Cabometyx (cabozantinib tablet) / Takeda, Exelixis, Ipsen - LARVOL DELTA

Young Onset Metastatic Pheochromocytoma: A Case Report of Clinical and Social Challenges (ENDO 2025) - "Treatment included alpha and beta blockade, surgical resection of the brain lesion, radiation therapy to bony and hepatic lesions, Zoledronic acid for bone metastasis, and chemotherapy (Cyclophosphamide, Vincristine, Dacarbazine)...Additional treatment with Lutathera and Cabozantinib was suggested but delayed due to cost and need for manufacturer assistance...Second, the case reveals the limitations of current available treatments for metastatic PCC, even when specific genetic mutations are identified. And third, the case underscores how social determinants of health can significantly impact patient care and outcomes."

Case report • Clinical • Metastases • Endocrine Cancer • Hepatology • Liver Failure • Musculoskeletal Diseases • Neuroendocrine Tumor • Oncology • Orthopedics • Palliative care • Solid Tumor • NF1 • TERT

management of an adrenal cortical carcinoma induced non-islet cell tumor hypoglycemia: a case report (ENDO 2025) - "She was started on adjuvant mitotane, and chemotherapy (cisplatin, etoposide, and doxorubicin) which induced cardiomyopathy...Osilodrostat was initiated for Cushing syndrome...Patient had recurrent severe hypoglycemic episodes, which were refractory to increasing carbohydrate intake, high-dose prednisone, and octreotide...She also received pasireotide 60mg and diazoxide 5mg/kg every 8 hours...Upon applying for alpelisib approval, her clinical status rapidly declined, and she died after 3 months of NITCH diagnosis...IGF2-mediated hypoglycemia was refractory to numerous therapeutic interventions, including paseriotide, pembrolizumab, olaparib, ivosidenib, and cabozantinib. This case emphasizes rare manifestations of metastatic ACC and limitations of existing medical therapies for NITCH."

Case report • Clinical • Adrenal Cortex Carcinoma • Cardiomyopathy • Cardiovascular • Cushing’s Disease • Diabetes • Endocrine Disorders • Hypoglycemia • Metabolic Disorders • Oncology • Pancreatic Cancer • Severe Hypoglycemia • Solid Tumor • IGF1 • IGF2

Tumor Induced Osteomalacia Caused by Metastatic Thyroid Cancer (ENDO 2025) - "Cabozantinib, a tyrosine kinase inhibitor, and palliative radiation therapy were initiated along with zoledronate... TIO is a rare paraneoplastic disorder often associated with phosphaturic mesenchymal tumors. The patient's clinical presentation of severe FGF23 mediated hypophosphatemia and diffuse skeletal fragility was initially misattributed to metastatic poorly differentiated thyroid cancer. Despite extensive evaluation, no secondary mesenchymal tumor was identified on imaging."

Metastases • Endocrine Cancer • Endocrine Disorders • Fatigue • Musculoskeletal Diseases • Oncology • Pain • Renal Disease • Solid Tumor • Thyroid Gland Carcinoma • Thyroid Gland Papillary Carcinoma • FGF23

The c-MET Inhibitor Tepotinib Elicits Growth Inhibitory Activity on Pancreatic Neuroendocrine Tumors Associated with MEN1 (ENDO 2025) - "Analysis of bulk RNA-seq data showed that Tepotinib treatment decreased the level of transcripts associated with cell cycle progression suggesting that a combination therapy with cell cycle inhibitors may enhance Tepotinib efficacy in PNET cells. Further preclinical and clinical evaluation will be important to consider Tepotinib as a treatment option for PNETs."

Endocrine Cancer • Neuroendocrine Tumor • Oncology • Pancreatic Cancer • Solid Tumor • NTRK3

Novel Therapies for Neurofibromatosis Type 1-Associated Inoperable Plexiform Neurofibromas: A Systematic Literature Review (ISPOR 2025) - "These reported on a total of 21 unique studies on 11 emerging therapies: 6 MEK inhibitors (selumetinib, mirdametinib, trametinib, binimetinib, FCN-159, tunlametinib) and 5 targeted anti-cancer agents (cabozantinib, tipifarnib, sorafenib, sirolimus, everolimus)... The promising results of MEK inhibitors in therapeutic trials for NF1 demonstrate that targeting the MAPK/ERK pathway is an attractive therapeutic avenue for unresectable PNs. Because selumetinib is only approved for children, future reimbursement will require advanced studies establishing MEK inhibitor efficacy in adults."

Review • Brain Cancer • Genetic Disorders • Neurofibromatosis • Oncology • Solid Tumor • NF1

Comparative efficacy of cabozantinib or lenvatinib following atezolizumab plus bevacizumzb in patients with advanced hepatocellular cancer. (ASCO 2025) - "This retrospective cohort study found no significant difference in OS in pts with metastatic HCC treated with lenvatinib or cabozantinib as second-line therapy following atezolizumab plus bevacizumab. These findings suggest that both agents may offer comparable survival benefits, highlighting the need for further prospective studies to refine second-line treatment strategies for advanced HCC."

Clinical • Metastases • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor

Risk of tumor lysis syndrome associated with kinase inhibitors: Analysis of FAERS and real-world data. (ASCO 2025) - " The current study presents a Disproportionality Analysis (DA) of FAERS data and explores Real-World Data (RWD) from TriNetX for the class of drugs known as Kinase Inhibitors (KIs) including Lenvatinib, Sorafenib, Sunitinib, Pazopanib, Axitinib and Cabozantinib... Analysis of FAERS and RWD revealed a considerable number of TLS cases post therapy with specified KIs indicating a need for inclusion of preventive measures including prophylaxis with hypouricemic agents, monitoring and early intervention for these therapies. The product labeling for this drug class may also require update to include relevant safety information, warnings and precautions. References: Tumor Lysis Syndrome in Patients With Solid Tumors: A Systematic Review of Reported Cases - PMC July - September 2024; Potential Signals of Serious Risks/New Safety Information Identified by the FDA Adverse Event Reporting System (FAERS); FDA Disproportionality analysis with FAERS data (Data cut-off 30 Sep 2024).*..."

Clinical • Real-world • Real-world evidence • Acute Kidney Injury • CNS Disorders • Endocrine Disorders • Epilepsy • Metabolic Disorders • Nephrology • Oncology • Renal Disease • Solid Tumor

Elevated hernia risk in renal cell carcinoma (RCC) patients treated with tyrosine kinase inhibitors (TKIs): A FAERS analysis. (ASCO 2025) - "Reports mentioning both RCC and TKI use were identified, specifically focusing on sunitinib, pazopanib, axitinib, cabozantinib, lenvatinib, bevacizumab, and tivozanib... Our FAERS analysis suggests a potential association between TKI use and increased hernia risk in patients with RCC. Further investigation using observational studies is crucial to confirm this association and explore underlying mechanisms. Future research should focus on validating these findings, elucidating the biological mechanisms involved, identifying risk factors, and developing clinical guidelines for hernia prevention and management in this patient population."

Clinical • Gastroenterology • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

Efficacy and safety of tyrosine kinase inhibitors and low dose immunotherapy in metastatic renal cell carcinoma. (ASCO 2025) - "First-line treatment with TKI and low-dose IO was given to 72.1% (n=31/43) and 62.5% (n=15/24) patients with a clear-cell and non-clear cell histology respectively, while TKI alone (sunitinib, pazopanib, cabozantinib) was given to 27.9% (n=12/43) and 37.5% (n=9/24) patients respectively... Limited follow-up showed higher response rates, and a trend towards improved survival with the addition of low-dose nivolumab to TKI in patients with clear-cell RCC. Keyword: Low dose nivolumab, Clear-cell renal cell carcinoma, Tyrosine kinase inhibitors"

Clinical • Metastases • Clear Cell Renal Cell Carcinoma • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

Hypertension and VEGF-A level correlation in cancer patients starting VEGF inhibitors: A prospective study. (ASCO 2025) - "VEGFi used were Sunitinib (n=21), Lenvatinib (n=14), Cabozantinib (n=8), Bevacizumab (n=5), Axitinib (n=4), and Pazopanib (n=3)... Approximately 70% of patients developed grade 2 HTN within one week of starting VEGFi, and about half had onset of sub-nephrotic proteinuria by week 12. VEGF-A levels after treatment correlated positively with diastolic BP at week 1. This highlights the importance of early monitoring of HTN and proteinuria."

Clinical • Cardiovascular • Diabetes • Endocrine Cancer • Genito-urinary Cancer • Hypertension • Kidney Cancer • Metabolic Disorders • Nephrology • Neuroendocrine Tumor • Oncology • Renal Cell Carcinoma • Renal Disease • Sarcoma • Solid Tumor

The impact of nephrectomy on the clinical outcomes of first-line immunotherapy and tyrosine kinase inhibitors combinations versus sunitinib in advanced renal cell carcinoma: A meta-analysis of randomized studies. (ASCO 2025) - "The IO+TKI regimens comprised: Avelumab+Axitinib, Nivolumab+Cabozantinib, and Toripalimab plus Axitinib... This meta-analysis highlights the differential impact of therapeutic nephrectomy on the efficacy of first-line IO+TKI combinations compared to Sunitinib in advanced RCC. It suggests that therapeutic nephrectomy may enhance the survival benefits of IO+TKI therapy in advanced RCC. Further prospective studies are warranted to better define the role of nephrectomy in optimizing outcomes for patients receiving modern combination therapies."

Clinical data • Metastases • Retrospective data • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

The impact of firstline immunotherapy and tyrosine kinase inhibitors combinations versus sunitinib on clinical outcomes of favorable risk advanced renal cell carcinoma: A meta-analysis of randomized studies. (ASCO 2025) - "The IO+TKI regimens comprised: Avelumab+Axitinib, Nivolumab+Cabozantinib, Pembrolizumab+Lenvatinib, and Pembrolizumab+Axitinib... This meta-analysis demonstrates that while first-line IO+TKI combinations significantly improve PFS compared to Sunitinib in IMDC favorable risk advanced RCC, they do not confer a statistically significant OS benefit. Nevertheless, this superior PFS observed with IO+TKI regimens suggests enhanced disease control, which is still a meaningful clinical outcome."

Clinical data • Metastases • Retrospective data • Clear Cell Carcinoma • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

Comparative outcomes of HBV-related vs. HCV-related hepatocellular carcinoma under targeted therapy: A retrospective study. (ASCO 2025) - " This retrospective cohort study included patients diagnosed with early stage HBV-related or HCV-related HCC between 2010 and 2020, who underwent targeted therapy with Cabozantinib, Lenvatinib, Sorafenib, and Regorafenib... HCV-related HCC patients demonstrated longer follow-up durations and better overall survival compared to HBV-related HCC patients under targeted therapy. These findings underscore the need for tailored treatment approaches to optimize outcomes for HBV and HCV patients with HCC."

Retrospective data • Hepatitis B • Hepatitis C • Hepatocellular Cancer • Hepatology • Oncology • Solid Tumor

Targeted therapies for metastatic pheochromocytoma and paraganglioma: A systematic review and meta-analysis. (ASCO 2025) - "This meta-analysis suggests that targeted therapies, particularly sunitinib, may offer a modest clinical benefit for patients with MPPGs. However, the limited sample size and number of studies, particularly for cabozantinib and pazopanib, and the inclusion of single-arm trials, warrant cautious interpretation. Larger randomized controlled trials are needed to confirm these findings."

Metastases • Retrospective data • Review • Endocrine Cancer • Neuroendocrine Tumor • Oncology • Solid Tumor

Second interim analysis of CABONEN: An international, multicenter phase II trial investigating cabozantinib in patients with advanced, low proliferative NEN G3. (ASCO 2025) - P2 | "At interim analysis, Cabozantinib represents a safe and effective treatment option in patients with low proliferative NEN G3. The CABONEN study will continue to the next planned analysis."

Clinical • Metastases • P2 data • Cardiovascular • Endocrine Cancer • Gastrointestinal Cancer • Gastrointestinal Disorder • Infectious Disease • Neuroendocrine Tumor • Oncology • Pain • Pancreatic Cancer • Solid Tumor

Exploring survival outcomes in sarcomatoid hepatocellular carcinomas in the era of immunotherapy. (ASCO 2025) - "Systemic Therapy used included combined Atezolizumab/ Bevacizuamab, Pembrolizumab/ Lenvatinib, Gemcitabine/ Docetaxel, Gemcitabine/ Cisplatin, Cabozantinib, and pembrolizumab monotherapy... Sarcomatoid HCC remains aggressive and difficult to treat cancer with limited overall survival. Immunotherapy based regimens have limited efficacy for this HCC variant."

IO biomarker • Fibrosis • Hepatocellular Cancer • Hepatology • Immunology • Metabolic Dysfunction-Associated Steatohepatitis • Oncology • Sarcoma • Solid Tumor • AFP • EGFR • IDH2 • MLH1 • NTRK3 • PALB2 • TP53

An integrative analysis of circulating and tumor microenvironment (TME) determinants of patient response in the Checkmate 9ER (CM 9ER) trial of nivolumab and cabozantinib (NIVO+CABO) in advanced renal cell carcinoma (aRCC). (ASCO 2025) - "Funded by No funding sources reported Background: The CM 9ER trial demonstrated increased objective response rate (ORR), progression-free and overall survival in patients with aRCC treated with NIVO+CABO compared to sunitinib (SUN)... Taken together, these findings indicate that the state of the tumor microenvironment and circulating factors together have an effect on patient responsiveness to NIVO+CABO in aRCC and provides a framework for integrative analysis for biomarker discovery."

Biomarker • Clinical • IO biomarker • Metastases • Tumor microenvironment • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor • CD4 • FOXP3 • PD-L1 • VIM

Phase II trial of zanzalintinib (XL-092) in combination with durvalumab and tremelimumab in unresectable hepatocellular carcinoma (ZENOBIA). (ASCO 2025) - P2 | "A Phase II study (CHECKMATE 040) reported an impressive objective response rate (ORR) of 29% with the combination of ipilimumab/nivolumab and Cabo but noted high toxicity rates...Comprehensive translational analyses include bulk RNA sequencing, spatial transcriptomics of baseline tumor biopsies, & serial ctDNA monitoring. Trial enrollment commenced in December 2024."

Combination therapy • P2 data • Gastroenterology • Gastrointestinal Disorder • Hepatocellular Cancer • Immunology • Oncology • Solid Tumor • AXL

Molecular profile of hepatocellular carcinoma (HCC) in older (OA) versus younger adults (YA) receiving tyrosine kinase inhibitors: Does age matter? (ASCO 2025) - "OA was not associated with MKI-TOT such as sorafenib or cabozantinib. However, OA was associated with a shorter TOT (HR 1.6, p < 0.01) on lenvatinib (len)...Multivariate analysis revealed that OA was independently associated with shorter len-TOT (HR 1.6, p 0.01), while CTNNB1 mutations and DNAJB1-PRKACA fusions were independently associated with longer len-TOT (HR 0.4-0.5, both p < 0.05)... OA was associated with differing survival trends between whites and Asian/PI compared to Black/AA. Further, OA was associated with shorter len-TOT, potentially due to anti-angiogenic toxicity. Our limitations include an inability to investigate race-based differences on len-TOT due to small sample sizes (Black/AA: n = 18, Asian/PI: n = 15) and the lack of toxicity and geriatric assessment data."

Clinical • IO biomarker • Hepatocellular Cancer • Oncology • Solid Tumor • CDKN2A • CTNNB1 • DNAJB1 • PD-L1 • PRKACA • TP53

Effect of the combination of systemic and locoregional therapy on tumor recurrence and survival after liver transplantation for hepatocellular carcinoma (HCC). (ASCO 2025) - "Systemic therapies included Nivolumab + Ipilimumab, Atezolizumab + Bevacizumab, Sorafenib, Lenvatinib, Ramuricumab, and Cabozantinib... Our results show that combination therapy using systemic options in addition to LRT is an effective downstaging strategy for high-risk HCC patients and may improve post-transplant survival and reduce post-LT tumor recurrence. This preliminary data suggests that combination therapy may have a favorable impact on the time to post-LT tumor recurrence in patients with higher risk tumors. This further attests the strong need for sustainable downstaging pre transplantation."

Hepatocellular Cancer • Hepatology • Infectious Disease • Oncology • Solid Tumor • Transplantation

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). (ASCO 2025) - P3 | "Although cabo is associated with pt-reported AEs consistent with its known safety profile, global HRQOL was maintained. A higher proportion of patients receiving cabo reported improved overall status. Results support cabo as a treatment option for pts with previously treated advanced NET that preserves patient HRQOL."

Clinical • HEOR • Metastases • P3 data • Dermatology • Endocrine Cancer • Neuroendocrine Tumor • Oncology • Solid Tumor

Final results of a phase II study of cabozantinib in patients with MET-altered lung cancers. (ASCO 2025) - P2 | "Four out of five patients who achieved a partial response had received prior MET TKI: two with crizotinib, one with tepotinib, and one with capmatinib... This trial met its primary endpoint. Cabozantinib demonstrated activity in MET-altered NSCLC, and prospective clinical proof-of-concept that type II MET TKI switching can rescue type I MET TKI progression was established."

Clinical • P2 data • Anorexia • Dermatology • Fatigue • Hypertension • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Renal Disease • Solid Tumor • MET

Integrating quality of life and overall survival to quantify benefit from frontline systemic therapy options in unresectable/advanced hepatocellular carcinoma: a network meta-analysis (EASL 2025) - " Ten studies, enrolling 7,268 patients treated with Sorafenib, Lenvatinib, Nivolumab, Tislelizumab, Durvalumab, Atezolizumab+Bevacizumab, Sintilimab+IBI305, Durvalumab+Tremelimumab, Nivolumab+Ipilimumab, Atezolizumab+Cabozantinib, Lenvatinib+Pembrolizumab, Camrelizumab+Apatinib were included... Atezolizumab plus Bevacizumab was associated with the highest magnitude in reducing the risk of deterioration of most HR-QoL domains compared to other systemic therapies. Integrated assessment of OS with HR-QoL assessed by MDC suggests atezolizumab plus bevacizumab to provide the best balance between QoL preservation and OS benefit compared to other systemic therapy options in unresectable/advanced HCC."

HEOR • Metastases • Retrospective data • Fatigue • Hepatocellular Cancer • Hepatology • Oncology • Pain • Solid Tumor

Ipilimumab and nivolumab in patients with metastatic clear cell renal cell carcinoma (mccRCC) treated on the phase 3 PDIGREE (Alliance A031704) trial: Results from Step 1 analysis. (ASCO 2025) - P3 | "The PDIGREE trial enrolled a representative US-based population with mccRCC. Step 1 pt characteristics and associated outcomes with induction ipi/nivo reveal new insights into the tolerability and tx response of 1L ipi/nivo. ClinicalTrials.gov: NCT03793166"

Clinical • Metastases • P3 data • Clear Cell Renal Cell Carcinoma • Endocrine Disorders • Fatigue • Gastroenterology • Gastrointestinal Disorder • Genito-urinary Cancer • Immunology • Nephrology • Oncology • Renal Disease • Solid Tumor

HIF family transcription factor expression in a cohort of 4362 patients with renal cell carcinoma (RCC). (ASCO 2025) - "This comprehensive analysis revealed distinct HIF TF expression patterns across RCC subgroups. Notably, elevated HIF2α expression was observed in clear cell RCC, VHL-mutated tumors, and was linked to improved OS and prolonged TOT with cabozantinib, suggesting a potential prognostic role for HIF2α in RCC, warranting further clinical investigation."

Clinical • Clear Cell Renal Cell Carcinoma • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Sarcoma • Solid Tumor • BAP1 • CHEK2 • EPAS1 • HIF1A • PALB2 • PBRM1 • PTEN • RB1 • SMARCB1 • TP53 • TSC1 • VHL