FDA Grants Lexicon Orphan Drug Designation for Telotristat Etiprate for the Treatment of Carcinoid Syndrome
THE WOODLANDS, Texas, March 20, 2012 /PRNewswire/ — Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), announced today that the U.S. Food and Drug Administration (FDA) has granted its request for Orphan Drug designation for telotristat etiprate (LX1032) for the treatment of carcinoid syndrome. Carcinoid syndrome is a chronic condition caused by neuroendocrine tumors that usually originate from the gastrointestinal tract and is characterized by severe diarrhea and flushing episodes with long-term consequences that may include cardiac valve disease. Symptoms of carcinoid syndrome have been linked to excess production of serotonin by metastatic tumor cells. Telotristat etiprate is an orally-delivered serotonin synthesis inhibitor (SSI) drug that acts to reduce serotonin levels.
“The Orphan Drug designation of telotristat etiprate by the FDA provides important benefits for this drug’s clinical development for carcinoid syndrome,” commented Dr. Pablo Lapuerta, senior vice president of clinical development and chief medical officer at Lexicon. “We reported positive Phase 2 results in August 2011 in patients with carcinoid syndrome and are proceeding with preparations for a global Phase 3 development plan in this indication.”
In the U.S., Orphan Drug designation is generally granted for drugs intended for the treatment of diseases that affect fewer than 200,000 people and provides various incentives and preferences, such as seven years marketing exclusivity from the date of approval and certain tax advantages. The marketing of drugs which have received Orphan Drug designation remains subject to the receipt of marketing approval from the FDA. Lexicon has previously received Orphan Drug designation from the European Medicines Agency and Fast Track status from the FDA for telotristat etiprate.
About Telotristat Etiprate
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. Excessive levels of serotonin have been implicated in symptoms associated with carcinoid syndrome, especially diarrhea and carcinoid heart disease. In preclinical studies, telotristat etiprate reduced peripheral serotonin in several different species without affecting serotonin levels in the brain. In clinical studies, telotristat etiprate reduced serotonin levels consistent with preclinical results and reduced bowel movement frequency in patients with carcinoid syndrome. Telotristat etiprate is being developed under Fast Track and Orphan Drug designation from the U.S. Food and Drug Administration and Orphan Drug designation from the European Medicines Agency. 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 currently planning a Phase 2 trial of telotristat etiprate in mild to moderate ulcerative colitis.
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, irritable bowel syndrome, carcinoid 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.
Safe Harbor Statement
This press release contains “forward-looking statements,” including statements relating to Lexicon’s clinical development of telotristat etiprate (LX1032), including characterizations of the results of and projected timing of clinical trials of such compounds, and the potential therapeutic and commercial potential of telotristat etiprate. 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 those relating to Lexicon’s ability to successfully conduct clinical development of telotristat etiprate and preclinical and clinical development of its other 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. Unless specifically indicated otherwise, results reported as trends were not statistically significant. 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.
SOURCE Lexicon Pharmaceuticals, Inc.