LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 09, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that Dinko Valerio, a co-founder of the Company, and Alison Lawton will rotate off the Board at the Company’s next Annual General Meeting (AGM) as their terms conclude. The planned changes to ProQR’s Board compositio ...

Core Insights - ProQR Therapeutics announced initial safety and pharmacokinetic (PK) data from its Phase 1 trial of AX-0810, indicating a positive early milestone for the company [2][5] - The company is advancing its pipeline with new development candidates for AX-2402 and AX-2911, targeting Rett syndrome and metabolic-associated steatohepatitis (MASH) respectively [7][12] AX-0810 Phase 1 Study - AX-0810 is a lead investigational RNA editing oligonucleotide targeting NTCP for cholestatic diseases [3][16] - The Phase 1 study is a randomized, double-blind, placebo-controlled trial with up to 33 participants, assessing safety, tolerability, and pharmacokinetics [4] - Initial data from the first cohort (3 mg/kg) showed no serious adverse events and PK observations aligned with non-clinical data, supporting continued dosing [5][9] Pipeline Progress - ProQR has selected development candidates for AX-2402 targeting MECP2 for Rett syndrome and AX-2911 targeting PNPLA3 for MASH [10][12] - AX-2402 demonstrated significant functional improvements in a mouse model, supported by funding from the Rett Syndrome Research Trust [11] - AX-2911 showed over 80% reduction in hepatic fat content in a humanized mouse model, outperforming a clinical-stage therapy [13] Strategic Collaboration - The collaboration with Eli Lilly achieved $4.5 million in milestones in 2025, validating ProQR's Axiomer platform and providing non-dilutive capital [14] Corporate Outlook - ProQR plans to report target engagement data for AX-0810 in the first half of 2026 and initiate a patient cohort in the trial [6][18] - The company aims to advance AX-2402 to a first-in-human trial in the first half of 2027 and continue to strengthen its financial position [18]

ProQR Therapeutics Conference Call Summary Company Overview - ProQR Therapeutics is a biopharmaceutical company based in the Netherlands, focused on developing RNA editing medicines to treat genetic and common diseases by modifying human messenger RNA [1][2]. Core Technology and Partnerships - The company has developed a proprietary RNA editing technology over the past 10 years and controls all foundational intellectual property [2]. - In 2021, ProQR entered a $4 billion collaboration with Eli Lilly, receiving $125 million upfront and the potential for $250 million in milestone payments for each of the 15 targets [2]. Pipeline and Clinical Programs - ProQR is pursuing a wholly owned pipeline focusing on liver and CNS indications, with the lead program AX-0810 targeting cholestatic diseases [3]. - The company has initiated a clinical study for AX-0810, expecting first human clinical data in the first half of next year [3][11]. Trial Design and Expectations - The AX-0810 trial will enroll 33 healthy volunteers across three cohorts, with a randomized, placebo-controlled, and double-blinded design [10]. - Initial safety and pharmacokinetic data are expected by the end of this year, while pharmacodynamic data will be available in the first half of next year [11]. Unmet Medical Needs - The target diseases, Primary Sclerosing Cholangitis (PSC) and Biliary Atresia (BA), have no approved therapies and represent a significant unmet medical need [7][14]. - The company aims to develop a medicine that addresses this need while generating a robust dataset to validate its technology [8]. Safety and Efficacy Considerations - ProQR believes that pruritus (itching) associated with high bile acid levels is driven by inflammation rather than bile acids themselves, supported by data from healthy individuals with high bile acid levels who do not experience pruritus [17][22]. - The company aims for a twofold increase in bile acid levels in serum to halt disease progression, with a linear correlation between editing percentage and bile acid concentration [23][25]. Future Development Plans - Following the healthy volunteer study, ProQR plans to conduct a Phase 1B study in PSC patients, with results expected before the end of next year [13]. - The company is exploring accelerated development plans for potential approval based on efficacy data [31]. Rett Syndrome Program - ProQR is also developing a program for Rett syndrome, a neurodevelopmental disease caused by the absence of the MECP2 protein, with the potential to restore normal function through RNA editing [34]. - The program is co-financed by the Rett Syndrome Research Trust, and a clinical candidate is expected to be selected soon [35]. Differentiation from Gene Therapy - The RNA editing approach allows for precise restoration of the MECP2 protein without the risk of overexpression, which is a concern in gene therapy [37]. - ProQR plans to target specific mutations, particularly stop codon mutations, which affect approximately 5,000 patients each [38]. Conclusion - ProQR Therapeutics is positioned to address significant unmet medical needs in liver diseases and Rett syndrome through innovative RNA editing technologies, with ongoing clinical trials and strategic partnerships enhancing its development pipeline [1][2][34].

Core Insights - ProQR Therapeutics is participating in the 8th Annual Evercore Healthcare Conference on December 4, 2025, at 8:20 am ET, indicating its active engagement in the healthcare investment community [1] Company Overview - ProQR Therapeutics is focused on developing transformative RNA therapies using its proprietary Axiomer technology platform, which utilizes ADAR-mediated RNA editing to create targeted medicines for various diseases [3][4] - The Axiomer platform allows for specific single nucleotide changes in RNA, potentially reversing mutations or modulating protein expression, which could lead to new treatments for both rare and common diseases [3][4] Technology Details - Axiomer employs Editing Oligonucleotides (EONs) to facilitate precise edits in RNA, converting Adenosine (A) to Inosine (I), which is then translated as Guanosine (G), thereby correcting disease-causing mutations [3] - The technology aims to leverage the body's own molecular machinery to develop a new class of medicines, addressing unmet medical needs [4]

Core Insights - Biotech and healthcare stocks experienced significant movements in after-hours trading, driven by corporate updates and investor anticipation [1] Company Updates - Butterfly Network, Inc. (BFLY) shares increased by 6.04% to $2.81 after a 14.22% rise to $2.65 at market close, following the announcement of CEO Joseph DeVivo and Interim CFO Megan Carlson's participation in the 8th Annual Evercore Healthcare Conference [2] - Citius Pharmaceuticals Inc. (CTXR) saw a 7.90% increase to $1.23 after a 15.15% rally to $1.14, attributed to a deeper collaboration with Verix for the commercialization of LYMPHIR, an FDA-approved immunotherapy expected to launch in Q4 2025 [3] - ProQR Therapeutics N.V. (PRQR) rose 6.80% to $2.20 after a flat close at $2.06, with no specific news indicating that the movement may be due to investor sentiment [4] - Perrigo Company plc (PRGO) advanced 3.97% to $13.14, building on a 3.10% gain at the close, reflecting steady investor interest despite no new announcements [4] - Tempest Therapeutics, Inc. (TPST) added 4.86% to $3.67 after reversing a decline, following the announcement of plans to acquire CAR-T programs from Factor Bioscience, expected to close in early 2026 [5] - Iterum Therapeutics plc (ITRM) rose 2.77% to $0.3969 after a 4.38% gain at the close, with no new updates released [6] - Processa Pharmaceuticals, Inc. (PCSA) edged up 1.86% to $0.26 despite a steep decline at the close, with no news issued [6]

Group 1 - The article does not contain relevant content regarding company or industry insights [1]

Financial Performance - Revenue for the three-month period ended September 30, 2025, was €2,882,000, a decrease of 25% compared to €3,843,000 in the same period of 2024[4]. - The net result for the nine-month period ended September 30, 2025, was a loss of €33,264,000, compared to a loss of €18,458,000 in 2024, reflecting an increase in losses of 80%[4]. - The total comprehensive income for the nine-month period ended September 30, 2025, was a loss of €34,340,000, compared to a loss of €18,559,000 in 2024, reflecting an increase in losses of 85%[4]. - Basic loss per share for the nine-month period ended September 30, 2025, was €0.32, compared to €0.23 in 2024, indicating a 39% increase in loss per share[4]. Operating Costs - Total operating costs for the nine-month period ended September 30, 2025, were €45,980,000, up 30% from €35,493,000 in 2024[4]. - Research and development costs for the nine-month period ended September 30, 2025, were €34,811,000, an increase of 35% from €25,745,000 in 2024[4]. - General and administrative costs for the nine-month period ended September 30, 2025, were €11,169,000, compared to €9,748,000 for the same period in 2024[62]. - The Company recognized €2,784,000 in share-based compensation expenses for the nine-month period ended September 30, 2025, up from €1,976,000 in the prior year[51]. Cash and Equivalents - Cash and cash equivalents at the end of the period on September 30, 2025, were €106,883,000, down 29% from €149,408,000 at the end of 2024[3]. - Cash used in operations for the nine-month period ended September 30, 2025, was €40,928,000, an increase of 41% from €29,046,000 in 2024[8]. - As of September 30, 2025, the company's cash and cash equivalents were €106,883,000, a decrease from €149,408,000 at December 31, 2024[32]. Equity and Liabilities - Total equity attributable to owners of the Company decreased to €57,071,000 as of September 30, 2025, down 36% from €88,560,000 at the end of 2024[3]. - Other current liabilities at September 30, 2025, amount to €8,550,000, slightly down from €8,849,000 at December 31, 2024[36]. - The company has total borrowings of €4,800,000 as of September 30, 2025, compared to €4,582,000 at December 31, 2024[37]. - The Company has a current income tax liability of €nil as of September 30, 2025, with no significant temporary differences between accounting and tax results[66]. Deferred Revenue and Income - The total deferred revenue balance related to the collaboration agreement with Eli Lilly amounts to €41,492,000 as of September 30, 2025, down from €50,930,000 at December 31, 2024[26]. - The company's total deferred income at September 30, 2025, is €41,519,000, compared to €51,371,000 at December 31, 2024[44]. Share Issuance and Financing - The Company completed a public offering of 18,000,000 ordinary shares at a price of $3.50 per share, generating gross proceeds of approximately $69,790,000 (€64,600,000) after a partial exercise of the underwriters' option[47]. - A concurrent private placement with Lilly resulted in the sale of 3,523,538 ordinary shares for total gross proceeds of approximately $12,300,000 (€11,400,000)[49]. - The company has not issued any shares under the ATM facility as of September 30, 2025, despite having a maximum aggregate offering price of $300,000,000 available[46]. Partnerships and Collaborations - The Company reached milestones amounting to $2,000,000 (€1,781,000) under the collaboration agreement with Lilly during the nine-month period ended September 30, 2025[56]. - The Company received an upfront payment of $60,000,000 (€56,412,000) from Lilly in February 2023 as part of an expanded collaboration agreement[55]. - The Company expanded its partnership with RSRT in December 2024, which includes an additional $8,150,000 in funding to support clinical trials[59]. Company Outlook - The company expects to continue as a going concern based on its existing funding and projected cash flows[16]. - The company operates in one reportable segment focused on the discovery and development of innovative RNA-based therapeutics[17]. - The company’s financial results are expected to vary significantly from period to period, with no particular seasonal factors affecting its ordinary activities[15].

Core Insights - ProQR Therapeutics reported its financial and operational results for Q3 2025, highlighting significant advancements in its RNA editing technology platform, Axiomer, and the initiation of a first-in-human study for its lead program AX-0810 [1][2]. Financial Highlights - As of September 30, 2025, ProQR held cash and cash equivalents of approximately €106.9 million, a decrease from €149.4 million at the end of 2024 [4]. - The net cash used in operating activities for the nine-month period ended September 30, 2025, was €39.4 million, compared to €27.0 million for the same period in 2024 [4][6]. - The net loss for the nine-month period was €33.3 million, or €0.32 per diluted share, compared to a net loss of €18.5 million, or €0.23 per diluted share, for the same period last year [7][16]. Recent Progress and Upcoming Events - The company received CTA authorization for the Phase 1 trial of AX-0810, targeting NTCP for cholestatic diseases, and is initiating the study in healthy volunteers [4][5]. - Initial safety, tolerability, and pharmacokinetic (PK) data from Cohort 1 are expected by year-end 2025, with target engagement data anticipated in the first half of 2026 [5]. - ProQR's Axiomer pipeline includes programs targeting various diseases, such as AX-2402 for Rett syndrome and AX-2911 for fatty liver disease, with updates expected in the coming months [5]. Collaboration and Milestones - The company achieved certain milestones in its collaboration with Eli Lilly, amounting to $2.0 million (~€1.8 million) [6]. - ProQR continues to execute its partnership with Eli Lilly, with potential data updates and milestone income from the ongoing collaboration [5]. Research and Development Costs - Research and development costs for the nine-month period ended September 30, 2025, were €34.8 million, compared to €25.7 million for the same period last year [6]. - General and administrative costs were €11.2 million for the same period, up from €9.7 million in 2024 [6].

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Core Points - ProQR Therapeutics hosted a virtual investor and analyst event to discuss company updates and future prospects [1] Group 1 - The event was led by Sarah Kiely, Vice President of Investor Relations and Corporate Affairs at ProQR Therapeutics [1]