SEATTLE, Feb. 3, 2020 /PRNewswire/ -- CTI BioPharma
Corp. (Nasdaq: CTIC) today announced that following a meeting with
the U.S. Food and Drug Administration ("FDA" or "the Agency"), CTI
has reached agreement on an accelerated approval pathway for
pacritinib for the treatment of myelofibrosis patients with severe
thrombocytopenia (platelet counts <50,000/µL). CTI will be
amending the PACIFICA pivotal Phase 3 trial protocol to allow for
the primary analysis of SVR rates on the first 168 patients, with
an end-of-study analysis of TSS and OS following the full
enrollment of 348 patients. If the primary endpoint of SVR is met
following the planned review of data from the first 168 patients,
CTI intends to submit a New Drug Application (NDA) under the FDA's
subpart H regulations, subject to review of all available efficacy
and safety data. Conversion to a regular approval of pacritinib
would be anticipated following the successful end-of-study
assessment of the secondary efficacy endpoints, and the completion
of post-marketing requirements.
"Since the initiation of the PACIFICA trial in September 2019, we have been working diligently
with the FDA to identify an expedited approval pathway for
pacritinib for the treatment of myelofibrosis patients with severe
thrombocytopenia," said Adam R.
Craig, M.D., Ph.D., President and Chief Executive Officer of
CTI Biopharma. "Severely thrombocytopenic myelofibrosis patients
(platelet counts <50,000/µL) have reduced survival and very
limited therapeutic options. Pacritinib has now demonstrated
clinical benefit in this population in three clinical trials,
including two prior randomized Phase 3 studies, so we believe that
pacritinib has the potential to change the treatment paradigm in
this area of serious unmet medical need."
Based on the new trial design, CTI expects to report primary SVR
data by the end of 2021, with a potential NDA filing in early 2022
if the SVR data is positive. Final study efficacy data is expected
in 2023.
Concurrent with this press release, CTI is announcing a
$60 million rights offering. For
further details, see the concurrent press release relating to the
rights offering.
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 anemia,
weakness, fatigue and an 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. 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. There are
currently no approved therapies available to treat myelofibrosis
patients with severe thrombocytopenia or patients who have failed
ruxolitinib treatment, thereby making this a significant unmet
medical need.
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 (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.
CTI BioPharma Corp. is 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. In particular,
we are focused on evaluating pacritinib for the treatment of adult
patients with myelofibrosis. CTI BioPharma is
headquartered in Seattle, Washington.
Forward-Looking Statements
This press release contains 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 include statements regarding our expectations regarding:
the anticipated trial design of the PACIFICA Phase 3 trial,
including changes to the protocol as discussed in this press
release; the anticipated enrollment of the PACIFICA Phase 3 trial;
the effectiveness of, and potential changes to, the PACIFICA Phase
3 trial design; the timing of, and results from, clinical trials
and other development activities related to pacritinib, including
the PACIFICA Phase 3 trial and its related protocol; the potential
efficacy, safety profile, future development plans, addressable
market, regulatory success and commercial potential of pacritinib;
the anticipated timing of regulatory submissions and interactions,
including any potential NDA submission; our ability to expedite the
regulatory approval process; our ability to successfully develop
and achieve milestones in the development of pacritinib; and the
anticipated benefits of pacritinib.
Risks Related to Forward-Looking Statements
The forward-looking statements contained in this press release 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, many of which are beyond our control, include, but
are not limited to: clinical trials may not demonstrate safety and
efficacy of pacritinib; the FDA may determine that the
benefit/risk profile of pacritinib at the dose selected for the
PACIFICA Phase 3 trial does not support approval based on the
results of such trial, previously identified FDA concerns
regarding safety and dosing limitations or otherwise; pacritinib
may fail in development, may not receive required regulatory
approvals, or may be delayed to a point where it is not
commercially viable; our assumptions regarding our planned
expenditures and sufficiency of our cash to fund operations may be
incorrect; we may not achieve additional milestones in our
pacritinib development program; the impact of competition; the
impact of expanded product development and clinical activities on
operating expenses; adverse conditions in the general domestic and
global economic markets; as well as the other risks identified in
our filings with the Securities and Exchange Commission. 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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