Pemazyre (pemigatinib) / Incyte, Innovent Biologics, Knight Therap, Specialised Therap - LARVOL DELTA

PEMIGATINIB-ASSOCIATED CALCIPHYLAXIS VERSUS CALCINOSIS CUTIS IN METASTATIC CHOLANGIOCARCINOMA (ISN-WCN 2026) - "This case illustrates pemigatinib-associated calcific skin disease presenting as probable non-uremic calciphylaxis. Accurate differentiation from calcinosis cutis is essential for guiding therapy, including the judicious use of sodium thiosulfate and other treatments."

Metastases • Biliary Cancer • Calciphylaxis • Cholangiocarcinoma • Infectious Disease • Metabolic Disorders • Nephrology • Oncology • Rare Diseases • Renal Disease • Septic Shock • Solid Tumor • Vasculitis • BICC1 • FGF23 • 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

First Report of Psoriasis Flare Following Pemigatinib Therapy for Cholangiocarcinoma (AAD 2026) - "Hydrocortisone was discontinued and treatment with fluocinonide 0.05% solution and ketoconazole 2% shampoo resulted in resolution of the scalp lesions. Known side effects of pemigatinib include ocular toxicity, hyperphosphatemia, and soft tissue mineralization[5]. To our knowledge, this is the first reported case of pemigatinib-induced psoriasis, highlighting the need for awareness of this potential adverse effect."

Biliary Cancer • Cholangiocarcinoma • Dermatology • Immunology • Infectious Disease • Metabolic Disorders • Mood Disorders • Nephrology • Oncology • Psoriasis • Renal Disease • Solid Tumor • FGFR

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

Hair loss in cancer patients receiving small molecule inhibitors: Evidence from clinical trials (AACR 2026) - "Hedgehog pathway inhibitors induced the highest overall rates, ranging from 20-30% with saridegib and glasdegib, and up to 63% with vismodegib. FGFR inhibitors (pemigatinib, infigratinib) and other kinase inhibitors (ripretinib, vemurafenib, sorafenib, AZD0424) demonstrated moderate rates (22-48%). Lower alopecia incidence was reported with aromatase inhibitors (anastrozole, lenostrozole) and the aurora B kinase inhibitor BI 811283, ranging from 9 to 21%...The higher incidences seen with Hedgehog inhibitors, compared with aromatase inhibitors, highlight how pathway-specific disruption of follicular signaling contributes to hair loss. Further research into the molecular mechanisms of these agents may help clinicians better anticipate and manage this side effect."

Clinical • Oncology • BRAF

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

Prognostic impact of concomitant genomic alterations in FGFR2-positive cholangiocarcinoma treated with pemigatinib: Molecular analysis of the Italian real-world study (ESMO-TAT 2026) - "In this real-world, multicenter cohort of patients with FGFR2-positive CCA treated with pemigatinib, TP53 mutations were associated with significantly worse clinical outcomes, suggesting a negative prognostic role."

Biomarker • Clinical • Real-world • Real-world evidence • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • BAP1 • CDKN2A • CDKN2B • FGFR2 • IDH1 • PTEN • TP53

Clinical insights and treatment outcomes of SDH deficient gastrointestinal stromal tumors (GIST): A retrospective analysis from a tertiary care center in India (Sarcoma-RC 2026) - "Response rates were: imatinib, 0% (n=8); sunitinib, 14.3% (2/14); regorafenib, 37.5% (3/8); and temozolomide, 20% (1/5). No responses were noted for cabozantinib (n=2), pemigatinib (n=1), pazopanib (n=1), or ripretinib (n=1); however, one patient had a partial response to doxorubicin...Legal entity responsible for the study The authors. Funding Has not received any funding."

Retrospective data • Stroma • Gastrointestinal Stromal Tumor • Hepatology • Oncology • Sarcoma • SDHB

Use of FGFR inhibitors for treatment of primary CNS tumors (Sarcoma-RC 2026) - "Prior treatments included surgical resection (8, 89%), radiation (7, 78%), temozolomide (7, 78%), bevacizumab (3, 33%), TPCV (1, 11%) and regorafenib (1, 11%)...FGFR inhibitors used included FGFR1-3 inhibitors infigratinib (n=1, 11%) and pemigatinib (n=4, 44%) as well as FGFR1-4 inhibitor erdafitinib (n=4, 44)...Legal entity responsible for the study The authors. Funding Has not received any funding."

Astrocytoma • Brain Cancer • CNS Tumor • Dental Disorders • Glioblastoma • Glioma • Metabolic Disorders • Nephrology • Oncology • Pilocytic Astrocytoma • Renal Disease • Solid Tumor • Stomatitis • FGFR2 • FGFR3

Expanding the Kidney–Skin Spectrum: Pemigatinib-Induced Nonuremic Calciphylaxis (NKF-SCM 2026) - "RESULTS/CASE DISCUSSION 58-year-old woman with HBV-related decompensated cirrhosis and FGFR2-altered intrahepatic cholangiocarcinoma—previously treated with durvalumab/tremelimumab and then carboplatin/gemcitabine/durvalumab—presented after completing four cycles of pemigatinib 13.5 mg daily (2 weeks on/1 week off), with her last dose 10 days earlier...Because of the extensive body-surface involvement, she received intravenous rather than intralesional sodium thiosulfate (20 mg IV three times weekly), pentoxifylline 400 mg three times weekly, phosphate binders, wound care, and analgesia...Further study is needed to clarify mechanisms and define true incidence. Clinicians should maintain vigilance for early cutaneous changes in patients receiving FGFR-targeted therapies."

Biliary Cancer • Calciphylaxis • Cholangiocarcinoma • Fibrosis • Hepatology • Immunology • Metabolic Disorders • Nephrology • Rare Diseases • Renal Disease • Solid Tumor • FGFR2

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

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

Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement and Pemigatinib. (PubMed, Blood) - "Outside allogeneic stem cell transplantation (ASCT), survival with conventional therapy is dismal, representing an unmet clinical need. We summarize here the data that led to approval of pemigatinib, a FGFR1 inhibitor, showing unprecedented efficacy in M/LN-FGFR1."

Journal • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Oncology • Transplantation • FGFR1

VPS33A Promotes Pemigatinib Resistance in Cholangiocarcinoma via Autophagy. (PubMed, Dig Dis Sci) - "VPS33A-mediated autophagy sustains Pemigatinib resistance in CCA through ULK1, revealing a targetable vulnerability that could help overcome current therapeutic limitations."

Journal • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • ULK1

FIGHT-302: A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (clinicaltrials.gov) - P3 | N=432 | Recruiting | Sponsor: Incyte Corporation | Trial completion date: Oct 2024 ➔ Jun 2026

Trial completion date • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • FGFR2

The FGFR1 N546K mutation confers resistance to pemigatinib in MLN-ZMYM2::FGFR1. (PubMed, Leukemia) - No abstract available

Journal • FGFR1 • ZMYM2

FIGHT-302: A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (clinicaltrials.gov) - P3 | N=167 | Active, not recruiting | Sponsor: Incyte Corporation | Recruiting ➔ Active, not recruiting | N=434 ➔ 167

Enrollment change • Enrollment closed • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • FGFR2

FIGHT-302: A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (clinicaltrials.gov) - P3 | N=434 | Recruiting | Sponsor: Incyte Corporation | Trial completion date: Jun 2026 ➔ Feb 2028 | Trial primary completion date: Oct 2023 ➔ Mar 2027

Trial completion date • Trial primary completion date • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • FGFR2

FIGHT-302: A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (clinicaltrials.gov) - P3 | N=432 | Recruiting | Sponsor: Incyte Corporation | Trial completion date: Mar 2023 ➔ Oct 2024 | Trial primary completion date: Mar 2022 ➔ Oct 2023

Trial completion date • Trial primary completion date • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • FGFR2

PEMIGIST: PH 2 Pemigatinib in SDH-deficient GIST (clinicaltrials.gov) - P2 | N=24 | Not yet recruiting | Sponsor: Dana-Farber Cancer Institute

New P2 trial • Gastrointestinal Stromal Tumor • Oncology • Sarcoma

FIGHT-302: A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (clinicaltrials.gov) - P3 | N=167 | Terminated | Sponsor: Incyte Corporation | Trial completion date: Jul 2028 ➔ Jul 2025 | Active, not recruiting ➔ Terminated | Trial primary completion date: Oct 2027 ➔ Jul 2025; The study was terminated due to lack of enrollment resulting from a change in the standard of care for the first-line treatment of patients with cholangiocarcinoma. There were no safety concerns that contributed to this decision.

Trial completion date • Trial primary completion date • Trial termination • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • FGFR2

FIGHT-302: A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (clinicaltrials.gov) - P3 | N=434 | Recruiting | Sponsor: Incyte Corporation | Trial completion date: Feb 2028 ➔ Jul 2028 | Trial primary completion date: Mar 2027 ➔ Oct 2027

Trial completion date • Trial primary completion date • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • FGFR2

FIGHT-302: A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (clinicaltrials.gov) - P3 | N=432 | Not yet recruiting | Sponsor: Incyte Corporation

New P3 trial • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • FGFR2

FIGHT-302: A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (clinicaltrials.gov) - P3 | N=432 | Recruiting | Sponsor: Incyte Corporation | Not yet recruiting ➔ Recruiting

Enrollment open • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • FGFR2