CTI Announces United Kingdom National Cancer Research Institute AML Cooperative Group Initiates Randomized Phase 2 Trial Evalua

     CTI Announces United Kingdom National Cancer Research Institute AML
  Cooperative Group Initiates Randomized Phase 2 Trial Evaluating Tosedostat
         plus Cytarabine for Older Patients with AML or High-Risk MDS

PR Newswire

SEATTLE, June 12, 2014

SEATTLE, June 12, 2014 /PRNewswire/ -- CTI BioPharma Corp. (CTI) (NASDAQ and
MTA: CTIC) today announced the initiation of an international cooperative
group Phase 2 clinical trial of tosedostat in combination with low-dose
cytarabine in older patients with Acute Myeloid Leukemia (AML) or high risk
Myelodysplastic Syndrome (MDS). Tosedostat is a first-in-class selective
inhibitor of aminopeptidases, which are required by tumor cells to provide
amino acids necessary for growth and tumor cell survival. The trial is being
conducted by the National Cancer Research Institute Haematological Oncology
Study Group under the sponsorship of Cardiff University. The trial management
group is led 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. Chroma Therapeutics Ltd., from whom CTI
licensed tosedostat, is facilitating drug supply for the trial.

"As the population of patients 65 years of age and older with AML continues to
grow, there are a large number of patients who are not suitable for
conventional intensive chemotherapy and for these patients there is a
significant unmet need for an alternative therapy that is effective and well
tolerated," said Professor Burnett. "New agents that are being developed to
target the underlying biology of the disease, such as tosedostat, may provide
hope for these patients where there has been very little improvement in the
outcome of these patients over the last couple of decades. Tosedostat has
demonstrated encouraging clinical activity in AML with manageable toxicities
that can be administered primarily as out-patient therapy, and we are pleased
to get this study underway."

In this Phase 2/3 trial, referred to as the AML Less Intensive (LI-1) trial,
patients will be randomized to standard treatment, low dose cytarabine, versus
one of five novel investigational treatments, one of which is tosedostat, each
in combination with low dose cytarabine. The trial will utilize a "Pick a
Winner" trial design.^1 Under such a design, the Phase 2 portion of the trial
is expected to initially enroll 50 patients per arm, and, if the complete
response rate of cytarabine plus novel therapy appears satisfactory, an
additional 50 patients per arm would be enrolled in the Phase 2 portion. Based
on an interim analysis of complete response results, the trial management
group may determine to move the applicable trial into the Phase 3 portion,
which would then undertake to enroll 100 additional patients (for a total of
200 patients per arm). Overall survival will serve as the primary endpoint of
the trial.

About Acute Myeloid Leukemia

Approximately 18,860 new cases of AML were expected to be diagnosed in the
United States in 2013.^2 As of January 2008 an estimated 30,993 people were
living with (or were in remission from) AML.^3 While AML may occur at any age,
adults at least 60 years of age are more likely to develop the disease than
younger people.^3 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.^3 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 Tosedostat

Tosedostat is an oral aminopeptidase inhibitor that has demonstrated
anti-tumor responses in blood-related cancers and solid tumors in Phase 1–2
clinical trials. CTI has an exclusive marketing and co-development agreement
withChroma Therapeutics Ltd.for drug candidate tosedostat in North, Central
andSouth America.

About CTI BioPharma

CTI BioPharma Corp. (NASDAQ and MTA: CTIC) 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. The Company has a
commercial presence in Europe and a late-stage development pipeline, including
pacritinib, CTI's lead product candidate that is currently being studied in a
Phase 3 program 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'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, the
expected efficacy and potential benefits of tosedostat and the expected
attributes (including the size) of the AML Less Intensive (LI-1) trial. 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 AML Less Intensive (LI-1) trial
will not occur as planned, that CTI 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,
that CTI may experience delays in the commencement of preclinical and clinical
studies, risks related to the cost of developing tosedostat 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 theSecurities 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 future developments.

References:

1. Hills RK & Burnett AK. Applicability of a "Pick a Winner" trial design to
acute myeloid leukemia. Blood. Sept 2011. Vol 118:2389-2394.
2. American Cancer Society, Cancer Facts & Figures 2014. Available at
http://tinyurl.com/m3nxmhl. Accessed June 2014.
3. The Leukemia and Lymphoma Society, Acute Myeloid Leukemia, Rev. 2011.
http://tinyurl.com/d72ycja. Accessed June 2014.

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

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

In Europe:

CTI Life Sciences Limited, Milan Branch
Laura Villa
+39 02 94751572
lvilla@cti-lifesciences.com
CTI_EUInvestors@CTI-Lifesciences.com

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SOURCE CTI BioPharma

Website: http://www.ctibiopharma.com
 
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