Portola Pharmaceuticals Announces Initiation of Phase 1/2 Clinical Study of PRT2070
09 Octobre 2013 - 2:00PM
Portola Pharmaceuticals, Inc. (Nasdaq:PTLA) today announced that it
has initiated a Phase 1/2 clinical study of PRT2070, a novel, oral,
dual spleen tyrosine kinase (Syk) and janus kinase (JAK) inhibitor.
The compound is being developed for patients with
genetically-defined hematologic cancers, as well as for patients
who have failed therapy due to relapse or acquired mutations.
PRT2070's dual mechanism of action, which targets two validated
pathways for tumor survival and proliferation, has strong
scientific rationale in these settings, where other compounds have
failed to demonstrate clinical benefit.
"We are pleased to advance our third wholly-owned program
focused on addressing an unmet medical need into the clinic. Our
goal with PRT2070 is to develop an effective and well- tolerated
oral drug for the thousands of lymphoma and leukemia patients who
are impacted by difficult- to-treat genetic subtypes or who become
unresponsive to available therapies," said John T. Curnutte, M.D.,
Ph.D., executive vice president of research and development for
Portola. "With PRT2070, we have seen promising in vitro activity in
cell lines with specific mutations and in tumor cells that have
lost sensitivity to other compounds due to acquired mutations. We
look forward to seeing initial clinical data for this agent in
2014."
The open-label, multicenter, Phase 1/2 proof-of-concept study
will assess the safety, pharmacokinetics, pharmacodynamics and
clinical activity of oral PRT2070. In the multi-dose,
dose-escalation Phase 1 part of the study, PRT2070 will be
administered to sequential dose cohorts at increasing dose levels
until the maximum tolerated dose is identified. The Phase 1
portion of the study can include patients with relapsed or
refractory chronic lymphocytic leukemia (CLL)/small lymphocytic
lymphoma and non-Hodgkin lymphoma (NHL) (including diffuse large
B-cell lymphoma, follicular lymphoma and mantle cell lymphoma). The
Phase 2 part of the study is a cohort expansion that will evaluate
measures of safety and efficacy in cancer types identified based on
the responses seen in the dose-escalation phase. Portola has
engaged the Sarah Cannon Research Institute, a global strategic
research organization based in Nashville, Tenn., to conduct the
study.
About PRT2070
PRT2070 is an oral, potent inhibitor of both the B-cell receptor
(BCR) pathway (via Syk) and important cytokine signaling (via JAK)
that promote survival and proliferation of hematologic cancers,
such as chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma
(NHL), and are also implicated in autoimmune diseases. PRT2070 is
unique in that it simultaneously targets these two validated
pathways in one therapy, which may particularly distinguish it in
difficult-to-treat patient populations with certain genetic
subtypes, such as diffuse large B-cell lymphoma (including patients
with identified mutations) and in patients who have failed therapy
due to relapse or acquired mutations.
Portola is currently evaluating PRT2070 in a Phase 1/2 clinical
study and has worldwide rights to develop and commercialize the
agent for hematologic cancers and other systemic indications.
Rights for certain nonsystemic indications are shared 50/50 with
Aciex Therapeutics.
About Portola Pharmaceuticals, Inc.
Portola Pharmaceuticals is a biopharmaceutical company focused
on the development and commercialization of novel therapeutics in
the areas of thrombosis, other hematologic disorders and
inflammation. Portola's wholly-owned lead compound, betrixaban, is
a novel, oral, once-daily Factor Xa inhibitor in Phase 3
development for extended-duration prophylaxis of a form of
thrombosis known as venous thromboembolism (VTE) in acute medically
ill patients. Currently, there is no anticoagulant approved for
extended-duration VTE prophylaxis in this population. Portola's
second lead development candidate, andexanet alfa (PRT4445*), has
the potential to be the first universal antidote to reverse the
effects of Factor Xa inhibitors in patients who suffer an
uncontrolled bleeding episode or trauma, or who require emergency
surgery. Portola retains full, worldwide commercial rights to
andexanet alfa. Portola's third product candidate, PRT2070, is an
orally available kinase inhibitor that uniquely inhibits two
validated tumor proliferation pathways -- spleen tyrosine kinase
(Syk) and janus kinase (JAK). It is currently being studied in
patients with genetically-defined hematologic cancers, as well as
for 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.
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
the use of PRT2070 as a kinase inhibitor and any of Portola's
clinical trials, including Portola's Phase 1/2 proof-of-concept
study for PRT2070. 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 most
recent filings with the Securities and Exchange Commission. 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.
*PRT4445 has a proposed International Nonproprietary Name (pINN)
of andexanet alfa.
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)
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