Lexicon Pilot Study of Telotristat Etiprate In Ulcerative Colitis Achieves Primary Objective Demonstrating Safety, Tolerabilty

Lexicon Pharmaceuticals

today announced top-line results from a pilot study exploring the use of telotristat etiprate in ulcerative colitis. Telotristat etiprate, a serotonin synthesis inhibitor, is currently in Phase 3 development for carcinoid syndrome, a condition caused by metastatic, functioning neuroendocrine tumors. Elevated serotonin is a hallmark of carcinoid syndrome, and two previous Phase 2 clinical trials have shown that clinical benefits of telotristat etiprate treatment were associated with significant reductions in 5-HIAA, a biomarker of serotonin production.  Genetic and pharmacological studies in preclinical models had also suggested that reducing serotonin levels might benefit patients with other forms of gastrointestinal disease such as ulcerative colitis. In this pilot study of mild to moderate ulcerative colitis, telotristat etiprate achieved the primary objective of demonstrating safety and tolerability. Adverse events and discontinuations were evenly distributed between treatment and placebo and there were no serious adverse events attributed to the investigational drug. In addition, telotristat etiprate showed dose-dependent, statistically significant reductions in 5-HIAA from baseline compared to placebo (p < 0.001), with a relationship between the biomarker and certain measures of clinical benefit being observed in the high dose group. Although these results