Preclinical Data on Proprietary AKT Inhibitor, ARQ 092, Demonstrating Effectiveness in the Treatment of Sickle Cell Disease P...
December 03 2016 - 05:45PM
Business Wire
The study supports further exploration of ARQ
092 in sickle cell disease
ArQule, Inc. (Nasdaq: ARQL) today announced that preclinical
data was presented on its proprietary AKT inhibitor, ARQ 092, in an
oral presentation by the University of Illinois College of Medicine
at the American Society of Hematology (ASH) Annual Meeting. The
presentation highlighted preclinical studies of ARQ 092 in sickle
cell disease (SCD). ARQ 092 is an orally available, selective
pan-AKT inhibitor.
ARQ 092 Oral Presentation Highlights
Title: Specific inhibition of AKT with ARQ 092, an
orally-available selective allosteric AKT inhibitor, attenuates
acute vaso-occlusive events in sickle cell disease
- ARQ 092 inhibits activation, and
effectively blocks heterotypic aggregation, of neutrophils and
platelets in SCD patients in vitro.
- Ex vivo studies indicate that ARQ 092
inhibits activation of neutrophils and platelets isolated from SCD
mice after oral administration.
- In vivo studies with SCD mice
demonstrate that co-administration of hydroxyurea and ARQ 092
effectively attenuates acute vaso-occlusive events and improves
survival.
- This study provides proof of concept in
an established preclinical model and enables a clinical pathway for
ARQ 092 in the treatment of SCD with or without hydroxyurea.
The presentation can be viewed at
https://www.arqule.com/wp-content/uploads/ASH-2016-ARQ-092-in-Sickle-Cell-Disease-.pdf.
“SCD is a debilitating lifetime disease with a worldwide
presence. A report from the American Society of Hematology shows
that approximately 100,000 people suffer from this disease in the
U.S.” said Dr. Jaehyung Cho, PhD., of the University of Illinois
College of Medicine. “Our research has been focused on identifying
novel therapeutic targets to prevent and treat vaso-occlusive
diseases. The work done at the University of Illinois, in
collaboration with ArQule, shows the importance of the AKT pathway
in inducing vaso-occlusive events during SCD and the beneficial
effect of ARQ 092 in attenuating adhesive function of neutrophils
and platelets in SCD mice. This work warrants further exploration
of ARQ 092 in patients with SCD.”
“Our AKT program, specifically ARQ 092, continues to progress
with the phase 1 trial in Proteus syndrome sponsored by the
National Institutes of Health (NIH) and with the recently approved
Investigational New Drug (IND) application for expansion into the
PROS family of rare over-growth diseases,” said Dr. Brian Schwartz,
M.D., Head of Research and Development and Chief Medical Officer at
ArQule. “The PI3K/AKT pathway has been implicated in multiple
diseases including SCD. We would like to thank the University of
Illinois College of Medicine for lending its expertise and helping
to establish valuable proof of concept for ARQ 092 in this disease
setting.”
About the AKT Pathway and ARQ 092
ARQ 092 is an orally bioavailable, selective small molecule
inhibitor of the AKT kinases. The AKT pathway when abnormally
activated is implicated in multiple oncogenic processes such as
cell proliferation and apoptosis. This pathway has emerged as a
target of potential therapeutic relevance for compounds that
inhibit its activity, which has been linked to a variety of cancers
as well as to select non-oncology indications.
ARQ 092, the lead compound in ArQule’s AKT program, has
completed phase 1a clinical testing and has advanced into phase 1b
expansion testing in cohorts of patients with endometrial cancer,
lymphomas and tumors harboring either AKT or PI3K mutations. It is
also in a phase 1 trial being conducted by the NIH for Proteus
syndrome, a rare over-growth disease from the PROS family.
Collaborators are exploring, in preclinical testing, other
indications for ARQ 092 including sickle cell disease.
About ArQule
ArQule is a biopharmaceutical company engaged in the research
and development of targeted therapeutics to treat cancers and rare
diseases. Our 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 lead product, in phase 3 clinical development,
is tivantinib (ARQ 197), an oral, selective inhibitor of the c-MET
receptor tyrosine kinase, for second-line treatment of
hepatocellular carcinoma in partnership with Daiichi Sankyo in the
West and Kyowa Hakko Kirin in Asia. ArQule’s proprietary pipeline
includes: ARQ 087, a multi-kinase inhibitor designed to
preferentially inhibit the fibroblast growth factor receptor (FGFR)
family, in phase 2 for iCCA and in phase 1b for multiple oncology
indications; ARQ 092, a selective inhibitor of the AKT
serine/threonine kinase, in phase 1 for multiple oncology
indications as well as ultra-rare Proteus syndrome, in partnership
with the National Institutes of Health (NIH); ARQ 751, a next
generation AKT inhibitor, in phase 1 for patients with AKT1 and
PI3K mutations; 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. In addition, we
have advanced ARQ 531, an investigational, orally bioavailable,
potent and reversible inhibitor of both wild type and C481S-mutant
BTK, into toxicology testing and plan to file an Investigational
New Drug Application in early 2017. ArQule’s current discovery
efforts are focused on the identification and development of novel
kinase inhibitors, leveraging the Company’s proprietary library of
compounds. You can follow us on Twitter and LinkedIn.
This press release contains forward-looking statements regarding
certain pre-clinical experiments conducted by the Company’s
collaborators and the Company’s clinical trials with ARQ 092. 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 pre-clinical, and early stage clinical trial, results,
including in SCD and Proteus syndrome, does not ensure that later
stage or larger scale clinical trials will be successful. For
example, ARQ 092 may not demonstrate promising therapeutic effect;
in addition, the drug candidate may not demonstrate appropriate
safety profiles 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 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 the compound
that could lead the Company or its partners, including the National
Institutes of Health, 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. Drug
development involves a high degree of risk. Only a small number of
research and development programs result in the commercialization
of a product. Positive pre-clinical data may not be supported in
later stages of development. 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 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: http://www.businesswire.com/news/home/20161203005009/en/
ArQuleDawn Schottlandt, 781-994-0300Sr. Director, Investor
Relations/Corp. Communicationswww.arqule.com
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