Phase II Gene Therapy Trial for CHF

This European milestone follows the announcement of the first participant randomized in the US arm of GenePHIT in 2024.

GenePHIT is an adaptive, double-blind, placebo-controlled, randomized, multicenter trial to evaluate the safety and efficacy of a single intracoronary infusion of AB 1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms who have been medically stable for at least four weeks (NCT05598333),. This clinical milestone in the development of AB 1002 potentially brings this investigational one-time gene therapy one step closer to treating patients in Europe with high unmet medical need.

GenePHIT will include between 90 and 150 adults with heart failure with reduced ejection fraction (HFrEF) between 15 and 35 percent who continue to suffer from symptoms despite guideline-recommended therapy. The study is enrolling participants across clinical centers in the United States and Europe (Austria, Germany, Hungary, Netherlands, Spain, UK). The successful randomization of the first participant in Europe, which happened in Spain, is an important landmark in the Phase II  GenePHIT clinical trial, which will include the largest number of participants to receive AB-1002 to date.

“Cardiovascular disease is the most common cause of death in Europe, claiming nearly four million lives each year, and it accounts for 45 percent of all deaths in females and 39 percent of all deaths in males,” said Dr, Timothy D. Henry, GenePHIT Principal Investigator and Steering Committee Member. “As society ages, the burden heart failure places on individuals and healthcare systems will only increase unless a solution is found. Today’s announcement is important as it represents meaningful progress in the development of a potentially disease-modifying therapy."

“The potential of gene therapy to address the intracellular abnormalities that characterize heart failure is immense, and we are excited to have achieved this critical milestone for the GenePHIT study in Europe,” said Dr.. Canwen Jiang  Chief Development Officer and Chief Medical Officer, AskBio. “This news demonstrates AskBio’s ability to advance AB 1002 gene therapy for the potential treatment of congestive heart failure, which is deadly and devastating for affected patients and has a significant impact on their loved ones.”