On August 3, 2015 Ipsen (Euronext: IPN; ADR: IPSEY) reported that its partner, Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), disclosed positive results from the pivotal Phase 3 TELESTAR study (Press release, Ipsen, AUG 3, 2015, View Source [SID:1234506977]). TELESTAR evaluated the efficacy and safety of telotristat etiprate for carcinoid syndrome patients with metastatic neuroendocrine tumor (NET) inadequately controlled by somatostatin analog (SSAs), the current standard of care.

Top-line results from the Phase 3 study show that patients who added telotristat etiprate to the standard of care at both the 250 mg and 500 mg doses experienced a statistically significant reduction from baseline compared to placebo in the average number of daily bowel movements over the 12-week study period (p<0.001), meeting the study’s primary endpoint. Complete results from the Phase 3 TELESTAR study will be presented at an upcoming scientific conference. Claude Bertrand, Executive Vice President R&D and Chief Scientific Officer, Ipsen stated: "We are pleased with the positive top-line Phase 3 results for telotristat etiprate. Should telotristat etiprate be approved, its oral formulation would satisfy an unmet medical need for patients with carcinoid syndrome not adequately controlled with SSAs therapy. After the approval of Somatuline Autogel as the first and only SSA for tumor control of gastro-intestinal and pancreatic neuroendocrine tumors (NETs) in the US and Europe, and the more recent acquisition of Octreopharm’s nuclear medicine platform, these results are an important milestone in our strategy to become a global leader in NETs." Professor Marianne Pavel, Charité-Universitätsmedizin Berlin, Principal Investigator, study lead in Germany stated: "There is a need for effective treatment to improve the health and comorbidities of patients whose carcinoid syndrome is not adequately controlled with somatostatin analogue therapy. Results of this positive phase 3 study with telotristat etiprate are promising in improving symptom management, and outcome of patients with carcinoid syndrome not adequately controlled with SSA therapy". In October 2014, Ipsen and Lexicon announced that they had entered into an exclusive licensing agreement for Ipsen to commercialize telotristat etiprate, excluding the US and Japan, with a focus on the symptomatic treatment of carcinoid syndrome inadequately controlled with SSAs. Lexicon retains sole rights to commercialize telotristat etiprate in the United States and Japan. About the study The double-blind Phase 3 study known as TELESTAR (Telotristat Etiprate for Somatostatin Analogue Not Adequately Controlled Carcinoid Syndrome) enrolled 135 patients with carcinoid syndrome who were not adequately controlled on SSA therapy, the current standard of care. The three-arm study evaluated two doses of oral telotristat etiprate – 250 mg and 500 mg, each taken three times daily – against placebo over a 12-week period and measured the reduction from baseline in the average number of daily bowel movements. Patients in both the treatment and placebo arms continued their SSA therapy throughout the study. Top-line results from TELESTAR 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 to placebo in the average number of daily bowel movements over the 12-week study period (p<0.001), meeting the study’s primary endpoint. In another key finding, a substantially greater proportion of patients on telotristat etiprate achieved a durable response (44 percent and 42 percent in the 250 mg and 500 mg arms, respectively), defined as at least a 30 percent reduction in daily bowel movements over at least half the days of the study period, as compared to 20 percent response on placebo (p<0.040). Patients who received 250 mg of telotristat etiprate experienced a 29 percent reduction in the average number of daily bowel movements during the final week (week 12) of the study period compared to baseline, and those in the 500 mg arm had a 35 percent reduction, while the placebo group showed a 17 percent reduction. These results are consistent with those seen in the 12-week Phase 2 study of telotristat etiprate. The proportion of patients with treatment-emergent adverse events (AEs), serious AEs and discontinuation due to AEs were generally similar in all three treatment arms. The tolerability profile of telotristat etiprate 250 mg tid appeared similar to placebo and somewhat better than 500 mg tid with respect to gastrointestinal discomfort and mood. Further in depth analysis of safety and tolerability data will be conducted. The 12-week double-blind study period is being followed by a 36-week open-label extension where all patients receive telotristat etiprate 500 mg three times daily.