Celator Announces Phase 3 Trial for VYXEOS™ (CPX-351) in Patients
Acute Myeloid Leukemia Demonstrates Statistically Significant Improvement in
-- First therapy to demonstrate statistically significant
improvement in overall survival and induction
response rate in a pivotal Phase 3 trial in high-risk AML --
-- NDA submission for VYXEOS planned for later this year --
-- Conference Call on Tuesday, March 15, 2016 at 8:00am EDT --
EWING, N.J., March 14, 2016 /PRNewswire/ -- Celator Pharmaceuticals, Inc.
(Nasdaq: CPXX) today announced positive results from the Phase 3 trial of
VYXEOS™ (cytarabine: daunorubicin) Liposome for Injection (also known as
CPX-351) in patients with high-risk (secondary) acute myeloid leukemia (AML)
compared to the standard of care regimen of cytarabine and daunorubicin
known as 7+3. The trial met its primary endpoint demonstrating a
statistically significant improvement in overall survival. Data will be
submitted for presentation at the American Society of Clinical Oncology 2016
The median overall survival for patients treated with VYXEOS in the study
was 9.56 months compared to 5.95 months for patients receiving 7+3,
representing a 3.61 month improvement in favor of VYXEOS. The hazard ratio
(HR) was 0.69 (p=0.005) which represents a 31 percent reduction in the risk
of death versus 7+3. The percentage of patients alive 12 months after
randomization was 41.5% on the VYXEOS arm compared to 27.6% on the 7+3 arm.
The percentage of patients alive 24 months after randomization was 31.1% on
the VYXEOS arm compared to 12.3% on the 7+3 arm.
"The overall survival advantage seen with CPX-351 compared to 7+3, along
with a superior response rate and no increase in serious toxicity indicates
that we'll likely have a new standard of care for treating older patients
with secondary AML," said Jeffrey E. Lancet, M.D., senior member and chief
of the Leukemia/Myelodysplasia Program at Moffitt Cancer Center and the
principal investigator for the study. "This represents a major step forward
for a very difficult-to-treat patient population."
VYXEOS also demonstrated a statistically significant improvement in
induction response rate (CR+CRi of 47.7% versus 33.3%; p=0.016) and this
significance was maintained for the analysis of CR alone (CR of 37.3% versus
Sixty-day all-cause mortality was 13.7% versus 21.2%, in favor of patients
treated with VYXEOS.
No substantial difference in Grade 3 or higher adverse events was observed
between VYXEOS and 7+3. In the intent-to-treat population, Grade 3 or
higher, hematologic adverse events were similar for overall infections,
febrile neutropenia, and bleeding events. In the intent-to-treat population,
Grade 3 or higher, non-hematologic adverse events were similar across all
organ systems, including cardiac, gastrointestinal, general systems,
metabolic disorders, musculoskeletal, nervous system,
respiratory, skin and renal.
"These findings confirm that VYXEOS provides the first opportunity we've had
in decades to extend survival for patients with high-risk AML," added Gail
Roboz, M.D., Professor of Medicine and Director of the Leukemia Program at
the Weill Medical College of Cornell University and the New
York-Presbyterian Hospital in New York. "Also, more patients in remission
means more who are eligible for potentially curative therapy."
Based on these results the company expects to submit a New Drug Application
(NDA) for VYXEOS with the U.S. Food and Drug Administration (FDA) later this
year and submit a Marketing Authorization Application (MAA) with the
European Medicines Agency (EMA) in the first quarter of 2017.
"The successful outcome of this Phase 3 trial represents an important
advance for AML patients, their families and clinicians," said Scott
Jackson, Chief Executive Officer of Celator Pharmaceuticals. "It also marks
a major milestone for Celator, for VYXEOS, and for our CombiPlex® platform.
We offer our sincere thanks to the patients and investigators who
participated in this study and we will work closely with regulatory
authorities to make this new treatment available to the AML community as
soon as possible."
The clinical trial was conducted in partnership with The Leukemia & Lymphoma
Society® (LLS) through its Therapy Acceleration Program (TAP), which has
supported the clinical development of VYXEOS beginning in Phase 2.
Conference Call Information
Celator will host a conference call and live audio webcast on Tuesday, March
15, 2016 at 8:00am EDT to discuss the results of the Phase 3 trial. To
participate in the conference call, please dial 877-303-6316 (domestic) or
650-521-5176 (international) and refer to conference ID 71930208. The live
webcast of the call can be accessed in the Investors section of Celator's
website at www.celatorpharma.com. An archived webcast will be available on
Celator's website beginning
approximately two hours after the event.
VYXEOS (cytarabine:daunorubicin) Liposome for Injection, also known as
CPX-351, is a nano-scale co-formulation of cytarabine and daunorubicin at a
synergistic 5:1 molar ration. VYXEOS represents a novel approach to
developing combinations of drugs in which molar ratios of two drugs with
synergistic anti-tumor activity are encapsulated in a nanoscale liposome in
order to maintain the desired ratio following administration. VYXEOS was
granted orphan drug status by the FDA and the European Commission for the
treatment of acute myeloid leukemia (AML). VYXEOS was also granted Fast
Track designation for the treatment of elderly patients with secondary AML.
In addition to the Phase 3 trial, Celator published results from two
randomized, controlled, Phase 2 trials with VYXEOS. The first trial was
conducted in newly diagnosed elderly AML patients and the second trial was
conducted in patients with AML in first relapse.
Phase 3 Trial Design
The randomized, controlled, Phase 3 trial (Protocol NCT01696084), enrolled
309 patients at 39 sites in the United States and Canada, and compared
VYXEOS to the conventional cytarabine and daunorubicin treatment regimen
(commonly referred to as 7+3) as first-line therapy in older (60-75 years of
age) patients with high-risk (secondary) AML. Patients were stratified for
age (60 to 69 and 70 to 75 years of age) and AML type; treatment-related
AML, AML with documented history of MDS with prior treatment with
hypomethylating agent therapy, AML with documented history of MDS without
hypomethlyating agent therapy, AML with a documented history of chronic
myelomonocytic leukemia (CMMoL), and de novo AML with a karyotype
characteristic of myelodysplastic syndrome (MDS).
Patients were randomized 1:1 to receive either VYXEOS or 7+3. Patients could
receive one or two inductions, and responding patients could receive one or
two consolidations. First induction for VYXEOS was 100u/m2; days 1, 3, and 5
by 90-minute infusion and for the control arm was cytarabine 100mg/m2/day by
continuous infusion for 7 days and daunorubicin 60mg/m2 on days 1, 2, and 3
(7+3). Second induction for VYXEOS-treated patients was 100u/m2 on days 1
and 3 and the control arm was cytarabine 100mg/m2/day by continuous infusion
for 5 days and daunorubicin 60mg/m2 on
days 1 and 2 (5+2).
Only patients with documented CR or CRi were eligible to receive
chemotherapy consolidation. Consolidation for VYXEOStreated patients was
65u/m2 on days 1 and 3 and the control arm was cytarabine 100mg/m2/day by
continuous infusion for 5 days and daunorubicin 60mg/m2 on days 1 and 2
Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood
characterized by the uncontrolled proliferation of immature blast cells in
the bone marrow. AML is generally a disease of older adults, and the median
age of a patient diagnosed with AML is about 67 years. The American Cancer
Society estimates that there will be 19,950 new cases of AML and 10,430
deaths from AML in the U.S. in 2016. In Europe the number of new cases is
estimated to be 18,000 and in Japan the number is 5,500. The Company
estimates that nearly 70 percent of AML patients are over the age of 60, and
approximately 75 percent are intermediate or high risk. Furthermore,
approximately half of those patients are considered suitable for intensive
Even with current treatment, overall survival for AML is poor. In patients
over 60 years of age, the 5 year survival rate is less than 10%. In
high-risk (secondary) AML, overall survival is lower, resulting in an acute
need for new treatment options for these patients.
About Celator Pharmaceuticals, Inc.
Celator Pharmaceuticals, Inc., with locations in Ewing, N.J., and Vancouver,
B.C., is an oncology-focused biopharmaceutical company that is transforming
the science of combination therapy, and developing products to improve
patient outcomes in cancer. Celator's proprietary technology platform,
CombiPlex®, enables the rational design and rapid evaluation of optimized
combinations of anti-cancer drugs, incorporating traditional chemotherapies
as well as molecularly targeted agents to deliver enhanced anti-cancer
activity. CombiPlex addresses several fundamental shortcomings of
combination regimens, as well as the challenges inherent in combination drug
development, by identifying the most effective synergistic molar ratio of
the drugs being combined in vitro, and fixing this ratio in a nano-scale
drug delivery complex to maintain the optimized combination after
administration and ensuring exposure of this ratio to the tumor. Celator's
pipeline includes the lead product, VYXEOS™ (also known as CPX-351), a
nano-scale liposomal formulation of cytarabine:daunorubicin being studied
for the treatment of acute myeloid leukemia; CPX-1, a nano-scale liposomal
formulation of irinotecan:floxuridine studied in colorectal cancer; and a
preclinical stage product candidate, CPX-8, a hydrophobic docetaxel prodrug
nanoparticle formulation. The Company is advancing its CombiPlex platform
and broadening its application to include molecularly targeted therapies.
The Company is seeking research and development collaborations with other
biotechnology/pharmaceutical companies where its proprietary technology may
For more information, please visit Celator's website at
www.celatorpharma.com. Information on ongoing trials is available at
To the extent that statements contained in this press release are not
descriptions of historical facts regarding Celator, they are forward-looking
statements reflecting the current beliefs and expectations of management
made pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995. Words such as "may," "will," "expect,"
"anticipate," "estimate," "intend," and similar expressions (as well as
other words or expressions referencing future events, conditions or
circumstances) are intended to identify forward-looking statements. Examples
of forward-looking statements contained in this press release include, among
others, statements regarding the safety, potential efficacy, therapeutic
potential, and commercial potential of VYXEOS™ (also known as CPX-351), our
expectations regarding the timing of our regulatory filings, our
expectations regarding our research and development programs and advancing
our CombiPlex platform and the potential to establish research and
development collaborations applying our proprietary technologies with other
biotechnology/pharmaceutical companies. Forward-looking statements in this
release involve substantial risks and uncertainties that could cause our
development programs, future results, or achievements to differ
significantly from those expressed or implied by the forward-looking
statements. Such risks and uncertainties include, among others, the
uncertainties inherent in the conduct of clinical studies, whether clinical
study results obtained to date will be predictive of
future results, whether the final results of our clinical studies will be
supportive of regulatory approval to market VYXEOS and other matters that
could affect the commercial potential of our drug candidates. Celator
undertakes no obligation to update or revise any forward-looking statements.
For a further description of the risks and uncertainties that could cause
actual results to differ from those expressed in these forward-looking
statements, as well as risks relating to the business of the company in
general, see Celator's Form 10-K for the year ended December 31, 2014,
subsequent reports on Form 10-Q and 8-K, and other filings by the company
with the U.S. Securities and Exchange Commission.
Sam Brown, Inc.
The Trout Group
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