AT-1001, Larazotide Acetate
Evidence Grade C — Moderate human evidence. 51 published studies, 34 human. 29 registered clinical trials.
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.
51 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.
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