US FDA Grants Fast Track Designation to Renova Therapeutics' RT-100 AC6 Gene Transfer for the Treatment of Heart Failure

FLOURISH Phase 3 clinical trial in patients with heart failure and reduced ejection fraction (HFrEF) to begin in Q1 2018

SAN DIEGO, CA -- Renova™ Therapeutics, a biotechnology company developing gene and peptide-based treatments for cardiovascular and metabolic diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company's lead product candidate, RT-100 AC6 gene transfer (Ad5.hAC6), for the treatment of heart failure with reduced ejection fraction (HFrEF).

The FDA's Fast Track program is designed to expedite the development and review of drugs and biologics with the potential to treat serious or life-threatening conditions and address an unmet medical need. A Fast Track designation enables more frequent communication with the FDA throughout a product candidate's development and review process, ensuring questions are answered and issues are resolved quickly. The designation allows for Rolling Review of a New Drug Application (NDA) or Biologic License Application (BLA), meaning a company can submit individual sections of its application to the FDA as they are completed, rather than waiting until every section is completed to submit. The designation also provides eligibility for Priority Review, if relevant criteria are met – potentially resulting in quicker access to patients.

"We're pleased to work closely with the FDA to advance rapidly the development of RT-100 to treat heart failure patients," said Dr. Jack W. Reich, CEO and Co-founder of Renova Therapeutics. "Despite some medical advancements, heart failure remains a progressive and fatal disease affecting millions of people worldwide. That's why we look forward to initiating a Phase 3 trial with RT-100 early next year, with hopes of eventually submitting a BLA and ultimately bringing this life-changing therapeutic to patients as quickly as possible."

RT-100 AC6 gene transfer involves infusing an inactivated adenovirus vector encoding human adenylyl cyclase type 6 (Ad5.hAC6) into the arteries that feed the heart during cardiac catheterization, a commonly performed procedure. AC6 is a protein found in heart muscle cells that regulates heart function and appears to be down-regulated in heart failure patients. Results of a Phase 2 clinical trial indicate that, through a one-time administration, RT-100 safely increased heart function beyond optimal heart failure therapy. The treatment also lowered the heart failure hospitalization rate at 12 months, which will be the primary endpoint in the program's upcoming Phase 3 trial.

Renova Therapeutics will conduct a randomized, placebo-controlled, double-blind multicenter Phase 3 trial of intracoronary delivery of RT-100 to evaluate safety and efficacy. This pivotal trial – known as FLOURISH (Heart Failure with Reduced Left Ventricular Ejection Fraction: One-time Gene Transfer Using RT-100 – Intracoronary Administration of Adenovirus 5 encoding Human AC6) – is expected to commence in Q1 2018 in the United States (ClinicalTrials.gov Identifier: NCT03360448).

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 In the United States, it is the most common cause of 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 heart failure and type 2 diabetes, two of the most common and devastating chronic diseases in the world. The company's lead product candidate, 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 heart failure. A Phase 3 trial – FLOURISH – of RT-100 is expected to commence in Q1 2018 (ClinicalTrials.gov Identifier: NCT03360448). 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.

References

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.

SOURCE Renova Therapeutics