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2 days ago
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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
31-07-2025
- Business
- Yahoo
WAVE Life Sciences Ltd (WVE) Q2 2025 Earnings Call Highlights: Progress in RNA Editing Amid ...
Release Date: July 30, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Positive Points WAVE Life Sciences Ltd (NASDAQ:WVE) has made significant progress in RNA editing, particularly with their AATD clinical program and Eli clinical program for obesity. The company has delivered positive data from their forward 53 clinical trial of N531 for DMD, showing statistically significant improvements. WVE 006, their RNA editing oligonucleotide for alpha one antitrypsin deficiency, has shown impressive durability and a favorable safety profile. The company has expanded its second cohort for the WVE 007 obesity program due to favorable safety and robust active reduction. WAVE Life Sciences Ltd (NASDAQ:WVE) has a strong cash position, with $208.5 million in cash and cash equivalents, expected to fund operations into 2027. Negative Points The company's revenue for the second quarter of 2025 decreased to $8.7 million from $19.7 million in the prior year quarter. Research and development expenses increased to $43.5 million, driven by spending in the inhibie program and RNA editing programs. General and administrative expenses rose to $18 million, primarily due to share-based compensation and other external expenses. The net loss for the second quarter of 2025 was $50.5 million, compared to a net loss of $32.9 million in the prior year quarter. There is uncertainty regarding the potential for future milestones and payments under the GSK collaboration, which are not included in the cash runway. Q & A Highlights Warning! GuruFocus has detected 5 Warning Signs with WVE. Q: For the inhibitE program, can you elaborate on your reasons for expanding cohort 2 over advancing to cohort 3 sooner? If the cohort 2 dose was well tolerated, why not just advance to cohort 3 versus expanding cohort 2? A: We didn't wait to start cohort 3; we're already dosing the 400 mg cohort. The focus on cohort 2 is due to its alignment with our DIO weight loss data and active and knee reduction modeling. Cohort 2 was modeled to align with weight loss similar to semaglutide based on the DIO model. We are encouraged by the clinical translation and are optimistic about the upcoming data readout. Q: Regarding the 006 AGD data readout, what is your guidance on the different expectations from the single-dose to multi-dose data readout? A: We expect larger liver concentration and exposure with the 200 mg multi-dose compared to the 400 mg single dose. We are encouraged by the M protein and total protein levels from the single dose and expect to see more with multi-dosing. The combination of M and total protein will provide good insights, similar to the early single-dose data. Q: On the DMD program, there were updates at the FDA level this morning. Would that affect your approach or strategy with regards to your DMD program? A: Our program goes to Cedar, which has a new division director. This division established the threshold for accelerated approvals for exon-skipping therapies. We are watching the agency's discussions, but there is nothing imminent suggesting changes to our strategy. Q: Can you discuss the extent to which you've focused on optimizing 006 so that it's competitive with others developing RNA editing therapeutics for AATD? A: We've developed 006 to have substantial editing properties that translate from pre-clinical to clinical data. Our optimized chemistry, specifically for Adar editing, and the use of Galnek for efficient delivery, differentiate our approach. This allows for subcutaneous administration and high hepatocyte uptake, distinguishing our program as best in class. Q: Regarding the ATD program, what do you believe the target conversion rate from Z to M should be? Are you looking for a near-complete conversion, or is there an acceptable amount of residual Z protein? A: The goal is to convert ZZ patients to MZ phenotype, providing sufficient protection against hepatic and lung disease. We achieved over 60% edited M protein versus Z with a single dose, which is encouraging. The target is to reach a 50% correction, aligning with the desired MZ phenotype. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. 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@
Yahoo
09-05-2025
- Business
- Yahoo
WAVE Life Sciences Ltd (WVE) Q1 2025 Earnings Call Highlights: Clinical Advancements and ...
Release Date: May 08, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. WAVE Life Sciences Ltd (NASDAQ:WVE) has made significant progress in its clinical pipeline, particularly in obesity, AATD, DMD, and HD programs. The company reported promising 48-week clinical results for WVE-531 in DMD, showing statistically significant improvements in muscle health and function. WVE-007 for obesity has shown potential for sustainable weight loss without muscle loss, differentiating it from current GLP-1 treatments. WVE-006 for AATD demonstrated RNA editing in humans, with promising durability and safety profiles. The company has a strong cash position, with $243.1 million in cash and equivalents, expected to fund operations into 2027. WAVE Life Sciences Ltd (NASDAQ:WVE) reported a decrease in revenue for Q1 2025 compared to the previous year, primarily due to timing of revenue recognition under a collaboration agreement. Research and development expenses increased significantly, contributing to a higher net loss of $46.9 million for the quarter. The company faces risks and uncertainties related to forward-looking statements and regulatory approvals, particularly for accelerated approval pathways. There is potential competition and differentiation challenges in the RNA editing space, particularly with DNA editing technologies. The company has not yet disclosed specific time points for data cutoffs in its clinical trials, which may impact investor confidence. Warning! GuruFocus has detected 4 Warning Signs with WVE. Q: For the InhibiE program, what's the trigger for data disclosure? Would you need to have completed dosing in all five cohorts for the disclosure, or once you have reached some other internal threshold? A: The disclosure will be triggered based on internal cutoffs at specific time points, such as 1 month, 3 months, and 6 months. We haven't specified which of these will be the time point for disclosure, but it will include target engagement, weight loss, and biomarkers. Q: You have a few drugs slated for accelerated approval, including for DMD and Huntington's. Can you confirm that they are all under CEDAR and not CBER, and any risks to the accelerated approval process for you guys? A: Yes, they are under CEDAR, not CBER. Our discussions with the agency have been consistent, and the accelerated approval pathway remains open. We are taking a comprehensive approach to advancing our programs, focusing on both biomarker and clinical data. Q: On the Huntington's program, the recent data from a competitor seems to imply that MRI of the caudate may not be as consistent. Any thoughts there? A: MRI has been highly consistent in our analyses and others, such as TRACK-HD and PREDICT-HD. Our allele-selective approach shows a strong correlation between mutant Huntington reduction and slowing of caudate atrophy, which is a key anatomical endpoint. Q: For the AATD program, even with single-dose data at the lowest dose, you're getting to the 11 micromolar threshold for total AAT protein. Do you think there are additional benefits to getting above that threshold? A: Yes, there are opportunities to continue correcting protein levels, not just for lung health but also for clearing hepatic aggregates. The 200 mg multi-dose data will be crucial in understanding how much higher we can push those levels. Q: How are you interpreting your increase in serum Z protein versus the significant decrease in M protein? What do you hypothesize is happening to the Z protein aggregates in the liver with DNA-based editing versus RNA-based editing? A: The Z protein increase is likely due to breaking up liver aggregates, which aligns with our preclinical models. RNA editing allows for functional protein production, which helps clear aggregates, unlike DNA editing, which may not effectively break down these aggregates. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
