Oncology · 3 h ago
Phase 2 FIDES-01 Reports Derazantinib Outcomes in FGFR2-Altered Cholangiocarcinoma
In the open-label, non-controlled Phase 2 FIDES-01 trial, derazantinib was evaluated in previously treated advanced intrahepatic cholangiocarcinoma with FGFR2 alterations. Among patients with FGFR2 fusions or rearrangements, the objective response rate was 22.3%; results in a separate, smaller subgroup with mutations or amplifications were also reported.
- Substudy 1 ORR was 22.3% in FGFR2 fusion/rearrangement iCCA.
- Substudy 2 PFS3 was 63% among 43 patients.
- No grade 4 or 5 drug-related events were reported.
- The open-label trial had no control group.
FIDES-01 was an open-label, non-controlled Phase 2 trial of once-daily oral derazantinib (300 mg) in patients with advanced intrahepatic cholangiocarcinoma (iCCA) who had received at least one prior chemotherapy regimen. The study enrolled patients with FGFR2 fusions or rearrangements in Substudy 1 and those with FGFR2 mutations or amplifications in Substudy 2. Tumour response was assessed by central radiology review using RECIST v1.1. The primary endpoints were objective response rate (ORR) in Substudy 1 and progression-free survival at three months (PFS3) in Substudy 2.
In Substudy 1, 23 of 103 patients had a partial response (ORR 22.3%); disease control rate was 75.7%, median progression-free survival was 8.1 months, and median overall survival was 17.2 months. In Substudy 2, PFS3 was 63% (27/43), ORR was 9.3% (four responses, including one complete response), and median overall survival was 11.9 months. No grade 4 or 5 drug-related adverse events were reported. Drug-related hyperphosphataemia occurred in 38% of patients (grade 3 in 3%) and dry eye in 36%.
The findings provide Phase 2 evidence of activity in previously treated, FGFR-inhibitor-naïve patients, but the trial had no control group. These results do not establish comparative benefit or support a change in treatment practice on their own.
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Hepatology: A pivotal Phase 2 study of derazantinib for previously-treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 aberrations: FIDES-01 ↗This is an automated AI-condensed summary that has not yet been reviewed by an editor. Always consult the full item at the original source.