Evidence Grade C — Moderate human evidence. 52 published studies, 34 human. 29 registered clinical trials.
Medically reviewed by a licensed medical professional
Larazotide is a synthetic peptide designed to tighten the seals between intestinal lining cells, reducing the leakiness that allows gluten fragments to trigger immune damage in coeliac disease. It was one of the most clinically advanced coeliac disease drug candidates, reaching Phase III trials, but the development programme has stalled after the sponsoring company went bankrupt.
Larazotide is also known by these brand and alternate names:
52 published studies: 34 human, 6 animal, 8 in-vitro, 24 reviews
Larazotide has no marketing authorisation but is one of the most clinically advanced coeliac disease drug candidates. A Phase IIb trial (342 patients) was the first coeliac disease therapeutic trial to meet a primary symptom endpoint. Phase III trials (GRASSROOTS programme) have been conducted.
Larazotide represents a novel approach — rather than suppressing the immune response, it aims to prevent the triggering event (gluten crossing the intestinal barrier). It is intended as an adjunct to a gluten-free diet, not a replacement. Clinical development is ongoing.
Research suggests larazotide works locally in the gut lumen (it is not absorbed into the bloodstream) by blocking the receptors that trigger tight junction opening in response to gluten exposure. By preventing this increased intestinal permeability, it is proposed to reduce the amount of gluten fragments that cross the intestinal barrier and trigger the immune response in coeliac disease.
Research suggests a Phase IIb trial (342 patients) was the first coeliac disease drug trial to meet a primary symptom endpoint, and the safety profile was excellent across over 828 subjects. Larazotide acts locally in the gut without being absorbed into the bloodstream, which limits systemic side effects. However, the Phase III results were disappointing. An unexplained inverted dose-response (only the lowest dose worked; higher doses were ineffective) was never resolved. The permeability biomarker endpoint was never met in outpatient settings. With the sponsor bankrupt and intellectual property being liquidated, no entity is actively developing larazotide as of early 2026.
PeptideTrace tracks 29 registered clinical trials for Larazotide sourced from ClinicalTrials.gov.
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
AT1001 for the Treatment of Long COVID
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
AT1001 for the Treatment of COVID-19 Related MIS-C
Larazotide (AT-1001) is a synthetic octapeptide derived from the zonula occludens toxin (ZOT) of Vibrio cholerae, which shares a sequence with zonulin, an endogenous tight junction modulator. Sequence: GGVLVQPG (Gly-Gly-Val-Leu-Val-Gln-Pro-Gly). MW (free base): 725.83 Da. CAS (free base): 258818-34-7. CAS (acetate salt): 881851-50-9. Acts locally in the gut with no systemic absorption (plasma levels remain below the limit of quantification at <0.05 ng/mL even at 36 mg). Formulated as oral enteric-coated capsules, TID before meals. Received FDA Fast Track designation for celiac disease. Developed by Alba Therapeutics, then Innovate Biopharmaceuticals, then 9 Meters Biopharma (which filed for Chapter 7 bankruptcy in July 2023). Development program is now defunct.
Research suggests larazotide blocks zonulin receptors (EGFR and PAR2), preventing the signaling cascade triggered when gliadin fragments bind CXCR3 on intestinal epithelium. This cascade normally proceeds through MYD88 to zonulin release to EGFR/PAR2 activation to actin rearrangement to tight junction disassembly to increased paracellular permeability. By blocking this pathway, larazotide research suggests prevents gliadin peptide transport from the intestinal lumen to the lamina propria, where they would otherwise trigger immune responses via tissue transglutaminase and Th1 pathways.
Phase 2b (Leffler et al., Gastroenterology 2015; N=342): The first celiac disease therapeutic trial to meet a primary symptom endpoint. The 0.5 mg TID dose achieved significance on the CeD-GSRS primary endpoint (P=0.022 ANCOVA; P=0.005 MMRM), with a 26% decrease in symptomatic days (P=0.017) and 31% increase in improved symptom days (P=0.034). An earlier Phase 2b (Kelly et al., 2013; N=184) showed larazotide 1 mg significantly limited gluten-induced symptoms (P=0.002) and reduced anti-tTG IgA levels (P=0.005-0.025). The LAMA permeability ratio primary endpoint was not met in either trial. Phase 3 (CedLara): Planned N=525 across three arms. Launched June 2020 at >100 US/Canadian sites. In June 2022, a pre-specified interim analysis determined trial discontinuation. In July 2023, 9 Meters Biopharma filed for Chapter 7 bankruptcy. Development is now defunct.
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