GPD1 modulators(glycerol-3-phosphate dehydrogenase 1 modulators), PRKAB1 activators(Protein kinase AMP-activated non-catalytic subunit beta 1 activators)

Therapeutic Areas

NeoplasmsEndocrinology and Metabolic DiseaseOther Diseases+ [10]

Active Indication

Mucinous NeoplasmPseudomyxoma PeritoneiEpendymoma+ [18]

Inactive Indication

Depressive DisorderDiabetes Mellitus, Type 2Differentiated Thyroid Gland Carcinoma+ [2]

Originator Organization

Elcelyx Therapeutics, Inc.

Active Organization

Peking Union Medical College Hospital

Baylor College of Medicine

University of California, Irvine

+ [10]

Inactive Organization

Anji Pharmaceuticals, Inc.

Elcelyx Therapeutics, Inc.Medical Research Agency

+ [3]

License Organization

Anji Pharmaceuticals (Shanghai) Co., Ltd.

Emory University

Elcelyx Therapeutics, Inc.

+ [3]

Drug Highest PhasePhase 2

First Approval Date-

Regulation-

Structure/Sequence

Molecular FormulaC4H11N5

InChIKeyXZWYZXLIPXDOLR-UHFFFAOYSA-N

CAS Registry657-24-9

345

Clinical Trials associated with Metformin

NCT07007221

/ WithdrawnPhase 2IIT

Clinical Trials to Evaluate Metformin to Treat Depression in People Living With HIV

Depression in HIV occurs commonly and causes poor HIV outcomes with public health implications.
Depression prevalence in HIV-infected persons is estimated at 26% compared to 5% in the general population. Adherence to antiretroviral medication is a life-saving treatment in HIV, but depression can reduce engagement in care and adherence to medication. Depression is also associated with poorer levels of viral suppression and even mortality. Overall, those with depression and HIV are less likely to return to health and remain more likely to transmit the virus. therefore, treatment of HIV is essential to prevention; lower engagement in and adherence to HIV treatment due to depression puts others at risk. This is a 3 part trial, an initial pilot, a placebo controlled pilot, and a full trial.
Part 1. Dose response, Tolerability/Acceptability of Metformin for Depression in people with HIV: To do Initial assessment of metformin for depression in people with HIV we will perform a randomized, double blind, 2-arm, 12 week randomized controlled trial to assess feasibility and acceptability of metformin for depression. We will test two doses of metformin 1000 mg daily versus 1500 mg daily.
Part 2. Preliminary Efficacy Trial of Metformin for Depression in People with HIV: This will be a two-arm, double-blind trial of metformin versus placebo in people with HIV and depression. We will randomize individuals 1:1 to metformin or placebo. Will then collect blood and rectal swabs for preliminary assessments of the mechanism of action for metformin in people with HIV and depression.
Part 3. Full efficacy factorial trial of metformin for depression and comparison/interaction with fluoxetine in people living with HIV: To assess metformin and fluoxetine as efficacious treatments for depression in people with HIV, we will conduct a 4-arm, 12-week, mechanistic, double-blinded, randomized controlled treatment trial (RCT) with an assessment of inflammation and the gut microbiome. Participants (n=400) will be HIV- positive patients on ART with comorbid depression receiving care at one of two Ugandan clinics. In addition, we will use the NIMH Research Domain Criteria (RDoC) of Acute Threat and loss under Negative Valence using biomarkers and self-reported tools.

Start Date01 Jul 2026

Sponsor / Collaborator

University of Minnesota

NCT07693452

/ RecruitingPhase 2IIT

Use of Metformin in Patients With Pseudomyxoma Peritonei Secondary to Appendiceal Mucinous Neoplasms

This is a pilot, open-label clinical trial determining the feasibility of metformin therapy in subjects with pseudomyxoma peritonei (PMP) secondary to appendiceal mucinous neoplasms (AMNs).

Start Date18 Jun 2026

Sponsor / Collaborator

University of California, Irvine

NCT07226453

/ RecruitingPhase 2IIT

A Target Validation and Efficacy Study of Metformin in Patients With Recurrent or Progressive Posterior Fossa Group A (PFA) Ependymoma

This is a multi-site study of the pharmacodynamic effects and efficacy of metformin in children and young adults with recurrent or progressive Posterior Fossa Group A (PFA) ependymoma.

Start Date05 May 2026

Sponsor / Collaborator

The University of California, San Francisco

100 Clinical Results associated with Metformin

100 Translational Medicine associated with Metformin

100 Patents (Medical) associated with Metformin

34,530

Literatures (Medical) associated with Metformin

01 Jan 2027·JOURNAL OF ETHNOPHARMACOLOGY

Mulberry leaf flavonoids alleviate type 2 diabetes by remodeling the gut microbiota-bile acid axis and activating FXR signaling

Article

Author: Dong, Yuekun ; Zhao, Baosheng ; Liu, Gaiting ; Xie, Jiamei ; Zhao, Yige ; Yang, Yang ; Liu, Shuchang ; He, Changhao ; An, Yongcheng ; Zhang, Huilin ; Shi, Lu ; Wang, Guohua ; Du, Yuhang ; Huang, Yan ; Fu, Wanxin ; Bu, Sitong

ETHNOPHARMACOLOGICAL RELEVANCE:

Mulberry leaf (Morus alba L.) has long been used in traditional Chinese medicine for treating "Xiaoke" (diabetes), as documented in "Compendium of Materia Medica" compiled by Li Shi-Zhen of the Ming Dynasty. Mulberry leaf flavonoids (MLF) are the major active components and have demonstrated anti-diabetic effects in modern studies. However, whether MLF alleviates type 2 diabetes mellitus (T2DM) by modulating the gut microbiota-bile acid axis and directly activating key metabolic receptors such as FXR remains unclear.

AIM OF THE STUDY:

This study aimed to investigate whether MLF ameliorates T2DM through remodeling the gut microbiota-bile acid axis and activating FXR signaling, and to identify potential flavonoid constituents within MLF that directly interact with FXR.

MATERIALS AND METHODS:

db/db mice were treated with low- or high-dose MLF (300 or 600 mg/kg/day) or metformin for 10 weeks. Blood glucose, insulin sensitivity, lipid profiles, and hepatic pathology were assessed. Gut microbiota composition was analyzed by 16S rRNA sequencing, and bile acid profiles in serum, liver, ileum, and feces were quantified by targeted metabolomics. The expression of FXR/TGR5 signaling pathway components was determined by qRT-PCR and Western blot. Molecular docking was performed to screen for potential FXR modulator among MLF constituents, followed by in vitro validation using HepG2 and Caco-2 cells.

RESULTS:

MLF treatment significantly reduced fasting blood glucose, improved glucose tolerance and insulin resistance, lowered serum and hepatic lipid levels, and alleviated hepatic steatosis in db/db mice. MLF reshaped the gut microbiota by increasing beneficial genera (e.g., Alloprevotella, Roseburia) and decreasing the Firmicutes/Bacteroidetes ratio. Concurrently, MLF reprogrammed bile acid profiles across multiple organs, elevating non-12-OH bile acids (e.g., CDCA) and reducing the FXR antagonist Tβ-MCA, thereby activating intestinal and hepatic FXR/TGR5 signaling, upregulating FGF15 and SHP, and increasing serum GLP-1. Molecular docking identified Morusin as a candidate FXR ligand, and in vitro assays confirmed that both MLF and Morusin activated FXR and modulated downstream targets (BSEP, NTCP, ASBT, CYP7A1) in hepatocytes and intestinal cells.

CONCLUSION:

MLF alleviates T2DM by remodeling the gut microbiota-bile acid axis and activating FXR/TGR5 signaling. Morusin, a flavonoid constituent of MLF, is a potential FXR-modulating compound. These findings provide a modern mechanistic rationale for the traditional use of mulberry leaf in treating diabetes and support the development of MLF as a gut-targeted therapeutic strategy for T2DM.

31 Dec 2026·JOURNAL OF MEDICAL ECONOMICS

Annual pharmacy cost per patient achieving composite treatment endpoints: a cost to target analysis of tirzepatide versus subcutaneous semaglutide 1 mg in patients with type 2 diabetes in the UK

Article

Author: Kanumilli, Naresh ; Evans, Jerome ; Cattin, Juliette ; Debackere, Nele ; Osumili, Beatrice ; Buckingham, Mike ; Hunt, Barnaby ; Webb, Joanne ; Nowakowski, Piotr

INTRODUCTION:

Tirzepatide is a treatment for type 2 diabetes associated with improvements in glycemic control and weight loss, and a low risk of hypoglycemia when not used in combination with insulin or insulin secretagogues. A cost to target analysis of tirzepatide 5, 10 and 15 mg versus subcutaneous semaglutide 1 mg in the UK setting was performed, calculating the annual pharmacy cost per patient treated to six composite endpoints combining glycemic control (glycated hemoglobin [HbA1c] ≤ 6.5% [48 mmol/mol] and <7.0% [53 mmol/mol]) with weight loss (≥5%, ≥10%, ≥15%) and avoidance of hypoglycemia. The costing analysis was based on the tirzepatide costs appraised by NICE as part of TA924.

METHODS:

The proportions of patients achieving composite treatment targets with tirzepatide and semaglutide (both in combination with metformin) were taken from a post-hoc analysis of the SURPASS-2 clinical trial (N = 1,845). Costs per patient treated to target were calculated by dividing the annual treatment costs associated with each intervention by the proportion of patients achieving the treatment target with each intervention.

RESULTS:

Tirzepatide 5, 10 and 15 mg were associated with a lower pharmacy cost per patient achieving treatment target than semaglutide 1 mg for the majority of endpoints evaluated. Differences became greater at more strict treatment targets. For example, the cost per patient achieving HbA1c ≤6.5%, weight loss ≥15%, and no hypoglycemia was GBP 5,650, GBP 8,665 and GBP 9,462 lower with tirzepatide 5, 10 and 15 mg, respectively, compared with semaglutide 1 mg over a 1-year time horizon. The only endpoint where semaglutide 1 mg was associated with a lower cost per patient achieving target was HbA1c <7.0%, weight loss ≥5%, and no hypoglycemia.

CONCLUSIONS:

Compared to semaglutide, tirzepatide was associated with a lower annual pharmacy cost per patient with diabetes achieving treatment target in the UK for the majority of endpoints, with greater differences at more strict treatment targets.

31 Dec 2026·PHARMACEUTICAL BIOLOGY

Qigui didang decoction alleviates renal injury in a diabetic kidney disease model with metabolic memory features: association with ferroptosis and the SIRT1/Nrf2 pathway

Article

Author: Lu, Yuhui ; Fan, Lifei ; You, Hongyi ; Lin, Min ; Wu, Yusen

OBJECTIVE:

This study aims to evaluate the renoprotective effects of QGDD in a DKD model exhibiting metabolic memory features and to explore its potential mechanism involving the regulation of ferroptosis via the SIRT1/Nrf2 signaling pathway.

METHODS:

A DKD rat model was induced using streptozotocin (STZ). The rats were assigned to the Blank Control Group (C), Model Group (M), Metformin group (Met), and low-, medium-, and high-dose QGDD groups (QGDD-L/M/H). Intervention effects were assessed by monitoring body weight, fasting blood glucose, renal function markers (24-UTP, BUN, Scr, Cys-C, β2-MG), renal histopathology (HE/Masson staining), oxidative stress markers (Fe²⁺, MDA, GSH, SOD), cell death indicators (TUNEL, ROS), and expression of genes and proteins associated with the SIRT1/Nrf2 pathway (RT-qPCR, Western blot).

RESULTS:

All QGDD dose groups decreased 24-hour urinary protein excretion and serum levels of BUN, Scr, Cys-C, and β2-MG. The medium-dose QGDD group notably reduced renal Fe²⁺ and MDA levels, increased GSH and SOD activity, and inhibited ROS accumulation and cellular apoptosis. QGDD activated the SIRT1/Nrf2 pathway, significantly upregulating the mRNA and protein expression of Nrf2, HO-1, and GPX4, while suppressing the accumulation of AGEs and Ferritin.

CONCLUSION:

QGDD mitigated the persistent elevation of AGEs, a hallmark of metabolic memory in DKD by activating the SIRT1/Nrf2 signaling pathway to inhibit ferroptosis-associated lipid peroxidation and oxidative stress. Its multi-target synergistic effects provide a solid experimental foundation for the use of Chinese herbal formulations in treating DKD. The medium-dose group demonstrated optimal therapeutic efficacy, emphasizing the significance of dose optimization.

195

News (Medical) associated with Metformin

07 Sep 2026

·www.prnewswire.com

Nature Biotechnology | Insilico's AI-Driven IPF Candidate Rentosertib Shows Potential for Biological Age Reversal, as Assessed by Six Proteomic Aging Clocks

First-in-Class Evaluation of an AI-Driven Candidate: This is the first de-novo drug designed with generative AI for a de-novo target discovered using AI and Aging Research (AI aging clocks, AI models and Hallmarks of Aging Assessment). First Universal Consensus Across Six Independently-Developed Proteomic Aging Clocks: Analyzing 12-week longitudinal Olink proteomic data from 42 IPF patients, six aging clock models (including ProtAge, OrganAge, and PAC) independently developed by the leading groups from Harvard, Oxford, PKU, and Insilico, unanimously indicated that rentosertib reversed predicted biological age. First Scalable Blueprint for Dual-Purpose Clinical Trials: The study is the first demonstration of a novel drug for a novel target discovered using aging research purposed to the age-related disease with aging biomarkers applied in a context of a clinical study. It establishes a standardized framework for embedding geroscience endpoints into standard disease trials, aligned with FDA Biomarker/BEST guidelines for dual-purpose aging and disease therapeutics. CAMBRIDGE, Mass., Sept. 7, 2026 /PRNewswire/ -- Insilico Medicine ("Insilico"), a clinical-stage company focused on generative AI-driven drug discovery and development, today announced the publication of a new study conducted in collaboration with an international team of scientists from Harvard Medical School, Stanford University, The Broad Institute, RWTH Aachen University, Peking University and Westlake University. Using longitudinal Olink proteomic data (CNCB OMIX accession: OMIX008341) collected during a Phase IIa clinical trial of rentosertib, researchers evaluated biological age using six internationally recognized, independently developed proteomic aging clocks. All six models consistently indicated a reduction in predicted biological age among patients treated with rentosertib. In addition, the Forced Vital Capacity (FVC), the essential measure of lung function declining with age, showed promising dose-dependent reversal compared to placebo aligning with the proteomic aging clock age reversal. Recently published in Nature Biotechnology, the study represents the first clinical evaluation of a potentially first-in-class drug candidate featuring both an AI-discovered target with relevance to aging and disease biology and an AI-designed molecule, rather than a repurposed generic drug such as rapamycin or metformin. The findings also provide a proof-of-concept framework for integrating aging biomarkers into standard clinical trials to accelerate the discovery of geroprotective drugs and longevity therapeutics. The results will be presented by first author Alex Zhavoronkov, founder and CEO of Insilico Medicine, at the Nature conference: Redefining Healthcare in the Age of AI, to be held at Sorbonne University in Paris on September 8, 2026. From aging biology to a clinical drug Unlike traditional geroscience studies that rely on repurposed drugs like rapamycin or metformin, Insilico pursued a different approach by incorporating aging biology into its drug discovery strategy from the outset. Using its AI-powered target discovery platform, Insilico identified TNIK as a novel target implicated in aging and fibrosis and performed hallmarks of aging assessment where TNIK scored highly in 6 hallmarks. TNIK was subsequently prioritized as a dual-purpose target relevant to both underlying aging biology and idiopathic pulmonary fibrosis (IPF). Insilico's generative chemistry platform, Chemistry42, was then deployed to design the small-molecule candidate rentosertib targeting TNIK. The program progressed from target identification to preclinical candidate nomination in approximately 18 months, with the preclinical findings published in Nature Biotechnology in 2024. In June 2025, Insilico reported the results of a Phase IIa trial of Rentosertib for IPF (NCT05938920) in Nature Medicine. The trial met its primary safety endpoint and demonstrated a promising dose-dependent trend in efficacy. One of the common efficacy outcome measures is Forced Vital Capacity (FVC), the essential measure of lung function. The typical age of onset of IPF is around 65 years. Interestingly, in healthy individuals older than 65, FVC declines as well typically at the rate of 20-50 mL/year and may also serve as a potential physiological biomarker of aging. In the previous publication, it was revealed that in the 60-mg once-daily group, patients experienced a mean improvement in forced vital capacity (FVC) of +98.4 mL, compared with a mean decline of -20.3 mL in the placebo group (-62.3 excluding outlier). Crucially, the trial protocol prospectively included longitudinal serum proteomic screening for exploratory biomarker analyses, which proved consistent with the drug's proposed anti-fibrotic and anti-inflammatory activity. All data collected from the trial were subsequently deposited with the China National Center for Bioinformation (CNCB), enabling the current investigation of biological aging biomarkers. Six different clocks point to strong biological age reversal measured by proteomics In the current study, researchers evaluated serum proteome profiles from 42 trial participants across 2,841 proteins. The team applied six independently developed proteomic aging clocks, including ProtAge, OrganAge (chronological and mortality variants), PAC, ipfP3GPT, and PAOPAC. Despite relying on diverse methodologies and training criteria, all six clocks consistently exhibited a trend toward reversed biological age among rentosertib-treated patients compared to placebo. The peak effect is observed at Week 4 in participants receiving 30 mg BID with approximately 3–4 years reversal in biological age and up to 6 years in a certain aging clock. Beyond changes in biological age scores, the study showed that rentosertib's anti-aging effects operate partially independent of its respiratory benefits. Notably, the dose associated with the greatest improvement in lung function differed from that producing the strongest age-reversal signal, suggesting that the drug's geroprotective activity was not merely a downstream consequence of disease improvement. This systemic impact was further validated through comparisons with 55,319 UK Biobank profiles, which showed that rentosertib directly reversed the typical age-related protein-expression trajectories. Mechanistically, the drug acted as a senomorphic agent, suppressing key drivers of cellular senescence, including EREG, ESM1, IGFBP4, ITGA2, MMP10, MMP13, and SPP1 while downregulating growth-factor signaling pathways associated with accelerated aging, including RTK–PI3K and RAS–ERK, and modulating antioxidant and cholesterol metabolisms. A framework for the future To support reproducibility and further research, all research data have been deposited at CNCB (accession OMIX008341), and the underlying pipeline code has been published as the open-source Python library on GitHub. Beyond these specific findings, the study lays out a scalable blueprint for embedding geroscience endpoints into disease-focused drug development. The research proposes a stepwise framework: collecting aging and senescence biomarkers prospectively as exploratory endpoints in disease trials, and ultimately pursuing biomarker qualification or composite clinical endpoints under the FDA Biomarker Qualification Program and FDA–NIH BEST framework. Such an approach could surface geroprotective candidates years or even decades earlier than the traditional path of post-approval drug repurposing. Insilico's dual-purpose therapeutic strategy is also proving its viability as a sustainable commercial model. Recently, Insilico reported total revenue of approximately $106 million in the first half of 2026, a 287% year-over-year increase, and achieved its first profitable half-year since listing, with an adjusted net profit exceeding $51 million. This milestone was driven by a series of out-licensing, co-development, and R&D collaborations with global partners, including Eli Lilly, Servier, Takeda, SK Biopharmaceuticals, Qilu Pharmaceutical, Hygtia Therapeutics, CMS, and Tenacia. As of the latest practicable date, the total contract value of transactions announced by Insilico in 2026 reached approximately $7.3 billion, pushing the cumulative contract value of its major collaborations since 2021 approximately $11 billion. On the AI-driven R&D front, Insilico nominated nine development candidates within nine months of 2026 as of late August, setting a new company record for annual pipeline productivity and achieving eight clinical milestones across its proprietary and co-developed programs. Leading this progress is rentosertib (ISM001-055), the world's first drug candidate discovered and developed using generative AI, which has advanced to a Phase III trial evaluating for idiopathic pulmonary fibrosis (IPF). In addition, Insilico Medicine launched a comprehensive set of benchmarks that allow foundation models to be evaluated in all tasks needed for drug discovery and launched state of the art (SOTA) foundation models outperforming other models and even internal tools in benchmarks. Using this new capability Insilico hopes to expand and accelerate longevity drug discovery and development both in terms of scale, therapeutic modalities, and indications. Industry Commentary and Additional Information "Six proteomic clocks from six independent groups, applied to the same 42 patients, all reported a younger biological age in the treated arms. What convinces me is not the size of the effect but the agreement, because these models share neither their features nor their training data." said Michael Levitt, PhD, 2013 Nobel Prize laureate in Chemistry. "This trial cannot yet separate slower aging from a treated lung, and the authors say so plainly. The experiment in healthy volunteers is the one I want to see next." "Insilico Medicine was built with one purpose – to develop a credible and sustainable business model for the development of frontier deep learning technology and its application to practical aging research and drug discovery with the purpose of developing using AI and practical discovery and development of longevity therapeutics. This first program demonstrates the first proof of concept for the novel dual-purpose therapeutic targeting aging but proposed toward the age-related disease discovered in record time making exploratory development in multiple indications commercially viable. Today, the majority of Insilico's 40+ drug programs have dual purpose targeting aging and disease in a broad range of therapeutic areas. By pursuing a credible biotechnology business model, the company reached profitability and established the longevity board, which is rare for the publicly-traded frontier biotechnology company. I am also happy that the drug carries the name of the true hero of the AI drug discovery revolution, our co-CEO, Dr. Ren. The social and economic opportunity for drugs reversing biological age is vast - trillions of dollars and billions of life years. If you add just 3 extra years to everyone's lifespan, that translates into roughly 25 billion life years on the global scale - at current life expectancy, that is roughly 340 million human lifetimes. More lifetimes than humanity lost in all the wars ever fought. If you manage to add 3 years to everyone's life, the drug should be able to significantly extend the healthy portion of life as well translating into trillions of dollars in productivity and savings", said Alex Zhavoronkov, PhD, founder and co-CEO of Insilico Medicine. "In this study, we evaluated clinical trial data to compare the blood proteomic profiles of pulmonary fibrosis patients treated with Rentosertib versus placebo", said Ludger Goeminne, Research Fellow in Medicine at Harvard Medical School, and co-author of the paper. "Using six proteomic aging clocks including models our team published in Cell Metabolism, we observed significant reductions in predicted biological age across multiple organ-specific clocks, and further pathway analysis confirmed that Rentosertib's biological impact extends far beyond merely reducing fibrosis." "As the developer of PAOPAC (Proteome-Aware Organ Proxy Aging Clock), I am thrilled to see our tool applied to the analysis of the Phase IIa clinical trial for rentosertib. Dr. Zhavoronkov and his colleagues are the first to systematically evaluate a novel drug candidate using multiple proteomic aging clocks within a single clinical trial cohort. The methodologies underlying these clocks range from traditional machine learning (OrganAge, PAOPAC, PAC) to deep learning (ProtAge, ipfP3GPT), with training targets encompassing both chronological age (ProtAge, OrganAgechrono, ipfP3GPT, PAOPAC) and mortality risk (PAC, OrganAgemortality). Across the board, these clocks consistently predicted a reduction in biological age within the rentosertib treatment group. This cross-model consistency demonstrates that rentosertib's effect on aging-related proteomic signals is not a model-specific artifact, but rather a highly robust biological phenomenon.", said Professor Jing-Dong Jackie Han, Peking-Tsinghua Center for Life Sciences, Academy for Advanced Interdisciplinary Studies, Center for Quantitative Biology (CQB), China. "Furthermore, this study establishes a relatively comprehensive workflow for the standardized application of aging clocks in clinical trials, covering cohort establishment, omics profiling, multi-clock parallel analysis, result cross-validation, and mechanistic interpretation. It also introduces an innovative practical approach to clinical trial design: evaluating aging biomarkers in parallel during trials targeting specific indications, particularly age-related diseases. This strategy holds great promise for accelerating the discovery and validation of longevity interventions. Finally, my congratulations on the breakthrough achieved with rentosertib—an innovative drug discovered in China and clinically evaluated primarily in Chinese patient cohorts. As a domestic Chinese research team behind the development of aging clocks, we look forward to witnessing more industry-academia-research collaborations driving future clinical trials in aging interventions." References About Rentosertib (ISM001-055) Rentosertib is a potentially first-in-class small molecule targeting TNIK, developed using Insilico Medicine's generative AI platform Pharma.AI. TNIK was identified through AI-powered target discovery as a gene involved in six hallmarks of aging, making it a dual-purpose target for both aging biology and idiopathic pulmonary fibrosis (IPF). The drug's discovery, design, preclinical validation, and Phase I results were published in Nature Biotechnology in March 2024. Phase IIa results were published in Nature Medicine in June 2025. Rentosertib has entered Phase III clinical development in China. About Idiopathic Pulmonary Fibrosis (IPF) Idiopathic Pulmonary Fibrosis is a chronic, scarring lung disease characterized by progressive and irreversible decline in lung function. Affecting approximately 5 million people worldwide, IPF carries a poor prognosis with a median survival of 3–4 years. Current approved treatments can slow disease progression but cannot stop or reverse it, leaving a significant unmet need for disease-modifying therapies. About Insilico Medicine Insilico Medicine is a pioneering global biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery, drive innovation in the life sciences, and extend healthy longevity to people on the planet. The company was listed on the Main Board of the Hong Kong Stock Exchange on December 30, 2025, under the stock code 03696.HK. By integrating AI and automation technologies and deep in-house drug discovery capabilities, Insilico is delivering innovative drug solutions for unmet needs including fibrosis, oncology, immunology, pain, and obesity and metabolic disorders. Additionally, Insilico extends the reach of Pharma.AI across diverse industries, such as advanced materials, agriculture, nutritional products and veterinary medicine. For more information, please visit SOURCE Insilico Medicine 21% more press release views with Request a Demo

Phase 2Clinical ResultPhase 3Phase 1

07 Sep 2026

·www.biospace.com

Nature Biotechnology | Insilico’s AI-Driven IPF Candidate Rentosertib Shows Potential for Biological Age Reversal, as Assessed by Six Proteomic Aging Clocks

First-in-Class Evaluation of an AI-Driven Candidate : This is the first de-novo drug designed with generative AI for a de-novo target discovered using AI and Aging Research (AI aging clocks, AI models and Hallmarks of Aging Assessment). First Universal Consensus Across Six Independently-Developed Proteomic Aging Clocks: Analyzing 12-week longitudinal Olink proteomic data from 42 IPF patients, six aging clock models (including ProtAge, OrganAge, and PAC) independently developed by the leading groups from Harvard, Oxford, PKU, and Insilico, unanimously indicated that rentosertib reversed predicted biological age. First Scalable Blueprint for Dual-Purpose Clinical Trials : The study is the first demonstration of a novel drug for a novel target discovered using aging research purposed to the age-related disease with aging biomarkers applied in a context of a clinical study. It establishes a standardized framework for embedding geroscience endpoints into standard disease trials, aligned with FDA Biomarker/BEST guidelines for dual-purpose aging and disease therapeutics. CAMBRIDGE, Mass. , Sept. 7, 2026 /PRNewswire/ -- Insilico Medicine ("Insilico"), a clinical-stage company focused on generative AI-driven drug discovery and development , today announced the publication of a new study conducted in collaboration with an international team of scientists from Harvard Medical School, Stanford University, The Broad Institute, RWTH Aachen University, Peking University and Westlake University. Using longitudinal Olink proteomic data (CNCB OMIX accession: OMIX008341) collected during a Phase IIa clinical trial of rentosertib, researchers evaluated biological age using six internationally recognized, independently developed proteomic aging clocks. All six models consistently indicated a reduction in predicted biological age among patients treated with rentosertib. In addition, the Forced Vital Capacity (FVC), the essential measure of lung function declining with age, showed promising dose-dependent reversal compared to placebo aligning with the proteomic aging clock age reversal. Recently published in Nature Biotechnology , the study represents the first clinical evaluation of a potentially first-in-class drug candidate featuring both an AI-discovered target with relevance to aging and disease biology and an AI-designed molecule, rather than a repurposed generic drug such as rapamycin or metformin. The findings also provide a proof-of-concept framework for integrating aging biomarkers into standard clinical trials to accelerate the discovery of geroprotective drugs and longevity therapeutics. The results will be presented by first author Alex Zhavoronkov, founder and CEO of Insilico Medicine, at the Nature conference: Redefining Healthcare in the Age of AI, to be held at Sorbonne University in Paris on September 8, 2026. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); From aging biology to a clinical drug Unlike traditional geroscience studies that rely on repurposed drugs like rapamycin or metformin, Insilico pursued a different approach by incorporating aging biology into its drug discovery strategy from the outset. Using its AI-powered target discovery platform, Insilico identified TNIK as a novel target implicated in aging and fibrosis and performed hallmarks of aging assessment where TNIK scored highly in 6 hallmarks. TNIK was subsequently prioritized as a dual-purpose target relevant to both underlying aging biology and idiopathic pulmonary fibrosis (IPF) . Insilico's generative chemistry platform, Chemistry42, was then deployed to design the small-molecule candidate rentosertib targeting TNIK. The program progressed from target identification to preclinical candidate nomination in approximately 18 months, with the preclinical findings published in Nature Biotechnology in 2024. In June 2025, Insilico reported the results of a Phase IIa trial of Rentosertib for IPF (NCT05938920) in Nature Medicine . The trial met its primary safety endpoint and demonstrated a promising dose-dependent trend in efficacy. One of the common efficacy outcome measures is Forced Vital Capacity (FVC), the essential measure of lung function. The typical age of onset of IPF is around 65 years. Interestingly, in healthy individuals older than 65, FVC declines as well typically at the rate of 20-50 mL/year and may also serve as a potential physiological biomarker of aging. In the previous publication, it was revealed that in the 60-mg once-daily group, patients experienced a mean improvement in forced vital capacity (FVC) of +98.4 mL, compared with a mean decline of -20.3 mL in the placebo group (-62.3 excluding outlier). ezstandalone.cmd.push(function () { ezstandalone.showAds(132); }); Crucially, the trial protocol prospectively included longitudinal serum proteomic screening for exploratory biomarker analyses, which proved consistent with the drug's proposed anti-fibrotic and anti-inflammatory activity. All data collected from the trial were subsequently deposited with the China National Center for Bioinformation (CNCB), enabling the current investigation of biological aging biomarkers. Six different clocks point to strong biological age reversal measured by proteomics In the current study, researchers evaluated serum proteome profiles from 42 trial participants across 2,841 proteins. The team applied six independently developed proteomic aging clocks, including ProtAge, OrganAge (chronological and mortality variants), PAC, ipfP3GPT, and PAOPAC. Despite relying on diverse methodologies and training criteria, all six clocks consistently exhibited a trend toward reversed biological age among rentosertib-treated patients compared to placebo. The peak effect is observed at Week 4 in participants receiving 30 mg BID with approximately 3–4 years reversal in biological age and up to 6 years in a certain aging clock. Beyond changes in biological age scores, the study showed that rentosertib's anti-aging effects operate partially independent of its respiratory benefits. Notably, the dose associated with the greatest improvement in lung function differed from that producing the strongest age-reversal signal, suggesting that the drug's geroprotective activity was not merely a downstream consequence of disease improvement. ezstandalone.cmd.push(function () { ezstandalone.showAds(133); }); This systemic impact was further validated through comparisons with 55,319 UK Biobank pro which showed that rentosertib directly reversed the typical age-related protein-expression trajectories. Mechanistically, the drug acted as a senomorphic agent, suppressing key drivers of cellular senescence, including EREG, ESM1, IGFBP4, ITGA2, MMP10, MMP13, and SPP1 while downregulating growth-factor signaling pathways associated with accelerated aging, including RTK–PI3K and RAS–ERK, and modulating antioxidant and cholesterol metabolisms. A framework for the future To support reproducibility and further research, all research data have been deposited at CNCB (accession OMIX008341), and the underlying pipeline code has been published as the open-source Python library on GitHub. Beyond these specific findings, the study lays out a scalable blueprint for embedding geroscience endpoints into disease-focused drug development. The research proposes a stepwise framework: collecting aging and senescence biomarkers prospectively as exploratory endpoints in disease trials, and ultimately pursuing biomarker qualification or composite clinical endpoints under the FDA Biomarker Qualification Program and FDA–NIH BEST framework. Such an approach could surface geroprotective candidates years or even decades earlier than the traditional path of post-approval drug repurposing. ezstandalone.cmd.push(function () { ezstandalone.showAds(134); }); Insilico's dual-purpose therapeutic strategy is also proving its viability as a sustainable commercial model. Recently, Insilico reported total revenue of approximately $106 million in the first half of 2026, a 287% year-over-year increase, and achieved its first profitable half-year since listing, with an adjusted net profit exceeding $51 million. This milestone was driven by a series of out-licensing, co-development, and R&D collaborations with global partners, including Eli Lilly, Servier, Takeda, SK Biopharmaceuticals, Qilu Pharmaceutical, Hygtia Therapeutics, CMS, and Tenacia. As of the latest practicable date, the total contract value of transactions announced by Insilico in 2026 reached approximately $7.3 billion, pushing the cumulative contract value of its major collaborations since 2021 approximately $11 billion. On the AI-driven R&D front, Insilico nominated nine development candidates within nine months of 2026 as of late August, setting a new company record for annual pipeline productivity and achieving eight clinical milestones across its proprietary and co-developed programs. Leading this progress is rentosertib (ISM001-055), the world's first drug candidate discovered and developed using generative AI, which has advanced to a Phase III trial evaluating for idiopathic pulmonary fibrosis (IPF). In addition, Insilico Medicine launched a comprehensive set of benchmarks that allow foundation models to be evaluated in all tasks needed for drug discovery and launched state of the art (SOTA) foundation models outperforming other models and even internal tools in benchmarks. Using this new capability Insilico hopes to expand and accelerate longevity drug discovery and development both in terms of scale, therapeutic modalities, and indications. Industry Commentary and Additional Information "Six proteomic clocks from six independent groups, applied to the same 42 patients, all reported a younger biological age in the treated arms. What convinces me is not the size of the effect but the agreement, because these models share neither their features nor their training data." said Michael Levitt, PhD, 2013 Nobel Prize laureate in Chemistry. "This trial cannot yet separate slower aging from a treated lung, and the authors say so plainly. The experiment in healthy volunteers is the one I want to see next." ezstandalone.cmd.push(function () { ezstandalone.showAds(135); }); "Insilico Medicine was built with one purpose – to develop a credible and sustainable business model for the development of frontier deep learning technology and its application to practical aging research and drug discovery with the purpose of developing using AI and practical discovery and development of longevity therapeutics. This first program demonstrates the first proof of concept for the novel dual-purpose therapeutic targeting aging but proposed toward the age-related disease discovered in record time making exploratory development in multiple indications commercially viable. Today, the majority of Insilico's 40+ drug programs have dual purpose targeting aging and disease in a broad range of therapeutic areas. By pursuing a credible biotechnology business model, the company reached profitability and established the longevity board, which is rare for the publicly-traded frontier biotechnology company. I am also happy that the drug carries the name of the true hero of the AI drug discovery revolution, our co-CEO, Dr. Ren. The social and economic opportunity for drugs reversing biological age is vast - trillions of dollars and billions of life years. If you add just 3 extra years to everyone's lifespan, that translates into roughly 25 billion life years on the global scale - at current life expectancy, that is roughly 340 million human lifetimes. More lifetimes than humanity lost in all the wars ever fought. If you manage to add 3 years to everyone's life, the drug should be able to significantly extend the healthy portion of life as well translating into trillions of dollars in productivity and savings", said Alex Zhavoronkov, PhD, founder and co-CEO of Insilico Medicine. "In this study, we evaluated clinical trial data to compare the blood proteomic profiles of pulmonary fibrosis patients treated with Rentosertib versus placebo", said Ludger Goeminne, Research Fellow in Medicine at Harvard Medical School, and co-author of the paper . "Using six proteomic aging clocks including models our team published in Cell Metabolism, we observed significant reductions in predicted biological age across multiple organ-specific clocks, and further pathway analysis confirmed that Rentosertib's biological impact extends far beyond merely reducing fibrosis." "As the developer of PAOPAC (Proteome-Aware Organ Proxy Aging Clock), I am thrilled to see our tool applied to the analysis of the Phase IIa clinical trial for rentosertib. Dr. Zhavoronkov and his colleagues are the first to systematically evaluate a novel drug candidate using multiple proteomic aging clocks within a single clinical trial cohort. The methodologies underlying these clocks range from traditional machine learning (OrganAge, PAOPAC, PAC) to deep learning (ProtAge, ipfP3GPT), with training targets encompassing both chronological age (ProtAge, OrganAge chrono , ipfP3GPT, PAOPAC) and mortality risk (PAC, OrganAge mortality ). Across the board, these clocks consistently predicted a reduction in biological age within the rentosertib treatment group. This cross-model consistency demonstrates that rentosertib's effect on aging-related proteomic signals is not a model-specific artifact, but rather a highly robust biological phenomenon.", said Professor Jing-Dong Jackie Han, Peking-Tsinghua Center for Life Sciences, Academy for Advanced Interdisciplinary Studies, Center for Quantitative Biology (CQB), China. "Furthermore, this study establishes a relatively comprehensive workflow for the standardized application of aging clocks in clinical trials, covering cohort establishment, omics profiling, multi-clock parallel analysis, result cross-validation, and mechanistic interpretation. It also introduces an innovative practical approach to clinical trial design: evaluating aging biomarkers in parallel during trials targeting specific indications, particularly age-related diseases. This strategy holds great promise for accelerating the discovery and validation of longevity interventions. Finally, my congratulations on the breakthrough achieved with rentosertib—an innovative drug discovered in China and clinically evaluated primarily in Chinese patient cohorts. As a domestic Chinese research team behind the development of aging clocks, we look forward to witnessing more industry-academia-research collaborations driving future clinical trials in aging interventions." References [1] Zhavoronkov, A., Galkin, F., Chen, S. et al. Integration of proteomic aging clocks in a phase 2a clinical trial supports simultaneous geroprotective assessment. Nat Biotechnol (2026). [2] Pun, F. W., Leung, G. H. D., Leung, H. W. et al. Hallmarks of aging-based dual-purpose disease and age-associated targets predicted using PandaOmics AI-powered discovery engine. Aging (Albany NY) 14, 2475–2506 (2022). [3] Tang, Q., Xiao, D., Veviorskiy, A. et al. AI-Driven Robotics Laboratory Identifies Pharmacological TNIK Inhibition as a Potent Senomorphic Agent. Aging Dis 17, 432–451 (2026). [4] Ren, F., Aliper, A., Chen, J. et al. A small-molecule TNIK inhibitor targets fibrosis in preclinical and clinical models. Nat Biotechnol 43, 63–75 (2025). [5] Xu, Z., Ren, F., Wang, P. et al. A generative AI-discovered TNIK inhibitor for idiopathic pulmonary fibrosis: a randomized phase 2a trial. Nat Med 31, 2602–2610 (2025). About Rentosertib (ISM001-055) Rentosertib is a potentially first-in-class small molecule targeting TNIK, developed using Insilico Medicine's generative AI platform Pharma.AI. TNIK was identified through AI-powered target discovery as a gene involved in six hallmarks of aging, making it a dual-purpose target for both aging biology and idiopathic pulmonary fibrosis (IPF). The drug's discovery, design, preclinical validation, and Phase I results were published in Nature Biotechnology in March 2024. Phase IIa results were published in Nature Medicine in June 2025. Rentosertib has entered Phase III clinical development in China. About Idiopathic Pulmonary Fibrosis (IPF) Idiopathic Pulmonary Fibrosis is a chronic, scarring lung disease characterized by progressive and irreversible decline in lung function. Affecting approximately 5 million people worldwide, IPF carries a poor prognosis with a median survival of 3–4 years. Current approved treatments can slow disease progression but cannot stop or reverse it, leaving a significant unmet need for disease-modifying therapies. About Insilico Medicine Insilico Medicine is a pioneering global biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery, drive innovation in the life sciences, and extend healthy longevity to people on the planet. The company was listed on the Main Board of the Hong Kong Stock Exchange on December 30, 2025, under the stock code 03696.HK. By integrating AI and automation technologies and deep in-house drug discovery capabilities, Insilico is delivering innovative drug solutions for unmet needs including fibrosis, oncology, immunology, pain, and obesity and metabolic disorders. Additionally, Insilico extends the reach of Pharma.AI across diverse industries, such as advanced materials, agriculture, nutritional products and veterinary medicine. For more information, please visit View original content: SOURCE Insilico Medicine

Phase 2Clinical ResultPhase 3

06 Aug 2026

·www.prnewswire.com

MetaVia Reports Second Quarter 2026 Financial Results and Provides Corporate Update

All Active Patients Successfully Reached Highest Planned Dose Levels of 48 mg and 64 mg in Ongoing Phase 1 Part 3 Study of DA-1726 16-Week Topline Data Expected in the Fourth Quarter of 2026 CAMBRIDGE, Mass., Aug. 6, 2026 /PRNewswire/ -- MetaVia Inc. (Nasdaq: MTVA), a clinical-stage biotechnology company focused on transforming cardiometabolic diseases, today announced financial results for the second quarter ended June 30, 2026, and provided a corporate strategic update. "During the second quarter of 2026, and subsequently, we continued to execute across both of our clinical development programs while further strengthening the differentiated profile of DA-1726 as a dual GLP-1/glucagon receptor agonist," stated Hyung Heon Kim, President and Chief Executive Officer of MetaVia. "Most recently, all active patients in both cohorts of our ongoing Phase 1 Part 3 study successfully reached the highest planned dose levels of 48 mg and 64 mg, representing an important execution milestone as we prepare for our 16-week topline data readout, expected in the fourth quarter of 2026. Also of note, during the quarter, we presented additional data from our 48 mg Phase 1 study of DA-1726 at both the EASL and ADA scientific conferences, which demonstrated up to 9.1% mean weight loss after just eight weeks of treatment, meaningful reductions in waist circumference and encouraging liver-related findings, reinforcing our confidence in DA-1726's potential best-in-class profile and its ability to address multiple aspects of cardiometabolic disease. Based on the continued trajectory of weight loss through eight weeks without evidence of plateau on the 48 mg dosage, we believe the ongoing 16-week study evaluating the 48 mg and higher 64 mg dosages has the potential to provide a more complete assessment of the drug's effects on weight loss, waist circumference, cardiometabolic health and liver-related outcomes." "In parallel, we continued to broaden the potential clinical utility of vanoglipel (DA-1241). During the quarter, we presented preclinical combination data at the ADA 2026 Scientific Sessions, demonstrating synergistic effects with both resmetirom in MASH and metformin in type 2 diabetes, while a peer-reviewed publication further highlighted the anti-fibrotic potential of GPR119 agonism. Together, these findings reinforce our strategy of advancing vanoglipel as a differentiated combination backbone for metabolic and liver diseases, designed to complement established and emerging therapies across cardiometabolic disorders." Second Quarter 2026 and Subsequent Highlights July 2026: Announced the completion of dose titration in the 16-week, Phase 1 Part 3 study of DA-1726, with all active patients successfully reaching the highest planned dose levels of 48 mg and 64 mg. June 2026: Presented new late-breaking data at the ADA 2026 Scientific Sessions supporting DA-1726's differentiated obesity profile and demonstrating the combination potential of vanoglipel with resmetirom in MASH and metformin in type 2 diabetes. May 2026: Presented additional data from the 48 mg Phase 1 trial of DA-1726 at EASL Congress 2026, demonstrating up to 9.1% mean body weight reduction without evidence of plateau and exploratory improvements in liver-related biomarkers, supporting the potential of DA-1726 in obesity and MASH. May 2026: Announced the publication of peer-reviewed preclinical research in Biomolecules & Therapeutics, supporting the anti-fibrotic potential of vanoglipel and the differentiated role of GPR119 agonism in MASH. April 2026: Dosed the first patient in Part 3 of the 16-week Phase 1 clinical trial evaluating DA-1726 in obese, otherwise healthy adults using one-step titration to 48 mg and two-step titration to 64 mg to safely achieve higher target doses and optimize tolerability. Anticipated Clinical Milestones DA-1726 in Obesity: Data readout from Phase 1 Part 3, 16-week titration studies, evaluating titration to 48 mg in one step and 64 mg via a two-step regimen, is expected in the fourth quarter of 2026. Vanoglipel (DA-1241) in MASH: The Company is currently working to schedule an End-of-Phase 2 meeting with the FDA during the second half of 2026 to discuss the clinical development pathway for the vanoglipel combination therapy. Second Quarter Financial and Operating Results Research and Development (R&D) Expenses were approximately $3.5 million for the second quarter ended June 30, 2026, as compared to approximately $2.3 million for the second quarter ended June 30, 2025. The increase of approximately $1.2 million was primarily attributable to higher direct R&D expenses related to DA-1726 product development. R&D expenses were approximately $5.6 million for the six months ended June 30, 2026, as compared to approximately $4.6 million for the six months ended June 30, 2025. The approximately $0.9 million increase was primarily attributable to $1.2 million in higher direct R&D expenses related to DA-1726 product development, partially offset by (i) $0.2 million in lower direct R&D expenses related to vanoglipel product development and (ii) $0.1 million in lower indirect employee compensation and benefits costs. General and Administrative (G&A) Expenses were approximately $1.9 million for the second quarter ended June 30, 2026, as compared to approximately $2.0 million for the second quarter ended June 30, 2025. The approximately $0.1 million decrease was primarily attributable to lower legal and professional fees. G&A expenses were approximately $3.8 million for the six months ended June 30, 2026, as compared to approximately $3.5 million for the six months ended June 30, 2025. The approximately $0.3 million increase was primarily attributable to (i) $0.1 million in higher consulting expenditures, (ii) approximately $0.1 million in higher employee compensation and benefits costs, and (iii) $0.1 million in higher other G&A expenses, which was primarily related to higher franchise tax expenses. Total Operating Expenses were approximately $5.4 million for the second quarter ended June 30, 2026, compared to approximately $4.3 million for the second quarter ended June 30, 2025. The approximately $1.1 million increase was primarily attributable to higher R&D expenses and was partially offset by lower G&A expenses. Total operating expenses were approximately $9.4 million for the six months ended June 30, 2026, compared to approximately $8.2 million for the six months ended June 30, 2025. The approximately $1.2 million increase was primarily attributable to higher R&D and G&A expenses for the six months ended June 30, 2026. Total Other Income was approximately $0.1 million for the three months ended June 30, 2026, as compared to approximately $0.3 million for the three months ended June 30, 2025. The approximately $0.2 million decrease was primarily attributable to an approximately $0.2 million negative impact from the change in fair value of warrant liabilities due to the impact of its common stock's decline in stock price and approximately $0.1 million in lower interest income, net, due to lower balances of cash and cash equivalents and lower interest rates. Total other income was approximately $0.3 million for the six months ended June 30, 2026, as compared to approximately $0.5 million for the six months ended June 30, 2025. The approximately $0.2 million decrease was primarily attributable to (i) $0.1 million in lower gain from change in fair value of warrant liabilities due to the impact of the Company's common stock's decline in stock price and (ii) $0.1 million in lower interest income, net, due to lower balances of cash and cash equivalents and lower interest rates. Net Loss was $5.3 million, or $0.90 per basic and diluted share, for the second quarter ended June 30, 2026 based on 5,931,875 weighted average shares of common stock outstanding, compared with a net loss of $4.0 million, or $2.87 per basic and diluted share, based on 1,389,753 weighted average shares of common stock outstanding for the second quarter ended June 30, 2025. Net loss for the six months ended June 30, 2026, was approximately $9.1 million, or $1.69 per basic and diluted share, based on 5,398,683 weighted average shares of common stock, basic and diluted, compared with a net loss of approximately $7.7 million, or $6.59 per basic and diluted share, based on 1,162,692 weighted average shares of common stock, basic and diluted, for the six months ended June 30, 2025. Cash and cash equivalents was $12.6 million as of June 30, 2026, compared with $10.3 million as of December 31, 2025. The company expects its cash position will be adequate to fund operations through 2026. About MetaVia MetaVia Inc. is a clinical-stage biotechnology company focused on transforming cardiometabolic diseases. The company is currently developing DA-1726 for the treatment of obesity, and is developing vanoglipel (DA-1241) for the treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH). DA-1726 is a novel oxyntomodulin (OXM) analogue that functions as a glucagon-like peptide-1 receptor (GLP1R) and glucagon receptor (GCGR) dual agonist. OXM is a naturally-occurring gut hormone that activates GLP1R and GCGR, thereby decreasing food intake while increasing energy expenditure, thus potentially resulting in superior body weight loss compared to selective GLP-1 receptor agonists such as semaglutide. In a Phase 1 multiple ascending dose (MAD) trial in obesity, DA-1726 demonstrated best-in-class potential for weight loss, glucose control, and waist reduction. Vanoglipel is a potential first-in-class drug candidate targeting G-protein-coupled receptor 119 (GPR119). In preclinical studies, vanoglipel demonstrated a positive metabolic effect on glucose and lipid control, and also proved differentiated hepatic benefits reducing hepatic steatosis, hepatic inflammation, and liver fibrosis independent of metabolic improvement. In a Phase 2a clinical study, vanoglipel demonstrated direct hepatic action in addition to its glucose lowering effects. For more information, please visit . Forward Looking Statements Certain statements in this press release may be considered forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "believes", "expects", "anticipates", "may", "will", "should", "seeks", "approximately", "potential", "intends", "projects", "plans", "estimates" or the negative of these words or other comparable terminology (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this press release, including, without limitation, those risks associated with MetaVia's history of net losses, the sufficiency of its existing cash on hand to fund operations and raising additional capital; adverse global economic conditions; MetaVia's ability to execute on its commercial strategy; the timeline for regulatory submissions; the ability to obtain regulatory approval through the development steps of MetaVia's current and future product candidates; the ability to realize the benefits of the license agreement with Dong-A ST Co. Ltd., including the impact on future financial and operating results of MetaVia; the cooperation of MetaVia's contract manufacturers, clinical study partners and others involved in the development of MetaVia's current and future product candidates; potential negative interactions between MetaVia's product candidates and any other products with which they are combined for treatment; MetaVia's ability to initiate and complete clinical trials on a timely basis; MetaVia's ability to recruit subjects for its clinical trials; whether MetaVia receives results from MetaVia's clinical trials that are consistent with the results of preclinical and previous clinical trials; impact of costs related to the license agreement, known and unknown, including costs of any litigation or regulatory actions relating to the license agreement; the effects of changes in applicable laws, regulations or Nasdaq listing rules; the effects of changes to MetaVia's stock price; and other risks and uncertainties described in MetaVia's filings with the Securities and Exchange Commission, including MetaVia's most recent Annual Report on Form 10-K. Forward-looking statements speak only as of the date when made. MetaVia does not assume any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contacts: MetaVia Marshall H. Woodworth Chief Financial Officer +1-857-299-1033 [email protected] Rx Communications Group Michael Miller +1-917-633-6086 [email protected] - Tables to Follow - SOURCE MetaVia Inc. 21% more press release views with Request a Demo

Phase 1Clinical ResultFinancial StatementPhase 2License out/in

100 Deals associated with Metformin

External Link

KEGG	Wiki	ATC	Drug Bank
D04966	Metformin	A10BA02	DB00331

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Differentiated Thyroid Gland Carcinoma	Phase 3	Poland	Medical Research Agency	01 Nov 2022
Ischemic stroke	Phase 3	United States	The Medical University of South Carolina	01 Oct 2008
Diabetes Mellitus, Type 2	Phase 3	United States	National Institute of Diabetes & Digestive & Kidney Diseases	01 May 2004
Depressive Disorder	Phase 2	-	University of Minnesota	01 Jul 2026
HIV Infections	Phase 2	-	University of Minnesota	01 Jul 2026
Mucinous Neoplasm	Phase 2	United States	University of California, Irvine	18 Jun 2026
Pseudomyxoma Peritonei	Phase 2	United States	University of California, Irvine	18 Jun 2026
Ependymoma	Phase 2	United States	The University of California, San Francisco	05 May 2026
Monoclonal Gammopathy of Undetermined Significance	Phase 2	United States	Dana-Farber Cancer Institute, Inc.	27 Apr 2021
Plasma Cell Leukemia	Phase 2	United States	Dana-Farber Cancer Institute, Inc.	27 Apr 2021

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04850846 (CTgov)	Phase 2	Smoldering Multiple Myeloma \| Monoclonal Gammopathy of Undetermined Significance \| Multiple Myeloma ... View more	60	Metformin XR (Metformin)	cgzgavfhum(iwikakcbgo) = uocdojkdfm pxhfmqlwxf (ervynbxxmj, eiswoqrrum - cccapararn) View more	-	09 Feb 2026
				Placebo (Placebo)	cgzgavfhum(iwikakcbgo) = hyixmgootv pxhfmqlwxf (ervynbxxmj, ufweiyrwjl - uajuqwkawu) View more
NCT03076281 (CTgov)	Phase 2	Lymphoid Interstitial Pneumonia \| Squamous Cell Carcinoma of Head and Neck \| Laryngeal Diseases	7	Metformin Hydrochloride (Arm A (Metformin Hydrochloride))	diyznaoocz(jtpwcfkzft) = xjesrwqlwk riuivygbgw (rbqortcjdq, afrshjpkfb - dwlceiqiso) View more	-	29 Jan 2026
				Doxycycline (Arm B (Doxycycline))	diyznaoocz(jtpwcfkzft) = nnzpbiecoh riuivygbgw (rbqortcjdq, ylnenebrsq - txjekbzgdj) View more
NCT02946996 (CTgov)	Phase 2	Prostatic Cancer \| Metastatic Prostate Carcinoma \| Adenocarcinoma of prostate	41	Metformin (Metformin Only)	kykudaqbnm(ujttpdyhyl) = eqvzeqsiey kvxhfqnerh (bodijsqhtq, bsnbdlqhga - bvwxghmhny) View more	-	03 Feb 2025
				OPC (OPC Only)	kykudaqbnm(ujttpdyhyl) = vqvhgcqxgz kvxhfqnerh (bodijsqhtq, nqngffebdg - lrrjhddgzv) View more
Corporate Publications Manual	Phase 3	Diabetes Mellitus, Type 2	-	恒格列净+二甲双胍 (恒格列净5 mg联合组)	nbyuocaixe(eglreldmnr) = bjbyczjtnx ipfbfvizoq (gjqzmvlqji ) View more	Positive	08 Jan 2024
				恒格列净+二甲双胍 (恒格列净10 mg联合组)	nbyuocaixe(eglreldmnr) = zjpezjdwgz ipfbfvizoq (gjqzmvlqji ) View more
NCT04899349 (EPIK-B4, CTgov)	Phase 2	Advanced breast cancer	2	Fulvestrant+Alpelisib+Dapagliflozin+Metformin XR+Dapagliflozin + metformin XR (Alpelisib + Fulvestrant + Dapagliflozin + Metformin XR)	mahixufyok = mehqhdpavf bzwdewhlog (jiimjfdxhe, yoxqyynzjm - mlablicpde) View more	-	12 Dec 2023
				Fulvestrant+Alpelisib+Metformin XR (Alpelisib + Fulvestrant + Metformin XR)	nyiiglxhiy = jlgyewkgoh sckiqbhaey (aaclxpftfx, lvvosiffoq - pfpiqvyfae) View more
FDA Manual	Not Applicable	Diabetes Mellitus, Type 2	1,091	Placebo	hakbfgxebe(wbuoeiavmc) = jfvwgdkzii ddxfvyicki (omrcuguwvx ) View more	Positive	03 Nov 2023
				Sitagliptin 100 mg	hakbfgxebe(wbuoeiavmc) = xexptzwqrx ddxfvyicki (omrcuguwvx ) View more
NCT01968317 (CTgov)	Phase 2	Endometrioid intraepithelial neoplasia \| uterus adenocarcinoma	150	Megestrol acetat+metformin (Megestrol Acetate and Metformin)	lmvcwzsxka = eczvjgshlf nkbrozyuat (snsawmmvmg, upmngjbpec - yfqcogppqg) View more	-	13 Sep 2021
				megestrol acetate (Megestrol Acetate)	lmvcwzsxka = mnlnbwblnh nkbrozyuat (snsawmmvmg, jzzihvrxjh - qigmiachav) View more
CTRI/2018/07/014865 (P-24, ESMO_GI2021)	Phase 1	Advanced Hepatocellular Carcinoma	40	Sorafenib 800mg	svcsqnkash(foljilhrfq) = ijlwvhepxx dzgthcpnlt (fjrwjujmxg ) View more	Positive	03 Jul 2021
				Sorafenib 600mg	jdmbizsmjl(rjyfwkiixk) = bdtyeafcaf vchawlgsol (fegvuyfsyy, 1 - 11)
NCT02496741 (Pubmed \| Cancers (Basel)) Manual	Phase 1/2	IDH1 positive Solid Tumors IDH1 Mutation	12	Metformin+Chloroquine	oemoipbmnb(cokocbbexh) = ipwysfdqmr oebtiinwoq (cnqyegzpco ) View more	Negative	19 May 2021
NCT03981861 (CTgov)	Phase 2/3	Polyendocrine Metabolic Ovarian Syndrome	12	Spironolactone+Metformin	mnrtdszrdy(cfnzxyfsrj) = sunhnywszw yjratoagzu (xbszdkuinm, 2.9) View more	-	23 Feb 2021

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