SAN DIEGO, Jan. 5, 2017 /PRNewswire-USNewswire/ -- Renova™ Therapeutics, a biotechnology company developing gene therapy treatments for congestive heart failure and type 2 diabetes, announced today that it has selected Worldwide Clinical Trials as its Clinical Research Organization (CRO) for a Phase 3 study of RT-100 AC6 gene transfer for the treatment of patients with reduced ejection fraction heart failure.
Selecting a CRO for its lead gene therapy product candidate is a key milestone for Renova Therapeutics, which completed a successful End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for RT-100 last year.
"We're thrilled to bring Worldwide Clinical Trials on board to help us conduct the Phase 3 trial of RT-100," said Jack W. Reich, Ph.D., CEO and Co-founder of Renova Therapeutics. "Their expertise and capabilities in managing pivotal trials in heart failure are a tremendous asset to us."'
With support from Worldwide Clinical Trials, 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.
"Upon learning that we were selected to partner with Renova to further explore its ground-breaking RT-100 AC6 gene transfer therapy, there was a buzz of excitement across Worldwide," said Peter Benton, President and Chief Operating Officer at Worldwide Clinical Trials. "Knowing that this therapy could bring hope for millions of people at risk and suffering from heart failure, our medical and scientific teams are eager to be a part of Renova's clinical development team. We're honored to be partnering with Renova and helping them take this next step with RT-100."
Worldwide Clinical Trials experts will work closely with Dr. Richard McCloskey, MD, Executive Vice President of Clinical Development, and Jennifer Spinella, Senior Vice President of Regulatory Affairs and Quality Assurance, on executing this Phase 3 study.
This pivotal trial is expected to commence in the second half of 2017, with 536 patients to be enrolled across 60 medical centers in the United States.
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.
About Renova Therapeutics Renova Therapeutics is developing definitive, one-time gene therapies and peptide infusion treatments to restore the health of people suffering from chronic diseases. The first indications the company is pursuing are gene therapy treatments for congestive heart failure (CHF) and type 2 diabetes, two of the most common and devastating chronic diseases in the world. The company's lead product, RT-100, is a treatment that delivers a therapeutic gene directly to the heart during a routine outpatient procedure and has the potential to increase heart function in millions of patients with CHF. The company's product pipeline also includes a groundbreaking gene therapy in preclinical stage for sufferers of type 2 diabetes, as well as a peptide infusion therapy for the treatment of acute decompensated heart failure. Renova Therapeutics was founded in 2009 and is led by an experienced management team in biopharmaceuticals and gene therapy. For additional information about the company, please visit www.renovatherapeutics.com.
About Worldwide Clinical Trials Worldwide Clinical Trials employs more than 1,400 professionals around the world, with offices in North and South America, Eastern and Western Europe, Russia and Asia. One of the world's leading, full-service contract research organizations (CROs), we partner with sponsors in the pharmaceutical and biotechnology industries to deliver fully integrated clinical development and bioanalytical services, extending from first-in-human through phase IV studies. Grounded in medicine and science, we help sponsors move from discovery into clinical development and commercialization across a range of therapeutic areas, including neuroscience, cardiovascular diseases, immune-mediated inflammatory disorders (IMID), and rare diseases. For more information, visit www.worldwide.com.
References 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.