Initiation of Randomization AskBio Launches Phase II Gene Therapy Trial for Congestive Heart Failure with First Patient

Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, have jointly announced a significant milestone in their efforts to combat congestive heart failure (CHF). The first patient has been randomized in the GenePHIT (Gene PHosphatase Inhibition Therapy) Phase II trial of AB-1002 (also referred to as NAN-101) for the treatment of CHF.

GenePHIT stands as an adaptive, double-blind, placebo-controlled, randomized, multicenter trial aimed at assessing 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, who have maintained medical stability for a minimum of four weeks. This development marks a crucial step forward in the progression of AB-1002 towards addressing the needs of patients facing significant medical challenges.

The trial, GenePHIT, anticipates enrolling between 90 and 150 adults characterized by a left ventricular ejection fraction ranging between 15 and 35 percent, despite continued experience of heart failure symptoms despite adherence to guideline-recommended therapy. The primary efficacy endpoint at 52 weeks will be a modified win ratio derived from several clinically significant assessments.

Dr. Timothy D. Henry, MD, MSCAI, Principal Investigator, and Steering Committee Member, emphasized the importance of this milestone, 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.” He expressed optimism that GenePHIT would not only assess the safety and efficacy of AB-1002 in the largest patient cohort to date but also enhance understanding of gene therapy’s broader potential in treating congestive heart failure.

Dr. Roger J. Hajjar, MD, Scientific Chair CHF at AskBio, echoed this sentiment, highlighting the significance of the trial’s commencement during Heart Failure Awareness Week. He noted that the enrollment of the first patient in the Phase II trial signifies the culmination of extensive research and development efforts in cardiac gene therapy, underscoring AskBio’s commitment to advancing AB-1002 as a potential treatment option for CHF patients.

Christian Rommel, PhD, Member of Bayer’s Pharmaceuticals Division Executive Committee and Head of Research and Development, emphasized the pressing medical need for innovative solutions in addressing heart failure, particularly in an aging population. He expressed excitement about the potential of gene therapy to address the root cause of the disease, underscoring Bayer’s dedication to delivering groundbreaking treatment options for patients.

It’s important to note that AB-1002 remains an investigational gene therapy without marketing authorization, and its efficacy and safety are yet to be fully established or evaluated. AB-1002 is manufactured by Viralgen Vector Core, S.L., a wholly owned and independently operated subsidiary of AskBio.

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