AskBio Completes Final Randomization In Phase 2 GenePHIT Trial For AB-1002, Advancing New Hope For HFrEF Patients

AskBio Inc., a gene therapy company that operates independently as a subsidiary of Bayer AG, announced that it has completed the randomization of the final participant in GenePHIT, its Phase 2 clinical trial evaluating AB-1002. This investigational gene therapy is being studied as a potential treatment for heart failure with reduced ejection fraction (HFrEF). The last participant was randomized earlier this year, marking a significant step forward in the program’s progress.

According to Timothy D. Henry, MD, MSCAI, who serves as the Principal Investigator and a member of the Steering Committee for GenePHIT, heart failure remains a major global health burden. The number of people affected continues to rise, placing increasing pressure on healthcare systems around the world. He noted that completing enrollment brings the study team closer to evaluating a promising new therapeutic approach for patients living with HFrEF, who currently have limited options.

The GenePHIT trial includes 173 participants. Completing enrollment is an important milestone because it allows the research team to fully assess AB-1002, which is administered as a single intracoronary infusion in addition to standard-of-care treatments. The therapy is being evaluated in adults 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.

Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio, said the organization is pleased to have reached the final randomization milestone. She explained that the Phase 2 program is designed to study the potential of AB-1002 to reduce symptoms of HFrEF, improve survival outcomes, and enhance quality of life for patients. The information collected from participants will be critical in understanding how this investigational therapy may address a significant unmet medical need.

Heart failure affects an estimated 64 million people worldwide, and despite improvements in available treatments, both mortality and morbidity remain high. This underscores the importance of developing new, innovative therapies and highlights the value of moving AB-1002 to the next stage of evaluation.

AskBio has previously studied AB-1002 in a Phase 1 non-randomized, sequential dose-escalation trial involving participants with NYHA Class III non-ischemic HFrEF. Twelve-month data from this study were published online in Nature Medicine in October 2025 and later appeared in print in November 2025. These findings had also been shared earlier that year, in May 2025, during a late-breaker presentation at the European Society of Cardiology Heart Failure Meeting. 

It is important to note that AB-1002 is still an investigational gene therapy. It has not been approved by any regulatory authority, and its safety and effectiveness have not yet been fully evaluated.