ITF-2984

Exelixis said Thursday that a Phase III trial evaluating Cabometyx (cabozantinib) against placebo has been stopped early due to a "dramatic improvement" in efficacy observed at an interim analysis. "As there is no standard of care for patients with advanced pancreatic or extra-pancreatic neuroendocrine tumours whose disease has progressed after prior therapy, we are pleased to see that [Cabometyx] improved outcomes for two additional patient populations living with advanced, difficult-to-treat cancers," remarked Will Berg, head of medical affairs at Exelixis.The CABINET trial enrolled a total of 290 patients with either advanced pancreatic neuroendocrine tumours or advanced extra-pancreatic neuroendocrine tumours who experienced progression after at least one FDA-approved line of prior therapy other than somatostatin analogues. Participants were randomised into the Cabometyx or placebo arms of the study in each of the two cohorts. Upon disease progression, patients were unblinded, and those receiving placebo were permitted to cross over to open-label therapy with Cabometyx.The primary endpoint was progression-free survival (PFS), while secondary goals assessed overall survival (OS) and radiographic response rate. According to Exelixis, the interim analysis showed that Cabometyx "substantially prolonged" the time without disease progression or death in both cohorts of the trial. The results prompted an independent data and safety monitoring board to unanimously recommend that the CABINET study be unblinded and stopped early. Detailed findings will be presented at an upcoming medical meeting and discussed with the FDA, the company said.Back-to-back positive readoutsThe latest readout marks another clinical trial win for the drug. Earlier this week, Exelixis and partner Ipsen reported that Cabometyx in combination with Roche's PD-L1 inhibitor Tecentriq (atezolizumab) significantly extended PFS in the Phase III CONTACT-02 study of patients with metastatic castration-resistant prostate cancer. A trend toward improvement in OS was also observed, although those data remain immature.Cabometyx is currently approved in the US for certain patients with renal cell carcinoma, hepatocellular carcinoma and thyroid cancer. It has also received regulatory nods in the EU and other countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialisation and further clinical development of Cabometyx outside of the US and Japan. The following year, Exelixis granted Takeda rights to the tyrosine kinase inhibitor in Japan. 

DEERFIELD, Ill., Dec. 7, 2022 /PRNewswire/ -- TerSera Therapeutics announced today a new sponsorship with the Carcinoid Cancer Foundation (CCF) and Alira Health to expand and optimize a holistic patient journey digital platform for patients with Neuroendocrine Tumors and Carcinoid Syndrome. Carcinoid syndrome (CS) is a rare condition that occurs in patients living with metastatic neuroendocrine tumors (mNETs) and is characterized by frequent and debilitating diarrhea that often prevents patients from leading active, predictable lives. Other symptoms of CS include facial flushing, abdominal pain, fatigue and, over time, for some people, heart valve damage.1-3 To support patients in coping with the impact NETs have on their quality of life and collaborate with their care team on treatment decisions, Alira Health and the Carcinoid Cancer Foundation have collaborated since 2015 to build "NET Cancer Health Storylines," a customized version of the Health Storylines™ app that makes it easy for patients to record their symptoms, nutritional concerns, moods, medication and more. The NET Health Storylines app provides patients with resources developed by NET experts. Specific tools include a Stool Diary, NET Nutrition tool, Medication tracker, and additional educational resources. The app is available for download at the NET Cancer Health Storylines website or through the Apple App Store or Google Play. "We are grateful for this support from TerSera to continue to enhance the NET Health Storylines platform to distribute meaningful educational content to NETs patients. The resources available through this platform are important tools in supporting NET patients throughout their journey," said Keith Warner, Chief Executive Officer of the Carcinoid Cancer Foundation. TerSera Therapeutics is the manufacturer of Xermelo (telotristat ethyl), the only oral medication approved for the treatment of carcinoid syndrome diarrhea (CSD), in combination with somatostatin analog (SSA) therapy.4 "Collaborating with CCF and the NET Cancer Health Storylines platform is a fantastic opportunity for us to support patients with Carcinoid Syndrome Diarrhea beyond our existing clinical nurse educator program. We are also looking forward to learning from patients how we can continue to support them better on their journey," said Pamela Gaytan, Executive Director, Clinical Nurse Educators and Patient Advocacy of TerSera Therapeutics. About Carcinoid Syndrome and Carcinoid Syndrome Diarrhea Carcinoid syndrome (CS) is a rare condition which affects approximately 1 out of every 5 patients with neuroendocrine tumors (NETs).5 In CS, patients have NETs that overproduce certain hormones such as serotonin, bradykinin, and histamine.1 One key hormone that is overproduced in CS is serotonin. Elevated serotonin levels in patients with CS can cause carcinoid syndrome diarrhea (CSD) which is characterized by increased motility in the gastrointestinal (GI) tract and reduced absorption of water and nutrients.2 Patients with CSD can experience multiple, urgent, loose and watery stools several times a day. CSD can have a prominent effect on patients' quality of life. Patients with CSD may avoid social activities and have increased fatigue, anxiety, and weight loss.2,3 About Xermelo Xermelo is the first and only approved oral therapy for CSD.4 Xermelo targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within metastatic neuroendocrine tumor (mNET) cells, providing an additional treatment option for patients suffering from CSD. Xermelo is approved in the United States, the European Union, and certain additional countries for the treatment of CSD in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy. Patients and healthcare professionals with questions about Xermelo should call 1-844-334-4035 or visit . IMPORTANT SAFETY INFORMATION ABOUT XERMELO: Indication Xermelo is a tryptophan hydroxylase inhibitor indicated for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy. Contraindications: XERMELO is contraindicated in patients with a history of a hypersensitivity reaction to telotristat. Reactions have included angioedema, rash and pruritis. Warnings and Precautions: XERMELO may cause constipation, which can be serious. Serious complications of constipation have been reported during clinical trials and post marketing with individual reports of intestinal perforation, obstruction, and fecaloma. 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: In a clinical trial of patients with carcinoid syndrome diarrhea and 4-12 bowel movements per day, the most common adverse reactions (≥5%) include nausea, headache, increased gamma-glutamyl transferase, depression, flatulence, decreased appetite, peripheral edema, and pyrexia. In a second clinical trial of patients with carcinoid syndrome diarrhea and less than 4 bowel movements per day, additional adverse reactions of abdominal pain and constipation were reported in ≥5% of patients. Drug Interactions: If necessary, consider increasing the dose of concomitant CYP3A4 and CYP2B6 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. Use in Special Populations: Xermelo is not recommended in patients with moderate and severe hepatic impairment. For more information, please see the full Prescribing Information for Xermelo. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. You can also contact TerSera Therapeutics at 1-844-334-4035 or [email protected]. About TerSera Therapeutics TerSera Therapeutics acquires, develops, and markets specialty pharmaceutical products with a focus on oncology and non-opioid pain. Its mission is to provide products which truly make a difference for patients. For more information about TerSera Therapeutics, please visit . About Carcinoid Cancer Foundation: The Carcinoid Cancer Foundation is the oldest nonprofit carcinoid and neuroendocrine cancer organization in the United States, founded in 1968. The mission of the foundation is to increase awareness and educate the general public and healthcare professionals regarding neuroendocrine tumors (NETs), to support NET cancer patients and their families, and to serve as patient advocates. Visit the CCF website for more information, . About Alira Health Alira Health is a global healthcare firm whose mission is to humanize healthcare and life sciences in partnership with patients. From development to medical care, we complement the expertise of our Pharma, Biotech, and MedTech clients with a full spectrum of services across their entire solutions lifecycle, including clinical operations, real-world evidence, and patient-centric technology offerings. Our integrated and multidisciplinary team of over 600 scientists, strategists, economists, clinicians, and biostatisticians collaborate across our North American, European, and Asian offices and advise 80% of the top 50 MedTech companies and 75% of the top 50 Pharma companies. More information may be obtained at . Media Contacts: References: Clement D, Ramage J, Srirajaskanthan R. Update on Pathophysiology, Treatment, and Complications of Carcinoid Syndrome. J Oncol. 2020 Jan 21;2020:8341426. Naraev BG, Halland M, Halperin DM, et al. Management of Diarrhea in Patients With Carcinoid Syndrome. Pancreas. 2019 Sep;48(8):961-972. Anthony L, Ervin C, Lapuerta P, Kulke MH, Kunz P, Bergsland E, Hörsch D, Metz DC, Pasieka J, Pavlakis N, Pavel M, Caplin M, et al. Understanding the Patient Experience with Carcinoid Syndrome: Exit Interviews from a Randomized, Placebo-controlled Study of Telotristat Ethyl. Clin Ther. 2017 Nov;39(11):2158-2168. XERMELO® (telotristat ethyl) Prescribing Information. TerSera Therapeutics LLC. Halperin DM, Shen C, Dasari A, Xu Y, et al. Frequency of carcinoid syndrome at neuroendocrine tumour diagnosis: a population-based study. Lancet Oncol. 2017 Apr;18(4):525-534. Xermelo® is a registered trademark of TerSera Therapeutics LLC or its affiliates. ©2022 TerSera Therapeutics LLC. All rights reserved. SOURCE TerSera Therapeutics LLC