ESMO 2025: new data reinforces breadth and depth of Ipsen’s growing oncology pipeline and portfolio

• Eight Ipsen and partner abstracts demonstrate how bold science is leading to new standards of cancer care and meaningful progress for patients across tumor types
• Subgroup analysis from the landmark Phase III CABINET trial shows improved progression-free survival with Cabometyx^® versus placebo in advanced lung and thymic neuroendocrine tumors¹
• Ipsen’s pioneering early science is reflected in two further abstracts, introducing for the first time two novel MAP-kinase inhibitors now progressing to Phase I/II trials^2,3

PARIS, FRANCE, 13 October 2025 – Ipsen announced today data to be presented at the upcoming European Society of Medical Oncology (ESMO) Symposium on 17-21 October 2025 for rare cancers and complex tumors, offering opportunities to change the course of disease for people with limited options.

Presentations include a subgroup analysis from the landmark Phase III CABINET trial of Cabometyx® (cabozantinib) versus placebo in people living with advanced, previously treated advanced pancreatic neuroendocrine tumors (pNETs) or extra-pancreatic neuroendocrine tumors (epNETs). Consistent with the results of the overall trial, Cabometyx demonstrated improved progression-free survival versus placebo for patients in the epNET cohort who had confirmed lung or thymic neuroendocrine tumors (NETs).¹ These data follow the recent approval of Cabometyx in Europe for previously treated advanced NETs, based on the findings from the CABINET Phase III trial as presented at the 2024 ESMO Congress⁴ and simultaneously published in the New England Journal of Medicine.⁵ Where previously no approved options have existed for lung and thymic NETs upon disease progression, these latest subgroup analysis data reinforce Cabometyx as an effective treatment option (presentation #1712P).^1,6,7

Additionally, two first-in-human studies will be shown for the first time, showcasing the strong potential of Ipsen’s biomarker-driven approach to pipeline expansion. Translating newly added early development programs to therapeutic opportunities for patients, Ipsen is progressing two novel mitogen-activated protein kinase (MAPK) inhibitors to Phase I/II trials. Reinforced by pre-clinical data and strength of expertise in the MAPK pathway, IPN01194 and IPN01195 present the opportunity to reduce tumor growth and overcome treatment resistance in solid tumors, challenges few other treatments have historically addressed.^2,3 

• IPN01194 is an investigational ERK1/2 inhibitor entering a Phase I/IIa study for people living with solid tumors. Inhibition of ERK1/2 represents a promising opportunity, as the final kinase in the MAPK pathway, to inhibit MAPK signaling (presentation 1020eTiP).²
• IPN01195 is a novel RAF inhibitor developed for the treatment of advanced MAPK mutant solid tumors, an area of significant unmet need. (presentation 1026eTiP).³

“Our presence at ESMO reflects Ipsen’s bold approach to innovation – harnessing our deep expertise to tackle the toughest challenges in oncology where people have had so few treatment options,” said Sandra Silvestri, MD, PhD, EVP and Chief Medical Officer, Ipsen. “When we see an opportunity to bring best and first-in-class treatments, we give them the best possible chance of success, pursuing the most pressing questions and generating robust data to translate these opportunities into longer and more fulfilling lives for people living with cancer.”

About Cabometyx

Cabometyx is a small molecule that inhibits multiple receptor tyrosine kinases, including VEGFRs, MET, RET and the TAM family (TYRO3, MER, AXL).⁸ These receptor tyrosine kinases are involved in both normal cellular function and pathological processes such as oncogenesis, metastasis, tumor angiogenesis (the growth of new blood vessels that tumors need to grow), drug resistance, immune modulation, and maintenance of the tumor microenvironment.^8,9,10,11

Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of Cabometyx outside of the U.S. and Japan. Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited (Takeda) for the commercialization and further clinical development of Cabometyx for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize Cabometyx in the U.S.

In over 65 countries outside of the United States and Japan, including in the European Union, Cabometyx is currently approved in several indications including:⁹

• Monotherapy for advanced renal cell carcinoma (aRCC).
  • as first-line treatment of adults with intermediate- or poor-risk disease.
  • in adults following prior VEGFR-targeted therapy.
• A combination with nivolumab for the first-line treatment of aRCC in adults.
• Monotherapy for the treatment of adults living with locally advanced or metastatic differentiated thyroid carcinoma, refractory or not eligible to radioactive iodine who have progressed during or after prior systemic therapy.
• Monotherapy for the treatment of hepatocellular carcinoma in adults who have previously been treated with sorafenib.
• Monotherapy for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues.

About CABINET (Alliance A021602)

CABINET (randomized, double-blinded Phase III trial of CABozantinib versus placebo In patients with advanced Neuroendocrine Tumors after progression on prior therapy) is sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health in the U.S., and is being led and conducted by the NCI-funded Alliance for Clinical Trials in Oncology with participation from the NCI-funded National Clinical Trials Network, as part of Exelixis’ collaboration through a Cooperative Research and Development Agreement with the NCI’s Cancer Therapy Evaluation Program.

The multicenter Phase III CABINET pivotal trial enrolled a total of 298 patients in the U.S. at the time of the analysis. Patients were randomized 2:1 to Cabometyx or placebo in two separately powered cohorts. Each cohort was randomized separately and had its own statistical analysis plan. The epNET cohort included patients with the following primary tumor sites: gastrointestinal tract, lung, unknown primary and other organs Patients must have had measurable disease per RECIST 1.1 criteria and must have experienced disease progression or intolerance after at least one U.S. Food and Drug Administration-approved line of prior systemic therapy other than somatostatin analogues. The primary endpoint in each cohort was PFS per RECIST 1.1 by retrospective blinded independent central review. Upon confirmation of disease progression, patients were unblinded, and those receiving placebo were permitted to cross over to open-label therapy with Cabometyx. Secondary endpoints included overall survival, objective response rate and safety. More information about this trial is available at ClinicalTrials.gov.

About IPN01194

IPN01194 is a next generation investigational MAPK inhibitor which targets the ERK1/2 proteins within the MAPK pathway. ERK1/2 are the proteins at the end of the MAPK pathway which regulates cell proliferation, survival and differentiation.

Ipsen acquired the worldwide rights to IPN01194 from AGV discovery as an early development program in 2022. In 2024, the IPN01194 progressed into Phase I/IIa clinical trials in people living with metastatic melanoma, colorectal cancer, pancreatic adenocarcinoma or head and neck squamous cell carcinoma, assessing the appropriate dosage, safety and effectiveness.

About IPN01195

IPN01195 is a next-generation investigational MAPK inhibitor which targets multiple RAF proteins within the MAPK pathway, including dimeric forms. Broad inhibition of RAF blocks signaling within the MAPK pathway caused by mutations which can lead to uncontrolled cell growth.

Ipsen acquired worldwide rights to IPN01195 as part of the option agreement entered into with IRICoR and Université de Montréal for a discovery-stage oncology program in 2020. In 2025, IPN01195 progressed to Phase I/II first in human studies to assess the safety, tolerability, pharmacokinetic, pharmacodynamic and antitumor activity for people with advanced solid tumors.

About Ipsen

We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.

Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries.

Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com.

Ipsen Contacts

Investors
Henry Wheeler   henry.wheeler@ipsen.com   +33 7766471149
Khalid Deojee   khalid.deojee@ipsen.com   +33 666019526

Media
Sally Bain sally.bain@ipsen.com +1 8573200517
Anne Liontas anne.liontas.ext@ipsen.com +33 0767347296

Disclaimers and/or forward-looking statements
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¹ Wolin et al. Efficacy and Safety of Cabozantinib (CABO) for Advanced Lung and Thymic Neuroendocrine Tumors (NET) after Progression on Prior Therapy: Subgroup Analysis of Phase 3 CABINET Trial (Alliance A021602). As presented at ESMO Congress 2025 during the ‘Poster lunch session’ Monday 20 October at 12:00 p.m. CEST Berlin, Germany.
² Tabernero et al. A phase I/IIa first-in-human, open label study to investigate ERK1/2 inhibitor IPN01194 in adults with advanced solid tumours. As published as an ePoster, Developmental therapeutics, ESMO Congress 2025 Berlin, Germany.
³ McKean et al. Phase I/II first-in-human (FIH) study to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD) and antitumour activity of IPN01195 as single agent in adult participants with advanced solid tumours. As published as an ePoster, Developmental therapeutics, ESMO Congress 2025 Berlin, Germany.
⁴ Chan et al. Cabozantinib Versus Placebo for Advanced Neuroendocrine Tumors (NET) after Progression on Prior Therapy (CABINET Trial/Alliance A021602): Updated Results Including Progression Free-Survival (PFS) by Blinded Independent Central Review (BICR) and Subgroup Analyses. As presented at ESMO Congress 2024 during the ‘Proffered Paper: NETs and Endocrine Tumors at 2:45 p.m. CEST Barcelona, Spain
⁵ Chan et al. Phase 3 Trial of Cabozantinib to Treat Advanced Neuroendocrine Tumors. 2024 New England Journal of Medicine. DOI: 10.1056/NEJMoa2403991
⁶ Pavel M, et al. Gastroenteropancreatic neuroendocrine neoplasms: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2020;31(7):844-860.
⁷ Baudin E, et al. Lung and thymic carcinoids: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2021 Nov;32(11):1453-1455.
⁸ El-Khoueiry A. et al., Cabozantinib: An evolving therapy for hepatocellular carcinoma. Cancer Treatment Reviews. 2021 Jul;98:102221. DOI: 10.1016/j.ctrv.2021.102221.
⁹ European Medicines Agency. Cabometyx® (cabozantinib) EU Summary of Product Characteristics. Available from: https://www.ema.europa.eu/en/documents/product-information/cabometyx-epar-product-information_en.pdf. Accessed September 2025.
¹⁰ Yakes M. et al., Cabozantinib (XL184), a novel MET and VEGFR2 inhibitor, simultaneously suppresses metastasis, angiogenesis, and tumor growth. Mol Cancer Ther. 2011;10:2298–2308. DOI: 10.1158/1535-7163.MCT-11-0264
¹¹ Hsu et al., AXL and MET in Hepatocellular Carcinoma: A Systematic Literature Review. Liver Cancer 2021 DOI: 10.1159/000520501