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 with Chroma
Therapeutics Ltd. for drug candidate tosedostat in North,
Central and South 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 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 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