Portola Pharmaceuticals (Nasdaq:PTLA) today announced that
andexanet alfa, a U.S. Food and Drug Administration
(FDA)-designated breakthrough therapy, has been granted orphan drug
designation by the FDA's Office of Orphan Products Development for
reversing the anticoagulant effect of direct or indirect Factor Xa
inhibitors in patients experiencing a serious uncontrolled bleeding
event or who require urgent or emergent surgery. Currently, there
is no approved antidote for these patients.
Andexanet alfa has the potential to become the first approved
universal antidote for Factor Xa inhibitors and the standard of
care to manage bleeding associated with these anticoagulants.
"Orphan drug designation for andexanet alfa recognizes its
potential to address a significant unmet medical need and to
advance the field by helping patients who currently have no
treatment options. Four factor prothrombin complex concentrates
(PCCs), which are indicated for warfarin reversal, have not
demonstrated reversal of Factor Xa inhibitor activity and have a
black box warning for fatal and non-fatal thromboembolic events,"
said Bill Lis, chief executive officer of Portola. "This
designation also provides significant business value to Portola,
including market exclusivity, tax credits and a waiver of FDA
marketing application fees."
The FDA's Orphan Drug Designation program provides orphan status
to drugs and biologics that are defined as those intended for the
safe and effective treatment, diagnosis or prevention of rare
diseases/disorders that currently affect fewer than 200,000 people
in the United States.i
Currently, millions of patients are treated with Factor Xa
inhibitors for short-term use or chronic conditions, and the
anticoagulant market is expected to continue to grow. Recent
patient dataii confirm earlier clinical trial results showing that,
annually, between 1-4 percent of patients treated with Factor Xa
inhibitors may experience major bleeding and an additional 1
percent may require emergency surgery. Annually, more than 100,000
U.S. patients are currently impacted and may benefit from an
antidote. With the expected increase in adoption of these
anticoagulants, Portola projects that this number will increase by
the year 2020 to up to 200,000 patients in the United States and an
estimated 500,000 patients in the G7 countries. Development of
a specific antidote designed to reverse the anticoagulant activity
of Factor Xa inhibitors may provide an important treatment option
for these patients.
About Andexanet Alfa
Andexanet alfa 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 has the potential to address numerous
clinical scenarios where an antidote is needed by allowing for
flexible and controlled reversal. This can be short-acting through
the administration of an IV bolus or longer-acting with the
addition of an extended infusion.
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 the Andexanet Alfa Clinical Development
Program
Portola is evaluating andexanet alfa in two randomized,
placebo-controlled Phase 3 ANNEXA™ (Andexanet Alfa a Novel Antidote
to the Anticoagulant Effects of fXA Inhibitors) registration
studies using pharmacodynamic endpoints agreed to with the FDA,
including anti-Factor Xa inhibitor units, to demonstrate efficacy.
The Company reported statistically significant results from the
first part of the Phase 3 ANNEXA-A study, which evaluated andexanet
alfa administered as a single IV bolus dose with Bristol-Myers
Squibb Company and Pfizer Inc.'s direct Factor Xa inhibitor
apixaban, and from the first part of the Phase 3 ANNEXA-R study
with Bayer HealthCare and Janssen's direct Factor Xa inhibitor
rivaroxaban. The second parts of the ANNEXA-A and ANNEXA-R studies
are ongoing and are evaluating a bolus plus a continuous infusion
of andexanet alfa to sustain the reversal of anticoagulation
activity.
ANNEXA-4, a Phase 4 single-arm 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 is also ongoing. Data from the
ANNEXA-A and ANNEXA-R studies, as well as data from a small number
of patients from ANNEXA-4, will serve as the clinical basis of a
Biologics License Application (BLA), which Portola plans to submit
under an Accelerated Approval pathway.
Results from four separate Phase 2 proof-of concept studies with
apixaban, rivaroxaban, edoxaban and enoxaparin in healthy
volunteers demonstrated that andexanet alfa immediately reversed
the anticoagulation activity of each Factor Xa inhibitor and that
the reversal could be sustained. Andexanet alfa has been shown to
be well tolerated in clinical studies, which have included more
than 140 healthy volunteers. No thrombotic events or antibodies to
Factor Xa or Factor X have been observed.
A Phase 2 proof-of-concept study with Portola's investigational
Factor Xa inhibitor betrixaban is planned.
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. Portola's partnered program is focused on developing
selective Syk inhibitors for inflammatory conditions.
Betrixaban
Portola's wholly-owned, oral, once-daily Factor Xa inhibitor
betrixaban is being evaluated in the only biomarker-based Phase 3
study for hospital-to-home prophylaxis of venous thromboembolism
(VTE) in acute medically ill patients. Betrixaban's distinct
properties may have the potential to allow the agent to demonstrate
efficacy without the significant increase in the rate of major
bleeding that was seen in this patient population with other Factor
Xa inhibitors. If approved, betrixaban could be the first
anticoagulant for both hospital and post-discharge VTE prophylaxis
and the standard of care in this large market of more than 20
million patients in the G7 countries alone.
Andexanet Alfa
Andexanet alfa, a U.S. Food and Drug Administration
(FDA)-designated breakthrough therapy, is a recombinant protein
designed to reverse the anticoagulant activity in patients treated
with an oral or injectable Factor Xa inhibitor. Andexanet alfa has
the potential to be a first-in-class antidote for anticoagulated
patients who suffer a major bleeding episode or require emergency
surgery. Portola has entered into Phase 3 clinical collaboration
agreements with all of the manufacturers of direct Factor Xa
inhibitors – Bristol-Myers Squibb and Pfizer (Eliquis
[apixaban]), Bayer HealthCare and Janssen Pharmaceuticals (XARELTO®
[rivaroxaban]), and Daiichi Sankyo (edoxaban) – while retaining all
commercial rights to andexanet alfa. The Company is currently
evaluating andexanet alfa in the Phase 3 and Phase 4 ANNEXA™
(Andexanet Alfa a Novel Antidote to the Anticoagulant Effects of
fXA Inhibitors) registration studies.
Cerdulatinib
Portola's product candidate in the area of hematologic cancer,
cerdulatinib, is an orally available molecule that uniquely
inhibits two validated tumor proliferation pathways – spleen
tyrosine kinase (Syk) and janus kinase (JAK). It is currently being
evaluated in a Phase 1/2a proof-of-concept study in patients with B
cell leukemias or lymphomas with a focus on genetically-defined
subtypes, as well as in patients who have failed therapy due to
relapse or acquired mutations.
For more information, visit www.portola.com and follow the
Company on Twitter @Portola_Pharma.
Forward-Looking Statement
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:
Portola's plans for future clinical studies and pursuit of an
accelerated approval process for andexanet alfa, timing of the
submission of our BLA for andexanet alfa, anticipated benefits of
an orphan drug designation, anticipated growth in the market for
anticoagulants, the potential market, indications, efficacy, safety
and activity of andexanet alfa, and the potential market and
indications for Portola's other product candidates. Risks that
contribute to the uncertain nature of the forward-looking
statements include: the accuracy of Portola's estimates regarding
its ability to initiate and/or complete its clinical trials; the
success of Portola's clinical trials and the demonstrated efficacy
of Portola's product candidates thereunder; the accuracy of
Portola's estimates regarding its expenses and capital
requirements; Portola's ability to manufacture andexanet alfa;
regulatory developments in the United States and foreign countries;
Portola's ability to obtain and maintain intellectual property
protection for its product candidates; and the loss of key
scientific or management personnel. These and other risks and
uncertainties are described more fully in Portola's most recent
filings with the Securities and Exchange Commission, including its
Annual Report on Form 10-K and most recent Quarterly Report on Form
10-Q. All forward-looking statements contained in this press
release speak only as of the date on which they were made. Portola
undertakes no obligation to update such statements to reflect
events that occur or circumstances that exist after the date on
which they were made.
i
http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm.
Accessed February 24, 2015.
ii Source: Truven MARKETSCAN® Commercial, Medicare Supplemental
and Medicaid Database (12 months ending September 2013).
CONTACT: Media: Joey Fleury, BrewLife,
jfleury@brewlife.com, 415.946.1090
Investors: Alexandra Santos, Portola Pharmaceuticals,
ir@portola.com, 650.246.7239
Portola Pharmaceuticals (NASDAQ:PTLA)
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