ArQule Announces Data Presentations with Tivantinib to Be Featured at ASCO 2014

  ArQule Announces Data Presentations with Tivantinib to Be Featured at ASCO

  Company Also Provides Update on Phase 3 Trial in Hepatocellular Carcinoma

2014 ASCO Annual Meeting

Business Wire

WOBURN, Mass. -- May 30, 2014

ArQule, Inc. (Nasdaq: ARQL) today announced that tivantinib will be included
in seven presentations during the 2014 Annual Meeting of the American Society
of Clinical Oncology (ASCO) to be held from May 30, 2014 to June 3, 2014 in
Chicago, Illinois.

The presentations will feature tivantinib in clinical trials across multiple
diseases and therapeutic combinations. Data will relate to the safety and
combinability of tivantinib with approved anti-cancer agents, supporting the
ongoing development of this compound.

The most advanced ongoing clinical trial with tivantinib is a pivotal Phase 3
randomized, double-blind controlled study (the METIV-HCC trial) of the
compound as single agent therapy in previously treated patients with
MET-diagnostic-high, inoperable hepatocellular carcinoma (HCC).

“Enrollment in the METIV-HCC trial continues to gather momentum, with the Data
Monitoring Committee having completed its most recent meeting,” said Paolo
Pucci, chief executive officer of ArQule. “We believe that recruitment figures
reflect our investigators’ enthusiasm, which has been further enhanced by
recent publications^1 that underscore the potential of MET as a target of
interest in HCC,” said Mr. Pucci.

Logistical information for the ASCO presentations featuring tivantinib follows

Abstract #TPS3661
ONC-2012-001: A single-arm phase II study oftivantinib(ARQ 197) plus
cetuximab in EGFR inhibitor-resistantMEThigh patients (pts) with locally
advanced or metastatic colorectal cancer (CRC) with wild-typeKRAS.

Saturday, May 31, 2014, 8:00 AM to 11:45 AM
S Hall A2, Poster Board: #117B
Lorenza Rimassa, MD

Abstract #8044
Tivantinib plus erlotinib versus placebo plus erlotinib in Asian patients with
previously treated nonsquamous NSCLC with wild-typeEGFR:First report of a
phase IIIATTENTIONtrial.

Saturday, May 31, 2014, 1:15 PM to 5:00 PM
S Hall A2, Poster Board #225
Koichi Azuma, MD, PhD

Abstract #8052
Phase II study of erlotinib plus tivantinib in patients
withEGFR-mutation–positive NSCLC who failed in immediately previous EGFR-TKI

Saturday, May 31, 2014, 1:15 PM to 5:00 PM
S Hall A2, Poster Board: #233
Tomonori Hirashima, MD

Abstract #TPS7610
Phase I-Ib trial of tivantinib in combination with carboplatin and pemetrexed
as first-line treatment in patients (pts) with advanced nonsquamous NSCLC or
malignant pleural mesothelioma (MPM).

Saturday, May 31, 2014, 1:15 PM to 5:00 PM
S Hall A2, Poster Board #217A
Paolo A. Zucali, MD

Abstract #1106
A phase II study oftivantinib(ARQ-197) for metastatic triple-negative breast

Monday, June 2, 2014, 8:00 AM to 11:45 AM
S Hall A2, Poster Board #199
Sara M. Tolaney, MD, MPH

Abstract #2555
A phase I study of ARQ 197 in combination with temsirolimus in patients (Pts)
with advanced solid tumors.

Sunday, June 1, 2014, 8:00 AM to 11:45 AM
S Hall A2, Poster Board #18
Amy M. Braden, DO

Abstract #2627
A phase 1 study of the c-Met inhibitor tivantinib (ARQ 197, IND#112603) in
children with relapsed or refractory solid tumors: A Children’s Oncology Group

Sunday, June 1, 2014, 8:00 AM to 11:45 AM
S Hall A2, Poster Board #90
James I. Geller, MD

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, selective and do not compete with ATP (adenosine
triphosphate) for binding to the kinase.

This press release contains forward-looking statements regarding clinical
trials with tivantinib, information about which is being presented at the 2014
ASCO Annual Meeting. 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 the Phase 3 METIV-HCC trial in
hepatocellular carcinoma, 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 or
its partners Daiichi Sankyo and 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 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.

^1 Future Oncol. (2014) 10(2), 285–304 and Journal of Hepatology 2014 vol. 60,


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