ArQule Presents Orphan Disease Clinical Data at the American Society of Human Genetics (ASHG) 2018 Annual Meeting for Its pan-AKT Inhibitor, Miransertib (ARQ 092)

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Three poster presentations confirm miransertib’s potential for
treating patients with Proteus syndrome and PROS (PIK3CA-Related
Overgrowth Spectrum)
BURLINGTON, Mass.–(BUSINESS WIRE)–Oct. 19, 2018–
ArQule, Inc. (Nasdaq:ARQL) today announced the presentation of
preliminary clinical data on miransertib (ARQ 092) in three poster
presentations at the American Society of Human Genetics (ASHG) 2018
Annual Meeting held from October 16 to 20, 2018 in San Diego. The data
presented relate to patients affected by either Proteus syndrome or PROS
who have been receiving miransertib as part of a clinical trial or in a
compassionate use setting.

Two presentations feature data from two patients treated as part of
ArQule’s named patient/compassionate use program. Presentation
highlights include:

1.Personalized medicine in rare diseases and
cancer: A case report of a lasting response in a young teenage patient
with Proteus syndrome and secondary ovarian cancer

Treatment for the Proteus syndrome patient with concomitant relapsed
ovarian cancer was administered at a dose level of 100mg and was well
tolerated for over 19 months, resulting in a clinically
significant/durable partial response of tumor and improvement of
Proteus syndrome symptoms including improved mobility/bone changes

2.Severe PI3Kinase overgrowth syndrome treated with
the AKT inhibitor miransertib

Treatment for the PROS patient was well tolerated for over 25 months
resulting in clinical stabilization and radiological improvement of

“Our understanding of the potential for miransertib to target and treat
these rare and devastating diseases that arise due to genetic
alterations of the PI3K/AKT pathway has grown significantly since we
partnered with The National Human Genome Research Institute in 2015 to
conduct the first clinical trial in Proteus syndrome,” said Brian
Schwartz, M.D., Chief Medical Officer of ArQule. “The data we are
presenting at ASHG continues to highlight the promise of ArQule’s
precision medicine approach for both rare diseases and oncology. We
remain deeply committed to advancing miransertib for rare PI3K/AKT
pathway overgrowth diseases as rapidly as possible, as there are
currently no approved therapeutics for these patients.”

A third presentation features data from patients treated as part of
ArQule’s ongoing Phase 1/2, open label study of miransertib for the
treatment of PROS. Study objectives include the evaluation of dosing
schedule, safety, PK profile and preliminary efficacy of miransertib.
Presentation highlights include:

3.An open-label, phase 1/2 study of miransertib
(ARQ 092), an oral pan-AKT inhibitor, in patients (pts) with
PIK3CA-related Overgrowth Spectrum (PROS): Preliminary results

Preliminary evidence of clinical efficacy was demonstrated by
improvements in disease related symptoms and objective radiologic and
photographic measures

Miransertib was well tolerated with a demonstrated manageable toxicity
profile in patients as young as two years old

The recommended dose of miransertib, defined as 15 mg/m2
QD, with potential dose escalation to 25 mg/m2 QD, provided
appropriate inhibition of the activated PIK3CA pathway for long-term
use without inhibition of growth in normal healthy cells

Peter Lawrence, President and Chief Operating Officer of ArQule said:
“This initial presentation of clinical data from our Phase 1/2 study in
PROS lays the foundation for potentially expanding the miransertib rare
disease program beyond Proteus syndrome, for which the drug already has
received Rare Pediatric Disease and Fast Track Designation. We look
forward to continuing our productive discussions with regulators to
define a pivotal trial design and rapidly advance miransertib.”

All posters presented by ArQule at the ASHG 2018 Annual Meeting are
available on the company’s website at

About MiransertibMiransertib (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. Miransertib has been granted Rare
Pediatric Disease Designation and Fast Track Designation by the U.S.
Food and Drug Administration (FDA), as well as Orphan Designation by the
FDA and European Medicines Agency in the rare overgrowth disease,
Proteus syndrome.

About PROSPROS 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 SyndromeProteus 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 ArQuleArQule 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 StatementsThis press release contains
forward-looking statements regarding the planned clinical development of
miransertib, including statements regarding its rapid advancement in
overgrowth diseases. 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
sufficient 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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Source: ArQule, Inc.
Corporate:ArQule, Inc.Marc Schegerin, M.D.Senior
Vice PresidentHead of Strategy, Finance and Communicationir@arqule.comorMedia:LifeSci
Public RelationsAllison Blum, Ph.D.,

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2018-10-20 05:12:42