Exelixis' Cabozantinib Granted Orphan Drug Designation for the Treatment of Hepatocellular Carcinoma
March 06 2017 - 9:04AM
Business Wire
Exelixis, Inc. (Nasdaq: EXEL) today announced that the U.S. Food
& Drug Administration (FDA) has granted orphan drug designation
to cabozantinib for the treatment of hepatocellular carcinoma
(HCC). This information was posted to FDA’s website on March 4,
2017 and can be accessed here. A pivotal phase 3 trial (CELESTIAL)
of cabozantinib is ongoing in patients with advanced HCC, and
Exelixis has guided that data from the trial are expected in
2017.
Orphan drug status is granted to treatments for diseases that
affect fewer than 200,000 people in the U.S. and provides certain
incentives for medications intended for the treatment, diagnosis or
prevention of rare diseases. At present, these incentives include
seven years of marketing exclusivity for the orphan indication,
certain federal grants, tax credits and waiver of certain FDA
fees.
About the CELESTIAL
Trial
CELESTIAL is designed to enroll 760 patients with advanced HCC
who received prior sorafenib. Patients are randomized 2:1 to
receive 60 mg of cabozantinib daily or placebo. The primary
endpoint for the trial is overall survival, and secondary endpoints
include objective response rate and progression-free survival.
Exploratory endpoints include patient-reported outcomes, biomarkers
and safety. The CELESTIAL trial is being conducted at more than 100
sites globally in 19 countries.
About Exelixis
Exelixis, Inc. (Nasdaq: EXEL) is a biopharmaceutical company
committed to the discovery, development and commercialization of
new medicines to improve care and outcomes for people with cancer.
Since its founding in 1994, three products discovered at Exelixis
have progressed through clinical development, received regulatory
approval, and entered the marketplace. Two are derived from
cabozantinib, an inhibitor of multiple tyrosine kinases including
MET, AXL and VEGF receptors: CABOMETYX™ tablets approved for
previously treated advanced kidney cancer and COMETRIQ® capsules
approved for progressive, metastatic medullary thyroid cancer. The
third product, Cotellic®, is a formulation of cobimetinib, a
selective inhibitor of MEK, is marketed under a collaboration with
Genentech (a member of the Roche Group), and is approved as part of
a combination regimen to treat advanced melanoma. Both cabozantinib
and cobimetinib have shown potential in a variety of forms of
cancer and are the subjects of broad clinical development programs.
For more information on Exelixis, please visit www.exelixis.com or follow @ExelixisInc on
Twitter.
Forward-Looking
Statements
This press release contains forward-looking statements,
including, without limitation, statements related to: the
expectation for CELESTIAL data results in 2017;
Exelixis’ commitment to the discovery, development and
commercialization of new medicines with the potential to improve
care and outcomes for people with cancer; the clinical potential of
cabozantinib and cobimetinib in a variety of forms of cancer; and
the continued development of cabozantinib and cobimetinib. Words
such as “guided,” “expected,” “committed,” “potential,” or other
similar expressions identify forward-looking statements, but the
absence of these words does not necessarily mean that a statement
is not forward-looking. In addition, any statements that refer to
expectations, projections or other characterizations of future
events or circumstances are forward-looking statements. These
forward-looking statements are based upon Exelixis’ current plans,
assumptions, beliefs, expectations, estimates and projections.
Forward-looking statements involve risks and uncertainties. Actual
results and the timing of events could differ materially from those
anticipated in the forward-looking statements as a result of these
risks and uncertainties, which include, without limitation: the
availability of data at the referenced time; Exelixis’ ability to
conduct clinical trials of cabozantinib sufficient to achieve a
positive completion; risks related to the potential failure of
cabozantinib to demonstrate safety and efficacy in clinical
testing; risks and uncertainties related to regulatory review and
approval processes and Exelixis’ compliance with applicable legal
and regulatory requirements; the degree of market acceptance of
CABOMETYX and COMETRIQ; Exelixis’ dependence on its relationship
with its cabozantinib collaboration partners, including, the
level of their investment in the resources necessary to
successfully commercialize cabozantinib in the territories where it
is approved; Exelixis’ dependence on its relationship with
Genentech/Roche with respect to cobimetinib and Exelixis’
ability to maintain its rights under the collaboration;
Exelixis’ dependence on third-party vendors; Exelixis’ ability
to protect the company’s intellectual property rights; market
competition; changes in economic and business conditions, and other
factors discussed under the caption “Risk Factors” in Exelixis’
annual report on Form 10-K filed with the Securities and
Exchange Commission (SEC) on February 27, 2017, and in
Exelixis’ future filings with the SEC. The forward-looking
statements made in this press release speak only as of the date of
this press release. Exelixis expressly disclaims any
duty, obligation or undertaking to release publicly any updates or
revisions to any forward-looking statements contained herein to
reflect any change in Exelixis’ expectations with regard thereto or
any change in events, conditions or circumstances on which any such
statements are based.
Exelixis, the Exelixis logo, COMETRIQ
and COTELLIC are registered U.S. trademarks, and CABOMETYX is a
U.S. trademark.
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version on businesswire.com: http://www.businesswire.com/news/home/20170306005745/en/
Investors Contact:Exelixis, Inc.Susan Hubbard,
650-837-8194EVP, Public Affairs and Investor
Relationsshubbard@exelixis.comorMedia Contact:For Exelixis,
Inc.Hal Mackins, 415-994-0040hal@torchcomllc.com
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