Renova Therapeutics announces successful End-of-Phase 2 meeting with FDA for RT-100 AC6 gene transfer therapy
November 1, 2016
Phase 3 trial in patients with heart failure and reduced ejection fraction to commence in H2 2017 across 60 medical centers in the United States
San Diego, CA – Renova™ Therapeutics, a biotechnology company developing gene therapy treatments for congestive heart failure and type 2 diabetes, announced today that it has completed an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for its lead gene therapy product candidate, RT-100 AC6 gene transfer for the treatment of patients with heart failure and reduced ejection fraction.
The FDA meeting – for which Renova Therapeutics submitted AC6 gene transfer Phase 2 trial results along with a proposed Phase 3 clinical trial protocol – was the next step in the company’s efforts to propel forward the development of this novel gene therapy for heart failure patients.
“Renova’s FDA interactions, which were quite constructive, reinforce our plans to develop RT-100 for patients living with heart failure,” said Jack W. Reich, Ph.D., CEO and Co-founder of Renova Therapeutics. “We look forward to advancing to pivotal trials and eventually bringing this life-changing therapeutic to market.”
Renova Therapeutics will proceed with conducting a randomized, placebo-controlled, double-blind multicenter Phase 3 trial of a one-time intracoronary administration of adenovirus 5 encoding human AC6 (known as RT-100) for patients with heart failure and reduced left ventricular ejection fraction. The primary endpoint will be the reduction of the event rate of all – first and repeat – heart failure hospitalizations occurring after RT-100 intracoronary injection from baseline to 12 months (the study period). Patient safety will continue to be monitored during a follow-up period following completion of the study.
This pivotal trial is expected to commence in the second half of 2017 across 60 medical centers in the United States. Additional details on the clinical trial – including patient enrollment information – will be available next year.
About RT-100 AC6 gene transfer
Adenylyl cyclase type 6 (AC6) is a protein found in cardiac muscle cells that regulates heart function and appears to be down-regulated in heart failure patients. RT-100 AC6 gene transfer involves infusing an inactivated adenovirus vector encoding human AC6 (Ad5.hAC6) into the arteries that feed the heart during cardiac catheterization, a commonly performed procedure.
A randomized, double-blind, placebo-controlled Phase 2 trial assessed the safety of five doses of Ad5.hAC6 versus placebo in 56 heart failure patients who were studied for up to one year at seven medical centers throughout the United States. Forty-two participants received Ad5.hAC6; 14 received a placebo.
The trial demonstrated that two endpoints showed differences between the two highest doses of AC6 (combined) versus placebo:
- AC6 gene transfer increased left ventricular peak –dP/dt (p=0.029). This is a direct measure of the heart’s ability to fill.
- AC6 gene transfer increased left ventricular ejection fraction in participants with non-ischemic heart failure (p=0.024). Non-ischemic heart failure is a type of heart failure not associated with extensive coronary artery disease.
In addition, symptoms of heart failure were reduced 12 weeks after therapy in Ad5.hAC6 participants (p=0.0005) but not in placebo subjects. For all trial participants, there were no differences in rates of serious adverse events between the AC6 and placebo groups.
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.1 It is the most common cause for emergency hospital admissions in patients 65 and older.
1Go 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.