GERMANTOWN, Md., Jan. 6, 2020 /PRNewswire/ -- Precigen, Inc., a
biopharmaceutical company specializing in the development of
innovative gene and cellular therapies to improve the lives of
patients, today announced that the US Food and Drug Administration
(FDA) has granted orphan drug designation (ODD) to PRGN-3006,
a first-in-class investigational therapy using Precigen's
non-viral UltraCAR-T™ therapeutic platform for patients with
relapsed or refractory acute myeloid leukemia (AML) (clinical trial
identifier: NCT03927261). Precigen announced in Q3 2019 that
it had completed enrollment for the first cohort of this
clinical trial and expects an initial data readout in the second
half of 2020.
The FDA grants ODD status to medicines intended for the
treatment, diagnosis or prevention of rare diseases or disorders
that affect fewer than 200,000 people in the US. Medicines that
receive the ODD designation may qualify for a number of incentives
that help to expedite and reduce the cost of development, approval
and commercialization.
"This regulatory designation underscores the critical medical
need for new therapies to treat AML patients. AML is a progressive,
debilitating and often fatal disease with limited treatment
options," said Helen Sabzevari, PhD,
President and CEO of Precigen*. "As the first regulatory
designation for our proprietary UltraCAR-T platform, this orphan
drug designation helps to advance the PRGN-3006 investigational
therapy and provides important incentives and support to deliver
this medicine as rapidly as possible for those patients suffering
from this disease."
PRGN-3006 utilizes Precigen's transformative UltraCAR-T
therapeutic platform, which eliminates ex vivo expansion,
reduces manufacturing time and provides the ability to administer
CAR-T therapy to patients only one day after non-viral gene
transfer at the cancer center. PRGN-3006 UltraCAR-T is a multigenic
CAR-T cell treatment utilizing Precigen's advanced non-viral gene
delivery system to co-express a chimeric antigen receptor,
membrane-bound interleukin‐15 (mbIL15), and a kill switch for
better precision and control in targeting relapsed or
refractory AML and higher risk MDS.
Preclinical data for PRGN-3006 UltraCAR-T™ evaluating
non-viral, multigenic autologous CAR-T cells administered one day
after gene transfer for the treatment of AML and MDS were presented
at the 2019 American Society for Hematology (ASH) annual meeting
and exposition in Orlando,
Florida.
About Acute Myeloid Leukemia (AML)
AML is a cancer
that starts in the bone marrow, but most often moves into the
blood1. Though considered rare, AML is among
the most common types of leukemia in adults2. In 2019,
it was estimated that 21,450
new cases of AML would be diagnosed
in the US2. AML is uncommon before the age of 45 and the
average age of diagnosis is about 682. The prognosis for
patients with AML is poor with an average 5‐year survival rate of
approximately 25 percent overall, and less than a 5 percent 5‐year
survival rate for patients older than 653. Amongst
elderly AML patients (≥ 65 years of age), median survival is short,
ranging from 3.5 months for patients 65 to 74 years of age to 1.4
months for patients ≥ 85 years of age3.
About the FDA Orphan Drug Designation
FDA orphan drug
designation is granted to drugs intended to treat rare diseases or
disorders that affect fewer than 200,000 people in the US.
Medicines that receive the ODD designation may qualify for a number
of incentives that help to expedite and reduce the cost of
development, approval and commercialization, such as grants, clinical design support,
FDA fee waivers, tax incentives, and seven years of market
exclusivity.
Precigen : Advancing Medicine with
PrecisionTM
Precigen is a dedicated discovery and
clinical stage biopharmaceutical company advancing the next
generation of gene and cellular therapies using precision
technology to target the most urgent and intractable diseases in
immuno-oncology, autoimmune disorders, and infectious diseases.
Precigen also follows the science opportunistically in pursuit of
promising programs in emerging therapeutics. Our technologies
enable us to find innovative solutions for affordable
biotherapeutics in a controlled manner. Precigen operates as an
innovation engine progressing a preclinical and clinical pipeline
of well-differentiated unique therapies toward clinical
proof-of-concept and commercialization. Precigen leverages a diverse portfolio of
technology platforms to advance human health. For more information
about Precigen, visit www.precigen.com or follow us on Twitter
@Precigen and LinkedIn.
*Precigen's parent company, Intrexon Corporation (Nasdaq: XON),
announced on January 2,
2020 that it will refocus the company on healthcare,
will change its name to Precigen, Inc., and has appointed Helen Sabzevari, PhD, as President and CEO. The
parent company will continue to hold, among its several health
assets, all of Precigen's discovery and clinical stage technology
and programs.
Precigen's UltraCAR-TTM Therapeutic
Platform
Precigen's UltraCAR-T platform has the potential to
disrupt the CAR-T treatment landscape by increasing patient access
through shortening manufacturing time, decreasing
manufacturing-related costs, and improving outcomes using advanced
approaches for precise tumor targeting and control of the immune
system. The platform brings several key advancements: 1) Non-viral
gene transfer using multigenic vectors for expression of multiple
effector genes leads to better precision and control of tumor
targeting and eliminates the need for virus; 2) Sustained
persistence and desired phenotype of infused UltraCAR-T helps
address T-cell exhaustion, a common issue with current CAR-T
therapies; 3) T-cell control by incorporation of kill switch
technology to potentially improve the safety profile; and 4) Rapid
manufacturing of UltraCAR-T cells using our proprietary non-viral
gene transfer process, which eliminates the need for ex vivo
propagation, thus dramatically reducing wait times for patients
from weeks to fewer than two days.
Safe Harbor Statement
Some of the statements made in
this press release are forward-looking statements. These
forward-looking statements are based upon our current expectations
and projections about future events and generally relate to plans,
objectives and expectations for the development of our business,
including the timing and progress of preclinical and clinical
trials and discovery programs, and the anticipated refocusing and
renaming of Precigen's parent company. Although management believes
that the plans and objectives reflected in or suggested by these
forward-looking statements are reasonable, all forward-looking
statements involve risks and uncertainties and actual future
results may be materially different from the plans, objectives and
expectations expressed in this press release.
1 American Cancer Society. What is Acute Myeloid
Leukemia (AML)? (Accessed January
2020)
2 American Cancer Society. Key Statistics for Acute
Myeloid Leukemia (AML) (Accessed January 2020)
3 Thein, M., et al., Outcome of older patients with
acute myeloid leukemia: an analysis of SEER data over 3 decades.
Cancer, 2013. 119(15): p.2720-7.
Precigen Media Contact:
Donelle M. Gregory
press@precigen.com
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SOURCE Precigen, Inc.
