SEATTLE, July 13, 2017 /PRNewswire/ -- CTI BioPharma
Corp. (NASDAQ and MTA: CTIC) today announced that European
Medicines Agency (EMA) has validated the Marketing Authorization
Application (MAA) for pacritinib for the treatment of patients with
myelofibrosis who have thrombocytopenia (platelet counts less than
100,000 per microliter). Validation confirms that the submission is
complete and initiates the centralized review process by the
EMA's Committee for Medicinal Products for Human
Use (CHMP). The CHMP review period is 210 days,
excluding question or opinion response periods, after which the
CHMP opinion is reviewed by the European Commission, which
usually issues a final decision on EU authorization within three
months. If authorized, pacritinib would be granted a marketing
license valid in all 28 EU member states.
"The MAA validation is a significant milestone for CTI BioPharma
as we seek to bring pacritinib to patients with myelofibrosis who
have thrombocytopenia that could benefit from its unique profile,"
said Adam R. Craig, M.D., Ph.D.,
President and CEO of CTI BioPharma. "We look forward to working
with the CHMP/EMA during their review of this application."
The MAA is primarily supported by data from two randomized Phase
3 clinical trials, PERSIST-1 and PERSIST-2, that evaluated
pacritinib in patients with myelofibrosis.
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.
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 resume. CTI BioPharma is initiating a Phase 2
dose exploration study that was a condition of the clinical hold
being removed.
About Myelofibrosis and Myeloproliferative Neoplasms
Myelofibrosis is one of three main types of myeloproliferative
neoplasms (MPN), which are a closely related group of progressive
blood cancers. The three main types of MPNs are primary
myelofibrosis (PMF), polycethemia vera (PV) and essential
thrombocythemia (ET).1
Myelofibrosis is a serious and life-threatening bone marrow
disorder caused by the accumulation of malignant bone marrow cells
that triggers an inflammatory response and scars the bone marrow.
The replacement of bone marrow with scar tissue limits its ability
to produce red blood cells, prompting the spleen and liver to take
over this function. Symptoms that arise from this disease include
enlargement of the spleen, anemia, extreme fatigue and pain.
The estimated prevalence of MPNs suggest there are approximately
300,000 people living with the disease in the U.S., of which
myelofibrosis accounts for approximately 18,000
patients.2 In Europe,
there is a wide variation of prevalence observed across data
sources. Myelofibrosis has a median age of 64 at the time of
diagnosis3 and is a progressive disease with
approximately 20 percent of patients eventually developing acute
myeloid leukemia (AML).4 The median survival for
high-risk myelofibrosis patients is less than 1.5 years, while the
median survival for patients with myelofibrosis overall is
approximately 6 years.4
About CTI BioPharma Corp.
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 late-stage development pipeline, including
pacritinib for the treatment of patients with myelofibrosis. CTI
BioPharma is headquartered in Seattle,
Washington. 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, which
are within the meaning of the Safe Harbor provisions of the Private
Securities Litigation Reform Act of 1995, including statements
regarding expectations with respect to the potential therapeutic
utility of pacritinib, including pacritinib's potential to achieve
treatment goals across patients with myelofibrosis, and
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 for the PERSIST-2
clinical trial and expectations with respect to the potential
therapeutic utility of pacritinib. 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 the issuers' securities, including risks related
to the satisfaction of regulatory and other requirements; the
actions of regulatory bodies and other governmental authorities;
other clinical trial results; changes in laws and regulations;
product quality, product efficacy, study protocol, data integrity
or patient safety issues; product development risks; and other
risks identified in each of the issuer's most recent filings on
Forms 10-K and 10-Q and other Securities and Exchange
Commission filings.
- MPN Research Foundation. Accessed June
2017. Available at www.mpnresearchfoundation.org.
- Based on Mesa R, ASH 2012 poster.
- Cervantes F, et al., New prognostic scoring system for primary
myelofibrosis based on a study of the International Working Group
for Myelofibrosis Research and Treatment. Blood. 2009;
113:2895-2901.
- Vannucchi, A. Management of Myelofibrosis. ASH Education Book.
2011; 1:222-230.
CTI BioPharma Contacts:
Ed Bell
+1 206-272-4345
ebell@ctibiopharma.com
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