Drug intelligence / Profile preview

telotristat ethyl + cisplatin + gemcitabine

Development stage
Unknown
Lead developer
Lexicon Pharmaceuticals
Modality
Small Molecules
Administration
Oral, Intravenous
01

Overview

This is a **combination therapy** consisting of three drugs: - **Telotristat ethyl** is an orally administered prodrug converted into telotristat etiprate, a potent inhibitor of tryptophan hydroxylase (TPH). By blocking TPH, it interferes with **serotonin biosynthesis**, thereby reducing serotonin levels. Telotristat ethyl is primarily used to treat carcinoid syndrome diarrhea and is being investigated for potential antitumor additive effects in cancers where serotonin overexpression contributes to tumor growth, such as cholangiocarcinoma[1][3][5]. - **Cisplatin** is a platinum-based chemotherapy agent that causes DNA crosslinking and subsequent apoptosis in rapidly dividing tumor cells. - **Gemcitabine** is a nucleoside analog that inhibits DNA synthesis, leading to cell cycle arrest and apoptosis. This triple combination is being studied as a first-line treatment for unresectable, locally advanced, recurrent, or metastatic **biliary tract cancer**, including cholangiocarcinoma and gallbladder cancer. Preclinical and early clinical studies indicate that telotristat ethyl may enhance the antitumor efficacy of standard GemCis therapy, particularly in patients with tumors exhibiting increased serotonin metabolism[1][3].

02

Targets

DNA polymerase familyTPH2 (Tryptophan hydroxylase 2)DNATPH1 (Tryptophan 5-hydroxylase 1)

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