Moxetumomab pasudotox BLA granted Priority
Review
AstraZeneca and MedImmune, its global biologics research and
development arm, today announced that the US Food and Drug
Administration (FDA) has accepted the Biologics License Application
(BLA) for moxetumomab pasudotox, an investigational anti-CD22
recombinant immunotoxin and a potential new medicine for the
treatment of adult patients with hairy cell leukemia (HCL) who have
received at least two prior lines of therapy. The FDA has granted
the moxetumomab pasudotox BLA Priority Review status with a
Prescription Drug User Fee Act (PDUFA) date set for the third
quarter of 2018.
The Phase III (‘1053’) moxetumomab pasudotox clinical trial met
its primary endpoint of durable complete response in adult patients
with relapsed or refractory HCL, for which there is currently no
established standard of care and few treatments available.1,2
Results from the 1053 Phase III trial will be presented at a
forthcoming medical meeting.
Priority Review is granted by the FDA to applications for
medicines that, if approved, would offer a significant improvement
in the safety or effectiveness of the treatment, diagnosis, or
prevention of serious conditions.3
NOTES TO EDITORS
About Moxetumomab Pasudotox
Moxetumomab pasudotox (formerly CAT-8015 or HA22) is an
investigational anti-CD22 recombinant immunotoxin and a potential
new medicine with the opportunity to be a first-in-class treatment
in the US for patients with relapsed or refractory hairy cell
leukemia (HCL) who have received at least two prior lines of
therapy. Immunotoxins are a class of anticancer agents that combine
the selectivity of antibodies to target drug delivery and the
potency of toxins to kill target cancer cells.4 Moxetumomab
pasudotox is composed of a binding portion of an anti-CD22 antibody
fused to a toxin. CD22 is a B-lymphocyte restricted transmembrane
protein with a higher receptor density in HCL cells relative to
normal B cells, making it an attractive therapeutic target for the
treatment of this cancer.5 After binding to CD22, the molecule is
internalized, processed and releases its modified protein toxin
that inhibits protein translation, leading to apoptotic cell death.
Moxetumomab pasudotox has been granted Orphan Drug Designation by
the FDA for the treatment of HCL.
About Hairy Cell Leukemia
HCL is a rare, incurable slow-growing leukemia in which the bone
marrow overproduces abnormal B cells or lymphocytes.6 HCL can
result in serious and life-threatening conditions, including
infections, bleeding and anemia.7 Approximately 1,000 people are
diagnosed with HCL in the US each year.8,9,10 While many patients
initially respond to treatment, up to 40% will relapse.11 With no
established standard of care and very few treatments available,
there remains significant unmet medical need for people with
relapsed or refractory HCL.1,2
About the ‘1053’ Phase III Trial
The ‘1053’ trial is a single-arm, multicenter Phase III clinical
trial assessing the efficacy, safety, immunogenicity and
pharmacokinetics of moxetumomab pasudotox monotherapy in patients
with relapsed or refractory HCL who have received at least two
prior therapies. The trial is being conducted in 80 patients across
34 sites in 14 countries.12
About AstraZeneca in Hematology
Leveraging its collective heritage in oncology, AstraZeneca has
established hematology as one of four key oncology disease areas of
focus, and is accelerating development of a broad portfolio of
potential blood cancer treatments. AstraZeneca and Acerta Pharma,
its hematology research and development center of excellence,
recently received FDA approval for CALQUENCE® (acalabrutinib), the
first medicine in this franchise.
About AstraZeneca in Oncology
AstraZeneca has a deep-rooted heritage in Oncology and offers a
quickly-growing portfolio of new medicines that have the potential
to transform patients’ lives and the Company’s future. With at
least six new medicines aimed to be launched between 2014 and 2020
and a broad pipeline of small molecules and biologics in
development, we are committed to advance New Oncology as one of
AstraZeneca’s five Growth Platforms focused on lung, ovarian,
breast and blood cancers. In addition to our core capabilities, we
actively pursue innovative partnerships and investments that
accelerate the delivery of our strategy as illustrated by our
investment in Acerta Pharma in hematology.
By harnessing the power of four scientific platforms –
Immuno-Oncology, Tumor Drivers and Resistance, DNA Damage Response
and Antibody Drug Conjugates – and by championing the development
of personalized combinations, AstraZeneca has the vision to
redefine cancer treatment and one day eliminate cancer as a cause
of death.
About MedImmune
MedImmune is the global biologics research and development arm
of AstraZeneca, a global, innovation-driven biopharmaceutical
business that focuses on the discovery, development and
commercialization of small molecule and biologic prescription
medicines. MedImmune is pioneering innovative research and
exploring novel pathways across Oncology; Respiratory,
Cardiovascular & Metabolic Diseases; and Infection and
Vaccines. The MedImmune headquarters is located in Gaithersburg,
MD, one of AstraZeneca’s three global R&D centers, with
additional sites in Cambridge, UK, and Mountain View, CA. For more
information, please visit www.medimmune.com.
About AstraZeneca
AstraZeneca is a global, science-led biopharmaceutical company
that focuses on the discovery, development and commercialization of
prescription medicines, primarily for the treatment of diseases in
three therapy areas - Oncology, Cardiovascular, Renal &
Metabolic Diseases and Respiratory. The Company also is selectively
active in the areas of autoimmunity, neuroscience and infection.
AstraZeneca operates in over 100 countries and its innovative
medicines are used by millions of patients worldwide.
For more information, please visit www.astrazeneca-us.com and
follow us on Twitter @AstraZenecaUS.
References
1.
López-Rubio, M., Garcia-Marco, J. A.
Current and emerging treatment options for hairy cell leukemia.
OncoTargets and Therapy. 2015; 8: 2147–2156. Available Online.
Accessed January 2018.
2.
National Institutes of Health. Hairy Cell
Leukemia. Available Online. Accessed December 2017.
3.
US Food and Drug Administration. Priority
Review. Available Online. Accessed December 2017.
4.
G Aruna. Immunotoxins: A review of their
use in cancer treatment. J Stem Cells Regn Med. 2006; 1(1): 31-36.
Published online 2006 Dec 26. Available Online. Accessed December
2017.
5.
Park JH, Ross LL. Targeted Immunotherapy
for Hairy cell leukemia. Journ Clin Onc 2012; 30: 1888–1890.
Available Online. Accessed October 2017.
6.
National Institutes of Health. Hairy Cell
Leukemia. Available Online. Accessed December 2017.
7.
Hairy Cell Leukemia Foundation.
Complications. Available Online. Accessed December 2017.
8.
Troussard X, Cornet E. Hairy cell leukemia
2018: Update on diagnosis, risk‐stratification, and treatment. AM J
Hematol. 2017; 92(12):1382-1390. Published online 2017 Nov 7.
Available Online. Accessed March 2018.
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Teras LR, DeSantis CE, Cerhan JR, Morton
LM, Jemal A, Flowers CR. 2016 US Lymphoid Malignancy Statistics by
World Health Organization Subtypes. CA Cancer J Clin. 2016;
66:443-459. Available Online. Accessed March 2018.
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Grever MR, Blachly JS, Andritsos LA. Hairy
cell leukemia: Update on molecular profiling and therapeutic
advances. Blood. 2014; 28(5):197-203. Available Online. Accessed
March 2018.
11.
Hairy Cell Leukemia Foundation. Treatment.
Available Online. Accessed December 2017.
12.
ClinicalTrials.gov. Moxetumomab Pasudotox
for Advanced Hairy Cell Leukemia. Available Online. Accessed
December 2017.
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