Larazotide Acetate

clinical trials

Also known as: AT-1001, INN-202

**Mechanism of Action** Larazotide acetate (AT-1001) is a synthetic octapeptide that functions as a tight junction regulator. It inhibits paracellular permeability by preventing the disassembly of intercellular tight junction complexes, particularly zonula occludens-1 (ZO-1) and occludin. This action counteracts the gliadin-induced increase in intestinal permeability, a hallmark of celiac disease, by blocking the release of zonulin, a modulator of tight junction dynamics. The peptide does not directly target gluten but stabilizes epithelial barrier integrity, reducing the passage of immunogenic gluten fragments into the lamina propria. **Key Research Findings** In phase 2 and 3 clinical trials, larazotide acetate demonstrated a reduction in gluten-induced gastrointestinal symptoms (e.g., bloating, abdominal pain) in celiac patients on a gluten-free diet, though it did not consistently normalize intestinal permeability biomarkers (e.g., lactulose/mannitol ratio). A 2020 phase 3 trial (NCT03569007) showed a modest but significant improvement in symptom scores versus placebo, with a favorable safety profile (mild adverse events, no systemic immunosuppression). Preclinical studies confirm its specificity for tight junction regulation without altering nutrient absorption or immune cell function. **Clinical Relevance** Larazotide acetate is the first-in-class tight junction modulator advanced to late-stage trials for celiac disease, targeting residual symptoms in patients adhering to a gluten-free diet. It is not a cure but an adjunctive therapy to mitigate accidental gluten exposure. Regulatory approval remains pending; further studies are needed to confirm long-term efficacy and mucosal healing. The peptide’s mechanism also holds potential for other permeability-related disorders (e.g., inflammatory bowel disease, irritable bowel syndrome), though clinical data in these indications are limited. For research purposes only — not medical advice.

Key data

Category
Gut & GI
Molecular weight
785.9 g/mol
Molecular formula
C34H59N9O12
CAS number
881851-50-9
Administration
oral
Research status
clinical trials
References
44
Tags
tight-junction, celiac, barrier

Research & studies

Coeliac disease and the intestinal barrier: mechanisms of disruption and strategies for restoration
Gut · 2026 · PubMed

Increased intestinal permeability is central to coeliac disease pathophysiology.; Mechanisms involve zonulin, inflammatory cytokines, microbial alterations, and immune responses to gliadin.; Therapies targeting barrier integrity include diets, supplements, and experimental medications like larazotide acetate and IMU-856.; Reliable biomarkers for evaluating increased permeability are needed, and further research on barrier-normalizing therapies is advocated.

Antibacterial hyaluronic acid hydrogel with sustained release of larazotide as effective colitis treatment
Journal of controlled release : official journal of the Controlled Release Society · 2025 · PubMed
New Therapeutic Challenges in Pediatric Gastroenterology: A Narrative Review
Healthcare (Basel, Switzerland) · 2025 · PubMed
Targeting endothelial tight junctions to predict and protect thoracic aortic aneurysm and dissection
European heart journal · 2023 · PubMed

Endothelial TJ expression was aberrant in thoracic aortas of TAAD patients by single-cell RNA sequencing.; In BAPN-fed mice, TJ function was disrupted early (5-10 days) before TAAD formation at 14 days.; MRI probes detecting TJ dysfunction predicted TAAD: increased signal from 5 to 10 days indicated later TAAD.; AT-1001 (TJ sealer) alleviated TJ impairment and reduced TAAD incidence; ZO-1 knockout increased TAAD.

Larazotide acetate for treatment of celiac disease: A systematic review and meta-analysis of randomized controlled trials
Clinics and research in hepatology and gastroenterology · 2022 · PubMed
Larazotide acetate: a pharmacological peptide approach to tight junction regulation
American journal of physiology. Gastrointestinal and liver physiology · 2021 · PubMed
The Therapeutic use of the Zonulin Inhibitor AT-1001 (Larazotide) for a Variety of Acute and Chronic Inflammatory Diseases
Current medicinal chemistry · 2021 · PubMed
Targeting zonulin and intestinal epithelial barrier function to prevent onset of arthritis
Nature communications · 2020 · PubMed

Zonulin family peptide is highly expressed in autoimmune mice and humans and predicts transition from autoimmunity to inflammatory arthritis.; Increased serum zonulin levels are associated with a leaky intestinal barrier, dysbiosis, and inflammation.; Restoration of the intestinal barrier with butyrate or a cannabinoid type 1 receptor agonist inhibits arthritis development.; Treatment with the zonulin antagonist larazotide acetate reduces arthritis onset by specifically increasing intestinal barrier integrity.

Frequently asked questions

What is Larazotide Acetate?

**Mechanism of Action** Larazotide acetate (AT-1001) is a synthetic octapeptide that functions as a tight junction regulator. It inhibits paracellular permeability by preventing the disassembly of intercellular tight junction complexes, particularly zonula occludens-1 (ZO-1) and occludin. This action counteracts the gl

How does Larazotide Acetate work?

Tight-junction-regulating octapeptide that reduces intestinal permeability ('leaky gut'); trialed as an adjunct for celiac disease.

What is the research status of Larazotide Acetate?

Larazotide Acetate is currently classified as clinical trials, with 44 research references on record. This is for research purposes only and is not medical advice.

What is the molecular weight of Larazotide Acetate?

Larazotide Acetate has a molecular weight of approximately 785.9 g/mol (formula C34H59N9O12).

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