ArQule Announces Publication of Manuscript Highlighting Preclinical Activity of FGFR Inhibitor, ARQ 087, in Peer Reviewed Jou...
September 15 2016 - 7:00AM
Business Wire
PLOS ONE publication supports phase 1/2
intrahepatic cholangiocarcinoma trial with ARQ 087 in patients with
FGFR2 genetic alterations
ArQule, Inc. (Nasdaq: ARQL) today announced the publication of a
paper detailing the preclinical profile of ARQ 087, an orally
available fibroblast growth factor receptor (FGFR) inhibitor. The
findings, published on-line by PLOS ONE,
http://dx.plos.org/10.1371/journal.pone.0162594, demonstrate that
ARQ 087 has anti-proliferative activity in cell lines driven by
FGFR dysregulation, including amplifications, fusions, and
mutations.
ARQ 087 is being dosed in intrahepatic cholangiocarcinoma (iCCA)
patients with FGFR2 genetic alterations as part of the phase 2
portion of a biomarker driven phase 1/2 trial. The company has been
granted orphan drug designation by the U.S. Food and Drug
Administration and European Medicines 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 activity 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
clinical-stage pipeline consists of five 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 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; ARQ 092, a selective inhibitor of 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 751, a next
generation AKT inhibitor, in phase 1 for patients with AKT1 and
PI3K mutations; 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 Results3
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ArQule, Inc.Dawn Schottlandt, 781-994-0300Sr. Director, Investor
Relations/Corp. Communicationswww.arqule.com
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