FDA grants fast track designation to Renova Therapeutics’ RT-100 AC6 gene transfer for the treatment of heart failure

San Diego, Calif. — 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).