On June 30, 2016 ArQule, Inc. (Nasdaq:ARQL) reported that preliminary data presented at ESMO (Free ESMO Whitepaper) GI demonstrate evidence of anticancer activity, defined by objective response rate and disease control rate in an ongoing phase 1/2 biomarker driven trial with ARQ 087 in intrahepatic cholangiocarcinoma (iCCA) (Press release, ArQule, JUN 30, 2016, View Source [SID:1234513641]). Activity was observed in patients with FGFR2 genetic alterations. ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family.

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The presentation titled "ARQ 087, an Oral Pan-Fibroblast Growth Factor Receptor (FGFR) Inhibitor, in Patients with Advanced and/or Metastatic Intrahepatic Cholangiocarcinoma (iCCA)" can be viewed at www.arqule.com/wp-content/uploads/ARQ-087-iCCA-ESMO-GI-2016.pdf.

Preliminary Data Results from Phase 1/2 iCCA Trial with ARQ 087

The data is comprised of 21 patients dosed with ARQ 087, 14 of which presented with FGFR2 genetic alterations and seven of which did not.
Of the 14 iCCA patients with FGFR2 genetic alterations, 12 patients were evaluable.
Among the 12 evaluable patients with FGFR2 genetic alterations, the objective response rate was 25% (three partial responses) and disease control rate was 75% (three partial responses and six patients with stable disease). Patients were evaluated using Standard RECIST (Response Evaluation Criteria in Solid Tumors).
In addition to the three patients with partial responses, three patients had a minor response, defined as a 15-29% tumor reduction. Durable disease control, defined as greater than 16 weeks, was observed in 50% of patients. Progressive disease was the best response in 25% of the patients.
ARQ 087 showed a manageable safety profile with mostly Grade 1 and 2 adverse events.
This data is derived from the phase 1 and 2 portions of the phase 1/2 trial in iCCA.
"Intrahepatic cholangiocarcinoma is a rare liver cancer with a high mortality rate and limited treatment options," said Dr. Brian Schwartz, Head of Research and Development and Chief Medical Officer at ArQule. "There is scientific evidence that this disease can be caused by a number of different genetic alterations, one being FGFR2, thus making the use of precision medicine essential in treating these patients. The data we presented, while preliminary, are very encouraging and offer evidence that ARQ 087 is active in those patients with a FGFR2 genetic alteration. We look forward to concluding the study late in the third quarter of 2016."

The company has been granted orphan drug designation by the U.S. Food and Drug Administration and European Medicine’s Agency for ARQ 087 in this indication.

About Intrahepatic Cholangiocarcinoma

Cholangiocarcinoma (CCA) 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 genetic alterations. 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 in vivo inhibition of tumor growth and downstream signaling in tumors whose growth is driven by FGFR targets.

Signals of single agent activity with this drug 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.