ProQR Announces CTA Submission for Phase 1 Study of AX-0810 Targeting NTCP
ProQR Therapeutics (Nasdaq: PRQR) has submitted a Clinical Trial Application (CTA) to the European Medicines Agency for its first-in-human Phase 1 trial of AX-0810. This marks the first clinical development milestone for ProQR's Axiomer™ RNA editing platform.
The trial will evaluate AX-0810, an ADAR-mediated RNA editing oligonucleotide targeting NTCP, a liver cell protein. The study will assess safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers, with initial data expected in Q4 2025. The treatment aims to reduce toxic bile acid accumulation in the liver for patients with cholestatic diseases.
ProQR Therapeutics (Nasdaq: PRQR) ha presentato una domanda di sperimentazione clinica (CTA) all'Agenzia Europea per i Medicinali per la sua prima sperimentazione di fase 1 sull'uomo di AX-0810. Questo rappresenta il primo traguardo nello sviluppo clinico della piattaforma di editing RNA Axiomer™ di ProQR.
Lo studio valuterà AX-0810, un oligonucleotide di editing RNA mediato da ADAR che prende di mira NTCP, una proteina delle cellule del fegato. La sperimentazione analizzerà sicurezza, tollerabilità, farmacocinetica e coinvolgimento del target in volontari sani, con dati iniziali attesi nel quarto trimestre 2025. Il trattamento mira a ridurre l'accumulo tossico di acidi biliari nel fegato nei pazienti con malattie colestatiche.
ProQR Therapeutics (Nasdaq: PRQR) ha presentado una solicitud de ensayo clínico (CTA) a la Agencia Europea de Medicamentos para su primer ensayo de fase 1 en humanos de AX-0810. Esto marca el primer hito en el desarrollo clínico de la plataforma de edición de ARN Axiomer™ de ProQR.
El ensayo evaluará AX-0810, un oligonucleótido de edición de ARN mediado por ADAR que apunta a NTCP, una proteína de las células hepáticas. El estudio evaluará la seguridad, tolerabilidad, farmacocinética y la interacción con el objetivo en voluntarios sanos, con datos iniciales esperados en el cuarto trimestre de 2025. El tratamiento pretende reducir la acumulación tóxica de ácidos biliares en el hígado en pacientes con enfermedades colestásicas.
ProQR Therapeutics (나스닥: PRQR)는 유럽 의약품청(EMA)에 AX-0810의 최초 인간 대상 1상 임상시험 신청서(CTA)를 제출했습니다. 이는 ProQR의 Axiomer™ RNA 편집 플랫폼에서 첫 임상 개발 이정표입니다.
이번 임상시험은 ADAR 매개 RNA 편집 올리고뉴클레오티드인 AX-0810을 평가하며, 간세포 단백질인 NTCP를 표적으로 합니다. 연구는 건강한 지원자를 대상으로 안전성, 내약성, 약동학 및 표적 결합을 평가하며, 초기 데이터는 2025년 4분기에 예상됩니다. 이 치료법은 담즙 정체성 질환 환자의 간 내 독성 담즙산 축적을 줄이는 것을 목표로 합니다.
ProQR Therapeutics (Nasdaq : PRQR) a soumis une demande d'essai clinique (CTA) à l'Agence européenne des médicaments pour son premier essai de phase 1 chez l'humain d'AX-0810. Cela marque une première étape dans le développement clinique de la plateforme d'édition ARN Axiomer™ de ProQR.
L'essai évaluera AX-0810, un oligonucléotide d'édition ARN médié par ADAR ciblant NTCP, une protéine des cellules hépatiques. L'étude portera sur la sécurité, la tolérance, la pharmacocinétique et l'engagement de la cible chez des volontaires sains, avec des premières données attendues au quatrième trimestre 2025. Le traitement vise à réduire l'accumulation toxique d'acides biliaires dans le foie chez les patients atteints de maladies cholestatiques.
ProQR Therapeutics (Nasdaq: PRQR) hat einen Antrag auf Durchführung einer klinischen Studie (CTA) bei der Europäischen Arzneimittelagentur für seine erste klinische Phase-1-Studie am Menschen mit AX-0810 eingereicht. Dies markiert den ersten klinischen Entwicklungsschritt für die Axiomer™ RNA-Editing-Plattform von ProQR.
Die Studie wird AX-0810 evaluieren, ein ADAR-vermitteltes RNA-Editing-Oligonukleotid, das auf NTCP, ein Leberzellprotein, abzielt. Die Untersuchung bewertet Sicherheit, Verträglichkeit, Pharmakokinetik und Zielbindung bei gesunden Freiwilligen, mit erwarteten ersten Daten im vierten Quartal 2025. Die Behandlung zielt darauf ab, die toxische Anhäufung von Gallensäuren in der Leber bei Patienten mit cholestatischen Erkrankungen zu reduzieren.
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Insights
ProQR's CTA submission for AX-0810 marks first clinical advancement of their RNA editing platform, targeting liver disease with Q4 2025 data expected.
ProQR has submitted a Clinical Trial Application for AX-0810, advancing their proprietary Axiomer™ RNA editing technology into clinical testing for the first time. This represents a significant development milestone for the company's platform technology.
AX-0810 targets the NTCP protein that transports bile acids into liver cells. By employing ADAR-mediated RNA editing, the candidate aims to modulate NTCP function to reduce toxic bile acid accumulation in the liver. This mechanism could potentially address the inflammation, fibrosis, and progression toward liver failure that characterize cholestatic diseases.
The pending Phase 1 study will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers, with initial data expected in Q4 2025. Importantly, the trial will assess biomarkers for target engagement, providing early indicators of whether the approach is working as designed.
The therapeutic rationale appears scientifically sound, with the company noting the approach is underpinned by human genetics and preclinical evidence. Cholestatic liver diseases represent conditions with significant unmet medical needs, where patients experience bile flow impairment leading to progressive liver damage.
While this CTA submission represents a crucial step forward, it's worth noting this is still early-stage development. The regulatory review process must first clear the application before the trial can commence at the planned Netherlands site.
- 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 www.proqr.com.
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 Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
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Investor contact:
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LifeSci Advisors
T: +1 617 430 7579
pkelleher@lifesciadvisors.com
FAQ
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