^
7d
FOENIX-CCA2: A Study of TAS-120 in Patients With Advanced Solid Tumors (clinicaltrials.gov)
P1/2, N=407, Completed, Taiho Oncology, Inc. | Active, not recruiting --> Completed
Trial completion • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR3 mutation • FGFR fusion • FGFR2 rearrangement • FGFR3 fusion
|
Lytgobi (futibatinib)
11d
Biliary tract cancers: French national clinical practice guidelines for diagnosis, treatments and follow-up (TNCD, SNFGE, FFCD, UNICANCER, GERCOR, SFCD, SFED, AFEF, SFRO, SFP, SFR, ACABi, ACHBPT). (PubMed, Eur J Cancer)
These guidelines are intended to provide a personalised therapeutic strategy for daily clinical practice. Each individual BTC case should be discussed by a multidisciplinary team.
Clinical guideline • Review • Journal • PD(L)-1 Biomarker • IO biomarker
|
HER-2 (Human epidermal growth factor receptor 2) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
|
BRAF V600E • MSI-H/dMMR • HER-2 amplification • BRAF V600 • IDH1 mutation • FGFR2 mutation • FGFR2 rearrangement
|
cisplatin • Imfinzi (durvalumab) • gemcitabine • 5-fluorouracil • capecitabine • leucovorin calcium
2ms
Journal • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR2 rearrangement
|
Pemazyre (pemigatinib)
2ms
FIGHT-209: Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations (clinicaltrials.gov)
P2, N=83, Active, not recruiting, Incyte Corporation | Recruiting --> Active, not recruiting | N=164 --> 83 | Trial completion date: Jan 2026 --> Nov 2024 | Trial primary completion date: Jan 2026 --> Nov 2024
Enrollment closed • Enrollment change • Trial completion date • Trial primary completion date
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 mutation • FGFR2 fusion • FGFR1 mutation • FGFR fusion • FGFR2 rearrangement • FGFR1 fusion • FGFR1 rearrangement
|
Pemazyre (pemigatinib)
3ms
FOENIX-CCA3: Futibatinib Versus Gemcitabine-Cisplatin Chemotherapy as First-Line Treatment of Patients With Advanced Cholangiocarcinoma Harboring FGFR2 Gene Rearrangements (clinicaltrials.gov)
P3, N=216, Active, not recruiting, Taiho Oncology, Inc. | Trial completion date: Dec 2023 --> Jun 2024 | Trial primary completion date: Dec 2023 --> Jun 2024
Trial completion date • Trial primary completion date • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 rearrangement
|
cisplatin • gemcitabine • Lytgobi (futibatinib)
4ms
Circulating tumor DNA enables sensitive detection of actionable gene fusions and rearrangements across cancer types. (PubMed, Clin Cancer Res)
In contrast to some prior literature, this study reports detection of a wide variety of rearrangements in ctDNA. The prevalence of driver rearrangements in tissue and LBx was comparable when TF ≥1%. LBx presents a viable alternative when TBx is not available, and there may be less value in confirmatory testing when TF is sufficient.
Journal • Circulating tumor DNA
|
BRAF (B-raf proto-oncogene) • ALK (Anaplastic lymphoma kinase) • FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • RET rearrangement • FGFR2 rearrangement
|
FoundationOne® CDx • FoundationOne® Liquid CDx
4ms
Real-world use of pemigatinib (pemi) for cholangiocarcinoma (CCA) among racial and ethnic minorities in the United States. (ASCO-GI 2024)
The diverse population in this RWS is reflective of the heterogeneous CCA pt population in the US. These real-world overall response rates support the clinical benefit of pemi across racial and ethnic groups and complement the results of the clinical trial. >BL, baseline (date of 1st pemi prescription); CI, confidence interval; FGFR2, fibroblast growth factor receptor 2; P25-P75, 25-75th percentile; rwORR, real-world overall response rate; y, years.
Clinical • Real-world evidence • Real-world
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR2 rearrangement
|
Pemazyre (pemigatinib)
4ms
FGFR1/2/3-rearranged carcinoma of the head and neck: expanded histological spectrum crossing path with high-risk HPV in the sinonasal tract. (PubMed, Histopathology)
FHNC include a morphological spectrum of carcinomas with squamous features and may occur in different HN locations, such as parotid gland and the sinonasal tract. Sinonasal cases can harbour FGFR2 rearrangement with or without associated high-risk HPV. Timely recognition of FHNC could help select patients potentially amenable to targeted therapy with FGFR inhibitors. Further studies are needed (1) to determine if FGFR2 rearranged/HPV-positive sinonasal carcinomas are biologically distinct from HMSC, and (2) to elucidate the biological and clinical significance of FGFR2 rearrangement in the context of high-risk HPV.
Journal
|
FGFR2 (Fibroblast growth factor receptor 2) • FGFR3 (Fibroblast growth factor receptor 3) • FGFR1 (Fibroblast growth factor receptor 1) • TACC3 (Transforming acidic coiled-coil containing protein 3) • TACC2 (Transforming Acidic Coiled-Coil Containing Protein 2)
|
FGFR2 fusion • FGFR2 rearrangement • FGFR1 fusion • FGFR3 fusion
6ms
FOENIX-CCA3: Futibatinib Versus Gemcitabine-Cisplatin Chemotherapy as First-Line Treatment of Patients With Advanced Cholangiocarcinoma Harboring FGFR2 Gene Rearrangements (clinicaltrials.gov)
P3, N=216, Active, not recruiting, Taiho Oncology, Inc. | Trial completion date: Sep 2023 --> Dec 2023 | Trial primary completion date: Sep 2023 --> Dec 2023
Trial completion date • Trial primary completion date • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 rearrangement
|
cisplatin • gemcitabine • Lytgobi (futibatinib)
6ms
Cost-Effectiveness Analysis of Pemigatinib for the Treatment of Adult Patients with Locally Advanced or Metastatic Cholangiocarcinoma with a FGFR2 Fusion or Rearrangement That Have Progressed After Systemic Therapy in Greece (ISPOR-EU 2023)
OBJECTIVES: To evaluate the cost-effectiveness of pemigatinib compared to oxaliplatin-L-folinic-acid and fluorouracil plus active symptom control (mFOLFOX+ASC) and ASC alone for the treatment of patients with advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) rearrangement or fusion who have progressed on at least one line of prior systemic therapy in Greece. Pemigatinib, a therapy covering the unmet medical need of patients with previously treated locally advanced or metastatic CCA with an FGFR2 fusion or rearrangement, was estimated to be cost-effective compared with mFOLFOX+ASC and ASC alone in Greece.
Clinical • HEOR • Cost-effectiveness • Cost effectiveness • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR2 rearrangement
|
5-fluorouracil • oxaliplatin • Pemazyre (pemigatinib) • leucovorin calcium
6ms
Impact of mutation status on efficacy outcomes in TOPAZ-1: A Phase 3 study of durvalumab (D) or placebo (PBO) plus gemcitabine and cisplatin (+GC) in advanced biliary tract cancer (BTC) (DGHO 2023)
BTC mutation prevalence in TOPAZ-1 by primary tumour location and geographical region was consistent with other studies. Although pt numbers were low and the confidence intervals were broad, pts with clinically actionable alterations ( IDH1 , BRAF and BRCA1/2 mutations and FGFR2 rearrangements) appeared to benefit from D+GC. Previously presented at ESMO Asia Congress, FPN 680, Juan Valle et al.
Clinical • P3 data • BRCA Biomarker • PD(L)-1 Biomarker • Metastases
|
HER-2 (Human epidermal growth factor receptor 2) • KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • TP53 (Tumor protein P53) • BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • ARID1A (AT-rich interaction domain 1A) • MTAP (Methylthioadenosine Phosphorylase) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B)
|
BRCA2 mutation • BRCA1 mutation • BRAF mutation • FGFR2 mutation • FGFR2 rearrangement
|
cisplatin • Imfinzi (durvalumab) • gemcitabine
6ms
Molecular Detection of FGFR2 Rearrangements in Resected Intrahepatic Cholangiocarcinomas: FISH Could Be An Ideal Method in Patients with Histological Small Duct Subtype. (PubMed, J Clin Transl Hepatol)
FISH achieved satisfactory concordance with NGS, has potential value for FGFR2 screening for targeted therapies. FGFR2 detection should be prioritized for unique clinical subgroups in ICC, which features a histological small duct subtype, early clinical stage, and reduced mucus production.
Journal • PD(L)-1 Biomarker • IO biomarker
|
PD-L1 (Programmed death ligand 1) • KRAS (KRAS proto-oncogene GTPase) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • BAP1 (BRCA1 Associated Protein 1) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B) • BICC1 (BicC Family RNA Binding Protein 1)
|
PD-L1 expression • KRAS mutation • IDH1 mutation • FGFR2 mutation • FGFR2 fusion • FGFR2 rearrangement • FGFR2 expression • FGFR2b expression
7ms
Evaluation of the Mass Balance and Metabolic Profile of Futibatinib in Healthy Participants. (PubMed, Clin Pharmacol Drug Dev)
These data indicate the primary metabolic pathways of futibatinib are O-desmethylation and glutathione conjugation, with cytochrome P450 enzyme-mediated desmethylation as the main oxidation pathway. C-futibatinib was well tolerated in this Phase 1 study.
Journal
|
FGFR2 (Fibroblast growth factor receptor 2) • FGFR1 (Fibroblast growth factor receptor 1)
|
FGFR2 rearrangement
|
Lytgobi (futibatinib)
8ms
Prognostic significance of somatic DNA gene rearrangement and structural atypia in metastatic breast cancer (ESMO 2023)
Conclusions MBC patients with one or more DNA gene rearrangements and structural atypia had a significantly better prognosis. Further investigations of the prognostic and biological significance of these alterations in MBC patients are warranted.
Metastases
|
FGFR2 (Fibroblast growth factor receptor 2) • FGFR1 (Fibroblast growth factor receptor 1) • NOTCH2 (Notch 2)
|
FGFR2 rearrangement • FGFR1 rearrangement
|
FoundationOne® CDx
9ms
Futibatinib, an Irreversible FGFR1-4 Inhibitor for the Treatment of FGFR-Aberrant Tumors. (PubMed, Oncologist)
These data show clinically meaningful benefit with futibatinib in FGFR2-rearrangement-positive cholangiocarcinoma and provide support for further investigation of futibatinib across other indications. Future directions for this agent include elucidating mechanisms of resistance and exploration of combination therapy approaches.
Journal
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR mutation • FGFR2 rearrangement
|
Lytgobi (futibatinib)
9ms
A Study to Assess Safety, Tolerability, and Pharmacokinetics of ABSK121-NX in Patients With Advanced Solid Tumors (clinicaltrials.gov)
P1, N=169, Recruiting, Abbisko Therapeutics Co, Ltd | Not yet recruiting --> Recruiting | Initiation date: Dec 2022 --> Jun 2023
Enrollment open • Trial initiation date • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 mutation • FGFR2 fusion • FGFR2 amplification • FGFR3 mutation • FGFR3 S249C • FGFR2 rearrangement • FGFR3 Y373C • FGFR3 G370C • FGFR3 R248C
|
ABSK121
10ms
Enrollment open • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR2 rearrangement
|
Lytgobi (futibatinib)
11ms
Are FGFR and IDH1-2 alterations a positive prognostic factor in intrahepatic cholangiocarcinoma? An unresolved issue. (PubMed, Front Oncol)
On the other hand, the positive prognostic role of IDH1/2 mutation seems much more uncertain. In this scenario, better designed clinical trials in these subsets of iCCA patients are needed in order to get definitive conclusions on this issue.
Review • Journal
|
FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • FGFR (Fibroblast Growth Factor Receptor)
|
FGFR2 mutation • FGFR2 rearrangement
11ms
Early-onset biliary tract cancer: An overview of clinical presentation, risk factors, molecular profile and outcome in a multicentric Italian cohort (ESMO-GI 2023)
Although family history seems not relevant in developing BTC in unselected population, in our experience a family and/or personal history of cancer was reported in a not negligible percentage of cases of EOBTC while the other known risk factor were less represented. This information, together with those given by an extensive molecular assessment, could help in identifying not only druggable but even genetic alterations involved in hereditary syndromes, and in selecting patients to address to a genetic counselling.
Clinical • BRCA Biomarker
|
HER-2 (Human epidermal growth factor receptor 2) • BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset) • MSI (Microsatellite instability) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
|
BRCA2 mutation • HER-2 overexpression • IDH1 mutation • FGFR2 mutation • FGFR2 rearrangement
11ms
Clinicopathological, etiological and molecular characteristics of intrahepatic cholangiocarcinoma subtypes classified by mucin production and immunohistochemical features. (PubMed, Expert Rev Mol Diagn)
Furthermore, positive FGFR2 rearrangement occurred only in small duct type ICC and IDH1/2 was mutated mainly in small duct type ICC. The subclassification system was applicable and the ICC subtypes had distinct clinicopathological characteristics, prognostic outcome, and IDH1/2 mutation pattern.
Journal
|
KRAS (KRAS proto-oncogene GTPase) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
|
KRAS mutation • FGFR2 mutation • FGFR2 rearrangement • FGFR2 translocation
12ms
FOENIX-CCA3: Futibatinib Versus Gemcitabine-Cisplatin Chemotherapy as First-Line Treatment of Patients With Advanced Cholangiocarcinoma Harboring FGFR2 Gene Rearrangements (clinicaltrials.gov)
P3, N=216, Active, not recruiting, Taiho Oncology, Inc. | Trial completion date: Mar 2023 --> Sep 2023 | Trial primary completion date: Mar 2023 --> Sep 2023
Trial completion date • Trial primary completion date • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 rearrangement
|
cisplatin • gemcitabine • Lytgobi (futibatinib)
1year
Resistance to selective FGFR inhibitors in FGFR-driven urothelial cancer (AACR 2023)
Patients progressed to erdafitinib (n = 14), futibatinib (n = 4) or pemigatinib (n = 3)...In patient-derived models, erdafitinib combined with pictilisib was synergic in the presence of PIK3CA E545K, while erdafitinib-gefitinib combination overcame bypass resistance mediated by EGFR activation.Conclusions We detected a high frequency of FGFR kinase domain mutations at progression to selective FGFR inhibitors in urothelial cancer. Unlike FGFR2-driven cholangiocarcinoma, polyclonality of FGFR3 kinase domain mutations does not seem to be a hallmark of FGFR-driven urothelial cancer. The identification of off-target events and the preclinical validation sustain the potential clinical applicability of combinatorial treatment strategies.
EGFR (Epidermal growth factor receptor) • PIK3CA (Phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha) • FGFR2 (Fibroblast growth factor receptor 2) • PTEN (Phosphatase and tensin homolog) • FGFR3 (Fibroblast growth factor receptor 3) • TACC3 (Transforming acidic coiled-coil containing protein 3) • FGFR4 (Fibroblast growth factor receptor 4) • TSC1 (TSC complex subunit 1) • STING (stimulator of interferon response cGAMP interactor 1)
|
PTEN mutation • FGFR2 mutation • PIK3CA E545K • FGFR2 fusion • FGFR3 mutation • FGFR2 rearrangement • FGFR3 fusion • FGFR4 mutation • TSC1 mutation • PIK3CA E545 • FGFR3 V555M
|
FoundationOne® CDx
|
gefitinib • Balversa (erdafitinib) • Lytgobi (futibatinib) • Pemazyre (pemigatinib) • pictilisib (GDC-0941)
1year
New P2 trial
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR2 rearrangement
|
Lytgobi (futibatinib)
1year
FOENIX-CCA3: Futibatinib Versus Gemcitabine-Cisplatin Chemotherapy as First-Line Treatment of Patients With Advanced Cholangiocarcinoma Harboring FGFR2 Gene Rearrangements (clinicaltrials.gov)
P3, N=216, Active, not recruiting, Taiho Oncology, Inc. | Recruiting --> Active, not recruiting | Trial completion date: Apr 2028 --> Mar 2023 | Trial primary completion date: Apr 2027 --> Mar 2023
Enrollment closed • Trial completion date • Trial primary completion date • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 rearrangement
|
cisplatin • gemcitabine • Lytgobi (futibatinib)
1year
Futibatinib for FGFR2-Rearranged Intrahepatic Cholangiocarcinoma. (PubMed, N Engl J Med)
In previously treated patients with FGFR2 fusion or rearrangement-positive intrahepatic cholangiocarcinoma, the use of futibatinib, a covalent FGFR inhibitor, led to measurable clinical benefit. (Funded by Taiho Oncology and Taiho Pharmaceutical; FOENIX-CCA2 ClinicalTrials.gov number, NCT02052778.).
Journal
|
TP53 (Tumor protein P53) • FGFR2 (Fibroblast growth factor receptor 2)
|
TP53 mutation • FGFR2 fusion • FGFR2 rearrangement
|
Lytgobi (futibatinib)
over1year
Clinical and mutational profiles of microsatellite instability (MSI-H) in intrahepatic cholangiocarcinoma (iCCA). (ASCO-GI 2023)
Gemcitabine/Cisplatin was given to 3 of 6 patients as first-line (1L) therapy, with median progression free survival of 4 months. MSI-H represents a small subset of patients with iCCA. High-TMB noted amongst MSI-H iCCA. Currently, MSI testing is not universally performed and may be necessary to identify and best treat these patients.
Clinical • Microsatellite instability • Tumor mutational burden • MSi-H Biomarker • BRCA Biomarker • IO biomarker
|
TP53 (Tumor protein P53) • PIK3CA (Phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha) • TMB (Tumor Mutational Burden) • BRCA2 (Breast cancer 2, early onset) • MSI (Microsatellite instability) • FGFR2 (Fibroblast growth factor receptor 2) • PTEN (Phosphatase and tensin homolog) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • ARID1A (AT-rich interaction domain 1A) • MTAP (Methylthioadenosine Phosphorylase) • MLH1 (MutL homolog 1) • BRCA (Breast cancer early onset)
|
TP53 mutation • TMB-H • MSI-H/dMMR • ARID1A mutation • FGFR2 mutation • FGFR2 fusion • FGFR2 rearrangement • BRCA mutation
|
FoundationOne® CDx
|
cisplatin • gemcitabine
over1year
New P1 trial • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 mutation • FGFR2 fusion • FGFR2 amplification • FGFR3 mutation • FGFR3 S249C • FGFR2 rearrangement • FGFR3 Y373C • FGFR3 G370C • FGFR3 R248C
|
ABSK121
over1year
Pemigatinib in cholangiocarcinoma with a FGFR2 rearrangement or fusion. (PubMed, Expert Rev Anticancer Ther)
This article reviews FGFR and its dysregulation in oncogenesis, FGFR inhibitors, especially pemigatinib, utilized in treatment of CCA, common adverse events associated with FGFR inhibitors, and future directions in the field of targeted drug development for CCA. FGFR inhibitors, including pemigatinib, have shown promise in the management of CCA with FGFR2 fusion or rearrangement; however, acquired resistance remains a major barrier in the field of FGFR inhibitors and requires further study.
Journal
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR2 rearrangement
|
Pemazyre (pemigatinib)
over1year
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, Incyte Corporation | Trial completion date: Feb 2028 --> Jul 2028 | Trial primary completion date: Mar 2027 --> Oct 2027
Trial completion date • Trial primary completion date • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 rearrangement
|
cisplatin • gemcitabine • Pemazyre (pemigatinib)
over1year
CIBI375A201: Pemigatinib in Treating Patients With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma Including FGFR2 Rearrangement (clinicaltrials.gov)
P2, N=34, Active, not recruiting, Innovent Biologics (Suzhou) Co. Ltd. | Trial completion date: Jun 2022 --> Dec 2023 | Trial primary completion date: Jan 2021 --> Jun 2022
Trial completion date • Trial primary completion date • Metastases
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 rearrangement
|
Pemazyre (pemigatinib)
over1year
Profiling of gene fusion involving targetable genes in Chinese gastric cancer. (PubMed, World J Gastrointest Oncol)
We characterized the landscape of fusions involving targetable genes in a Chinese GC cohort and found that 1.68% of patients with GC harbor potential targetable gene fusions, which were enriched in patients with ERBB2 amplification. Gene fusion detection may provide a potential treatment strategy for patients with GC with disease progression following standard therapy.
Journal • Tumor Mutational Burden • IO biomarker
|
EGFR (Epidermal growth factor receptor) • BRAF (B-raf proto-oncogene) • ALK (Anaplastic lymphoma kinase) • TMB (Tumor Mutational Burden) • MSI (Microsatellite instability) • FGFR2 (Fibroblast growth factor receptor 2) • FGFR3 (Fibroblast growth factor receptor 3) • EML4 (EMAP Like 4) • NTRK2 (Neurotrophic tyrosine kinase, receptor, type 2) • NRG1 (Neuregulin 1) • SEPTIN14 (Septin 14) • NTRK (Neurotrophic receptor tyrosine kinase)
|
HER-2 amplification • NTRK2 fusion • FGFR2 fusion • ALK fusion • NRG1 fusion • FGFR2 rearrangement • FGFR3 fusion • NRG1 fusion • NTRK positive • NTRK fusion
|
Vitrakvi (larotrectinib) • zenocutuzumab (MCLA-128)
over1year
LCK inhibition downregulates YAP activity and is therapeutic in patient-derived models of cholangiocarcinoma. (PubMed, J Hepatol)
A novel LCK inhibitor, NTRC 0652-0, inhibited YAP signaling and demonstrated preclinical efficacy in CCA cell-lines, and patient derived organoid and xenograft models.
Journal
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR2 rearrangement
over1year
Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study. (PubMed, Cancer Med)
The encouraging antitumor activity and favorable safety profile support the use of pemigatinib as a treatment in previously treated Chinese patients with cholangiocarcinoma and FGFR2 rearrangements.
P2 data • Journal
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 fusion • FGFR2 rearrangement
|
Pemazyre (pemigatinib)
over1year
Trial completion date • Trial primary completion date
|
EGFR (Epidermal growth factor receptor) • FGFR2 (Fibroblast growth factor receptor 2) • TERT (Telomerase Reverse Transcriptase)
|
EGFR amplification • FGFR2 mutation • FGFR2 fusion • FGFR1 mutation • FGFR fusion • FGFR2 rearrangement • FGFR1 fusion • TERT mutation • FGFR1 rearrangement • IDH wild-type • TERT promoter mutation
|
Pemazyre (pemigatinib)
over1year
Molecular therapeutic targets for cholangiocarcinoma: Present challenges and future possibilities. (PubMed, Adv Cancer Res)
As treatment resistance is an inevitability in advanced disease, resistance mechanisms require characterization to guide the development of combination therapies to increase the duration of clinical benefit. Patient-focused clinical, technological and analytical synergy is needed to deliver future solutions to these present therapeutic challenges.
Journal • IO biomarker
|
FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
|
IDH1 mutation • FGFR2 mutation • FGFR2 rearrangement
over1year
FOENIX-CCA3: Futibatinib Versus Gemcitabine-Cisplatin Chemotherapy as First-Line Treatment of Patients With Advanced Cholangiocarcinoma Harboring FGFR2 Gene Rearrangements (clinicaltrials.gov)
P3, N=216, Recruiting, Taiho Oncology, Inc. | Trial completion date: Feb 2026 --> Apr 2028 | Trial primary completion date: Apr 2025 --> Apr 2027
Trial completion date • Trial primary completion date
|
FGFR2 (Fibroblast growth factor receptor 2)
|
FGFR2 rearrangement
|
cisplatin • gemcitabine • Lytgobi (futibatinib)
over1year
Tumor Mutational Burden • MSi-H Biomarker
|
EGFR (Epidermal growth factor receptor) • HER-2 (Human epidermal growth factor receptor 2) • KRAS (KRAS proto-oncogene GTPase) • TP53 (Tumor protein P53) • TMB (Tumor Mutational Burden) • MSI (Microsatellite instability) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • FGFR3 (Fibroblast growth factor receptor 3) • FGFR1 (Fibroblast growth factor receptor 1) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • FGFR (Fibroblast Growth Factor Receptor) • BAP1 (BRCA1 Associated Protein 1) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B) • ATRX (ATRX Chromatin Remodeler) • STAG2 (Stromal Antigen 2)
|
TMB-H • MSI-H/dMMR • FGFR2 mutation • FGFR2 rearrangement
almost2years
Clinical, Genomic, and Transcriptomic Data Profiling of Biliary Tract Cancer Reveals Subtype-Specific Immune Signatures. (PubMed, JCO Precis Oncol)
BTC subtypes exhibit diverse DNA alterations, RNA inflammatory signatures, and immune biomarkers. The association between specific BTC anatomical subsets, molecular alterations, and immunophenotypes highlights new opportunities for therapeutic development.
Journal • IO biomarker
|
KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • TP53 (Tumor protein P53) • FGFR2 (Fibroblast growth factor receptor 2) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • ATM (ATM serine/threonine kinase) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • ARID1A (AT-rich interaction domain 1A) • PBRM1 (Polybromo 1) • BAP1 (BRCA1 Associated Protein 1) • LRP1B (LDL Receptor Related Protein 1B) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B) • KMT2C (Lysine Methyltransferase 2C)
|
TP53 mutation • KRAS mutation • ATM mutation • FGFR2 mutation • FGFR2 fusion • BAP1 mutation • FGFR2 rearrangement