ArQule Presents Phase 1/2 Clinical Data with ARQ 087 in Intrahepatic Cholangiocarcinoma at the 2017 American Society of Clini...
June 03 2017 - 9:00AM
Business Wire
83% disease control rate observed in
intrahepatic cholangiocarcinoma FGFR2 positive patients
ArQule, Inc. (Nasdaq: ARQL) today announced that data from a
phase 1/2 trial with fibroblast growth factor receptor (FGFR)
inhibitor, ARQ 087, presented at ASCO demonstrate a meaningful
clinical benefit to intrahepatic cholangiocarcinoma (iCCA) patients
harboring FGFR2 fusions. The data show a robust response rate and
prolonged duration of therapy for these patients well in excess of
that reported for second-line chemotherapy. These data support
future development of ARQ 087 as second-line treatment, and a
registrational phase 3 trial in iCCA FGFR2 fusion positive patients
is planned to begin in the third quarter of 2017. ARQ 087 is a
multi-kinase inhibitor designed to preferentially inhibit the
fibroblast growth factor receptor (FGFR) family.
The presentation titled “ARQ 087, an oral pan-Fibroblast Growth
Factor Receptor (FGFR) inhibitor, in patients with advanced
intrahepatic cholangiocarcinoma (iCCA) with FGFR2 genetic
aberrations” can be viewed at
https://www.arqule.com/wp-content/uploads/ARQ-087-101_Poster_ASCO-2017.pdf.
ARQ 087 Results from Phase 1/2 iCCA Trial Presented at
ASCO
- The data is comprised of 35 iCCA
patients harboring FGFR2 genetic alterations, of which 29 patients
were FGFR2 fusion positive. All 29 of these patients were evaluable
for this data presentation.
- The objective response rate for iCCA
FGFR2 fusion positive patients was 21% (six partial responses) and
the disease control rate was 83% (six partial responses and 18
patients with stable disease). Patients were evaluated using
Standard RECIST (Response Evaluation Criteria in Solid
Tumors).
- Clinical benefit was observed in 72% of
FGFR2 fusion positive patients defined as partial response (six
patients) and stable disease (15 patients) for at least 16
weeks.
- The median duration of therapy observed
in iCCA FGFR2 fusion positive patients was 182 days. In these same
patients, the median duration of front-line chemotherapy was 119
days.
- ARQ 087 showed a manageable safety
profile with mostly Grade 1 and 2 adverse events.
- Nine patients with FGFR2 fusions are
on-going in the trial.
“Clinical evidence is accumulating on the role of FGFR
inhibitors in cholangiocarcinoma and other FGFR driven tumors such
as urothelial and gastric cancers,” said Dr. Brian Schwartz,
M.D., Head of Research and Development and Chief Medical Officer
at ArQule. “We are encouraged to see that both the response
rate and disease control rate were consistent throughout the trial.
Patients with iCCA often have a poor prognosis for front-line
treatment with chemotherapy, and there are no currently approved
second-line therapeutic options.”
Patients with advanced iCCA who relapse after first-line
multi-agent chemotherapy have limited treatment options with poor
prognosis. In recent years, FGFR2 fusions have been recognized as a
potential iCCA-specific therapeutic target. The company has been
granted orphan drug designation by the U.S. Food and Drug
Administration and European Medicines Agency for ARQ 087 in this
indication.
About Intrahepatic Cholangiocarcinoma
Cholangiocarcinoma (CCA) is the most common biliary malignancy
and the second most common hepatic malignancy after hepatocellular
carcinoma (HCC)1. Depending on the anatomic location, CCA is
classified as intrahepatic (iCCA), perihilar (pCCA), and
extrahepatic (eCCA). iCCA originates from the intrahepatic biliary
ductal system and forms an intrahepatic mass. The average age
adjusted incidence rate for iCCA is approximately one in 100,000
per year in the United States and Europe2,3.
About FGFR and ARQ 087
ARQ 087 is a multi-kinase inhibitor designed to preferentially
inhibit the fibroblast growth factor receptor (“FGFR”) family with
demonstrated efficacy in FGFR2 genetic alterations. The FGFR
pathway is disrupted in several ways in human cancer, thus
providing numerous therapeutic targets for an inhibitor of this
pathway. ARQ 087 has demonstrated in vivo inhibition of tumor
growth and downstream signaling in tumors whose growth is driven by
FGFR.
Signals of single agent activity with this drug were observed in
phase 1a testing. Phase 1b expansion cohorts with ARQ 087 include
patients with cholangiocarcinoma and adrenocortical tumors, as well
as those with FGFR translocations, amplifications and mutations.
Clinical development of ARQ 087 advanced into phase 2 for
intrahepatic cholangiocarcinoma (iCCA) in patients with FGFR2
fusions following the observation of two confirmed responses in
this patient population in the phase 1 portion of the program, and
a phase 3 registrational trial is planned to begin in the third
quarter of 2017 in this same patient population.
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
1/2 company sponsored study for Overgrowth Diseases, in phase 1 for
ultra-rare Proteus syndrome conducted by the National Institutes of
Health (NIH), as well as in multiple oncology indications; 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.
Forward Looking Statements
This press release contains forward-looking statements regarding
the Company’s clinical trials with ARQ 087. 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,
ARQ 087 may not demonstrate promising therapeutic effect; in
addition, these drugs 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 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. In addition, we plan to develop and use a
companion diagnostic to identify patients with FGFR2 and possibly
other fusions for our future ARQ 087 clinical trials. We intend to
outsource the development of such companion diagnostics to one or
more third party collaborators. There can be no assurance that we
will successfully enter into an agreement or agreements with any
such collaborators; in addition, any such collaborator may
encounter difficulties in developing and obtaining approval for
such companion diagnostic, 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 diagnostic could
delay or prevent approval of ARQ 087. 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.
1 Welzel TM, et al. Impact of classification of hilar
cholangiocarcinomas (Klatskin tumors) on the incidence of intra-
and extrahepatic cholangiocarcinoma in the United States. J
Natl Cancer Inst. 2006; 98(12),873–875.2 National
Cancer Institute: Surveillance, Epidemiology, and End Results3
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ArQule, Inc.Dawn Schottlandt, 781-994-0300Vice President,
Investor Relations/Corp. Communicationswww.ArQule.com
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