A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of the NOX1/4 Inhibitor Setanaxib in Patients With Alport Syndrome

The purpose of the study is to learn about the safety and tolerability of setanaxib in subjects with Alport syndrome, when added to their standard of care treatment. The study will assess how safe setanaxib is when compared to placebo. Study participants will be asked if they are experiencing any side effects at each study visit. In addition, tests in blood, urine and other examinations will be used to look at the safety of setanaxib. The study will also measure how well setanaxib works in comparison to a placebo, by measuring urine protein and certain markers in the blood and urine. The concentration of setanaxib in the blood will also be measured throughout the course of the study.
Setanaxib is planned for use together with the current standard of care to hopefully provide additional therapeutic benefits by preserving kidney function.
The study will be conducted at multiple research sites in the UK, Spain, and Czech Republic. Eligible participants will be randomly assigned to receive either setanaxib or placebo. Setanaxib dose level will depend on age and all participants will receive their standard of care in addition to setanaxib or placebo. The study consists of a Screening period of up to 4 weeks, a 24-week Treatment period and a 4- week Follow-up period.

Start Date27 Nov 2023

Sponsor / Collaborator

Calliditas Therapeutics AB

NCT05323656

/ CompletedPhase 2

A Phase 2, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Setanaxib, When Administered With Pembrolizumab, in Patients With Recurrent or Metastatic SCCHN

The primary objective of this study is to compare the change in tumour size per Response Evaluation Criteria in Solid Tumours Version 1.1 (RECIST v1.1) in recurrent or metastatic SCCHN patients treated with setanaxib and pembrolizumab versus patients treated with placebo and pembrolizumab.

Start Date06 Apr 2022

Sponsor / Collaborator-

NCT05014672

/ CompletedPhase 2

TRANSFORM: A 24-week, Randomized, Placebo-controlled, Double-blind, Phase 2b Trial of Setanaxib in Patients With Primary Biliary Cholangitis (PBC) and Elevated Liver Stiffness

The primary objective of this study is to evaluate the effect of setanaxib on alkaline phosphatase (ALP) at Week 24 in participants with PBC and with elevated liver stiffness and intolerance or inadequate response to ursodeoxycholic acid (UDCA).

Start Date14 Feb 2022

Sponsor / Collaborator-

100 Clinical Results associated with Setanaxib

100 Translational Medicine associated with Setanaxib

100 Patents (Medical) associated with Setanaxib

165

Literatures (Medical) associated with Setanaxib

01 Sep 2026·INTERNATIONAL IMMUNOPHARMACOLOGY

Nox4 mediates chondrocyte senescence and cartilage degradation in post-traumatic osteoarthritis via the ROS/p38MAPK signaling pathway

Article

Author: Huang, Danni ; Wang, Shengnan ; Wu, Qian ; Shen, Ke ; Yu, Bin ; Qin, Hanjun ; Chen, Hong ; Jiang, Guiyong ; Chen, Jinhua

Osteoarthritis (OA) is a degenerative joint disease characterized by cartilage degradation and chondrocyte senescence. Emerging evidence implicates NADPH oxidase 4 (Nox4) in OA pathogenesis. This study investigated the role of Nox4 in regulating extracellular matrix (ECM) homeostasis and chondrocyte senescence through the p38 mitogen-activated protein kinase (p38MAPK) signaling pathway. We found that Nox4 expression was significantly upregulated in OA chondrocytes and cartilage. In a post-traumatic OA mouse model (Anterior cruciate ligament transection, ACLT), Nox4 knockout attenuated cartilage damage and chondrocyte senescence, while preserving ECM proteins such as type II collagen and aggrecan. In vitro experiments further demonstrated that Nox4 overexpression accelerated chondrocyte senescence, impaired ECM synthesis, and upregulated senescence markers (p16, p21, p53). Conversely, pharmacological inhibition of Nox4 with GKT137831 or scavenging reactive oxygen species (ROS) with N-acetylcysteine (NAC) alleviated these effects, suggesting that Nox4 contributes to chondrocyte senescence potentially via ROS accumulation. Furthermore, Nox4-induced p38MAPK activation was closely associated with increased senescence and ECM degradation, and these pathological changes were reversed by the p38MAPK inhibitor SB203580. These findings highlight the Nox4-p38MAPK axis as acritical driver of OA progression and suggest that targeting this pathway may offer a promising therapeutic strategy to mitigate cartilage degradation and senescence.

01 Sep 2026·MOLECULAR THERAPY

Treatment with the Nox1/4 inhibitor Setanaxib ameliorates cardiac function in mouse models of Duchenne muscular dystrophy

Article

Author: Wickramage, Ishani ; Chen, Hong ; Teng, Mingxiang ; Mably, John D ; Lin, Tian ; Wang, Da-Zhi ; Zhang, Shiju ; Liu, Xiang ; Semerci, Nihan ; Huang, Zhan-Peng ; Seok, Hee Young ; Guo, Haipeng ; Taheri, Saeid ; Wu, Jingjing ; De Oliveira Silva, Tabatha ; Deng, Rui ; Lu, Yao Wei ; Diniz, Gabriela P ; Fan, Cheng

Duchenne muscular dystrophy (DMD) is caused by mutations in dystrophin, leading to degeneration and weakness of skeletal and cardiac muscle. Despite great progress in the development of gene replacement therapies, DMD remains a devastating disease. In our recent work, we demonstrated that loss of the cardiac Isl1-interacting protein (CIP), which interacts with dystrophin in sarcolemma of cardiomyocytes, accelerates the progression of dystrophic cardiomyopathy and identified Nox4 as one of the downstream mediators of this process. Here, we report that setanaxib, a Nox1/4 inhibitor, protected the heart of CIP/Mdx double knockout (dKO) mice from heart failure; significantly, this compound also reduced cardiac fibrosis and protected against heart failure in Mdx/Utrn dKO mouse. At a molecular level, Nox1/4 inhibition reduced the expression of genes associated with cardiomyopathy in these animals. Further transcriptomic analysis of the hearts treated with setanaxib revealed an enrichment in genes associated with fatty acid metabolism, oxidative phosphorylation, and protein binding, while genes related to epithelial-mesenchymal transition were downregulated. Collectively, these findings suggest that oxidative stress plays a key role in development of myocardial fibrosis and heart failure caused by dystrophin deficiency and suggest that Nox1/4 inhibitor treatment could be a novel therapy to treat DMD-associated cardiomyopathy.

01 Aug 2026·INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES

NOX4 promotes cardiomyocyte ferroptosis-related injury in septic cardiomyopathy in associated with mitochondrial dysfunction and impaired NRF2 nuclear translocation

Article

Author: Zhang, Song ; Zhu, Chaoying ; Cao, Yu ; Qin, Sirun ; Zhu, Can ; Wang, Jian ; Wang, Xinran ; Deng, Lijie ; Chen, Chenyang

Septic cardiomyopathy is a common early complication of sepsis, and its underlying mechanisms remain incompletely understood. In this study, we integrated transcriptomic differential expression analysis, a ferroptosis gene set, protein-protein interaction networks, and consensus machine-learning-based screening to identify NOX4 as a core ferroptosis-related candidate gene. Single-cell transcriptomic analysis further revealed that NOX4 upregulation was most pronounced in cardiomyocytes and was accompanied by enhanced intercellular communication. In the cecal ligation and puncture mouse model, pharmacological inhibition of NOX4 with Setanaxib improved survival and cardiac function, reduced myocardial injury biomarkers, alleviated histopathological damage, and partially reversed the dysregulation of ferroptosis-related markers. In the lipopolysaccharide-stimulated cardiomyocyte model, Setanaxib treatment or genetic silencing with si-NOX4 mitigated the accumulation of ferrous iron, lipid peroxidation, and reactive oxygen species, and partially restored the expression of NRF2, GPX4, SLC7A11, and ACSL4. Mechanistically, NOX4 inhibition attenuated mitochondrial dysfunction, including mitochondrial membrane depolarization, fragmentation, and respiratory dysfunction, and restored nuclear NRF2 levels and downstream antioxidant signaling. In addition, candidate compounds identified from the HERB database and evaluated by molecular docking and molecular dynamics simulations were predicted to exhibit stable binding features with NOX4. Collectively, these findings support a model in which NOX4 contributes to ferroptosis-related injury in septic cardiomyopathy in association with mitochondrial dysfunction and impaired NRF2-dependent antioxidant signaling.

News (Medical) associated with Setanaxib

04 May 2026

·www.medpace.com

ASN Kidney Week: Transformative Nephrology Insights

The 2025 ASN Kidney Week in Houston, TX marked a pivotal moment in nephrology, signaling a shift from broad-stroke treatments toward precision-driven approaches tailored to individual patient needs. From groundbreaking therapies for rare glomerular diseases to the first major advances in Type 1 Diabetes-related CKD in decades, the meeting highlighted how innovation is reshaping care. Generative AI emerged as a powerful tool for uncovering disease subtypes and guiding drug development, while advances in xenotransplantation, APOL1-targeted therapies, and novel biological pathways pointed to a future where treatment is increasingly personalized, predictive, and proactive. In this blog, Dr. Ajay Srivastava, Vice President, Medical Department, reflects on these key takeaways from 2025 ASN Kidney Week and what they mean for the future of renal drug development. Marked a transition for nephrology, moving from “broad-stroke” treatments like SGLT2 inhibitors toward Precision Nephrology. Meeting was dominated by breakthroughs in rare glomerular diseases, the first major therapeutic advance for Type 1 Diabetes (T1D) with CKD in decades, and the rising role of generative AI in drug discovery. The 2025 meeting featured several “practice-changing” trials, particularly in the fields of T1D, IgA Nephropathy (more detailed review later in this summary) and Alport Syndrome. Several notable biological pathways emerged as the primary focus for future drug development: BAFF/APRIL Dual Inhibition: This was the highlight for IgA Nephropathy. Drugs like Atacicept and Telitacicept target these two cytokines to deplete the pathogenic B-cell populations responsible for Gd-IgA1 production. Aldosterone Synthase Inhibition (ASI): Unlike traditional MRAs, ASIs like Lorundrostat block the production of aldosterone itself. This addresses “aldosterone escape,” a common issue where aldosterone levels rise despite ACE/ARB therapy. See also below additional meeting highlights. APOL1-Mediated Kidney Disease (AMKD): A major focus on precision medicine for Black patients. Discussion centered on small molecule inhibitors (like Inaxaplin) that specifically target the toxic function of APOL1 risk variants (G1/G2) in podocytes. See also below additional meeting highlights. NOX1/4 & Oxidative Stress: The Setanaxib data highlighted a shift toward targeting Reactive Oxygen Species (ROS) to combat interstitial fibrosis, particularly in genetic conditions like Alport Syndrome. Anti-CD38 Platform: Felzartamab was highlighted for its ability to target long-lived plasma cells, which are often resistant to standard anti-CD20 (Rituximab) therapy in refractory glomerular diseases. The 2025 meeting confirmed that IgAN treatment has moved well past simple BP control and steroid use. Phase 3 data for BAFF/APRIL inhibitors showed remarkable efficacy with surprisingly manageable safety profiles. The “Advances in Research” conference showcased how AI is moving beyond “chatbots” into structured science. Spatial Proteomics/Lipidomics: Sessions (e.g., Pinaki Sarder, PhD) discussed how AI can now map specific proteins and lipids directly onto a biopsy slide with sub-cellular resolution. This allows us to see exactly where a drug like an SGLT2i is active versus where fibrosis is still pervasive. “Digital Twins” [virtual models] in Trials: Dr. Navdeep Tangri’s group presented on using AI to create prognostic enrichment models. Using AI to select only the “fast progressors” for trials, which significantly increases the power of a study with a smaller sample size. Pathology 2.0: Generative AI is being used to “reconstruct” 3D models of the glomerulus from 2D slides, identifying podocyte foot process effacement that was previously only visible via expensive Electron Microscopy. APOL1-mediated kidney disease (AMKD) was a major focus, specifically for its role in health disparities. Inaxaplin: New data showed that small molecule inhibition of the APOL1 pore is not just stopping progression but potentially reversing some podocyte damage in FSGS patients with high-risk genotypes. Mechanism: Focus is on the G1/G2 variants which cause a “gain of toxicity” through pore formation in the podocyte membrane, leading to cell death. Inaxaplin: New data showed that small molecule inhibition of the APOL1 pore is not just stopping progression but potentially reversing some podocyte damage in FSGS patients with high-risk genotypes. Mechanism: Focus is on the G1/G2 variants which cause a “gain of toxicity” through pore formation in the podocyte membrane, leading to cell death. This was the first major trial in 30 years to show a significant reduction in albuminuria (25%) specifically in Type 1 Diabetes patients. Until now, they were largely excluded from the big MRA/SGLT2 trials. With decades of experience conducting phase I-IV nephrology clinical trials, Medpace is a globally recognized leader in nephrology clinical research. Our seasoned medical in-house nephrologists, operational, and regulatory teams are fully embedded throughout your project – providing unmatched support for your program. Combined with our strong site network and KOL relationships to accelerate development and enhance patient recruitment, we’ve successfully led Phase III studies (several to regulatory approval) in key indications such as acute kidney injury, chronic kidney disease, glomerulonephritis, genetic and rare kidney diseases, and renal transplantation.

Phase 3

30 Mar 2026

·www.prnewswire.com

Calliditas Therapeutics AB to Present Exploratory Biomarker and Post-Hoc NefIgArd Analyses at the World Congress of Nephrology

Calliditas will present new secondary, biomarker, and mechanistic analyses from the Phase 3 NefIgArd study at the 2026 World Congress of Nephrology. Data further support Nefecon's gut–targeted, disease–modifying mechanism of action in IgA nephropathy. Calliditas to present seven posters and sponsor a scientific symposium highlighting the evolving IgAN treatment paradigm. STOCKHOLM, March 30, 2026 /PRNewswire/ -- Calliditas Therapeutics AB, an Asahi Kasei company, announced today that new data from the Phase 3 NefIgArd study in patients with primary immunoglobulin A nephropathy (IgAN) will be presented at the 2026 World Congress of Nephrology (WCN), taking place from March 28 to 31 in Yokohama, Japan. Nefecon's approval status, labeled indication, and availability vary globally. It is approved in the United States where it is marketed as TARPEYO® (budesonide) delayed-release capsules. It is available in Europe where it is marketed as Kinpeygo® by STADA Arzneimittel AG and in select countries in Asia, including Mainland China, Hong Kong, Macau, Taiwan, Singapore, and South Korea by Everest Medicines. It is also the subject of clinical trials in Japan by Viatris Pharamaceticals Japan Inc. The presentations will highlight secondary analyses from the Phase 3 NefIgArd study evaluating Nefecon, its effects on serum biomarkers associated with gut-associated lymphoid tissue (GALT) activity and the immunological impact of orally administered budesonide on B- and T-cell subsets in murine Peyer's patches following in vivo stimulation. These findings reinforce observations from earlier exploratory biomarker analyses and demonstrate the immunomodulatory effects in the Peyer's patches that underlie Nefecon's mechanism of action in modulating the mucosal immune system in the gut. Collectively, these data support a disease-modifying role for Nefecon in reducing levels of pathogenic galactose-deficient IgA1 in patients with IgAN. In total, 7 abstracts, of which 3 are based on secondary clinical efficacy and safety analyses from the Phase 3 NefIgArd study and 2 on mechanistic/biomarker analyses, will be presented. In addition, the initial demographics of the real-world PERFORM registry will be presented. Calliditas will also sponsor a symposium for educational scientific exchange, highlighting the evolving treatment paradigm in IgA nephropathy. Symposium and Presentation details as follows: Company Sponsored Symposium: Title: "The Evolving IgAN Treatment Paradigm: Why Target the Gut?" Date and time: March 30, 12:50 pm to 13:50 pm JST Presenters: Dr. Yusuke Suzuki, Juntendo University, Tokyo, Japan Dr. Sayna Norouzi, Loma Linda University Medical Center, Loma Linda, California, USA Dr. Chee Kay Cheung, University of Leicester, Leicester, UK Poster Presentations: Abstract No. WCN26-AB-8040 Title: "Impact of treatment with Nefecon on serum biomarkers of gut-associated lymphoid tissue function in patients with immunoglobulin A nephropathy from the Phase 3 NefIgArd trial" Date and time: March 30, 17:00 pm – 18:00 pm JST Presenter: Nadia Nawaz, College of Life Sciences, University of Leicester, Leicester, United Kingdom Abstract No. WCN26-AB-8197 Title: "Budesonide Modulates B- and T-Cell Immunity in Peyer's Patches: Implications for IgA Nephropathy" Date and time: March 28, 17:30 pm – 18:30 pm JST Presenter: Roza Ghotbi, Calliditas Therapeutics AB, Stockholm, Sweden Encore Poster Presentations: Title: "Urine protein–creatinine ratio response at 12 months in patients with immunoglobulin A nephropathy receiving Nefecon vs placebo: Analysis of NefIgArd trial data" Date and time: March 28, 17:30 pm – 18:30 pm JST Presenter: Richard Lafayette, Stanford University, CA Title: "Duration of absolute proteinuria response with Nefecon in patients with primary immunoglobulin A nephropathy: Results from the 2-year NefIgArd trial" Date and time: March 30, 17:00 pm – 18:00 pm JST Presenter: Brad Rovin , The Ohio State University Wexner Medical Center Title: "Absolute proteinuria over time with Nefecon vs placebo in patients with immunoglobulin A nephropathy: Results from the Phase 3 NefIgArd trial" Date and time: March 30, 17:00 pm – 18:00 pm JST Presenter: Heather Reich, University of Toronto Title: "The creation and initial demographics of the PERFORM™ Patient Registry: A novel real-world registry of patients treated for IgA nephropathy in the United States" Date and time: March 29, 17:00 pm – 18:00 pm JST Presenter: Russell Jones, Calliditas Therapeutics AB, Stockholm, Sweden Title: "Safety and preliminary efficacy findings from a Phase 2a randomized, double-blind, placebo-controlled trial of setanaxib in patients with Alport syndrome" Date and time: March 28, 17:30 pm – 18:30 pm JST Presenter: Daniel Gale , University College London The World Congress of Nephrology (WCN) is the flagship scientific, educational, and networking event of the International Society of Nephrology (ISN). Each year, WCN brings together kidney care professionals from around the world to exchange knowledge, showcase groundbreaking research, and participate in regionally-relevant symposia, training programs, and courses. About TARPEYO ® /Kinpeygo® TARPEYO is an oral 4mg delayed-release formulation of budesonide, designed to dissolve in the pH of the distal ileum. Each capsule contains coated beads of budesonide that target mucosal B-cells present in the ileum, including the Peyer's patches, which are responsible for the production of galactose-deficient IgA1 antibodies (Gd-IgA1), causing IgA nephropathy. About Primary Immunoglobulin A Nephropathy Primary immunoglobulin A nephropathy (IgA nephropathy or IgAN or Berger's Disease) is a rare, progressive, chronic immune-mediated disease that attacks the kidneys and occurs when galactose-deficient IgA1 is recognized by autoantibodies, creating IgA1 containing immune complexes that become deposited in the glomerular mesangium of the kidney. This deposition in the kidney can lead to progressive kidney damage, potentially resulting in end-stage kidney disease. IgAN most often develops between late teens and late 30s. IMPORTANT SAFETY INFORMATION FOR US AUDIENCE INDICATION TARPEYO is indicated to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS TARPEYO is contraindicated in patients with hypersensitivity to budesonide or any of the ingredients of TARPEYO. Serious hypersensitivity reactions, including anaphylaxis, have occurred with other budesonide formulations. WARNINGS AND PRECAUTIONS Hypercorticism and adrenal axis suppression: When corticosteroids are used chronically, systemic effects such as hypercorticism and adrenal suppression may occur. Corticosteroids can reduce the response of the hypothalamus-pituitary-adrenal (HPA) axis to stress. In situations where patients are subject to surgery or other stress situations, supplementation with a systemic corticosteroid is recommended. When discontinuing therapy or switching between corticosteroids, monitor for signs of adrenal axis suppression. Patients with moderate to severe hepatic impairment (Child-Pugh Class B and C respectively) could be at an increased risk of hypercorticism and adrenal axis suppression due to an increased systemic exposure to oral budesonide. Avoid use in patients with severe hepatic impairment (Child-Pugh Class C). Monitor for increased signs and/or symptoms of hypercorticism in patients with moderate hepatic impairment (Child-Pugh Class B). Immunosuppression and Increased Risk of Infection: Corticosteroids, including TARPEYO, suppress the immune system and increase the risk of infection with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic pathogens. Corticosteroids can: reduce resistance to new infections, exacerbate existing infections, increase the risk of disseminated infections, increase the risk of reactivation or exacerbation of latent infections, and mask some signs of infection. Corticosteroid-associated infections can sometimes be serious. Monitor for infection and consider TARPEYO withdrawal as needed. Avoid corticosteroid therapy, including TARPEYO, in patients with active or quiescent tuberculosis or hepatitis B infection; untreated fungal, bacterial, systemic viral, or parasitic infections; ocular herpes simplex; or Kaposi's sarcoma. Avoid exposure to active, easily-transmitted infections (e.g., chickenpox, measles). Corticosteroid therapy may decrease the immune response to some vaccines. Other corticosteroid effects: TARPEYO is a systemically available corticosteroid and is expected to cause related adverse reactions. Monitor patients with hypertension, prediabetes, diabetes mellitus, osteoporosis, peptic ulcer, glaucoma or cataracts, or with a family history of diabetes or glaucoma, or with any other condition where corticosteroids may have unwanted effects. ADVERSE REACTIONS In clinical studies, the most common adverse reactions with TARPEYO (occurring in ≥5% of TARPEYO treated patients, and ≥2% higher than placebo) were peripheral edema (17%), hypertension (12%), muscle spasms (12%), acne (11%), headache (10%), upper respiratory tract infection (8%), face edema (8%), weight increased (7%), dyspepsia (7%), dermatitis (6%), arthralgia (6%), and white blood cell count increased (6%). DRUG INTERACTIONS Budesonide is a substrate for CYP3A4. Avoid use with potent CYP3A4 inhibitors, such as ketoconazole, itraconazole, ritonavir, indinavir, saquinavir, erythromycin, and cyclosporine. Avoid ingestion of grapefruit juice with TARPEYO. Intake of grapefruit juice, which inhibits CYP3A4 activity, can increase the systemic exposure to budesonide. USE IN SPECIFIC POPULATIONS Pregnancy: The available data from published case series, epidemiological studies, and reviews with oral budesonide use in pregnant women have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with IgAN. Infants exposed to in-utero corticosteroids, including budesonide, are at risk for hypoadrenalism. Please see Full Prescribing Information About Calliditas Therapeutics Calliditas Therapeutics is a biopharma company with offices in Stockholm, Sweden, focused on identifying, developing, and commercializing novel treatments in orphan indications with significant unmet medical needs. Visit Calliditas.com for further information. About Asahi Kase i Asahi Kasei is a diversified global company that contributes to life and living for people around the world. Since its foundation in 1922, with businesses in ammonia and cellulose fiber, Asahi Kasei has consistently grown through proactive portfolio transformation to meet the evolving needs of every age. With 50,000 employees worldwide, the company contributes to sustainability by providing solutions to the world's challenges across its three business sectors: Healthcare, Homes, and Material. For more information, visit . SOURCE Calliditas Therapeutics 21% more press release views with Request a Demo

Phase 3Drug ApprovalClinical ResultPhase 2

18 Nov 2025

·www.drugs.com

American Society of Nephrology, Nov. 5-9

The annual meeting of the American Society of Nephrology (Kidney Week) was held this year from Nov. 5 to 9 in Houston, attracting attendees from around the world, including nephrology specialists, researchers, scientists, and other health care professionals. The conference featured presentations focusing on the latest advances in the management of patients with kidney diseases and related disorders. In one study, Daniel Gale, Ph.D., of University College London, and colleagues showed that an NADPH oxidase synthase (NOX) 1/4 inhibitor, setanaxib, has an acceptable safety profile, with some evidence of reduced proteinuria among patients with Alport syndrome. The researchers analyzed the safety and efficacy of setanaxib in Alport syndrome. In a 2:1 randomized controlled trial of 20 patients, 24 weeks of treatment was well tolerated, and there was some evidence of reduced proteinuria in the treatment group compared with placebo. The study was a small phase 2a exploratory study and none of the differences observed were statistically significant. "We conclude that these results justify a further (larger and longer) randomized controlled study to quantify the safety and efficacy of this drug to treat people with Alport syndrome," Gale said. The study was funded by Calliditas Therapeutics, which is developing setanaxib. Abstract No. SA-OR082 In another study, Charmaine E. Lok, M.D., of the University Health Network in Toronto, and colleagues found that daily oral supplementation of eicosapentaenoic acid (EPA) and docosahexaenoic acid (DHA) in the form of fish oil is safe and reduces the rate of serious cardiovascular events in patients with kidney failure on maintenance hemodialysis. As part of the PISCES study, 1,228 patients who were treated with chronic hemodialysis were randomly assigned to oral daily fish oil or placebo capsules. The investigators found that the rate of serious cardiovascular events (heart attack, stroke, peripheral vascular disease leading to amputation, and cardiac death) was reduced by approximately 40 percent in patients treated with maintenance hemodialysis who received daily oral supplementation of 4 g of fish oil (total EPA, 1.6 g; total DHA, 0.8 g) compared with placebo. The rate of serious cardiovascular events was 0.31/1,000 days in the fish oil group versus 0.61/1,000 days in the placebo group. Adverse events, including bleeding, did not differ between the groups. "Given the underlying high risk of cardiovascular events, lack of other preventative measures, and safety of fish oil, I would not be surprised if there is quick uptake of fish oil supplementation in patients receiving hemodialysis," Lok said. "It should be emphasized that the dose and formulation of fish oil, and how it is taken, is important." Abstract No. FR-OR082 Natalie Staplin, Ph.D., of the University of Oxford Medical Sciences Division in the United Kingdom, and colleagues demonstrated clear absolute benefits of sodium-glucose cotransporter 2 (SGLT2) inhibitors on kidney, hospitalization, and mortality outcomes irrespective of diabetes status and level of urine albumin-to-creatinine ratio. The authors conducted a meta-analysis of 58,816 participants enrolled in eight large randomized trials of SGLT2 inhibitors, including empagliflozin, dapagliflozin, and canagliflozin, compared with placebo. The investigators found that SGLT2 inhibitors substantially reduced the risks for kidney disease progression, acute kidney injury, hospitalization, and death among individuals with and without diabetes and across all levels of albuminuria. Compared with placebo, allocation to SGLT2 inhibition reduced the hazard of kidney disease progression by 35 percent among participants with diabetes and 26 percent among participants without diabetes. Allocation to SGLT2 inhibition reduced the risk for acute kidney injury to a similar extent in participants with and without diabetes by 23 and 28 percent, respectively. The risk for hospitalization for heart failure was 32 and 25 percent lower in people with and without diabetes, respectively, who were allocated to an SGLT2 inhibitor, and the risk for any hospitalization was reduced by 10 and 11 percent, respectively. People with lower albumin levels, who are often at lower risk for kidney failure, experienced benefit as a result of taking an SGLT2 inhibitor, particularly through reduced hospital admissions for heart failure and any other cause. Participants with higher albuminuria (≥200 mg/g) had larger absolute reductions in kidney disease progression. "The findings suggest that guideline recommendations distinguishing between patients based on diabetes status or albuminuria level may no longer be necessary," Staplin said. "Current international kidney disease guidelines give stronger recommendations for using SGLT2 inhibitors in patients with high albumin levels, but the new results indicate that all patients with chronic kidney disease can benefit." Abstract No. FR-OR086 In the multicenter, randomized OPTIMIZE trial, Stefan P. Berger, Ph.D., of the Universitair Medisch Centrum Groningen in the Netherlands, and colleagues found that a reduced-dose immunosuppressive regimen combining low-dose tacrolimus plus everolimus is safe among older kidney transplant recipients (age 65 years and older), but the regimen does not lead to improved outcomes compared with the current standard regimen. The authors investigated whether older kidney transplant recipients could benefit from a reduced and less nephrotoxic immunosuppressive regimen. Three hundred seventy-nine kidney transplant recipients aged 65 years or older, across six Dutch university medical centers and the University Hospitals Leuven, were included in the study. The study compared a "standard" immunosuppressive regimen (prednisolone + standard-dose tacrolimus + mycophenolate mofetil) versus an "adapted" regimen (prednisolone + low-dose tacrolimus + everolimus). The primary outcome was defined as a "successful transplantation" at two years: patient alive with functioning graft and minimum estimated glomerular filtration rate thresholds (≥30 mL/min/1.73m 2 for older deceased-donor kidneys; ≥45 mL/min/1.73m 2 for younger deceased donor or living donor kidneys). At two years, the authors observed no statistically significant difference between arms -- 50 percent success in the low-dose arm versus 57 percent in the standard-dose arm. "Importantly, the adapted regimen was safe, but it did not demonstrate superiority over the standard regimen in this older population," Berger said. "Clinicians should be reassured that older transplant recipients achieve very good outcomes under current standard regimens (89 percent patient survival; only 5 to 6 percent graft loss at two years) and that a reduced regimen is safe. We should move toward stratified immunosuppression in older patients with an awareness that immunosuppression can be reduced safely in most cases. Clearly the OPTIMIZE trial had not hit the threshold for reduction of immunosuppression. The study highlights the need for further research to tailor the immunosuppressive regimen to the individual older patient (taking into account donor age, immunosenescence, comorbidities, frailty, etc.)." The study was funded by Chiesi Pharmaceuticals. Abstract No. FR-OR085 ASN: Nearly All Adults With CKD Would Benefit from Systolic BP Target <120 mm Hg WEDNESDAY, Nov. 12, 2025 -- Nearly all adults with chronic kidney disease would benefit from a systolic blood pressure target <120 versus <140 mm Hg, according to a study presented at Kidney Week, the annual meeting of the American Society of Nephrology, held from Nov. 5 to 9 in Houston. Read Full Text ASN: Similar Risks Seen for Young, Older Patients With Primary Glomerular Diseases WEDNESDAY, Nov. 12, 2025 -- For patients with primary glomerular diseases, young patients face similar risks of kidney function decline as older patients, according to a study presented at Kidney Week, the annual meeting of the American Society of Nephrology, held from Nov. 5 to 9 in Houston. Read Full Text ASN: Expanding Kidney Transplant Wait-List Criteria to Include Risk for Kidney Failure Could Improve Outcomes WEDNESDAY, Nov. 12, 2025 -- Expanding the wait-listing criteria for kidney transplantation to include risk for kidney failure -- rather than taking a single measurement of kidney function -- could improve outcomes, according to a study presented at Kidney Week, the annual meeting of the American Society of Nephrology, held from Nov. 5 to 9 in Houston. Read Full Text ASN: New Tool Predicts CKD Survival With Conservative Management Versus Dialysis WEDNESDAY, Nov. 12, 2025 -- A new tool can predict individualized probability of survival with conservative management versus dialysis transition among individuals with advanced chronic kidney disease, according to a study presented at Kidney Week, the annual meeting of the American Society of Nephrology, held from Nov. 5 to 9 in Houston. Read Full Text ASN: Large Gap Between Creatinine-Based eGFR and Cystatin C-Based eGFR Tied to Worse Outcomes MONDAY, Nov. 10, 2025 -- In the outpatient setting, the presence of a cystatin C-based estimated glomerular filtration rate that is at least 30 percent lower than creatinine-based eGFR is associated with significantly higher rates of all-cause mortality, cardiovascular events, and kidney failure, according to a study published online Nov. 7 in the Journal of the American Medical Association to coincide with Kidney Week, the annual meeting of the American Society of Nephrology, held from Nov. 5 to 9 in Houston. Read Full Text ASN: Prevalence of Chronic Kidney Disease Increasing Globally MONDAY, Nov. 10, 2025 -- The prevalence of chronic kidney disease is increasing globally, according to a study published online Nov. 7 in The Lancet to coincide with Kidney Week, the annual meeting of the American Society of Nephrology, held from Nov. 5 to 9 in Houston. Read Full Text

Clinical ResultPhase 2

100 Deals associated with Setanaxib

External Link

KEGG	Wiki	ATC	Drug Bank
-	Setanaxib	-	-

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Nephritis, Hereditary	Phase 2	Austria	Calliditas Therapeutics AB	27 Nov 2023
Nephritis, Hereditary	Phase 2	Czechia	Calliditas Therapeutics AB	27 Nov 2023
Nephritis, Hereditary	Phase 2	France	Calliditas Therapeutics AB	27 Nov 2023
Nephritis, Hereditary	Phase 2	Spain	Calliditas Therapeutics AB	27 Nov 2023
Nephritis, Hereditary	Phase 2	United Kingdom	Calliditas Therapeutics AB	27 Nov 2023
Idiopathic Pulmonary Fibrosis	Phase 2	Sweden	Calliditas Therapeutics France SAS	30 Jan 2022
Type 1 diabetes mellitus with established diabetic nephropathy	Phase 2	Australia	Calliditas Therapeutics France SAS	12 Dec 2017
Primary Biliary Cholangitis	Phase 2	United States	Calliditas Therapeutics AB	26 Jun 2017
Primary Biliary Cholangitis	Phase 2	Belgium	Calliditas Therapeutics AB	26 Jun 2017
Primary Biliary Cholangitis	Phase 2	Canada	Calliditas Therapeutics AB	26 Jun 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03865927 (GKT137831, CTgov)	Phase 2	Idiopathic Pulmonary Fibrosis	58	Placebo Oral Tablet (Placebo Oral Tablet)	kxsgbbwclu(rivhaujvrd) = lhgmddmrqy rebktjpmlz (wdkqofpioh, 255.5) View more	-	11 May 2026
				GKT137831 (GKT137831)	kxsgbbwclu(rivhaujvrd) = pstmmumscj rebktjpmlz (wdkqofpioh, 39.3) View more
NCT06274489 (CTgov)	Phase 1/2	Nephritis, Hereditary	20	Setanaxib (Setanaxib)	kkkwbdizcu = gnsmwgpqvw qyxptbxwxz (lhtobybryq, aiaqmwnluh - obcqiyzrad) View more	-	13 Apr 2026
				Placebo (Setanaxib Placebo)	tkkdbhuvcq(itwyxgghjk) = qzjfoscwjv imkysatere (wavzadnszm, zoukdrnblx - hnlfsupvbw) View more
NCT06274489 (ISN WCN2026)	Phase 2	Nephritis, Hereditary	20	Setanaxib + Setanaxibd therapy	ibvpktliwk(rymesnbuac) = zdjsnicvls toewnexfjq (vcmmebbxgr ) View more	Positive	01 Apr 2026
NCT05323656 (GSN000400, CTgov)	Phase 2	Squamous cell carcinoma of head and neck metastatic	55	Pembrolizumab+Setanaxib (Setanaxib 1600 mg and Pembrolizumab 200 mg)	wvdbtdfefh(pdycjpxhww) = aqayoceaiq vvxikljiyq (ipdyqohhic, 9.323) View more	-	09 May 2025
				Placebo+Pembrolizumab (Placebo and Pembrolizumab 200 mg)	wvdbtdfefh(pdycjpxhww) = extpkhouro vvxikljiyq (ipdyqohhic, 8.977) View more
NCT05014672 (TRANSFORM, CTgov)	Phase 2	Primary Biliary Cholangitis \| Stiffness	76	Setanaxib (Setanaxib 1200 mg/Day)	iugibblnoy(zymkmoqqbv) = vebqcixgtw hofemjhwdl (tbffdwdjnd, rabwookwoy - boqiuanbxm) View more	-	24 Apr 2025
				Setanaxib (Setanaxib 1600 mg/Day)	iugibblnoy(zymkmoqqbv) = uqhumcusoa hofemjhwdl (tbffdwdjnd, ghayaskmnn - nntuochwbz) View more
NCT02010242 (CTgov)	Phase 2	Diabetes Mellitus, Type 2 \| Type 2 diabetes mellitus with established diabetic nephropathy \| Albuminuria	136	GKT137831 (GKT137831)	qbkvxdlkss(arxrehizzx) = cfsytncmvl kuicrlbezd (nswbhtxtuc, buwznadepb - uihvfhziru) View more	-	28 Feb 2025
				Placebo (Placebo)	qbkvxdlkss(arxrehizzx) = unxrsphhxd kuicrlbezd (nswbhtxtuc, ikcctfbnld - zdctamrnfj) View more
NCT05323656 (GSN000400, ESMO2024) Manual	Phase 2	Squamous Cell Carcinoma of Head and Neck First line	55	Setanaxib 800 mg BID + Pembrolizumab 200 mg IV every 3 weeks	jpeijgozwj(yjkfkqinum) = tgxylmybbx laxucjlrjx (jilqzbjlip, 9.323) View more	Positive	14 Sep 2024
				Placebo + Pembrolizumab 200 mg IV every 3 weeks	jpeijgozwj(yjkfkqinum) = prvdzfyhqq laxucjlrjx (jilqzbjlip, 8.997) View more
NCT05014672 (TRANSFORM, PRNewswire) Manual	Phase 2	Primary Biliary Cholangitis	76	setanaxib	yupgshjcgw(efwvqewmch) = Patients treated with setanaxib showed statistically significant improvements in the primary endpoint of ALP of 19% in the 1600mg arm and 14% in the 1200mg arm and showed positive trends on liver stiffness assessed by FibroScan® at 24 weeks. byndodpnrs (lgfpaedexj ) Met View more	Positive	26 Jul 2024
				Placebo
NCT05323656 (PRNewswire) Manual	Phase 2	Squamous Cell Carcinoma of Head and Neck	55	Setanaxib+Pembrolizumab	jeuqjwmazl(ewxwtuterj) = evniwdmsih lruzjgwtlq (xvytsgjber ) View more	Positive	06 May 2024
				Pembrolizumab + Placebo	jeuqjwmazl(ewxwtuterj) = kjfgiqpdjk lruzjgwtlq (xvytsgjber ) View more
NCT03226067 (Pubmed)	Phase 2	Primary Biliary Cholangitis Second line	111	Setanaxib 400 mg OD	zrlgtofwnn(ucozfqynsh) = sadmlsoslj pjfweprphe (qjixolfjrx, 59.6) View more	Positive	15 May 2023
				Setanaxib 400 mg BID	zrlgtofwnn(ucozfqynsh) = cjawbaolkm pjfweprphe (qjixolfjrx, 28.9) View more

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