ArQule to Present Clinical Update for Miransertib in Rare Disease at the American Society of Human Genetics (ASHG) 2018 Annual Meeting

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Three poster presentations highlight miransertib clinical data in
patients with Proteus syndrome and PROS (PIK3CA-Related Overgrowth
ArQule, Inc. (Nasdaq: ARQL) today announced that it will be presenting
clinical data on the company’s lead AKT inhibitor, miransertib (ARQ
092), in three poster presentations at the American Society of Human
Genetics (ASHG) 2018 Annual Meeting to be held from October 16 to 20,
2018 in San Diego, CA.

The data to be presented is from the Phase 1/2 company-sponsored trial
in PROS and the single patient use program with select physicians.

Presentation Details
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. 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 Contact:ArQule, Inc.Marc Schegerin, M.D.Senior
Vice President, Head of Strategy, Finance and Communicationir@arqule.comorMedia
Contact:LifeSci Public RelationsAllison Blum, Ph.D.,

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2018-10-05 06:23:28