• Thyroid cancer is the most rapidly increasing cancer diagnosis in the US with incidence tripling over the last 30 years
• Cabometyx is approved for renal cell carcinoma and hepatocellular carcinoma
• It has orphan drug status in the US for advanced hepatocellular carcinoma

Exelixis, Inc.  (Nasdaq: EXEL) today announced the initiation of a phase 3 pivotal trial (COSMIC-311) of single-agent cabozantinib in patients with radioiodine-refractory differentiated thyroid cancer (DTC) who have progressed after up to two prior vascular endothelial growth factor receptor (VEGFR)-targeted therapies. The co-primary endpoints for the trial are progression-free survival and objective response rate.
“Cabozantinib has demonstrated encouraging clinical activity in patients with radioiodine-refractory differentiated thyroid cancer in phase 1 and 2 studies, suggesting it may be a promising treatment option for patients who have progressed after prior VEGFR-targeting therapy,” said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. “We look forward to enrolling patients in this global trial to learn more about the potential of cabozantinib for this intractable form of thyroid cancer.”

“With the incidence of thyroid cancer increasing more rapidly than any other type of cancer in the U.S., and limited options available to patients whose disease has progressed following anti-VEGFR therapy, there is an urgent need for new treatments,”  said Marcia Brose, M.D., Ph.D., Associate Professor of Otorhinolaryngology: Head and Neck Surgery and Director of the Center for Rare Cancers and Personalized Therapy at the Abramson Cancer Center of the University of Pennsylvania, and principal investigator of the trial. “Given the positive results from earlier stage trials, we are eager to learn more from this phase 3 study about cabozantinib’s potential benefit in this patient population.”

Cabometyx is a tyrosine kinase inhibitor that  inhibits key receptors, including VEGFR, MET, and AXL, which are involved in normal and pathologic processes such as tumor growth, invasiveness, angiogenesis, and metastasis.  Cabometyx tablets are approved in the United States for the treatment of patients with advanced renal cell carcinoma (RCC). Cabometyx tablets are also approved in: the European Union, Norway, Iceland, Australia, Switzerland and South Korea for the treatment of advanced RCC in adults who have received prior VEGF-targeted therapy; in the European Union for previously untreated intermediate- or poor-risk advanced RCC; and in Canada for adult patients with advanced RCC who have received prior VEGF targeted therapy.

In March 2017, the FDA granted orphan drug designation to cabozantinib for the treatment of advanced hepatocellular carcinoma (HCC). In May 2018, the FDA accepted Exelixis’ supplemental New Drug Application for Cabometyx as a treatment for patients with previously treated HCC and assigned it a Prescription Drug User Fee Act action date of January 14, 2019. On March 28, 2018, Ipsen announced that the European Medicines Agency validated its application for a new indication for cabozantinib as a treatment for previously treated advanced HCC in the European Union; on September 20, 2018 the CHMP provided a positive opinion for Cabometyx as a monotherapy for the treatment of HCC in adults who have been previously treated with sorafenib. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan.

This year, an estimated 53,990 adults (13,090 men and 40,900 women) in the United States will be diagnosed with thyroid cancer. Thyroid cancer is the fifth most common cancer in women. It is the most common cancer in women 20 to 34. About 2% of cases occur in children and teens. It is the most rapidly increasing cancer diagnosis in the US with incidence tripling over the last 30 years.