• Final participant randomized in the GenePHIT Phase 2 clinical trial assessing safety and efficacy of AB-1002 for adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms
• Initial results are expected in the first half of 2027
• Completion of enrollment marks significant milestone in progression of heart failure clinical program and included the largest number of participants to date to receive AB-1002

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG,  announced that the randomization of the last participant was completed earlier this year for GenePHIT, its Phase 2 clinical trial of AB-1002, an investigational gene therapy being developed as a potential treatment for heart failure with reduced ejection fraction (HFrEF).

“Heart failure is a major public health challenge and places a massive strain on healthcare systems around the world,” said Timothy D. Henry, MD, MSCAI, GenePHIT Principal Investigator and Steering Committee Member. “Prevalence is increasing, and the need for innovative therapies has never been greater. Completing enrollment in this trial brings us another step closer to evaluating a potential treatment strategy for heart failure with reduced ejection fraction.”

GenePHIT includes 173 participants, and the completion of enrollment marks a significant milestone in the development of AB-1002, an investigational gene therapy administered via a single intracoronary infusion on top of the standard of care in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms.¹ Initial trial results are expected in the first half of 2027.

“We are pleased to have randomized the last participant in our GenePHIT trial,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “AskBio’s Phase 2 heart failure program investigates the efficacy and safety of AB-1002, which is designed to potentially mitigate the symptoms of heart failure with reduced ejection fraction and improve survival rates and quality of life. The data we receive from the participants will help us better understand the potential of our investigational gene therapy in an area of significant medical need.”

An estimated 64 million people worldwide are living with heart failure, and despite advances in treatment, mortality and morbidity remain very high.^2,3 The completion of enrollment represents a significant step in potentially bringing a new treatment to those who need it most.

AskBio explored AB-1002 in a Phase 1 non-randomized, sequential dose escalation trial for participants with NYHA Class III non-ischemic HFrEF.⁴ Twelve-month data were published online in Nature Medicine in October 2025 and in print in November 2025.⁵ These data had previously been presented in May 2025 as a late-breaker presentation at the European Society of Cardiology Heart Failure Meeting.⁶

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