Portola Pharmaceuticals Announces Full Results of Phase 3 APEX Study of Betrixaban Presented at International Society on Thro...
27 Mai 2016 - 2:45PM
--Company Plans to Submit NDA and MAA for
Betrixaban in Second Half of 2016--
Portola Pharmaceuticals Inc.® (NASDAQ:PTLA) announced that the full
results of its pivotal Phase 3 APEX (Acute Medically Ill VTE
Prevention with Extended Duration Betrixaban) Study were presented
today for the first time in a late-breaking clinical trial session
(http://investors.portola.com/) at the 62nd Annual International
Society on Thrombosis and Haemostasis (ISTH) Scientific and
Standardization Committee (SSC) Meeting. Full results from the APEX
trial were published simultaneously online in
The
New England Journal of
Medicine (http://www.nejm.org/doi/full/10.1056/NEJMoa1601747).
Portola announced topline results (http://topline.portola.com) from
the APEX trial in March 2016.
APEX, which enrolled 7,513 patients at more than 450 clinical
sites worldwide, assessed the superiority of extended-duration
betrixaban for 35 days compared to standard-duration enoxaparin for
10+4 days. The trial was designed in cooperation with the FDA and
EMA to incorporate a novel patient enrichment and statistical
analysis plan derived from the 2012 FDA guidance document on
enrichment strategies for clinical trials.
“In a pre-specified subgroup of medically ill patients who were
D-dimer positive, extended- duration betrixaban demonstrated a
reduction in VTE events approaching statistical significance. In
the pre-specified exploratory analyses of central lab D-dimer
values and in progressively larger cohorts, including all study
patients, the data demonstrated a consistent and significant
reduction in VTE with betrixaban with no statistical difference in
major bleeding between the betrixaban and enoxaparin arms,” said C.
Michael Gibson, M.S., M.D., senior author of the NEJM publication
and APEX Executive Committee Member and Steering Committee
Chairman.
Betrixaban, an investigational drug, is an oral, once-daily
Factor Xa inhibitor anticoagulant. It is an FDA-designated Fast
Track therapy for extended-duration VTE prophylaxis in acute
medically ill patients. Acute medically ill patients are
hospitalized for serious common medical conditions, such as heart
failure, stroke, infection and pulmonary disease. Despite the use
of standard of care anticoagulation, a significant number of
patients will suffer a VTE event. Over half of these events will
occur after discontinuation of enoxaparin and hospital discharge.
Currently no anticoagulant, including any of the marketed oral
Factor Xa inhibitors, is approved or guideline-recommended for
extended-duration VTE prophylaxis in this patient population.
“The APEX Study results show consistent evidence that VTE events
can be reduced with betrixaban with no statistical difference in
major bleeding between the betrixaban and enoxaparin arms. This is
particularly true for the most clinically relevant symptomatic
disease where we observed a 30 to 45 percent reduction in events
over the duration of the study,” said lead author of the NEJM
publication Alexander (Ander) T. Cohen, MBBS, M.Sc., M.D., FRACP,
APEX Co-Principal Investigator and Co-Chairman of the APEX
Executive Committee and Consultant Physician at Guy’s and St
Thomas’ NHS Foundation Trust. “Such meaningful results in an area
where there is currently no available recommended therapy offer
important potential benefits for public health worldwide. Based on
the number of reduced events in APEX, this could translate into
preventing over 100,000 additional VTE events per year and over
50,000 deaths from pulmonary embolism in the G7 countries if
betrixaban is approved.”
Later this year, Portola plans to submit the APEX Study data as
part of a New Drug Application (NDA) for betrixaban in the United
States and as part of a Marketing Authorization Application (MAA)
in the EU.
“We recently held a pre-NDA meeting with the FDA where we
reconfirmed Fast Track Designation for betrixaban in this
indication and agreed to upcoming meetings in preparation for an
NDA submission in the second half of 2016,” said Bill Lis, chief
executive officer of Portola.
About VTE in Acute Medically Ill PatientsAn
estimated 20 million acute medically ill patients in the G7
countries are at risk of developing VTE either while in the
hospital or following discharge. Each year, more than 1 million
VTEs and 150,000 VTE-related deaths occur in acute medically ill
patients in the G7 countries, despite the standard use of
injectable enoxaparin and other heparins in the hospital. Although
more than half of VTE events occur after the patient is discharged
from the hospital, no anticoagulant, including enoxaparin or any of
the marketed oral Factor Xa inhibitors, is approved for extended
VTE prophylaxis in the more than 24 million medically ill patients
hospitalized in the G7 countries annually.
About Betrixaban Betrixaban directly inhibits
the activity of Factor Xa, an important validated target in the
blood coagulation pathway, to prevent life-threatening thrombosis.
Betrixaban has distinct properties that may allow it to demonstrate
clinical benefit without the significant imbalance in the risk of
major bleeding seen with other agents in the class. These include a
19-25-hour half-life for once-daily dosing; a low peak-to-trough
drug concentration ratio that minimizes anticoagulant variability;
low renal clearance; and no significant CYP3A4 metabolism, which
may reduce the risk of drug-drug interactions.
Webcast DetailsPortola will host an investor
event today, May 27, 2016, at 5:00-6:00 p.m. CEST/11 a.m.-12 p.m.
EDT in which members of the Company’s senior management team and
Drs. Gibson and Cohen will present and discuss the full APEX Study
data. Webcast participants will be able to participate in a live
Q&A session via the webcast portal. To access the webcast of
the live event, go to the Investor Relations section of the
Company's website at investors.portola.com. A replay will be
available on the Company’s website for 30 days following the live
event, also at investors.portola.com.
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
three programs, including betrixaban, an oral, once-daily Factor Xa
inhibitor; AndexXa™ (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
portola.com and follow the Company on Twitter @Portola_Pharma.
Forward-looking StatementsStatements 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 the potential for study results to
support an application for regulatory approval of betrixaban, the
potential for betrixaban, subject to regulatory approval, to play a
role in both in-hospital and post-discharge reduction of VTE events
and deaths, our interpretation and characterization of APEX Study
results and the anticipated timing of our submission of filings
seeking regulatory approval. Risks that contribute to the uncertain
nature of the forward-looking statements include the results of
discussions with regulatory authorities regarding interpretation of
full APEX Study results, that the FDA and other regulatory
authorities may not approve betrixaban, whether the clinical
results of betrixaban will meet the regulatory requirements for
approval, whether regulatory submissions will occur or will be
submitted in a timely manner and that marketing approvals may not
be granted or, if granted, may have significant limitations on
their use or require additional studies. These and other risks and
uncertainties are described more fully in our most recent filings
with the Securities and Exchange Commission, including our most
recent quarterly report on Form 10-Q, which was filed on May 6,
2016. 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.
Investor Contact:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
1.650.246.7252
Media Contact:
Julie Normart
W2O Group
jnormart@w2ogroup.com
1.559.974.3245
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