Telotristat ethyl, or LX1032, is currently in development for the treatment of carcinoid syndrome. We have filed a New Drug Application for telotristat ethyl and have been granted priority review by the FDA with a Prescription Drug User Fee Act (“PDUFA”) target action date of February 28, 2017.

If approved, telotristat ethyl will be the first and only oral therapy approved for the treatment of carcinoid syndrome that targets tryptophan hydroxylase (TPH), an enzyme that triggers the excess serotonin production within metastatic neuroendocrine tumor (mNET) cells that leads to carcinoid syndrome, without affecting the beneficial production of serotonin in the brain.

While existing treatments for carcinoid syndrome work to reduce the release of serotonin outside tumor cells, telotristat ethyl works at the source to reduce serotonin production within the tumor cells. By specifically inhibiting serotonin production, telotristat ethyl seeks to control this important driver of carcinoid syndrome and, in turn, provide patients with more control over their disease.

Telotristat ethyl was specifically designed to achieve enhanced systemic exposure to address disorders such as carcinoid syndrome that require regulation of serotonin levels beyond the EC cells in the gastrointestinal tract without impacting brain serotonin production.

Telotristat ethyl has received Orphan Drug status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA and EMA Orphan Drug programs are intended to promote the development of drugs which prevent or treat life-threatening or chronically debilitating conditions that affect limited populations.

In addition, telotristat ethyl has received Fast Track designation from the FDA. The FDA’s Fast Track program facilitates the development of potential new drugs and expedites the review of new drugs intended to serve unmet medical needs in serious or life-threatening conditions.

Lexicon retains rights to market telotristat ethyl in the U.S. and Japan, and is building the in-house commercial infrastructure to serve the U.S. market. Lexicon has a license and collaboration agreement with Ipsen to commercialize telotristat ethyl in Europe and other countries outside the U.S. and Japan.

Telotristat ethyl reduces peripheral serotonin production by inhibiting the enzyme TPH in metastatic carcinoid tumors.