SEATTLE, Jan. 9, 2017 /PRNewswire/ -- CTI BioPharma
Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) today announced
positive progress on its lead programs in addition to key business
priorities for 2017.
"Throughout 2016 we maintained our commitment to bringing new
therapies to patients with unmet medical needs, and were successful
in working with the FDA to remove the full clinical hold on
pacritinib and get it back on the development track for the benefit
of myelofibrosis patients," said Richard
Love, Interim President and Chief Executive Officer of CTI
BioPharma. "The PERSIST-2 clinical trial of pacritinib was
highlighted as one of six late-breaking data presentations at the
American Society of Hematology conference in December. We
believe this oral presentation was well received by the
hematology/oncology community, which recognizes the unmet need for
myelofibrosis patients who are ineligible to receive or are
not benefitting from the approved JAK1/JAK2 inhibitor, ruxolitinib.
Additionally, the PIX306 confirmatory trial of our commercial
product PIXUVRI(R)(pixantrone) continues to progress
toward an announcement of top-line results later this
year. If positive, this trial could provide the opportunity
for full approval and label expansion by EMA, and discussions with
the FDA about accelerated PIXUVRI approval in the US for the
treatment of patients with relapsed or refractory aggressive B-cell
non-Hodgkin lymphoma. We have also made significant effort at
reducing our expenses and believe we are well positioned moving
into 2017."
Recent Progress Update
Pacritinib
In January 2017, CTI BioPharma
announced the U.S. Food and Drug Administration (FDA) removed the
full clinical hold on studies being conducted under the
Investigational New Drug (IND) application for pacritinib.
In December 2016, data from the
randomized Phase 3 PERSIST-2 clinical trial comparing pacritinib
with physician-specified best available therapy (BAT), including
ruxolitinib, for treatment of patients with myelofibrosis whose
baseline platelet counts are less than 100,000 per microliter was
one of six late-breaking oral presentations at the American Society
of Hematology Annual Meeting. Patients in the trial were
randomized to receive 200 mg pacritinib twice daily (BID), 400 mg
pacritinib once daily (QD), or BAT. In those patients who had a
chance to reach Week 24 (the primary analysis time point) at the
time the clinical hold was imposed, the trial showed a
statistically significant response rate in spleen volume reduction
(SVR) in patients treated with pacritinib compared to BAT
irrespective of prior treatment with ruxolitinib. The co-primary
endpoint of reduction of Total Symptom Score (TSS) was not achieved
but trended toward improvement in TSS. Although secondary
objectives could not be evaluated formally due to the study not
achieving one of the primary objectives, when the two pacritinib
dosing arms were evaluated separately versus BAT, pacritinib BID
showed a higher percent of SVR and TSS responses compared to BAT;
whereas, pacritinib given QD showed only a higher percent SVR
responses compared to BAT. There was no significant difference in
overall survival (OS) across treatment arms, censored at the time
of clinical hold. The most common treatment-emergent adverse
events (AEs), occurring in 20 percent or more of patients treated
with pacritinib within 24 weeks, of any grade, were
gastrointestinal (generally manageable diarrhea, nausea and
vomiting) and hematologic (anemia and thrombocytopenia) and were
generally less frequent for BID versus QD administration. The
most common serious treatment-emergent AEs (incidence of ≥5 percent
reported in any treatment arm irrespective of grade) were anemia,
thrombocytopenia, pneumonia and acute renal failure none of which
exceeded 8 percent individually in any arm. The presentation was
also selected to be part of the "2017 Highlights of ASH" program
designed to review significant scientific updates presented at ASH
with hematologists/oncologists at five locations across the
U.S.
PIXUVRI®
In January 2017, CTI BioPharma
received a €7.5 million milestone payment from its partner Servier
following achievement of a milestone associated with patient
enrollment in the Phase 3 PIX306 clinical trial of PIXUVRI. The
trial is a post-authorization trial as part of the conditional
marketing authorization of PIXUVRI in the European Union (E.U.) The
PIX306 is comparing PIXUVRI and rituximab with gemcitabine and
rituximab in the setting of aggressive B-cell non-Hodgkin lymphoma
(NHL). The trial continues to enroll patients.
2017 Key Objectives
Advance Marketing Authorization Application in E.U. and
define regulatory pathway in U.S. for pacritinib. CTI BioPharma
continues to have dialogue with the European Medicines Authority
(EMA) on the Marketing Authorization Application (MAA) for
pacritinib that had been previously filed by its former partner,
Baxalta. At the time of the filing only data from the first
Phase 3 clinical trial of pacritinib, PERSIST-1, was available.
With the availability of results from the PERSIST-2 clinical
trial and the recent completion of the PERSIST-2 clinical study
report, CTI BioPharma believes that the best strategy currently to
achieve marketing authorization is to utilize the combined clinical
evidence from both Phase 3 trials. Accordingly, CTI BioPharma
is evaluating whether to update the current application with the
additional data from PERSIST-2 or to resubmit the MAA. Under either
plan, CTI BioPharma would expect to pursue marketing authorization
for the treatment of patients with myelofibrosis who are ineligible
to receive, intolerant of or have insufficient response to the
approved JAK1/JAK2 inhibitor, ruxolitinib.
CTI BioPharma also intends to discuss with the FDA the future
development of pacritinib.
Initiate PAC203 trial. CTI BioPharma expects to initiate
the PAC203 trial in the second quarter of 2017. The trial plans to
enroll up to approximately 105 patients with primary myelofibrosis
who have failed prior ruxolitinib therapy to evaluate the safety
and the dose response relationship for efficacy (spleen volume
reduction at 24 weeks) of three dose regimens: 100 mg once-daily,
100 mg twice-daily (BID) and 200 mg BID.
Secure ex-U.S. partner for pacritinib. CTI BioPharma
intends to secure a partnership for the development and
commercialization of pacritinib in certain territories outside the
U.S.
Release top-line results of PIX306. CTI BioPharma expects
to complete enrollment in the ongoing PIX306 trial of PIXUVRI and
release top-line results by the end of 2017.
Financial
CTI BioPharma's preliminary, unaudited estimates of its cash and
cash equivalents balance as of December 31,
2016 is approximately $44.0
million. In January 2017, we
received a €7.5 million milestone payment from Servier. CTI
BioPharma expects that its cash burn (a non-GAAP financial
measure), excluding cash inflows from future business development
activities and proceeds from capital markets financing activities,
would be approximately $65-75 million
for 2017. The Company expects to meet its cash requirements for
2017 with existing cash and by partnering one or more product
assets during the course of the year.
About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with
specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK family of
enzymes is a central component in signal transduction pathways,
which are critical to normal blood cell growth and development, as
well as inflammatory cytokine expression and immune responses.
Mutations in these kinases have been shown to be directly related
to the development of a variety of blood-related cancers, including
myeloproliferative neoplasms, leukemia and lymphoma. In addition to
myelofibrosis, the kinase profile of pacritinib suggests its
potential therapeutic utility in conditions such as acute myeloid
leukemia, or AML, myelodysplastic syndrome, or MDS, chronic
myelomonocytic leukemia, or CMML, and chronic lymphocytic leukemia,
or CLL, due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.
In August 2014, pacritinib was
granted Fast Track designation by the FDA for the treatment of
intermediate and high risk myelofibrosis including, but not limited
to, patients with disease-related thrombocytopenia (low platelet
counts); patients experiencing treatment-emergent thrombocytopenia
on other JAK2 inhibitor therapy; or patients who are intolerant of,
or whose symptoms are not well controlled (sub-optimally managed)
on other JAK2 therapy.
Pacritinib was evaluated in two Phase 3 clinical trials, known
as the PERSIST program, for patients with myelofibrosis, with one
trial in a broad set of patients without limitations on platelet
counts, the PERSIST-1 trial; and the other in patients with low
platelet counts, the PERSIST-2 trial. The PERSIST-1 trial met its
primary endpoint of spleen volume reduction (35 percent or greater
from baseline to Week 24 by MRI/CT scan). The PERSIST-2 trial met
one of its co-primary endpoints, that of spleen volume reduction.
The co-primary endpoint of reduction of Total Symptom Score (TSS)
was not achieved but trended toward improvement in TSS.
Clinical studies under the investigational new drug (IND) for
pacritinib were subject to a full clinical hold issued by the FDA
in February 2016. In January 2017, the FDA removed the full clinical
hold and stated that clinical trials may be resumed.
About PIXUVRI® (pixantrone)
PIXUVRI is a novel aza-anthracenedione with unique structural
and physiochemical properties. In May 2012, the European
Commission granted conditional marketing authorization for
PIXUVRI as a monotherapy for the treatment of adult patients with
multiply relapsed or refractory B-cell aggressive NHL. The
benefit of PIXUVRI treatment has not been established in patients
when used as fifth line or greater chemotherapy in patients who are
refractory to last therapy. The Summary of Product Characteristics
(SmPC) has the full prescribing information, including the safety
and efficacy profile of PIXUVRI in the approved indication.
The SmPC is available at www.pixuvri.eu. PIXUVRI does not
have marketing approval in the United States.
In September 2014, CTI BioPharma
entered into an exclusive license and collaboration agreement, with
Servier with respect to the development and commercialization of
PIXUVRI. Under the agreement, CTI BioPharma retains full
commercialization rights to PIXUVRI in Austria, Denmark, Finland, Germany, Israel, Norway, Sweden, Turkey, the United
Kingdom and the U.S. while Servier has exclusive rights to
commercialize PIXUVRI in all other countries.
About CTI BioPharma
CTI BioPharma Corp. is a biopharmaceutical company focused on
the acquisition, development and commercialization of novel
targeted therapies covering a spectrum of blood-related cancers
that offer a unique benefit to patients and healthcare providers.
CTI BioPharma has a commercial presence in Europe with respect to PIXUVRI® and
a late-stage development pipeline, including pacritinib for the
treatment of patients with myelofibrosis. CTI BioPharma is
headquartered in Seattle,
Washington, with offices in London and Milan under the name CTI Life Sciences
Limited. For additional information and to sign up for email alerts
and get RSS feeds, please visit www.ctibiopharma.com.
Forward-Looking Statements
This press release includes forward-looking statements within
the meaning of the Safe Harbor provisions of the Private Securities
Litigation Reform Act of 1995. Such statements are subject to
a number of risks and uncertainties, the outcome of which could
materially and/or adversely affect actual future results and the
trading price of CTI BioPharma's securities. Such
statements include, but are not limited to, expectations with
respect to our ability to be able to interpret clinical trial data
and results despite not satisfying the pre-specified minimum
evaluable patient goal, expectations with respect to the potential
therapeutic utility of pacritinib, including pacritinib's potential
to achieve treatment goals across patients with myelofibrosis,
regardless of baseline characteristics, and statements
regarding CTI BioPharma's expectations with respect to
the development of CTI BioPharma, its financial
position and its product and product candidate portfolio,
including strategies and plans for achieving marketing
authorization and other approvals, partnerships, and the initiation
or completion of clinical strials. Investors are cautioned not
to place undue reliance on these forward-looking statements, which
speak only as of the date of this release. In particular,
meaningful interpretation of PERSIST-2 may not be possible because
the pre-specified minimum evaluable patient goal was not met. The
statements are based on assumptions about many important factors
and information currently available to us to the extent we have
thus far had an opportunity to fully and carefully evaluate such
information in light of all surrounding facts, circumstances,
recommendations and analyses. Risks that contribute to the
uncertain nature of the forward-looking statements include, among
others: risks associated with the biopharmaceutical industry in
general and with CTI BioPharma and its product and
product candidate portfolio in particular, risks associated with
the pace or geography of enrollment of its clinical trials, risks
associated with the commencement or outcome of preclinical and
clinical studies, that trial results observed to date may differ
from future results or that different conclusions or considerations
may qualify such results once existing data has been more fully
evaluated, that CTI BioPharma may not obtain favorable
determinations by other regulatory, patent and administrative
governmental authorities or will not be in a position to submit
regulatory submissions as or when projected, risks related to the
actions of regulatory bodies and other governmental authorities,
risks related to changes in laws and regulations, product quality,
product efficacy, study protocol, data integrity or patient safety
issues, risks related to the costs of developing, producing and
selling PIXUVRI, pacritinib and CTI BioPharma's other
product candidates, and other risks, including, without limitation,
competitive factors, technological developments, and
that CTI BioPharma may not achieve previously announced
goals and objectives as or when projected as well as other risks
listed or described from time to time in CTI
BioPharma's most recent filings with the SEC on
Forms 10-K, 10-Q and 8-K. Except as required by
law, CTI BioPharma does not intend to update any of the
statements in this press release upon further developments.PIXUVRI
is a registered trademark of CTI BioPharma Corp.
CTI BioPharma Contacts:
Ed Bell
+1 206-272-4345
ebell@ctibiopharma.com
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/cti-biopharma-announces-progress-of-lead-programs-and-strategic-objectives-for-2017-300387308.html
SOURCE CTI BioPharma Corp.