GERMANTOWN, Md., Nov. 7, 2019 /PRNewswire/ -- Triple-Gene LLC, a
clinical stage cardiovascular gene therapy company and majority
owned subsidiary of Intrexon Corporation (NASDAQ: XON), today
announced the completion of enrollment and dosing in its Phase 1
trial of INXN-4001, a multigenic investigational therapeutic
candidate under evaluation for the treatment of heart failure, the
leading cause of death worldwide. The Phase 1 open label
study is designed to investigate the safety of INXN-4001 delivered
via Retrograde Coronary Sinus Infusion (RCSI) in patients with an
implanted Left Ventricular Assist Device (LVAD) for mechanical
support of end-stage heart failure, either as a bridge to
transplant or destination therapy (clinical trial identifier:
NCT03409627).
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"We are excited to have reached this important milestone in the
clinical evaluation of INXN-4001 for treatment of end-stage heart
failure," stated Amit Patel, MD, MS,
Co-Founder and Medical Director of Triple‑Gene. "Heart
failure rarely results from a single genetic defect, and while
single gene therapy approaches have been studied, these treatments
may not fully address the causes of the disease. Our unique
multigenic approach is designed to stimulate biological activity
targeting multiple points in the disease progression pathway."
Triple-Gene's investigational therapy uses non-viral delivery of
a constitutively expressed multigenic plasmid designed to express
human S100A1, SDF-1α, and VEGF165 gene products, which affect
progenitor cell recruitment, angiogenesis, and calcium handling,
respectively, and target the underlying molecular mechanisms of
pathological myocardial remodeling. The plasmid therapy is
delivered via RCSI which allows for cardiac-specific delivery to
the ventricle.
"Heart failure is the leading cause of death worldwide and
represents a significant and growing global health problem. Aside
from heart transplant and LVAD, current treatment options for those
patients with end-stage disease are limited," commented
Timothy Henry, MD, FACC, MSCAI,
Medical Director of the Carl and Edyth Lindner Center for Research
and Education at The Christ Hospital and a member of the
Triple-Gene Medical Advisory Board. "The INXN‑4001
investigational therapy represents a biologically-based method
focused on repairing the multiple malfunctions of cardiomyocytes,
and I look forward to seeing the results of this initial safety
study and further exploring the promise of this innovative
treatment approach."
Triple-Gene will present preliminary data from the Phase 1 study
at the American Heart Association Scientific Sessions at the
Pennsylvania Convention Center in
Philadelphia. A poster titled "Safety of First in Human
Triple-Gene Therapy Candidate for Heart Failure Patients" will be
presented on Sunday, November
17th from 3:00 pm - 3:30 pm ET in Zone 4 of
the Science and Technology Hall.
About the Phase 1 Trial of INXN-4001
INXN-4001 is
being evaluated in a Phase I open label study in adult patients
with implanted Left Ventricular Assist Device (LVAD). The
study is designed to investigate the safety and feasibility of
supplemental cardiac expression of S100A1, SDF-1α and VEGF-165 from
a single, multigenic plasmid delivered via Retrograde Coronary
Sinus Infusion (RCSI) in stable patients implanted with a LVAD for
mechanical support of end-stage heart failure. Twelve stable
patients with an implanted LVAD were allocated into 2 cohorts (6
subjects each) to evaluate the safety and feasibility of infusing
80mg of INXN‑4001 in either a 40mL (Cohort 1) or 80mL (Cohort 2)
volume. The primary endpoint of safety and feasibility is
assessed at the 6-month endpoint. Daily activity data are also
collected throughout the study using a wearable biosensor.
Dosing on both Cohorts 1 and 2 has been completed, and patients
continue follow-up per protocol.
About Triple-Gene
Triple-Gene LLC is a clinical stage
gene therapy company focused on advancing targeted, controllable,
and multigenic gene therapies for the treatment of complex
cardiovascular diseases. The Company's lead product is a
non-viral investigational gene therapy candidate that drives
expression of three candidate effector genes involved in heart
failure. Triple-Gene is a majority owned subsidiary of
Intrexon Corporation (NASDAQ: XON) co-founded by Amit Patel, MD, MS, and Thomas D. Reed, PhD, Founder and Chief Science
Officer of Intrexon. Learn more about Triple-Gene at
www.3GTx.com.
About Intrexon Corporation
Intrexon Corporation
(NASDAQ: XON) is Powering the Bioindustrial Revolution with Better
DNA® to create biologically-based products that
improve the quality of life and the health of the planet through
two operating units – Intrexon Health and Intrexon
Bioengineering. Intrexon Health is focused on addressing
unmet medical needs through a diverse spectrum of therapeutic
modalities, including gene and cell therapies, microbial
bioproduction, and regenerative medicine. Intrexon
Bioengineering seeks to address global challenges across food,
agriculture, environmental, energy, and industrial fields by
advancing biologically engineered solutions to improve
sustainability and efficiency. Our integrated technology suite
provides industrial-scale design and development of complex
biological systems delivering unprecedented control, quality,
function, and performance of living cells. We call our synthetic
biology approach Better DNA®, and we invite you to
discover more at www.dna.com or follow us on Twitter at
@Intrexon, on Facebook, and LinkedIn.
Trademarks
Intrexon, Powering the Bioindustrial
Revolution with Better DNA, and Better DNA are trademarks of
Intrexon and/or its affiliates. Other names may be trademarks of
their respective owners.
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 our
plans, objectives and expectations for the development of our
business. 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.
For more information contact:
Investor
Contact:
Steven
Harasym
Vice President,
Investor Relations
Intrexon
Corporation
Tel: +1 (301)
556-9850
investors@dna.com
|
Corporate
Contact:
Marie Rossi,
PhD
Vice President,
Communications
Intrexon
Corporation
Tel: +1 (301)
556-9850
publicrelations@dna.com
|
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SOURCE Triple-Gene LLC