FDA fast tracks investigational gene therapy for heart failure

The US Food and Drug Administration (FDA) has granted Fast Track designation to invesigational gene therapy AB-1002 for treatment of congestive heart failure (CHF).AB-1002 is an investigational one-time gene therapy designed to inhibit the action of protein phosphatase 1, which has been linked to heart failure.

Inhibiting the function of this protein, which is linked to congestive heart failure (CHF), could potentially lead to a therapeutic effect on the heart.

“The FDA Fast Track Designation for AB-1002 is an important accomplishment for the clinical development of this program and highlights our goal of potentially bringing effective treatments to patients with advanced congestive heart failure,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We look forward to completing our Phase II GenePHIT clinical trial, which is currently enrolling patients with severe heart failure, and are committed to exploring the full potential of AB-1002 for the treatment of this devastating disease.”

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill unmet medical needs. The purpose of the Program is to get important new therapeutics to patients earlier. Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.

“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.”

AB-1002 is an investigational gene therapy that has not received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AskBio is currently enrolling patients in the Phase II GenePHIT (Gene PHosphatase Inhibition Therapy) trial of AB-1002 (also known as NAN-101) for the treatment of CHF.

About GenePHIT

GenePHIT is a Phase II adaptive, double-blinded, placebo-controlled, randomized, multi-center trial to evaluate the safety and efficacy of the one-time administration of AB-1002, via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms. Subjects are randomized into one of three treatment groups in a 1:1:1 fashion to either low dose, high dose, or placebo. Primary outcome measures include cardiovascular related death and change from baseline in NYHA classification, left ventricular ejection fraction (LVEF), peak oxygen uptake (pVO2), and Six Minute Walk Test (6MWT). For more information, please visit clinicaltrials.gov or visit askbio.com.

About Congestive Heart Failure

Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body’s needs, including providing sufficient oxygen to the organs. Congestive heart failure results in the slowing of the blood flow out of the heart, which causes the blood returning to the heart through the veins to back up. This causes congestion in the body’s tissues. Symptoms include swelling in the legs and ankles. Sometimes, fluid collects in the lungs and interferes with breathing. Approximately 26 million people worldwide are living with congestive heart failure.