Portola Pharmaceuticals Presents Interim Phase 2a Safety and Efficacy Data for Cerdulatinib at the International Congress of ...
15 Juin 2017 - 2:30PM
Portola Pharmaceuticals Inc.® (Nasdaq:PTLA) today announced the
presentation of interim data from a Phase 2a study evaluating
cerdulatinib in patients with relapsed/refractory B-cell
malignancies. Cerdulatinib is an investigational oral, dual SYK/JAK
kinase inhibitor for the treatment of relapsed/refractory B-cell
and other hematological malignancies, specifically in patients who
have not responded to prior therapies. The data were presented
by Paul Hamlin, M.D., chief of the Medical Oncology Service at
Memorial Sloan Kettering Basking Ridge during an oral presentation
given earlier today at the International Congress on Malignant
Lymphoma (ICML) in Lugano, Switzerland.
The interim results presented at ICML demonstrated
evidence of clinical activity in patients with relapsed/refractory
(r/r) B-cell malignancies. To date, overall response rates are as
follows:
- 12 out of 18 (67%) partial responses (PRs) in patients with r/r
CLL/SLL
- 5 out of 9 (56%) PRs in patients with r/r FL
- 1 out of 7 (14%) PRs in patients with other r/r iNHL (marginal
zone lymphoma and Waldenstrom macroglobulinemia)
- A complete response (CR) was seen in the first r/r peripheral T
cell lymphoma (PTCL) patient evaluated in the study
Results also showed that cerdulatinib was generally
well-tolerated in these heavily pre-treated patients (at target
drug levels). However, three patients at 35 mg BID achieved higher
than expected drug concentrations and had severe adverse events
(SAEs) including two grade 5 infections and one case of grade three
pancreatitis. The dose was subsequently reduced to 30 mg BID and a
PK monitoring strategy was implemented. This has resulted in an
improved PK and safety profile without compromising clinical
activity.
“While we continue to focus our efforts on gaining regulatory
approval of our two lead programs, betrixaban and AndexXa®
(andexanet alfa), we are pleased that our third molecule,
cerdulatinib, continues to show promising results in hematologic
cancers — an area of great unmet need,” said John Curnutte, M.D.,
Ph.D., executive vice president, research and development of
Portola. “We believe these results of our Phase 2a trial further
validate cerdulatinib’s potential to control relapsed/refractory
B-cell malignancies by a differentiated mechanism of action,
inhibiting two key cell signaling pathways that promote
cancer. We anticipate an update on the ongoing study and a decision
regarding future development by the end of 2017.”
“Patients with relapsed/refractory CLL and NHL are difficult to
treat and have limited options after failing standard therapy,”
said Dr. Hamlin. “Presently, there are few effective treatments for
patients who have failed prior therapies. The interim results of
this clinical trial are very encouraging, and cerdulatinib could
represent an important treatment option for these patients if
confirmed in further trials.”
Additional detail on the interim Phase 2a data will be provided
at the upcoming European Hematology Association (EHA) 22nd Annual
Congress from June 22-25, 2017.
About the Phase 1/2a StudyThe open-label,
multicenter, Phase 1/2a proof-of-concept study assessed the safety,
pharmacokinetics, pharmacodynamics and clinical activity of oral
cerdulatinib in patients with CLL and NHL. In the multi-dose,
dose-escalation Phase 1 part of the study, cerdulatinib was
administered orally once or twice daily in sequential dose cohorts
at increasing dose levels until the maximum tolerated dose was
identified. The clinical expansion cohorts in the Phase 2a part of
the study are evaluating the safety and efficacy of cerdulatinib in
cancer types identified based on results from the dose-escalation
phase of the study. Up to 40 patients each will be enrolled in the
clinical expansion cohorts including patients with
relapsed/refractory chronic lymphocytic leukemia (CLL)/ small
lymphocytic leukemia (SLL), and indolent lymphomas such as
follicular lymphoma (FL) and peripheral T cell lymphoma (PTCL).
About CerdulatinibCerdulatinib is an oral, dual
Syk-JAK inhibitor with a unique mechanism of action. It 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 may have 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
three programs, including betrixaban, an oral, once-daily Factor Xa
inhibitor; AndexXa (andexanet alfa), a recombinant protein designed
to reverse the anticoagulant effect in patients treated with an
oral or injectable Factor Xa inhibitor; and cerdulatinib, a SYK/JAK
inhibitor in development to treat hematologic
cancers. Portola's partnered program is focused on
developing selective SYK inhibitors for inflammatory conditions.
For more information, visit www.portola.com and follow
the Company on Twitter @Portola_Pharma.
Forward-looking StatementsStatements 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, cerdulatinib’s potential as a treatment for hematologic
cancers and the potential of Portola’s other product candidates.
Risks that contribute to the uncertain nature of the
forward-looking statements include: failure to
obtain FDA and/or EMA approval for one or more of our
product candidates, regulatory developments in the United
States and foreign countries; our expectation that we will
incur losses for the foreseeable future and will need additional
funds to finance our operations; the accuracy of our estimates
regarding our ability to initiate and/or complete our clinical
trials and the timing and expense of these trials; the results of
our clinical trials related to the efficacy and safety of our
product candidates; our potential inability to manufacture our
product candidates on a commercial scale in a timely or
cost-efficient manner; the accuracy of our estimates regarding
expenses and capital requirements; our ability to successfully
build a hospital-based sales force and commercial infrastructure;
our ability to obtain and maintain intellectual property protection
for our product candidates; and our ability to retain key
scientific or management personnel. 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.
Investors:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
650.246.7431
Media:
Julie Normart
Pure Communications
jnormart@purecommunications.com
415.946.1087
Portola Pharmaceuticals (NASDAQ:PTLA)
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