Derazantinib May Demonstrate Promising Disease Control in Some Patients With Cholangiocarcinoma


Interim results from a phase 2 study demonstrated that patients inoperable or advanced intrahepatic cholangiocarcoma and a FGFR2 gene mutation had a disease control rate of 79% when treated with derazantinib.

Treatment with derazantinib in patients with inoperable or advanced intrahepatic cholangiocarcinoma and FGFR2 gene fusions demonstrated promising disease control, according to a press release from Basilea Pharmaceutica announcing results from an analysis of a phase 2 study.

“This supports the relevance of derazantinib in a group of patients with (inoperable or advanced intrahepatic cholangiocarcinoma) where there has been very limited clinical evidence of successful treatment with other FGFR inhibitors and confirms the broad potential of derazantinib as a monotherapy for the treatment of (inoperable or advanced intrahepatic cholangiocarcinoma) patients with diverse FGFR2 gene aberrations,” said Dr. Marc Engelhardt, chief medical officer at Basilea Pharmaceutica, in the release.

The interim analysis consisted of 14 evaluable patients with locally advanced or inoperable cholangiocarcinoma with at least one tumor assessment after the start of the study.

The disease control rate (percentage of patients who have achieved complete response, partial response and stable disease) was 79%. This included one patient who had a complete response, one patient with an unconfirmed partial response and nine patients with a best response of stable disease.

In addition, at least eight patients met the primary endpoint of progression-free survival (the time during and after treatment when a patient lives with the disease without worsening) of at least three months. Progression-free survival in the other patients will be assessed at a later analysis.

“We are very pleased with the positive interim results or this cohort of (inoperable or advanced intrahepatic cholangiocarcinoma) patients with FGFR2 gene mutations or amplifications,” Engelhardt said in the release. “The clinical benefit with derazantinib is similar to that reported for (inoperable or advanced intrahepatic cholangiocarcinoma) patients with FGFR2 gene fusions earlier this year.”

In the release, Engelhardt added that additional results are tentatively slated to be released next year.

“This outcome is very encouraging and further strengthens the evidence for the differentiation of derazantinib versus other FGFR inhibitors both from the efficacy and safety perspective,” Engelhardt said in the release. “We are now progressing the study to the next stage and expect topline results for cohort 2 in the first half of 2022.”

In a previous study with 29 patients, derazantinib showed antitumor activity with a favorable tolerability profile in this patient population with an overall response rate (percentage indicating patients with a partial or complete response to treatment) of 20.7% and median progression-free survival of 5.7 months. Common side effects included weakness/fatigue (69%), eye toxicity (41.4%) and increased phosphate levels in blood (75.9%). Side effects considered severe or worse occurred in 27.6% of patients.

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