ArQule Announces Dosing In Registrational Trial Of FGFR Inhibitor Derazantinib

(RTTNews) - ArQule, Inc. (ARQL) on Tuesday announced dosing of the initial patients in a registrational trial with its FGFR inhibitor, derazantinib or ARQ 087 in FGFR2 fusion driven second-line intrahepatic Cholangiocarcinoma or iCCA.

The trial is planned to enroll up to 100 iCCA patients and will provide an opportunity for a conditional approval as part of a fast-to-market strategy. Derazantinib is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family.

The open-label single-arm trial will be recruiting initially in the U.S. and subsequently in Europe with objective response rate or ORR as the primary endpoint. Derazantinib will be dosed orally once a day at 300 mg.

FGFR2 fusion status will be determined by a break apart FISH assay. An interim analysis will be performed after the first 40 patients have been enrolled and evaluated for response.

Patients with advanced iCCA who relapse after first-line multi-agent chemotherapy have limited treatment options with poor prognosis. In recent years, FGFR2 fusions have been recognized as a potential iCCA-specific therapeutic target.

ArQule has been granted orphan drug designation by the U.S. Food and Drug Administration and European Medicines Agency for derazantinib in this indication.