AskBio Begins GenePHIT Phase 2 Trial for AB-1002 in Heart Failure Treatment

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the first participant has been randomized in Europe in GenePHIT (Gene PHosphatase Inhibition Therapy), a phase 2 clinical trial of AB-1002 for the treatment of congestive heart failure (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. 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 per cent 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 2 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 Timothy D. Henry, MD, MSCAI, 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 Canwen Jiang MD, PhD, chief development officer and chief medical officer, AskBio. “Today’s 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.”

AB-1002 is an investigational gene therapy that has been approved for experimental clinical trials only and has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

GenePHIT is a phase 2 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 NYHA Class III heart failure symptoms.

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 may include shortness of breath, swelling in the legs and ankles caused by fluid retention, and fatigue.  More than 64 million people worldwide are living with congestive heart failure.

AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programmes across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease.

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