ArQule Announces Preliminary Results from Its Phase 1/2 Study of Miransertib (ARQ 092), in Patients with PIK3CA-related Overg...
June 17 2019 - 9:00AM
Business Wire
Encouraging preliminary safety and clinical
activity data confirm miransertib’s potential for treating PS and
PROS patients
Registrational study, MOSAIC (Miransertib in
Overgrowth Syndromes in Adults and Children), expected to begin
enrollment in Q3 2019
ArQule, Inc. (Nasdaq:ARQL) today announced preliminary results
from the company’s phase 1/2 study of its pan-AKT inhibitor,
miransertib (ARQ 092), in patients with PIK3CA-related Overgrowth
Spectrum (PROS) and Proteus syndrome (PS) in an oral presentation
at the European Society of Human Genetics Conference in Gothenburg,
Sweden.
“The results presented from our ongoing phase 1/2 study
highlight the potential for miransertib to provide a molecularly
targeted treatment for patients with rare PI3K/AKT driven
overgrowth diseases,” said Dr. Brian Schwartz, Chief Medical
Officer of ArQule. “We have been committed to rapidly advancing
miransertib for patients with these devastating diseases since
collaborating with The National Human Genome Research Institute in
2015 and are now poised to start the registrational study, MOSAIC,
in the third quarter of this year. We are thrilled with the
preliminary safety and clinical activity data and look forward to
continued clinical progress for the program.”
Dr. Chiara Leoni, an Investigator on the study from the
Fondazione Policlinico Universitario Agostino Gemelli, said, “The
precision medicine approach of the study has led to very
encouraging preliminary results with the observed safety and
tolerability profile, along with clinically meaningful improvement
in disease related symptoms in patients. In addition, the majority
of patients have had stable disease while on treatment,
demonstrating the potential of miransertib to halt disease
progression. The ability to attain durable treatment responses with
a manageable safety profile is an important step forward for this
patient population that currently has no medicinal treatment
options, and I look forward to advancing the registrational MOSAIC
study in the coming months.”
The reported interim data are from the phase 1/2 study, an
international, multi-center, open-label 2-part study evaluating
miransertib in patients with PS and PROS. The first part of the
study is evaluating the safety, tolerability, PK profile and
preliminary evidence of clinical activity of miransertib at
different dose levels. MOSAIC, the registrational part of the
study, is expected to begin patient enrollment in Q3 2019.
Key findings presented include:
- Recommended initial dose for the
registrational study was defined as 15mg/m2 QD with subsequent
maximum dose increase to 25mg/m2
- A manageable safety profile was
observed in patients as young as 2 years old, with mostly Grade 1
or 2 AEs
- Improvement in disease related symptoms
and performance status as measured by Karnofsky/Lansky scale was
reported in the majority of patients
- The majority of patients demonstrated
improvement or no disease progression extending beyond 1 year on
treatment
The presentation, Preliminary results from the company’s phase
1/2 study of its pan-AKT inhibitor, miransertib (ARQ 092), in
patients with PIK3CA-related Overgrowth Spectrum (PROS) and Proteus
syndrome (PS), is available on the company’s website at
www.arqule.com/publications-presentations/.
About MiransertibMiransertib (ARQ 092) is an orally
available, selective, pan-AKT (protein kinase B) inhibitor that
potently inhibits AKT 1, 2 and 3 isoforms. Additionally, it 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
single agent or 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 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 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 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 planned registrational trial with
cohorts in Proteus syndrome and PROS to initiate in 2019, and in
phase 1b in combination with the hormonal therapy, anastrozole, in
patients with advanced endometrial cancer; ARQ 751, a next
generation highly potent and selective AKT inhibitor, in phase 1
for patients 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 StatementsThis press release contains
forward-looking statements, including without limitation those
regarding the ongoing phase 1/2 study with miransertib and the
planned 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/20190617005207/en/
Corporate:Kathleen FarrenInvestor Relations&
Executive Assistant to the CFOir@arqule.com
Media:Cait Williamson, Ph.D.LifeSci Public Relations(646)
751-4366cait@lifescipublicrelations.com
www.ArQule.com
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