SEATTLE, Oct. 13, 2020 /PRNewswire/ -- CTI BioPharma
Corp. (Nasdaq: CTIC) today announced that it has commenced a
rolling New Drug Application (NDA) submission to the U.S. Food and
Drug Administration ("FDA" or "the Agency") seeking approval of
pacritinib as a treatment for myelofibrosis patients with severe
thrombocytopenia (platelet counts less than 50 x 109/L).
CTI has previously announced the results of a recent Pre-NDA
meeting with FDA where agreement was reached on an NDA submission
package based upon available data from the completed Phase 3
PERSIST-1 and PERSIST-2, and the Phase 2 PAC203 trials.
"Today we are pleased to announce the start of a rolling NDA
submission that seeks to address the important unmet medical need
of myelofibrosis patients with severe thrombocytopenia, a
population that includes both front-line treatment-naïve patients
and patients with prior exposure to JAK2 inhibitors," said
Adam R. Craig, M.D., Ph.D.,
President and Chief Executive Officer of CTI Biopharma. "We have
started pre-commercial activities and are planning for a commercial
launch in 2021, subject to priority review."
About Myelofibrosis and Severe Thrombocytopenia
Myelofibrosis is a type of bone marrow cancer that results in
formation of fibrous scar tissue and can lead to severe
thrombocytopenia and anemia, weakness, fatigue and enlarged spleen
and liver. Patients with severe thrombocytopenia are estimated to
make up more than one-third of patients treated for myelofibrosis,
or approximately 17,000 people in the
United States and Europe.
Severe thrombocytopenia, defined as blood platelet counts of less
than 50,000 per microliter, has been shown to result in overall
survival rates of just 15 months. Thrombocytopenia in patients with
myelofibrosis is associated with the underlying disease but has
also been shown to correlate with treatment with ruxolitinib, which
can lead to dose reductions, and as a result, may potentially
reduce clinical benefit. Survival in patients who have discontinued
ruxolitinib therapy is further compromised, with an average overall
survival of seven to 14 months. Myelofibrosis patients with severe
thrombocytopenia have limited treatment options, creating a
significant area of unmet medical need.
About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with
specificity for JAK2, 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 (AML), myelodysplastic syndrome (MDS), chronic
myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia
(CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.
About CTI BioPharma Corp.
We are a
biopharmaceutical company focused on the acquisition, development
and commercialization of novel targeted therapies for blood-related
cancers that offer a unique benefit to patients and their
healthcare providers. We concentrate our efforts on treatments that
target blood-related cancers where there is an unmet medical need.
In particular, we are focused on evaluating pacritinib, our sole
product candidate currently in active late-stage development, for
the treatment of adult patients with myelofibrosis. In addition, we
have recently started developing pacritinib for use in hospitalized
patients with severe COVID-19, in response to the COVID-19
pandemic. We are headquartered in Seattle, Washington.
Forward-Looking Statements
Statements included in this
press release that are not historical in nature are forward-looking
statements within the meaning of Section 27A of the Securities Act
of 1933 and Section 21E of the Securities Exchange Act of 1934 and
the Private Securities Litigation Reform Act of 1995. These
forward-looking statements are based on current assumptions that
involve risks, uncertainties and other factors that may cause the
actual results, events or developments to be materially different
from those expressed or implied by such forward-looking statements.
These risks and uncertainties include, but are not limited to: our
ability to successfully demonstrate the safety and efficacy of
pacritinib; our ability to complete a rolling NDA for pacritinib in
the timeline currently anticipated; our ability to receive
regulatory approval for pacritinib pursuant to the accelerated
approval pathway or at all; our planned commercialization of
pacritinib; our ability to enter into potential partnerships
relating to our commercial launch of pacritinib; and those risks
more fully discussed in the section entitled "Risk Factors" in our
Annual Report on Form 10-K for the year ended December 31, 2019 and subsequent quarterly
reports on Form 10-Q. These forward-looking statements speak only
as of the date hereof and we assume no obligation to update these
forward-looking statements, and readers are cautioned not to place
undue reliance on such forward-looking statements. "CTI BioPharma"
and the CTI BioPharma logo are registered trademarks or trademarks
of CTI BioPharma Corp. in various jurisdictions. All other
trademarks belong to their respective owner.
CTI BioPharma Investor Contacts:
Maeve Conneighton/Maghan
Meyers
+212-600-1902
cti@argotpartners.com
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SOURCE CTI BioPharma Corp.