INXN-4001 / Precigen - LARVOL DELTA

Novel INXN-4001 Triple Effector Plasmid in Heart Failure (clinicaltrials.gov) - P1; N=12; Completed; Sponsor: Triple-Gene, LLC; Active, not recruiting ➔ Completed; Trial completion date: Nov 2020 ➔ Aug 2020

Clinical • Trial completion • Trial completion date • Cardiovascular • Congestive Heart Failure • Heart Failure

"Make sure to speak to @Intrexon subsidiary @Precigen on INXN-4001 https://t.co/A6VuKOJYNO" (@Biotech2050)

Safety of First in Human Triple-Gene Therapy Candidate for Heart Failure Patients (AHA 2019) - "The INXN-4001 investigational new drug is a non-viral, multigenic, triple effector plasmid designed with a self-cleaving linker to constitutively express human SDF-1α, S100A1, and VEGF-165 to affect progenitor cell recruitment, calcium metabolism, and angiogenesis... This is the first in-human experience with a triple gene construct."

Clinical • P1 data • VEGFA

"Check out INXN-4001 at #AHA19" (@Biotech2050)

Novel INXN-4001 Triple Effector Plasmid in Heart Failure (clinicaltrials.gov) - P1; N=12; Active, not recruiting; Sponsor: Triple-Gene, LLC; Recruiting ➔ Active, not recruiting

Enrollment closed

Novel INXN-4001 Triple Effector Plasmid in Heart Failure (clinicaltrials.gov) - P1; N=12; Recruiting; Sponsor: Triple-Gene, LLC; Trial completion date: Jun 2020 ➔ Sep 2020; Trial primary completion date: Dec 2019 ➔ Mar 2020

Clinical • Trial completion date • Trial primary completion date

Novel INXN-4001 Triple Effector Plasmid in Heart Failure (clinicaltrials.gov) - P1; N=12; Recruiting; Sponsor: Xogenex, LLC; Trial completion date: Dec 2019 ➔ Jun 2020; Trial primary completion date: Apr 2019 ➔ Dec 2019

Clinical • Trial completion date • Trial primary completion date

Intrexon and Xogenex present at American Society of Gene and Cell Therapy Annual Meeting (PRNewswire) - "Intrexon Corporation...and its majority owned Intrexon Health subsidiary Xogenex, today announced that four abstracts will be presented as posters at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will be held April 29 - May 2, 2019, at the Washington Hilton in Washington, D.C....Preclinical studies described in these posters set the basis for demonstrating safety and clinically relevant findings with INXN-4001 and provided support for translation into the clinical setting. Evaluation of the Phase 1 trial data for three patients reflecting the patients' status six months after being given the INXN-4001 appears to indicate that the drug material and the delivery process were both well-tolerated."

P1 data • Preclinical

Minimally Invasive Novel Triple Effector Gene Therapy for Adriamycin Cardiomyopathy (ASGCT 2019) - "Most treatments for Adriamycin (ADM) induced cardiomyopathy have failed as there are multiple underlying mechanisms contributing to heart deterioration. Echocardiographic measurements of cardiac structure and function were performed at baseline (before ADM treatments), 3 weeks after ADM treatments, 2 weeks after UTMD-based delivery of the two test plasmids, as well as 4 weeks after UTMD. Echocardiographic measurements performed at 4 weeks post UTMD delivery demonstrated that UTMD-INXN-4001 gene therapy significantly improved fractional shortening index, LV posterior wall diameter, and Interventricular Septal Thickness at Diastole (IVSd) compared to treatment with plasmid control treatments."

Human Cardiac iPSC Evaluation of Novel Triple Effector Gene Therapy for Dilated Cardiomyopathy (ASGCT 2019) - "INXN-4001 demonstrated the most significant improvement, restoring beat rate, contraction duration and contraction rate of DCM iPSC-CM to healthy levels without issues of toxicity."

Retrograde Delivery of Novel Triple Effector pDNA Gene Therapy for Heart Failure (ASGCT 2019) - "Coronary sinus delivery of INXN-4001 was safe and improved myocardial function in a large animal ischemic heart failure model."

First Human Experience with Novel Triple Effector Gene Therapy for Heart Failure (ASGCT 2019) - "...Our goal is to evaluate INXN-4001, a triple effector gene therapy, which impacts angiogenesis, cell homing and contractility in a clinical setting...Triple effector gene therapy is demonstrating safety thus far with promising efficacy signals. Further follow-up is ongoing to evaluate clinical endpoints."