- 21% objective response rate with six
confirmed partial responses from 29 evaluable
patients
- 83% disease control rate
- Safety profile and tolerability of
continuous dosing schedule confirmed
ArQule, Inc.’s (Nasdaq: ARQL) partner, Basilea Pharmaceutica
Ltd. (SIX: BSLN), today announced results from the interim analysis
of the registrational Phase 2 study with the orally administered
pan-fibroblast growth factor receptor (FGFR) kinase inhibitor
derazantinib (BAL087). The analysis showed promising activity in
patients with FGFR2 gene fusion-expressing intrahepatic
cholangiocarcinoma (iCCA) and also confirmed the safety profile and
tolerability of the drug candidate observed in previous clinical
studies.
The interim analysis in the ongoing registrational Phase 2 study
was conducted after 42 patients had been enrolled in the study,
with a subset of 29 evaluable patients who had at least one
post-baseline imaging assessment. The objective response rate (ORR)
in the 29 evaluable patients was 21%. The disease control rate
(DCR), reflecting the proportion of patients with a partial
response or with stable disease, was 83%. The safety data obtained
from all 42 patients enrolled to date was consistent with the
results from previous clinical studies with derazantinib.
http://www.basilea.com/Investor-Relations/News-and-Media/
Peter Lawrence, President and Chief Operating Officer of ArQule,
said, “We are pleased with derazantinib’s continued progress since
it was licensed to Basilea Pharmaceutica in April 2018 in the US,
EU, Japan and rest of world excluding Greater China. Under the
terms of the license agreement, ArQule is eligible to receive up to
$326 million in regulatory and commercial milestone payments, and
we look forward to further progress and updates from Basilea.”
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 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 derazantinibDerazantinib (BAL087, formerly ARQ 087)
is an investigational orally administered small molecule inhibitor
of the FGFR family of kinases with strong activity against FGFR 1,
2, and 3. Therefore, it is called a pan-FGFR kinase inhibitor. FGFR
kinases are key drivers of cell proliferation, differentiation and
migration. FGFR alterations, e.g., gene fusions, overexpression or
mutations, have been identified as potentially important
therapeutic targets for various cancers, including iCCA, bladder,
breast, gastric and lung cancers.2 Current scientific literature
suggests that FGFR alterations exist in a range of 5% to 30% in
these cancers.3 In iCCA, FGFR2 gene fusions have been reported in
13-22% of the cases4, 5 and FGFR gene mutations have been reported
in up to 5% of the cases.3 Basilea in-licensed derazantinib from
ArQule Inc. in April 2018. The drug candidate has demonstrated
favorable clinical data in previous clinical studies, including a
biomarker-driven Phase 1/2 study in iCCA patients.6 Derazantinib
has U.S. and EU orphan drug designation for this disease.
About intrahepatic cholangiocarcinoma (iCCA)Intrahepatic
cholangiocarcinoma (iCCA) is a cancer originating from the biliary
system. The age-adjusted incidence rate of iCCA in the United
States has been increasing over the past decade and is currently
estimated to be approximately 1.2 per 100,000.7 Patients are often
diagnosed with advanced or metastatic disease that cannot be
surgically removed. Current first-line standard of care is the
chemotherapy combination of gemcitabine and platinum-derived
agents. The prognosis for patients with advanced disease is poor,
with a median survival of less than one year. There is no proven
effective treatment for patients who progress on first-line
chemotherapy, thus there is a high unmet medical need.8
About BasileaBasilea Pharmaceutica Ltd. is a commercial
stage biopharmaceutical company focused on the development of
products that address the medical challenge in the therapeutic
areas of oncology and anti-infectives. With two commercialized
drugs, the company is committed to discovering, developing and
commercializing innovative pharmaceutical products to meet the
medical needs of patients with serious and life-threatening
conditions. Basilea Pharmaceutica Ltd. is headquartered in Basel,
Switzerland and listed on the SIX Swiss Exchange (SIX: BSLN).
Additional information can be found at Basilea's website
www.basilea.com.
Forward Looking StatementsThis press release contains
forward-looking statements regarding clinical trials with
derazantinib as well as the potential for future milestone and
royalty payments under the Company’s exclusive license agreement
with Basilea. 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 does not ensure that later stage or larger scale clinical
trials will be successful. For example, derazantinib may not
demonstrate promising therapeutic effect. In addition, derazantinib
may not demonstrate an acceptable 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 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 derazantinib that could lead the Company
or Basilea to discontinue its 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 Basilea’s view of the data or
require additional data or information or additional studies. In
addition, we or Basilea plan to develop and use a companion
diagnostic to identify patients with FGFR2 fusions and possibly
other fusions for our future derazantinib clinical trials. We or
Basilea intend to outsource the development of such companion
diagnostics to one or more third party collaborators. Such
collaborators may encounter difficulties in developing and
obtaining approval for such companion diagnostics, including issues
relating to selectivity/specificity, analytical validation,
reproducibility, concordance or clinical validation. Any delay or
failure to develop or obtain regulatory approval of such companion
diagnostics could delay or prevent approval of derazantinib.
Moreover, Basilea has only a limited track record of drug
development in oncology. If derazantinib is not successfully
developed and as a result of any of the foregoing or other issues,
risks or uncertainties, ArQule may not receive any future
milestones or royalties under the license agreement with Basilea.
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.
References
1 ClinicalTrials.gov identifier: NCT03230318 2 R. Porta, R.
Borea, A. Coelho et al. FGFR a promising druggable target in
cancer: Molecular biology and new drugs. Critical Reviews in
Oncology/Hematology 2017 (113), 256-267 3 T. Helsten, S. Elkin, E.
Arthur et al. The FGFR landscape in cancer: Analysis of 4,853
tumors by next-generation sequencing. Clinical Cancer Research 2016
(22), 259-267 4 R. P. Graham, E. G. Barr Fritcher, E. Pestova et
al. Fibroblast growth factor receptor 2 translocations in
intrahepatic cholangiocarcinoma. Human Pathology 2014 (45),
1630-1638 5 A. Jain, M. J. Borad, R. K. Kelley et al.
Cholangiocarcinoma with FGFR genetic abberations: a unique clinical
phenotype. JCO Precision Oncology 2018 (2), 1-12 6 V. Mazzaferro,
B. F. El-Rayes, M. Droz dit Busset et al. Derazantinib (ARQ 087) in
advanced or inoperable FGFR2 gene fusion-positive intrahepatic
cholangiocarcinoma. British Journal of Cancer. Published online on
November 13, 2018. https://doi.org/10.1038/s41416-018-0334-0 7 S.
K. Saha, A. X. Zhu, C. S. Fuchs et al. Forty-year trends in
cholangiocarcinoma incidence in the U.S.: intrahepatic disease on
the rise. The Oncologist 2016 (21), 594-599 8 S. Sahu, W. Sun,
Targeted therapy in biliary tract cancers – current limitations and
potentials in the future. Journal of Gastrointestinal Oncology 2017
(8), 324-336
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version on businesswire.com: https://www.businesswire.com/news/home/20190109005207/en/
Corporate Contact:Marc Schegerin, M.D.Senior Vice
PresidentHead of Strategy, Finance and
Communicationir@arqule.com
Media Contact:Allison Blum, Ph.D.LifeSci Public
Relations(646) 627-8383Allison@lifescipublicrelations.com
www.ArQule.com
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