Dec 15, 2015

ArQule Announces Orphan Drug Designation in Cholangiocarcinoma and Clinical Update for ARQ 087

Orphan drug designation marks significant regulatory milestone for phase 2 trial

Additional response in phase 1b further validates development for ARQ 087 in cancers with FGFR2 genetic alterations

BURLINGTON, Mass., Dec. 15, 2015 (GLOBE NEWSWIRE) -- ArQule, Inc. (NASDAQ:ARQL) today announced receipt of orphan drug designation from the Food and Drug Administration (FDA) for ARQ 087 in cholangiocarcinoma. ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth receptor (FGFR) family.

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-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.

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 prioritized clinical-stage pipeline consists of four drug candidates, all of which are in targeted, biomarker-defined patient populations, making ArQule an early leader in precision medicine. ArQule's lead product, in phase 2 and phase 3 clinical development, is tivantinib (ARQ 197), an oral, selective inhibitor of the c-MET receptor tyrosine kinase. The Company's pipeline includes: ARQ 092, designed to inhibit the AKT serine/threonine kinase; ARQ 087, a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family; and ARQ 761, a Beta lapachone analog being evaluated as a promoter of NQ01-mediated programmed cancer cell necrosis. ArQule's current discovery efforts are focused on the identification 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;98:873-5.
2 National Cancer Institute: Surveillance, Epidemiology, and End Results

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

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Source: ArQule, Inc.

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