Results from Pivotal Phase 3 Clinical Study of Telotristat Ethyl Show Clinical Benefit in Patients with Carcinoid Syndrome
The Woodlands, Texas, October 31, 2016 – Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), announced today that the Journal of Clinical Oncology published detailed results from Lexicon’s pivotal Phase 3 TELESTAR clinical study of telotristat ethyl in patients with carcinoid syndrome. The article, titled “Telotristat Ethyl, a Tryptophan Hydroxylase Inhibitor for the Treatment of Carcinoid Syndrome,” is available online at the Journal of Clinical Oncology website at ascopubs.org/journal/jco.
“Inhibition of tryptophan hydroxylase with telotristat ethyl represents a novel approach to reducing tumoral serotonin production in patients with carcinoid tumors,” said TELESTAR primary investigator and lead author, Matthew H. Kulke, M.D., Director, Program in Neuroendocrine and Carcinoid Tumors and Senior Physician, Dana Farber Cancer Institute, and Professor of Medicine, Harvard Medical School. “These results are encouraging for patients who suffer from the debilitating condition of carcinoid syndrome.”
TELESTAR was a global double-blind Phase 3 study which enrolled 135 patients from 12 countries with carcinoid syndrome whose symptoms were not adequately controlled on somatostatin analog therapy (SSA), the current standard of care. Data show that patients who added telotristat ethyl 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. There was also a statistically significant reduction in the levels of urinary 5-hydroxyindole acetic acid (5-HIAA), the main metabolite of serotonin, from baseline to week 12 with a reduction of 40 mg/24 hours (250 mg arm) and 58 mg/24 hours (500 mg arm) versus an increase of 11 mg/24 hours in the placebo arm (p<0.001). Treatment with telotristat ethyl was generally well tolerated during the double-blind treatment period.
Eighty-five percent of the patients originally enrolled in TELESTAR opted to continue study participation, receiving treatment with 500 mg telotristat ethyl in a 36-week open-label extension (OLE) study. Results from the OLE showed sustained bowel movement responses to treatment and no additional safety signals. Additional results detailed in the publication showed evidence that telotristat ethyl may also improve stool consistency, reduce the urgency to defecate and reduce the use of rescue short-acting octreotide.
“The Journal of Clinical Oncology publication provides key data on the benefits of telotristat ethyl for patients with carcinoid syndrome. It includes meaningful results extending out nearly a year,” said Pablo Lapuerta, M.D., Lexicon’s executive vice president and chief medical officer. “We are very encouraged about the potential benefits telotristat ethyl may bring in terms of bowel movement frequency and other measures of carcinoid syndrome severity.”
About Carcinoid Syndrome
Carcinoid syndrome is a rare disease affecting thousands of cancer patients with metastatic neuroendocrine tumors (mNETs) that have spread to the liver and other organs from the gastrointestinal tract. The condition is characterized by frequent and debilitating diarrhea that often prevents patients from leading active, predictable lives, as well as by facial flushing, abdominal pain, fatigue and, over time, heart valve damage.
About Telotristat Ethyl
Discovered using Lexicon’s unique approach to gene science, telotristat ethyl is the first investigational drug in clinical studies to target tryptophan hydroxylase, an enzyme that triggers the excess serotonin production within mNET cells that is a key driver of carcinoid syndrome. While existing treatments for carcinoid syndrome work to reduce the release of serotonin outside tumor cells, telotristat ethyl works at the source to reduce serotonin production within the tumor cells. By specifically inhibiting serotonin production, telotristat ethyl seeks to control this important driver of carcinoid syndrome and, in combination with SSA therapy, the current standard of care, to provide patients with more control over their disease.
Telotristat ethyl has received Fast Track and Orphan Drug designation from the U.S. Food and Drug Administration and has been granted priority review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2017.
Lexicon retains rights to market telotristat ethyl in the U.S. and Japan, and is building the in-house commercial infrastructure to serve the U.S. market. Lexicon has a license and collaboration agreement with Ipsen to commercialize telotristat ethyl in Europe and other countries outside the U.S. and Japan.
Lexicon is a fully integrated biopharmaceutical company that is applying a unique approach to gene science based on Nobel Prize-winning technology to discover and develop precise medicines for patients with serious, chronic conditions. Through its Genome5000™ program, Lexicon scientists have studied the role and function of nearly 5,000 genes over the last 20 years and have identified more than 100 protein targets with significant therapeutic potential in a range of diseases. Through the precise targeting of these proteins, Lexicon is pioneering the discovery and development of innovative medicines to safely and effectively treat disease. Lexicon has a pipeline of promising drug candidates in clinical and pre-clinical development in oncology, diabetes and metabolism. For additional information please visit www.lexpharma.com.
Safe Harbor Statement
This press release contains “forward-looking statements,” including statements relating to Lexicon’s clinical development of telotristat ethyl (formerly referred to as telotristat etiprate and LX1032) and the results of and projected timing of clinical trials and the potential therapeutic and commercial potential of telotristat ethyl. In addition, this press release also contains forward-looking statements relating to Lexicon’s growth and future operating results, discovery and development of products, strategic alliances and intellectual property, as well as other matters that are not historical facts or information. All forward-looking statements are based on management’s current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the risk that clinical studies of telotristat ethyl may be halted, delayed or otherwise not demonstrate safety or efficacy, the risk that the FDA and other regulatory authorities may not grant regulatory approval of telotristat ethyl in accordance with Lexicon’s currently anticipated timelines or at all, and the risk that such regulatory approvals, if granted, may have significant limitations on the approved use of telotristat ethyl. As a result, telotristat ethyl may never be successfully commercialized. Other risks include Lexicon’s ability to meet its capital requirements, successfully conduct preclinical and clinical development and obtain necessary regulatory approvals of its other potential drug candidates, achieve its operational objectives, obtain patent protection for its discoveries and establish strategic alliances, as well as additional factors relating to manufacturing, intellectual property rights, and the therapeutic or commercial value of its drug candidates. Any of these risks, uncertainties and other factors may cause Lexicon’s actual results to be materially different from any future results expressed or implied by such forward-looking statements. Information identifying such important factors is contained under “Risk Factors” in Lexicon’s annual report on Form 10-K for the year ended December 31, 2015, as filed with the Securities and Exchange Commission. Lexicon undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.