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Metabolic Modulators

Amlexanox

Old canker-sore and asthma drug that blocks inflammatory kinases in fat and liver; small human trials suggest benefit in some diabetics.

Approved · other useEvidence level 3 of 5: Limited human dataAlso: Aphthasol, AA-673, Solfa

Overview

Amlexanox is an anti-inflammatory and anti-allergic drug first approved as a paste for canker sores and, in Japan, for asthma and allergic rhinitis. Researchers later found that it inhibits two kinases, TBK1 and IKKε, that are switched on in fat and liver tissue during obesity and that dampen energy expenditure. In obese mice, blocking them caused weight loss and improved insulin sensitivity. In a small human trial in obese people with type 2 diabetes and fatty liver, about a third responded with better blood sugar and less liver fat, often those with more baseline inflammation. It is a repurposing candidate, not an established metabolic treatment.

Classification
Small-molecule TBK1/IKKε inhibitor (benzopyrano-pyridine); not a peptide
Route
Oral (off-label); topical oral paste (approved form)
Half-life
~3.5 hours (oral)

Mechanism of action

Primary targetTBK1 and IKKε kinases
  1. Inhibits TBK1/IKKεBlocks two non-canonical inflammatory kinases induced by obesity.
  2. Restores cAMP signalingRelieves kinase-driven suppression of adrenergic and cAMP pathways in fat.
  3. Increases energy expenditureAdipose tissue increases thermogenesis and fat oxidation.
  4. Reduces tissue inflammationLower inflammatory signaling in adipose tissue and liver.
  5. Improves insulin actionBetter insulin sensitivity and reduced hepatic glucose output.
Downstream effects
  • Lower HbA1c (responders)
  • Reduced liver fat
  • Modest weight loss

Researched for

  • Type 2 diabetes
  • Obesity
  • Nonalcoholic fatty liver disease (MASLD)
  • Aphthous ulcers (approved topical)
  • Asthma and allergic rhinitis (Japan)

Research notes

  • Mouse studies (Reilly 2013, Nat Med) showed reversal of obesity, insulin resistance and fatty liver.
  • Human RCT (Oral 2017, Cell Metab): responders had reduced HbA1c and liver fat; responders had higher baseline adipose inflammation.
  • Only about 30–40% of patients appear to respond; predictors are not validated.
  • Larger, longer phase 2/3 metabolic trials have not been reported.

Reported side effects

  • Mild nausea or dyspepsia
  • Diarrhea
  • Transient liver-enzyme elevation
  • Rash (rare)

Contraindications & cautions

  • Hypersensitivity to amlexanox
  • Significant liver disease
  • Pregnancy or breastfeeding

Lab monitoring

  • HbA1c, fasting glucose, fasting insulin
  • AST and ALT
  • Lipid panel
  • hsCRP
  • Weight and waist circumference

Dosing and pricing for Amlexanox

Dosing references and compound pricing are available to established patients in the secure portal after a medical evaluation.

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