Xermelo approved for Carcinoid Syndrome

FOR IMMEDIATE RELEASE FDA APPROVES LEXICON DRUG XERMELO™ (TELOTRISTAT ETHYL) 250 MG AS FIRST AND ONLY ORAL TREATMENT FOR CARCINOID SYNDROME DIARRHEA IN CANCER PATIENTS WITH METASTATIC NEUROENDOCRINE TUMORS First-In-Class: Tryptophan Hydroxylase Inhibitor Orphan Drug XERMELO Now Available to Treat Rare and Debilitating Condition Characterized by Frequent, Severe Diarrhea Conference Call and Webcast at 5:00 pm Eastern Time The Woodlands, Texas, February 28, 2017 – Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) announced today that the U.S. Food and Drug Administration (FDA) has approved XERMELO™ (telotristat ethyl) 250 mg as a first and only orally administered therapy for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapyi. Carcinoid syndrome is a rare and debilitating condition that affects people with metastatic neuroendocrine tumors (mNETs)ii. XERMELO targets the overproduction of serotonin inside mNET cellsiii, providing a new treatment option for patients suffering from carcinoid syndrome diarrhea. This new treatment is now available by prescription and will be in select specialty pharmacies beginning March 6, 2017. “Today’s approval of XERMELO represents a shift in the treatment paradigm of carcinoid syndrome diarrhea for cancer patients who are inadequately controlled by SSA therapy, and until now, have had limited options to manage this debilitating condition,” said Lonnel Coats, Lexicon’s president and chief executive officer. “We are proud to have discovered and developed this ground-breaking orphan drug, and it is an honor to make it available for the thousands of patients currently suffering from this condition who wish to lead a more routine life with fewer incidences of severe diarrhea.” Carcinoid syndrome is a rare condition that occurs in patients living with mNETsiv and 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. “The approval of XERMELO establishes a new treatment option for patients with carcinoid syndrome diarrhea that is inadequately controlled by SSA therapy,” said Matthew H. Kulke, M.D., TELESTAR primary investigator, director of the Program in Neuroendocrine and Carcinoid Tumors at Dana Farber Cancer Institute and Professor of Medicine, Harvard Medical School. “Inhibition of tumoral serotonin production represents a novel approach for patients with this condition. Studies have shown that XERMELO can reduce the debilitating effects of carcinoid syndrome diarrhea and has a favorable efficacy and safety profile in patients who currently have limited treatment options.” Page 2 About XERMELO™ Discovered using Lexicon’s unique approach to gene science, XERMELO is the first and only approved oral therapy for carcinoid syndrome diarrhea. XERMELO targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within 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 telotristat ethyl in Japan. Lexicon has established a license and collaboration agreement with Ipsen to commercialize telotristat ethyl in Europe and other countries outside of U.S. and Japan. For more information about XERMELO, please visit www.xermelo.com. XERMELO™ 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 GGT, depression, peripheral edema, flatulence, decreased appetite, and pyrexia.  Drug Interactions: If necessary, consider increasing the dose of concomitant CYP3A4 substrates, as XERMELO may decrease their systemic exposure. For more information about XERMELO, see Full Prescribing Information at www.xermelo.com.

About Lexicon 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 preclinical development in diabetes and metabolism and neuropathic pain. For additional information please visit www.lexpharma.com. In an effort to ensure all appropriate patients have fast access to affordable treatment, Lexicon offers LexCares—a comprehensive program designed to guide patients through each step of their treatment journey. Through LexCares, patients will have access to financial assistance programs that may reduce or eliminate out-of-pocket costs associated with their XERMELO prescription. For additional information about the LexCares program, visit www.xermelo.com/lexcares. Page 3 Safe Harbor Statement This press release contains “forward‑looking statements,” including statements relating to the safety and efficacy and the therapeutic and commercial potential of XERMELO (telotristat ethyl) 250 mg. 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, 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.


For Additional Information Contact: Investors Chas Schultz Senior Director, Finance and Communications (281) 863-3421 cschultz@lexpharma.com Media Lori Rodney Vice President GCI Health (212) 798-9915 lori.rodney@gcihealth.com i Data on file – XERMELO label. Lexicon Pharmaceuticals, Inc. 2017. ii National Organization for Rare Disorders. Carcinoid Syndrome. Retrieved from: https://rarediseases.org/rare-diseases/carcinoid-syndrome/. Accessed November 22, 2016. iii Data on file. Lexicon Pharmaceuticals, Inc. 2017. iv Rorstad O. Prognostic indicators for carcinoid neuroendocrine tumors of the gastrointestinal tract. J Surg Oncol. 2005; 89(3):151-60.

Healing NET Foundation Summit delivers on NET-Cancer discussions


For further information contact:
Cindy Lovelace TheHealingNetFoundation@gmail.com 615.369.6463
Freddie Evans fevans@the-lynx-group.com 732.992.1895

NET-Cancer Summit Delivers on Diagnosis and Treatment Debates

Nashville ---The inaugural international Healing NET Foundation Summit delivered on its promise to bring key discussions on diagnosis and care for the complicated NET-Cancer to the forefront of debate by many of the world’s leading physician and research experts. Topics covered the use of Somatostatin analogues and other targeted drugs and chemotherapies along with the combinations of treatment at the micro/molecular level and the macro/surgical levels to help patients live longer and more productive lives. One of the drugs, Afinitor, received Food and Drug Administration (FDA) approval for GI and Lung NETs tumors this week. The Summit’s faculty also looked at the use of biomarkers in clinical practice, staging of disease and the use of nuclear medicine. New surgical techniques and tools, and the care of children as well as adults were presented the NET-Cancer experts.

The Summit, organized in partnership with the Association for Value-Based Cancer Care (AVBCC), a successful division of The Lynx Group, brought leading researchers together with the physicians from across the United States and Europe who have had years of experience in treating patients with NET-Cancer. This disease is often misdiagnosed and treated, due in part to its previous reference as carcinoid tumors, and seen as a benign disease.

Summit organizers acknowledged that the meeting is a first step in examining and debating protocols for how patients with NET-Cancer are viewed by clinicians in locations across the United States. The overall goal is to diagnose the disease earlier and help define the best course of treatment once people are diagnosed. Patients and physicians, anxious for information of the Summit’s discussions, will be able to watch the panel discussions on video in the coming weeks and a summary document will be made available as quickly as possible for the medical community.

“The problem is that people who have NET-Cancers experience sets of symptoms that can easily be written off as common illnesses,” said Dr. Eric Liu, Chief Medical Advisor for The Healing NET Foundation.

 “A typical patient experience is about five-seven years of doctor-hopping before a clinician might begin to put the puzzle pieces together and look for NET-Cancer tumors as the root cause of all the symptoms,” Liu explained.

Liu noted that there are medications and new diagnostic tests and treatments that can make a big difference in patient quality of life and long-term survival, but the clinician first has to suspect NET-Cancer.

The Healing NET Foundation is a 501(c)3 organization established to address the lack of information available to physicians and in particular, the first person a patient exhibiting these symptoms might see – their community doctor. The Foundation supports NET-Cancer education in the medical community and advocates for the best possible care for NET-Cancer patients.

“Physicians have a saying that can complicate the rare disease diagnosis,” said Cindy Lovelace, Executive Director of The Healing NET Foundation.

“In medical school, they are taught to think horses, when they hear hoofbeats, not zebras. In other words, consider the more common suspects and not a rare condition when a patient presents with symptoms. However, the NET-Cancer patient IS the zebra.”

Lovelace, a NET-Cancer survivor herself, was once referred to by an ER physician as a ‘zebra’ years before her diagnosis. “My cancer was discovered incidentally because of a scan for an unrelated condition and then took another eight months to figure out what it was,” she said. “Ironically, I was already being followed by an oncologist due to a breast cancer diagnosis five years previously, but there was no information and no protocol for this new cancer,” she said. It would take another six months for Lovelace to find an expert in NET-Cancer, but Lovelace considers herself one of the lucky ones. “Most people go years without answers to the puzzle,” she said. Liu agreed, “Just like no two zebra stripes are alike, no two NET-Cancer patients have exactly the same manifestation of the disease. Sometimes a patient has symptoms and sometimes, like Lovelace, the tumors are found incidentally with no symptoms.”

NET-Cancer which affects both sexes, all ages and all ethnicities can be managed if caught early with patients being then able to live productive lives. Liu noted that 90 percent of cancer patients in the United States are treated by community-based physicians operating outside the academic and research communities. This means that even when a patient is ultimately and properly diagnosed with NET-Cancer they will likely be followed by a doctor who has little experience with the disease.

“The Healing NET Foundation wants to help doctors pull all those pieces of the puzzle together by connecting them with expert physicians and facts – and ultimately help patients receive the best possible care for this rare disease,” said Lovelace.

The discussions at The Healing NET Foundation Summit are a step toward bringing the academic and research community experts together to begin sharing knowledge and experience that can help community-based physicians and specialists. Attendees were selected based on their experience in the diagnosis and treatment of NET-Cancer patients and represent 30 leading international centers of research and clinical care. They come from subspecialties of oncology, radiology, endocrinology, cardiology, gastroenterology, basic science and surgery.

Statistics that would reflect the incidence and prevalence of NET-Cancer in the United States are difficult to estimate. Examination of large government databases suggest that there are greater than 100,000 patients with this disease with the number of new cases increasing each year. However, tracking these patients is challenging because the terminology for this class of disease was only recently established.

The Lynx Group, an alliance of medical education and communication companies which includes the AVBCC, will be supporting the Summit with a team of writers who are tasked with capturing the discussions and areas of consensus to formulate information that will be made available to physicians nationwide to aid in the treatment of NET-Cancer patients.

“Our goal is to help assemble discussions from the expert panels of physicians and researchers and begin creating materials that will reflect the latest and most accurate information about NET-Cancer,” said Brian Tyburski, President and CEO of The Lynx Group.

“We are very excited to be able to be part of this inaugural Summit that has the potential to create a profound impact on the lives of many cancer patients and their families,” he said.

Abigail Adair, President of The Lynx Group said, “We are committed to getting the Summit information published and available to physicians and patients as rapidly as possible and are sensitive to the fact that these discussions can have an immediate impact in saving lives.”

New information gleaned from the Summit will serve as a companion and update to The Healing NET Foundation’s Primer for Healthcare Professionals, published in October, 2015, now available at www.TheHealingNET.org
Participants at The Healing NET Foundation Summit are prepared to share good news about the effective use of some existing cancer drugs in treating NETs and eminent FDA approval of a new tool that will enable physicians to make earlier and more definitive diagnoses.

Neuroendocrine cells produce hormones that control breathing and digestion. Sometimes these cells go haywire and become tumors that grow anywhere in the body, but are most commonly found in the intestines, pancreas and liver. They can also develop in the lung and are even associated with a heart valve defect. The fact that the tumors can be anywhere means that patients may have confusing symptoms sending them to a series of specialists who, not seeing a bigger picture, will treat symptoms but miss the root cause.
Among the frequently misdiagnosed symptoms are cramping, intestinal upsets, diarrhea, flushing faces, breathing difficulty, deep vein thrombosis or even hypoglycemia.

The Healing NET Foundation is a non-profit 501(c)3 formed to raise awareness, support and to help fund the best possible care for patients with neuroendocrine tumors (NETs). By developing access to state-of-the-art information and training for physicians in the United States to catch up with successful practitioners in other parts of the world, The Healing NET Foundation seeks to then enable multi-disciplinary practitioners to provide the comprehensive care essential for NET-Cancer patients. By educating the public, The Healing Net Foundation seeks to connect patients with physicians prepared to provide comprehensive care. For more about The Healing NET Foundation go to: www.TheHealingNET.org

The mission of AVBCC is to provide a forum for payers, providers and the entire oncology team to consider and evaluate the cost-value issues particular to cancer treatments and its impact on patient care and outcomes. This unique focus is achieved through discussions and collaborations with those involved in evaluating therapies, treating patients and paying for care.
AVBCC is the fastest growing national specialty organization dedicated to improving the care of cancer patients and their quality of life by discussing, considering and evaluating the value equation of how it relates to new and existing cancer therapies. This organization, which currently consists of more than 500 members was established to provide a network for payers and oncology healthcare professionals to interact and network in order to promote optimal care for patients and their families. For more: www.avbcconline.org

The Lynx Group, one of the fast growing medical education and communications companies, is a global strategic alliance of medical communications and education companies. Through its unique business model, The Lynx Group strives to provide pivotal and contemporary information and education for all stakeholders in healthcare. The Lynx Group is the official association management and marketing company of the Academy of Oncology Nurse & Patient Navigators, Association for Value-Based Cancer Care, Association for Oncology Practice Management and the National Association of Specialty Pharmacy. For more information visit: the-lynx-group.com

Allen Cohn (Oncology, Rocky Mountain Cancer Centers, Denver, CO), Eric Liu (Neuroendocrinology/Surgery, Rocky Mountain Cancer Centers, Denver CO), Charles Nutting (Interventional Radiology, RIA, Denver, CO)

Scientific Advisory Committee:
George Fisher (Oncology, Stanford University, Palo Alto, CA), David Metz (Gastroenterology, University of Pennsylvania, Philadelphia, PA), Irvin Modlin (WREN Laboratories, New Haven, CT), Kjell Oberg (Neuroendocrinology, Uppsala University, Sweden), Tom O’Dorisio (Endocrine, University of Iowa, Iowa City, IA)

Lowell Anthony (Oncology, University of Kentucky, Lexington, KY), Joy Ardill (Basic Science, Belfast, Ireland), Emily Bergsland (Oncology, UCSF, San Francisco, CA), Lisa Bodei (Nuclear Medicine, Deputy Director, Nuclear Medicine Division, European Institute of Oncology. Località Milano, Italia), Phillip Boudreaux (Surgery, Oschner Clinic, New Orleans, LA), Jennifer Chan (Oncology, DFCI, Boston, MA), Wouter de Herder (Endocrinology, Erasmus, Rotterdam, Netherlands), Hal Gerstein (Oncology, Private Practice, Long Island, NY), Dan Granberg (Neuroendocrinology, Uppsala, Sweden), Anthony Heaney (Endocrinology, UCLA, Los Angeles, CA), Mark Kidd (WREN Laboratories, New Haven, CT), Sue O’Dorisio (Pediatric Oncology, University of Iowa, Iowa City, IA), Richard K. Parker (Surgery, Cardiothoracic Surgery Associates), Scott Paulson (Oncology, Texas Oncology, Dallas, TX), Rodney Pommier (Surgery, Oregon Health Sciences, Portland, OR), Robert Ramirez (Oncology, Oschner Clinic, New Orleans, LA), Guido Rindi (Pathology, Catholic University, Rome, Italy), Michael Soulen (Interventional Radiology, University of Pennsylvania, Philadelphia, PA), Jonathan Strosberg (Oncology, Moffitt Cancer Center, Tampa, FL), Laura Tang (Pathology, Memorial Sloan Kettering, New York, NY), Arthur Vinik (Endocrinology, Eastern Virginia Medical School, Norfolk, VA), Jerry Zacks (Cardiology, Mt. Sinai, New York, NY)

HEALING NET SUMMIT UNDERWRITERS: Norvartis, Lexicon, Advanced Accelerator Applications, BioTheranostics, Chiasma, Rocky Mountain Cancer Centers/ U.S. Oncology Research, Angio Dynamics, Neuroendocrine Cancer Awareness Network, Sarah Cannon Cancer Network/HealthONE, Foundation Medicine.

Supported in part by educational grants from: Ipsen, Sirtex


World Cancer Day Provides Springboard for NET-Cancer Summit

NASHVILLE – Diagnosis and treatment of Neuroendocrine (NET) Cancers will be debated Feb. 24- 26 when physicians and bench researchers from more than 30 research hospitals across the United States and Europe gather in Beaver Creek, Colorado, for a Summit hosted by The Healing NET Foundation in partnership with the Association for Value-Based Cancer Care.

Celebrities such as Steve Jobs, Dave Thomas and Audrey Hepburn are among the thousands of individuals who have fought NET-Cancer, formerly referred to as carcinoid cancer. Patients tend to present unusual and what could be viewed as common sets of symptoms which can cause them to be misdiagnosed with more common benign diseases, or have a tumor discovered incidentally without any symptoms at all. The zebra has become symbolic of NET-Cancer patients, because medical students are encouraged to think of common illness rather than to view every patient as having an obscure problem, but sometimes common symptoms can be caused by something unusual, “like zebras”. Just like no two zebra stripes are alike, no two NET-Cancer patients have exactly the same manifestation of the disease, which makes it more difficult to establish a protocol treatment.

Attendees of the inaugural Healing NET Foundation Summit will share their clinical experiences in the diagnosis and treatment of NET-Cancer patients. It presents in people of both sexes, all ages and all ethnicities, causing confusing symptoms and sometimes no symptoms. Many NET-Cancer patients often spend five to seven years “doctor hopping” before an accurate diagnosis, dramatically reducing chances of successful treatment and possible recovery. If caught early, however, NET-Cancer can be manageable, and patients may live productive lives.

With a mission to further awareness of and education about NET-Cancer in the medical community and to profoundly impact the quality of patient care, The Healing NET Foundation views the Summit as a step toward bringing the academic and research community experts in the disease together to begin sharing knowledge and experience with community-based physician specialists.

The goal of the Summit is to develop material to distribute accurate information about NETs cancers that prompts physicians to reflect NET-Cancer as the primary diagnosis rather than continue to treat random symptoms in difficult-to-diagnose patients. Patients with an array of symptoms are frequently shuffled among specialized practices like oncology, radiology, endocrinology, pulmonology, cardiology, gastroenterology and surgery.

With 90 percent of cancer patients in the United States treated by community-based physicians operating outside academic and research communities, even if a patient is ultimately properly diagnosed with NETs, a community-based doctor may have little access to information that will allow them to successfully treat NET-Cancer patients.

Participants at the Healing NET Foundation Summit are prepared to share good news about the effective use of some existing cancer drugs in treating NETs and eminent FDA approval of a new tool that will enable physicians to make earlier and more definitive diagnoses.

Summary documents resulting from the Summit are being distributed to physicians to help better identify NET-Cancers and provide proper care for NET patients. The new information will serve as a companion and update to The Healing NET Foundation’s Primer for Healthcare Professionals published in October 2015, which is available at www.TheHealingNET.org.

Novartis, Lexicon, AAA, Angio Dynamics, BioTheranostics, Foundation Medicine, Neuroendocrine Cancer Awareness Network, Anonymous Donor, Sarah Cannon Cancer Network, and Chiasma

PSA: World NET Cancer Awareness Day is November 10

Trey Anastasio, guitarist, composer and vocalist, speaks out about a rare cancer that affected his family, and advocates for more education and awareness of Neuroendocrine Cancer.  Trey supports the mission of the Healing NET Foundation to educate and support the medical community at large about NET cancer, and promotes World NET Cancer Awareness Day on November 10, 2016.

During the month of November the HNF will feature one story each day of the month on our website to promote NET Cancer Awareness.  Click here to Tell Your Story

Watch our public service announcement video below. Or visit our YouTube channel to view it.