Intracoronary Gene Therapy Fast Tracked for Congestive Heart Failure

The Food and Drug Administration (FDA) has granted Fast Track designation to AB-1002 for the treatment of congestive heart failure (CHF).

AB-1002 is an investigational one-time gene therapy that promotes increased production of protein inhibitor 1. It is designed to inhibit the action of protein phosphatase 1, which has been linked to heart failure. 

Preliminary results from a phase 1 study (ClinicalTrials.gov Identifier: NCT04179643) showed clinically meaningful improvements in left ventricular ejection fraction, NYHA Functional Class, Minnesota Living with Heart Failure Questionnaire, cardiopulmonary exercise test, and 6-minute walk test at 12 months following intracoronary infusion of AB-1002.

The Company is currently enrolling participants in the phase 2 GenePHIT trial (ClinicalTrials.gov Identifier: NCT05598333) to evaluate the efficacy and safety of a single dose of AB-1002 administered via antegrade intracoronary artery infusion in patients with nonischemic cardiomyopathy and New York Heart Association (NYHA) Class III symptoms of heart failure.

“The Fast Track Designation for AB-1002 emphasizes the need to rapidly advance new therapeutic modalities such as gene therapy for people living with congestive heart failure,” said Christian Rommel, PhD, Head of Research and Development at Bayer’s Pharmaceuticals Division. “This designation underpins the potential of AB-1002 to address currently high unmet medical need, and we are excited about the opportunity to accelerate its development.”