First -in-Class Oral Tryptophan Hydroxylase Inhibitor to be Considered for Approval in Europe for the Treatment of Carcinoid Syndrome Diarrhea in Combination with Somatostatin Analog Therapy
Decision on Marketing Authorization Application Expected in Q3 2017
The Woodlands, Texas, July 21, 2017 - Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), announced today that the Committee for Medicinal Products for Human Use (CHMP), the committee of the European Medicines Agency (EMA) responsible for human medicines, has adopted a positive opinion for the Marketing Authorization Application (MAA) filed by Lexicon collaborator Ipsen for XERMELO® (telotristat ethyl) 250 mg to treat carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy.
The CHMP’s recommendation is now referred to the European Commission (EC), which is expected to render its final decision in the third quarter of 2017. If approved by the EC, the marketing of XERMELO will be authorized in all 28 countries of the European Union (EU), as well as Norway, Liechtenstein and Iceland.
"XERMELO was approved in the U.S. in February, and the CHMP’s positive opinion marks another important step forward in providing the first oral treatment option for adults affected by this rare disease in Europe. We thank the carcinoid syndrome community for their continued support, including the adults, families and caregivers who gave their time to participate in the clinical trials of telotristat ethyl with the goal of making this treatment a reality for patients," said Pablo Lapuerta, M.D., executive vice president and chief medical officer.
The XERMELO MAA was based on two randomized Phase 3 studies, TELESTAR and TELECAST. The TELESTAR study was a global, pivotal, double-blind Phase 3 study for telotristat ethyl in 135 patients from 12 countries with carcinoid syndrome diarrhea whose symptoms were not adequately controlled on SSA therapy. The primary objectives were to evaluate the safety and tolerability of telotristat ethyl and the effectiveness of the drug on the reduction in the average number of daily bowel movements over a 12-week study period. The TELECAST study was designed similarly to the TELESTAR study to provide additional efficacy and safety information in 76 patients with carcinoid syndrome diarrhea. The primary endpoints were the percent change from baseline in u5-HIAA at Week 12 and incidence of treatment emergent adverse events (TEAEs).
In July 2016, the EMA validated and accepted the MAA to begin the Centralized Procedure.
The CHMP is a scientific committee composed of representatives from the 28-member states of the EU, as well as Norway, Liechtenstein and Iceland. The committee reviews medical product applications on their scientific and medicinal merit and provides advice to the EC, which has the authority to approve medicines for the EU. The EC is expected to make its final decision on the XERMELO MAA in the third quarter of 2017.
About XERMELO (Telotristat Ethyl)
Discovered using Lexicon’s unique approach to gene science, XERMELO (telostristat ethyl) is the first and only approved oral therapy for carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSAs. XERMELO targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within metastatic neuroendocrine tumor (mNET) cells. Lexicon has built the in-house capability and infrastructure to launch and market XERMELO in the U.S., where it retains all commercialization rights. Lexicon also retains rights to market XERMELO in Japan. Lexicon has established a license and collaboration agreement with Ipsen to commercialize XERMELO in Europe and other countries outside of U.S. and Japan.
XERMELO was approved by the U.S. Food and Drug Administration on February 28, 2017 for the treatment of carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSA therapy. Carcinoid syndrome is a rare condition that occurs in patients living with metastatic NETs (mNETs) and is characterized by frequent and debilitating diarrhea. XERMELO targets the overproduction of serotonin inside mNET cells, providing a new treatment option for patients suffering from carcinoid syndrome diarrhea.
XERMELO (Telotristat Ethyl) Important Safety Information
- Warnings and Precautions: XERMELO may cause constipation, which can be serious. Monitor for signs and symptoms of constipation and/or severe, persistent, or worsening abdominal pain in patients taking XERMELO. Discontinue XERMELO if severe constipation or severe, persistent, or worsening abdominal pain develops.
- Adverse Reactions: The most common adverse reactions (=5%) include nausea, headache, increased gamma-glutamyl-transferase, depression, flatulence, decreased appetite, peripheral edema, and pyrexia.
- Drug Interactions: If necessary, consider increasing the dose of concomitant CYP3A4 substrates, as XERMELO may decrease their systemic exposure. If combination treatment with XERMELO and short-acting octreotide is needed, administer short-acting octreotide at least 30 minutes after administering XERMELO.
For more information about XERMELO, see Full Prescribing Information at www.xermelo.com.
About Lexicon Pharmaceuticals
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. In addition to XERMELO, Lexicon has a pipeline of promising drug candidates in clinical and pre-clinical development in diabetes and metabolism and neuropathic pain. For additional information please visit www.lexpharma.com.
Safe Harbor Statement
This press release contains "forward-looking statements," including statements relating to the safety and efficacy and therapeutic and commercial potential of XERMELO (telotristat ethyl) 250mg. 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 degree of market acceptance of XERMELO, the availability of coverage and reimbursement for XERMELO, Lexicon’s dependence on third parties for manufacturing and distribution of XERMELO, Lexicon’s compliance with applicable legal and regulatory requirements and other factors relating to the commercialization of XERMELO. 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, 2016, 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.
Kimberly Lee, D.O.
Head of Investor Relations and Corporate Strategy
Senior Director, Corporate Communications and Advocacy