ArQule Receives Clearance of Investigational New Drug Application from the FDA for Proprietary Reversible BTK Inhibitor, ARQ ...
April 11 2017 - 7:00AM
Business Wire
Plan to initiate a phase 1 trial by Q3 of
2017
ArQule, Inc. (Nasdaq: ARQL) today announced that it has received
clearance from the U.S. Food and Drug Administration (FDA) for the
Investigational New Drug (IND) application to conduct a phase 1
clinical trial with ARQ 531 in patients with B-cell malignancies
who are refractory to other therapeutic options. ARQ 531 is an
investigational, orally bioavailable, potent and reversible
inhibitor of both wild type and C481S-mutant Bruton’s tyrosine
kinase (BTK).
ArQule plans to initiate a phase 1a/b dose escalation and signal
generation trial by Q3 of 2017. The phase 1a portion will be a dose
escalation study open to patients with B-cell malignancies, with
the aim of establishing a recommended dose. Upon completion of the
phase 1a trial, the company plans to begin a phase 1b trial in a
number of expansion cohorts that will include patients with the
C481S mutation who are refractory to other therapies. The goal
would be to establish target engagement and early proof of
concept.
“Given the emerging data on BTK resistance and the extensive
preclinical work the team at The Ohio State University have
done with ARQ 531, we are looking forward to moving this drug
from the bench to the bedside,” said Dr.
Jennifer Woyach, M.D., of The Ohio State University
College of Medicine. “A clear need is emerging for a BTK inhibitor
that addresses resistance.”
“There is an emerging body of evidence that is defining the
potential clinical need related to BTK resistance, and new
molecules are needed to treat patients who have developed
resistance,” said Dr. Brian Schwartz, M.D., Head of Research and
Development and Chief Medical Officer at ArQule. “We have been
working with The Ohio State University in the preclinical
development of ARQ 531, and we are looking forward to extending
that partnership into clinical testing.”
B-cell malignancies, like chronic lymphocytic leukemia,
Waldenstrom’s macroglobulinemia, diffuse large B-cell lymphoma and
mantle cell lymphoma are driven by BTK. The only approved BTK
inhibitor, ibrutinib, is irreversible and makes a covalent bond
with the C481 residue of the targeted protein. Although ibrutinib
has demonstrated excellent responses in patients with elevated
B-cell receptor signaling, clinical resistance has been observed,
and the BTK C481S mutation is emerging as a predominant mechanism
of resistance. As a reversible inhibitor, ARQ 531 does not require
interaction with the C481 residue, a binding site essential for
irreversible ibrutinib binding to BTK, thus positioning ARQ 531 as
a targeted therapy for patients harboring C481S-mutant BTK who have
developed resistance to irreversible BTK inhibitors.
About BTK and ARQ 531
ARQ 531 is an investigational, orally bioavailable, potent and
reversible Bruton’s tyrosine kinase (BTK) inhibitor. Biochemical
and cellular studies have shown that ARQ 531 inhibits both the wild
type and C481S-mutant forms of BTK. The C481S mutation is a known
emerging resistance mechanism for first generation irreversible BTK
inhibitors. In preclinical studies ARQ 531 has demonstrated high
oral bioavailability as well as good ADME, pharmacokinetic and
metabolic properties. The company plans to initiate a phase 1 trial
by the third quarter of 2017. BTK is a therapeutic target that has
been clinically proven to inhibit B-cell receptor signaling in
blood cancers.
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 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, through toxicology testing and plan to
initiate a phase 1 trial by the third quarter of 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
preclinical experiments and planned clinical trials with ARQ 531.
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 results does not ensure that clinical trials
will be successful. For example, ARQ 531 may not demonstrate
promising therapeutic effect in man; in addition, it may not
exhibit an adequate safety profile in planned, 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 these compounds 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. Drug
development involves a high degree of risk. 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 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/20170411005159/en/
ArQule, Inc.Dawn Schottlandt, 781-994-0300Sr. Director, Investor
Relations/Corp. Communicationswww.arqule.com
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