Evrenzo (roxadustat) / Astellas, AstraZeneca, Kyntra Bio - LARVOL DELTA

Phosphate Binder Selection, Dosing Interval Adherence, and Suboptimal Anemia Control in Maintenance Hemodialysis Patients on Roxadustat During the Volume-Based Procurement Transition Period: A Case-Control Study (ChiCTR) - P=N/A | N=50 | Not yet recruiting | Sponsor: Mindong Hospital of Ningde City; Mindong Hospital of Ningde City

New trial • Anemia • Chronic Kidney Disease • Hematological Disorders • Nephrology • Renal Disease

Roxadustat in CKD and transplant patients: A new era in anemia management (TTS 2026) - "Roxadustat appears to be an effective and generally safe therapeutic option for managing anemia in CKD patients, including those who are HDD, NDD, or RTRs. However, monitoring for cardiovascular events, such as myocardial infarction, is essential in these patients."

Clinical • Anemia • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Myocardial Infarction • Nephrology • Renal Disease • Transplantation

HIF-PH Inhibitor Promotes Stabilization of HIF-1α via Inhibition of Its Degradation and Exerts Chondroprotective Effects in a Rat Osteoarthritis Model. (PubMed, Int J Mol Sci) - "These findings demonstrate that Roxadustat promotes anabolic responses in chondrocytes through stabilization of HIF-1α and suppresses cartilage degeneration in OA. Intra-articular administration of HIF-PH inhibitors may represent a novel disease-modifying therapeutic strategy for OA."

Journal • Preclinical • Hematological Disorders • Immunology • Osteoarthritis • Pain • Rheumatology • ACAN • HIF1A • SOX9 • YBX1

Dose Conversion Ratio Between Roxadustat and Daprodustat in Patients With Non-dialysis Chronic Kidney Disease. (PubMed, Cureus) - "Our study results suggest that the dose conversion ratio between roxadustat and daprodustat is approximately 10:1 in non-dialysis CKD patients."

Journal • Chronic Kidney Disease • Hematological Disorders • Nephrology • Renal Disease

Epicatechin alleviates ulcerative colitis by restoring intestinal mucosa via regulating the NCR+ILC3 cell counts. (PubMed, Phytomedicine) - "Epicatechin alleviates DSS-induced colitis by directly targeting NLE1, enhancing NLE1-NICD binding and reducing NICD-FBXW7 interaction to stabilize NICD protein, thereby activating the NLE1/NICD/HIF-1α signaling axis and restoring NCR⁺ILC3 numbers and IL-22 secretion. These findings establish a molecular link between dietary flavonoids and ILC3-mediated immunity, positioning epicatechin as a phytochemical lead for UC mucosal repair."

Journal • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammation • Inflammatory Bowel Disease • Targeted Protein Degradation • Ulcerative Colitis • FBXW7 • HIF1A • IL17A • IL22 • NCR1 • NICD • OCLN • RORC • TJP1

Open-label, Phase 2 study of roxadustat for the treatment of anemia in patients receiving chemotherapy for non-myeloid malignancies. (PubMed, Am J Hematol) - "Roxadustat increased Hb in patients with CIA regardless of tumor type and chemotherapy regimen. Adverse events were consistent with observations in patients with advanced-stage malignancies."

Journal • P2 data • Anemia • Cardiovascular • Chemotherapy-Induced Anemia • Hematological Disorders • Hematological Malignancies • Oncology • Pulmonary Embolism • Respiratory Diseases • Venous Thromboembolism

Efficacy and Safety of Roxadustat for Treatment of Anemia in Patients with Lower-Risk Myelodysplastic Syndrome (LR-MDS) with Low Red Blood Cell (RBC) Transfusion Burden: Results of Phase III Matterhorn Study (ASH 2023) - P3 | "Despite not meeting the primary endpoint, roxadustat plus BSC was well-tolerated, and a high percentage of pts with LR-MDS and LTB were TI responders. The high TI response rate in the PBO arm, historically poor outcomes in pts with ESA-refractory disease, and the inclusion of pts who were not transfusion-dependent (1 pRBC unit Q8W) may have contributed to the lack of a statistically significant difference in TI response rates between arms. MATTERHORN outcomes highlight the continued unmet need for effective and safe therapies that reduce RBC transfusion burden in LR-MDS."

Clinical • P3 data • Acute Myelogenous Leukemia • Anemia • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Hematological Malignancies • Infectious Disease • Ischemic stroke • Leukemia • Myelodysplastic Syndrome • Myocardial Infarction • Nephrology • Oncology • Pneumonia • Renal Disease • Respiratory Diseases

Efficacy and Safety of Roxadustat for Anemia in Patients Receiving Chemotherapy for Nonmyeloid Malignancies: A Randomized, Open-Label, Active-Controlled Phase III Study. (PubMed, J Clin Oncol) - "Roxadustat was noninferior to rHuEPO-α in treating CIA in participants with nonmyeloid malignancies receiving multicycle treatments of myelosuppressive chemotherapy. The oral formulation of roxadustat may potentially increase compliance."

Journal • P3 data • Anemia • Chemotherapy-Induced Anemia • Hematological Disorders • Oncology

Comparative efficacy and safety of methoxy polyethylene glycol-epoetin beta and roxadustat in the management of renal anemia in maintenance hemodialysis. (PubMed, Front Pharmacol) - "Hb target attainment and hypertension-rate comparisons did not show statistically significant treatment-by-time interaction effects. Because treatment allocation reflected clinical practice and patient preference, residual selection bias remains an important competing explanation."

Journal • Anemia • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Hypertension • Nephrology • Renal Disease • CRP

Roxadustat Versus Erythropoietin for Anaemia Treatment in Diabetic Nephropathy (clinicaltrials.gov) - P=N/A | N=64 | Completed | Sponsor: Hanzla Arshad

New trial • Anemia • Chronic Kidney Disease • Diabetic Nephropathy • Hematological Disorders • Nephrology • Renal Disease

Rapid, HIF-1α-independent, response of VEGF secretion to decreased O2 in mouse retina. (PubMed, Proc Natl Acad Sci U S A) - "Moreover, increased HIF-1α induced by the drug Roxadustat, did not stimulate VEGF secretion at atmospheric (21%) O2 and a steady rate of VEGF secretion was sustained for over 120 min following elimination of HIF-1α and protein synthesis by exposure to the protein synthesis inhibitor cycloheximide...These stores are depleted within 30 min once [O2] is decreased. Taken together, these findings indicate that VEGF is constitutively made (under the influence of HIF-1α and other transcription factors) and stored within the retina, and rapid release occurs after a substantial decrease in available O2 independent of HIF-1α."

Journal • Preclinical • Retinal Disorders • HIF1A • VEGFA

Roxadustat-Associated Cholestatic Jaundice in Haemodialysis Patients with Positive Rechallenge. (PubMed, Eur J Case Rep Intern Med) - "This case series provides clinical evidence supporting a causal association between roxadustat and cholestatic jaundice in haemodialysis patients, warranting clinical awareness of this potential adverse effect."

Journal • Cholestasis • Chronic Kidney Disease • Hematological Disorders • Hepatology • Nephrology • Renal Disease

…Upcoming Milestones:…Roxadustat (GlobeNewswire) - "Pivotal Phase 3 trial protocol of roxadustat for the treatment of anemia in patients with LR-MDS and high transfusion burden (HTB) has been finalized....Company continues to explore the opportunity to develop roxadustat internally or with a strategic partner, with the goal of initiating the Phase 3 trial in the fourth quarter of 2026."

New P3 trial • Anemia • Myelodysplastic Syndrome

Bioresponsive gelatin-coated silk fibroin nanoparticles enhance roxadustat therapy in experimental pulmonary fibrosis. (PubMed, Front Pharmacol) - "Integrated recovery analysis showed the highest overall fibrosis recovery index for GT-RXS-SKNPs (85.3%), outperforming uncoated RXS-SKNPs (60.8%) and free RXS (34.1%). Overall, GT-RXS-SKNPs improved pulmonary retention and translated enhanced lung delivery into superior antifibrotic efficacy while limiting systemic exposure."

Journal • Fibrosis • Immunology • Pulmonary Disease • Respiratory Diseases • MMP2 • MMP9 • TGFB1

Association of gut microbiome signatures with iron metabolism response to roxadustat in peritoneal dialysis patients with anemia: a cross-sectional study. (PubMed, Front Pharmacol) - "The final model achieved an AUC exceeding 0.8, with favorable calibration and clinical utility, and showed good discriminative performance for iron metabolism non-response to roxadustat in PD patients. Distinct gut microbiome signatures are associated with the iron metabolism response to roxadustat in anemic PD patients."

Journal • Observational data • Anemia • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Nephrology • Renal Disease

Roxadustat and incidence of peritoneal dialysis-associated peritonitis: a propensity score-matched cohort study. (PubMed, Clin Exp Nephrol) - "Roxadustat was associated with a reduced incidence of PDAP compared to rhuEPO, suggesting that HIF stabilization may offer protective benefits beyond anemia correction. Prospective validation is warranted."

Journal • Hematological Disorders • Inflammation

Clinical characteristics and influencing factors of roxadustat-induced central hypothyroidism in patients receiving peritoneal dialysis. (PubMed, Perit Dial Int) - "Multivariate analysis identified hypoalbuminemia as an independent risk factor for CEH. Therefore, thyroid function should be routinely monitored in PD patients before and during roxadustat therapy."

Journal • Endocrine Disorders • Hematological Disorders

Efficacy of roxadustat for anaemia in kidney transplant recipients and its drug-drug interaction with tacrolimus: A retrospective cohort study. (PubMed, Br J Clin Pharmacol) - "Roxadustat is an effective oral treatment for PTA, demonstrating superior efficacy to rhEPO at 6 months. However, it is associated with a significant drug-drug interaction that may increase TAC exposure. These findings warrant further prospective studies to validate this drug-drug interaction."

Journal • Retrospective data • Anemia • Hematological Disorders • Transplantation

HIF1 Stabilization by Roxadustat Improves Cognition and Prevents Neuron Loss in Alzheimer's Diseases In Vivo. (PubMed, Biology (Basel)) - "In addition, animals treated with Roxadustat exhibited significantly increased expression of the brain-derived neurotrophic factor (BDNF) in the cerebral cortex. Our findings suggest that stabilization of the HIF-1 complex through inhibition of HIF prolyl hydroxylase may represent a promising strategy for neuroprotection in AD."

Journal • Preclinical • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • BDNF • HIF1A

A Study to Investigate the Efficacy and Safety of Roxadustat (FG-4592) for Treating Anemia in Participants With Myelodysplastic Syndromes (MDS) (clinicaltrials.gov) - P3 | N=201 | Not yet recruiting | Sponsor: Kyntra Bio

New P3 trial • Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Comparison of Efficacy and Safety between MIRCERA and Roxadustat in the Treatment of Renal Anemia (ChiCTR) - P4 | N=60 | Completed | Sponsor: Guangdong Second Provincial General Hospital; Guangdong Second Provincial General Hospital

New P4 trial • Anemia • Hematological Disorders

The Clinical Significance of Enarodustat,Roxadustat and Erythropoiesis Stimulatig Agents in Non-Dialysis Patients with Renal Anemia (ChiCTR) - P=N/A | N=150 | Not yet recruiting | Sponsor: The Affiliated Hospital of Xuzhou Medical University; The Affiliated Hospital of Xuzhou Medical University

New trial • Anemia • Chronic Kidney Disease • Hematological Disorders • Nephrology • Renal Disease

Roxadustat-loaded pH-sensitive Anionic Liposomes Ameliorating Experimental Colitis in Mice: Formulation, Cellular Uptake and In vivo Evaluation. (PubMed, Curr Drug Deliv) - "ROX-MAE@LP is an effective intestine-targeted delivery system that improves roxadustat's solubility, bioavailability and therapeutic outcomes in inflammatory bowel disease, while offering additional options for the development of novel roxadustat-based preparations and pHresponsive nanocarriers."

Journal • Preclinical • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammation • Inflammatory Bowel Disease

Association of Roxadustat Versus Erythropoiesis-Stimulating Agents With Renal Outcomes in Non-Dialysis CKD: A Propensity-Matched Real-World Cohort Study. (PubMed, Clin Ther) - "Among patients with non-dialysis-dependent CKD and anemia, roxadustat and ESAs were associated with similar renal outcomes overall. Subgroup findings suggest possible heterogeneity in treatment effects by age. Further studies are warranted to clarify the clinical relevance of these findings."

Journal • Real-world evidence • Chronic Kidney Disease • Hematological Disorders • Nephrology • Renal Disease

Roxadustat Versus ESA Therapy for Sustained Hemoglobin Response in ESA-Hyporesponsive Hemodialysis Patients. (PubMed, Hemodial Int) - "In ESA-hyporesponsive hemodialysis patients, roxadustat was associated with higher adjusted odds of sustained Hb ≥ 10 g/dL achievement but did not clearly reduce month-to-month Hb variability over 6 months. These retrospective findings support sustained target achievement as a clinically relevant response metric and warrant prospective validation."

Journal • Hematological Disorders • Renal Disease