ArQule Presents Preliminary Clinical Data for ARQ 087 Demonstrating Evidence of Anticancer Activity in Intrahepatic Cholangiocarcinoma at the ESMO 18th World Congress on Gastrointestinal Cancer

75% disease control rate, including a 25% objective response rate, was observed in 12 evaluable patients harboring FGFR2 genetic alterations

BURLINGTON, Mass., June 30, 2016 (GLOBE NEWSWIRE) -- ArQule, Inc. (Nasdaq:ARQL) today announced that preliminary data presented at ESMO 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). 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.

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

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.

About ArQule

ArQule is a biopharmaceutical company engaged in the research and development of targeted therapeutics to treat cancers and rare diseases. Our mission is to discover, develop and commercialize novel small molecule drugs in areas of high unmet need that will dramatically extend and improve the lives of our patients. Our proprietary clinical-stage pipeline consists of four drug candidates, all of which are in targeted, biomarker-defined patient populations, making ArQule a leader among companies our size in precision medicine. ArQule’s lead product, in phase 3 clinical development, is tivantinib (ARQ 197), an oral, selective inhibitor of the c-MET receptor tyrosine kinase, for second-line treatment of hepatocellular carcinoma in partnership with Daiichi Sankyo in the West and Kyowa Hakko Kirin in Asia. ArQule’s proprietary pipeline includes: ARQ 092, designed to inhibit the AKT serine/threonine kinase, in phase 1 for multiple oncology indications as well as ultra-rare Proteus syndrome, in partnership with the National Institutes of Health (NIH); ARQ 087, a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family, in phase 2 for iCCA and in phase 1b for multiple oncology indications; and ARQ 761, a β-lapachone analog being evaluated as a promoter of NQO1-mediated programmed cancer cell necrosis, in phase 1/2 in multiple oncology indications in partnership with the University of Texas Southwestern Medical Center. ArQule’s current discovery efforts are focused on the identification and development of novel kinase inhibitors, leveraging the Company’s proprietary library of compounds.

This press release contains forward-looking statements regarding the Company’s clinical trials with ARQ 087. These statements are based on the Company’s current beliefs and expectations, and are subject to risks and uncertainties that could cause actual results to differ materially. Positive information about pre-clinical and early stage clinical trial results does not ensure that later stage or larger scale clinical trials will be successful. For example, ARQ 087 may not demonstrate promising therapeutic effect; in addition, it may not demonstrate appropriate safety profiles in current or later stage or larger scale clinical trials as a result of known or as yet unanticipated side effects. The results achieved in later stage trials may not be sufficient to meet applicable regulatory standards or to justify further development. Problems or delays may arise during clinical trials or in the course of developing, testing or manufacturing ARQ 087 that could lead the Company to discontinue development. Even if later stage clinical trials are successful, unexpected concerns may arise from subsequent analysis of data or from additional data. Obstacles may arise or issues may be identified in connection with review of clinical data with regulatory authorities. Regulatory authorities may disagree with the Company’s view of the data or require additional data or information or additional studies. In addition, we plan to develop and use a companion diagnostic to identify patients with FGFR2 fusions. We intend to outsource the development of such companion diagnostic to one or more third party collaborators. There can be no assurance that we will successfully enter into an agreement or agreements with any such collaborator; in addition, any such collaborator may encounter difficulties in developing and obtaining approval for such companion diagnostic, including issues relating to selectivity/specificity, analytical validation, reproducibility, or clinical validation. Any delay or failure by our collaborators or us to develop or obtain regulatory approval of such companion diagnostic could delay or prevent approval of ARQ 087. Drug development involves a high degree of risk. Only a small number of research and development programs result in the commercialization of a product. Positive pre-clinical data may not be supported in later stages of development. Furthermore, ArQule may not have the financial or human resources to successfully pursue drug discovery in the future. For more detailed information on the risks and uncertainties associated with the Company’s drug development and other activities, see the Company’s periodic reports filed with the Securities and Exchange Commission. The Company does not undertake any obligation to publicly update any forward-looking statements.

1 Welzel TM, et al. Impact of classification of hilar cholangiocarcinomas (Klatskin tumors) on the incidence of intra- and extrahepatic cholangiocarcinoma in the United States. J Natl Cancer Inst. 2006; 98(12),873–875.
2 National Cancer Institute: Surveillance, Epidemiology, and End Results

Contacts: Dawn Schottlandt Sr. Director, Investor Relations/ Corp. Communications (781) 994-0300

Source:ArQule, Inc.