AskBio Completes Enrollment in Phase 2 Gene Therapy Trial for Heart Failure

AskBio查看公司主页 Inc., a gene therapy subsidiary of Bayer AG查看公司主页, has completed enrollment in its pivotal Phase 2 clinical trial of AB-1002查看药品详情, an investigational gene therapy for heart failure with reduced ejection fraction (HFrEF). The GenePHIT trial enrolled 173 participants across 46 locations in 12 countries, marking the largest cohort to date to receive the experimental treatment.

The completion represents a significant milestone in addressing a major unmet medical need, as an estimated 64 million people worldwide are living with heart failure, with mortality and morbidity remaining very high despite treatment advances.

Novel Gene Therapy Approach

AB-1002查看药品详情 is administered as a single intracoronary infusion directly to the heart, where it promotes production of a modified version (I-1c) of the naturally occurring protein inhibitor-1. The therapy is designed to block the action of protein phosphatase 1, which is linked to heart failure pathophysiology.

"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."

Trial Design and Patient Population

GenePHIT is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial evaluating the safety and efficacy of AB-1002查看药品详情 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms. The trial is designed to evaluate cardiovascular-related deaths and changes from baseline in NYHA classification, left ventricular ejection fraction, and six-minute walking distance.

The therapy is administered on top of standard of care treatment, potentially offering an additional therapeutic option for patients with advanced heart failure symptoms.

Promising Phase 1 Foundation

The Phase 2 advancement builds on encouraging Phase 1 data published in Nature Medicine in October 2025. The earlier non-randomized, sequential dose escalation trial evaluated AB-1002查看药品详情 in participants with NYHA Class III non-ischemic HFrEF, with twelve-month data demonstrating preliminary safety and efficacy signals.

"AskBio查看公司主页's Phase 2 heart failure program investigates the efficacy and safety of AB-1002查看药品详情, which is designed to potentially mitigate the symptoms of heart failure with reduced ejection fraction and improve survival rates and quality of life," said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio.

Timeline and Clinical Impact

Initial results from the GenePHIT trial are expected in the first half of 2027. The completion of enrollment represents a significant step toward potentially bringing a new treatment option to patients with advanced heart failure, a condition characterized by the heart's inability to pump blood efficiently enough to meet the body's oxygen needs.

Heart failure symptoms include shortness of breath, swelling in the legs and ankles caused by fluid retention, and fatigue. The condition causes congestion in the body's tissues and significantly impacts quality of life.

AskBio查看公司主页 maintains a broader clinical-stage pipeline that includes investigational therapeutics for limb-girdle muscular dystrophy, multiple system atrophy, Parkinson's disease, and Pompe disease, positioning the company as a leader in gene therapy development across multiple therapeutic areas.