Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced the randomization of the first patient in GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase II trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF).

GenePHIT represents an adaptive, double-blind, placebo-controlled, randomized, multicenter trial designed to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults diagnosed with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms. This significant milestone in the development of AB-1002 for CHF underscores its potential to address the unmet medical needs of patients suffering from this debilitating condition.

The trial aims to enroll between 90 and 150 adults with left ventricular ejection fraction ranging between 15 and 35 percent, who continue to experience heart failure symptoms despite receiving guideline-recommended therapy. The primary efficacy endpoint at 52 weeks will be a modified win ratio based on various clinically meaningful assessments.

Principal Investigator and Steering Committee Member, Timothy D. Henry, MD, MSCAI, expressed the importance of this 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. GenePHIT will evaluate the safety and efficacy of AB-1002 in the largest number of patients to date and improve our understanding of gene therapy for the treatment of congestive heart failure."

Roger J. Hajjar, MD, Scientific Chair of CHF at AskBio, highlighted the significance of this milestone, stating, "Being able to announce this important GenePHIT trial update during Heart Failure Awareness Week adds special significance to this milestone. The enrollment of this first patient in the Phase II trial represents the culmination of many years of dedicated research and development in all aspects of cardiac gene therapy for congestive heart failure."

Christian Rommel, PhD, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development, emphasized the potential impact of gene therapy in addressing heart failure, stating, "Heart failure is a devastating disease with increasing unmet medical need, especially in a progressively aging population. The potential impact of gene therapy to address this disease at its root cause is immense, and we are thrilled about this step in our path to deliver truly innovative treatment options for patients."

AB-1002 is an investigational gene therapy developed by AskBio and has not yet received marketing authorization. Its efficacy and safety are currently being evaluated in clinical trials. The manufacturing of AB-1002 is conducted by Viralgen Vector Core, S.L., a wholly owned subsidiary of AskBio.