European Medicines Evaluation Agency Grants Orphan Drug Designation to ARQ 197 for Treatment of Soft Tissue Sarcoma

WOBURN, Mass.--(Business Wire)--
ArQule, Inc. (NASDAQ: ARQL) today announced that the European Medicines
Evaluation Agency (EMEA) has designated ARQ 197 as an orphan medical product for
the treatment of soft tissue sarcoma.

Under the guidelines of the EMEA, medicinal products are designated as orphan
drugs when they are used to treat illnesses affecting small numbers of patients,
such as soft tissue sarcoma. Such designation provides for incentives for
investment in the research and development of such drugs, including exclusive
distribution rights throughout the European Union for ten years, during which
period no other product with the same active ingredient would be authorized for
use for the same therapeutic application.

ArQule is conducting a Phase 2 clinical trial with ARQ 197 in a sub-group of
soft tissue sarcoma known as MiT (Microphthalmia Transcription
Factor)-associated tumors. The designation of ARQ 197 as an orphan medical
product in soft tissue sarcoma, combined with the evaluation of clinical data
from this trial expected to be available in early 2010, will inform the
decisions related to the advancement of the program.

About ARQ 197 and c-MET

In addition to soft tissue sarcoma, ARQ 197 is currently being evaluated in
Phase 2 clinical trials in non-small cell lung cancer, pancreatic cancer and
hepatocellular carcinoma.

ARQ 197 is a selective inhibitor of c-MET, a receptor tyrosine kinase. When
abnormally activated, c-MET plays multiple roles in aspects of human cancer,
including cancer cell growth, survival, angiogenesis, invasion and metastasis.
Pre-clinical data have demonstrated that ARQ 197 inhibits c-MET activation in a
wide range of human tumor cell lines and shows anti-tumor activity against
several human tumor xenografts. In clinical studies to date, treatment with ARQ
197 has been well tolerated and has resulted in tumor responses and prolonged
stable disease across broad ranges of tumors and doses.

About ArQule and Daiichi Sankyo Co., Ltd.

On December 19, 2008, ArQule and Daiichi Sankyo signed a license, co-development
and co-commercialization agreement to co-develop ARQ 197 in the U.S., Europe,
South America and the rest of the world, excluding Japan, China (including Hong
Kong), South Korea and Taiwan, where Kyowa Hakko Kirin Co., Ltd. has exclusive
rights for development and commercialization.

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 cancer. ArQule`s lead product, in Phase 2 clinical
development, is ARQ 197, an inhibitor of the c-MET receptor tyrosine kinase. The
Company has also initiated Phase 1 clinical testing with ARQ 621, designed to
inhibit the Eg5 kinesin motor protein. An additional clinical-stage program
includes compounds that activate the cell`s DNA damage response mechanism
mediated by the E2F-1 transcription factor. The Company`s pre-clinical pipeline
includes a compound designed to inhibit the BRAF kinase. 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 the orphan drug
designation of ARQ 197 by the European Commission and the future clinical
development of ARQ 197 in a sub-group of soft tissue sarcoma known as
MiT(Microphthalmia Transcription Factor)-associated tumors. 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 and statements about such designation by the
European Commission do not ensure that regulatory review and approval of ARQ 197
will take place on a timely basis or at all in Europe, the United States or
other parts of the world.In addition, early stage clinical trial results do not
ensure that later stage or larger scale clinical trials will be successful. For
example, ARQ 197 may not demonstrate promising therapeutic effect; in addition,
this compound may not demonstrate an appropriate safety profile in further
pre-clinical testing and in current, 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. Problems or delays may arise during clinical trials or in the course
of developing, testing or manufacturing these compounds that could lead the
Company or its partner to discontinue development.Even if later stage clinical
trials are successful, the risk exists that unexpected concerns may arise from
analysis of data or from additional data or that obstacles may arise or issues
be identified in connection with review of clinical data with regulatory
authorities or that regulatory authorities may disagree with the Company`s view
of the data or require additional data, information or studies.In addition, the
planned timing of initiation and completion of clinical trials for ARQ 197 is
subject to the ability of the Company to enroll patients, enter into agreements
with clinical trial sites and investigators, and other technical hurdles and
issues that may not be 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. Furthermore, ArQule may not have the financial
or human resources to pursue drug discovery successfully 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.

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

Copyright Business Wire 2009