[Translation] A single-center, randomized, open-label, two-sequence, two-period crossover, single-dose, fasting and postprandial bioequivalence study of tauroursodeoxycholic acid capsules in healthy subjects.

主要目的：比较空腹和餐后给药条件下受试制剂福建省宝诺医药研发有限公司提供的牛磺熊去氧胆酸胶囊（规格：0.25 g ) 与参比制剂Bruschettini S.r.l （意大利贝斯迪大药厂）持证的牛磺熊去氧胆酸胶囊（商品名：滔罗特®/TAUROLITE®；规格：250mg ) 在中国健康人群吸收程度和吸收速度的差异，评价两制剂的生物等效性。次要目的：评价空腹及餐后条件下，受试制剂与参比制剂在中国健康试验参与者体内的安全性。

[Translation]

Primary objective: To compare the absorption extent and rate of absorption of the test formulation (Tauroursodeoxycholic acid capsules, 0.25 g) provided by Fujian Baonuo Pharmaceutical R&D Co., Ltd., and the reference formulation (Tauroursodeoxycholic acid capsules, 250 mg, certified by Bruschettini S.r.l., Italy) in a healthy Chinese population under fasting and postprandial administration conditions, and to evaluate the bioequivalence of the two formulations. Secondary objective: To evaluate the safety of the test and reference formulations in healthy Chinese participants under fasting and postprandial administration conditions.

Start Date26 May 2026

Sponsor / Collaborator

Fujian Boro Pharmaceutical Research Co., Ltd.

NCT07517276

/ Not yet recruitingPhase 2IIT

A Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial of Maternal Oral TUDCA in High-Risk Lactating Mother-Infant Dyads Identified by Early Postpartum Breast Milk Bile Acid Hydrophobicity Index

This is a randomized, double-blind, placebo-controlled Phase 2 proof-of-concept trial in mother-infant dyads. The study aims to evaluate the safety, tolerability, and biological effects of maternal oral tauroursodeoxycholic acid (TUDCA) in lactating mothers with metabolic dysfunction-associated steatotic liver disease (MASLD).
Eligible mother-infant dyads will be screened in the early postpartum period using breast milk bile acid hydrophobicity index. Dyads identified as high risk will be randomized 1:1 to maternal oral TUDCA or placebo.
The primary objectives are to assess maternal and infant safety and to evaluate changes in breast milk bile acid hydrophobicity index. Secondary objectives include assessment of infant ketone-related metabolic biomarkers and gut microbiome features. Exploratory outcomes include early infant neurodevelopment during follow-up.

Start Date02 Apr 2026

Sponsor / Collaborator

Peking University First Hospital

NCT07328009

/ RecruitingPhase 2IIT

The HCC-SIGHT: A Therapy Platform Trial for Hepatocellular Carcinoma Guided by Stratification Imaging and Genetic Horizon Testing

This is a phase II, multi-arm, Bayesian adaptive platform trial designed to efficiently evaluate novel therapies for advanced hepatocellular carcinoma (HCC) after first-line treatment failure. The study aims to rapidly identify the most effective investigational regimens and discover predictive biomarker signatures (from tumor tissue, blood, and imaging) to guide personalized second-line therapy.

Start Date19 Jan 2026

Sponsor / Collaborator

Fudan University

100 Clinical Results associated with Tauroursodeoxycholate sodium

100 Translational Medicine associated with Tauroursodeoxycholate sodium

100 Patents (Medical) associated with Tauroursodeoxycholate sodium

2,553

Literatures (Medical) associated with Tauroursodeoxycholate sodium

01 Jan 2027·THERIOGENOLOGY

Mitigation of ER stress improves bovine in vitro embryo development and pregnancy rates

Article

Author: Agostini Xavier, Livia ; da Costa, Daniele Campos Araújo ; Bordignon, Vilceu ; Gutierrez, Karina ; Souza de Aguiar, Maria H ; Glanzner, Werner G ; Barreta, Marcos Henrique ; Lemos do Prado Welter, Nicole ; Manenti, Nadielle P ; Fontana Goetten, André ; Braga Rissi, Vitor

Endoplasmic reticulum (ER) stress compromises cellular homeostasis in multiple cell types, gametes and early embryos are particularly susceptible, with negative consequences for developmental competence. This study investigated the effects of ER stress and its attenuation by TUDCA at specific developmental stages of bovine embryos and evaluated ER stress-modulating compounds in a commercially in vitro embryo production (IVEP) system. To induce ER stress, embryos were exposed to tunicamycin (TN; 0.1, 1, and 10 ng/mL) during in vitro culture (IVC). Stage-specific sensitivity to ER stress was assessed by treating embryos 1 ng/mL TN during periods associated with embryonic genome activation (EGA): pre-EGA (days 1-3), EGA (days 3-5), and post-EGA (days 5-7). The ability of the bile acid tauroursodeoxycholic acid (TUDCA) to alleviate ER stress was then examined. Embryos received combined TN and TUDCA treatments either throughout culture or specifically during EGA, and outcomes were compared with TN alone. Finally, TUDCA supplementation was tested under commercial IVEP conditions. TN at 1 and 10 ng/ml dose-dependently reduced embryo development during in vitro culture (Days 1-7). Embryo development and total cell number were also decreased by 1 ng/ml TN during EGA (Days 3-5). TUDCA supplementation (100 μM) increased total cell number and the proportion of expanded blastocysts compared to controls and effectively mitigated TN-induced damage. In commercial IVEP, TUDCA supplementation increased blastocyst yield by ∼11% in beef cattle and improved pregnancy rates by 24.1% in dairy cattle. These results highlight the importance of ER stress during key embryo developmental stages and support the use of TUDCA as a beneficial supplement to enhance outcomes in commercial bovine IVEP systems.

01 Dec 2026·BIORESOURCE TECHNOLOGY

Genetic algorithm-guided sample design enables efficient machine learning-driven optimization of tauroursodeoxycholic acid production in engineered Escherichia coli

Article

Author: Jin, Lina ; Yang, Li ; Zhao, Shujuan ; Li, Chenghan ; Wang, Zhengtao

Data-driven machine learning (ML) approaches offer powerful tools for modeling and optimizing complex fermentation processes, yet their application is often limited by the need for large experimental datasets, which is difficult to meet in low-throughput microbial fermentation systems. Here, we present a practical genetic algorithm (GA)-assisted adaptive sampling strategy to support sample-efficient ML-assisted optimization of microbial biotransformation processes. In this strategy, GA is employed upstream to iteratively select informative experimental conditions and generate a compact dataset for artificial neural network (ANN) modeling, rather than relying on uniformly distributed or predefined experimental designs. Using tauroursodeoxycholic acid (TUDCA) biotransformation in engineered Escherichia coli as a case study, the proposed GA-assisted adaptive sampling strategy coupled with ANN (GA-ANN) workflow identified favorable fermentation medium compositions under a limited experimental budget. Experimental validation yielded a maximum TUDCA/TCDCA ratio of 2.18 ± 0.06 and a TUDCA titer of 7.11 ± 0.12 g/L using 20 g/L chicken bile powder as substrate. ANN-based response surface analysis further revealed a biphasic effect of corn dextrin concentration on TUDCA conversion, indicating a nonlinear behavior that was not fully captured by quadratic regression models. This study provides a feasible and sample-efficient strategy for low-throughput fermentation optimization under constrained experimental resources.

01 Dec 2026·POULTRY SCIENCE

Tauroursodeoxycholic acid alleviates LPS-induced intestinal injury by reducing endoplasmic reticulum stress and restoring Mucin2 in broilers

Article

Author: Zhang, Xinran ; Xu, Haoqing ; Wang, Guanran ; Song, Zhigang ; Zhou, Huajin ; Sun, Haojian

Lipopolysaccharide (LPS)-induced intestinal inflammation in broilers is closely associated with endoplasmic reticulum (ER) stress and mucus barrier disruption. This study investigated the effects of tauroursodeoxycholic acid (TUDCA) on LPS-induced intestinal injury in broilers using a 2 × 2 factorial design with LPS challenge (0 or 1 mg/kg BW) and TUDCA administration (0 or 10 mg/kg BW). Ninety-six male Arbor Acres broilers were randomly assigned to four treatment groups, and intraperitoneal injections were administered from day 7 to day 9. Serum endotoxin and inflammatory cytokines were determined, along with ileal goblet cell density, Mucin2 content, and the gene expression of Mucin2. In addition, ER stress-related gene expression, particularly components of the PERK-eIF2α pathway, and ileal microbiota composition were evaluated. The results showed that LPS challenge significantly increased serum endotoxin and the concentrations of IL-1β, IL-8, and TNF-α, with significant LPS × TUDCA interaction effects detected (P < 0.05). TUDCA treatment reduced circulating endotoxin and these inflammatory cytokines across treatments (P < 0.05). LPS challenge significantly reduced ileal goblet cell density and decreased Mucin2 content and Mucin2 gene expression, whereas TUDCA treatment increased these indices (P < 0.05). LPS challenge increased the expression of ER stress-related genes, particularly Grp78, while significant LPS × TUDCA interactions were observed for Grp78 and eIF2α (P < 0.05). TUDCA treatment reduced IRE1 expression (P < 0.05) and tended to decrease PERK expression (P = 0.066). TUDCA significantly increased the relative abundance of Lactobacillus (P = 0.015), with a significant LPS × TUDCA interaction (P = 0.006). Correlation analysis revealed that Lactobacillus abundance was negatively associated with serum IL-1β, while IL-8 and TNF-α were positively correlated with ER stress-related genes (Grp78, PERK, and eIF2α), suggesting a link between inflammatory responses and ER stress activation in the ileum. In conclusion, TUDCA alleviated LPS-induced intestinal injury in broilers by improving mucus barrier function and modulating the transcription of ER stress-related genes, particularly those associated with PERK-eIF2α signaling. These effects were accompanied by an increase in Lactobacillus abundance, suggesting the potential of TUDCA for maintaining intestinal homeostasis under inflammatory conditions.

149

News (Medical) associated with Tauroursodeoxycholate sodium

11 Sep 2026

·www.icepharma.com

The History of Bile Acids: From Traditional Chinese Medicine to Modern Pharmaceuticals

The history of bile acids is a remarkable journey spanning more than two millennia, from the apothecary traditions of ancient China to the sophisticated pharmaceutical pipelines of the 21st century. What began as an empirical understanding of animal bile has evolved, through centuries of scientific enquiry, into one of the most clinically significant and therapeutically versatile classes of molecules in modern medicine. Today, bile acid science sits at the heart of treatments for liver disease, gallstone dissolution, and emerging therapies for metabolic disorders. The story of how we arrived here is as compelling as the chemistry itself. Long before scientists had the tools to isolate or characterise individual molecules, practitioners of Traditional Chinese Medicine (TCM) had identified animal bile as a valuable therapeutic substance. The use of bear bile is documented as far back as 659 CE in the Tang Ben Cao (Newly Revised Materia Medica), one of the most important pharmacological texts of the Tang Dynasty. Within the framework of TCM, bear bile was described as being able to “clear heat” and “relieve toxin”. While these concepts are distinct from modern biomedical terminology, contemporary research has identified anti-inflammatory and hepatoprotective properties in ursodeoxycholic acid (UDCA), one of the major bile acids found in bear bile. Bear bile was prescribed for a variety of conditions, including: Jaundice and liver disorders Eye ailments Haemorrhoids Inflammatory conditions Over centuries of practice, bear bile earned the designation “king of animal biles” among many TCM practitioners. Modern chemical analysis suggests that its perceived therapeutic value may be related, at least in part, to the unusually high concentration of ursodeoxycholic acid (UDCA) found in bear bile. Other animal biles, including those derived from cattle, pigs, snakes, and chickens, also featured prominently in traditional pharmacopoeias, each recognised as possessing distinct medicinal properties. This diversity of use reflects a form of observation-led pharmacology that, while lacking the analytical tools of modern chemistry, was nevertheless grounded in genuine biological activity. The transition from traditional knowledge to scientific understanding began in earnest during the early twentieth century. German chemist Heinrich Otto Wieland played a central role in this transformation. Beginning his investigations into bile acids in 1912, Wieland and his collaborators undertook a series of painstaking chemical studies to determine the structures of cholic acid and related compounds. Their work revealed that bile acids belong to the steroid family of molecules and share a close structural relationship with cholesterol, fundamentally reshaping scientific understanding of lipid biochemistry. For these discoveries, Wieland was awarded the 1927 Nobel Prize in Chemistry for his investigations into the constitution of bile acids and related substances. Key milestones from this period include: 1912 – Wieland begins systematic structural research on bile acids 1927 – Wieland receives the Nobel Prize in Chemistry; Japanese chemist Shoda isolates UDCA from bear bile 1928 – Adolf Windaus receives the Nobel Prize in Chemistry for his work on sterols and their relationship to vitamins 1936 – Iwasaki further characterises the structure of UDCA 1955 – Japanese researchers achieve laboratory synthesis of UDCA, enabling pharmaceutical production independent of animal sources Together, Wieland and Windaus helped establish the structural relationship between bile acids, sterols, and cholesterol, laying the foundations for modern steroid chemistry and decades of subsequent pharmaceutical development. Bile acids are steroid molecules synthesised in the liver from cholesterol. They are secreted into bile, stored in the gallbladder, and released into the small intestine during digestion, where they facilitate the absorption of dietary fats and fat-soluble vitamins. Bile acids can be classified according to their carbon skeleton, although the compounds most relevant to human physiology and medicine are the C24 bile acids. The two primary bile acids produced directly by the liver are: Cholic acid (CA) Chenodeoxycholic acid (CDCA) Once secreted into the intestine, these molecules are modified by gut bacteria to produce secondary bile acids, including: Deoxycholic acid (DCA) Lithocholic acid (LCA) Ursodeoxycholic acid (UDCA) is normally a minor component of the human bile acid pool but occurs at substantially higher levels in bear bile. It is this molecule that has become one of the most important therapeutic bile acids in modern medicine. The laboratory synthesis of UDCA in 1955 opened the door to widespread clinical investigation. Researchers soon discovered that UDCA could dissolve certain cholesterol gallstones by altering bile composition, providing a non-surgical treatment option for selected patients. As clinical evidence accumulated, its therapeutic applications expanded considerably. UDCA exerts protective effects on liver and bile duct cells through multiple mechanisms and is commonly described as having: Anticholestatic properties that improve bile flow Anti-apoptotic effects that protect cells from premature death Anti-inflammatory activity Antifibrotic effects that may help limit liver scarring Today, UDCA is the evidence-based first-line treatment for primary biliary cholangitis (PBC), the most common chronic cholestatic liver disease, and is approved in numerous jurisdictions worldwide for this indication. UDCA has also been investigated and used in selected patients with primary sclerosing cholangitis (PSC), although its role remains considerably more controversial than in PBC. In addition, it is widely used in the management of intrahepatic cholestasis of pregnancy and several other cholestatic disorders. One of the most transformative discoveries in modern bile acid biology has been the recognition that bile acids function not only as digestive molecules but also as signalling molecules. Bile acids interact with receptors throughout the body, influencing metabolism, inflammation, immune function, and energy homeostasis. This insight has opened entirely new avenues for pharmaceutical development. Two receptors are of particular significance: FXR is a nuclear receptor that regulates bile acid synthesis, transport, and homeostasis. Activation of FXR influences multiple pathways involved in inflammation, fibrosis, and metabolic regulation. Obeticholic acid (OCA), a semisynthetic derivative of chenodeoxycholic acid, received regulatory approval in 2016 as a second-line treatment for PBC, representing the first new approved therapy for the disease in more than two decades. TGR5 is a G protein-coupled receptor expressed in the gut, liver, immune cells, and other tissues. Activation of TGR5 has demonstrated promising metabolic effects in preclinical and early clinical research, making it an attractive target for future therapies aimed at conditions such as metabolic syndrome, type 2 diabetes, and metabolic dysfunction-associated steatohepatitis (MASH). One of the most dynamic areas of contemporary research concerns the relationship between bile acids and the gut microbiome. The intestinal microbiota plays a critical role in converting primary bile acids into secondary bile acids, while the composition of the bile acid pool influences the structure and function of microbial communities within the gut. This bidirectional relationship has linked bile acid biology to a wide range of physiological and pathological processes. Researchers are investigating how bile acid–microbiome interactions may contribute to: Inflammatory bowel disease Obesity and metabolic disorders Cardiovascular disease Liver disease Neurological and neuropsychiatric processes through the gut–brain axis Particularly noteworthy is ongoing research into tauroursodeoxycholic acid (TUDCA), a conjugated bile acid derivative that has demonstrated neuroprotective effects in preclinical studies. Investigators continue to explore its potential relevance to neurodegenerative and neuropsychiatric disorders. While many of these applications remain under active investigation, they highlight the expanding therapeutic significance of bile acid biology beyond the digestive system. The history of bile acids illustrates the long and often surprising dialogue between empirical observation and scientific discovery. The practitioners who recognised the medicinal value of animal bile centuries ago could not have known the molecular structures involved, nor the complex receptor networks that modern researchers continue to uncover. Yet many of the biological effects they observed would eventually be explained through advances in chemistry, biochemistry, pharmacology, and clinical medicine. The structural insights achieved by Wieland, Windaus, and their contemporaries laid the foundations for synthetic manufacturing programmes that now produce UDCA and related compounds at industrial scale. Meanwhile, modern research continues to reveal new functions for bile acids as signalling molecules involved in metabolism, immunity, and human health. Today, bile acids represent far more than digestive detergents. They are a scientifically rich and therapeutically important class of molecules whose story continues to evolve, linking ancient medical traditions with some of the most promising areas of modern pharmaceutical research. At ICE Pharma, we have dedicated over 70 years to the development and manufacture of bile acid derivatives. Understanding where these molecules have come from, scientifically and historically, is part of what drives our commitment to advancing where they are going next.

Drug Approval

20 Aug 2026

·allsci.com

Amylyx ups fundraising target to USD 500m after avexitide's Phase III win in hypoglycemia

Cambridge, Massachusetts-based Amylyx Pharmaceuticals (Nasdaq: AMLX) announced the pricing of an upsized underwritten public offering of common stock, raising USD 500.2 million in gross proceeds to fund pre-commercial preparations for avexitide, its lead GLP-1 receptor antagonist for post-bariatric hypoglycemia (PBH). The raise comes two days after avexitide met its FDA-agreed primary endpoint in the Phase III LUCIDITY trial, and represents a significant upsizing from the company's initial USD 350 million target announced the prior day. The offering comprises 14,090,000 shares priced at USD 35.50 per share, with underwriters granted a 30-day option to purchase up to an additional 2,113,500 shares. Amylyx said it intends to use net proceeds, together with existing cash, for avexitide pre-commercial activities including securing additional manufacturing capacity, research and development, and working capital. The offering was expected to close on or about August 21, 2026. The USD 35.50 pricing compares with USD 10.00 per share in a USD 175 million offering priced in September 2025, reflecting the stock's appreciation following the LUCIDITY readout. Avexitide, acquired from Eiger BioPharmaceuticals in a bankruptcy auction for USD 35.1 million in July 2024, carries FDA Breakthrough Therapy Designation for PBH in patients following Roux-en-Y gastric bypass surgery. The LUCIDITY trial demonstrated a 55% reduction in composite Level 2 and Level 3 hypoglycemic events versus placebo (p=0.000003), with all secondary endpoints met. Amylyx has said the data support an NDA filing targeting a potential approval and launch in 2027. Beyond avexitide, Amylyx is advancing AMX0035 (sodium phenylbutyrate/taurursodiol) in Wolfram syndrome, where a pivotal Phase III trial is targeted for initiation in H2 2026, and AMX0114, an antisense oligonucleotide targeting calpain-2 for ALS, currently in Phase I dose escalation. Access the AllSci platform to explore the science behind the news. Spot something wrong? Report an issue with this article

Phase 3Phase 1Breakthrough TherapyClinical ResultOligonucleotide

13 Aug 2026

·www.biospace.com

ProQR Announces Second Quarter 2026 Operating and Financial Results

First clinical validation of Axiomer™ RNA editing established through NTCP modulation with positive AX-0810 clinical target engagement data; full Phase 1 dataset to be presented at a medical or scientific conference later this year Multiple clinical milestones, including initial AX-0811 Phase 1 data expected by year-end 2026, followed in H1 2027 by: biliary atresia IIT initiation and initial data, initial AX-0422 (IDUA) patient data in MPS I Hurler syndrome, and advancement of AX-2911 (PNPLA3) to the clinic Strengthened balance sheet through $59.2 million financing during the quarter; ended Q2 2026 with € 117.1 million cash and cash equivalents, supporting runway through mid-2028 LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 13, 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 reported its financial and operating results for the second quarter ended June 30, 2026, and provided a business update. “The second quarter marked an important inflection point for ProQR as we established the first clinical validation of our Axiomer RNA editing platform by modulating NTCP with AX-0810,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “We believe these data, together with the significant unmet need that remains in cholestatic liver diseases, including biliary atresia, support the continued advancement of our NTCP franchise. Combined with our recent financing, we are well positioned to deliver a series of important clinical milestones, including presentation of the full AX-0810 Phase 1 dataset later this year, initial Phase 1 data from AX-0811 by year end, initiation of our biliary atresia clinical program and initial data in the first half of 2027, and continued advancement of our MPS I Hurler syndrome and MASH programs. Together, these milestones reflect ProQR’s evolution into a multi-program clinical-stage RNA editing company, strengthen the growing body of evidence supporting the broader potential of our Axiomer platform, and bring us closer to delivering transformative RNA editing therapies for patients.” Recent Progress and Anticipated Upcoming Events First Clinical Validation of Axiomer and Next Milestones with NTCP Franchise ProQR’s NTCP RNA editing approach is designed to modulate NTCP and reduce bile acid uptake directly in hepatocytes, with the goal of reducing intrahepatic cholestasis. In June, ProQR announced positive AX-0810 Phase 1 target engagement data from healthy volunteers, establishing the first clinical validation of the Company’s proprietary Axiomer RNA editing platform. Key findings included: Up to 8-fold increase in total serum bile acids (6 mg/kg) exceeding the Company’s pre-defined 2-fold threshold for meaningful NTCP modulation; Dose-dependent changes in conjugated bile acids, together with concordant changes in total serum bile acids and TUDCA, supporting the therapeutic rationale that modulating NTCP reduces the accumulation of toxic bile acids in the liver; Favorable safety and tolerability pro with AX-0810 to date, with no serious adverse events or pruritus reported; pharmacokinetic findings to date support sustained target engagement, including a half-life of eight weeks; and Data supporting advancement into patient study. ProQR plans to present the full dataset from the Phase 1 study of AX-0810, including Cohort 3, at a medical or scientific conference later this year. In July, the Company submitted a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of AX-0811, its next-generation editing oligonucleotide (EON) targeting NTCP for the treatment of cholestatic diseases. AX-0811 is generated by ProQR’s AI-enabled discovery engine and demonstrated higher potency and longer durability than AX-0810 preclinically, which will lead to lower dose levels and less frequent dosing in the clinic. Pending CTA authorization, initial data with AX-0811 is anticipated by year end 2026. ProQR continues preparations for an investigator-initiated trial (IIT) in pediatric biliary atresia in China, with initial clinical data expected in the first half of 2027. Strengthened Financial Position In June, ProQR closed an underwritten registered direct offering of $50.0 million, alongside a concurrent private placement to an existing shareholder and strategic partner Eli Lilly and Company (Lilly) of $9.2 million, for total gross proceeds of approximately $59.2 million. The financing further strengthened the Company’s balance sheet providing runway into mid-2028, and is expected to support the continued advancement of its wholly owned Axiomer RNA editing pipeline, including multiple anticipated clinical milestones. Platform and Research Progress ProQR continues to strengthen the scientific foundation of its Axiomer RNA editing platform through ongoing research and publication of preclinical findings. During the quarter, the Company’s preclinical research describing RNA editing of B4GALT1 was published online as a peer-reviewed Journal Pre-proof in Molecular Therapy – Nucleic Acids . The publication further expands the body of evidence supporting the breadth and versatility of the Axiomer platform beyond the Company’s clinical pipeline. Upcoming anticipated milestones NTCP franchise AX-0810 data from the 9 mg/kg cohort and 12-week follow up from the ongoing Phase 1 study expected by year-end 2026 AX-0811 initial data in healthy volunteers expected by year-end 2026, pending CTA authorization IIT in pediatric biliary atresia in China, with initial data targeted for first half of 2027 Potentially registration-enabling Phase 2 program, subject to regulatory interactions, expected to start in mid-2027 with first interim analysis data expected by mid-2028 Other pipeline candidates AX-0422 for MPS I Hurler Syndrome (IDUA) CTA filing for a first-in-patient study anticipated in early 2027 and initial data expected in the first half of 2027 AX-2911(PNPLA3) FIH IIT in China expected in the first half of 2027 Partnership Continue to execute on Lilly collaboration, with potential data updates and milestone payments Financial Highlights At June 30, 2026, ProQR held cash and cash equivalents of approximately € 117.1 million, compared to € 92.4 million at December 31, 2025. In June 2026, the Company closed an underwritten registered direct offering of 27,624,310 ordinary shares and a concurrent private placement of 5,100,780 ordinary shares with Lilly for gross proceeds totaling approximately $59.2 million. Net cash used in operating activities during the second quarter ended June 30, 2026 was € 12.3 million, compared to € 11.4 million used for the same period in 2025. Research and development (R&D) costs for the quarter ended June 30, 2026 were € 12.7 million, compared to € 11.4 million for the same period last year. General and administrative costs for the quarter ended June 30, 2026 were € 4.9 million, compared to € 4.8 million for the same period in 2025. Net loss for the quarter ended June 30, 2026 was € 8.7 million, or € 0.08 per diluted share, compared to € 12.2 million, or € 0.12 per diluted share, for the same period in 2025. For further financial information for the period ended June 30, 2026, please refer to our Q2 financial report filing available on our website, under Financials and Filings. 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 a clinical-stage company 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; the ongoing Phase 1 clinical study of AX-0810 in NTCP for cholestatic diseases, including the timing and presentation of the full Phase 1 dataset, including Cohort 3 and 12-week follow-up data, and the potential advancement into patient studies; our expectations regarding the safety, tolerability, target engagement and potential therapeutic benefits of AX-0810; our ability to collaborate with investigators to initiate, conduct and recruit for an IIT of our NTCP program in China in pediatric participants with biliary atresia and to generate meaningful data therefrom, including the anticipated timing of initial data readout in H1 2027; the anticipated initiation of our registration-enabling Phase 2 program in mid-2027 with first interim analysis data expected by mid-2028, subject to regulatory interactions; our pipeline targets, including the planned Phase 1 clinical trial of AX-0811 in healthy volunteers; our ability to obtain authorization for, initiate, enroll, and complete a Phase 1 clinical trial for AX-0811 and the anticipated timing of initial data readout by year end 2026, pending CTA authorization; the anticipated first-in-human study of AX-0422 targeting IDUA for MPS I Hurler syndrome, with a CTA filing expected in early 2027 and anticipated initial clinical data readout in H1 2027; the anticipated IIT in China of AX-2911 targeting PNPLA3 for MASH in H1 2027; our expectations regarding clinical updates across multiple programs in 2026 and 2027; the potential design, initiation and timing of a potentially registration-enabling Phase 2 program for the NTCP franchise, subject to regulatory interactions; the therapeutic potential and development timeline regarding AX-0810, AX-0811, AX-0422, and AX-2911;our participation at upcoming scientific conferences; 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 and pipeline 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, and potential milestones; our ability to selectively form new partnerships and enter into future collaborations; our financial position and expected cash-runway to fund our operations through mid 2028. 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, risks and uncertainties associated with conducting IITs in China, including evolving regulatory requirements, 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 due to shortage and pressure on supply chains and logistics in the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and our 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, inflationary pressures, fluctuating 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 or Investor contact: Peter Kelleher LifeSci Advisors T: +1 617 430 7579 pkelleher@lifesciadvisors.com PROQR THERAPEUTICS N.V. Unaudited Interim Condensed Consolidated Statement of Financial Position June 30, December 31, 2026 2025 €1 ,000 €1 ,000 Assets Property, plant and equipment 11,923 12,630 Investments in financial assets — — Non-current assets 11,923 12,630 Cash and cash equivalents 117,125 92,413 Trade and other receivables 3,908 6,800 Other taxes 597 913 Current assets 121,630 100,126 Total assets 133,553 112,756 Equity and liabilities Equity Equity attributable to owners of the Company 79,477 49,374 Total equity 79,477 49,374 Liabilities Borrowings — — Lease liabilities 8,692 9,547 Deferred income 10,510 21,394 Non-current liabilities 19,202 30,941 Borrowings 5,017 4,872 Lease liabilities 1,603 1,545 Derivative financial instruments 213 234 Trade payables — 298 Social securities and other taxes 214 — Deferred income 17,644 17,552 Other current liabilities 10,183 7,940 Current liabilities 34,874 32,441 Total liabilities 54,076 63,382 Total equity and liabilities 133,553 112,756 PROQR THERAPEUTICS N.V. Unaudited Interim Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income (€ in thousands, except share and per share data) Three month period Six month period ended June 30, ended June 30, 2026 2025 2026 2025 €1 ,000 €1 ,000 €1 ,000 €1 ,000 Revenue 8,759 3,817 10,792 8,336 Other income — 158 — 380 Research and development costs (12,667) (11,408) (24,497) (23,731) General and administrative costs (4,914) (4,816) (8,766) (8,050) Total operating costs (17,581) (16,224) (33,263) (31,781) Operating result (8,822) (12,249) (22,471) (23,065) Finance income and expense 152 192 364 647 Results related to financial liabilities measured at fair value through profit or loss (33) (104) 21 178 Result before corporate income taxes (8,703) (12,161) (22,086) (22,240) Income taxes (25) (18) (25) (18) Result for the period (8,728) (12,179) (22,111) (22,258) Other comprehensive income (foreign exchange differences on foreign operation) 48 (682) 227 (1,053) Total comprehensive income (8,680) (12,861) (21,884) (23,311) Result attributable to Owners of the Company (8,728) (12,179) (22,111) (22,258) Total comprehensive income attributable to Owners of the Company (8,680) (12,861) (21,884) (23,311) Share information Weighted average number of shares outstanding 1 109,958,613 105,343,897 107,673,118 105,320,495 Earnings per share attributable to owners of the Company (Euro per share) Basic loss per share 1 (0.08) (0.12) (0.21) (0.21) Diluted loss per share 1 (0.08) (0.12) (0.21) (0.21) 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 N.V. Unaudited Interim Condensed Consolidated Statement of Changes in Equity Attributable to owners of the Company Number of shares Share Capital Share Premium Equity settled Employee Benefit Reserve Translation Reserve Accumulated Deficit Total Equity €1 ,000 €1 ,000 €1 ,000 €1 ,000 €1 ,000 €1 ,000 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 vested 131,525 — 67 (181) — 181 67 Balance at June 30, 2025 107,710,916 4,308 483,879 26,272 297 (447,773) 66,983 Balance at January 1, 2026 107,710,916 4,308 483,881 28,426 265 (467,506) 49,374 Result for the period — — — — — (22,111) (22,111) Other comprehensive income — — — — 227 — 227 Recognition of share-based payments — — — 2,786 — — 2,786 Issuance of ordinary shares 35,755,393 1,430 47,771 — — — 49,201 Treasury shares transferred (4,623) — — — — — — Share options lapsed — — — (2,199) — 2,199 — Share options exercised / RSUs vested 4,623 — — (6) — 6 — Balance at June 30, 2026 143,466,309 5,738 531,652 29,007 492 (487,412) 79,477 PROQR THERAPEUTICS N.V. Unaudited Interim Condensed Consolidated Statement of Cash Flows Three month period Six month period ended June 30, ended June 30, 2026 2025 2026 2025 €1 ,000 €1 ,000 €1 ,000 €1 ,000 Cash flows from operating activities Net result (8,728) (12,179) (22,111) (22,258) Adjustments for: — Other income — (158) — (380) — Depreciation 707 675 1,401 1,353 — Share-based compensation 1,358 909 2,786 1,667 — Financial income and expenses (110) (139) (369) (647) — Results related to financial liabilities measured at fair value through profit or loss 33 104 (21) (178) — Income tax expenses 25 18 25 18 Changes in working capital (5,754) (1,178) (5,645) (7,900) Cash used in operations (12,469) (11,948) (23,934) (28,325) Corporate income tax (paid)/received (25) (18) (25) (18) Interest received 359 617 726 1,405 Interest paid (135) (52) (182) (261) Net cash used in operating activities (12,270) (11,401) (23,415) (27,199) Cash flow from investing activities Purchases of property, plant and equipment (291) (101) (455) (325) Net cash used in investing activities (291) (101) (455) (325) Cash flows from financing activities Proceeds from issuance of shares, net 49,201 — 49,201 — Proceeds from exercise of share options — — — 67 Repayment of lease liability (621) (293) (925) (860) Net cash generated by / (used in) financing activities 48,580 (293) 48,276 (793) Net increase / (decrease) in cash and cash equivalents 36,019 (11,795) 24,406 (28,317) Currency effect cash and cash equivalents 18 (854) 306 (1,326) Cash and cash equivalents, at beginning of the period 81,088 132,414 92,413 149,408 Cash and cash equivalents at the end of the period 117,125 119,765 117,125 119,765

100 Deals associated with Tauroursodeoxycholate sodium

External Link

KEGG	Wiki	ATC	Drug Bank
D11836	Tauroursodeoxycholate sodium	-	DB08834

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Cholelithiasis	China	Bruschettini Srl	25 Jul 2006

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cholestatic liver disease	Phase 3	China	Beijing Quanfu Kangjian Medicine Information Consulting Co., Ltd.	01 Aug 2009
Primary Biliary Cholangitis	Phase 3	China	Beijing Quanfu Kangjian Medicine Information Consulting Co., Ltd.	01 Aug 2009
Liver Diseases	Phase 2	-	Tanabe Pharma Corp.	-
Fibrosis, Liver	Preclinical	China	Lanzhou University	09 Apr 2025
Colitis, Ulcerative	Preclinical	China	Zhejiang Chinese Medical University	30 May 2024
Colitis, Ulcerative	Preclinical	China	University of Macau	30 May 2024
Colitis, Ulcerative	Preclinical	China	Xi'an Jiaotong University	30 May 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02218619 (CTgov)	Phase 2	Diabetes Mellitus, Type 1	20	Sugar Pill (placebo) (Sugar Pill (Placebo))	jfrfuylbzh(fzsznzlvpz) = hyocaujvri itxvteuxfz (zhfnzskwaz, 0.11) View more	-	05 Jun 2025
				Tauroursodeoxycholic Acid (TUDCA) (Taurourodeoxycholic Acid (TUDCA))	jfrfuylbzh(fzsznzlvpz) = nzfwdjvswf itxvteuxfz (zhfnzskwaz, 0.10) View more
NCT03423121 (CTgov)	Phase 1/2	Multiple Sclerosis, Chronic Progressive	59	Tauroursodeoxycholic Acid (TUDCA Treatment)	orljvsfvqg = zlmksptdhf vlpknrmqaj (cyztoecbaa, wtwbvnguln - updwxbkqvu) View more	-	03 May 2023
				Placebo oral capsule (Placebo Oral Capsule)	orljvsfvqg = ynzvnuqqto vlpknrmqaj (cyztoecbaa, mxdppkiqft - iafgseihmy) View more
DDW2021	Phase 1	Colitis, Ulcerative ER stress markers Bip \| eIF2a \| p-eIF2a	9	Tauroursodeoxycholic acid (TUDCA) 2000 mg	tbkfsysahd(acwkojqqms) = dfnuschnbx bzxuqeahlx (odrrsdolwi ) View more	Positive	23 May 2021
NCT01855360 (CTgov)	Phase 1/2	Transthyretin Amyloid Cardiomyopathy \| Senile cardiac amyloidosis	40	Doxycycline+Tauroursodeoxycholic Acid	ehbsbkypwu(afpcaqdzon) = adrijoicsm enrftkosyv (ethgzxyudf, .9) View more	-	04 Dec 2020
NCT01877551 (CTgov)	Not Applicable	Insulin Resistance	27	Tauroursodeoxycholic acid (Tauroursodeoxycholic Acid)	bjwkjtqjvx(avqgnvawgi) = ygpirlmlqq qvtdcwwfwd (dpkubaufqy, 11) View more	-	27 Jan 2020
				Placebo tablet (Placebo)	bjwkjtqjvx(avqgnvawgi) = hbkjnlssae qvtdcwwfwd (dpkubaufqy, 7) View more
NCT00771901 (TUDCA/PBA, CTgov)	Not Applicable	Insulin Resistance \| Diabetes Mellitus \| Obesity	101	placebo (Placebo)	hkkgonnddn(jdhscyopyj) = hwbxoytupn btzoounyax (ubdnbcybfh, 7) View more	-	29 May 2018
				tauroursodeoxycholic acid (Tauroursodeoxycholic Acid)	hkkgonnddn(jdhscyopyj) = drzstyzxdj btzoounyax (ubdnbcybfh, 8) View more
NCT00877604 (Pubmed \| Eur J Neurol)	Phase 2	Amyotrophic Lateral Sclerosis	34	Tauroursodeoxycholic acid	jegzeqmudi(cjxakucpek) = ntpbmsbpem ivnhnvvoit (falkavuopq )	Positive	01 Jan 2016
				Placebo	jegzeqmudi(cjxakucpek) = logumczkhw ivnhnvvoit (falkavuopq )
NCT00877604 (TUDCA-ALS, CTgov)	Phase 2	Amyotrophic Lateral Sclerosis	34	tauroursodeoxycholic acid (TUDCA) (TUDCA)	fnoaeprook = zsphzfmlbq vohdpazqdu (ekriqdaqzc, gvrxsiaqgf - nsxxwibzpm) View more	-	26 Nov 2014
				Placebo (Placebo)	fnoaeprook = zwyxdygixp vohdpazqdu (ekriqdaqzc, avehygituy - fofedfiyxr) View more

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