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Explore  /  Larazotide (AT-1001, Larazotide Acetate)
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Larazotide (AT-1001, Larazotide Acetate)

D
lead outcome
Celiac disease (the lead, best-studied use)
grades vary by outcome ↓
Peptide
also called — Larazotide · larazotide acetate · AT-1001 · INN-202
intestinal barrier / tight-junction regulationceliac disease (adjunct, investigational)"leaky gut" (the concept it actually targets)gut health

The "leaky gut" drug that got furthest — and still failed Phase 3. Larazotide is unusual in this register: a first-in-class, FDA Fast-Tracked, Phase 3-stage pharmaceutical — the most clinically advanced non-dietary celiac-disease drug ever — not a typical gray-market peptide. Three threads: (1) its

In brief

Larazotide (AT-1001) is a synthetic octapeptide and a first-in-class "tight-junction regulator" — the first drug ever aimed at intestinal permeability, the "leaky gut" concept. Taken orally and acting only inside the gut, it works as a zonulin antagonist: in celiac disease, gluten makes the junctions between intestinal cells spring open (via zonulin), letting gliadin through to trigger the immune attack — and larazotide is designed to hold those junctions shut. It's genuinely notable for how far it got: developed across three companies (Alba → Innovate → 9 Meters), it became the most clinically advanced non-dietary celiac drug, the only Phase 3-stage one, with FDA Fast Track and positive Phase 2b data. But in June 2022 its pivotal Phase 3 (CeDLara) was stopped for futility — an interim analysis showed it couldn't feasibly beat placebo — a major blow to the whole zonulin/"leaky gut" drug hypothesis. As of 2026 it's approved for nothing. The honest, and broadly important, takeaway: the most rigorously developed "leaky gut" drug in history failed its decisive trial — which should temper the far larger, far less tested wellness-market hype around "leaky gut" supplements. A real drug, a real mechanism, a real (negative) answer.

Legal standing, by region
United States · your region
Not approved

not approved for any indication (the Phase 3 celiac program failed in 2022). It had FDA Fast Track during development, but never reached approval. Investigational.

Evidence, by outcome

An honest grade per outcome — drawn from the evidence, not any catalogue. Hype and undemonstrated marketing claims grade low.

OutcomeEvidence base · effectGrade
Celiac disease (the lead, best-studied use)
The defining fact: reached Phase 3 and failed — couldn't feasibly beat placebo. Not "untested" — tested and negative at the pivotal stage
Positive Phase 2b (342 pts) → but Phase 3 CeDLara discontinued for futility (2022)
D
Tight-junction / permeability mechanism
The biology of tight-junction regulation is real and elegant; it didn't translate to a positive clinical endpoint
Well-characterized: zonulin antagonism; MLCK inhibition; rodent + porcine barrier-repair models
C
"Leaky gut" / general gut health (the gray-market pitch)
The broad wellness claim is not supported; even the targeted celiac trial failed
No approved indication; concept-level only
F
Adjacent indications (MIS-C, intestinal ischemia)
Exploratory; nothing established
Early/preclinical (porcine ischemia repair, pediatric research)
F
Safety
Generally well-tolerated in trials (a genuine plus — its problem was efficacy, not safety); long-term chronic-modulation effects unestablished
Extensive trial exposure; gut-restricted (minimal systemic absorption)
B

Identity a synthetic single-chain octapeptide (8 amino acids) that is a first-in-class intestinal tight-junction regulator — the first drug ever to target intestinal permeability ("leaky gut") as a therapeutic mechanism. It's orally administered and "gut-restricted" (acts locally in the intestinal lumen, essentially not absorbed systemically), developed as an adjunct to the gluten-free diet for celiac disease. Structurally related to cholera's Zot toxin (which opens junctions); larazotide is designed to help close them. ## Mechanism (as proposed) the intestinal epithelium is sealed by tight junctions — protein complexes (claudins, occludin, ZO-1) that control what passes between cells (the paracellular route). In celiac disease, gliadin triggers zonulin release, which signals the tight junctions to disassemble, increasing permeability and letting gliadin reach the immune cells beneath — driving inflammation. Larazotide is a zonulin receptor antagonist that blocks this opening, promoting redistribution/reassembly of tight-junction proteins and actin filaments (partly via myosin-light-chain-kinase inhibition, which relaxes the actin tension that pulls junctions apart) — thereby restoring/maintaining barrier integrity. Being gut-restricted, it does this locally with minimal systemic exposure (a safety advantage). The mechanism is coherent and animal-validated — and yet the Phase 3 clinical endpoint wasn't met, the register's recurring reminder that a beautiful mechanism and even positive Phase 2 data don't guarantee pivotal success.

Sources — 4 cited
01Slifer ZM, Krishnan BR, Madan J, Blikslager AT. Larazotide acetate: a pharmacological peptide approach to tight junction regulation. Am J Physiol Gastrointest Liver Physiol. 2021 (mechanism; zonulin/MLCK).
02Phase 2b (Alba Therapeutics, 2014; 342 patients) — reduced symptoms; non-monotonic dose response.
03CeDLara Phase 3 (CeD-LA-3001, 525 patients): discontinued for futility, June 2022 (9 Meters Biopharma press release; BioSpace, BeyondCeliac coverage) — interim analysis, couldn't feasibly beat placebo.
04Fasano A, et al. Zonulin, a newly discovered modulator of intestinal permeability, and its expression in coeliac disease. Lancet. 2000 (the zonulin concept). Porcine ischemia tight-junction repair (2021). (No FDA approval as of 2026; celiac program halted.)
Review status
Not yet reviewed

A credentialed reviewer (PharmD / PhD / MD) will be named before this entry is finalised. Until then, treat it as a working draft. Last updated July 2026 (status: celiac Phase 3 failed 2022, no approved indication; watch only for revived development in adjacent barrier-disease indications).

Grades reflect the published evidence, not our interest. No dosing, reconstitution, or administration is published for research compounds — that restraint is deliberate.

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Larazotide (AT-1001) — the tight-junction "leaky gut" peptide that reached Phase 3 and failed: the honest story · Vallydia