SOUTH SAN FRANCISCO, Calif.,
Dec. 9, 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 relapsed/refractory follicular lymphoma
(FL) receiving cerdulatinib alone or in combination with rituximab.
The data will be presented today during a poster session at the
61st American Society of Hematology (ASH) Annual Meeting
in Orlando (December 7-10).
Data included safety and efficacy findings as of November 2019 for 42 patients who received single
agent cerdulatinib at 30 mg twice daily (with the exception of two
patients who initiated treatment at 35 mg) and 21 patients who
received cerdulatinib at 30 mg twice daily in combination with a
standard dosing regimen of rituximab. The number of prior treatment
regimens including anti-CD20 antibody, bendamustine and other
alkylating agents, and PI3K inhibitors ranged from one to 10, with
a median of three.
Among the 42 patients in the cerdulatinib-only cohort, the
overall response rate (ORR) was 48%; 7 patients (17%) achieved a
complete response (CR), 13 patients (31%) achieved a partial
response (PR) and 10 patients (24%) achieved stable disease (SD).
To date, 16 of the 42 patients (38%) in the cerdulatinib-only
cohort have been on study drug for at least 10 months.
Among the 21 patients evaluated for efficacy in the cerdulatinib
and rituximab combination cohort, the ORR was 76%; 5 patients (24%)
achieved a CR, 11 patients (52%) achieved a PR and 5 patients (24%)
achieved SD. Of the 11 patients in this combination cohort who have
been on one to three prior therapies, the ORR was 91% with a
complete response rate of 36%.
Cerdulatinib was generally well-tolerated and the safety profile
appeared similar in both the cerdulatinib-only and rituximab
combination cohorts. The most common adverse events (AEs) occurring
in ≥5% of all evaluable study patients were lipase increase (27%),
neutropenia (18%), diarrhea (13%) and amylase increase (9%). The
most common AEs in the combination cohort included lipase increase
(32%), neutropenia (22%) and diarrhea (14%). The lipase and amylase
changes were generally asymptomatic and not associated with
pancreatitis. Additionally, there was no emergence of late-stage
colitis, cardiac or liver abnormalities, or other evidence of
cumulative toxicity.
"These interim results demonstrate that cerdulatinib provides
sustained clinical activity and good tolerability in patients with
relapsed/refractory follicular lymphoma, and that the tumor
response to both monotherapy and combination therapy appears to
deepen over time," said Paul Hamlin,
M.D., medical director for the David H. Koch Center for Cancer Care
at Memorial Sloan Kettering Cancer Center. "It is encouraging that
the combination of cerdulatinib and rituximab achieved an improved
objective response rate (ORR) in heavily pre-treated patients
compared to prior interim data, indicating it may have potential as
a second-line therapy. We look forward to continuing the study and
exploring an optimized dose of cerdulatinib and rituximab in this
setting."
"Cerdulatinib is the most advanced SYK/JAK inhibitor of its kind
in development for oncology, and has demonstrated its potential to
inhibit two key survival pathways in Non-Hodgkin Lymphoma," said
Jeff Myers, Portola's interim chief medical officer. "The
data presented at ASH continues to demonstrate its safety and
efficacy across a range of malignancies. Specific to follicular
lymphoma, we are excited that the updated data showed an improved
ORR with greater anti-tumor activity in combination with rituximab,
and we look forward to exploring lower doses for potential use in
the second-line setting."
ASH Poster Session Details – Monday,
December 9, 2019, at 6:00 p.m. EST
Title:
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Rapid and Durable
Responses with the SYK/JAK Inhibitor Cerdulatinib in a Phase 2
Study in Relapsed/Refractory Follicular Lymphoma—Alone or in
Combination with Rituximab
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Session:
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623. Mantle Cell,
Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies:
Poster III
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Presenter:
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Paul A. Hamlin, M.D.,
David H. Koch Center for Cancer Care at Memorial Sloan Kettering
Cancer Center
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Location:
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Hall B, Level 2
(Orange County Convention Center)
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About the Phase 2a Study
The Phase 2a, open-label
study was designed to assess the safety and efficacy of
cerdulatinib in patients with relapsed/refractory FL (alone or in
combination with rituximab), small lymphocytic lymphoma (SLL) and
specific subtypes of T-cell Non-Hodgkin Lymphoma, including PTCL,
AITL and CTCL.
Tumor response in the two cohorts evaluating patients with
relapsed/refractory FL was assessed by Lugano classification, with
treatment continued until disease progression or unacceptable
toxicity. Tumor response assessments were performed at the end of
cycle two and every three cycles thereafter.
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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