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Miransertib (ARQ 092) Granted Fast Track Designation for the Treatment of PIK3CA-Related Overgrowth Spectrum (PROS)

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ArQule, Inc. (NASDAQ:ARQL) today announced that the U.S. Food and Drug
Administration (FDA) has granted Fast Track Designation to miransertib
(ARQ 092) for the treatment of PIK3CA-Related Overgrowth Spectrum
(PROS), a group of related, ultra-rare genetic disorders characterized
by excessive tissue growth in various parts of the body. The FDA's Fast
Track program aims to expedite the development and review of drugs which
treat serious or life-threatening conditions and have demonstrated the
potential to address unmet clinical needs.

Miransertib has already been granted Rare Pediatric Disease Designation
by the FDA and Orphan Designation by the FDA and European Medicines
Agency in the rare overgrowth disease, Proteus syndrome. During the past
three years, miransertib has been tested in a Phase 1
NIH/NHGRI-sponsored trial in Proteus Syndrome. More recently, we have
been conducting a Phase 1/2 company-sponsored trial in PROS and a single
patient use program with select physicians.

"We are very excited by this important regulatory milestone for
miransertib and look forward to rapidly advancing this program for
patients suffering from this devastating condition with no approved
therapies," said ArQule's Chief Medical Officer, Dr. Brian Schwartz,
M.D. "Fast Track Designation and its associated FDA interactions come at
an ideal time for miransertib as we move forward in this genetically
targeted population and define the optimal path to registration."

Dr. Andrea Bartuli, Director of the Genetic and Rare Disease Unit of
Bambino Gesù Pediatric Hospital and the principal investigator of the
PROS trial added, "PROS is a group of genetic diseases affecting mostly
children that severely impacts the quality of life of patients and their
families. I am committed to helping advance miransertib as the potential
first effective therapeutic for patients with PROS, addressing the
significant unmet need in this patient population."

About Miransertib
Miransertib (ARQ 092) is an orally
available, selective, pan-AKT (protein kinase B) inhibitor that potently
inhibits AKT1, 2 and 3 isoforms. Dysregulation of AKT has been
implicated in a variety of rare overgrowth diseases and cancers;
however, there are currently no approved inhibitors of AKT. AKT
inhibitors, either as single agent or combination therapy, show
significant promise in molecularly defined patient populations.
Miransertib is currently in a Phase 1/2 company-sponsored study for
PIK3CA-Related Overgrowth Spectrum (PROS), a Phase 1 study for
ultra-rare Proteus syndrome conducted by the National Institutes of
Health (NIH/NHGRI), and a Phase 1b study in combination with the
hormonal therapy, anastrozole, in patients with advanced endometrial
cancer with AKT and PI3K mutations.

About PROS
PROS is a term used to refer to a spectrum of
rare diseases identified by somatic mutations in the PIK3CA gene, that
result in excess growth in certain areas of the body. While the
individual diseases that fall within the overgrowth spectrum have
similar symptoms, each disease is defined by unique clinical
characteristics. The implementation of genetic sequencing has led to the
identification of the underlying genetic mutations that drive these
overgrowth disorders, allowing for the development of medicines that
target the specific causes of disease.

About Proteus Syndrome
Proteus syndrome is an ultra-rare
condition characterized by the aberrant overgrowth of multiple tissues
of the body. Patients with Proteus syndrome experience changes in the
shapes of certain body structures over time, including abnormal, often
asymmetric, massive growth (overgrowth) of the skeleton, skin, adipose
tissue and central nervous system out of proportion to the rest of the
body. Although patients may have minimal or no manifestations at birth,
the disease develops and becomes apparent in early childhood (6-18
months) and rapidly progresses with intense growth in the first 10 years
of life. The worldwide incidence is believed to be approximately one in
a million. There are currently no approved medicinal treatments for
Proteus syndrome, leaving patients with minimal treatment options to
manage the disease and a mortality of 25% by age 22.

About ArQule
ArQule is a biopharmaceutical company engaged
in the research and development of targeted therapeutics to treat
cancers and rare diseases. ArQule's 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 pipeline
includes: ARQ 531, an orally bioavailable, potent and reversible
inhibitor of both wild type and C481S-mutant BTK, in Phase 1 for
patients with B-cell malignancies refractory to other therapeutic
options; Miransertib (ARQ 092), a selective inhibitor of the AKT
serine/threonine kinase, in a Phase 1/2 company-sponsored study for
Overgrowth Diseases, in a Phase 1 study for ultra-rare Proteus syndrome
conducted by the National Institutes of Health (NIH), and in Phase 1b in
combination with the hormonal therapy, anastrozole, in patients with
advanced endometrial cancer; ARQ 751, a next generation AKT inhibitor,
in Phase 1 for patients with AKT1 and PI3K mutations; Derazantinib, a
multi-kinase inhibitor designed to preferentially inhibit the fibroblast
growth factor receptor (FGFR) family, in a registrational trial for
iCCA; 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.

Forward Looking Statements
This press release contains
forward-looking statements regarding the planned clinical development of
miransertib, including a statement regarding its rapid advancement in
PROS. 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 early
clinical results does not ensure that later-stage clinical trials will
be successful. For example, miransertib may not demonstrate promising
therapeutic effect in man; in addition, it may not exhibit an adequate
safety profile in planned 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 miransertib 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 are utilizing diagnostic tests to identify
patients in the Phase 1/2 trial with miransertib in PROS diseases and
expect to utilize diagnostic tests in other clinical trials with
miransertib. We or our collaborators may need to develop and register
these or other diagnostic tests as companion diagnostics with the FDA.
We or our collaborators may encounter difficulties in developing and
obtaining regulatory approval for companion diagnostics, including
issues relating to access to certain technologies,
selectivity/specificity, analytical validation, reproducibility, or
clinical validation. Any delay or failure by our collaborators or us to
develop or obtain regulatory approval of companion diagnostics could
delay or prevent approval of our product candidates. Drug development
involves a high degree of risk. Only a small number of research and
development programs result in the commercialization of a product. 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.

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