Study of Efficacy, Safety, and Tolerability of LYS006, in Patients With Mild to Moderate Ulcerative Colitis

A Randomized, Multi-center, Subject and Investigator-blinded, Placebo-controlled, Parallel-group Study to Assess the Efficacy Safety and Tolerability of LYS006 in Patients With Mild to Moderate Ulcerative Colitis

The purpose of the study was to assess preliminary efficacy, safety, and tolerability of LYS006 in adult patients with mild to moderate ulcerative colitis and to determine if LYS006 has an adequate clinical profile for further development in this indication.

This was a randomized, placebo-controlled, subject and investigator blinded, multicenter, non-confirmatory, parallel group, proof of concept study in patients with mild to moderate ulcerative colitis. This study consisted of a screening period of up to 4 weeks, and a 8 week treatment period followed by a 30 day post treatment period safety follow up. The maximum study duration for each participant including the 4 week screening period was 16 weeks.

At the beginning of the treatment period, subjects were randomized to one of the two following treatment groups in 2:1 ratio

• LYS006
• matching placebo

Key Inclusion Criteria:

• Male and female subjects 18-75 years of age with an established diagnosis of ulcerative colitis at least 3 months prior to screening are eligible for the study.
• Patients must have active disease with a full Mayo Score between 5 and 10 (inclusive) with an endoscopy score of 2 or 3; rectal bleeding and stool frequency scores 1 to 3 and a physician's global assessment of 1 or 2.
• Patients must have responded inadequately to conventional therapy with oral 5-ASA prior to screening.

Key Exclusion Criteria:

• Has previous diagnosis with Crohn's disease, indeterminate colitis, microscopic colitis or acute diverticulitis based on medical history.
• History of toxic megacolon, abdominal abscess, symptomatic colonic stricture, or stoma; history or is at risk of colectomy.
• Treatment with biologics within 3 months or 5 half-lives (whichever is longer) prior to screening endoscopy, non-biologics advanced therapies within 4 weeks prior to screening endoscopy, systemic immunosuppressant or immunomodulator within 6 week, topical treatment with 5-ASA or steroids within 2 weeks prior to screening endoscopy