Bayer announces start of Phase 2 GenePHIT trial for AB-1002

Bayer AG and its subsidiary Asklepios BioPharmaceutical, Inc. (AskBio), have achieved a significant milestone in the fight against congestive heart failure (CHF) with the randomization of the first patient in the Phase II GenePHIT trial for AB-1002 (NAN-101). This trial represents a critical step forward in the development of new treatments for CHF, a condition with a high unmet medical need affecting millions globally.

Understanding the GenePHIT Trial

The GenePHIT trial is an adaptive, double-blind, placebo-controlled, randomized, multicenter study designed to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms. This innovative approach to gene therapy aims to improve the quality of life for patients suffering from heart failure symptoms despite undergoing guideline-recommended therapy.

First patient Randomized in Pioneering Gene Therapy Trial of AB-1002 by Bayer AG and AskBio for Heart Failure

The Promise of AB-1002 for Heart Failure Patients

AB-1002 is under investigation as a potentially groundbreaking gene therapy for the treatment of congestive heart failure. Manufactured by Viralgen Vector Core, S.L., another subsidiary of AskBio, AB-1002 has yet to receive marketing authorization, and its safety and efficacy are still under evaluation. The GenePHIT trial will include between 90 and 150 adults, assessing the primary efficacy endpoint at 52 weeks through a modified win ratio of several clinically meaningful assessments.

Timothy D. Henry, MD, MSCAI, Principal Investigator and Steering Committee Member, emphasized the importance of the GenePHIT trial, stating, “The randomization of the first patient as a part of the Phase II GenePHIT trial is an important moment for the heart failure community.” Roger J. Hajjar, MD, Scientific Chair CHF at AskBio, also highlighted the significance of announcing this trial update during Heart Failure Awareness Week, marking a culmination of years of research in cardiac gene therapy.

Christian Rommel, PhD, from Bayer’s Executive Committee, expressed enthusiasm about the potential of gene therapy to address heart failure at its root cause, underscoring Bayer’s commitment to delivering innovative treatment options for patients.

The Impact of Gene Therapy on Heart Failure Treatment

The development of AB-1002 and the initiation of the GenePHIT trial underscore the ongoing efforts to find more effective treatments for congestive heart failure, a condition that continues to pose a significant challenge in a progressively aging population. The successful completion of this trial could mark a turning point in how heart failure is treated, offering hope to millions of patients worldwide.