Updated Interim Results from Ongoing Phase 2a Study of Portola Pharmaceuticals’ Oral Syk/JAK Inhibitor Cerdulatinib Contin...
04 Décembre 2018 - 3:15AM
– Early Signs of Durable Response Across Patients
with Relapsed/Refractory PTCL –
Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced
updated 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 60th American Society of Hematology
(ASH) Annual Meeting in San Diego (December 1-4).
As of the November 1, 2018 cut-off date, 41 PTCL
patients and 27 CTCL patients were evaluable for response. The
objective response rate (ORR) was 34 percent in the PTCL cohort and
26 percent in the CTCL cohort. Among the subset of patients in the
PTCL cohort with AITL, the ORR was 57 percent.
PTCL Cohort Data
- Eleven of 41 patients (27 percent) achieved a complete response
(CR), and three patients (7 percent) achieved a partial response
(PR).
- In the subgroup of 14 patients with AITL, seven patients (50
percent) achieved a CR and one patient (7 percent) achieved a
PR.
- One patient who achieved a CR went on to transplant.
- Eight responding patients have remained on drug for three to
more than 12 months and five patients have had a duration of
response of six months or greater. Follow-up is ongoing.
CTCL Cohort Data
- In the CTCL cohort, two patients (7 percent) achieved a CR and
five patients (19 percent) achieved a PR. Responses have been seen
in patients with Mycosis Fungoides and Sezary Syndrome.
- Eleven of 23 patients (48 percent) achieved a ≥ 50 percent
reduction in skin lesions, based on the Modified Severity Weighted
Assessment Tool (mSWAT).
- 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.
- The CTCL cohort continues to enroll and data analysis is
ongoing.
Cerdulatinib has demonstrated 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
percent were lipase increase (23 percent), amylase increase (18
percent), sepsis/bacteremia (8 percent), and neutropenia,
pneumonia/lung infection and diarrhea (7 percent each). These
events are manageable and further accrual is ongoing.
“The ongoing Phase 2a cerdulatinib data continue
to demonstrate meaningful clinical activity, with particularly
compelling complete response rates among AITL patients and the
potential for durable response among patients with PTCL and CTCL,”
said Steven M. Horwitz, M.D., Memorial Sloan Kettering Center, and
study investigator. “Like many others with T-cell malignancies, the
patients in this study have failed prior therapies and are in need
of additional treatment options. It is encouraging to see such a
strong early signal in both disease response and quality-of-life
measures, such as itching.”
“While still early, the continuing and durable
response rates in PTCL and the encouraging responses in CTCL
underscore the potential of cerdulatinib to control
relapsed/refractory T-cell malignancies,” said John Curnutte, M.D.,
Ph.D., executive vice president and head of research and
development at Portola. “We will apply our learnings from this
study to inform the design of a pivotal trial, and look forward to
continuing discussions with the U.S. Food and Drug Administration
about the potential for an accelerated approval pathway for
cerdulatinib in PTCL.”
ASH Oral Session Details – Monday,
December 3, 2018 at 7:15 p.m. PST
Title:
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1001. The Novel Syk/JAK
Inhibitor Cerdulatinib Demonstrates Good Tolerability |
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and Clinical Response
in a Phase 2a Study in Relapsed/Refractory Peripheral |
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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: T Cell |
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Lymphoma: Chemotherapy
and Targeted Approaches) |
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Presenter: |
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Steven M. Horwitz,
M.D., Memorial Sloan-Kettering Cancer Center |
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Session
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6:15 – 7:45 p.m. PST;
Room 6F |
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About CerdulatinibCerdulatinib
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. In addition, pre-clinical data
suggest an important role for Syk and JAK in PTCL tumor
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 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 diseases.
The Company’s two FDA-approved medicines are Andexxa® [coagulation
factor Xa (recombinant), inactivated-zhzo], the first and only
antidote for patients treated with rivaroxaban and apixaban when
reversal of anticoagulation is needed due to life-threatening or
uncontrolled bleeding, and Bevyxxa® (betrixaban), the first and
only oral, once-daily Factor Xa inhibitor for the prevention of VTE
in adult patients hospitalized for an acute medical illness. The
company also is advancing cerdulatinib, a Syk/JAK inhibitor for the
treatment of hematologic cancers.
Forward-Looking StatementsThis
announcement contains forward-looking statements, including
statements regarding next steps for the development of
cerdulatinib, including the potential for an accelerated approval
pathway in the U.S. for certain tumor subtypes. Risks that
contribute to the uncertain nature of the forward-looking
statements include: failure to obtain FDA agreement to an
accelerated or other regulatory approval pathway, regulatory
developments in the U.S. 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.
Investor
Contact: |
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Media
Contact: |
Cara Miller |
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Patrick Ryan |
Portola
Pharmaceuticals |
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Pure
Communications |
IR@portola.com |
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pryan@purecommunications.com |
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