SEATTLE, March 9, 2018 /PRNewswire/ -- CTI BioPharma Corp.
(CTI BioPharma) (NASDAQ: CTIC) today announced that results from
the Phase 3 PERSIST-2 clinical trial of pacritinib (an
investigational JAK2 inhibitor) have been published online in
JAMA Oncology. The randomized, international, multicenter
study compared the efficacy and safety of pacritinib at two dose
levels, compared with best available therapy (BAT), which included
ruxolitinib (a JAK1/JAK2 inhibitor), in patients with myelofibrosis
and thrombocytopenia (defined as platelet counts ≤100 x
109/L). The publication can be accessed at:
https://jamanetwork.com/journals/jamaoncology/fullarticle/2674384.
In the intent-to-treat patient population of the study, the
combined pacritinib arms (400mg once daily and 200mg twice daily
dosing, 149 patients total) demonstrated a significant improvement
of 35% or more in spleen volume reduction (SVR) at 24 weeks of
treatment in 27 patients (18%) compared to 2 patients (3%) out of
72 patients in the BAT arm, which included treatment with
ruxolitinib (P=0.001). The combined pacritinib treatment arms also
demonstrated a greater than 50% reduction in total symptom score
(TSS) in 37 patients (25%), compared to 10 patients (14%) in the
BAT arm (P=0.079). Additionally, an exploratory analysis of
the 74 patients who received pacritinib 200mg twice daily showed an
improvement of 35% or more reduction of SVR in 16 patients (22%;
P=0.001 vs BAT) and 50% or greater reduction of TSS in 24 patients
(32%; P=0.01 vs BAT).
Pacritinib was generally well tolerated. The most commonly
reported (≥15%) nonhematologic adverse events with pacritinib were
gastrointestinal events, fatigue, peripheral edema, and dizziness,
and those with BAT (including 19 patients with watchful-waiting
only) were abdominal pain, fatigue, diarrhea, and peripheral edema.
The majority of common nonhematologic adverse events were grade 1
or 2 in severity. Diarrhea was the most frequently observed adverse
event with pacritinib (53% grade 1/2; 4% grade 3) most often
occurring during weeks 1 to 8. The incidence of diarrhea was lower
with pacritinib twice daily dosing compared to once daily dosing
(48% vs 67%, respectively). Diarrhea was manageable with standard
antidiarrheal agents (e.g., loperamide) and generally resolved
within 1 to 2 weeks. The rate of on-study death was lowest with
pacritinib twice daily (6%) compared to BAT (9%) and pacritinib
once daily (14%).
Cardiac events were reported at similar rates in all arms (32%,
pacritinib once daily or twice daily; 28%, BAT) and were most
commonly peripheral edema in all arms. Grade 3 or 4 cardiac events
were reported in 13 patients (13%) treated with pacritinib once
daily, 7 patients (7%) treated with pacritinib twice daily, and 9
patients (9%) treated with BAT.
Bleeding events were reported at similar rates in all arms (36%,
42%, and 41% of patients treated with pacritinib once daily, twice
daily, and BAT, respectively) and were most commonly epistaxis in
all arms. Grade 3 or 4 bleeding events were reported in 7 patients
(7%), 15 patients (14%), and 7 patients (7%) treated with
pacritinib once daily, twice daily, and BAT, respectively.
"Pacritinib was shown to reduce both spleen volume and total
symptom score, two very important clinical measures, in
myelofibrosis patients with thrombocytopenia including those
patients who received prior treatment with ruxolitinib," stated
John Mascarenhas, M.D., Adult
Leukemia Program, Tisch Cancer Institute, Icahn School of Medicine
at Mount Sinai. "Clinical improvements in hemoglobin levels and
reduction in transfusions were also seen in patients who received
pacritinib, and pacritinib had a generally manageable safety
profile."
Dr. Mascarenhas continued, "Myelofibrosis is a difficult and
progressive disease, and patients with thrombocytopenia that have
already received JAK2 inhibitor therapy have an especially poor
prognosis. These study results indicate there is important
potential clinical benefit of pacritinib for these patients."
The PERSIST-2 trial results were previously presented at the
58th American Society of Hematology (ASH) Annual Meeting, in
December 2016.
CTI BioPharma is currently enrolling patients in the PAC203
study, which is evaluating the safety and efficacy of three dosing
schedules, including 200mg twice daily (BID), 100mg twice daily
(BID), and 100mg once daily (QD).
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.
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 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).
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. In
Europe, there is a wide variation
of prevalence observed across data sources. Myelofibrosis has a
median age of 64 at the time of diagnosis and is a progressive
disease with approximately 20 percent of patients eventually
developing acute myeloid leukemia (AML). 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.
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. 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 BioPharma's securities. Such
statements include, but are not limited to, statements regarding
expectations with respect to the potential important clinical
benefits of pacritinib and the potential therapeutic utility of
pacritinib. Investors are cautioned not to place undue reliance on
these forward-looking statements, which speak only as of the date
of this release. The statements are based on assumptions about many
important factors and information currently available to us to the
extent we have thus far had an opportunity to fully and carefully
evaluate such information in light of all surrounding facts,
circumstances, recommendations and analyses. A number of results
and uncertainties could cause actual results to differ materially
from those in the forward-looking statements, including those
related to the satisfaction of regulatory and other requirements;
the actions of regulatory bodies and other governmental
authorities; changes in laws and regulations; other clinical trial
results; that results observed to date may differ from future
results or that different conclusions or considerations may qualify
such results once existing data has been more fully evaluated;
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. Except as required by law, CTI BioPharma
does not intend to update any of the statements in this press
release upon further developments.
Investor Contacts:
Tricia Truehart
+1 646-378-2953
ttruehart@troutgroup.com
View original content with
multimedia:http://www.prnewswire.com/news-releases/cti-biopharma-announces-publication-of-pacritinib-phase-3-persist-2-clinical-trial-in-jama-oncology-300611396.html
SOURCE CTI BioPharma Corp.