Triple-Gene Announces Completion of Enrollment and Dosing in Phase 1 Trial of INXN‑4001, First Multigenic Investigational Therapeutic Candidate for Treatment of Heart Failure

– Company to Present Preliminary Data at American Heart Association Scientific Sessions –

GERMANTOWN, Md., Nov. 7, 2019 — Triple-Gene LLC, a clinical stage cardiovascular gene therapy company and majority owned subsidiary of Intrexon Corporation, 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).

"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

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 or follow us on Twitter at @Intrexon, on Facebook, and LinkedIn.

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.