Portola Pharmaceuticals (Nasdaq:PTLA) today announced that it has
initiated a Phase 2 proof-of-concept study to evaluate andexanet
alfa, the company's investigational Factor Xa inhibitor reversal
agent, as a reversal agent for Daiichi Sankyo's oral, once-daily,
direct Factor Xa inhibitor edoxaban in healthy volunteers. Portola
is developing andexanet alfa, an FDA-designated breakthrough
therapy, as a potential first-in-class antidote to reverse the
anticoagulation activity of Factor Xa inhibitor-treated patients
who are suffering a major bleeding episode or who require emergency
surgery. The Company is pursuing an Accelerated Approval pathway
for andexanet alfa, which is the only agent that has demonstrated
reversal of the anticoagulation activity of Factor Xa inhibitors as
measured by biomarkers, including anti-Factor Xa activity, in human
studies.
"Portola was one of the first companies to identify an important
unmet need for an agent to reverse the anticoagulation activity of
the Factor Xa inhibitor class of drugs. Our development team
leveraged our cutting-edge science in the area of thrombosis to
create andexanet alfa as a possible solution," said John T.
Curnutte, M.D., Ph.D., executive vice president, research and
development at Portola. "The initiation of this new Phase 2 study
with edoxaban moves us closer to our goal of bringing andexanet
alfa to market as a universal antidote for Factor Xa
inhibitors."
In the randomized, double-blind, placebo-controlled, cohort
dose-escalation Phase 2 study, healthy volunteers will be treated
on days 1-6 with edoxaban 60 mg once a day and then randomized in a
6:3 ratio to intravenous andexanet alfa or placebo on day 6. The
first dose cohort will evaluate a 600 mg bolus dose of andexanet
alfa or placebo in 9 healthy volunteers. Pharmacodynamic and safety
data will be collected through day 48.
Portola announced in June 2013 that it had entered into a
clinical collaboration agreement with Daiichi Sankyo for the Phase
2 study. Daiichi Sankyo provides funding for the study while
Portola retains full worldwide commercial rights to andexanet
alfa.
About the Need for a Factor Xa Inhibitor Reversal
Agent
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. Clinical
trial results suggest 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, depending on the patient's underlying medical condition.
Development of an agent specifically designed to reverse the
activity of Factor Xa inhibitors may provide an important treatment
option for patients who experience a major bleeding event or
require emergency surgery.
About Andexanet Alfa
Andexanet alfa is a first-in-class recombinant, modified Factor
Xa molecule being developed as a direct reversal agent for patients
receiving a Factor Xa inhibitor who suffer a major bleeding episode
or who may require emergency surgery. Andexanet alfa acts as a
Factor Xa decoy that targets and sequesters with high specificity
both direct and indirect 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.
About Andexanet Alfa Clinical Development
Program
Portola is currently evaluating andexanet alfa in a Phase 3
study with Bristol-Myers Squibb Company and Pfizer Inc.'s Factor Xa
inhibitor Eliquis® (apixaban). The randomized, double-blind,
placebo-controlled study is designed to evaluate the efficacy of
andexanet alfa in reversing Eliquis-induced anticoagulation rapidly
after an IV bolus and sustaining that effect through a continuous
infusion. The Company expects to report the bolus infusion data in
the fourth quarter of this year, followed by bolus-plus-continuous
infusion data in early 2015.
Another Phase 3 study is expected to begin in the first half of
2014 that will evaluate andexanet alfa with Bayer HealthCare and
Janssen Pharmaceuticals Inc.'s XARELTO® (rivaroxaban).
Results from two Phase 2 proof-of concept studies demonstrated
that andexanet alfa immediately reversed the anti-Factor Xa
activity of Eliquis and XARELTO in healthy volunteers. Andexanet
alfa provided temporary reversal through the administration of an
IV bolus infusion or sustained reversal with the addition of an
extended infusion. In clinical practice, patients with bleeding due
to a traumatic injury may require short-acting reversal of their
anticoagulant, while those who need emergency surgery may require
long-acting reversal. Andexanet alfa has the potential to address
numerous clinical scenarios by allowing for flexible and controlled
reversal. Andexanet alfa has been shown to be well tolerated in
clinical studies, which have included more than 90 volunteers, with
no thrombotic events, serious adverse events or antibodies to
Factor Xa or Factor X observed.
Phase 2 proof-of-concept studies with Daiichi Sankyo's edoxaban,
Portola's Factor Xa inhibitor betrixaban, and the indirect Factor
Xa inhibitor enoxaparin are ongoing or planned.
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 thrombosis and hematology.
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
first-in-class therapies.
Betrixaban
Portola's lead compound, betrixaban, is an oral, once-daily
Factor Xa inhibitor 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
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 lead development candidate, 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) and PRT2607
Portola's third wholly-owned product candidate, cerdulatinib
(PRT2070), 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. Portola's
fourth program is partnered with Biogen Idec and is focused on the
development of PRT2607, a selective Syk inhibitor.
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, anticipated growth
in the market for anticoagulants, clinical trial cost, design and
timing, and the potential efficacy, safety and activity of
andexanet alfa, betrixaban 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).
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)
Graphique Historique de l'Action
De Juin 2024 à Juil 2024
Portola Pharmaceuticals (NASDAQ:PTLA)
Graphique Historique de l'Action
De Juil 2023 à Juil 2024