– Acute myeloid leukemia (AML) is
among the most common types of leukemia in adults –
– PRGN-3006 UltraCAR-T
previously received orphan drug designation
(ODD) in patients with AML by the US Food and Drug
Administration (FDA) –
– PRGN-3006 UltraCAR-T has
demonstrated a favorable safety profile to date with no
dose-limiting toxicities or neurotoxicity, and has demonstrated
dose-dependent in vivo expansion and durable persistence
–
GERMANTOWN, Md., April 4,
2022 /PRNewswire/ -- Precigen, Inc. (Nasdaq:
PGEN), a biopharmaceutical company specializing in the development
of innovative gene and cell therapies to improve the lives of
patients, today announced that the FDA has granted Fast Track
designation for PRGN-3006 UltraCAR-T® in patients with
relapsed or refractory (r/r) AML (clinical trial identifier:
NCT03927261). PRGN-3006 was previously granted FDA Orphan Drug
Designation.
PRGN-3006 UltraCAR-T is a multigenic autologous chimeric antigen
receptor (CAR)-T cell treatment utilizing Precigen's non-viral
Sleeping Beauty system to simultaneously express a CAR
specifically targeting CD33, which is over expressed on AML blasts;
membrane bound IL-15 for enhanced in vivo expansion and
persistence; and a kill switch to conditionally eliminate CAR-T
cells for an improved safety profile.
Precigen's UltraCAR-T platform is designed to overcome
limitations of currently available CAR-T therapies by utilizing an
advanced overnight non-viral gene delivery manufacturing process at
a medical center's cGMP facility without the need for ex
vivo expansion. Current CAR-T cell therapies are limited due
to, inter alia, the prolonged interval between apheresis to
product infusion and an exhausted phenotype of T cells resulting
from lengthy ex vivo expansion. UltraCAR-T cells for the
PRGN-3006 study are manufactured overnight using Precigen's
proprietary UltraPorator™ system.
"We are very pleased to receive the FDA's Fast Track
designation, which facilitates development and expedites the review
process of drugs that address serious conditions and high unmet
medical needs," said Helen
Sabzevari, PhD, President and CEO of Precigen. "AML is a
rapidly progressing disease with a very poor prognosis. The Fast
Track designation will help facilitate the timely development of
this program and we look forward to working more closely with the
FDA to potentially bring this new and highly differentiated
overnight UltraCAR-T therapy to patients."
About AML
AML is a cancer that starts in the bone
marrow, but most often moves into the blood.1 Though
considered rare, AML is among the most common types of
leukemia in adults.2 In 2019, it was estimated that
21,450 new cases of AML would be diagnosed in the US.2
AML is uncommon before the age of 45 and the average age of
diagnosis is about 68.2 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 65.3 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 age.3
Precigen: Advancing Medicine with Precision™
Precigen
(Nasdaq: PGEN) is a dedicated discovery and clinical stage
biopharmaceutical company advancing the next generation of gene and
cell therapies using precision technology to target the most urgent
and intractable diseases in our core therapeutic areas of
immuno-oncology, autoimmune disorders, and infectious diseases. 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 therapies toward clinical proof-of-concept
and commercialization. For more information about Precigen, visit
www.precigen.com or follow us on Twitter @Precigen,
LinkedIn or YouTube.
Trademarks
Precigen, UltraCAR-T, UltraPorator and
Advancing Medicine with Precision are trademarks
of Precigen and/or its affiliates. Other names may be
trademarks of their respective owners.
Cautionary Statement Regarding Forward-Looking
Statements
Some of the statements made in this press release
are forward-looking statements. These forward-looking statements
are based upon the Company's current expectations and projections
about future events and generally relate to plans, objectives, and
expectations for the development of the Company's business,
including the timing and progress of preclinical studies, clinical
trials, discovery programs and related milestones, the promise of
the Company's portfolio of therapies, and in particular its CAR-T
and AdenoVerse therapies. 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, including the
possibility that the timeline for the Company's clinical trials
might be impacted by the COVID-19 pandemic, and actual future
results may be materially different from the plans, objectives and
expectations expressed in this press release. The Company has no
obligation to provide any updates to these forward-looking
statements even if its expectations change. All forward-looking
statements are expressly qualified in their entirety by this
cautionary statement. For further information on potential risks
and uncertainties, and other important factors, any of which could
cause the Company's actual results to differ from those contained
in the forward-looking statements, see the section entitled "Risk
Factors" in the Company's most recent Annual Report on Form 10-K
and subsequent reports filed with the Securities and Exchange
Commission.
References
1 American Cancer Society. What
is Acute Myeloid Leukemia (AML)?
2 American Cancer Society. Key Statistics for Acute
Myeloid Leukemia (AML)
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
Investor Contact:
Steven Harasym
Vice President, Investor Relations
Tel: +1 (301) 556-9850
investors@precigen.com
Media Contacts:
Donelle M.
Gregory
press@precigen.com
Glenn Silver
Lazar-FINN Partners
glenn.silver@finnpartners.com
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SOURCE Precigen, Inc.