Stivarga (regorafenib) / Amgen, Bayer - LARVOL DELTA

Radiotherapy Combined with Regorafenib and PD-1 Inhibitor as Third-Line treatment for Microsatellite Stable Metastatic Colorectal Cancer: A Prospective, single arm, Multicenter, Phase II Study (ChiCTR) - P=N/A | N=59 | Not yet recruiting | Sponsor: The First Affiliated Hospital of Army Medical University; The First Affiliated Hospital of Army Medical University

New trial • Colorectal Cancer • Oncology • Solid Tumor

The Clinical Trial of Tirelizumab, Regorafenib and Raltitrexed for Patients With Microsatellite Stable Colorectal Cancer and Disease Progression With Prior Chemotherapy (ChiCTR) - P4 | N=38 | Recruiting | Sponsor: Army Medical Center of PLA; Army Medical Center of PLA

New P4 trial • Colorectal Cancer • Oncology • Solid Tumor

Trifluridine/tipiracil plus bevacizumab (TTB) versus regorafenib (R) in refractory advanced colorectal cancer patients: A real-word evidence efficacy study (ESMO-GI 2025) - "All statistical analyses were conducted utilizing the TriNetX Analytics function in the online research platform. A total of 1483 patients met the study criteria, including previous treatment with oxaliplatin, irinotecan and fluoropyrimidine-based chemotherapy (capecitabine or fluorouracil), also with an antiangiogenic drug (bevacizumab, aflibercept or regorafenib) and with an anti-EGFR therapy (cetuximab or panitumumab) as indicated. In this study, based on Real World Data, trifluridine/tipiracil plus bevacizumab was associated with better overall survival compared to regorafenib in refractory advanced colorectal cancer patients."

Clinical • Metastases • Colorectal Cancer • Oncology • Solid Tumor

ASCO 2025: Current Insights and Emerging Approaches in Managing Treatment-Resistant Metastatic Colorectal Cancer : Episode 6: A Review of the Treatment Options in Third-Line mCRC and Therapy Sequencing Strategies (OncLive) - "Panelists discuss how 3 drugs (regorafenib, TAS-102, and fruquintinib) offer modest but meaningful survival benefits in treatment-refractory colorectal cancer, with TAS-102 plus bevacizumab being the preferred combination when tolerated."

Video

Case Report WIN-MTB-2023001 WIN International Molecular Tumor Board A 62-year-old male with metastatic colorectal cancer with 5 prior lines of treatment. (PubMed, Oncotarget) - "We present a 62-year-old male with mCRC harboring BRAF, MET, APC, TP53 and NRAS alterations, following FOLFOX and FOLFIRI, dabrafenib plus panitumumab, and a BRAF inhibitor clinical trial, each leading to initial responses followed by disease progression...Personalized combinations suggested included amivantamab-vmjw (anti-MET/EGFR antibody) (one-third standard dose) (for MET amplification and due to prior response to anti-EGFR antibody), trametinib, 1 mg po daily (MEK inhibitor for BRAF V600E mutation), and regorafenib (may have WNT inhibitor activity relevant to APC mutation; VEGFR activity relevant since TP53 alterations upregulate VEGF/VEGFR axis) starting at 40 mg po daily three weeks on, one week off. Another option was trametinib at 1 mg daily, cetuximab (EGFR antibody), 250 mg/m² IV every two-weeks, and cabozantinib (MET and VEGFR inhibitor), 40 mg po daily. FOLFOXFIRI combined with bevacizumab, or liver-directed therapies for hepatic metastases, or..."

Biomarker • Journal • Colorectal Cancer • Hepatocellular Cancer • Oncology • Solid Tumor • APC • NRAS • TP53

Current perspectives on the pharmacological treatment of advanced hepatocellular carcinoma: a narrative review. (PubMed, Ewha Med J) - "Historically, first-line systemic therapies included sorafenib and lenvatinib, with regorafenib, cabozantinib, or ramucirumab serving as second-line options. Since 2020, immune checkpoint inhibitors have shown superior overall survival than sorafenib, leading to the adoption of combination therapies such as atezolizumab with bevacizumab and durvalumab with tremelimumab as first-line treatments...Second-line therapies now include regorafenib, cabozantinib, ramucirumab, nivolumab with ipilimumab, and pembrolizumab, each offering benefits for specific patient populations...Clinicians must be well-versed in these treatments and their potential side effects to provide optimal patient care. The emergence of combination therapies targeting complex biological pathways signifies a new paradigm in HCC treatment, emphasizing the importance of continuous education and vigilant monitoring to optimize patient outcomes."

Journal • Review • Cardiovascular • Gastrointestinal Disorder • Hepatocellular Cancer • Hypertension • Liver Cancer • Oncology • Solid Tumor

Autophagy inhibition improves sensitivity to the multi-kinase inhibitor regorafenib in preclinical mouse colon tumoroids. (PubMed, Front Cell Dev Biol) - "In contrast, primary mouse colon fibroblasts exhibited greater resistance to both single-agent and combination regorafenib treatments. In summary, our findings using an organoid model suggest that autophagy inhibition may represent a promising strategy to overcome chemoresistance to regorafenib in mCRC patients."

Journal • Preclinical • Colon Cancer • Colorectal Cancer • Oncology • Palliative care • Solid Tumor

Regorafenib versus local standard of care in patients with grade 2-3 meningioma no longer eligible for loco-regional treatments: a phase II randomized controlled trial (the MIRAGE study). (PubMed, Trials) - P2 | "MIRAGE is a phase 2 trial designed to determine the role of regorafenib in prolonging the PFS of grade 2-3 meningioma patients ineligible for further surgery and/or radiotherapy."

Clinical protocol • Journal • P2 data • Brain Cancer • Meningioma • Oncology • Solid Tumor • BRAF • FGFR1 • FLT1 • PDGFRB

Early Nivolumab addition to Regorafenib in patients with Hepatocellular Carcinoma after first-line therapy (GOING trial). (ESMO 2025) - No abstract available

Clinical • Hepatocellular Cancer • Oncology • Solid Tumor

Managing hepatocellular carcinoma recurrence after liver transplantation: emerging role of immune checkpoint inhibitors. (PubMed, Discov Oncol) - "Surgery or locoregional therapies are preferred but are often unsuitable for disseminated disease, leaving systemic therapies-including tyrosine kinase inhibitors (TKIs) such as sorafenib, lenvatinib, and regorafenib-as primary options. However, ICIs should be reserved for select cases or clinical trials, with multidisciplinary team collaboration being critical. Prospective studies are needed to establish clear guidelines for their use in post-LT care."

Checkpoint inhibition • IO biomarker • Journal • Review • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor • Transplant Rejection • Transplantation

LEAF-02: Tauroursodeoxycholic Acid (TUDCA) Plus Camrelizumab and Regorafenib in Hepatocellular Carcinoma Previously Treated With Anti-PD1/PD-L1 and Bevacizumab (clinicaltrials.gov) - P2 | N=141 | Not yet recruiting | Sponsor: Fudan University

New P2 trial • Hepatocellular Cancer • Oncology • Solid Tumor

GBM AGILE: A Trial to Evaluate Multiple Regimens in Newly Diagnosed and Recurrent Glioblastoma (clinicaltrials.gov) - P2/3 | N=1280 | Recruiting | Sponsor: Global Coalition for Adaptive Research | Trial completion date: Jun 2028 ➔ Jun 2030 | Trial primary completion date: Jun 2026 ➔ Jun 2028

Trial completion date • Trial primary completion date • Brain Cancer • Glioblastoma • Hematological Disorders • Oncology • Solid Tumor

Tyrosine Kinase Inhibitors for Gastrointestinal Stromal Tumor After Imatinib Resistance. (PubMed, Pharmaceutics) - "Sunitinib, regorafenib, and ripretinib are currently approved as standard second-, third-, and fourth-line therapies, each demonstrating efficacy against distinct mutational profiles. Avapritinib, notably effective against PDGFRA D842V mutations, represents a milestone for previously untreatable subgroups. Several alternative agents-such as nilotinib, masitinib, sorafenib, dovitinib, pazopanib, and ponatinib-have shown varying degrees of success in refractory cases or specific genotypes. Investigational compounds, including crenolanib, bezuclastinib, famitinib, motesanib, midostaurin, IDRX-42, and olverembatinib, are under development to address resistant or wild-type GISTs...Future strategies include precision medicine approaches such as ctDNA-guided therapy, rational drug combinations, and novel drug delivery systems to optimize bioavailability and reduce toxicity. Ongoing research will be crucial for refining treatment sequencing and expanding therapeutic options,..."

Journal • Review • Gastrointestinal Cancer • Gastrointestinal Disorder • Gastrointestinal Stromal Tumor • Oncology • Sarcoma • KIT • PDGFRA

Cost-effectiveness analysis of first-line treatment for advanced soft tissue sarcomas in China. (PubMed, J Med Econ) - "To evaluate the cost-effectiveness of four first-line treatment strategies-anlotinib, pazopanib, regorafenib, and conventional chemotherapy-for patients with advanced soft tissue sarcomas (STSs) in China. Conventional chemotherapy, specifically using doxorubicin injection, represents the most cost-effective first-line treatment for advanced STS. However, this conclusion is sensitive to the chemotherapy composition; if the more expensive liposomal doxorubicin is used, anlotinib emerges as the preferred cost-effective option."

HEOR • Journal • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

A Study of Regorafenib Combined With Envafolimab for Metastatic Gastrointestinal Stromal Tumors With Kit Gene Exon 17 Mutation That Failed Standard Treatment (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: Peking University | Trial completion date: Aug 2026 ➔ Jul 2028 | Initiation date: Jan 2025 ➔ Jul 2025 | Trial primary completion date: Aug 2025 ➔ Jul 2027

Trial completion date • Trial initiation date • Trial primary completion date • Gastrointestinal Stromal Tumor • Oncology • Sarcoma

Real-World Impact of Frontline Anti-VEGF and Anti-EGFR Exposure on Survival Outcomes with Trifluridine/Tipiracil (Without Bevacizumab) and Regorafenib: Findings from the Multicenter Retrospective ReTrITA Study (ESMO 2025) - No abstract available

Real-world • Real-world evidence • Retrospective data • Oncology

Liver Transplantation for Hepatocellular Carcinoma in Patients Treated with Tyrosine Kinase Inhibitors Therapy: Efficacy and Safety (WTC 2025) - "The aim of this study was to define the efficacy and safety of Tyrosine Kinase Inhibitors therapy in patients waiting liver transplantation in term of survival and complications.* Previously Sorafenib and then Lenvatinib, Regorafenib, and Cabozantinib provided a survival benefit, but it has been reported many adverse events, such as hand-foot syndrome, diarrhea and more severe cardiovascular disorders.In 2022 and 2023, we performed 5 liver transplants in patients undergoing Tyrosine Kinase Inhibitors therapy at our liver transplant center in Italy... In our small experience, the use of Tyrosine Kinase Inhibitors therapy until Liver Transplant has been associated to very severe complications after the transplant.We need more data to manage those therapies to reduce complications, in terms of timing to stop the therapy and other supportive medications."

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

Evolution of second-line practices in the era of immunotherapy: Real-life data from the French prospective CHIEF cohort (ESMO-GI 2025) - "We aimed to study the access to second-line treatment, comparing patients initially treated with Sorafenib (Sor) to those with atezolizumab-bevacizumab (AB). This study included patients from the real-world prospective CHIEF cohort and aimed to get insight into Systemic treatment sequences in patients with advanced HCC (STRETCH)...No significant differences were found between Sor (n=78), lenvatinib (n=35), regorafenib, or cabozantinib (n=23) (p=0.83), though lenvatinib showed a trend for improved mPFS. This unique prospective cohort provides real-world data on second-line treatment practices. This unique prospective cohort provides real-world data on second-line treatment practices. Access to second-line treatment was lower after AB than Sor, but survivals under second-line TKI were similar. While second-line immunotherapy following immunotherapy shows promising results, these are likely influenced by patient selection for rechallenge."

Clinical • Gastrointestinal Cancer • Hepatocellular Cancer

A RARE CASE OF HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH)-ASSOCIATED STEVEN JOHNSON'S SYNDROME/TOXIC EPIDERMAL NECROLYSIS (SJS/TEN) FROM MULTIKINASE INHIBITOR (REGORAFENIB) USE (CHEST 2025) - No abstract available

Clinical • Hemophagocytic lymphohistiocytosis • Immunology • Rare Diseases • Steven-Johnson Syndrome

Present management of gastrointestinal stromal tumors. (PubMed, Klin Onkol) - "In advanced or metastatic disease, standard care involves sequential treatment with tyrosine kinase inhibitors, including imatinib, sunitinib, and regorafenib. The expanding range of targeted therapies, such as avapritinib for the PDGFRA D842V mutation, underscores the importance of molecular profiling in guiding optimal treatment strategies. This review aims to summarize current knowledge on the diagnosis and treatment of GIST, with a focus on the role of molecular-genetic profiling, the therapeutic value of individual tyrosine kinase inhibitors, and emerging options for advanced disease, with particular emphasis on ripretinib."

Journal • Review • Chronic Myeloid Leukemia • Gastrointestinal Cancer • Gastrointestinal Stromal Tumor • Hematological Malignancies • Leukemia • Oncology • Sarcoma • PDGFRA

Efficacy and safety of hepatic arterial infusion chemotherapy (HAIC) combined with tislelizumab and regorafenib for advanced intrahepatic cholangiocarcinoma (ICC): a single arm, open label, phase Ⅱ study (ESMO 2025) - No abstract available

Clinical • Metastases • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor

A phase 2 clinical trial of regorafenib (REGO) combined with BRAF-/MEK-inhibitors in advanced pretreated BRAFV600-mutant (mut) melanoma (RegoMel) (ESMO 2025) - No abstract available

Clinical • Metastases • P2 data • Melanoma • Oncology • Solid Tumor

Neoadjuvant short-course radiotherapy (SCRT) plus iparomlimab and tuvonralimab (QL1706), CAPOX, and regorafenib in locally advanced rectal cancer (LARC): A randomized, multicenter, non-comparative phase 2 trial in progress (ESMO 2025) - No abstract available

Clinical • Metastases • P2 data • Colorectal Cancer • Oncology • Rectal Cancer • Solid Tumor

A Phase I trial of Botensilimab, Balstilimab and Regorafenib (BBR) in Chemotherapy-Resistant Patients with Microsatellite Stable (MSS) Metastatic Colorectal Cancer (ESMO 2025) - No abstract available

Clinical • Metastases • P1 data • Colorectal Cancer • Oncology • Solid Tumor

Regorafenib plus Irinotecan versus regorafenib alone in metastatic colorectal cancer after standard therapies failure in cyclin D1 A/A(870) genotype patients : the randomized phase III NEXT-REGIRI trial. (ESMO 2025) - No abstract available

Clinical • Metastases • P3 data • Colorectal Cancer • Oncology • Solid Tumor • CCND1