--Updated Safety and Efficacy Data From Phase 1
Part of Ongoing Phase 1/2a Study to be Presented--
--Company Opens Enrollment in Expansion Cohorts
in Phase 2a Part of Study--
Portola Pharmaceuticals (Nasdaq:PTLA) today announced that it will
present updated safety and efficacy data from the Phase 1 part of
its ongoing Phase 1/2a proof-of-concept study of cerdulatinib in
patients with hematologic cancers who have failed multiple
therapies. The data will be presented in a poster discussion
session at the upcoming 2015 American Society of Clinical Oncology
(ASCO) Annual Meeting, which is taking place from May 29-June 2 in
Chicago. The Company also announced that it has initiated two
clinical expansion cohorts in the Phase 2a part of the study, which
will enroll patients with chronic lymphocytic leukemia (CLL) or
follicular lymphoma (FL) who have progressed or relapsed on other
therapies. The Phase 1 part of the study is ongoing as the maximum
tolerated dose has not yet been achieved.
Cerdulatinib is an oral, dual Syk-JAK kinase inhibitor that
Portola is developing to treat patients with hematologic cancers,
specifically those who have relapsed or who have not responded to
prior therapies.
"We have demonstrated initial clinical proof-of-concept in the
ongoing dose-escalation Phase 1 part of the study showing that
cerdulatinib is active and well tolerated. We have seen partial
responses with this unique dual pathway anti-cancer agent and
clinical activity in patients who failed or could not tolerate
prior therapy with BTK and PI3K kinase inhibitors," said John T.
Curnutte, M.D., Ph.D., executive vice president, research and
development for Portola. "Based on these promising preliminary
data, we have advanced cerdulatinib to the next stage of clinical
development to study larger expansion cohorts in the Phase 2a part
of the study. We will evaluate two cohorts of patients with CLL or
FL whose cancer has progressed or relapsed following treatment with
multiple agents. These patients have limited treatment options. Our
goal is to identify patient populations with significant unmet need
in whom the inhibition of both Syk and JAK by cerdulatinib may
provide clinical benefit."
The cerdulatinib abstract is now available at
abstracts.asco.org. Details regarding the cerdulatinib poster
presentation, which will include additional data not currently
available in the published abstract, follow.
Abstract Title: Phase 1 open-label dose
escalation study of the dual SYK/JAK inhibitor cerdulatinib
(PRT062070) in patients with relapsed/refractory B-cell
malignancies: Safety profile and clinical activity (abstract #8531;
poster board #348)
Presenter: Ian W. Flinn, M.D., Ph.D., director,
Hematologic Malignancies Research, Program, Sarah Cannon Research
Institute, Memphis
Session Name: Poster Discussion Session --
Lymphoma and Plasma Cell Disorders
Session Date, Time and Location: Sunday, May
31, from 4:30-5:45 p.m. CT; S Hall A, McCormick Place
About the Phase 1/2a Study
The open-label, multicenter, Phase 1/2a proof-of-concept study
is assessing the safety, pharmacokinetics, pharmacodynamics and
clinical activity of oral cerdulatinib in patients with CLL and
non-Hodgkin lymphoma. In the multi-dose, dose-escalation Phase 1
part of the study, cerdulatinib is being administered orally once a
day to sequential dose cohorts at increasing dose levels until the
maximum tolerated dose is identified. The maximum tolerated dose
has not yet been identified and the study is continuing.
The clinical expansion cohorts in the Phase 2a part of the study
will evaluate the safety and efficacy of cerdulatinib in cancer
types identified based on responses seen in the dose-escalation
phase of the study. Two groups of up to 40 patients each will be
enrolled in the clinical expansion cohorts. One group will include
patients with CLL or small lymphocytic leukemia (SLL) whose disease
has progressed following prior therapies. The other group will
include patients with FL who have progressed or relapsed on prior
therapies (such as rituximab and bendamustine).
About Cerdulatinib
Cerdulatinib is an oral, dual Syk-JAK inhibitor with a unique
mechanism of action. It uniquely inhibits two key signaling
pathways that promote cancer cell growth in certain hematologic
malignancies: the B-cell receptor pathway via Syk and key cytokine
receptors via JAK. With its dual pathway mechanism, cerdulatinib
may be more effective in specific patients than a single pathway
agent, such as those resistant to current therapies or those with
known heterogeneous cellular mutations. Preclinical data suggested
that cerdulatinib has anti-tumor activity in patients who did not
adequately respond to, or relapsed on, other treatments due to
defined mutations.
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 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 programs are 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, an FDA-designated breakthrough therapy, is a
recombinant protein designed to reverse the anticoagulant effect in
patients treated with an oral or injectable Factor Xa inhibitor.
Andexanet alfa has the potential to be a first-in-class antidote
for anticoagulated patients who suffer a major bleeding episode or
require emergency surgery. Portola has entered into Phase 3
clinical collaboration agreements with all of the manufacturers of
direct Factor Xa inhibitors while retaining all commercial rights
to andexanet alfa. The Company is currently evaluating andexanet
alfa in the Phase 3 and Phase 4 ANNEXA™ (Andexanet Alfa a Novel
Antidote to the Anticoagulant Effects of FXa Inhibitors)
registration studies.
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:
Portola's plans for future clinical studies and pursuit of an
Accelerated Approval process for andexanet alfa, anticipated growth
in the market for anticoagulants, the potential indications,
efficacy, safety and activity of andexanet alfa, and the potential
market and indications for its other product candidates. 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; Portola's ability to manufacture andexanet alfa;
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-Q, which was filed on May 7, 2015. 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.
CONTACT: Investor Contact:
Michele Mantynen
Portola Pharmaceuticals
ir@portola.com
Media Contact:
Joey Fleury
BrewLife
jfleury@brewlife.com
Portola Pharmaceuticals (NASDAQ:PTLA)
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