Larazotide

Gut Health / Tight Junction Regulatorinvestigational (Phase III)

Also known as: Larazotide acetate, AT-1001, INN-202

A synthetic octapeptide that regulates intestinal tight junctions, developed as the first drug to target intestinal permeability ('leaky gut') in celiac disease and other conditions.

Overview

Larazotide acetate is a synthetic 8-amino acid peptide derived from Vibrio cholerae zonula occludens toxin (Zot) that acts as a tight junction regulator. It was developed by Innovate Biopharmaceuticals (now 9 Meters Biopharma) as a first-in-class oral therapy targeting intestinal permeability for celiac disease. Unlike a gluten-free diet, which avoids the trigger, larazotide addresses the downstream pathological mechanism โ€” the opening of intestinal tight junctions that allows gluten peptides and other antigens to cross the epithelial barrier and trigger immune responses. In Phase II and III clinical trials, larazotide reduced celiac disease symptoms in patients already on a gluten-free diet who continued to have residual symptoms. It acts locally in the gut lumen with minimal systemic absorption, giving it an excellent safety profile. Beyond celiac disease, larazotide has potential applications for other conditions associated with increased intestinal permeability, including inflammatory bowel disease, IBS, type 1 diabetes, and environmental enteropathy.

Mechanism of Action

Larazotide acts on intestinal tight junctions: (1) Blocks the zonulin receptor pathway that triggers tight junction disassembly; (2) Prevents gliadin-induced (and other antigen-induced) opening of paracellular pathways; (3) Maintains the integrity of the zonula occludens protein complex (ZO-1, occludin, claudins); (4) Reduces intestinal permeability to macromolecules including gluten fragments; (5) Acts locally in the gut lumen โ€” minimal systemic absorption; (6) Reduces downstream immune activation by preventing antigen translocation across the epithelium; (7) Does not digest gluten or prevent its initial contact with the mucosa.

Molecular Formula

C40H63N9O11

Molecular Weight

834.00 g/mol

Sequence

Gly-Gly-Val-Leu-Val-Gln-Pro-Gly

Dosage Protocols

Dose Range

0.25mg โ€“ 1.0mg

Frequency

Three times daily (before meals)

Route

oral

Cycle Length

Ongoing

Taken 15 minutes before each meal. The 0.5mg TID dose showed optimal efficacy in Phase IIb trials. Acts locally in the gut โ€” not systemically absorbed. Must be used alongside a gluten-free diet, not as a replacement.

Source: Phase II/III clinical trial protocols

Side Effects

EffectSeverity
Headachemild
GI symptomsmild
Upper respiratory infectionmild

Pros & Cons

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First-in-class mechanism targeting intestinal permeability directly โ€” addresses root pathophysiology of leaky gut

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Oral administration with local gut action and minimal systemic absorption โ€” excellent safety profile

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Human clinical trial data (Phase II/III) demonstrating efficacy in reducing celiac symptoms

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Potential broad applicability to multiple conditions involving increased intestinal permeability

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Not yet FDA-approved โ€” still in clinical development pipeline

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Adjunctive therapy only โ€” does not replace gluten-free diet for celiac patients

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Very limited availability as a research compound

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Phase III results were mixed โ€” primary endpoints not consistently met across trials

Research Studies

Legal Status

Not FDA-approved. Phase III clinical trials completed for celiac disease (as of 2024). Not yet available by prescription. Available as a research compound from some suppliers.

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