Basilea Announces Positive Interim Results from Registrational Phase 2 Study with Oncology Drug Candidate Derazantinib in Intrahepatic Cholangiocarcinoma (iCCA)

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 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 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 derazantinib
Derazantinib (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 cases^{4, 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 Basilea
Basilea 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 Statements
This 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

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