Safety and Tolerability Study of Larazotide Acetate in Celiac Disease Subjects

CLIN1001-004 was a randomized, double-blind, placebo controlled, dose-ranging, 7-arm,
multicenter study with a gluten challenge. The objects were multiple dose safety and
tolerance; efficacy (intestinal permeability [change in urinary LAMA ratio] and disease signs
and symptoms) following gluten challenge.

Following a 21-day screening period, subjects were randomized to one of seven treatments
groups: four groups received larazotide acetate (0.25 mg, 1 mg, 4 mg or 8 mg TID) along with
an 800 mg gluten challenge, one group received placebo with an 800 mg gluten challenge, a
safety control arm received the highest dose of larazotide acetate (8 mg TID) and gluten
placebo and the last group received drug placebo and gluten placebo. The gluten challenge was
administered as capsules (800 mg TID) with each main meal for a total of 2.4 g daily. Drug or
drug placebo was administered TID 15 minutes prior to each main meal. Subjects received their
assigned treatments for two weeks (Day 0 through Day 14) and came to clinic for a follow-up
visit one week later (Day 21). Subjects remained on their gluten-free diet for the duration
of the study.

Inclusion Criteria:

- Must have been diagnosed with celiac disease by biopsy for ≥ 6 months.

- Have a Anti-Tissue Transglutaminase (tTG) ≤ 10 EU as measured by serology.

- Must be on a gluten-free diet for at least the past 6 months.

Exclusion Criteria:

- Have any chronic active GI disease other than celiac disease (e.g., IBS, Crohn's,
Colitis).

- Have diabetes (Type 1 or Type 2).

- Chronically consumes non-steroidal anti-inflammatory agents ("NSAIDs") or takes
proton-pump inhibitors.

- Consuming oral corticosteroids or immune suppressants.