SEATTLE, Sept. 21,
2022 /PRNewswire/ -- CTI BioPharma Corp. (NASDAQ:
CTIC) today announced two poster presentations from the Company's
pacritinib program at the Society of Hematologic Oncology (SOHO)
Tenth Annual Meeting, to be held in Houston, Texas and virtually September 28 – October 1,
2022.
A new data analysis from the Phase 3 PERSIST-2 trial and an
in vitro analysis of pacritinib, a novel JAK2/IRAK1
inhibitor approved by the U.S. FDA for patients with myelofibrosis
and a platelet count below 50 x 109/L, will highlight
pacritinib's impact on anemia and inhibition of Activin A receptor
type 1 (ACVR1).
"Treatment with pacritinb at the approved dose of 200 mg twice
daily (BID) led to improvements in transfusion independence and
anemia when compared to best available therapy (BAT) in patients
treated on the PERSIST-2 Phase 3 study," said Dr. Stephen Oh, MD, PhD, Associate Professor of
Medicine, Hematology Division at Washington
University School of Medicine in St. Louis. "I am encouraged by these data,
given the limited options to address anemia in myelofibrosis,
especially high-risk patients with cytopenias who frequently
require blood transfusions. I look forward to further investigation
of pacritinib's potential to alleviate anemia and related symptoms
in this patient population."
"We are pleased to report that pacritinib is a highly potent
inhibitor of ACVR1. This inhibition is thought to lead to
improvements in blood transfusion requirements and anemia in
patients with cytopenic myelofibrosis," said Adam Craig, MD,
PhD, President and Chief Executive Officer of CTI BioPharma. "The
data presented at SOHO 2022 support our belief that pacritinib is a
simple, safe and effective JAK2 inhibitor. We plan to present
additional data on pacritinib's anemia benefit at a medical meeting
later this year."
Presentation materials will be available at
ctibiopharma.com.
Retrospective Analysis of Anemia Benefit of Pacritinib from
the PERSIST-2 Trial
Abstract Number: MPN-145
Session Name: Poster Session
Session Date: Wednesday,
September 28
Presentation Time: 5:05-6:30 p.m.
CDT (06:05-7:30 p.m. EDT)
Presenter: Dr. Stephen
Oh
Pacritinib is a novel JAK2/IRAK1 inhibitor approved by the U.S.
Food and Drug Administration (FDA) for patients with myelofibrosis
and severe thrombocytopenia (platelet count <50 x
109/L). Previous clinical trials have noted improvements
in spleen volume and symptom scores, as well as improvement in
anemia and a decline in transfusion burden. A retrospective
analysis of the Phase 3 PERSIST-2 trial was conducted to further
assess pacritinib's impact on anemia, and an in vitro
analysis was performed to explore pacritinib's inhibition of
Activin A receptor type 1 (ACVR1; also known as activin
receptor-like kinase 2 [ALK2]).
On duplicate assays, pacritinib was shown to be a more potent
inhibitor of ACVR1 compared to momelotinib, with a half maximal
inhibitory concentration (IC50) of 10.8 and 22.6 nM
versus34.9 and 70.2 nM, respectively. The percentage of patients
who achieved transfusion independence over any 12-week intervals
through week 24 was greater on pacritinib 200 mg twice daily than
best available therapy (27% vs 7%), among evaluable non-transfusion
independent patients (defined as any red blood cell transfusions in
90 days prior to the first dose or a baseline hemoglobin <8
g/dL). These data suggest an important role for pacritinib in
addressing anemia in patients with myelofibrosis.
Retrospective Comparison of Patient Outcomes on Pacritinib
Versus Ruxolitinib in Patients with Myelofibrosis (MF) and
Thrombocytopenia (encore)
Abstract Number: MPN-141
Session Name: Poster Session
Session Date: Wednesday, September 28
Presentation Time: 5:05-6:30 p.m. CDT (06:05-7:30 p.m.
EDT)
Presenter: Dr. Prithviraj
Bose
Pacritinib is a novel JAK2/IRAK1 inhibitor approved by the U.S.
FDA for patients with myelofibrosis and severe thrombocytopenia
(platelet count <50 x 109/L). Unlike the JAK 1/2
inhibitor ruxolitinib, which must be dose-reduced or held in
patients with thrombocytopenia, pacritinib has been studied at full
dose regardless of platelet count. This retrospective analysis
reported on the outcomes in patients treated with pacritinib versus
ruxolitinib in the Phase 3 PERSIST-2 study. Patients were
randomized 1:1:1 to pacritinib 200 mg BID, pacritinib 400 mg once
daily (QD) or BAT, with 45 percent of patients on BAT receiving
ruxolitinib.
Results show that pacritinib, administered at the full dose of
200 mg BID, yielded higher response rates and a similar safety
profile compared to lower-dose ruxolitinib in patients with
myelofibrosis who have moderate or severe thrombocytopenia.
About VONJO (pacritinib)
Pacritinib is an oral kinase
inhibitor with activity against wild type Janus Associated Kinase 2
(JAK2), mutant JAK2V617F form and FMS-like tyrosine
kinase 3 (FLT3), which contribute to signaling of a number of
cytokines and growth factors that are important for hematopoiesis
and immune function. Myelofibrosis is often associated with
dysregulated JAK2 signaling. Pacritinib has higher inhibitory
activity for JAK2 over other family members, JAK3 and TYK2. At
clinically relevant concentrations, pacritinib does not inhibit
JAK1. Pacritinib exhibits inhibitory activity against additional
cellular kinases (such as CSF1R and IRAK1), the clinical relevance
of which is unknown.
VONJO is indicated for the treatment of adults with intermediate
or high-risk primary or secondary (post-polycythemia vera or
post-essential thrombocythemia) myelofibrosis with a platelet count
below 50 × 109/L. This indication is approved under
accelerated approval based on spleen volume reduction. Continued
approval for this indication may be contingent upon verification
and description of clinical benefit in a confirmatory trial(s).
Important VONJO Safety Information
Hemorrhage:
Serious (11%) and fatal (2%) hemorrhages have occurred in
VONJO-treated patients with platelet counts <100 ×
109/L. Serious (13%) and fatal (2%) hemorrhages have
occurred in VONJO-treated patients with platelet counts <50 ×
109/L. Grade ≥3 bleeding events (defined as requiring
transfusion or invasive intervention) occurred in 15% of patients
treated with VONJO compared to 7% of patients treated on the
control arm. Due to hemorrhage, VONJO dose reductions, dose
interruptions, or permanent discontinuations occurred in 3%, 3%,
and 5% of patients, respectively.
Avoid use of VONJO in patients with active bleeding and hold
VONJO seven days prior to any planned surgical or invasive
procedures. Assess platelet counts periodically, as clinically
indicated. Manage hemorrhage using treatment interruption and
medical intervention.
Diarrhea:
VONJO causes diarrhea in approximately 48% of patients compared to
15% of patients treated on the control arm. The median time to
resolution in VONJO-treated patients was two weeks. The incidence
of reported diarrhea decreased over time, with 41% of patients
reporting diarrhea in the first eight weeks of treatment, 15% in
weeks 8 through 16, and 8% in weeks 16 through 24. Diarrhea
resulted in treatment interruption in 3% of VONJO-treated patients.
None of the VONJO-treated patients reported diarrhea that resulted
in treatment discontinuation. Serious diarrhea adverse reactions
occurred in 2% of patients treated with VONJO compared to no such
adverse reactions in patients in the control arm.
Control pre-existing diarrhea before starting VONJO treatment.
Manage diarrhea with antidiarrheal medications, fluid replacement,
and dose modification. Treat diarrhea with antidiarrheal
medications promptly at the first onset of symptoms. Interrupt or
reduce VONJO dose in patients with significant diarrhea despite
optimal supportive care.
Thrombocytopenia:
VONJO can cause worsening thrombocytopenia. VONJO dosing was
reduced due to worsening thrombocytopenia in 2% of patients with
pre-existing moderate to severe thrombocytopenia (platelet count
<100 × 109/L). VONJO dosing was reduced due to
worsening thrombocytopenia in 2% of patients with pre-existing
severe thrombocytopenia (platelet count <50 ×
109/L).
Monitor platelet count prior to VONJO treatment and as
clinically indicated during treatment. Interrupt VONJO in patients
with clinically significant worsening of thrombocytopenia that
lasts for more than seven days. Restart VONJO at 50% of the last
given dose once the toxicity has resolved. If toxicity recurs hold
VONJO. Restart VONJO at 50% of the last given dose once the
toxicity has resolved.
Prolonged QT interval:
VONJO can cause prolongation of the QTc interval. QTc prolongation
of >500 msec was higher in VONJO-treated patients than in
patients in the control arm (1.4% vs 1%). QTc increase from
baseline by 60 msec or higher was greater in VONJO-treated patients
than in control arm patients (1.9% vs 1%). Adverse reactions of QTc
prolongation were reported for 3.8% of VONJO-treated patients and
2% of control arm patients. No cases of torsades de pointes were
reported.
Avoid use of VONJO in patients with a baseline QTc of
>480 msec. Avoid use of drugs with significant potential for QTc
prolongation in combination with VONJO. Correct hypokalemia prior
to and during VONJO treatment. Manage QTc prolongation using VONJO
interruption and electrolyte management.
Major Adverse Cardiac Events (MACE):
Another JAK)-inhibitor has increased the risk of MACE, including
cardiovascular death, myocardial infarction, and stroke (compared
to those treated with TNF blockers) in patients with rheumatoid
arthritis, a condition for which VONJO is not indicated.
Consider the benefits and risks for the individual patient prior
to initiating or continuing therapy with VONJO, particularly in
patients who are current or past smokers and patients with other
cardiovascular risk factors. Patients should be informed about the
symptoms of serious cardiovascular events and the steps to take if
they occur.
Thrombosis:
Another JAK-inhibitor has increased the risk of thrombosis,
including deep venous thrombosis, pulmonary embolism, and arterial
thrombosis (compared to those treated with TNF blockers) in
patients with rheumatoid arthritis, a condition for which VONJO is
not indicated.
Patients with symptoms of thrombosis should be promptly
evaluated and treated appropriately.
Secondary Malignancies:
Another JAK-inhibitor has increased the risk of lymphoma and other
malignancies, excluding non-melanoma skin cancer (NMSC) (compared
to those treated with TNF blockers), in patients with rheumatoid
arthritis, a condition for which VONJO is not indicated. Patients
who are current or past smokers are at additional increased
risk.
Consider the benefits and risks for the individual patient prior
to initiating or continuing therapy with VONJO, particularly in
patients with a known malignancy (other than a successfully-treated
NMSC), patients who develop a malignancy, and patients who are
current or past smokers.
Risk of Infection:
Another JAK-inhibitor has increased the risk of serious infections
compared to best available therapy (BAT) in patients with
myeloproliferative neoplasms. Serious bacterial, mycobacterial,
fungal and viral infections may occur in patients treated with
VONJO. Delay starting therapy with VONJO until active serious
infections have resolved. Observe patients receiving VONJO for
signs and symptoms of infection and manage promptly. Use active
surveillance and prophylactic antibiotics according to clinical
guidelines.
Interactions with CYP3A4 Inhibitors or Inducers:
Co-administration of VONJO with strong CYP3A4 inhibitors or
inducers is contraindicated. Avoid concomitant use of VONJO with
moderate CYP3A4 inhibitors or inducers.
Drug interruptions due to an adverse reaction occurred in 27%
patients who received VONJO 200 mg twice daily compared to 10% of
patients treated with BAT. Dosage reductions due to an adverse
reaction occurred in 12% of patients who received VONJO 200 mg
twice daily compared to 7% of patients treated with BAT. Permanent
discontinuation due to an adverse reaction occurred in 15% of
patients receiving VONJO 200 mg twice daily compared to 12% of
patients treated with BAT.
Please
visit http://www.ctibiopharma.com/vonjo_prescribing_information for
full Prescribing Information and the Medication Guide.
About Myelofibrosis
Myelofibrosis is bone marrow
cancer that results in formation of fibrous scar tissue and can
lead to thrombocytopenia and anemia, weakness, fatigue and an
enlarged spleen and liver. Within the
United States, there are approximately 21,000 patients with
myelofibrosis, 7,000 of which have severe thrombocytopenia (defined
as blood platelet counts of less than 50 x109/L). Severe
thrombocytopenia is associated with poor survival and high symptom
burden and can occur as a result of disease progression or from
drug toxicity with other JAK2 inhibitors, such as JAKAFI and
INREBIC.
About CTI BioPharma Corp.
We are a commercial
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. CTI has one FDA-approved product,
VONJO® (pacritinib), a JAK2 and IRAK1 inhibitor,
that spares JAK1. VONJO is approved for the treatment of adults
with intermediate- or high-risk primary or secondary
(post-polycythemia vera or post-essential thrombocythemia)
myelofibrosis with a platelet count below 50 × 109/L.
This indication is approved under FDA accelerated approval based on
spleen volume reduction. Continued approval for this indication may
be contingent upon verification and description of clinical benefit
in a confirmatory trial(s). CTI is conducting the Phase 3 PACIFICA
study of VONJO in patients with myelofibrosis and severe
thrombocytopenia as a post-marketing requirement.
VONJO® is a registered trademark of CTI
BioPharma Corp.
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 commercialize VONJO and generate future
revenues with respect to VONJO; our limited experience in
generating revenue from product sales; the accuracy of our
assumptions regarding our planned expenditures and sufficiency of
our cash to fund operations; risks and uncertainties related to the
COVID-19 pandemic as it relates to our operations and ongoing
clinical trials; plans to present additional data on pacritinib's
anemia benefit at later scientific conferences or in scientific
journals; 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, 2021 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:
Argot Partners
+212-600-1902
cti@argotpartners.com
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