Lytgobi (futibatinib) / Otsuka - LARVOL DELTA

Recurrent resistance mutations to lirafugratinib inform treatment sequencing in FGFR2-driven tumors. (PubMed, Clin Cancer Res) - P1/2 | "The complementary activity of lirafugratinib and futibatinib against FGFR2 kinase domain mutations supports their sequential use, when precise resistance mutations are detected in patients."

Journal • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • BICC1 • FGFR2

Pharmacovigilance analysis of the FGFR inhibitor futibatinib using the FDA adverse event reporting system (FAERS) database. (PubMed, J Oncol Pharm Pract) - "These events may reflect direct drug effects and individual variability. Further studies are needed to clarify mechanisms and inform clinical management."

Adverse events • Journal • Oncology

The evolving role of futibatinib for advanced cholangiocarcinoma. (PubMed, Expert Rev Gastroenterol Hepatol) - "A comprehensive literature search was performed in PubMed/MEDLINE, Embase, and Scopus for studies published from January 2000 to February 2026, using combinations of the terms 'cholangiocarcinoma,' 'FGFR2,' 'fibroblast growth factor receptor,' and 'futibatinib.' Relevant clinical trials, translational studies, and review articles were screened for inclusion. Futibatinib represents the most biologically rational FGFR inhibitor currently available for FGFR2-altered CCA, as it directly addresses the dominant mechanism limiting the efficacy of earlier agent, on-target resistance driven by secondary FGFR2 kinase domain mutations."

Journal • Review • Biliary Cancer • Biliary Tract Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • FGFR2

Tinengotinib for adults with advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 trial. (PubMed, Lancet Gastroenterol Hepatol) - P2 | "These findings suggest that tinengotinib might have activity in patients with cholangiocarcinoma with FGFR2 fusions that progressed following FGFR inhibitor therapy. Anti-tumour activity was also observed in patients with other FGFR alterations. The data from this phase 2 study supported the initiation of a phase 3 registration trial."

Journal • P2 data • Biliary Cancer • Cardiovascular • Cholangiocarcinoma • CNS Disorders • Dental Disorders • Dermatology • Hypertension • Oncology • Solid Tumor • Stomatitis • FGFR2

Phosphorylated FRS2 (pFRS2) as a biomarker for FGFR mutation or fusion activity assessment in formalin-fixed paraffin-embedded tumor sections (AACR 2026) - "The FGFR inhibitors, pemigatinib, erdafitinib and futibatinib, have FDA approvals to treat selected solid tumors with FGFR pathway alterations. This enables quantitative distinction between specimens with high pathway activity (including fusions and some point mutations) from specimens with lower pathway activity (some amplifications), suggesting utility as a companion diagnostic for identification of likely responders to currently approved and newly emerging FGFR therapies. In particular, the ability to determine subcellular localization of pathway activity may prove useful for trials of both antibody-drug conjugates and bispecific antibodies which might be expected to be less effective in tumors where the oncogenic FGFR activity is primarily internal and not cell-surface associated."

Biomarker • Oncology • Solid Tumor • FGFR • FGFR1 • FGFR2 • FGFR3 • FGFR4 • FRS2

Comparative biochemical kinase activity analysis identifies lirafugratinib as a highly selective FGFR2 inhibitor (AACR 2026) - "A KINOMEscan Profiling Service scanMAX and TREEspot™ interaction maps were used for evaluation and visualization of inhibition of kinases: a panel of 468 human target kinases and disease-relevant kinase mutant variants were tested at a concentration of 500 nM for lirafugratinib and four other pan-FGFR inhibitors (futibatinib, pemigatinib, erdafitinib, and AZD4547). The head-to-head kinome analysis of lirafugratinib and four other FGFR inhibitors revealed that lirafugratinib inhibited FGFR2 with high selectivity, suggesting minimal off-target and off-isoform-related toxicities compared to pan-FGFR inhibitors used in clinical practice."

Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • FGFR2 • FGFR3 • FGFR4 • MKNK2

FGFR2 translocated sinonasal adenocarcinoma: A biphasic seromucinous adenocarcinoma with a distinctive and targetable molecular phenotype (AACR 2026) - "FGFR2::SORBS3 is rare even among the 150+ identified partners in other tumor types (i.e. cholangiocarcinoma) but appear to function similarly, upregulating phosphorylation pathways. Recognition of this tumor subtype could help select patients potentially amenable to FDA- approved oral FGFR2 inhibitors (pemigatinib, futibatinib)."

Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • Squamous Cell Carcinoma • ALOX15 • BMPR1A • BRAF • CTNNB1 • DICER1 • ETV6 • FGFR2 • IL12A • IL6 • KRAS • KRT14 • NTRK3 • OLFM4 • PIK3R1 • PTEN • SOX2 • SPP1 • STAT3

MULTISARC: Molecular Profiling of Advanced Soft-tissue Sarcomas (clinicaltrials.gov) - P3 | N=960 | Recruiting | Sponsor: Institut National de la Santé Et de la Recherche Médicale, France | Not yet recruiting ➔ Recruiting | Trial completion date: Mar 2024 ➔ Oct 2024 | Initiation date: Mar 2019 ➔ Oct 2019 | Trial primary completion date: Mar 2022 ➔ Oct 2022

Enrollment open • IO biomarker • Trial completion date • Trial initiation date • Trial primary completion date • Oncology • Sarcoma • Soft Tissue Sarcoma

Secondary resectability through individually personalised sequential therapy for metastatic CCC (DKK 2026) - "A palliative chemotherapy with Gemcitabin, Cisplatin and Durvalumab was started. This case shows the importance of gene mutation analysis and interdisciplinary re-evaluation in the treatment of CCC even in initially unresectable cases. Indication of source: 1. Goyal, L., et al., Futibatinib for FGFR2-Rearranged Intrahepatic Cholangiocarcinoma."

Clinical • Metastases • Biliary Cancer • Biliary Tract Cancer • Cholangiocarcinoma • Oncology • Palliative care • Solid Tumor • FGFR2

MULTISARC: Molecular Profiling of Advanced Soft-tissue Sarcomas (clinicaltrials.gov) - P3 | N=960 | Not yet recruiting | Sponsor: Institut National de la Santé Et de la Recherche Médicale, France

IO biomarker • New P3 trial • Oncology • Sarcoma • Soft Tissue Sarcoma

MULTISARC: Molecular Profiling of Advanced Soft-tissue Sarcomas (clinicaltrials.gov) - P3 | N=603 | Active, not recruiting | Sponsor: Institut National de la Santé Et de la Recherche Médicale, France | Recruiting ➔ Active, not recruiting | N=960 ➔ 603

Enrollment change • Enrollment closed • IO biomarker • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

MULTISARC: Molecular Profiling of Advanced Soft-tissue Sarcomas (clinicaltrials.gov) - P3 | N=960 | Recruiting | Sponsor: Institut National de la Santé Et de la Recherche Médicale, France | Trial completion date: Oct 2024 ➔ Oct 2025 | Trial primary completion date: Oct 2022 ➔ Oct 2023

IO biomarker • Trial completion date • Trial primary completion date • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

Futibatinib in Patients Previously Enrolled in an Antecedent Futibatinib as Monotherapy or Combination Therapy. (clinicaltrials.gov) - P2/3 | N=15 | Enrolling by invitation | Sponsor: Taiho Oncology, Inc. | Trial completion date: Dec 2026 ➔ Apr 2027 | Trial primary completion date: Dec 2026 ➔ Apr 2027

Monotherapy • Trial completion date • Trial primary completion date • Oncology

Management of Nail Toxicities From Fibroblast Growth Factor Receptor Inhibitors. (PubMed, J Drugs Dermatol) - "The incidence of FGFRi-associated nail toxicities varies by agent and can affect quality of life and treatment adherence. The pathogenesis remains unclear, and no predictive biomarkers exist. Further research into optimized management and preventative strategies is needed. Early recognition and proactive multidisciplinary management are essential to minimizing complications and maintaining oncologic treatment continuity. &nbsp."

Journal • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • Urothelial Cancer • FGFR

ESMART: European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors (clinicaltrials.gov) - P1/2 | N=285 | Recruiting | Sponsor: Gustave Roussy, Cancer Campus, Grand Paris

New P1/2 trial • Hematological Malignancies • Oncology • Pediatrics

ESMART: European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors (clinicaltrials.gov) - P1/2 | N=455 | Recruiting | Sponsor: Gustave Roussy, Cancer Campus, Grand Paris | Trial completion date: Aug 2027 ➔ Feb 2031 | Trial primary completion date: Aug 2027 ➔ Feb 2031

Trial completion date • Trial primary completion date • Hematological Malignancies • Oncology • Pediatrics

ESMART: European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors (clinicaltrials.gov) - P1/2 | N=397 | Recruiting | Sponsor: Gustave Roussy, Cancer Campus, Grand Paris | N=285 ➔ 397 | Trial completion date: Dec 2020 ➔ Jan 2022 | Trial primary completion date: Dec 2020 ➔ Jan 2022

Enrollment change • Trial completion date • Trial primary completion date • Pediatrics

SAFIR ABC10: Targeted maintenance therapy versus first-line standard of care in advanced biliary cancer. (BWG 2026) - "Introduction: The first-line standard of care (1L-SoC) for advanced biliary tract cancers (ABC), a heterogeneous group of malignancies of the bile ducts and gallbladder, is cisplatin and gemcitabine combined with anti-PD-(L)1...They were randomized to SoC (18 pts, 33%), Futibatinib (13 pts, 24%), Ivosidenib (13 pts, 24%), Zanidatamab (5 pts, 9%), Neratinib-Trastuzumab (1 pt, 2%), Binimetinib-Encorafenib (4 pts, 8%)... SAFIR ABC10 is recruiting at a high pace, with > 50% of the target sample screened, and 34.5% (55/159 pts) randomized. Targetable alterations were found in 104 pts (29%) of patients (including 1% MSI-High pts), of which 55 are randomized. Belgium centres are currently open(ing)."

Clinical • Metastases • Biliary Cancer • Biliary Tract Cancer • Cholangiocarcinoma • Gallbladder Cancer • Oncology • Solid Tumor • BRAF • FGFR2 • HER-2 • IDH1

ESMART: European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors (clinicaltrials.gov) - P1/2 | N=460 | Recruiting | Sponsor: Gustave Roussy, Cancer Campus, Grand Paris | Trial completion date: Jan 2022 ➔ Aug 2027 | Trial primary completion date: Jan 2022 ➔ Aug 2027

Trial completion date • Trial primary completion date • Hematological Malignancies • Oncology • Pediatrics

Saturation mutational scanning uncovers druggability of FGFR2 point mutations outside of the kinase domain (DKK 2026) - "While FGFR tyrosine kinase inhibitors like Pemigatinib and Futibatinib have been approved for FGFR translocations, their efficacy against FGFR point mutations is unclear. Our comprehensive catalog of all actionable FGFR2 mutations supports clinical decision-making in molecular tumor boards. The screening method developed here broadly enables the systematic identification of gain-of-function or loss-of-function mutations in other clinically relevant genes."

Oncology • CKB • FGFR2

FGFR2-Rearranged Biliary Tract Cancer: Biology, Resistance Mechanisms, and Emerging Therapeutic Strategies. (PubMed, Cancers (Basel)) - "Selective FGFR tyrosine kinase inhibitors, including the reversible inhibitor pemigatinib and the irreversible inhibitor futibatinib, have demonstrated clinically meaningful response rates and durable disease control in patients with previously treated FGFR2-altered iCCA, leading to regulatory approvals and the incorporation of FGFR inhibition into contemporary treatment paradigms. In addition, we highlight the growing role of circulating tumor DNA as a noninvasive tool for longitudinal molecular monitoring and treatment guidance. Together, these insights underscore the central role of FGFR2-directed therapy in precision oncology for biliary tract cancer and provide a framework for optimizing and extending targeted treatment in this molecularly defined disease subset."

Journal • Review • Biliary Cancer • Biliary Tract Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • FGFR2

Mutated FGFR1 is an oncogenic driver and therapeutic target in high-risk neuroblastoma. (PubMed, J Clin Invest) - "In addition, partial regression of FGFR1N546K mutant tumor lesions occurred upon treatment with the FGFR inhibitor futibatinib and low-intensity chemotherapy in a patient with refractory neuroblastoma. Together, our data demonstrate that FGFR1N546K is a strong oncogenic driver in neuroblastoma that is associated with failure of current standard chemotherapy, and suggest potential clinical benefit of FGFR-directed therapies in FGFR1 mutant high-risk patients."

Journal • Neuroblastoma • Oncology • Solid Tumor • FGFR1 • MYCN

Fibroblast growth factor receptor inhibitors in glioma: a narrative review of recent advances. (PubMed, Front Pharmacol) - "Pharmacologically, dedicated inhibitors like Infigratinib have demonstrated anti-tumor activity in clinical Phase II trials for FGFR-altered recurrent gliomas, while the multi-kinase inhibitor Regorafenib has shown a modest survival benefit in recurrent GBM; however, mechanistic studies indicate that effective response often relies on co-targeting bypass pathways (e.g., CLK2) and mitigating the tumor's metabolic dependency. Crucially, limited drug exposure through the blood-brain barrier (BBB) continues to be the foremost challenge, dictating optimization efforts toward compounds with favorable pharmacokinetic properties and novel delivery platforms, such as the covalent inhibitor futibatinib and liposomal formulations, to enhance brain penetrance. In conclusion, the evolving molecular landscape validates FGFR alterations as a targetable niche in gliomas, and future success depends critically on integrating comprehensive next-generation sequencing to identify..."

Journal • Review • Brain Cancer • Glioma • Oncology • Solid Tumor • CLK2 • FGFR • FGFR2 • FGFR3 • TACC3

Phase II study of futibatinib plus pembrolizumab in patients (pts) with advanced/metastatic urothelial carcinoma (mUC): Final analysis of efficacy and safety (ESMO 2024) - P2 | "Futibatinib plus pembrolizumab had encouraging antitumor activity with durable responses in pts with mUC, particularly among pts with FGFR3 mutations or FGFR1–4 f/r. The combination was well tolerated; TRAEs were consistent with the known safety profiles of futibatinib and pembrolizumab, with no new safety concerns."

Clinical • IO biomarker • Metastases • P2 data • Oncology • Solid Tumor • Urothelial Cancer • FGFR2 • FGFR3 • PD-L1

POTENTIATE: Study of the CHK1 Inhibitor BBI-355, an ecDNA-directed Therapy (ecDTx), and the RNR Inhibitor BBI-825, in Subjects With Tumors With Oncogene Amplifications (clinicaltrials.gov) - P1 | N=85 | Active, not recruiting | Sponsor: Boundless Bio | Recruiting ➔ Active, not recruiting | N=127 ➔ 85

Enrollment change • Enrollment closed • First-in-human • Breast Cancer • Cervical Cancer • Cervical Squamous Cell Carcinoma • Endometrial Cancer • Head and Neck Cancer • High Grade Serous Ovarian Cancer • Hormone Receptor Breast Cancer • Leiomyosarcoma • Lung Cancer • Oncology • Ovarian Cancer • Pancreatic Cancer • Sarcoma • Small Cell Lung Cancer • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck • Squamous Cell Skin Cancer • Triple Negative Breast Cancer • Undifferentiated Pleomorphic Sarcoma