Telotristat Etiprate Shows Promise in Treating Carcinoid Syndrome

Carcinoid syndrome frequently prevents patients from leading active lives.

Lexicon Pharmaceuticals, Inc recently released data that showed that telotristat etiprate had a clinical benefit in treating carcinoid syndrome in cancer patients not adequately controlled by long-acting somatostatin analog (SSA) therapy.

The new drug showed efficacy in reducing the amount of bowel movement frequency in patients with carcinoid syndrome whose condition was not being controlled by SSA therapy.

Carcinoid syndrome is characterized by frequent and debilitating diarrhea that often prevents patients from leading active, predictable lives, as well as by facial flushing, abdominal pain, heart valve damage, and other serious consequences.

“We are pleased with the efficacy and safety results of telotristat etiprate and also with the durability of the response shown in this study,” said Lexicon Executive Vice President and Chief Medical Officer Pablo Lapuerta, MD. “The data also support that the compound is acting directly on the cause of carcinoid syndrome, by reducing serotonin production within tumor cells.”

The double-blind phase 3 study evaluated 135 patients with carcinoid syndrome not adequately controlled by SSA therapy. Telotristat etiprate use was compared against a placebo group as patients continued their SSA therapy over the 12-week study period.

Data show that patients who added telotristat etiprate to SSA therapy at both the 250 mg and 500 mg doses experienced a statistically significant reduction from baseline compared with placebo in the average number of daily bowel movements over the 12-week study period, meeting the study’s primary endpoint.

“Telotristat etiprate represents a novel approach by specifically inhibiting serotonin synthesis and, as such, is a promising potential new treatment for patients whose lives can be significantly impacted by this debilitating condition,” said TELESTAR primary investigator Matthew H. Kulke, MD, director, Program in Neuroendocrine and Carcinoid Tumors and Senior Physician, Dana Farber Cancer Insitute, and Associate Professor of Medicine, Harvard Medical School. “These results are exciting from both an efficacy and safety perspective for carcinoid syndrome patients.”

Treatment with telotristat etiprate was generally well-tolerated with patients discontinuing treatment due to these effects at a rate of 7% in the telotristat etiprate arm as compared to 13% in the placebo arm. Patients mostly experienced nausea and depression in response to treatment with the investigational medication, but there were no discontinuations of medication due to these effects.

“Carcinoid syndrome has a significant impact on the lives of patients who already have been battling metastatic cancer,” said Maryann Wahmann, founder and president of the Neuroendocrine Cancer Awareness Network. “These patients can live for many years with their cancer, yet the symptoms of carcinoid syndrome are what frequently limit their lives and restrict their activities every single day. So there is a tremendous need for effective new treatment options.”