ArQule Announces First Patient Dosed in Registrational MOSIAC Trial of Miransertib for the Treatment of Proteus Syndrome & PI...
October 02 2019 - 7:00AM
Business Wire
MOSAIC trial represents the first
registrational trial for Proteus Syndrome and PIK3CA-related
overgrowth spectrum
ArQule, Inc. (Nasdaq: ARQL), today announced
that the first patient was dosed in the registrational MOSAIC
(Miransertib in Overgrowth Syndromes
in Adults and Children) trial of its oral, selective pan-AKT
inhibitor, miransertib, for the treatment of Proteus syndrome (PS)
and PIK3CA-related Overgrowth Spectrum disorders (PROS). The MOSAIC
trial represents the first registrational trial of its kind for
patients suffering from these rare genetic conditions characterized
by mutations in the PI3K/AKT pathway that emerge in childhood and
early adolescence.
“After years of extensive preclinical and clinical efforts among
ArQule and our collaborators, the MOSAIC trial represents an
important milestone for patients with Proteus syndrome and PROS,
their families and caregivers,” said Brian Schwartz, Chief Medical
Officer of ArQule. “These patients currently have extremely limited
therapeutic options and no approved or effective drug for their
treatment. We are pleased that the FDA has granted miransertib Fast
Track Designation for PROS and Rare Pediatric Disease Designation
for Proteus syndrome.”
“PS and PROS are chronic, progressive and debilitating
conditions affecting both children and adults. At Texas Children’s
Hospital (TCH) Vascular Anomalies Center we are very excited and
hopeful that miransertib may help our patients,” said Dr. Ionela
Iacobas, medical director of the Vascular Anomalies Center at Texas
Children’s Hospital and assistant professor of pediatrics –
hematology and oncology at Baylor College of Medicine. “With the
current research advances in the vascular anomalies field, we have
been able to provide early diagnosis for syndromes caused by
genetic abnormalities. Still, until now there was no open clinical
trial specifically targeted for PS and PROS. We have made a
commitment to our patients to offer comprehensive multidisciplinary
medical and surgical care and to bring them both state-of-the-art
standard-of-care as well as the newest promising experimental
therapies. Opening the MOSAIC study at TCH Vascular Anomalies
Center is part of that commitment.”
The MOSAIC trial is an international, multi-center, open-label
study that will evaluate the objective response to miransertib in
patients with PS and PROS. The study will enroll approximately
30-35 patients with documented somatic mutations in the AKT1 or
PIK3CA genes in the registrational cohorts for PS and PROS. Thirty
to 35 additional patients will be enrolled in 2 other cohorts for
patients under compassionate use or those ineligible to enter the
registrational cohorts.
About Miransertib Miransertib (ARQ 092) is an orally
available, selective, pan-AKT (protein kinase B) inhibitor that
potently inhibits AKT 1, 2 and 3 isoforms and binds both the active
and inactive forms of AKT which directly inhibits and prevents
membrane localization, respectively. 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 a single agent or in combination therapy,
show significant promise in molecularly defined patient
populations. Miransertib has been granted Rare Pediatric Disease
Designation for Proteus syndrome by the U.S. Food and Drug
Administration (FDA) as well as Orphan Drug Designation by both the
FDA and European Medicines Agency. Fast Track Designation has been
granted by the FDA for PROS.
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 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 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 four 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 dual inhibitor of both wild
type and C481S-mutant BTK, in phase 1/2 for patients with B-cell
malignancies refractory to other therapeutic options; miransertib
(ARQ 092), a potent and selective inhibitor of the AKT
serine/threonine kinase, in a registrational trial with cohorts in
Proteus syndrome and PROS; ARQ 751, a next generation highly potent
and selective AKT inhibitor, in phase 1 for patients with solid
tumors with AKT1 and PI3K mutations; and derazantinib, a
multi-kinase inhibitor designed to preferentially inhibit the
fibroblast growth factor receptor (FGFR) family, in a
registrational trial for iCCA in collaboration with Basilea and
Sinovant. 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, including statements regarding the
MOSAIC registrational study in Proteus syndrome and 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 from those set forth in
this press release. Positive information about early stage clinical
trial results does not ensure that later stage or larger scale
clinical trials will be successful. For example, miransertib may
not demonstrate adequate 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 current or
later stage trials may not be sufficient to meet applicable
regulatory standards or to justify further development. Problems or
delays may arise prior to the initiation of planned clinical
trials, during clinical trials or in the course of developing,
testing or manufacturing 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 or its collaborators’ view of data or require
additional data or information or additional studies. In addition,
the planned timing of completion of clinical trials is subject to
the ability of the Company and, in certain cases, its collaborators
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. 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 successfully pursue drug discovery in the future. For more
detailed information on the risks and uncertainties associated with
the Company's drug development, financial condition 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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version on businesswire.com: https://www.businesswire.com/news/home/20191002005266/en/
Corporate: Kathleen Farren Investor Relations
&Executive Assistant to the CFO ir@arqule.com
Media: Cait Williamson, Ph.D. LifeSci Public Relations
(646) 751-4366 cait@lifescipublicrelations.com
www.ArQule.com
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