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Ref Type Journal Article
PMID (30420614)
Authors Mazzaferro V, El-Rayes BF, Droz Dit Busset M, Cotsoglou C, Harris WP, Damjanov N, Masi G, Rimassa L, Personeni N, Braiteh F, Zagonel V, Papadopoulos KP, Hall T, Wang Y, Schwartz B, Kazakin J, Bhoori S, de Braud F, Shaib WL
Title Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma.
Journal British journal of cancer
Vol 120
Issue 2
Date 2019 01
URL
Abstract Text Next-generation sequencing has identified actionable genetic aberrations in intrahepatic cholangiocarcinomas (iCCA), including the fibroblast growth factor receptor 2 (FGFR2) fusions. Derazantinib (ARQ 087), an orally bioavailable, multi-kinase inhibitor with potent pan-FGFR activity, has shown preliminary therapeutic activity against FGFR2 fusion-positive iCCA.This multicentre, phase 1/2, open-label study enrolled adult patients with unresectable iCCA with FGFR2 fusion, who progressed, were intolerant or not eligible to first-line chemotherapy (NCT01752920). Subjects received derazantinib in continuous daily doses. Tumour response was assessed according to RECIST 1.1 every 8 weeks.Twenty-nine patients (18 women/11 men; median age, 58.7 years), 2 treatment-naive and 27 who progressed after at least one prior systemic therapy, were enrolled. Overall response rate was 20.7%, disease control rate was 82.8%. Estimated median progression-free survival was 5.7 months (95% CI: 4.04-9.2 months). Treatment-related adverse events (AE) were observed in 27 patients (93.1%, all grades), including asthenia/fatigue (69.0%), eye toxicity (41.4%), and hyperphosphatemia (75.9%). Grade ≥ 3 AEs occurred in 8 patients (27.6%).Derazantinib demonstrated encouraging anti-tumour activity and a manageable safety profile in patients with advanced, unresectable iCCA with FGFR2 fusion who progressed after chemotherapy. A pivotal trial of derazantinib in iCCA is ongoing (NCT03230318).

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
FGFR2 BFSP2 FGFR2 - BFSP2 fusion unknown FGFR2-BFSP2 results in a fusion of FGFR2 and BFSP2 (PMID: 30420614). FGFR2-BFSP2 has been identified in intrahepatic cholangiocarcinoma (PMID: 30420614), but has not been biochemically characterized and therefore, the effect on protein function is unknown (PubMed, Sep 2020).
FGFR2 SH3GLB1 FGFR2 - SH3GLB1 fusion unknown FGFR2-SH3GLB1 results from the fusion of FGFR2 and SH3GLB1 (PMID: 30420614). FGFR2-SH3GLB1 has been identified in intrahepatic cholangiocarcinoma (PMID: 30420614), but has not been biochemically characterized and therefore, the effect on protein function is unknown (PubMed, Sep 2020).
FGFR2 TACC1 FGFR2 - TACC1 fusion unknown FGFR2-TACC1 results from the fusion of FGFR2 and TACC1 (PMID: 30420614). FGFR2-TACC1 has been identified in intrahepatic cholangiocarcinoma (PMID: 30420614), but has not been biochemically characterized and therefore, the effect on protein function is unknown (PubMed, Sep 2020).
FGFR2 TACC2 FGFR2 - TACC2 fusion unknown FGFR2-TACC2 results from the fusion of FGFR2 and TACC2 (PMID: 30420614). FGFR2-TACC2 has been identified in intrahepatic cholangiocarcinoma (PMID: 30420614), but has not been biochemically characterized and therefore, the effect on protein function is unknown (PubMed, Sep 2020).
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FGFR2 - AHCYL1 intrahepatic cholangiocarcinoma predicted - sensitive Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, a patient with intrahepatic cholangiocarcinoma harboring FGFR2-AHCYL1 demonstrated stable disease when treated with Derazantinib (ARQ 087) (PMID: 30420614; NCT01752920). 30420614
FGFR2 - BFSP2 intrahepatic cholangiocarcinoma predicted - sensitive Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, a patient with intrahepatic cholangiocarcinoma harboring FGFR2-BFSP2 demonstrated stable disease when treated with Derazantinib (ARQ 087) (PMID: 30420614; NCT01752920). 30420614
FGFR2 - BICC1 intrahepatic cholangiocarcinoma predicted - sensitive Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, treatment with Derazantinib (ARQ 087) in four patients with intrahepatic cholangiocarcinoma harboring FGFR2-BICC1 resulted in one partial response, stable disease in two, and progressive disease in one (PMID: 30420614; NCT01752920). 30420614
FGFR2 - KIAA1217 intrahepatic cholangiocarcinoma predicted - sensitive Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, treatment with Derazantinib (ARQ 087) in two patients with intrahepatic cholangiocarcinoma harboring FGFR2-KIAA1217 resulted in one with a partial response and one with stable disease (PMID: 30420614; NCT01752920). 30420614
FGFR2 - TACC1 intrahepatic cholangiocarcinoma predicted - resistant Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, a patient with intrahepatic cholangiocarcinoma harboring FGFR2-TACC1 demonstrated progressive disease when treated with Derazantinib (ARQ 087) (PMID: 30420614; NCT01752920). 30420614
FGFR2 - CCAR2 intrahepatic cholangiocarcinoma predicted - sensitive Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, a patient with intrahepatic cholangiocarcinoma harboring FGFR2-CCAR2 demonstrated stable disease when treated with Derazantinib (ARQ 087) (PMID: 30420614; NCT01752920). 30420614
FGFR2 - CIT intrahepatic cholangiocarcinoma predicted - resistant Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, a patient with intrahepatic cholangiocarcinoma harboring FGFR2-CIT demonstrated progressive disease when treated with Derazantinib (ARQ 087) (PMID: 30420614; NCT01752920). 30420614
FGFR2 - SH3GLB1 intrahepatic cholangiocarcinoma predicted - sensitive Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, a patient with intrahepatic cholangiocarcinoma harboring FGFR2-SH3GLB1 demonstrated a partial response when treated with Derazantinib (ARQ 087) (PMID: 30420614; NCT01752920). 30420614
FGFR2 fusion intrahepatic cholangiocarcinoma sensitive Derazantinib Phase I Actionable In a Phase Ib/II trial, Derazantinib (ARQ 087) treatment resulted in an overall response rate of 20.7% (6/29), a disease control rate of 82.8% (24/29), and a median progression-free survival of 5.7 months in patients with intrahepatic cholangiocarcinoma harboring FGFR2 fusions (PMID: 30420614; NCT01752920). 30420614
FGFR2 - CCDC6 intrahepatic cholangiocarcinoma predicted - sensitive Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, a patient with intrahepatic cholangiocarcinoma harboring FGFR2-CCDC6 demonstrated stable disease when treated with Derazantinib (ARQ 087) (PMID: 30420614; NCT01752920). 30420614
FGFR2 - TACC2 intrahepatic cholangiocarcinoma predicted - sensitive Derazantinib Case Reports/Case Series Actionable In a Phase Ib/II trial, a patient with intrahepatic cholangiocarcinoma harboring FGFR2-TACC2 demonstrated sensitivity to treatment with Derazantinib (ARQ 087) (PMID: 30420614; NCT01752920). 30420614