Lexicon Pharmaceuticals announced the completion of a safety review of the initial run-in cohort of the Telotristat Ethyl for Advanced Biliary Tract Cancer, or TELE-ABC, study, which is a Phase 2 clinical study of telotristat ethyl in patients with biliary tract cancer. Telotristat ethyl is a novel, oral tryptophan hydroxylase (TPH) inhibitor approved as XERMELO® for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy alone.

Safety analysis from the first six patients who completed at least a 21-day cycle of treatment with telotristat ethyl in combination with cisplatin and gemcitabine supports the continuation of enrollment with no adjustment in the telotristat ethyl 500 mg three times daily dosing. To date, the first two patients enrolled have completed over six months of treatment.

"We are pleased that the safety review of the first dose cohort supports continuation of the study in patients with biliary tract cancer," said Pablo Lapuerta, MD, executive vice president and chief medical officer of Lexicon. "We believe telotristat ethyl’s mechanism of action has potential utility outside of carcinoid syndrome diarrhea. Inhibiting serotonin may help control tumor growth, and we look forward to obtaining topline data on progression-free survival for the first 20 biliary tract cancer patients next year."

The TELE-ABC study is a multicenter, open-label, two-stage Phase 2 study evaluating the safety and efficacy of telotristat ethyl in combination with standard of care, first-line chemotherapy (cisplatin plus gemcitabine) in approximately 53 patients with unresectable, locally advanced, recurrent or metastatic biliary tract cancer. The primary efficacy endpoint under evaluation is progression-free survival (PFS) rate at six months. Secondary endpoints include overall survival (OS), OS rate at six and 12 months, and PFS rate at 12 months.

Subject to completion of patient enrollment, Lexicon expects to report topline results on 6-month progression-free survival data from the first 20 patients of the TELE-ABC study in 2020.

Discovered using Lexicon’s unique approach to gene science, XERMELO is the first and only approved oral therapy for carcinoid syndrome diarrhea. XERMELO targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within metastatic neuroendocrine tumor (mNET) cells.

XERMELO is approved in the United States, the European Union and certain additional countries for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy. Carcinoid syndrome is a rare condition that occurs in patients living with mNETs and is characterized by frequent and debilitating diarrhea. XERMELO targets the overproduction of serotonin inside mNET cells, providing an additional treatment option for patients suffering from carcinoid syndrome diarrhea.

Lexicon has granted Ipsen an exclusive royalty-bearing right and license to commercialize XERMELO outside of the United States and Japan. Lexicon is commercializing XERMELO in the United States and Ipsen is commercializing XERMELO in multiple countries, including the United Kingdom and Germany.

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