On December 15, 2015 ArQule, Inc. (NASDAQ:ARQL) reported receipt of orphan drug designation from the Food and Drug Administration (FDA) for ARQ 087 in cholangiocarcinoma (Press release, ArQule, DEC 15, 2015, View Source [SID:1234508572]). ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth receptor (FGFR) family.
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ArQule is currently conducting a biomarker-driven phase 2 trial for ARQ 087 in the U.S. and Italy in intrahepatic cholangiocarcinoma (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. Subsequently, 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 further explored 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, respectively, while the incidence rate in Asia is believed 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.
"This year we have already received orphan drug designation for ARQ 092 in Proteus syndrome. We are now pleased to have received our second orphan drug designation from the FDA for ARQ 087 in CCA. This is part of our continued pursuit of precision medicine," said Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "CCA is a rare cancer that lacks approved therapeutic options to specifically target FGFR2 fusions. We are very encouraged by the clinical efficacy so far observed in the phase 1 portion of the trial which validates the pre-clinical data recently presented at the AACR (Free AACR Whitepaper)-NCI-EORTC conference that demonstrated efficacy across a number of FGFR2-driven tumors in xenograft models."
About Intrahepatic Cholangiocarcinoma
Cholangiocarcinoma is the most common biliary malignancy and the second most common hepatic malignancy after hepatocellular carcinoma (HCC)1. Depending on the anatomic location, CCA is classified as intrahepatic (iCCA), perihilar (pCCA), and extrahepatic (eCCA.) iCCA originates from the intrahepatic biliary ductal system and forms an intrahepatic mass. The average age adjusted incidence rate for iCCA is approximately one in 100,000 per year in the United States and Europe2,3.
About FGFR and ARQ 087
ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor ("FGFR") family with demonstrated efficacy in FGFR2 translocations. The FGFR pathway is disrupted in several ways in human cancer, thus providing numerous therapeutic targets for an inhibitor of this pathway. ARQ 087 has demonstrated inhibition of tumor growth and downstream signaling in vivo in tumors whose growth is driven by these targets.
Signals of single agent activity with this compound were observed in phase 1a testing. Phase 1b expansion cohorts with ARQ 087 include patients with cholangiocarcinoma and adrenocortical tumors, as well as those with FGFR translocations, amplifications and mutations. Clinical development of ARQ 087 has advanced into phase 2 for intrahepatic cholangiocarcinoma (iCCA) following the observation of two confirmed responses in this patient population in the phase 1 portion of the program.