LX 1032 (telotristat etiprate) filed with FDA for treatment of carcinoid syndrome- Lexicon Pharmaceuticals

Lexicon Pharmaceuticals announced that it has submitted a New Drug Application to the FDA seeking approval for the marketing and sale of LX 1032 (telotristat etiprate) an oral drug for the treatment of carcinoid syndrome. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing. Lexicon has requested a Priority Review by the FDA as part of the NDA filing.

The NDA filing is supported by the results from TELESTAR, a pivotal Phase III clinical trial of telotristat etiprate in patients with carcinoid syndrome, and TELECAST, a Phase III companion study to TELESTAR. Results from TELESTAR demonstrated a statistically significant reduction from baseline compared to placebo in the average number of daily bowel movements over the 12-week study period, meeting the study's primary endpoint. Top-line results from TELECAST demonstrated a statistically significant reduction in urinary 5-hydroxyindoleacetic acid (5-HIAA, the main metabolite of serotonin) at week 12 as compared to placebo, meeting that study's primary endpoint. The proportions of patients with treatment-emergent adverse events, serious adverse events and discontinuation due to adverse events were generally similar between the telotristat etiprate and placebo arms in both studies.

Comment: telotristat etiprate is the first investigational drug in clinical studies to target tryptophan hydroxylase, an enzyme that triggers the excess serotonin production within mNET cells that leads to carcinoid syndrome. While existing treatments for carcinoid syndrome work to reduce the release of serotonin outside tumor cells, telotristat etiprate works at the source to reduce serotonin production within the tumor cells.