ArQule Announces Commencement of Phase 3 Clinical Trial with Tivantinib in Hepatocellular Carcinoma by Partner Kyowa Hakko Kirin

  ArQule Announces Commencement of Phase 3 Clinical Trial with Tivantinib in
  Hepatocellular Carcinoma by Partner Kyowa Hakko Kirin in Japan

Business Wire

WOBURN, Mass. -- February 4, 2014

ArQule, Inc. (Nasdaq: ARQL) today reported the announcement by its partner,
Kyowa Hakko Kirin Co., Ltd. (Tokyo: 4151) of the initiation of a Phase 3
clinical trial evaluating tivantinib (ARQ 197) in Japanese patients with c-Met
diagnostic-high inoperable hepatocellular carcinoma treated with one prior
sorafenib therapy.

The trial is a randomized, double-blind placebo-controlled study to compare
progression-free survival (PFS) in patients treated with tivantinib with those
treated with placebo. Kyowa Hakko Kirin plans to enroll approximately 160
patients in this study.

“With the commencement of this study, two Phase 3 trials are now ongoing
worldwide to evaluate the impact of tivantinib therapy in second line HCC,”
said Brian Schwartz, M.D., chief medical officer of ArQule. “We are also
enrolling patients in the ongoing pivotal, randomized, double-blind METIV-HCC
trial, being conducted in the West by ArQule and our partner, Daiichi Sankyo
Co., Ltd., under a Special Protocol Assessment (SPA) agreement.”

About MET and Tivantinib (ARQ 197)

Tivantinib is an orally administered, selective inhibitor of MET, a receptor
tyrosine kinase, which is currently in Phase 2 and 3 clinical trials. In
certain healthy adult cells, MET is present in low to normal levels to support
natural cellular function, but in some cancer cells, MET is inappropriately
and continuously activated. When abnormally activated, c-Met plays multiple
roles in aspects of human cancer, including cancer cell growth, survival,
angiogenesis, invasion and metastasis. The activation of certain cell
signaling pathways, including MET, has also been associated with the
development of resistance to EGFR inhibitors such as cetuximab.

Pre-clinical data have demonstrated that tivantinib inhibits MET activation in
a range of human tumor cell lines and shows anti-tumor activity against
several human tumor xenografts. In clinical trials to date, treatment with
tivantinib has been generally well tolerated and has shown clinical activity
in the tumors studied. Tivantinib has not yet been approved for any indication
in any country.

About ArQule, Inc. and its Partners for the Development of Tivantinib

On December 19, 2008, ArQule and Daiichi Sankyo Co., Ltd. signed a license,
co-development and co-commercialization agreement to co-develop tivantinib in
the U.S., Europe, South America and the rest of the world, excluding Japan,
China (including Hong Kong), South Korea and Taiwan, areas for which Kyowa
Hakko Kirin has exclusive rights for development and commercialization under
an exclusive license agreement signed with ArQule in 2007.

About ArQule

ArQule is a biotechnology company engaged in the research and development of
next-generation, small-molecule cancer therapeutics. The Company’s targeted,
broad-spectrum products and research programs are focused on key biological
processes that are central to human cancers. 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 and ARQ
087, designed to inhibit fibroblast growth factor receptor (FGFR). ArQule’s
current discovery efforts, which are based on the ArQule Kinase Inhibitor
Platform (AKIP™), are focused on the identification of novel kinase inhibitors
that are potent and selective against their targets.

This press release contains forward-looking statements regarding the METIV-HCC
clinical trial with tivantinib in hepatocellular carcinoma (HCC) conducted
with Daiichi Sankyo and the Phase 3 clinical trial with tivantinib in HCC
conducted by Kyowa Hakko Kirin as well as the Company’s agreements with both
Daiichi Sankyo and Kyowa Hakko Kirin. 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. There can
be no assurance that tivantinib alone or in a combination therapy will
demonstrate promising therapeutic effects in pivotal or other trials; in
addition, tivantinib may ultimately not demonstrate an appropriate safety
profile in later stage or larger scale clinical trials, such as METIV-HCC or
the Kyowa Phase 3 trial in HCC, including among patients with underlying
cirrhosis and compromised liver function, 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 tivantinib that could lead the
Company, Daiichi Sankyo or Kyowa Hakko Kirin to discontinue development. Even
if later stage clinical trials are successful, unexpected concerns may arise
from analyses of data or from additional data. Obstacles may arise or issues
may be identified in connection with review of clinical data with regulatory
authorities, and regulatory authorities may disagree with the Company’s view
of the data or require additional data or information or additional studies.
In addition, the planned timing of completion of clinical trials like
METIV-HCC or the Phase 3 HCC trial conducted by Kyowa Hakko Kirin is subject
to the ability of the Company or its partners to enroll patients, enter into
agreements with clinical trial sites and investigators, and overcome ongoing
or emergent regulatory issues and address other technical hurdles and issues
related to the conduct of the trials for which each of them is responsible
that may not be resolved promptly, or at all. Drug development involves a high
degree of risk. Only a small number of research and development programs
result in the commercialization of a product. Furthermore, ArQule may not have
the financial or human resources to successfully pursue drug discovery in the
future. Moreover, Daiichi Sankyo and Kyowa Hakko Kirin have certain rights to
unilaterally terminate the tivantinib license agreement with the Company. If
it were to do so, the Company might not be able to complete development and
commercialization of tivantinib on its own. 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.

Contact:

ArQule, Inc.
William B. Boni, 781-994-0300
VP, Investor Relations/Corp. Communications
www.ArQule.com
 
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