Aphthasol (amlexanox) / Abeona Therap, Esteve - LARVOL DELTA

Sex-specific differences of amlexanox in a mouse model for atherosclerosis and MASLD. (PubMed, Mol Metab) - "Collectively, our findings indicate that therapeutic IKKε inhibition with amlexanox does not prevent progression of advanced atherosclerosis in this model but effectively ameliorates MASLD in male mice. In contrast, female mice experience aggravated hepatic lipid deposition. These results underscore the importance of incorporating sex-specific analyses in metabolic and cardiovascular research and highlight the need to evaluate therapeutic strategies such as amlexanox in both sexes."

Journal • Preclinical • Atherosclerosis • Cardiovascular • Hepatology • Inflammation • Metabolic Disorders • Metabolic Dysfunction-Associated Steatotic Liver Disease

To Evaluate and Compare the Effectiveness of Topical Amlexanox, Curcuma Longa Extract, and Clobetasol Propionate in the Treatment of Oral Lichen Planus: An Original Research. (PubMed, J West Afr Coll Surg) - "Amlexanox and Curcuma longa extract reduce the signs and symptoms of OLP to a tolerable level, with improvement remaining static after the cessation of therapy. Clobetasol propionate causes complete remission of signs and symptoms of OLP, but there is a higher chance of recurrence."

Journal • Dermatology • Dermatopathology • Immunology • Lichen Planus • Pain

Targeting the HMGB1 Pathway With Glycyrrhizic Acid and Amlexanox Attenuates RSV-Induced Injury and Viral Propagation. (PubMed, FASEB J) - "In experiments using lethal murine RSV infection models, treatment with GA and AMX significantly improved survival rates and reduced lung pathology. In conclusion, these findings suggest that HMGB1 is a promising therapeutic target for RSV infection and highlight GA and AMX as potential antiviral candidates that can modulate HMGB1-mediated immunopathology."

Journal • Infectious Disease • Inflammation • Respiratory Diseases • Respiratory Syncytial Virus Infections • HMGB1

Efficacy of Diode Laser Therapy in Managing Recurrent Aphthous Stomatitis and Oral Lichen Planus: A Systematic Review. (PubMed, Photobiomodul Photomed Laser Surg) - "We can conclude that photobiomodulation therapy was better for treating RAS and OLP with greater healing, patient satisfaction, and lower recurrence rate in comparison with other topical drug therapies."

Journal • Dental Disorders • Dermatology • Dermatopathology • Inflammation • Lichen Planus • Pain • Stomatitis

Amlexanox as a multi-target neuroregenerative modulator for traumatic brain injury. (PubMed, Neural Regen Res) - "This review summarizes current evidence suggesting that amlexanox functions as a system-level modulator of traumatic brain injury pathology by simultaneously targeting inflammatory signaling, regenerative transcriptional programs, and lysosomal homeostasis. Such multimodal pharmacology may represent a promising strategy for bridging the gap between acute neuroprotection and long-term neural repair following traumatic brain injury."

Journal • CNS Disorders • Inflammation • Vascular Neurology • TFEB

Pharmacological inhibition of STING-TBK1 signaling is associated with reduced PANoptosis-like features and fibrotic remodeling in keloids. (PubMed, Int Immunopharmacol) - "These findings suggest that STING-TBK1 signaling is associated with PANoptosis-like features and fibrotic remodeling in keloids and may represent a potential therapeutic target for antifibrotic immunomodulation."

Journal • Fibrosis • Immunology • STING • TBK1

A transcriptionally distinct population of human adipocytes with end-of-trajectory signature (hEOS) emerges during obesity to drive maladaptive inflammation. (PubMed, Pharmacol Res) - "Importantly, pharmacological intervention with the IKKε/TBK1 inhibitor amlexanox improved metabolic dysfunction in vivo, and this was accompanied by reduced adipocyte Lama4 expression and suppressed pro-inflammatory macrophage activation, consistent with attenuation of the hEOS-Mac3 module. Together, these findings define hEOS adipocytes as a disease-emergent, pharmacologically responsive population that is implicated in maladaptive adipocyte-macrophage crosstalk and prioritize the LAMA4-integrin-NF-κB axis as a potential therapeutic target."

Journal • Genetic Disorders • Inflammation • Metabolic Disorders • Obesity • HIF1A • ITGB1

A nutrient-responsive AMPK/TBK1 circuit restricts adipocyte catabolism. (PubMed, JCI Insight) - "Combined treatment of mice with amlexanox and the AMPK activator AICAR enhanced weight loss, improved glucose tolerance and insulin sensitivity, and suppressed inflammatory and lipogenic programs in adipose tissue, as well as fibrotic gene expression in the liver. Building on prior clinical observations linking TBK1 inhibition to metabolic health, these findings defined a nutrient-sensitive AMPK/TBK1 feedback loop that limited adipocyte catabolism and suggested that dual targeting of TBK1 and AMPK may help counteract metabolic adaptation and enhance the durability of obesity therapies."

Journal • Endocrine Disorders • Fibrosis • Genetic Disorders • Inflammation • Obesity • AMPK • NRF1

Effect of small molecule inhibitors of IRF3 and TBK1 signaling pathways on ovarian cancer cells (IMMUNOLOGY 2026) - "We are interested in the expression of this signaling pathway in tumor cells, and the possibility of targeting it as a therapeutic approach. In these studies, we treated mouse (ID8 and BPPNM) and human (A2780, SKOV3) OvCa cells with inhibitors of IRF3 (BXT-795, C10 [phenylmethimazole]), TBK1(GSK-8612, MRT-67307); and TBK1 and IKKe (Amlexanox) at different concentrations. These findings suggest that IRF3/TBK1 inhibition may influence metastatic potential and proliferative capacity of ovarian cancer cells and make them more prone to the action of oncolytic viruses."

IO biomarker • Gynecologic Cancers • High Grade Serous Ovarian Cancer • Infectious Disease • Oncology • Ovarian Cancer • Solid Tumor

Does ACE2 deficiency have a role in Parkinson's disease -exacerbated pulmonary fibrosis? (PubMed, Exp Neurol) - "ROC analysis validated hub genes (e.g., BDNF, FOSL2) with good diagnostic value (AUC > 0.7), and molecular docking identified Smilagenin, Fostamatinib, Olopatadine, and Amlexanox as potential therapeutics. This study confirms ACE2 deficiency is a central driver of PD-exacerbated pulmonary fibrosis via the FoxO1/TNF/JAK1-STAT3/AGE-RAGE pathways, providing novel biomarkers and drug candidates to address the clinical need for managing this comorbidity."

Journal • CNS Disorders • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Inflammation • Movement Disorders • Parkinson's Disease • Pneumonia • Pulmonary Disease • Respiratory Diseases • BDNF • FOSL2 • JAK1 • TGFB1

Pharmacological elevation of lactate alleviates sepsis via histone lactylation-induced IL-10 production. (PubMed, Free Radic Biol Med) - "Importantly, in vivo sodium lactate administration improves survival in endotoxemia. Our study clarifies that elevating lactate-mediated histone lactylation plays a protective role in sepsis, and amlexanox is a potential drug for sepsis."

Journal • Infectious Disease • Inflammation • Septic Shock • IL10

Interferon-Type-I Response and Autophagy Independently Regulate Radiation-Induced HLA-Class-I Molecule Expression in Lung Cancer. (PubMed, Curr Issues Mol Biol) - "The effect of RT (8 and 3 × 8 Gy) on Interferon beta (IFNβ), IFN-stimulated genes (ISGs), and HLA-class-I expression in combination with IFN-type-I-response inhibitors (Ruxolitinib, Tofacitinib, Amlexanox) targeting the JAK and TBK1 was studied with Flow cytometry and RT-PCR. The current study supports the theory that baseline autophagy, RT-induced autophagy blockage, and IFN-type-I response enhancement define the HLA-class-I levels in NSCLC cells. This complex interplay emerges as a promising target for the development of radio-vaccination strategies to enhance the efficacy of radio-immunotherapy."

IO biomarker • Journal • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • ATG7 • IFNB1 • MAP1LC3A

Potential Cardioprotective Effect of a GRK5 Inhibitor Against NF-κB-Mediated Inflammation in an Animal Model of Isoproterenol-Induced Myocardial Infarction. (PubMed, Int J Mol Sci) - "Thus, this study aims to investigate the effects of Amlexanox (AMX), a potential GRK5 inhibitor, in an animal model of MI by assessing its impact on GRK5-mediated NF-κB/inflammatory processes...Treatment with AMX led to a significant decrease in NF-κB(p65) and (p105) expression (p < 0.01 and p < 0.001, respectively), and GRK5 and MEF2α protein levels were also upregulated. In conclusion, AMX shows potential cardioprotective effects by modulating the GRK5/MEF2-mediated NF-κB inflammatory signaling pathway."

Journal • Preclinical • Cardiovascular • Congestive Heart Failure • Heart Failure • Inflammation • Myocardial Infarction • IL6 • RELA

Next generation sting agonist in combination with DNA methyltransferase inhibitors induce an apoptotic cell death mechanism driven by apoptosis in TP53-mutated Acute Myeloid Leukemia (ASH 2025) - "STING activation was also recently shown to trigger p53-independent apoptosis, andSTING agonists have shown a synergistic effect with BH3-mimetics in TP53-mutated AML cells.Here we studied the next-generation allosteric STING agonist C92 from Curadev Pharma, whichpotently binds and activates all human STING variants, in combination with the DNMTidecitabine (DAC) in TP53-mutated AML.MethodsTo test the hypothesis that DNMTis synergize with STING agonists, we treated AML cell lineswith wild-type (WT) (MOLM-14, OCI AML1) and mutated TP53 (KG1, KASUMI, U937) andpatient samples (N=5-10) with C92 in combination with decitabine (DAC)...To investigate apoptosis in TP53 KO vs WT, we measured Annexin Vlabeling by flow cytometry pre and post treatment with the 2 drugs, administered alone and incombination in the presence of the pan-caspase apoptosis inhibitor Z-VAD-FMK, the JAK/STATinhibitor ruxilitinib and the TBK1 inhibitor amlexanox...Finally, C92 and DAC combination decreased..."

Combination therapy • Epigenetic controller • IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • CXCL10 • CXCL11 • IFI27 • IFIH1 • IFIT2 • MX1 • OASL • STING • TNFA • TNFSF10

Repurposing amlexanox as a topical anti-inflammatory and antioxidant intervention for diabetic foot ulcers: insights from in-silico and in-vivo studies. (PubMed, Inflammopharmacology) - "Taken together, the present work highlights the mechanisms associated with ALX mediated wound healing in diabetic rats thereby demonstrating its potential as a promising strategy for DFU management."

Journal • Preclinical • Diabetes • Inflammation • Metabolic Disorders • IL1B • IL6 • MMP9 • TNFA

Therapeutic potential of IκB kinase epsilon inhibition in preventing meniscal degeneration of early osteoarthritis. (PubMed, Bone Joint Res) - "Furthermore, IKKε regulates meniscal degeneration through NF-κB signalling-mediated catabolism. Two IKKε/TBK1 inhibitors, amlexanox and BAY-985, are potential targets for the treatment of meniscal degeneration prior to OA."

Journal • Immunology • Osteoarthritis • Pain • Rheumatology • NFKBIA • RELA

Amlexanox Alleviates Renal Inflammation and Fibrosis by Inhibiting cGAS/STING/TBK1 and TGF-β1/Smad Signaling. (PubMed, Eur J Pharmacol) - "AMX exerts renoprotective effects by targeting both inflammation and fibrosis through the inhibition of the cGAS/STING/TBK1 and TGF-β1/Smad signaling pathway."

Journal • Fibrosis • Immunology • Inflammation • Renal Disease • CGAS • FN1 • STING • TGFB1

Combined treatment with amlexanox and cytarabine induces apoptosis via the S100A6-Akt pathway in KMT2A::AFF1-positive acute lymphoblastic leukemia. (PubMed, Leukemia) - No abstract available

Journal • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • AFF1 • KMT2A • S100A6

Targeting the cGAS-STING Pathway to Modulate Immune Inflammation in Diabetes and Cardiovascular Complications: Mechanisms and Therapeutic Insights. (PubMed, Curr Issues Mol Biol) - "Pharmacological inhibitors, including RU.521 (cGAS antagonist), C-176/H-151 (STING palmitoylation blockers), and the TBK1 inhibitor amlexanox, effectively lower pro-inflammatory cytokines (IL-1β, IL-6, TNF-α) and improve left ventricular ejection fraction in diabetic cardiomyopathy and ischemia-reperfusion injury models. Novel PROTAC degraders targeting cGAS/STING and natural products such as Astragaloside IV and Tanshinone IIA further support the pathway's druggability. Collectively, these findings position the cGAS-STING axis as a central molecular nexus linking metabolic derangement to cardiovascular pathology in T2DM and underscore its inhibition or targeted degradation as a promising dual cardiometabolic therapeutic strategy."

Journal • Review • Atherosclerosis • Cardiomyopathy • Cardiovascular • Diabetes • Inflammation • Metabolic Disorders • Reperfusion Injury • Targeted Protein Degradation • Type 2 Diabetes Mellitus • CGAS • IL1B • IL6 • NLRP3 • STING • TNFA

Amlexanox Ameliorates Traumatic Brain Injury by Restoring Autophagy-Lysosomal Function via cAMP Signaling Modulation. (PubMed, Int J Biol Sci) - "Behavioral assessments confirmed significant improvements in cognitive and neurological deficits following TBI. These findings establish that AMX is a promising therapeutic agent that restores lysosomal function and mitigates TBI-induced neuronal damage through multi-target PDE inhibition and anti-inflammatory actions."

Journal • CNS Disorders • Inflammation • Vascular Neurology

Combined amlexanox and anti-MCP-1 therapy suppresses tumor progression in a murine Lewis lung carcinoma model. (PubMed, Anticancer Drugs) - "Flow cytometric analysis indicated a significant decrease in M2 macrophages (F4/80+CD206+) in the intervention group, with no substantial change observed in the proportion of M1 macrophages (F4/80+CD86+). Combined administration of amlexanox and anti-MCP-1 mAb inhibited tumor cell proliferation, promoted apoptosis, and reduced infiltration of tumor-associated M2 macrophages, thereby contributing to suppression of tumor progression in the LLC murine model."

IO biomarker • Journal • Preclinical • Lung Cancer • Oncology • Solid Tumor • ARG1 • BCL2 • BCL2L1 • CCL2 • CD86 • MCL1 • MRC1 • TBK1

Development of translational read-through-inducing drugs as novel therapeutic options for patients with Fanconi anemia. (PubMed, Cell Death Discov) - "Amlexanox, an anti-inflammatory drug, also promotes read-through of premature stop codons caused by nonsense mutations. This study represents a milestone of drug development for FA as it paves the way for clinical development of TRIDs, indicating ataluren as a promising approach to address the genetic instability and reduce the risk of malignant transformation in FA cells. Moreover, these results highlight the importance of a reliable experimental pipeline to assess whether minimal protein rescue via translational read-through can yield meaningful phenotypic rescue."

Journal • Anemia • Hematological Disorders • Pediatrics • FANCA • FANCC • FANCF • LMNB1 • STAT2

Unlocking therapeutic potential of amlexanox in MASH with insights into bile acid metabolism and microbiome. (PubMed, NPJ Gut Liver) - "These findings uncover the multifaceted therapeutic potential of amlexanox in treating MASH and atherosclerosis by targeting bile acid metabolism, gut microbiota, hepatic inflammation, and fibrosis. Our study highlights amlexanox as a promising candidate for clinical applications."

Journal • Atherosclerosis • Cardiovascular • Diabetes • Dyslipidemia • Fibrosis • Genetic Disorders • Hepatocellular Cancer • Hepatology • Immunology • Liver Failure • Metabolic Disorders • Metabolic Dysfunction-Associated Steatohepatitis • Obesity • Oncology • Solid Tumor

Matrix stiffness-induced IKBKE and MAPK8 signaling drives a phenotypic switch from DCIS to invasive breast cancer. (PubMed, Cell Commun Signal) - "The IKBKE-inhibitor Amlexanox, clinically utilized for aphthous ulcers, as well as the MAPK8 inhibitor JNK-IN-8, reinstalled the DCIS-like phenotype of breast cancer cells on high matrix stiffness. This suggests that IKBKE and/or MAPK8 inhibitors could enhance the arsenal of treatments to prevent or treat breast cancer."

Journal • Breast Cancer • Oncology • Solid Tumor • IKBKE • MAPK8

Amlexanox inhibits production of type I interferon and suppresses B cell differentiation in vitro: a possible therapeutic option for systemic lupus erythematosus and other systemic inflammatory diseases. (PubMed, RMD Open) - "Our findings demonstrate inhibitory effects of amlexanox on type I IFN production and B cell differentiation in primary human cells. Inhibition of TBK1 could potentially be a therapeutic option for the treatment of type I IFN-driven systemic inflammatory diseases."

Journal • Preclinical • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Scleroderma • Sjogren's Syndrome • Systemic Lupus Erythematosus • Systemic Sclerosis • CD40LG • IL21 • MX1 • TBK1