Lexicon's Drug Candidate LX1032 For Carcinoid Syndrome Receives Orphan Drug Designation From EMEA

Main Category: Cardiovascular / Cardiology
Also Included In: Clinical Trials / Drug Trials;  Pharma Industry / Biotech Industry
Article Date: 22 Jul 2009 - 2:00 PDT

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Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), a biopharmaceutical company focused on discovering and developing breakthrough treatments for human disease, announced today that it has received orphan drug designation from the Committee for Orphan Medical Products (COMP) of the European Medicines Agency (EMEA) for LX1032, the company's oral drug candidate for managing gastrointestinal symptoms associated with carcinoid syndrome. LX1032 is designed to reduce serotonin production in patients with metastatic carcinoid tumors. Elevated levels of serotonin contribute to the gastrointestinal and possibly other symptoms experienced by these patients.

The EMEA is a regulatory agency in the European Union (EU) primarily responsible for the protection and promotion of public and animal health, through the evaluation and supervision of medicines for human and veterinary use. The goal of EMEA's orphan drug program is to promote the development of drugs intended for the prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU. Incentives for developing new drugs under orphan drug designation in the EU include 10 years of marketing exclusivity, regulatory assistance, reduced regulatory fees associated with applying for marketing approval, and assistance with clinical trial design. LX1032 is the first drug candidate for which Lexicon has obtained orphan drug status.

"Gaining orphan designation in Europe for LX1032 provides important regulatory and commercial advantages in the development of this new drug candidate for symptoms associated with carcinoid syndrome," said Philip Brown, M.D., J.D., senior vice president of clinical development at Lexicon. "We are now well positioned to conduct trials of LX1032 in Europe as part of an integrated global development strategy."

Lexicon recently initiated a Phase 2 clinical trial of LX1032 in the United States to evaluate the safety and tolerability of LX1032 and its effects on symptoms associated with carcinoid syndrome. The study is designed as a four-week, randomized, double-blind, placebo-controlled trial that will include up to 28 patients with carcinoid syndrome who are symptomatic despite treatment with currently available therapy. Up to four dose levels may be evaluated in a serial ascending fashion. Once an optimal or maximal dose is identified, additional patients will be added to confirm clinical observations.

In addition to LX1032, Lexicon has three other drug candidates progressing through clinical development, including LX1031 for irritable bowel syndrome, LX2931 for rheumatoid arthritis, and LX4211 for diabetes. For more information about Lexicon's clinical development programs, please visit www.lexpharma.com. LX1031 and LX1032 are being developed in a product development collaboration with Symphony Capital Partners, L.P. and its co-investors.

About LX1032

LX1032 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. Serotonin's breakdown product, 5-HIAA, is a biomarker used in the diagnosis of the condition. In preclinical studies, LX1032 reduced peripheral serotonin in several different species without affecting serotonin levels in the brain. In Phase 1 clinical studies, LX1032 reduced serotonin levels and urinary 5-HIAA in healthy volunteers consistent with preclinical results. LX1032 is being developed under Fast Track designation from the U.S. Food and Drug Administration and orphan drug designation from the European Medicines Agency.

About Carcinoid Syndrome

Carcinoid syndrome is a chronic condition caused by metastatic neuroendocrine tumors that usually originate from the gastrointestinal tract. These tumors secrete large amounts of serotonin, which can cause a variety of symptoms including severe diarrhea and abdominal discomfort. Patients with carcinoid syndrome currently have limited therapeutic options, and the standard of care includes chronic therapy with somatostatin analogues, which are delivered by injection. With current therapy, the gastrointestinal symptoms return over time in the vast majority of patients, hence the need for new agents.

About Lexicon

Lexicon is a biopharmaceutical company focused on discovering and developing breakthrough treatments for human disease. Lexicon currently has five drug candidates in development for autoimmune disease, carcinoid syndrome, diabetes, glaucoma and irritable bowel syndrome, all of which were discovered by the company's research team. The company 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.

Safe Harbor Statement

This press release contains "forward-looking statements," including statements relating to Lexicon's clinical development of LX1032 and the potential therapeutic and commercial potential of LX1032. 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 LX1032 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. Information identifying such important factors is contained under "Factors Affecting Forward-Looking Statements" and "Risk Factors" in Lexicon's annual report on Form 10-K for the year ended December 31, 2008, 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

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