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Clinical Trial
. 2024 Jun;9(6):103488.
doi: 10.1016/j.esmoop.2024.103488. Epub 2024 Jun 4.

An open-label study of pemigatinib in cholangiocarcinoma: final results from FIGHT-202

A Vogel  1 , V Sahai  2 , A Hollebecque  3 , G M Vaccaro  4 , D Melisi  5 , R M Al Rajabi  6 , A S Paulson  7 , M J Borad  8 , D Gallinson  9 , A G Murphy  10 , D-Y Oh  11 , E Dotan  12 , D V Catenacci  13 , E Van Cutsem  14 , C F Lihou  15 , H Zhen  15 , M L Veronese  16 , G K Abou-Alfa  17
Affiliations
Clinical Trial

An open-label study of pemigatinib in cholangiocarcinoma: final results from FIGHT-202

A Vogel et al. ESMO Open. 2024 Jun.

Abstract

Background: Fibroblast growth factor receptor 2 (FGFR2) fusions and rearrangements are clinically actionable genomic alterations in cholangiocarcinoma (CCA). Pemigatinib is a selective, potent, oral inhibitor of FGFR1-3 and demonstrated efficacy in patients with previously treated, advanced/metastatic CCA with FGFR2 alterations in FIGHT-202 (NCT02924376). We report final outcomes from the extended follow-up period.

Patients and methods: The multicenter, open-label, single-arm, phase II FIGHT-202 study enrolled patients ≥18 years old with previously treated advanced/metastatic CCA with FGFR2 fusions or rearrangements (cohort A), other FGF/FGFR alterations (cohort B), or no FGF/FGFR alterations (cohort C). Patients received once-daily oral pemigatinib 13.5 mg in 21-day cycles (2 weeks on, 1 week off) until disease progression or unacceptable toxicity. The primary endpoint was objective response rate (ORR) in cohort A assessed as per RECIST v1.1 by an independent review committee; secondary endpoints included duration of response (DOR), progression-free survival (PFS), overall survival (OS), and safety.

Results: FIGHT-202 enrolled 147 patients (cohort A, 108; cohort B, 20; cohort C, 17; unconfirmed FGF/FGFR alterations, 2). By final analysis, 145 (98.6%) had discontinued treatment due to progressive disease (71.4%), withdrawal by patient (8.2%), or adverse events (AEs; 6.8%). Median follow-up was 45.4 months. The ORR in cohort A was 37.0% (95% confidence interval 27.9% to 46.9%); complete and partial responses were observed in 3 and 37 patients, respectively. Median DOR was 9.1 (6.0-14.5) months; median PFS and OS were 7.0 (6.1-10.5) months and 17.5 (14.4-22.9) months, respectively. The most common treatment-emergent AEs (TEAEs) were hyperphosphatemia (58.5%), alopecia (49.7%), and diarrhea (47.6%). Overall, 15 (10.2%) patients experienced TEAEs leading to pemigatinib discontinuation; intestinal obstruction and acute kidney injury (n = 2 each) occurred most frequently.

Conclusions: Pemigatinib demonstrated durable response and prolonged OS with manageable AEs in patients with previously treated, advanced/metastatic CCA with FGFR2 alterations in the extended follow-up period of FIGHT-202.

Keywords: fibroblast growth factor receptor; intrahepatic cholangiocarcinoma; next-generation sequencing; pemigatinib; precision medicine; targeted therapy.

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Figures

Figure 1
Figure 1
Best percentage change from baseline in target lesion size based on IRC assessment among efficacy-assessable patients in cohort A (FGFR2 rearrangements or fusions). The dashed line indicates criterion for PR (≥30% decrease in sum of target lesion diameters). CR, complete response; FGFR, fibroblast growth factor receptor; IRC, independent review committee; PD, progressive disease; PR, partial response; SD, stable disease.
Figure 2
Figure 2
Kaplan–Meier estimates of (A) PFS based on IRC assessment, (B) OS for all cohorts, and (C) OS in cohort A stratified by response (efficacy-assessable population). IRC, independent review committee; NE, not estimable; OS, overall survival; PFS, progression-free survival.
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