Dedicated to Innovation
The Woodlands, Texas, October 12, 2012 – Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), a biopharmaceutical company focused on discovering and developing breakthrough treatments for human disease, announced today that it has initiated a registrational, Phase 3 clinical trial of telotristat etiprate, an investigational new drug candidate for the treatment of carcinoid syndrome. Carcinoid syndrome is a chronic condition caused by neuroendocrine tumors that usually originate from the gastrointestinal tract. It is characterized by severe diarrhea and flushing episodes with long-term consequences including malnutrition, heart disease, and death. Carcinoid syndrome has been linked to excess production of serotonin by metastatic tumor cells.
Telotristat etiprate is an oral drug candidate designed to treat carcinoid syndrome by reducing serotonin production in patients with metastatic carcinoid tumors. Telotristat etiprate has Fast Track status and Orphan Drug designation from the U.S. Food and Drug Administration, and Orphan designation from the European Medicines Agency.
“The registrational, Phase 3 study is designed to evaluate the efficacy and safety of telotristat etiprate in patients with carcinoid syndrome that is refractory to somatostatin therapy,” said Pablo Lapuerta M.D., senior vice president and chief medical officer at Lexicon. “We are encouraged by the consistent clinical results obtained to date in two Phase 2 trials that showed telotristat etiprate significantly improved multiple dimensions of carcinoid syndrome with a favorable safety profile.”
The Phase 3 registrational trial is a 12-week, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of telotristat etiprate as a new treatment for carcinoid syndrome that is refractory to current therapy. Two dose levels of telotristat etiprate, 250mg and 500mg three times daily (TID), will be tested along with placebo. The study will include approximately 105 patients with refractory carcinoid syndrome on background somatostatin analog therapy, assigned equally among the 3 treatment groups using a 1:1:1 randomization plan. After the 12-week treatment period, patients will enter a 36 week open-label extension period where all patients will receive telotristat etiprate 500mg TID.
About Telotristat Etiprate (LX1032)
Telotristat etiprate was discovered and developed at Lexicon to reduce serotonin production by inhibiting tryptophan hydroxylase (TPH), a key enzyme in the synthesis of serotonin. Serotonin’s breakdown product, 5-HIAA, is a biomarker used in the diagnosis of the condition. In preclinical studies, telotristat etiprate reduced 5-HIAA and peripheral serotonin in several different species without affecting serotonin levels in the brain. Telotristat etiprate is a member of a new class of oral drugs invented by Lexicon, the serotonin synthesis inhibitors, which are being developed in a spectrum of gastrointestinal indications. Lexicon is also carrying out a Phase 2 trial of telotristat etiprate in mild to moderate ulcerative colitis.
About Carcinoid Syndrome
Carcinoid syndrome is a chronic condition caused by metastatic neuroendocrine tumors that usually originate from the gastrointestinal tract. Patients with carcinoid syndrome currently have limited therapeutic options, and the standard of care includes chronic therapy with somatostatin analogs, which are delivered by injection. With current therapy, carcinoid syndrome symptoms return over time in most patients, hence the need for new agents.
Lexicon is a biopharmaceutical company focused on discovering breakthrough treatments for human disease. Lexicon currently has four drug programs in mid-stage development for diabetes, carcinoid syndrome, irritable bowel syndrome and rheumatoid arthritis, all of which were discovered by Lexicon’s research team. Lexicon has used its proprietary gene knockout technology to identify more than 100 promising drug targets. Lexicon has focused drug discovery efforts on these biologically-validated targets to create its extensive pipeline of clinical and preclinical programs. For additional information about Lexicon and its programs, please visit www.lexpharma.com.
This press release contains "forward-looking” statements, including 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 those relating to Lexicon's ability to successfully conduct preclinical and clinical development of its potential drug candidates, advance additional candidates into preclinical and clinical development, obtain necessary regulatory approvals, 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, that 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, 2011, 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.