AskBio Initiates Phase II Gene Therapy Trial for Heart Failure
Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, have 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 congestive heart failure (CHF) treatment.
GenePHIT is a double-blind, placebo-controlled, multicenter trial assessing the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy and NYHA Class III heart failure symptoms. The trial aims to address the unmet medical need for CHF treatment.
Between 90 and 150 adults with left ventricular ejection fraction between 15 and 35 percent will participate, with the primary efficacy endpoint at 52 weeks being a modified win ratio of clinically meaningful assessments.
Dr. Timothy D. Henry, Principal Investigator, emphasized the trial’s significance for the heart failure community, expressing hope for potentially transformative outcomes. Dr. Roger J. Hajjar, Scientific Chair CHF at AskBio, underscored the culmination of years of research in cardiac gene therapy and its promise for new treatment avenues.
Christian Rommel, PhD, from Bayer’s Pharmaceuticals Division, highlighted the immense potential of gene therapy in addressing heart failure’s root cause and the excitement about advancing innovative treatment options.
AB-1002, an investigational gene therapy manufactured by Viralgen Vector Core, S.L., aims to address heart failure’s underlying mechanisms.
GenePHIT, a Phase II adaptive trial, evaluates the safety and efficacy of AB-1002 in adults with non-ischemic cardiomyopathy and NYHA Class III heart failure symptoms. Participants are randomized into low dose, high dose, or placebo groups, with primary outcome measures including cardiovascular-related death and key functional assessments.
