AXL inhibitors(AXL receptor tyrosine kinase inhibitors), RET inhibitors(Tyrosine-protein kinase receptor RET inhibitors), ROS1 inhibitors(Proto-oncogene tyrosine-protein kinase ROS inhibitors)

Therapeutic Areas

NeoplasmsDigestive System DisordersEndocrinology and Metabolic Disease+ [11]

Active Indication

Well Differentiated Pancreatic Endocrine TumorExtra-pancreatic neuroendocrine tumorNeuroendocrine tumor of pancreas+ [191]

Inactive Indication

Advanced Bile Duct CarcinomaAdvanced breast cancerCarcinoma of extrahepatic bile duct+ [40]

Originator Organization

Exelixis, Inc.

Active Organization

Xspray Pharma ABIpsen Pharma SAS

Ipsen SA

+ [19]

Inactive Organization

University of Washington

EMD Serono, Inc.

Jiangsu Hansoh Pharmaceutical Co., Ltd.

License Organization

Ipsen SA

Takeda Pharmaceutical Co., Ltd.

Chugai Pharmaceutical Co., Ltd.

+ [7]

Drug Highest PhaseApproved

First Approval Date

United States (29 Nov 2012),

Thyroid Cancer, Medullary

RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States)

Structure/Sequence

Molecular FormulaC32H30FN3O10

InChIKeyHFCFMRYTXDINDK-WNQIDUERSA-N

CAS Registry1140909-48-3

271

Clinical Trials associated with Cabozantinib (s)-Malate

NCT06811116

/ RecruitingPhase 1/2IIT

A Phase I/II Trial of Sapanisertib in Combination With Cabozantinib in β-Catenin-Mutated Hepatocellular Carcinoma

This phase I/II trial studies the side effects and best dose of sapanisertib when given together with cabozantinib, and to see how well they work in treating patients with liver cancer that has spread from where it first started to other places in the body (metastatic) and contains a mutation (change) in the β-catenin gene. Sapanisertib and cabozantinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving sapanisertib and cabozantinib together may work better than giving cabozantinib alone in treating β-catenin-mutated metastatic hepatocellular carcinoma.

Start Date10 Feb 2026

Sponsor / Collaborator

National Cancer Institute

NCT07077161

/ Not yet recruitingPhase 2IIT

Cabozantinib Dose Skipping as an Alternative to Dose Reductions

The goal of this study is to determine if an alternative cabozantinib dosing regimens results in a similar drug exposure compared to the standard regimens in patients with metastatic renal cell carcinoma (mRCC). All dosages of cabozantinib (20 ,40, 60mg) have the same price, and cabozantinib is eliminated very slowly by the body. This means that using fewer tablets could potentially lead to cost savings, while remaining equally effective.
The main questions it aims to answer are:
* Is the drug exposure from our experimental regimens similar to the standard dosing regimens?
* Do the experimental regimens affect the number of side effect and the patients' quality of life?
Participants will:
* Take cabozantinib according to either the experimental or standard dosage regimen for 4 weeks
* After 4 weeks: visit the clinic to collect some blood samples and complete two questionnaires.
* 1 and 3 days after this visit: visit the clinic to collect 1 blood sample.
* The day after the hospital visit: switch to the other dosing regimen and according to that regimen for another 4 weeks.
* After 4 weeks: visit the clinic to collect some blood samples and complete two questionnaires
* 1 and 3 days after this visit: visit the clinic to collect 1 blood sample.

Start Date01 Sep 2025

Sponsor / Collaborator-

NCT06900595

/ Not yet recruitingPhase 2IIT

A Phase II Study of Cabozantinib in Combination With Cemiplimab (Cabo-Cemiplimab) Versus Cabozantinib Alone in Adolescents and Adults With Advanced Adrenocortical Cancer

This phase II trial compares the effect of giving cabozantinib with or without cemiplimab in patients with adrenocortical cancer that has spread to nearby tissue or lymph nodes (locally advanced), and that cannot be removed by surgery (unresectable) or that has come back after a period of improvement (recurrent) or that has spread from where it first started (primary site) to other places in the body (metastatic). Cabozantinib is in a class of medications called tyrosine kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply, which may help keep cancer cells from growing. Immunotherapy with monoclonal antibodies, such as cemiplimab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving cabozantinib with cemiplimab may kill more tumor cells in patients with locally advanced unresectable or recurrent/metastatic adrenocortical cancer.

Start Date31 Jul 2025

Sponsor / Collaborator

National Cancer Institute

100 Clinical Results associated with Cabozantinib (s)-Malate

100 Translational Medicine associated with Cabozantinib (s)-Malate

100 Patents (Medical) associated with Cabozantinib (s)-Malate

2,707

Literatures (Medical) associated with Cabozantinib (s)-Malate

01 Sep 2025·EUROPEAN JOURNAL OF PHARMACOLOGY

Investigating the ‘RT-PK’ phenomenon: Effects of X-ray radiation on cabozantinib metabolism

Article

Author: Zhang, Junxia ; Fan, Huirong ; Liang, Bohan ; Ma, Jiaojiao ; Shang, Yan ; Teng, Yunhua ; Li, Yanjie ; Wang, Lingmei ; Dong, Shiqi ; Zhu, Huazhen

BACKGROUND AND PURPOSE:

This study investigated whether the "RT-PK" phenomenon, referring to radiation therapy's effects on the pharmacokinetics of chemotherapeutic agents, occurs with cabozantinib, a multikinase inhibitor, after radiotherapy and investigated the underlying mechanisms.

METHOD:

A quantitative liquid chromatography-tandem mass spectrometry method was developed for cabozantinib. Sprague-Dawley (SD) rats were irradiated with X-rays, and changes in cabozantinib distribution and concentration in the blood and excreta were monitored. The RT-PK phenomenon was confirmed, and further studies examined liver injury and alterations in primary metabolic enzymes in rats and HepG2 cells using hematoxylin and eosin staining, flow cytometry, Western blot, quantitative reverse transcription polymerase chain reaction, and serum biochemical analysis. The mRNA expression of IL6/STAT3 and PXR was also determined.

RESULTS:

X-ray exposure increased the area under the drug-time curve (AUC_0-t) of cabozantinib by 3.6-fold, and excretion of the drug in the feces was 73 % lower in irradiated rats. Hematoxylin and eosin staining showed hepatocyte and interstitial edema, and serum Alanine transaminase and aspartate transaminase contents were decreased. Radiation induced redox imbalance, increased reactive oxygen species and apoptotic markers, and reduced superoxide dismutase and GSH/GSSG contents. As indicated by western blotting and RT-qPCR, the activation of the IL-6/STAT3 pathway reduced PXR expression, leading to decreased levels of CYP450 3A4, the key enzyme that metabolizes cabozantinib.

CONCLUSIONS:

Radiation increased reactive oxygen species, activated the IL-6/STAT3 pathway, and inhibited PXR, thereby reducing CYP450 3A4 expression. These changes led to altered cabozantinib metabolism and the "RT-PK" phenomenon.

01 Aug 2025·Clinical Genitourinary Cancer

Cabozantinib in the First-Line Treatment of Patients With Metastatic Renal Cell Carcinoma, Real-World Data From the Czech Republic and Poland: ICARO-RC Project

Article

Author: Lacek, Michal ; Poprach, Alexandr ; Domański, Piotr ; Bezděková, Monika ; Kopecky, Jindrich ; Richter, Igor ; Kolaríková, Eva ; Sikora-Kupis, Bożena ; Pinto, Alvaro ; Studentova, Hana ; Koloušková, Ivana ; Buchler, Tomas ; Kucharz, Jakub ; Fiala, Ondrej ; Darewicz, Marta

INTRODUCTION:

The treatment of metastatic renal cell carcinoma (RCC) has changed dramatically in the last few years, with different options being available, and with no data comparing these new systemic therapies. Therefore, the value of real-world outcomes (RWO) becomes of great interest to understand the effectiveness of these treatments. Here we analyze the outcome of metastatic RCC patients treated with first-line cabozantinib in a retrospective cohort from the Czech Republic and Poland METHODS: Patients with metastatic RCC treated with first-line cabozantinib in the Czech Republic and Poland were included in a retrospective fashion. Data were collected regarding progression-free survival (PFS), overall survival (OS), response rate and toxicity, with a focus in several subgroups of interest.

RESULTS:

We identified 146 patients, the majority of them (80.8%) with clear cell RCC (ccRCC). The median OS was 14.7 months, and the median PFS 8.2 months, with a response rate of 30.3%. CTCAE v3.0 grade 3+4 toxicity was presented in 34.2% of patients. The efficacy of Cabozantinib was maintained regardless of histologic subtype and the presence of sarcomatoid component, although PFS and OS data were numerically better for nonclear cell RCC. Bone and liver metastases were confirmed as independent factor for poor survival in the multivariate analysis.

CONCLUSIONS:

In our series, Cabozantinib demonstrated its activity in RCC patients in a RWO setting. Our data can be considered comparable with what has been seen in randomised clinical trials, considering the inherent bias present in RWO studies.

01 Aug 2025·JOURNAL OF PATHOLOGY

Involvement of KEAP1/NRF2 pathway in non‐BRAF mutated squamous cell carcinoma of the thyroid

Article

Author: Fagman, Henrik ; Dahr, Niklas ; Nilsson, Mikael ; Nilsson, Jonas A ; Elias, Erik ; Nilsson, Lisa M ; Dzanan, Jozefina J ; Carlsson, Therese ; Muth, Andreas ; Schoultz, Elin ; Muhammad, Ghayeb ; Sayin, Volkan I ; Andersson, Ellinor ; Dahlberg, Jakob

Abstract:

Squamous cell carcinoma (SCC) of the thyroid is a rare tumor that is classified as an anaplastic thyroid cancer (ATC) due to its similar unresponsiveness to chemoradiotherapy and an outstandingly poor prognosis. Due to its rarity, current knowledge about this tumor is mostly based on single‐case reports. The tumor‐cell‐origin and molecular pathogenesis remain unclear, although the presence of BRAF mutations in some cases suggest it may evolve from papillary thyroid carcinoma (PTC). Here we provide direct evidence of derivation of SCC of the thyroid from PTC, based on a unique combination of likely pathogenic mutations in KEAP1, STK11 (LKB1), and RB1 found in both tumor components, along with loss of one copy of chromosome 11 and additional somatic mutations in the SCC tumor. Transdifferentiation from PTC to SCC was also evident by immunohistochemistry. Out of eight attempted patient‐derived xenografts (PDX) from advanced thyroid cancers, only one derived from thyroid SCC successfully engrafted in immunodeficient NOG mice. Untreated PDXs showed high Ki67 indices but did not reproduce the conspicuous stromal invasion of CDH1low/SNAI2+/CDH2+ cells that characterized the primary tumor. Based on the mutation profile (NFE2L2, PIK3CA, CDKN2A, and TP53), experiments were designed to evaluate targeted drug therapy using third‐passage PDX transplants. The combination of TRK and PI3K inhibitors, cabozantinib and GDC‐0326, additively reduced PDX growth by nearly 90%. Remarkably, CB‐839 (telaglenastat), a glutaminase inhibitor targeting metabolic rewiring downstream of NRF2 activation, was equally effective. Both combined treatment with cabozantinib + GDC‐0326 and CB‐839 monotherapy diminished the expression of NQO1, an NRF2 transcriptional target, in tumor cells. Glutaminase inhibition further promoted squamous differentiation in engrafted tumors. Both investigated SCC tumors were negative for BRAFV600E or any other common driver mutation of thyroid cancer. Collectively, these findings indicate that aberrant activation of the KEAP1/NRF2 pathway due to somatic mutations is a previously unrecognized feature of thyroid SCC and suggest that glutaminase inhibition may serve as a potential therapeutic option for this subgroup of ATC patients. © 2025 The Author(s). The Journal of Pathology published by John Wiley & Sons Ltd on behalf of The Pathological Society of Great Britain and Ireland.

493

News (Medical) associated with Cabozantinib (s)-Malate

31 Jul 2025

·www.ipsen.com

Ipsen delivers strong results in the first half of 2025 and upgrades its full-year guidance

H1 total sales growth of 11.4% at CER1, or 9.7% as reported, driven by the three therapeutic areas: 95.7% in Rare Disease, 9.7% in Neuroscience, and 6.4% in Oncology H1 core operating income of €656m, growing by 21.9% as reported, with a core operating margin of 36.0% of total sales, increasing by 3.6 points Upgraded FY 2025 financial guidance2: total-sales growth greater than 7.0% at CER (prior guidance: greater than 5.0% at CER); core operating margin greater than 32.0% of total sales (prior guidance: greater than 30%) Pipeline progression including regulatory filing of tovorafenib in Europe, and initiation of a Phase II trial of LANT3 (IPN10200) in cervical dystonia European Commission approval on 23 July 2025 of Cabometyx® in advanced neuroendocrine tumors (NETs), the sixth indication Key upcoming milestones with the pivotal FALKON trial results for fidrisertib in FOP4 and the Proof-of-Concept data readout for the LANT in aesthetics PARIS, FRANCE, 31 July 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the first half of 2025. “Our half year results reflect continued strong momentum, with growth across all three therapeutic areas, particularly in our rare liver disease franchise, which is expanding rapidly and progressing well,” said David Loew, Chief Executive Officer, Ipsen. “Building on that performance, I am pleased to increase our full year guidance in terms of sales and profitability.” “I’m also delighted to report robust progression in our pipeline and portfolio, including the recent European Commission approval of Cabometyx® in advanced neuroendocrine tumors, an area where Ipsen has a strong legacy. In the second half of the year, we are anticipating the readout of our pivotal study of fidrisertib in fibrodysplasia ossificans progressiva and the proof-of-concept trial of our long-acting neurotoxin in aesthetics. Our focused strategy, our culture of excellence in execution and our commitment to science with purpose position us to provide a positive impact for patients and society.” Full-year 2025 guidance Based on the strong performance in the first half, Ipsen upgrades its financial guidance for 2025: Total-sales growth greater than 7.0%, at CER. Based on the average level of exchange rates in June 2025, an adverse impact on total sales of around 2% from currencies is expected Core operating margin greater than 32.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities Guidance includes a negative impact on Somatuline sales due to a potential increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions. Pipeline update since Q1 2025 In May 2025, Ipsen presented data on Iqirvo® (elafibranor) from the Phase II ELMWOOD study at the European Association for the Study of the Liver congress. It showed a favorable safety profile and demonstrated dose-dependent efficacy over 12 weeks for people living with PSC5, a rare liver disease with no approved treatment options. In June 2025, Ipsen initiated a Phase II study of LANT (IPN10200), in cervical dystonia. This marked the fourth study in the global long-acting neurotoxin development plan in therapeutics and aesthetics indications. On 23 July 2025, Ipsen received European Commission approval for Cabometyx® (cabozantinib) in previously treated advanced neuroendocrine tumors. This approval was based on positive outcomes from the Phase III CABINET trial. Consolidated financial statements The Board of Directors approved the condensed consolidated financial statements on 30 July 2025. The Company’s auditors performed a limited review of the H1 2025 condensed consolidated financial statements. The interim financial report, with regards to the regulated information, will be available on ipsen.com in due course, under the Reports and Accounts tab in the Investor Relations section. Conference call A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its year-to-date and third-quarter sales update on 22 October 2025. Notes All financial figures are in € millions (€m). The performance shown in this announcement covers the six-month period to 30 June 2025 (H1 2025) and the three-month period to 30 June 2025 (Q2 2025), compared to the six-month period to 30 June 2024 (H1 2024) and the three-month period to 30 June 2024 (H1 2024), respectively, unless stated otherwise. Commentary is based on the performance in H1 2025, unless stated otherwise. 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 80 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 Khalid Deojee khalid.deojee@ipsen.com +33 6 66 01 95 26 Media Sally Bain sally.bain@ipsen.com +1 857 32 00 517 Anne Liontas anne.liontas@ipsen.com +33 7 67 34 72 96 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period. 2 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions. 3 Long-acting neurotoxin. 4 Fibrodysplasia ossificans progressiva. 5 Primary sclerosing cholangitis Attachment H1 total sales growth of 11.4% at CER1, or 9.7% as reported, driven by the three therapeutic areas: 95.7% in Rare Disease, 9.7% in Neuroscience, and 6.4% in Oncology H1 core operating income of €656m, growing by 21.9% as reported, with a core operating margin of 36.0% of total sales, increasing by 3.6 points Upgraded FY 2025 financial guidance2: total-sales growth greater than 7.0% at CER (prior guidance: greater than 5.0% at CER); core operating margin greater than 32.0% of total sales (prior guidance: greater than 30%) Pipeline progression including regulatory filing of tovorafenib in Europe, and initiation of a Phase II trial of LANT3 (IPN10200) in cervical dystonia European Commission approval on 23 July 2025 of Cabometyx® in advanced neuroendocrine tumors (NETs), the sixth indication Key upcoming milestones with the pivotal FALKON trial results for fidrisertib in FOP4 and the Proof-of-Concept data readout for the LANT in aesthetics PARIS, FRANCE, 31 July 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the first half of 2025. “Our half year results reflect continued strong momentum, with growth across all three therapeutic areas, particularly in our rare liver disease franchise, which is expanding rapidly and progressing well,” said David Loew, Chief Executive Officer, Ipsen. “Building on that performance, I am pleased to increase our full year guidance in terms of sales and profitability.” “I’m also delighted to report robust progression in our pipeline and portfolio, including the recent European Commission approval of Cabometyx® in advanced neuroendocrine tumors, an area where Ipsen has a strong legacy. In the second half of the year, we are anticipating the readout of our pivotal study of fidrisertib in fibrodysplasia ossificans progressiva and the proof-of-concept trial of our long-acting neurotoxin in aesthetics. Our focused strategy, our culture of excellence in execution and our commitment to science with purpose position us to provide a positive impact for patients and society.” Full-year 2025 guidance Based on the strong performance in the first half, Ipsen upgrades its financial guidance for 2025: Total-sales growth greater than 7.0%, at CER. Based on the average level of exchange rates in June 2025, an adverse impact on total sales of around 2% from currencies is expected Core operating margin greater than 32.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities Guidance includes a negative impact on Somatuline sales due to a potential increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions. Pipeline update since Q1 2025 In May 2025, Ipsen presented data on Iqirvo® (elafibranor) from the Phase II ELMWOOD study at the European Association for the Study of the Liver congress. It showed a favorable safety profile and demonstrated dose-dependent efficacy over 12 weeks for people living with PSC5, a rare liver disease with no approved treatment options. In June 2025, Ipsen initiated a Phase II study of LANT (IPN10200), in cervical dystonia. This marked the fourth study in the global long-acting neurotoxin development plan in therapeutics and aesthetics indications. On 23 July 2025, Ipsen received European Commission approval for Cabometyx® (cabozantinib) in previously treated advanced neuroendocrine tumors. This approval was based on positive outcomes from the Phase III CABINET trial. Consolidated financial statements The Board of Directors approved the condensed consolidated financial statements on 30 July 2025. The Company’s auditors performed a limited review of the H1 2025 condensed consolidated financial statements. The interim financial report, with regards to the regulated information, will be available on ipsen.com in due course, under the Reports and Accounts tab in the Investor Relations section. Conference call A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its year-to-date and third-quarter sales update on 22 October 2025. Notes All financial figures are in € millions (€m). The performance shown in this announcement covers the six-month period to 30 June 2025 (H1 2025) and the three-month period to 30 June 2025 (Q2 2025), compared to the six-month period to 30 June 2024 (H1 2024) and the three-month period to 30 June 2024 (H1 2024), respectively, unless stated otherwise. Commentary is based on the performance in H1 2025, unless stated otherwise. 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 80 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 Khalid Deojee khalid.deojee@ipsen.com +33 6 66 01 95 26 Media Sally Bain sally.bain@ipsen.com +1 857 32 00 517 Anne Liontas anne.liontas@ipsen.com +33 7 67 34 72 96 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period. 2 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions. 3 Long-acting neurotoxin. 4 Fibrodysplasia ossificans progressiva. 5 Primary sclerosing cholangitis Attachment Cabometyx® approved in the EU for previously treated advanced neuroendocrine tumors Ipsen announces changes to its Executive Committee Ipsen receives positive CHMP opinion for Cabometyx® in previously treated advanced neuroendocrine tumors Late-breaking analysis demonstrates characteristics associated with long-term overall survival with Onivyde® regimen in metastatic pancreatic adenocarcinoma Late-breaking exploratory data highlights the impact of IQIRVO® (elafibranor) on fatigue and provides mechanistic insights into anti-inflammatory and symptom-related effects in patients with primary biliary... Ipsen appoints Laura Réveillon as EVP, Strategy & Transformation Late-breaking elafibranor primary sclerosing cholangitis (PSC) data demonstrates favorable safety profile and significant efficacy in second potential rare liver disease indication Ipsen delivers strong sales in the first quarter 2025 and confirms its full-year guidance Ipsen publishes its 2024 Universal Registration Document Ipsen announces issuance of €500 million inaugural Rated Public Bond You are now leaving the Ipsen group website. To continue, please click on Continue? These cookies are necessary for the website to function and cannot be switched off in our systems. They are usually only set in response to actions made by you which amount to a request for services, such as setting your privacy preferences, logging in or filling in forms. You can set your browser to block or alert you about these cookies, but some parts of the site will not then work. 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Clinical ResultPhase 2Drug ApprovalPhase 3Financial Statement

29 Jul 2025

·endpoints.news

Exelixis axes late-stage plans for TKI drug in head and neck cancer as Q2 earnings disappoint

Exelixis’ shares have taken a hit after the company shared a lackluster set of second-quarter earnings and nixed development of its tyrosine kinase inhibitor candidate in head and neck cancer. The California biotech said Monday it no longer plans to advance zanzalintinib into the Phase 3 portion of STELLAR-305 in patients with advanced squamous cell carcinoma of the head and neck. The decision was based on “emerging data” from the Phase 2 part of the study as well as new competition in the indication, Exelixis said. The company’s shares $EXEL fell about 14% when markets opened Tuesday. Jefferies analysts said Monday that while the decision to not move forward with the Phase 3 may be “disappointing” for investors, it is “prudent” from a cash perspective because zanzalintinib’s other indications offer larger commercial opportunities with better competitive dynamics. The drug is in registrational development for colorectal cancer, non-clear cell renal cell carcinoma and advanced neuroendocrine tumors. In June, Exelixis reported a Phase 3 win for zanzalintinib in combination with Roche’s Tecentriq in metastatic colorectal cancer. The company also plans to start “an additional wave” of pivotal zanzalintinib trials in the coming months. Exelixis also reported Q2 revenues of $568.3 million, which came in below Wall Street consensus estimates of $580 million, according to Stifel analysts. The miss was partly due to lower-than-expected income from licenses and other collaborations, which totaled $48.2 million compared with $199.6 million for the same period in 2024. Sales of cancer medicine Cabometyx in the quarter reached $517.9 million, also missing consensus estimates of $531 million. This was driven by higher-than-expected gross-to-net sales adjustments as a result of more sales volume under the 340B program, which allows hospitals and clinics to buy outpatient drugs at a significant discount from manufacturers. Editor’s note: The company’s share price was updated.

Phase 3Financial StatementPhase 2Clinical Trial FailureClinical Trial Termination

25 Jul 2025

·pipelinereview.com

Cabometyx® approved in the EU for previously treated advanced neuroendocrine tumors

PARIS, France I July 24, 2025 I Ipsen announced today that the European Commission has approved Cabometyx ® (cabozantinib) 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. Most forms of neuroendocrine tumors (NETs) develop slowly, can originate in various parts of the body 5 and require multiple lines of therapy as the disease progresses. 1,2 Treatment options upon progression are often limited depending on primary tumor site and other factors, making it challenging to define optimal sequencing of treatments specific to individual patient needs. 1,2,6 In particular, for the 27% of people diagnosed with lung NETs 7 , there have been no approved treatment options upon progression on a prior therapy. 1,2 “The complex nature of neuroendocrine tumors and lack of innovation in recent years has resulted in significant physical and emotional strain for patients as their disease progresses,” said Sandra Silvestri, MD, PhD, EVP and Chief Medical Officer, Ipsen. “We are pleased that for the first time, this approval offers a unique, simplified and efficacious treatment option upon progression, where few to no other options currently exist. We look forward to working with local health authorities to make Cabometyx ® available to even more patients, reinforcing our longstanding commitment to delivering transformational therapies in oncology.” NETs can have a significant impact on both individuals’ lives and wider society, with 71% of people reporting NETs having a negative effect on their daily life, with 92% reporting that they have to make lifestyle changes to accommodate their disease. 8 “Sequential use of systemic therapies remains challenging in the different types of neuroendocrine tumor (NET) that may arise from various organ sites. The number of available therapies is limited and not all NET patients may benefit from currently approved therapies,” said Professor Marianne Pavel, Endocrinologist and NET expert at the Department of Medicine 1, Friedrich-Alexander University of Erlangen, Germany. “Advancements like those achieved through the CABINET Phase III study, as recognized by the approval for Cabometyx in a wide range of NET, is offering new treatment opportunities to delay disease progression in patients with well differentiated NET irrespective of the type of neuroendocrine tumor.” The EC approval was based on data from the CABINET Phase III trial which investigated Cabometyx versus placebo in people living with advanced pNETs or epNETs whose disease had progressed after prior systemic therapy. Final results from the trial, as presented at the 2024 European Society for Medical Oncology Congress and simultaneously published in the New England Journal of Medicine , demonstrated progression-free survival (PFS) benefits in favor of Cabometyx versus placebo: 3,4 About Cabometyx Cabometyx is a small molecule that inhibits multiple receptor tyrosine kinases, including VEGFRs, MET, RET and the TAM family (TYRO3, MER, AXL). 10 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. 10,11,12,13 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 indicated as: 11 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. The epNET cohort included patients with the following primary tumor sites: gastrointestinal tract, lung, unknown primary and other organs. Each cohort was randomized separately and had its own statistical analysis plan. 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 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. References 1 Pavel M, et al . Gastroenteropancreatic neuroendocrine neoplasms: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2020;31(7):844-860. 2 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. 3 Chan et al. Phase 3 Trial of Cabozantinib in Previously Treated Advanced Neuroendocrine Tumors. 2024 New England Journal of Medicine. DOI: 10.1056/NEJMoa2403991 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 Neuroendocrine tumor (NET). https://www.cancer.gov/pediatric-adult-rare-tumor/rare-tumors/rare-endocrine-tumor/carcinoid-tumor. Accessed July 2025. 6 McClellan, K., Chen. E.Y, Kardosh A., et al. Therapy Resistant Gastroenteropancreatic Neuroendocrine Tumors. Cancers. 2022, 14(19), 4769. 7 Frilling et al. Neuroendocrine tumor disease: an evolving landscape. 2012. 19:R163-R185 8 Singh et al. Patient-Reported Burden of a Neuroendocrine Tumor (NET) Diagnosis: Results From the First Global Survey of Patients With NETs. J Glob Oncol. 2017 Feb; 3(1): 43–53. 9 Dueck et al . Health-related quality of life (HRQOL) in the phase 3 trial of cabozantinib vs placebo for advanced neuroendocrine tumors (NET) after progression on prior therapy (CABINET, Alliance A021602). As presented at ASCO Congress 2025. 10 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. 11 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 July 2025. 12 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 13 Hsu et al., AXL and MET in Hepatocellular Carcinoma: A Systematic Literature Review. Liver Cancer 2021 DOI: 10.1159/000520501 SOURCE: Ipsen

Phase 3Clinical ResultLicense out/inDrug Approval

100 Deals associated with Cabozantinib (s)-Malate

External Link

KEGG	Wiki	ATC	Drug Bank
D10095	Cabozantinib (s)-Malate	L01XE26	DB08875

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Well Differentiated Pancreatic Endocrine Tumor	European Union	Ipsen Pharma SAS	24 Jul 2025
Well Differentiated Pancreatic Endocrine Tumor	Iceland	Ipsen Pharma SAS	24 Jul 2025
Well Differentiated Pancreatic Endocrine Tumor	Liechtenstein	Ipsen Pharma SAS	24 Jul 2025
Well Differentiated Pancreatic Endocrine Tumor	Norway	Ipsen Pharma SAS	24 Jul 2025
Extra-pancreatic neuroendocrine tumor	United States	Exelixis, Inc.	26 Mar 2025
Neuroendocrine tumor of pancreas	United States	Exelixis, Inc.	26 Mar 2025
Differentiated Thyroid Gland Carcinoma	South Korea	Ipsen Ltd.	26 Sep 2017
Renal Cell Carcinoma	South Korea	Ipsen Ltd.	26 Sep 2017
Hepatocellular Carcinoma	European Union	Ipsen Pharma SAS	09 Sep 2016
Hepatocellular Carcinoma	Iceland	Ipsen Pharma SAS	09 Sep 2016
Hepatocellular Carcinoma	Liechtenstein	Ipsen Pharma SAS	09 Sep 2016
Hepatocellular Carcinoma	Norway	Ipsen Pharma SAS	09 Sep 2016
Advanced Renal Cell Carcinoma	United States	Exelixis, Inc.	25 Apr 2016
Thyroid Cancer, Medullary	United States	Exelixis, Inc.	29 Nov 2012

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Solid tumor	NDA/BLA	China	Jiangsu Hansoh Pharmaceutical Co., Ltd.	28 Sep 2020
Refractory Differentiated Thyroid Gland Carcinoma	Phase 3	United States	ECOG-ACRIN Medical Research Foundation, Inc.	22 Aug 2024
Refractory Thyroid Gland Carcinoma	Phase 3	United States	ECOG-ACRIN Medical Research Foundation, Inc.	22 Aug 2024
Metastatic osteosarcoma	Phase 3	United States	National Cancer Institute	03 Mar 2023
Advanced Urothelial Carcinoma	Phase 3	United States	National Cancer Institute	03 Jun 2022
Advanced Urothelial Carcinoma	Phase 3	Canada	National Cancer Institute	03 Jun 2022
Bladder Cancer	Phase 3	United States	National Cancer Institute	03 Jun 2022
Bladder Cancer	Phase 3	Canada	National Cancer Institute	03 Jun 2022
Metastatic Urethral Urothelial Carcinoma	Phase 3	United States	National Cancer Institute	03 Jun 2022
Metastatic Urethral Urothelial Carcinoma	Phase 3	Canada	National Cancer Institute	03 Jun 2022

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02302833 (CTgov)	Phase 2	Regional Adrenal Gland Pheochromocytoma \| Adrenal Gland Pheochromocytoma	21	Cabozantinib	veupdzwcfa = vjnjnchkck rltcezdphs (ycdjsxfjzp, bqbasnnryv - omtcmqukqh) View more	-	31 Jul 2025
NCT04220229 (CTgov)	Phase 1/2	Sarcoma of the Extremity	6	Radiation Therapy+Cabozantinib S-malate	qguogprrfk = zdiqykertg bxnuihplwk (rttunssihy, zqjurtjnwu - lifhxfbyzh)	-	17 Jul 2025
NCT05007613 (ESMO_GI2025)	Phase 2	Metastatic Esophageal Squamous Cell Carcinoma Second line	24	Cabozantinib 40mg	xacnpklgvf(kuqjxpnhow) = ulzqclzkjm owqpbpevmg (lhqupmwgfs, 11.7% - 60.1) View more	Positive	03 Jul 2025
EUCTR2019-004728-39-DE (IKF-t018 AURORA, ESMO_GI2025)	Phase 2	First line	22	Cabozantinib 60 mg/day	fvtxgoabco(fsfrmjqdjk) = DOR could not be analyzed rqfncteluq (ffcfgxfzoe )	Positive	03 Jul 2025
NCT05536141 (ARC-20, PipelineReview) Manual	Phase 1	Renal Cell Carcinoma	42	Casdatifan 100mg QD + Cabozantinib 60mg QD	lmgzibuxew(bkhvmxcdbz) = ofemodhart pakiinbcpr (lthsekosio, 26 - 67) View more	Positive	02 Jun 2025
NCT05536141 (ARC-20, ASCO2025) Manual	Phase 1	Renal Cell Carcinoma	27	Casdatifan 100 mg + Cabozantinib 60 mg	befyazszub(okuwvtfcvw) = cphnhmhhfe plyxwgoggk (scqqgoikdm ) View more	Positive	30 May 2025
CABONEN (ASCO2025)	Phase 2	Neuroendocrine Tumors Ki67	45	Cabozantinib	njpwlqejtk(pdiaujteqk) = zcxqtrpeth qguidfpapn (oiivzzaiym ) View more	Positive	30 May 2025
NCT01639508 (phase II study of cabozantinib in patients with MET-altered lung cancers, ASCO2025)	Phase 2	Lung Cancer MET exon 14 alterations \| MET amplification	28	Cabozantinib 60 mg daily	rskldyistx(aisavirpbw) = dpkrqqdpyc nohyizgjle (zrmkmmloju, 8.9 - 39.1) View more	Positive	30 May 2025
NCT04338269 (CONTACT-03, ASCO2025)	Phase 3	Advanced Renal Cell Carcinoma Second line	522	Cabozantinib (cabo)	tlcpkwacni(fxcsorobvt) = kbkdmvptda qskjacgldx (ojwfmggang ) View more	Positive	30 May 2025
NCT04338269 (CONTACT-03, ASCO2025)	Phase 3	Advanced Renal Cell Carcinoma Second line	522	Cabozantinib (cabo) + Atezolizumab (atezo)	tlcpkwacni(fxcsorobvt) = ytzcrzvubd qskjacgldx (ojwfmggang ) View more	Positive	30 May 2025
ASCO2025	Not Applicable	Advanced Hepatocellular Carcinoma Second line	552	Lenvatinib	gifzruaawq(raqhspnium) = nunjofvxnv owqdjngkzg (trimmalcet )	Negative	30 May 2025
ASCO2025	Not Applicable	Advanced Hepatocellular Carcinoma Second line	552	Cabozantinib	gifzruaawq(raqhspnium) = wlwyhwasat owqdjngkzg (trimmalcet )	Negative	30 May 2025

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