‘Human Gene Therapy’ publishes review of randomized clinical trials of gene transfer for heart failure with reduced ejection fraction
April 11, 2017
Review finds that gene transfer in patients with heart failure and reduced ejection fraction appears to be a safe treatment method, paving the way for further progress in the field of gene therapy
San Diego, CA – Renova™ Therapeutics, a biotechnology company developing gene and peptide therapies for cardiovascular and metabolic diseases, today announced its co-founder’s online publication of an article in Human Gene Therapy. The article reviews four published clinical trials using gene transfer in patients with heart failure with reduced ejection fraction (HFrEF). The review finds that, while efficacy varied among the clinical trials, gene transfer was not associated with an increased rate of adverse events, supporting further investigations using this treatment method in the HFrEF patient population.
The review, which will be published in the May 2017 print issue of Human Gene Therapy, was co-authored by Dr. H. Kirk Hammond and Dr. William Penny, both Professors of Medicine at the University of California – San Diego and cardiologists at the Veterans Affairs San Diego Healthcare System. Dr. Hammond is a co-founder of Renova Therapeutics and a consultant to the company.
The authors reviewed safety and efficacy results of four trials utilizing three gene transfer strategies: intracoronary delivery of adeno-associated virus type 1 encoding sarcoplasmic reticulum Ca2+-ATPase (AAV1.SERCA2a; CUPID 1 and CUPID 2); endocardial injection of a plasmid encoding stromal cell-derived factor-1 (pSDF-; STOP-HF; and intracoronary delivery of adenovirus-5 encoding human adenylyl cyclase type 6 (Ad5.hAC6).
Results of the AC6 clinical trial indicate that, through a one-time administration, AC6 gene transfer safely increased heart function beyond optimal heart failure therapy. This Phase 2 trial was funded by the National Institutes of Health (NIH), the Gene Therapy Resource Program and Renova Therapeutics, via an NIH public-private partnership.
“With safety results indistinguishable from placebo, we can confidently advance our gene transfer clinical trials,” says Dr. Jack W. Reich, CEO and Co-founder of Renova Therapeutics. “The potential therapeutic value of gene transfer in heart failure patients is tremendous.”
Renova Therapeutics will conduct a randomized, placebo-controlled, double-blind multicenter Phase 3 trial of intracoronary delivery of Ad5.hAC6 (known as RT-100) to evaluate safety and efficacy. The primary efficacy endpoint will be heart failure hospitalization rate 12 months after randomization. This pivotal trial is expected to commence this year at 60 medical centers in the United States.
About heart failure
Heart failure is a chronic disease characterized by the inability of the heart to pump sufficient blood to meet the body’s demands. It is a progressive and fatal chronic condition, and symptoms worsen over time. Heart failure afflicts more than 28 million people globally and is the only cardiovascular disease that is increasing in prevalence. In the United States, it is the most common cause for emergency hospital admissions in patients 65 and older.
- Dr. William F Penny and Dr. H. Kirk Hammond. Randomized Clinical Trials of Gene Transfer for Heart Failure with Reduced Ejection Fraction. Human Gene Therapy. March 2017, ahead of print. doi:10.1089/hum.2016.166.
- Go AS, Mozaffarian D, Roger VL, et al. Heart disease and stroke statistics–2013 update: a report from the American Heart Association. Circulation. 2013;127:e6–e245.