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Basilea Pharmaceutica Announces Pooled Results of FGFR Inhibitor for Patients With Bile Duct Cancer

NEW YORK – Switzerland-based Basilea Pharmaceutica on Monday announced pooled efficacy results for its investigational FGFR inhibitor, derazantinib, as a treatment for patients with bile duct cancer whose tumors harbor FGFR mutations or amplifications.

Across 23 patients with intrahepatic cholangiocarcinoma (iCCA), derazantinib resulted in a median progression-free survival of 7.2 months. The median duration of treatment across the 23 patients was 8.2 months.

The efficacy data, presented during the European Society for Medical Oncology's Molecular Analysis for Precision Oncology virtual congress, were derived from patients enrolled in two clinical trials and those who received derazantinib through an expanded access program. The first trial was a tumor-agnostic Phase I/II study evaluating derazantinib for patients with metastatic solid tumors with FGFR genetic alterations. The second was a Phase II trial, dubbed FIDES-01, evaluating the agent specifically for patients with inoperable or advanced iCCA whose tumors had either FGFR2 gene fusions, as confirmed by fluorescence in situ hybridization or FGFR2 mutations or amplifications, as confirmed by next-generation sequencing.

The remainder of the patients included in the pooled analysis were those with advanced iCCA with FGFR genomic alterations who received derazantinib from Basilea under an expanded access program that is ongoing while the drug is in development.

According to Basilea, the latest pooled data are consistent with previously reported efficacy and safety results among iCCA patients treated with derazantinib.

"To date there is limited clinical evidence for the benefit of FGFR inhibitors in iCCA patients with FGFR2 gene mutations and amplifications," Basilea Chief Medical Officer Marc Engelhardt said in a statement. "The data presented at the [ESMO meeting] show that derazantinib is active in this group of patients and underscore the broad therapeutic potential of derazantinib in FGFR2-positive iCCA."

In addition to patients with iCCA, Basilea is evaluating the agent as a treatment for patients with advanced urothelial cancer or advanced gastric cancers with FGFR genetic aberrations. In preclinical studies, derazantinib has demonstrated activity against FGFR1, FGFR2, and FGFR3.

Other drug developers have also been pursuing FGFR inhibition as a precision oncology treatment strategy. The US Food and Drug Administration has granted accelerated approval to two FGFR-inhibiting agents: the pan-FGFR inhibitor erdafitinib (Janssen's Balversa) as a treatment for patients with previously treated metastatic bladder cancer who harbor alterations in FGFR3 or FGFR2 genes, and pemigatinib (Incyte's Pemazyre), an inhibitor of FGFR isoforms 1, 2, and 3, for patients with previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. Recently, Relay Therapeutics began evaluating its FGFR2 inhibitor, RLY-4008, in patients with iCCA and other advanced solid tumors.