– Strong U.S. commercial launch of
VONJO™ (pacritinib) underway following accelerated FDA
approval for the treatment of adult patients with Myelofibrosis and
Thrombocytopenia –
– VONJO (pacritinib) net product revenue of
$2.3 million in less than one month
following launch, exceeding initial Company expectations –
– VONJO included as recommended first-
and second-line treatment in NCCN®
Clinical Practice Guidelines in Oncology for Myeloproliferative
Neoplasms –
– Abstracts accepted at ASCO 2022 and EHA 2022
in June –
– Management to host conference call today at
4:30 p.m. ET –
SEATTLE, May 12, 2022
/PRNewswire/ -- CTI BioPharma Corp. (Nasdaq: CTIC) today reported
its financial results for the first quarter ended March 31, 2022.
"With the accelerated FDA approval and U.S. commercial launch of
VONJO, the first quarter was transformational for CTI and the
myelofibrosis community. We are thrilled to now be delivering VONJO
for patients with cytopenic myelofibrosis who have platelet counts
below 50 x 109/L. Our U.S. commercial team has been in
the field since early March and has delivered net product revenue
of $2.3 million in less than a month,
exceeding our internal expectations and establishing a great
foundation for future performance," said Adam Craig, President and Chief Executive
Officer of CTI BioPharma. "We are also pleased that
NCCN® quickly recommended VONJO for the treatment of
myelofibrosis, making VONJO the only approved JAK inhibitor
recommended by NCCN for these patients regardless of platelet
counts."
Recent Accomplishments and Updates
- FDA accelerated approval for VONJO 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.
- U.S. commercial launch in early March by fully funded sales and
marketing team.
- $60 million payment from DRI
Healthcare Trust for the acquisition of a tiered royalty for
VONJO.
- VONJO included as recommended treatment in the latest National
Comprehensive Cancer Network®
(NCCN®) Clinical Practice Guidelines in Oncology
for Myeloproliferative Neoplasms, as a first-line treatment for
high-risk patients with myelofibrosis with platelet counts <50 x
109/L who are not candidates for transplant, and as a
second-line treatment for lower-risk and higher-risk patients with
myelofibrosis with platelet counts ≥50 x 109/L who are
not candidates for transplant.
- Accepted abstract at the 2022 American Society of Clinical
Oncology (ASCO) Annual Meeting being held June 3–7, 2022, in
Chicago and virtually:
-
- Abstract Title: Risk-adjusted safety analysis of
pacritinib (PAC) in patients (pts) with myelofibrosis (MF)
-
- Abstract Number: 7058
- Session Name: Hematologic Malignancies—Leukemia,
Myelodysplastic Syndromes, and Allotransplant
- Session Date: Saturday, June 4, 2022
- Presentation Time: 8:00 – 11:00 a.m. CDT (9:00 a.m. –
12:00 p.m. ET)
- Presenter: Dr. Naveen
Pemmaraju
- Accepted abstracts at the European Hematology Association (EHA)
2022 Congress being held June 9–12, 2022, in Vienna, Austria:
-
- Abstract Title: Risk-adjusted safety analysis of
pacritinib in patients with myelofibrosis
-
- Abstract Number: P1068
- Session Name: Poster session
- Session Date: Friday, June 10, 2022
- Presentation Time: 16:30 – 17:45 CEST (10:30 – 11:45
a.m. ET)
- Presenter: Dr Naveen
Pemmaraju
- Abstract Title: Retrospective comparison of patient
outcomes on pacritinib versus ruxolitinib in patients with
myelofibrosis and thrombocytopenia
-
- Abstract Number: P1069
- Session Name: Poster session
- Session Date: Friday, June 10, 2022
- Presentation Time: 16:30 – 17:45 CEST (10:30 – 11:45
a.m. ET)
- Presenter: Prof. Claire
Harrison
First Quarter Financial Results
Net product sales of $2.3 million
for the first quarter ended March 31, 2022 were attributable to
VONJO product sales in the United
States. There were no product sales for the comparable
period in 2021. Our realization of future product sales will be
dependent, in part, upon our commercialization efforts and the
market acceptance of VONJO among physicians, patients, healthcare
payers and the medical community.
Operating loss was $35.1 million
and $17.1 million for the three
months ended March 31, 2022 and 2021, respectively. The increase in
operating loss between periods resulted primarily from increases in
selling, general and administrative activities related to the
commercial-launch of VONJO and the growth in our commercial
infrastructure, as well as a $10.3
million milestone expense related to FDA approval of VONJO,
which was included in other operating expenses for the three months
ended March 31, 2022.
Net loss for the three months ended March 31, 2022 was
$37.2 million, or $0.37 for basic and diluted loss per share,
compared to net loss of $17.3
million, or $0.23 for basic
and diluted loss per share, for the same period in 2021.
As of March 31, 2022, our cash and cash equivalents totaled
$96.9 million. We expect our present
financial resources, including expected cash receipts from
receivables arising from historical net product sales of VONJO (but
excluding any proceeds of future net product sales of VONJO), will
enable us to fund our operations into the first quarter of 2023. In
accordance with applicable accounting standards, our evaluation of
our expected cash runway considers only relevant conditions and
events that are known or reasonably knowable at the date that the
financial statements are issued. As a result, our cash runway
evaluation did not include VONJO sales that we may recognize in the
future. We expect to include future net product sales of VONJO in
our cash runway projections once we have an established history of
such sales.
Conference Call and Webcast
CTI will host a conference call and webcast to review its
first quarter 2022 financial results and provide an
update on business activities today, May 12 at 4:30 p.m. ET. To
access the live call by phone please dial (877) 735-2860 (domestic)
or (602) 563-8791 (international); the conference ID is 7291915. A
live audio webcast of the event may also be accessed through the
"Investors" section of CTI's website at www.ctibiopharma.com. A
replay of the webcast will be available for 30 days following the
event.
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 7 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 2 weeks. The
incidence of reported diarrhea decreased over time with 41% of
patients reporting diarrhea in the first 8 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 anti–diarrheal
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 7 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 Janus associated kinase (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) 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, 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. 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 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; 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
(tables follow)
CTI BioPharma
Corp.
Condensed Statements
of Operations
(In thousands, except
per share amounts)
(unaudited)
|
|
Three Months Ended
March 31,
|
|
2022
|
|
2021
|
Net product
sales
|
$
2,295
|
|
$
—
|
Operating costs and
expenses:
|
|
|
|
Cost of sales
|
278
|
|
—
|
Research and development
|
8,048
|
|
9,444
|
Selling, general and administrative
|
18,046
|
|
7,626
|
Other operating expenses
|
11,023
|
|
—
|
Total operating costs and
expenses
|
37,395
|
|
17,070
|
Loss from
operations
|
(35,100)
|
|
(17,070)
|
Non-operating
expenses:
|
|
|
|
Interest expense, net
|
(2,063)
|
|
(187)
|
Foreign exchange loss
|
(12)
|
|
(9)
|
Total non-operating
expenses
|
(2,075)
|
|
(196)
|
Net loss
|
$
(37,175)
|
|
$
(17,266)
|
Basic and diluted net
loss per common share
|
$
(0.37)
|
|
$
(0.23)
|
Shares used in
calculation of basic and diluted net loss per common
share:
|
99,834
|
|
76,367
|
|
|
Balance Sheet Data
(unaudited):
|
|
(amounts in
thousands)
|
|
|
March 31,
|
|
December
31,
|
|
|
2022
|
|
2021
|
Cash and cash
equivalents
|
|
$
96,902
|
|
$
65,446
|
Accounts receivable,
net
|
|
2,426
|
|
—
|
Working
capital
|
|
4,351
|
|
1,728
|
Total assets
|
|
131,435
|
|
72,434
|
Current portion of
long-term debt
|
|
47,521
|
|
47,380
|
Total stockholders'
(deficit) equity
|
|
(27,917)
|
|
3,767
|
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SOURCE CTI BioPharma Corp.