AskBio, a gene therapy company and a wholly owned subsidiary of Bayer AG, has completed patient enrollment for its Phase II clinical trial, GenePHIT, evaluating the investigational gene therapy AB-1002 for the treatment of heart failure with reduced ejection fraction (HFrEF).

The study has enrolled 173 participants, marking a key milestone in the development of AB-1002, a one-time gene therapy administered via intracoronary infusion alongside standard treatment. The therapy is being tested in adult patients with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms. Initial results from the trial are expected in the first half of 2027.

Experts highlight the growing burden of heart failure globally, with an estimated 64 million people affected worldwide. Despite advancements in treatment, the condition continues to carry high mortality and morbidity, underscoring the need for innovative therapies.

AB-1002 is designed to enhance cardiac function by promoting the production of a modified protein that targets pathways associated with heart failure. Earlier Phase I data, published in Nature Medicine, indicated promising safety and efficacy signals, supporting further clinical evaluation.

The GenePHIT trial is a randomised, double-blind, placebo-controlled, multi-centre study conducted across 46 sites in North America and Europe. It aims to assess both safety and efficacy, including outcomes such as cardiovascular-related deaths, improvements in heart function, and patient mobility.

Company officials stated that the completion of enrollment brings them closer to determining the therapy’s potential to improve survival rates and quality of life for patients suffering from this chronic and life-threatening condition.

AB-1002 remains an investigational therapy and has not yet been approved by regulatory authorities.