Portola's Factor Xa Inhibitor Betrixaban Successfully Passes Futility Analysis in Phase 3 APEX Study; Trial Continues as Plan...
05 Février 2015 - 10:06PM
Portola Pharmaceuticals (Nasdaq:PTLA) today announced that the
Independent Data Monitoring Committee (IDMC) recommended that the
Phase 3 APEX (Acute Medically Ill VTE Prevention with Extended
Duration Betrixaban) Study of betrixaban continue as planned
without modification based on the successful completion of a
pre-specified futility analysis. In making this recommendation, the
IDMC evaluated preliminary efficacy trends and safety reports from
the first half of the study. The APEX Study is assessing
betrixaban, an oral, once-daily, investigational Factor Xa
inhibitor, for both hospital and post-discharge prevention of
venous thromboembolism (VTE), or blood clots, in acute medically
ill patients. Acute medically ill patients are those who are
hospitalized for serious medical conditions, such as heart failure,
stroke, infection and pulmonary disease.
The APEX Study is over 65 percent enrolled at over 450 sites
worldwide. The Company expects to complete enrollment by the end of
this year and report top-line results in early 2016.
"Given the positive outcome of the APEX futility analysis and
the observation that the blinded aggregate VTE event rates in the
study to date are on track, we are confident that we are enrolling
patients who are at high risk of blood clots and who are most
likely to benefit from betrixaban," said John T. Curnutte, M.D.,
Ph.D., executive vice president, research and development for
Portola. "Our goal is for betrixaban to be the first Factor Xa
inhibitor approved for both in-hospital and post-discharge
prevention of VTE in acute medically ill patients and to be the
standard of care for this patient population."
Bill Lis, chief executive officer of Portola added, "This
positive development with the APEX Study follows on the heels of
recent positive Phase 3 data for our FDA breakthrough-designated
Factor Xa inhibitor antidote, andexanet alfa, giving Portola a
robust franchise in the growing multi-billion dollar global
thrombosis market. Additionally, based on promising clinical data
seen to date with our Syk-JAK inhibitor, cerdulatinib, we are
making significant progress toward achieving our goal of becoming a
fully commercialized biopharmaceutical company with potentially
groundbreaking products in two of the largest fields of innovation
-- thrombosis and hematologic cancers."
About the APEX Study
The global, pivotal Phase 3 APEX Study is evaluating the
superiority of extended-duration anticoagulation with oral
betrixaban (for at least 35 days) compared with standard of care
anticoagulation with injectable enoxaparin (for up to 14 days) for
prevention of VTE in acute medically ill patients. The study is
using a biomarker-based trial design to identify and enroll
patients who are at highest risk of VTE and most likely to benefit
from betrixaban – specifically those with an elevated level of
D-dimer (a protein fragment present after a blood clot has
developed) and those over age 75. The APEX Study is the only
biomarker-based Phase 3 study for hospital-to-home prophylaxis of
VTE in acute medically ill patients. It is also the only pivotal
thrombosis trial evaluating a single anticoagulant for VTE
prophylaxis in both the in-hospital and post-discharge settings.
More than half of VTE events occur after hospital discharge in this
patient population.i No anticoagulant is approved to treat these
patients for this extended time period.
About Betrixaban
Betrixaban is an investigational small molecule anticoagulant
that directly inhibits the activity of Factor Xa, an important
validated target in the blood coagulation pathway, to prevent
thrombosis. Betrixaban has distinct properties that may allow it to
demonstrate clinical benefit without the significant imbalance in
the risk of fatal bleeding seen with other agents in the class.
These include a 19-25-hour half-life for true once-daily dosing; a
low peak-to-trough drug concentration ratio that minimizes
anticoagulant variability; the lowest renal clearance in the class;
and no significant CYP3A4 metabolism, which may reduce the risk of
drug-drug interactions. Betrixaban has been studied in patients
with all degrees of renal function, including those with severe
renal impairment (excluding dialysis patients).
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 recombinant modified human Factor Xa molecule,
has the potential to be a first-in-class antidote to reverse the
effects of Factor Xa inhibitors in patients who suffer a major
bleeding episode or who require emergency surgery. Andexanet alfa
has been designated as a breakthrough therapy by the U.S. Food and
Drug Administration (FDA). 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 ANNEXA™
(Andexanet Alfa a Novel Antidote to the Anticoagulant Effects of
fXA Inhibitors) registration studies and in a Phase 4 study to
support FDA approval under an Accelerated Approval pathway.
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:
enrollment of a patient population in APEX that is at a high risk
of blood clots and most likely to benefit from betrixaban; timing
of completion of APEX enrollment and Portola's report of trial
results; potential of betrixaban to demonstrate efficacy without
the significant increase in bleeding; potential indications and
market acceptance of betrixaban; potential for betrixaban to become
standard of care; and Portola's intention of becoming a
commercialized biopharmaceutical company with potentially
groundbreaking products for thrombosis and hematologic cancers.
Risks that contribute to the uncertain nature of the
forward-looking statements include: the accuracy of Portola's
estimates regarding its ability to 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 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 for the third quarter of 2014, which was filed
on November 4, 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.
i Source: Amin et al. (2012). Duration of Venous Thromboembolism
Risk Across a Continuum in Medically Ill Hospitalized Patients.
Journal of Hospital Medicine, 7(3), 231-238.
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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