ArQule Reports Orphan Drug Status For ARQ 087 In Cholangiocarcinoma

(RTTNews) - ArQule, Inc. (ARQL) Tuesday announced receipt of orphan drug designation from the Food and Drug Administration for ARQ 087 in cholangiocarcinoma.

ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth receptor or FGFR family.

ArQule is currently conducting a biomarker-driven phase 2 trial for ARQ 087 in the U.S. and Italy in intrahepatic cholangiocarcinoma or iCCA patients with FGFR2 fusions.

The commencement of the phase 2 trial followed the observation of two confirmed partial responses in iCCA with FGFR2 fusions in the phase 1a portion of the trial. A minor response was observed in a third patient also harboring an FGFR2 fusion.

The phase 2 trial is designed to enroll up to 20 patients and potentially provide a clinical signal to be explored further in an expedited clinical strategy in this rare indication.

The incidence rate for iCCA in the U.S. and Europe is approximately one in 100,000, while the incidence rate in Asia is said to be higher.

In addition to the phase 2 trial for iCCA, ArQule is continuing to enroll the phase 1b portion of the trial with ARQ 087 in solid tumors harboring FGFR2 genetic alterations. A partial response was recently observed in a bladder cancer patient with an FGFR2 genetic alteration.