ArQule Announces Publication of Clinical Data with Miransertib in Proteus Syndrome
February 25 2019 - 7:00AM
Business Wire
Data support miransertib as a potential first
systemic treatment for individuals affected by Proteus syndrome
ArQule, Inc. (Nasdaq:ARQL), today announced the publication of
clinical pharmacodynamic, safety and efficacy data in patients with
Proteus syndrome. These data, together with data already presented
at ASHG last year, support miransertib’s further development as a
potential first systemic treatment for patients suffering from
overgrowth diseases, such as Proteus syndrome. The study, published
in the American Journal of Human Genetics, and led by the National
Institutes of Health (NIH), demonstrated good target engagement,
tolerability and reductions in lesion size and pain, especially in
children.
Highlights from the study include:
- Generally well-tolerated safety
profile
- Reductions in pAKT (activated AKT) in
most patients
- Reductions in Cerebriform Connective
Tissue Nevus (CCTN) lesions in size (measured with standardized
photography) but also in firmness, depth of sulci and discomfort
(by patient report)
- Reduction in pain intensity in all (3
of 3) children in the study
“These data are highly encouraging and support further
investigation into the potential use of miransertib for this
devastating condition,” said Dr. Brian Schwartz, Chief Medical
Officer at ArQule. “The reduction in CCTN lesions were particularly
striking since these lesions are, in general, relentlessly
progressive, and cause severe morbidity, ulcerations and
intractable pain. We’d like to thank our academic collaborators and
the patients and their families for their support and tremendous
dedication to this cause.”
The study is available online at:
https://marlin-prod.literatumonline.com/pb-assets/journals/society/ajhg/AJHG2737.pdf
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.
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. 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 Proteus
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. 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.
Forward Looking Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995, including without limitation those regarding the current
clinical trial with miransertib and the statement by Dr. Schwartz.
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 results from early stage clinical
trials of miransertib are not necessarily indicative of results
that will be obtained in subsequent trials and does not ensure that
later stage or larger scale clinical trials will be successful. The
results achieved in ongoing or later stage trials may not be
sufficient to meet applicable regulatory standards or to justify
further development. In addition, miransertib 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. 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. 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 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. Only a small number of research and development
programs result in the commercialization of a product.
Furthermore, the Company 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 disclaims any obligation to update the
information contained in this press release as new information
becomes available.
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version on businesswire.com: https://www.businesswire.com/news/home/20190225005274/en/
Corporate Contact:Marc Schegerin, MDSenior Vice
PresidentHead of Strategy and FinanceIR@arqule.com
or
Media Contact:LifeSci Public RelationsAllison Blum,
Ph.D., 646-627-8383Allison@lifescipublicrelations.com
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