P2, N=80, Recruiting, Advenchen Pharmaceuticals, LLC. | N=36 --> 80 | Trial completion date: Dec 2026 --> Dec 2028 | Trial primary completion date: Dec 2025 --> Dec 2027 | Active, not recruiting --> Recruiting

P2/3, N=201, Recruiting, Janssen Cilag International | Active, not recruiting --> Recruiting

Pemigatinib plus paclitaxel demonstrated antitumor activity with an acceptable safety profile in FGFR2-amplified gastric cancer. Further investigation is warranted to elucidate resistance mechanisms and validate these findings in larger cohorts.

The most common baseline regimen was gemcitabine + cisplatin (34.4%). Treatment patterns, adherence, OS, HCRU, and costs demonstrated consistent trends across racial subgroups. Real-world pemigatinib treatment patterns and survival outcomes were consistent with findings from clinical trials and support continued use of pemigatinib across diverse patient groups as a key second-line treatment for CCA.

Treatment with the FGFR inhibitor BGJ398 reduced tumor growth and decreased stromal FGFR3-positive components, suggesting that stromal FGFR3 may represent a potential microenvironmental vulnerability in CRC with peritoneal dissemination. This autologous CRC-mesothelial system provides a physiologically relevant platform for dissecting tumor-stroma interactions in peritoneal metastasis and may advance stromal-targeted therapeutic strategies.

Lucitanib plus toripalimab showed encouraging antitumor activity with manageable safety in heavily pretreated advanced solid tumors, supporting further randomized evaluation, particularly in NPC and EC. Trial Registration: Chinese Clinical Trial Registry Identifier: ChiCTR2400087935.

P2, N=24, Recruiting, Dana-Farber Cancer Institute | Not yet recruiting --> Recruiting

P2, N=50, Active, not recruiting, Advenchen Pharmaceuticals, LLC. | N=30 --> 50 | Recruiting --> Active, not recruiting

Functional analyses demonstrated that ARRB2 promotes the malignant progression of ICC by facilitating YAP nuclear translocation while also modulating the sensitivity of ICC to pemigatinib through the Raf-MEK-ERK signaling axis. This study identifies the tumor-promoting activities of ARRB2 and elucidates the regulatory mechanism of the METTL3-ARRB2-YAP/Raf axis in ICC, which may provide a novel prognostic biomarker and potential therapeutic target for human ICC.

P1/2, N=22, Not yet recruiting, Qed Therapeutics Inc.

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.

Early termination limited the ability to draw definitive conclusions on the efficacy of infigratinib as first-line treatment of FGFR2-rearranged CCA. This study illustrates the challenges of powering confirmatory studies in biomarker-selected subpopulations of rare tumors and highlights the need for regulatory collaboration to develop pragmatic frameworks for assessing novel therapies in ultra-rare malignancies.