Portola Pharmaceuticals Inc.® (Nasdaq:PTLA) today announced that it
has submitted a New Drug Application (NDA) to the U.S. Food and
Drug Administration (FDA) seeking approval to market betrixaban for
extended-duration prophylaxis of venous thromboembolism (VTE) in
acute medically ill patients with risk factors for VTE. Betrixaban,
an FDA Fast Track-designated investigational drug, is an oral,
once-daily Factor Xa inhibitor anticoagulant.
“Hospitalized acute medically ill patients are at significant
risk of experiencing a potentially life-threatening VTE event, both
during their stay and after discharge, especially within the first
four weeks. Yet, no drug is approved in the United States for
extended use to prevent VTE in these patients. We are committed to
bringing betrixaban to market to address this urgent unmet medical
need,” said John Curnutte, M.D. Ph.D., executive vice president,
research and development at Portola. “If approved, betrixaban would
be the first anticoagulant indicated for the prevention of VTE in
acute medically ill patients both during hospitalization and for an
extended period after the patient returns home.”
An estimated 22.5 million acute medically ill patients in the G7
countries are at risk of developing VTE, which includes both deep
vein thrombosis (DVT) and pulmonary embolism (PE), either while in
the hospital or following discharge. Each year, more than 1 million
VTE events 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. More than
half of VTE events occur after the patient is discharged from the
hospital. However, no anticoagulant, including enoxaparin or any of
the marketed oral Factor Xa inhibitors, is approved for extended
VTE prophylaxis for acute medically ill patients who are
hospitalized.
The NDA for betrixaban is supported by data from the pivotal
Phase 3 APEX Study, which enrolled 7,513 patients at more than 450
clinical sites worldwide and assessed the superiority of
extended-duration anticoagulation with oral betrixaban for 35 - 42
days compared with standard-duration injectable enoxaparin for 10+4
days in preventing VTE in high-risk acute medically ill patients.
Results showed that betrixaban reduced the incidence of VTE
compared with enoxaparin at a p value approaching statistical
significance (p=0.054) in the primary efficacy analysis subgroup of
3,870 patients with elevated D-dimer levels. It also significantly
reduced VTE in several pre-specified analyses of the primary
efficacy analysis subgroup as well as in the overall study
population (p=0.006) of 7,513 patients. No statistical difference
in major bleeding was observed between the betrixaban and
enoxaparin arms in either of the primary analysis patient subgroup
or in the overall study population.
“The submission of the Betrixaban NDA as planned is an important
accomplishment for Portola and our regulatory and clinical teams as
a first step towards a potential approval in 2017,” said Bill Lis,
chief executive officer of Portola. “Based on the totality of data
from the pivotal Phase 3 APEX study, there is strong support among
the medical community for the use of betrixaban in preventing VTE
in medically ill patients. We believe our dossier with the APEX
Study as the basis shows clear evidence of betrixaban’s efficacy
and safety. We look forward to working with the FDA as they review
our application.”
Portola expects a response from the FDA within 60 days as to
whether the NDA is complete and acceptable for filing. The Company
plans to submit a Marketing Authorization Application for approval
of betrixaban in the EU by the end of this year.
About the Need for an Oral
Anticoagulant for Extended-Duration Prevention of VTE in Acute
Medically Ill PatientsAcute medically ill patients are
those hospitalized for serious, common medical conditions,
including heart failure, stroke, infection and pulmonary disease.
Because of their underlying disorder or immobilization during
hospitalization, they are at increased risk of VTE, a serious and
potentially life-threatening blood clot (thrombus). VTE includes
both DVT, a blood clot in a deep vein in the leg, and PE, a blood
clot in a deep vein in the leg that breaks loose and travels to the
lungs, where it lodges, blocking the pulmonary artery or one of its
branches, where it can be fatal.
VTE is a major cause of preventable morbidity and mortality and
re-hospitalization in the acute medically ill patient population.
The incidence of VTE in acute medically ill patients has been
reported to be at least as high as in patients undergoing general
surgery.i Approximately 2.5 percent of high-risk medical patients
will experience a symptomatic VTE or VTE-related
death.ii Hospitalized patients are at significant risk of
experiencing VTE events both during their stay and after
discharge.iii They remain at increased risk for up to three
months following discharge, with the peak incidence occurring
within the first four weeks.iv, v
About BetrixabanBetrixaban, an investigational
drug, 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.
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 www.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 timing of a response from the
FDA to our NDA and our submission of a Marketing Authorization
Application in the EU, and potential approval of betrixaban. Risks
that contribute to the uncertain nature of the forward-looking
statements include the risk that our data fail to demonstrate
safety and efficacy to the satisfaction of the FDA or similar
regulatory authorities outside the United States and the risk that
the results from our APEX clinical trial may cause betrixaban
regulatory approval to be delayed, more costly or not be obtained
at all. 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 August 9, 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.
i Turpie AGG, Leizorovicz A. Prevention of venous
thromboembolism in medically ill patients: a clinical update.
Postgrad Med J. 2006;82(974):806-809.
ii Spyropoulos AC, Anderson FA, FitzGerald G, Decousus H, Pini
M, et al. Predictive and associative models to identify
hospitalized medical patients at risk for VTE. Chest.
2011;140:706-714.
iii Amin AN, Varker H, Princic N, Lin J, Thompson S, et al.
Duration of venous thromboembolism risk across a continuum in
medically ill hospitalized patients. J Hosp Med. 2012;7:231-8.
iv Heit JA, Melton LJ, Lohse CM, Petterson TM, Silverstein
MD, et al. Incidence of venous thromboembolism in hospitalized
patients vs. community residents. Mayo Clin Proc.
2001;76:1102-10.
v Cohen AT. Results from the VEG Registry. Presented at the
23rd Congress of the International Society on Thrombosis and
Haemostasis 57th Annual SSC Meeting, July 23-28, 2011, Kyoto
Japan.
Investor Contact:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
Media Contact:
Julie Normart
W2O Group
jnormart@w2ogroup.com
415.946.1087
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