CTI Announces United Kingdom National Cancer Research Institute AML Cooperative Group Phase 2 Trial Evaluating Pacritinib for

     CTI Announces United Kingdom National Cancer Research Institute AML
   Cooperative Group Phase 2 Trial Evaluating Pacritinib for Patients with
                Relapsed FLT3 Positive Acute Myeloid Leukemia

- Trial Sponsored by Cardiff University and Supported by Cancer Research UK -

PR Newswire

SEATTLE, Jan. 30, 2014

SEATTLE, Jan.30, 2014 /PRNewswire/ -- Cell Therapeutics, Inc. (CTI) (NASDAQ
and MTA: CTIC) today announced the initiation of an international cooperative
group Phase 2 clinical trial of pacritinib in adult patients with relapsed
acute myeloid leukemia (AML) with mutations of the FLT3 gene. Mutation of the
FLT3 gene is found in approximately one-third of AML patients and is an
independent risk factor for poor prognosis. Pacritinib is an oral JAK2/FLT3
inhibitor that has demonstrated encouraging activity in preclinical models of
AML with mutated FLT3 gene, including additional FLT3 mutations that confer
resistance to other targeted FLT3 agents. The trial is being conducted by the
AML Working Group of the National Cancer Research Institute Haematological
Oncology Study Group in Acute Myeloid Leukemia (AML) and high risk
Myelodysplastic Syndrome (MDS) under the sponsorship of Cardiff University and
supported by Cancer Research UK. The trial management group is lead by
Professor Alan K. Burnett, Head of Haematology in the Department of Medical
Genetics, Haematology and Pathology at the School of Medicine at Cardiff
University.

"Mutation of the FLT3 gene in AML is associated with a high relapse rate and a
poor prognosis with standard therapy; therefore, novel agents capable of
inhibiting the activation of this gene are of great interest in the AML
field," said Professor Burnett. "Pacritinib is a specific, potent inhibitor of
the common FLT3 mutation and certain other generally drug resistant mutations.
Because pacritinib also inhibits JAK2, which is independently associated with
resistance to FLT3 inhibition and a poor prognosis in AML, this is an
attractive agent to test in patients with relapsed FLT3 mutated AML who have
limited options for beneficial therapy."

This Phase 2 trial is part of a larger ongoing study in AML, referred to as
the AML17 trial, which includes multiple arms evaluating first line regimens
for AML. Patients with the FLT3 mutation, who are enrolled in this study and
relapse following standard therapy, will be offered therapy with pacritinib.
Approximately 80 patients at sites in England and Wales will be enrolled and,
if an encouraging response rate is observed, a pacritinib arm may be adopted
in the first line therapy study.

About Acute Myeloid Leukemia

Although AML can occur at any age, adults aged 60 years and older are more
likely to develop the disease than younger people. AML is a cancer
characterized by the rapid growth of abnormal white blood cells that
accumulate in the bone marrow and interfere with the production of normal
blood cells. AML may develop from the progression of other diseases, such as
MDS, a blood cancer that also affects the bone marrow leading to a decrease in
circulating red blood cells. AML is the most common acute leukemia affecting
adults, and its incidence increases with age. Internal tandem duplication
mutations of the FLT3 gene are found in approximately one-third of those
diagnosed with AML.^1 The mutation is associated with an inferior outcome
attributed to a higher relapse rate. The symptoms of AML are caused by the
replacement of normal bone marrow with leukemic cells, which causes a drop in
red blood cells, platelets and normal white blood cells, leading to infections
and bleeding. AML progresses rapidly and is typically fatal within weeks or
months if left untreated. Although a substantial proportion of younger
individuals who develop AML can be cured, AML in the elderly typically
responds poorly to standard therapy with few complete remissions.

About Pacritinib

Pacritinib is an oral tyrosine kinase inhibitor with dual activity against
JAK2 and FLT3. 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. Pacritinib may offer an
advantage over other JAK inhibitors through effective treatment of symptoms
while having less treatment-emergent thrombocytopenia and anemia than has been
seen in currently approved and in-development JAK inhibitors.

In November 2013, CTI and Baxter International entered into a worldwide
license agreement to develop and commercialize pacritinib in which CTI and
Baxter will jointly commercialize pacritinib in the United States and Baxter
has exclusive commercialization rights for all indications outside the United
States.

As part of the new collaboration with Baxter, CTI is pursuing a broad approach
to advancing pacritinib for patients with myelofibrosis by conducting two
Phase 3 clinical trials: one in a broad set of patients without limitations on
blood platelet counts, the PERSIST-1 trial; and the other will be in patients
with low platelet counts, the PERSIST-2 trial, which is expected to begin in
early 2014. In October 2013, CTI reached agreement with the U.S. Food and Drug
Administration on a Special Protocol Assessment, or SPA, for the PERSIST-2
pivotal trial. A SPA is a written agreement between CTI and the FDA regarding
the design, endpoints and planned statistical analysis approach of the trial
to be used in support of a potential New Drug Application, or NDA, submission.


About Cell Therapeutics, Inc.

CTI (NASDAQ and MTA: CTIC) is a biopharmaceutical company committed to the
development and commercialization of an integrated portfolio of oncology
products aimed at making cancer more treatable. CTI is headquartered in
Seattle, WA. For additional information and to sign up for email alerts and
get RSS feeds, please visit www.CellTherapeutics.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's securities. Such statements include,
but are not limited to, statements regarding CTI's expectations with respect
to the development of CTI and its product and product candidate portfolio, any
current or potential collaborations or partnerships, the prevalence of people
with mutations of the FLT3 gene, the expected commencement of the PERSIST-2
clinical trial in early 2014 and the expected efficacy and potential benefits
of pacritinib (including that pacritinib may offer an advantage over other JAK
inhibitors through effective treatment of symptoms while having less
treatment-emergent thrombocytopenia and anemia than has been seen in currently
approved and in-development JAK inhibitors). 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
and its product and product candidate portfolio in particular including, among
others, risks associated with the following: that CTI cannot predict or
guarantee the pace or geography of enrollment of its clinical trials, that CTI
cannot predict or guarantee the outcome of preclinical and clinical studies,
that the second Phase 3 clinical trial of pacritinib will not occur as
planned, that CTI may not obtain favorable determinations by other regulatory,
patent and administrative governmental authorities, that CTI may experience
delays in the commencement of preclinical and clinical studies, risks related
to the costs of developing pacritinib and CTI's other product candidates, and
other risks, including, without limitation, competitive factors, technological
developments, that CTI's operating expenses continue to exceed its net
revenues, that CTI may not be able to sustain its current cost controls or
further reduce its operating expenses, that CTI may not achieve previously
announced goals and objectives as or when projected, that CTI's average net
operating burn rate may increase, that CTI will continue to need to raise
capital to fund its operating expenses, but may not be able to raise
sufficient amounts to fund its continued operation as well as other risks
listed or described from time to time in CTI's most recent filings with the
Securities and Exchange Commission on Forms 10-K, 10-Q and 8-K. Except as
required by law, CTI does not intend to update any of the statements in this
press release upon further developments.

References:

1. Brunet S (2012) Impact of FLT3 Internal Tandem Duplication on the Outcome
of Related and Unrelated Hematopoietic Transplantation for Adult Acute Myeloid
Leukemia in First Remission: A Retrospective Analysis. J Clin Oncol
30(7):735-41.

Contacts:
Monique Greer
+1 206-272-4343
mgreer@ctiseattle.com

Ed Bell
+1 206-282-7100
ebell@ctiseattle.com

In Europe:

CTI Life Sciences Limited, Milan Branch
Laura Villa
+39 02 89659706
lvilla@cti-lifesciences.com
CTI_EUInvestors@CTI-Lifesciences.com

SOURCE Cell Therapeutics, Inc.

Website: http://www.celltherapeutics.com
 
Press spacebar to pause and continue. Press esc to stop.