Positive Phase 2 Clinical Data with Tivantinib in Hepatocellular Carcinoma to Be Highlighted in Oral Presentation at 2012 Annual

  Positive Phase 2 Clinical Data with Tivantinib in Hepatocellular Carcinoma
  to Be Highlighted in Oral Presentation at 2012 Annual Meeting of American
  Society of Clinical Oncology

Business Wire

WOBURN, Mass. & TOKYO -- May 17, 2012

ArQule, Inc. (Nasdaq: ARQL) and Daiichi Sankyo, Co., Ltd. (TSE 4568) today
announced that an oral presentation at the Annual Meeting of the American
Society of Clinical Oncology (ASCO) will feature Phase 2 trial data with
tivantinib as a single agent investigational second-line treatment in
hepatocellular carcinoma (HCC). ArQule announced that this randomized,
double-blind study met its primary endpoint in January and will now present
the full results from this trial, including positive data in the pre-defined
c-MET high population. Additional clinical data with tivantinib will be
featured in two poster discussions and two general poster sessions. Abstracts
of these presentations with tivantinib have been published on www.asco.org.

“These findings represent the first randomized data reported with an
investigational c-MET inhibitor administered as a single agent second-line
treatment in HCC,” said Paolo Pucci, chief executive officer of ArQule. “They
clearly define c-MET high patients as a biological subgroup for potential
targeted therapy with tivantinib. The robust statistical significance achieved
in this trial reflects the anti-cancer activity of tivantinib alone and
expands its therapeutic potential.”

HCC Trial Summary: c-MET high patients

Data from the HCC trial demonstrated a statistically significant improvement
in time-to-progression (HR=0.43, log rank p-value=0.03), accompanied by
significant improvements in progression-free survival and disease control rate
among second-line patients with c-MET high tumors who were treated with
tivantinib. In addition, overall survival data were observed favoring
tivantinib-treated patients in this population. Efficacy was similar in the
two tivantinib dosing subgroups (360 milligrams twice daily and 240 milligrams
twice daily), with less frequent neutropenia in the lower dose.

Previously announced top-line data from the HCC trial demonstrate that
treatment with tivantinib produced a statistically significant 56 percent
improvement in TTP in the intent-to-treat (ITT) population by central
radiology review, the primary endpoint (HR = 0.64, log rank p-value = 0.04) in
this trial. Adverse events were reported at similar rates in the treatment and
placebo arms, except for a higher incidence of fatigue and hematologic events,
including neutropenia and anemia, in tivantinib-treated patients. The
incidence of hematologic events declined following dose reduction of
tivantinib from 360 milligrams twice daily to 240 milligrams twice daily.

The schedule of this and other presentations of data with tivantinib is
provided below. All times are Central Daylight Time.

Oral Presentation

Date and time: Saturday, June 2, 2012, 5:00 PM – 5:15 PM
Abstract number: 4006
Poster title: Tivantinib (ARQ 197) versus placebo in patients (Pts) with
hepatocellular carcinoma (HCC) who failed one systemic therapy: Results of a
randomized controlled phase II trial (RCT)
Presenter: Lorenza Rimassa, MD
Location: E Hall D1

Poster Discussion Sessions

Date and time: Saturday, June 2, 2012, 8:00 AM – 12:00 PM
Abstract number: 4545
Poster title: Safety and efficacy of MET inhibitor tivantinib (ARQ 197)
combined with sorafenib in patients (pts) with renal cell carcinoma (RCC) from
a phase 1 study
Poster board # 24
Presenter: Igor Puzanov, MD
Location: E450a

Date and time: Saturday, June 2, 2012, 1:15 PM – 5:15 PM
Abstract number: 8519
Poster title: Safety and efficacy of MET inhibitor tivantinib (ARQ 197)
combined with sorafenib in patients (pts) with NRAS wild-type or mutant
melanoma from a phase 1 study
Poster board # 8
Presenter: Julie A. Means-Powell, MD
Location: E450b

General Poster Session

Date and time: Monday, June 4, 2012, 8:00 AM – 12:00 PM
Abstract number: 4117
Poster title: Safety and efficacy of MET inhibitor tivantinib (ARQ 197)
combined with sorafenib in patients (pts) with hepatocellular carcinoma (HCC)
from a phase 1 study
Poster board # 50D
Presenter: Robert E. Martell, MD, PhD
Location: S Hall A2

Date and time: Monday, June 4, 2012, 8:00 AM – 12:00 PM
Abstract number: 4082
Poster title: A phase II study of tivantinib monotherapy in patients with
previously treated advanced or recurrent gastric cancer
Poster board # 46A
Presenter: Kei Muro, MD
Location: S Hall A2

Tivantinib is currently in Phase 3 development and has not yet been approved
for any indication.

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 candidate,
in Phase 2 and Phase 3 clinical development together with development and
commercialization partner, Daiichi Sankyo, Co. Ltd, is tivantinib, an oral,
selective inhibitor of the c-MET receptor tyrosine kinase. The Company’s
pipeline consists of ARQ 621, designed to inhibit the Eg5 kinesin motor
protein, and ARQ 736, designed to inhibit the RAF kinases. 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.

About Daiichi Sankyo

The Daiichi Sankyo Group is dedicated to the creation and supply of innovative
pharmaceutical products to address the diversified, unmet medical needs of
patients in both mature and emerging markets. While maintaining its portfolio
of marketed pharmaceuticals for hypertension, hyperlipidemia, and bacterial
infections, the Group is engaged in the development of treatments for
thrombotic disorders and focused on the discovery of novel oncology and
cardiovascular-metabolic therapies. Furthermore, the Daiichi Sankyo Group has
created a "Hybrid Business Model," which will respond to market and customer
diversity and optimize growth opportunities across the value chain. For more
information, please visit www.daiichisankyo.com.

This press release contains statements regarding the clinical trials with
tivantinib (ARQ 197) by ArQule and its business partner, Daiichi Sankyo.These
statements are based on the current beliefs and expectations of both
companies, 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, tivantinib may not
demonstrate a promising therapeutic effect; in addition, it may not
demonstrate an appropriate safety profile 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 these compounds that could lead ArQule or
its partners to discontinue development.Even if later stage clinical trials
are successful, unexpected concerns may arise from 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 ArQule’s view of the data or require additional
data or information or additional studies.In addition, the planned timing of
initiation and completion of clinical trials for tivantinib are subject to the
ability of ArQule, Daiichi Sankyo, and Kyowa Hakko Kirin, a licensee of
tivantinib, to enroll patients, enter into agreements with clinical trial
sites and investigators, and overcome technical hurdles and other issues
related to the conduct of the trials for which each of them is
responsible.There is a risk that these issues may not be successfully
resolved.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. Moreover, with respect to
partnered programs, even if certain compounds show initial promise, Daiichi
Sankyo or Kyowa Hakko Kirin may decide not to license or continue to develop
them, as the case may be.In addition, Daiichi Sankyo and Kyowa Hakko Kirin
have certain rights to unilaterally terminate their agreements with ArQule. If
either company were to do so, ArQule might not be able to complete development
and commercialization of the applicable licensed products on its own. For more
detailed information on the risks and uncertainties associated with the
ArQule’s drug development and other activities, see the ArQule’s periodic
reports filed with the Securities and Exchange Commission. Neither ArQule, nor
DaiichiSankyo 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
or
Daiichi Sankyo, Co., Ltd.
Dr. Michaela Paudler-Debus, +81-3-6225-1338 (office)
 
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