CT Poster Number: AMA1427 TIDES USA: Oligonucleotide and Peptide Therapeutics Conference May 19-22, 2025 | San Diego, California, USA Podium Presentation Title: Advancing Axiomer ADAR RNA Editing Platform - Editing oligonucleotides optimization for therapeutic use Presenter: Peter A. Beal, PhD, Chief ADAR Scientist Date/Time: May 22, 2025, 2:00 PM PST Session/Track: Genome Editing Technology and Applications Following the conferences, presentation materials will be made available in the Publications and Presentations section of the ProQR website at 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 Forward Looking Statements for ProQR 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 presentations at these conferences, our business, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our Axiomer™ RNA editing technology 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, and the potential of our technologies and product candidates, 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 anticipated in 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; 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 or suppliers to supply materials for research and development and the risk of supply interruption or delays from suppliers or contract manufacturers; 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; 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. 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 "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 participation in this conference, our business, technology, strategy, our Axiomer platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides, and the potential of our technologies and product candidates. 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 anticipated in 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@ contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@ in to access your portfolio
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10-05-2025
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ProQR: Q1 Earnings Snapshot
LEIDEN, Netherlands (AP) — LEIDEN, Netherlands (AP) — ProQR Therapeutics NV (PRQR) on Thursday reported a loss of $10.6 million in its first quarter. The Leiden, Netherlands-based company said it had a loss of 11 cents per share. The results missed Wall Street expectations. The average estimate of three analysts surveyed by Zacks Investment Research was for a loss of 9 cents per share. The Dutch drugmaker posted revenue of $4.8 million in the period. _____ This story was generated by Automated Insights ( using data from Zacks Investment Research. Access a Zacks stock report on PRQR at Sign in to access your portfolio
