SOUTH SAN FRANCISCO, Calif.,
Dec. 8, 2019 /PRNewswire/
-- Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA)
today announced new interim results from the Company's ongoing
Phase 2a study of cerdulatinib, an investigational, oral SYK/JAK
inhibitor, in patients with specific subtypes of T-cell Non-Hodgkin
Lymphoma, including relapsed/refractory peripheral T-cell lymphoma
(PTCL); angioimmunoblastic T-cell lymphoma (AITL), a subset of
PTCL; and cutaneous T-cell lymphoma (CTCL). The data will be
presented today during an oral session at the 61st
American Society of Hematology (ASH) Annual Meeting in Orlando (December
7-10).
As of the November 12, 2019
cut-off date, 64 PTCL patients and 40 CTCL patients treated with
cerdulatinib as a single agent were evaluable for response.
The overall response rate (ORR) was 34% in the PTCL cohort and 43%
in the CTCL cohort. Among the subset of patients in the PTCL cohort
with AITL, the ORR was 52% and the complete response rate (CR) was
37%.
"Relapsed or refractory PTCL and CTCL remain challenging
diseases to treat given their heterogenous nature and lack of
uniformly effective therapies," said Steven
M. Horwitz, M.D., an oncologist at Memorial Sloan Kettering
Cancer Center. "I am encouraged to see these updated results, which
underscore the potential of cerdulatinib to address a range of
rare, aggressive sub-types of T-cell malignancies as well as
benefits in terms of quality-of-life measures, such as pruritus,
which can be a significant burden for patients with cutaneous
lymphomas."
Among the 64 patients in the PTCL cohort, 14 patients (22%)
achieved a CR and eight patients (12%) achieved a partial response
(PR). In the subgroup of 27 patients with AITL, 10 patients (37%)
achieved a CR, and four patients (15%) achieved a PR. The median
duration of response is eight months for all PTCL patients and is
greater than nine months in AITL patients. Follow-up is
ongoing.
Among the 40 patients in the CTCL cohort, three patients (8%)
achieved a CR and 14 patients (35%) achieved a PR. Importantly,
rapid improvements in pruritus, or severe itching – a common and
often serious condition associated with CTCL – have been observed,
as measured by the Likert scale.
Cerdulatinib demonstrated good tolerability in both PTCL and
CTCL. The most common Grade 3 or greater adverse events across the
PTCL and CTCL cohorts with a frequency > 5% were lipase increase
(25%), amylase increase (19%), neutropenia (12%), anemia (10%),
diarrhea (8%), sepsis/bacteremia (6%), and febrile neutropenia
(4%). The lipase and amylase changes were generally asymptomatic
and not associated with pancreatitis.
"We are encouraged by the continued safety and efficacy profile
of cerdulatinib in patients with PTCL and CTCL, and the potential
it has to meet medical needs through dual SYK and JAK pathway
inhibition as the most advanced therapy of its kind currently in
development for oncology," said Jeff
Myers, Portola's interim
chief medical officer. "We look forward to the planned initiation
of our registrational trial in patients with PTCL in the coming
months, and to gathering additional evidence on the clinical
activity of cerdulatinib."
ASH Oral Session Details – Sunday,
December 8, 2019, at 12:45 p.m. EST
Title:
|
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A Phase 2 Study of
the Dual SYK/JAK Inhibitor Cerdulatinib Demonstrates Good
Tolerability and Clinical Response in Relapsed/Refractory
Peripheral T-Cell Lymphoma and Cutaneous T-Cell
Lymphoma
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Session:
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624. Hodgkin Lymphoma
and T/NK Cell Lymphoma—Clinical Studies: Novel Therapies in
Peripheral T-cell Lymphomas
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Presenter:
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Steven M. Horwitz,
M.D., Memorial Sloan Kettering Cancer Center
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Location:
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Valencia D (W415D),
Level 4 (Orange County Convention Center)
|
About the Phase 2a Study
The Phase 2a, open-label study was designed to assess the safety
and efficacy of cerdulatinib in patients with specific subtypes of
relapsed/refractory T-cell Non-Hodgkin Lymphoma – including PTCL
and CTCL, B-cell Non-Hodgkin lymphoma alone or in combination with
rituximab, chronic lymphocytic leukemia, and small lymphocytic
lymphoma.
About Cerdulatinib
Cerdulatinib is an investigational oral, dual spleen tyrosine
kinase (SYK) and janus kinase (JAK) inhibitor that uniquely
inhibits two key cell signaling pathways implicated in certain
hematologic malignancies and autoimmune diseases. There is a strong
rationale for inhibiting both SYK (B-cell receptor pathway) and JAK
(cytokine receptors) in B-cell malignancies where both targets have
been shown to promote cancer cell growth and survival.
The U.S. Food and Drug Administration granted cerdulatinib
Orphan Drug Designation for the treatment of PTCL in September 2018.
About Portola Pharmaceuticals, Inc.
Portola
Pharmaceuticals is a global, commercial-stage
biopharmaceutical company focused on the discovery, development and
commercialization of novel therapeutics that could significantly
advance the fields of thrombosis and other hematologic conditions.
The Company's first two commercialized products are
Andexxa® [coagulation factor Xa (recombinant),
inactivated-zhzo], marketed in Europe as
Ondexxya® (andexanet alfa), and
Bevyxxa® (betrixaban). The company also is
advancing cerdulatinib, a SYK/JAK inhibitor being developed for the
treatment of hematologic cancers. Founded in 2003
in South San Francisco, California, Portola has
operations in the United States and Europe.
Forward-Looking Statements
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 anticipated development plan for
cerdulatinib. Risks that contribute to the uncertain nature of the
forward-looking statements include: the risk of unfavorable results
from additional clinical trials involving cerdulatinib; our ability
to successfully execute on our development strategy; the risk of
unfavorable regulatory developments; our expectation that we will
incur losses for the foreseeable future and will need additional
funds to finance our operations; the effects of competition; the
accuracy of our estimates regarding expenses and capital
requirements; our ability to successfully market Andexxa; our
ability to obtain and maintain intellectual property protection for
our product candidates; our ability to retain key scientific or
management personnel; and general market conditions. 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. 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.
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