Zelboraf (vemurafenib) / Daiichi Sankyo, Roche, KeChow Pharma - LARVOL DELTA

Precision-guided therapy in dialysis-dependent classic hairy cell leukemia: a case report. (PubMed, Front Oncol) - "We present a novel case of a 43-year-old man on chronic hemodialysis secondary to suspected autosomal dominant polycystic kidney disease diagnosed with classic BRAF V600E-mutated HCL, treated with low-dose vemurafenib in combination with anti-CD20 therapy...This report highlights the feasibility and efficacy of BRAF-targeted therapy combined with an anti-CD20 antibody in a young dialysis-dependent HCL patient, expanding therapeutic options for this high-risk population. Further prospective studies and case series are needed to guide management in this unique clinical context."

Journal • Autosomal Dominant Polycystic Kidney Disease • Chronic Kidney Disease • Fibrosis • Genetic Disorders • Hairy Cell Leukemia • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Nephrology • Oncology • Polycystic Kidney Disease • Renal Disease

Combined cold atmospheric plasma and vemurafenib suppress MITF-P21 signaling and MMP-driven metastasis in cutaneous melanoma. (PubMed, Iran J Basic Med Sci) - "The CAP plus VEM enhances anti-tumor efficacy and suppresses metastasis-related pathways in melanoma, both in vitro and in vivo. This suggests a promising complementary therapeutic strategy."

Journal • Cutaneous Melanoma • Melanoma • Oncology • Solid Tumor • CDKN1A • MITF • MMP2 • MMP9

Comparative Efficacy of Second- and Later-Line BRAF-Targeted Regimens in BRAF V600E-Mutant Metastatic Colorectal Cancer: A Network Meta-Analysis (ACG 2026) - "Five trials enrolling 1,018 patients across eight treatment arms were included. All four experimental regimens demonstrated significant PFS benefit over irinotecan plus cetuximab. Encorafenib plus cetuximab plus alpelisib ranked highest for PFS (HR 0.29, 95% CI 0.19-0.47; P-score 0.923), followed by encorafenib plus binimetinib plus cetuximab (EBC; HR 0.38, 95% CI 0.29-0.49), encorafenib plus cetuximab (EC; HR 0.40, 95% CI 0.31-0.50), and vemurafenib plus irinotecan plus cetuximab (HR 0.48, 95% CI 0.32-0.72)."

Metastases • Retrospective data • Colorectal Cancer • Oncology • Solid Tumor • BRAF

Thyroid Cancer in the Modern Era: From Molecular Landscape and Multimodal Diagnostics to Integrative Traditional Chinese Medicine-A Comprehensive Review. (PubMed, Cancer Manag Res) - "Current comprehensive integrated therapeutic strategies cover robotic surgery, radioactive iodine ablation, external radiotherapy, molecular targeted drugs (sorafenib, lenvatinib, vemurafenib combined with trametinib) and novel immunotherapy. Most available studies are small-sample retrospective analyses lacking large-scale, multicenter randomized controlled trials to verify efficacy and safety. Future research should focus on standardized clinical trial design, in-depth pharmacological mechanism exploration, and construction of standardized thyroid cancer integrative diagnosis and treatment regimens combining Western precision therapy and TCM adjuvant intervention, to clarify the definite positioning of TCM in whole-course thyroid cancer management."

IO biomarker • Journal • Review • Endocrine Disorders • Oncology • Solid Tumor • Thyroid Gland Carcinoma • BRAF • PAX8 • PD-L1 • PPARG

Antitumor effects of CEP32496 on urothelial carcinoma with BRAF mutation (V595E) in dogs. (PubMed, Can J Vet Res) - "For reference, 7 BRAF inhibitors, including GDC0879, PLX4720, encorafenib, vemurafenib, dabrafenib, AZ628, and RAF265, were also evaluated. In the xenograft model, 10 mg/kg body weight of CEP32496 resulted in a significant decrease (P < 0.05) in tumor growth in mice, with severe and extensive necrosis on histopathological examination. These results suggested that CEP32496 exerts antitumor effects on both cell proliferation and tumor growth in UC with V595E."

Journal • Oncology • Solid Tumor • Urothelial Cancer • BRAF

Efficacy Study of Vemurafenib in Children With Recurrent or Progressive BRAFV600E- or BRAFInsT-Mutant Brain Tumors: PNOC002. (PubMed, JCO Precis Oncol) - "Vemurafenib showed efficacy in children with recurrent or progressive BRAFV600E-mutant brain tumors with acceptable toxicity. However, the rebound rate of 23% warrants further investigation."

Journal • Brain Cancer • CNS Tumor • Glioma • High Grade Glioma • Low Grade Glioma • Oncology • Pediatrics • Solid Tumor • BRAF

Cobimetinib Plus Vemurafenib in Patients With Colorectal Cancer With BRAF Mutations: Results From the Targeted Agent and Profiling Utilization Registry (TAPUR) Study. (PubMed, JCO Precis Oncol) - "The combination of C + V has antitumor activity in heavily pretreated patients with CRC with BRAF mutations."

Journal • Colorectal Cancer • Endocrine Disorders • Fatigue • Gastrointestinal Cancer • Gastrointestinal Disorder • Hematological Disorders • Metabolic Disorders • Oncology • Pulmonary Disease • Renal Disease • Solid Tumor • BRAF • MAP2K1 • NRAS

Overall survival with first-line atezolizumab in combination with vemurafenib and cobimetinib in BRAF mutation-positive advanced melanoma (IMspire150): second interim analysis of a multicentre, randomised, phase 3 study. (PubMed, Lancet Oncol) - P3 | "Additional follow-up of the IMspire150 trial showed that overall survival was not significantly improved with atezolizumab, vemurafenib, and cobimetinib compared with placebo, vemurafenib, and cobimetinib in patients with BRAF mutation-positive advanced melanoma. Results of the final analysis are awaited to establish whether a significant improvement in overall survival can be achieved with long-term treatment with this triplet combination versus vemurafenib plus cobimetinib."

Combination therapy • Journal • P3 data • P3 data: top line • Hepatology • Inflammation • Liver Failure • Melanoma • Oncology • Solid Tumor • BRAF

Combination of vemurafenib with cobimetinib in BRAF V600E/K mutated melanoma patients to normalize LDH and optimize immunotherapy with nivolumab and ipilimumab (COWBOY): A randomized, open-label phase 2 clinical trial. (ASCO 2026) - P2 | "BRAF/MEKi induction prior to dual ICI did not improve response rates to ICI and was associated with worse PFS and OS in pts with BRAFV600E/K mutated melanoma and elevated LDH compared to upfront dual ICI."

Clinical • IO biomarker • P2 data • Melanoma • Solid Tumor • BRAF

Long-Term Real-World Outcomes and Safety of Vemurafenib and Vemurafenib + Cobimetinib Therapy in Patients with BRAF-Mutated Melanoma. (PubMed, Target Oncol) - "We confirmed significant improvement in the mOS and mPFS of unresectable and/or metastatic BRAF mutated-melanoma patients treated outside clinical trials with V + C as compared with V, with no major increase in toxicity for the combination."

Journal • Real-world • Real-world evidence • Melanoma • Oncology • Solid Tumor • BRAF

Pan-Cancer Efficacy of Vemurafenib in BRAFV600-Mutant Non-Melanoma Cancers. (PubMed, Cancer Discov) - "Responses were observed in 13 unique cancer types, including historically treatment-refractory tumors such as cholangiocarcinoma, sarcoma, glioma, neuroendocrine carcinoma, and salivary gland carcinomas. Collectively, these data demonstrate that single-agent BRAF inhibition has broader clinical activity than previously recognized."

Clinical • Journal • Biliary Cancer • Brain Cancer • Cholangiocarcinoma • CNS Disorders • Colorectal Cancer • Gastrointestinal Cancer • Glioma • Neuroendocrine Tumor • Salivary Gland Cancer • Sarcoma • Solid Tumor • BRAF

Contribution of MEK Inhibition to BRAF/MEK Inhibitor Combination Treatment of BRAF-Mutant Melanoma: Part 2 of the Randomized, Open-Label, Phase III COLUMBUS Trial. (PubMed, J Clin Oncol) - "COMBO300 improved PFS, ORR, and tolerability compared with ENCO300, confirming the contribution of binimetinib to efficacy and safety."

Journal • P3 data • Melanoma • Oncology • Solid Tumor • BRAF

Cobimetinib Plus Vemurafenib in Patients With Solid Tumors With BRAF Mutations: Results From the Targeted Agent and Profiling Utilization Registry Study. (PubMed, JCO Precis Oncol) - "Cobimetinib plus vemurafenib showed antitumor activity in patients with advanced solid tumors with BRAF V600E mutations; additional study is warranted to confirm the antitumor activity in tumors with non-V600E BRAF mutations."

Journal • Oncology • Ovarian Cancer • Renal Disease • Solid Tumor • BRAF

Vemurafenib Neoadjuvant Trial in Locally Advanced Thyroid Cancer (clinicaltrials.gov) - P2 | N=24 | Active, not recruiting | Sponsor: M.D. Anderson Cancer Center | Trial completion date: Sep 2026 ➔ Sep 2028 | Trial primary completion date: Sep 2026 ➔ Sep 2028

Trial completion date • Trial primary completion date • Oncology • Solid Tumor • Thyroid Gland Carcinoma • Thyroid Gland Papillary Carcinoma • BRAF

Combination or sequence of vemurafenib, cobimetinib, and atezolizumab in high-risk, resectable melanoma (NEO-TIM): Primary results. (ASCO 2024) - P2 | "Pts treated with TT upfront had highest response to NAT but the pts who received IO as NAT had a better RFS."

Late-breaking abstract • Melanoma • Oncology • Solid Tumor • BRAF

COLUMBUS 5-Year Update: A Randomized, Open-Label, Phase III Trial of Encorafenib Plus Binimetinib Versus Vemurafenib or Encorafenib in Patients With BRAF V600-Mutant Melanoma. (PubMed, J Clin Oncol) - P3 | "In this 5-year update of part 1 of the COLUMBUS trial, encorafenib plus binimetinib treatment demonstrated continued long-term benefits and a consistent safety profile in patients with BRAF V600-mutant melanoma."

IO biomarker • Journal • P3 data • Melanoma • Oncology • Solid Tumor • BRAF

Atezolizumab, vemurafenib, and cobimetinib in patients with melanoma with CNS metastases (TRICOTEL): a multicentre, open-label, single-arm, phase 2 study. (PubMed, Lancet Oncol) - P2 | "Adding atezolizumab to vemurafenib plus cobimetinib provided promising intracranial activity in patients with BRAF-mutated melanoma with CNS metastases."

IO biomarker • Journal • P2 data • CNS Disorders • Dermatitis • Dermatology • Hematological Disorders • Immunology • Melanoma • Oncology • Solid Tumor • BRAF

A context-dependent modulatory role for eIF6 in acquired resistance to vemurafenib in melanoma. (PubMed, FEBS Lett) - "Silencing of eIF6 led to reduced proliferation and partially restored vemurafenib sensitivity, while its overexpression increased sensitivity irrespective of BRAF status via mTOR and MAPK modulation. These findings reveal a context-dependent role for eIF6, linking translation regulation to therapeutic response in melanoma."

Journal • Preclinical • Melanoma • Oncology • Solid Tumor • BRAF • EIF6

Determinants of clinical decision-making in first-line treatment of pleomorphic xanthoastrocytoma and the impact of BRAF status on subsequent therapies: a 10-year single-center series (EANO 2026) - "Grade 3 PXA pts underwent multimodality treatment, with 90% (9/10) receiving either radiochemotherapy with concurrent and adjuvant temozolomide (TMZ) (n=6) or radiotherapy followed by TMZ (n=3)...Second-line systemic chemotherapies included TMZ (n=4, mPFS 3.4 months) and fotemustine/lomustine with or without procarbazine (n=3, mPFS 4.5 m). Targeted therapies administered at first or second progression included dabrafenib/trametinib (n=2, DCR 50%, mPFS 25 m), vemurafenib (n=1, DCR 100%, mPFS 27 m), and regorafenib (n=1, DCR 0%, mPFS 1.1 m)... First-line treatment for PXA is primarily driven by grade, extent of resection, and patient-related factors. Despite the high prevalence of BRAF V600E mutations, targeted therapy was utilized in a minority of cases, likely due to the long analysis period and evolving therapeutic standards, including the approval of dabrafenib/trametinib in Italy in 2024 for high-grade gliomas. The clinical impact of BRAF inhibition and its potential..."

Clinical • Astrocytoma • Brain Cancer • High Grade Glioma • Oncology • Pleomorphic Xanthoastrocytoma • BRAF

Early switch from run-in with targeted to immunotherapy in advanced BRAFV600-positive melanoma: final results of the randomised phase II ImmunoCobiVem trial. (PubMed, ESMO Open) - "Early switch to ICIs after TT run-in (arm B) led to an improved, although not statistically significant, 4- and 5-year landmark OS compared with arm A. No subgroups were identified for which a TT run-in provided clinical benefit. The number of patients developing brain metastasis and the time to brain metastasis were not improved by an early TT to ICI switch."

Journal • P2 data • Melanoma • Oncology • Solid Tumor • PD-1

Early switch from targeted to immunotherapy in advanced BRAFV600-positive melanoma: Long-term OS and final PFS results of the randomized phase II ImmunoCobiVem trial (ESMO 2024) - P2 | "While first-line ICI is superior to TT in terms of overall survival (OS), it is still uncertain whether a short run-in with TT and switch to ICI before progression may improve benefit. The 1:1 randomised phase 2 ImmunoCobiVem trial compared – after a 3-month run-in phase with vemurafenib (V, 960 mg twice daily) and cobimetinib (C, 60 mg daily d 21-28, Q4W) – in Arm A continuous V+C until progressive disease (PD1) and second-line (2L) atezolizumab (ATEZO, 1200 mg Q3W) versus (Arm B) early switch to ATEZO after the run-in phase and then crossover to V+C at PD1. The early switch to ICI after TT run-in led to an improved landmark OS at 4- and at 5-years compared to Arm A, although not statistically significant. The better early tumor control (PFS1) in Arm A and treatment options outside the trial diluted the overall effect size for early switch to ICI, so that it remained small. No subgroups were identified for which a TT run-in (Arm B) provided clinical benefit."

Clinical • Late-breaking abstract • Metastases • P2 data • Melanoma • Oncology • Skin Cancer • Solid Tumor • PD-1

Atezolizumab (A), cobimetinib (C), and vemurafenib (V) in patients (pts) with BRAFV600 mutation–positive melanoma with central nervous system (CNS) metastases (mets): Primary results from phase 2 Tricotel study. (ASCO 2022) - P2 | "Prespecified subgroup analyses were performed in pts receiving corticosteroids (>2 mg/d dexamethasone) and/or with CNS-related symptoms at baseline vs asymptomatic pts. Addition of A to C + V provides promising intracranial activity in pts with BRAFV600-mutated melanoma with CNS mets, particularly in those receiving corticosteroids and/or in symptomatic pts. The safety profile of A + C + V is consistent with that observed in the IMspire150 study."

Clinical • IO biomarker • P2 data • Immunology • Melanoma • Oncology • Solid Tumor

A phase III, randomized, open-label study of tunlametinib plus vemurafenib versus investigator's choice of therapy in patients with previously treated BRAFV600E-mutant metastatic colorectal cancer. (ASCO 2026) - P1, P3 | "This is the first phase III randomized controlled trial to demonstrate that a BRAF/MEK inhibitor combination (tunlametinib plus vemurafenib) significantly improves PFS and ORR compared with conventional chemotherapy-based regimens in previously treated BRAF V600E–mutant mCRC, with a manageable safety profile and the added benefit of a convenient all-oral regimen."

Clinical • Late-breaking abstract • Metastases • P3 data • Colorectal Cancer • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor

Improving public cancer care by implementing precision medicine in Norway (clinicaltrialsregister.eu) - P1/2 | N=1000 | Recruiting | Sponsor: Oslo University Hospital HF | N=6000 ➔ 1000

Enrollment change • Oncology

Effects of BRAF-Targeted Therapy on Telomerase Expression in Thyroid Cancer (ETA 2026) - "Vemurafenibs effect on cell proliferation and viability seems to depend on cell genotype, being stronger in the B-CPAP cell line, which harbours the BRAF V600E mutation."

Oncology • Solid Tumor • Thyroid Gland Carcinoma • Thyroid Gland Papillary Carcinoma • BRAF • TERT