Basilea reports positive interim results from registrational phase 2 study with oncology drug candidate derazantinib in intrahepatic cholangiocarcinoma (iCCA)

Basilea Pharmaceutica AG / Basilea reports positive interim results from registrational phase 2 study with oncology drug candidate derazantinib in intrahepatic cholangiocarcinoma (iCCA) . Processed and transmitted by West Corporation. The issuer is solely responsible for the content of this announcement.

• 21% objective response rate with six confirmed partial responses from 29 evaluable patients
• 83% disease control rate

Safety profile and tolerability of continuous dosing schedule confirmed

             
Basel, Switzerland, January 09, 2019 - Basilea Pharmaceutica Ltd. (SIX: BSLN) announced today results from the interim analysis of the registrational phase 2 study with the orally administered pan-fibroblast growth factor receptor (FGFR) kinase inhibitor derazantinib (BAL087). The analysis showed promising efficacy in patients with FGFR2 gene fusion-expressing intrahepatic cholangiocarcinoma (iCCA) and also confirmed the safety profile and tolerability of the drug candidate observed in previous clinical studies.

The interim analysis in the ongoing registrational phase 2 study was conducted after 42 patients have been enrolled in the study, with a subset of 29 evaluable patients who had at least one post-baseline imaging assessment. The objective response rate (ORR) in these 29 patients was 21%. The disease control rate (DCR), reflecting the proportion of patients with a partial response or with stable disease, was 83%. The safety data obtained from all 42 patients enrolled to date was consistent with the results from previous clinical studies with derazantinib.

Dr. Marc Engelhardt, Chief Medical Officer of Basilea, said: "We are very pleased to have achieved this important milestone. 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. We are looking forward to the final data once the study is completed mid-2020."

He added: "The results of the interim analysis underscore the potential of derazantinib in the treatment of FGFR-driven tumors. As previously communicated, we are planning to extend the clinical development program of derazantinib, by starting a phase 2 study in other types of FGFR-driven solid tumors, mid-2019. We are also planning to expand the ongoing iCCA study by adding a separate cohort of patients with FGFR gene mutations to assess the potential expanded utility of derazantinib in the treatment of iCCA."