Portola Pharmaceuticals Completes BLA Submission to U.S. Food and Drug Administration for Andexanet Alfa
18 Décembre 2015 - 2:00PM
Portola Pharmaceuticals, Inc. (Nasdaq:PTLA) announced today
completion of the submission of a Biologics License Application
(BLA) to the U.S. Food and Drug Administration (FDA) for its
investigational agent andexanet alfa, an FDA-designated
breakthrough therapy specifically designed to reverse the
anticoagulant activity of both direct and indirect Factor Xa
inhibitors. Portola is developing andexanet alfa as a universal
reversal agent for patients anticoagulated with an oral or
injectable Factor Xa inhibitor who experience a serious
uncontrolled bleeding event or who require urgent or emergency
surgery.
The BLA submission includes data from Portola’s Phase 3 ANNEXA™
(Andexanet Alfa a Novel Antidote to the Anticoagulant Effects of
FXa Inhibitors) studies, which evaluated the safety and efficacy of
andexanet alfa in reversing the anticoagulant activity of the
Factor Xa inhibitors rivaroxaban and apixaban, respectively, in
healthy volunteers. Results of those studies were published online
by The New England Journal of Medicine in November 2015. In
addition, data from a small number of patients from ANNEXA-4 will
serve as the clinical basis for the BLA. Portola is currently
evaluating andexanet alfa in this global Phase 4 single-arm,
open-label confirmatory study in patients receiving apixaban,
rivaroxaban, edoxaban or enoxaparin (a low molecular weight heparin
and indirect Factor Xa inhibitor) who present with an acute major
bleed.
About the Need for a Factor Xa Inhibitor Reversal
Agent
Commensurate with the increase in use of Factor Xa inhibitors,
the number of hospital admissions due to bleeding associated with
these agents continues to grow. Annually, 1 to 4 percent of
patients treated with Factor Xa inhibitors may experience major
bleeding, and an additional 1 percent may require emergency
surgery. In the United States alone, during the 12 months ended
April 2015, there were over 50,000 rivaroxaban or apixaban treated
patients admitted to the hospital due to bleeding.1 This
number does not account for patients taking the injectable Factor
Xa inhibitor enoxaparin or those on a Factor Xa inhibitor
undergoing emergency surgery. In the United States, more than
100,000 patients annually may benefit from a reversal agent.
Currently, there is no FDA-approved reversal agent for Factor Xa
inhibitors for these patients.
About Andexanet Alfa
Andexanet alfa, an investigational drug, is a modified human
Factor Xa molecule that acts as a decoy to target and sequester
with high specificity both oral and injectable Factor Xa inhibitors
in the blood. Once bound, the Factor Xa inhibitors are unable to
bind to and inhibit native Factor Xa, thus allowing for the
restoration of normal hemostatic processes. Andexanet alfa is the
only compound being studied as a reversal agent for Factor Xa
inhibitors that directly and specifically corrects anti-Factor Xa
activity – the anticoagulant mechanism of these agents.
About Portola Pharmaceuticals, Inc.
Portola Pharmaceuticals is a biopharmaceutical company
developing product candidates that could significantly advance the
fields of thrombosis and other hematologic diseases. The Company is
advancing its three wholly-owned programs using novel biomarker and
genetic approaches that may increase the likelihood of clinical,
regulatory and commercial success of its potentially life-saving
therapies. These programs include betrixaban, an oral, once-daily
Factor Xa inhibitor being evaluated in the APEX Phase 3 study for
prophylaxis of venous thromboembolism; andexanet alfa, a
recombinant protein designed to reverse the anticoagulant effect in
patients treated with an oral or injectable Factor Xa inhibitor;
and cerdulatinib, a Syk/JAK inhibitor in development to treat
hematologic cancers. Portola's partnered program is focused on
developing selective Syk inhibitors for inflammatory conditions.
For more information, visit www.portola.com and follow the Company
on Twitter @Portola_Pharma.
Forward-looking Statements Statements contained
in this press release regarding matters that are not historical
facts are “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995. Because such
statements are subject to risks and uncertainties, actual results
may differ materially from those expressed or implied by such
forward-looking statements. Such statements include, but are
not limited to, statements regarding: andexanet alfa’s potential to
treat patients needing reversal of Factor Xa anticoagulation
effects, the projected number of patients that could benefit from
andexanet alfa, the potential for andexanet alfa to become the
first universal reversal agent for Factor Xa inhibitors and the
standard of care for managing major bleeding among patients on
Factor Xa inhibitors, our plans for pursuit of regulatory approval
of andexanet alfa, and the likelihood of clinical, regulatory and
commercial success for andexanet alfa and our other product
candidates. Risks that contribute to the uncertain nature of the
forward-looking statements include: our expectation that we will
incur losses for the foreseeable future and will need additional
funds to finance our operations; the accuracy of our estimates
regarding our ability to initiate and/or complete our clinical
trials and the timing and expense of these trials; the pace of
enrollment in our clinical trials; the results of our clinical
trials related to the efficacy and safety of our product
candidates; the risk that regulatory approval of our product
candidates may not be received in a timely manner, or at all; our
potential inability to manufacture andexanet alfa and our other
product candidates on a commercial scale in a timely or
cost-efficient manner; the accuracy of estimates regarding our
expenses and capital requirements; our ability to successfully
build a hospital-based sales force and commercial infrastructure;
regulatory developments in the United States and foreign countries;
our ability to obtain and maintain intellectual property protection
for our product candidates; and our ability to retain key
scientific or management personnel. These and other risks and
uncertainties are described more fully in our most recent filings
with the Securities and Exchange Commission, including our Annual
Report on Form 10-K, which was filed on March 2, 2015, and
Quarterly Report on Form 10-Q for the third quarter of 2015, which
was filed on November 9, 2015. All forward-looking statements
contained in this press release speak only as of the date on which
they were made. We undertake no obligation to update such
statements to reflect events that occur or circumstances that exist
after the date on which they were made.
1 Truven, MarketScan Commercial, Medicare Supplemental,
last 12 months ending April 30, 2015. Medicaid accounts for ~5% of
the total bleed-related admissions.
Investor Contact:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
Media Contact:
Julie Normart
W2O Group
jnormart@w2ogroup.com
Portola Pharmaceuticals (NASDAQ:PTLA)
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