Ipsen Opts-in to Join Exelixis With Ongoing Development of Cabometyx® for People Living With a Form of Thyroid Cancer, Based on Promising Interim Results

Regulatory News:

Ipsen (Euronext: IPN; ADR: IPSEY) today announced it has exercised its option to collaborate with Exelixis, Inc. (Exelixis) in the pivotal COSMIC-311 Phase III trial. COSMIC-311 is evaluating Cabometyx^® (cabozantinib) 60 mg versus placebo in people living with radioiodine-refractory differentiated thyroid cancer (DTC) who have progressed after up to two prior vascular endothelial growth factor receptor (VEGFR)-targeted therapies.² Radioactive iodine (RAI) is a treatment option for DTC when patients are at high risk of disease recurrence, have incompletely resected cancer, or distant metastases.³ Patients who develop RAI-refractory DTC, whereby they are resistant to RAI treatment, typically have a poor prognosis with an estimated survival of three to five years on average.⁴

Howard Mayer, Executive Vice President and Head of Research and Development at Ipsen, commented:

“A planned interim analysis of the COSMIC-311 Phase III trial has shown promising and clinically meaningful results in the use of Cabometyx in people living with radioiodine-refractory differentiated thyroid cancer who have progressed after prior therapy. We are delighted to build on our strong foundation and join Exelixis to further evaluate, and to work with regulatory authorities on, the potential of Cabometyx in a patient population who currently have limited treatment options.”

Results from the planned Phase III interim analysis of COSMIC-311 showed that the trial met the co-primary endpoint of demonstrating significant improvement in progression-free survival.¹ The detailed results from the analysis will be presented at the forthcoming ASCO Annual Meeting, taking place virtually from 4 to 8 June 2021.

Ipsen has an exclusive collaboration agreement with Exelixis for the commercialization of Cabometyx outside of the U.S. and Japan. Following the decision to opt-in to the COSMIC-311 trial, Ipsen gains access to the results to support potential future regulatory submissions in its territories.

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for Cabometyx in February 2021 as a potential treatment for people living with DTC who have progressed following prior therapy and who are RAI-refractory (if RAI is appropriate).

About radioiodine-refractory differentiated thyroid cancer

In 2020, over 580,000 new cases of thyroid cancer were diagnosed worldwide.⁵ Thyroid cancer is the ninth most commonly occurring cancer globally and incidence is three times higher in women than in men, with the disease representing one in every 20 cancers diagnosed among women.⁵ While cancerous thyroid tumors include differentiated, medullary and anaplastic forms, differentiated thyroid tumors make up about 90 to 95% of cases.^6,7 These include papillary, follicular and Hürthle cell cancer.^6,7 DTC is typically treated with surgery, followed by ablation of the remaining thyroid tissue with RAI, but approximately 5 to 15% of cases are resistant to RAI treatment.⁸ Patients who develop RAI-refractory DTC have a poor prognosis with an estimated survival of three to five years on average.⁴

About the COSMIC-311 trial

COSMIC-311 is a multicenter, randomized, double-blind, placebo-controlled Phase III trial that aimed to enroll approximately 300 patients at 150 sites globally.² Patients were randomized in a 2:1 ratio to receive either Cabometyx 60 mg or placebo once daily.² The co-primary endpoints are progression-free survival and objective response rate, evaluated by a blinded independent radiology committee. Additional endpoints include safety, overall survival and quality of life.² More information about this trial is available at ClinicalTrials.gov.

About Cabometyx (cabozantinib)

Cabometyx is currently approved in 58 countries, including in the E.U., the U.K., Norway, Iceland, Australia, New Zealand, Switzerland, South Korea, Canada, Brazil, Taiwan, Hong Kong, Singapore, Macau, Jordan, Lebanon, the Russian Federation, Ukraine, Turkey, the U.A.E., Saudi Arabia, Serbia, Israel, Mexico, Chile, Peru, Panama, Guatemala, Dominican Republic, Ecuador, Thailand and Malaysia for the treatment of advanced renal cell carcinoma (RCC) in adults who have received prior VEGF-targeted therapy; in the E.U., the U.K., Norway, Iceland, Canada, Australia, Brazil, Taiwan, Hong Kong, Singapore, Lebanon, Jordan, the Russian Federation, Ukraine, Turkey, the U.A.E., Saudi Arabia, Israel, Mexico, Chile, Peru, Panama, Guatemala, the Dominican Republic, Ecuador, Thailand and Malaysia for previously untreated intermediate- or poor-risk advanced RCC; and in the E.U., the U.K., Norway, Iceland, Canada, Australia, Switzerland, Saudi Arabia, Serbia, Israel, Taiwan, Hong Kong, South Korea, Singapore, Jordan, the Russian Federation, Ukraine, Turkey, Lebanon, the U.A.E., Peru, Panama, Guatemala, Chile, the Dominican Republic, Ecuador, Thailand and Malaysia for hepatocellular carcinoma (HCC) in adults who have previously been treated with sorafenib. In the E.U., Cabometyx is also approved in combination with nivolumab as first line treatment for people living with advanced RCC.

The detailed recommendations for the use of Cabometyx are described in the Summary of Product Characteristics (SmPC) and in the U.S. Prescribing Information (PI).

Ipsen has exclusive rights for the commercialization of Cabometyx outside of the U.S. and Japan. Cabometyx is marketed by Exelixis in the U.S. and by Takeda Pharmaceutical Company Limited in Japan. Cabometyx is a registered trademark of Exelixis.

About Ipsen

Ipsen is a global mid-size biopharmaceutical company with a focus on transformative medicines in Oncology, Rare Disease and Neuroscience. Ipsen also has a well-established Consumer Healthcare business. With total sales over €2.5 billion in 2020, Ipsen sells more than 20 drugs in over 110 countries, with a direct commercial presence in more than 30 countries. Ipsen’s R&D is focused on its innovative and differentiated technological platforms located in the heart of the leading biotechnological and life sciences hubs (Paris-Saclay, France; Oxford, UK; Cambridge, US; Shanghai, China). The Group has about 5,700 employees worldwide. Ipsen is listed in Paris (Euronext: IPN) and in the United States through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information on Ipsen, visit www.ipsen.com

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References

1. Exelixis announces cabozantinib significantly improved progression-free survival of COSMIC-311 Phase 3 pivotal trial in patients with previously treated radioiodine-refractory differentiated thyroid cancer. News release. Exelixis, 21 December 2020. Last accessed: April 2021. Available at: https://ir.exelixis.com/news-releases/news-release-details/exelixis-announces-cabozantinib-significantly-improved
2. Brose MS. et al. A phase 3 (COSMIC-311), randomized, double-blind, placebo-controlled study of cabozantinib in patients with radioiodine (RAI)-refractory differentiated thyroid cancer (DTC) who have progressed after prior VEGF-targeted therapy. J Clin Oncol. 2019;37(suppl 15):TPS6097. doi: 10.1200/JCO.2019.37.15_suppl.TPS6097
3. Cancer.net. ASCO Answers: Thyroid Cancer Fact Sheet. Last accessed: April 2021. Available at: https://www.cancer.net/sites/cancer.net/files/asco_answers_thyroid.pdf.
4. Fugazzola L. et al. 2019 European Thyroid Association Guidelines for the Treatment and Follow-Up of Advanced Radioiodine-Refractory Thyroid Cancer. Eur Thyroid J. 2019;8:227–245. doi: 10.1159/000502229.
5. Sung. H et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA: A Cancer Journal For Clinicians. doi: 10.3322/caac.21660.
6. Cancer.Net. ASCO. Thyroid Cancer: Introduction. Last accessed: April 2021. Available at: https://www.cancer.net/cancer-types/thyroid-cancer/introduction
7. Chen D. et al. Innovative analysis of distant metastasis in differentiated thyroid cancer. Oncol Lett 19: 1985-1992, 2020. doi: 10.3892/ol.2020.11304.
8. Worden F. Treatment strategies for radioactive iodine-refractory differentiated thyroid cancer. Ther Adv Med Oncol. 6:267–279. doi: 10.1177/1758834014548188.

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