Basilea posts interim pivotal data on ArQule-partnered cancer drug

Basel, Switzerland
Basel, Switzerland, where Basilea is based (Getty Images/Xantana)

Basilea Pharmaceutica has shared interim data from a registrational trial of ArQule-partnered cancer drug derazantinib. The oral FGFR kinase inhibitor achieved an objective response rate of 21% in the first 29 evaluable intrahepatic cholangiocarcinoma (iCCA) patients enrolled in the study.

Derazantinib is designed to counter the pan-fibroblast growth factor receptor dysregulation that drives the bile duct cancer iCCA and the growth of multiple other types of tumors. In the case of iCCA, ArQule and Basilea are hoping to offer patients with tumors harboring FGFR2 fusions a better option than surgery and chemotherapy, which are associated with low response rates and short progression-free survival times. 

A 2012 analysis of the efficacy of chemotherapy in biliary tract carcinomas, including iCCA, linked the treatment to response rates ranging from 10% to 40%, although those figures come with caveats about the designs of the studies. The five-year overall survival rate is less than 15%.


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To test whether derazantinib can deliver better outcomes, Basilea is enrolling up to 100 patients with inoperable or advanced iCCA expressing FGFR2 gene fusions in a registrational open-label phase 2 study. Basilea presented interim data on the first 29 patients enrolled in the trial today.

Six of the subjects experienced partial responses, resulting in an objective response rate of 21%. The partial responses contributed to a disease control rate of 83%. Basilea thinks the results suggest the drug may have a future. 

“The response rate and the safety profile at the time of the interim analysis are promising, especially considering the poor outcomes with chemotherapy in this group of patients reported in the literature,” Basilea Chief Medical Officer Marc Engelhardt said in a statement.

ArQule and Basilea will have a fuller picture of the efficacy of the drug around the middle of next year, when the study is due to finish. In parallel, Basilea is preparing to start a phase 2 study in other types of tumor that are driven by FGFR. That study is due to get underway around the middle of this year.

Shares in Basilea rose and dipped slightly following the release of the results.