GenePHIT: Bayer and AskBio Launch Pioneering Gene Therapy Trial for Heart Failure

Bayer AG and AskBio have initiated the GenePHIT Phase II trial to assess AB-1002, a gene therapy, for treating congestive heart failure. This trial marks a significant advancement in the field of gene therapy and could potentially revolutionize the treatment of congestive heart failure.

Bayer AG and its subsidiary Asklepios BioPharmaceutical (AskBio) announced that they have initiated the Phase II GenePHIT trial for the treatment of congestive heart failure (CHF). This groundbreaking study focuses on evaluating the efficacy and safety of AB-1002, a novel gene therapy, in adults with a specific category of heart failure. It could revolutionize CHF treatment, offering hope to millions suffering from this debilitating condition.

Jude Samulski, Co-Founder and Chief Scientific Officer at AskBio, expressed optimism about the potential of AB-1002 in treating congestive heart failure. “We believe this trial will help determine the potential of AB-1002 as a treatment for one of the world’s most devastating diseases, and we look forward to learning more about this important investigational cardiac gene therapy.”

The GenePHIT trial is an adaptive, double-blind, placebo-controlled, randomized, multicenter study. It aims to test the safety and effectiveness of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy classified as New York Heart Association (NYHA) Class III Heart Failure. This classification refers to patients with marked limitation of physical activity.

The trial plans to enroll between 90 and 150 adults who have been medically stable for at least four weeks, and who continue to experience heart failure symptoms despite following recommended guidelines. These participants will have a left ventricle ejection fraction between 15 and 35%.

The primary efficacy endpoint of the trial, set at 52 weeks, involves a comprehensive evaluation of several clinically meaningful assessments. A modified win ratio method will be used to gauge the effectiveness of the treatment. The trial also includes a four-year long-term follow-up period.

AB-1002, yet to be approved by regulatory authorities, is being manufactured by Viralgen Vector Core, another subsidiary of Bayer and AskBio. 
Viralgen specializes in producing AAV gene therapy treatments, utilizing the Pro10™ platform for enhanced scalability and yield.