Portola Pharmaceuticals (Nasdaq:PTLA) today announced publication
of the design and rationale of the Phase 3 APEX (Acute Medically
Ill VTE Prevention with Extended Duration Betrixaban) Study in the
March 2014 issue of the American Heart Journal.i Betrixaban, the
Company's wholly-owned, oral, once-daily Factor Xa anticoagulant,
is being evaluated in the only ongoing global, pivotal study of
in-hospital and post-discharge prevention of venous thromboembolism
(VTE), or blood clots, in patients who have been hospitalized for
medical conditions, such as heart failure, stroke, infection and
pulmonary disease.
"Extended VTE prevention in acute medically ill patients is an
important unmet medical need because studies have shown that
well-identified patients are at high risk for life-threatening
blood clots beginning at hospital admission and for several weeks
after discharge. However, no anticoagulant therapy has been
approved to protect patients during this period of risk. APEX is
the only ongoing pivotal study evaluating one continuous therapy
for hospital-to-home VTE prevention for these patients," said
Alexander T. Cohen, M.D., co-chairman of the APEX Executive
Committee and honorary consultant vascular physician, Department of
Haemostasis and Thrombosis, Guy's and St Thomas' Hospitals at
King's College London, UK. "Additionally, these patients are often
renally compromised, and because betrixaban has a lower level of
renal clearance compared with other drugs in its class, APEX is the
first pivotal study to allow inclusion of patients with severe
renal impairment."
The prospective, randomized, double-blind, multicenter,
multinational APEX Study is evaluating the superiority of
extended-duration anticoagulation with oral betrixaban (for up to
35 days in hospital and post-discharge) compared with standard of
care anticoagulation with injectable enoxaparin (for 10 days) for
VTE prevention in acute medically ill patients. The study will
enroll close to 7,000 patients at more than 425 study sites
worldwide. The primary efficacy endpoint is the composite of
asymptomatic proximal deep venous thrombosis, symptomatic deep
venous thrombosis, non-fatal pulmonary embolism or VTE-related
death through day 35. The primary safety outcome is the occurrence
of major bleeding.
"The APEX study is using biomarkers to identify and enroll
patients at highest risk of blood clots who will most likely
benefit from treatment with betrixaban, including those with
elevated blood levels of D-dimer (a protein fragment present after
a blood clot has developed) and those over age 75," said John T.
Curnutte, M.D., Ph.D., executive vice president, research and
development at Portola. "We believe that betrixaban, because of its
unique pharmacokinetic properties, has the potential to be the
first novel oral anticoagulant to prevent VTE in this patient
population without the significant increase in the rate of major
bleeding seen with other Factor Xa inhibitors. Betrixaban could be
the first anticoagulant approved for 'hospital to home' use and the
standard of care in this large market of more than 30 million
patients worldwide."
About VTE Prevention in Acute Medically Ill
Patients
Acute medically ill patients, who are at high risk for
developing blood clots, are hospitalized due to a serious medical
condition such as heart failure, stroke, infection, pulmonary
disease or rheumatic disease. They are often elderly, frail,
renally compromised and taking multiple concomitant medications.
Acute medically ill patients represent the single largest category
of patients at risk for thrombosis, with an estimated 30 million
patients worldwide. Enoxaparin, the current standard of care, is
limited to hospital use because it is administered by injection and
is associated with an increase in major bleeding when administered
in this population for extended use. The other novel oral Factor Xa
inhibitors that have been studied in this population have also been
associated with an increased rate of major bleeding. A
well-identified population of medical patients is at risk for
life-threatening blood clots during hospitalization and for several
weeks after discharge, but currently no anticoagulant is approved
to treat these patients for this extended time period.
About Betrixaban
Betrixaban is a small molecule anticoagulant that directly
inhibits the activity of Factor Xa, an important validated target
in the blood coagulation pathway, to prevent thrombosis. The
compound has three properties that differentiate it from other oral
anticoagulants: it has the longest half-life of all of the Factor
Xa inhibitors for true once-daily dosing; it has a low level of
clearance through the kidneys and has been studied in patients with
severe renal impairment (excluding dialysis patients); and it is
not metabolized by CYP3A4, a liver enzyme that metabolizes many
drugs and can lead to drug-drug interactions. These properties are
important for acute medically ill patients and may allow betrixaban
to demonstrate efficacy in this population without the increase in
major bleeding seen with other Factor Xa inhibitors.
Portola has full worldwide development and commercial rights to
betrixaban.
About Portola Pharmaceuticals, Inc.
Portola Pharmaceuticals is a biopharmaceutical company
developing product candidates that have the potential to represent
significant advances in the fields of hematology and inflammation.
The Company is advancing its three wholly-owned hematology programs
using novel biomarker and genetic approaches that may increase the
likelihood of clinical, regulatory and commercial success of its
first-in-class 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 a biomarker-based Phase 3 study
for "hospital to home" prophylaxis of venous thromboembolism (VTE)
in acute medically ill patients. Betrixaban's properties may be
particularly suited to potentially demonstrate efficacy without
significantly increasing bleeding in this patient population.
Currently, there is no anticoagulant approved for extended-duration
VTE prophylaxis in acute medically ill
patients.
Andexanet Alfa
Portola's second product candidate in the area of thrombosis,
andexanet alfa, has the potential to be a first-in-class reversal
agent to reverse the effects of Factor Xa inhibitors in patients
who suffer a major bleeding episode or who require emergency
surgery. Portola has entered into clinical collaboration agreements
with all of the manufacturers of direct Factor Xa inhibitors,
including 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. Andexanet alfa has been designated as a Breakthrough Therapy
by the U.S. Food and Drug Administration.
Cerdulatinib* (PRT2070)
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
studied in patients with 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:
anticipated growth in the market for anticoagulants, clinical trial
enrollment, cost, design and timing, and the potential efficacy,
safety and activity of betrixaban, andexanet alfa and cerdulatinib.
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; 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 2013
Annual Report on Form 10-K filed with the Securities and Exchange
Commission on March 3, 2014. 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.
*Cerdulatinib is a proposed International Nonproprietary Name
(pINN).
iCohen AT, Harrington R, Goldhaber SZ, Hull R, Gibson CM, et al.
The design and rationale for the Acute Medically Ill Venous
Thromboembolism Prevention with Extended Duration Betrixaban (APEX)
study. Am Heart J. 2014;167:335-41.
CONTACT: Investor Contact:
Alexandra Santos
Portola Pharmaceuticals
ir@portola.com
650.246.7239
Media Contact:
Joey Fleury
BrewLife
jfleury@brewlife.com
415.946.1090
Portola Pharmaceuticals (NASDAQ:PTLA)
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